4 May 2018 No. 3903

Scripscrip.pharmaintelligence.informa.com Pharma intelligence | informa VP and Global Head of CV Franchise Shel- don Koenig said in an interview, repeating a complaint the manufacturers have raised since the launch of the injectable high- cholesterol therapies. “This really simplifies the documentation necessary.” Express Scripts Chief Medical Officer Steve Miller said the negotiated price would be at the low end of the range rec- ommended by the third-party Institute for Clinical and Economic Review (ICER) as being cost-effective. After reviewing positive outcomes data on Praluent ear- lier this year, ICER concluded the net price for Praluent for high-risk patients with cholesterol of at least 100 mg/dL despite intensive statin therapy should be $4,500 to $8,000 per year, depending on the risk Shutterstock: LittlePerfectStock Shutterstock: to the patient. “We are really excited because it puts this drug now within the affordable range of /Regeneron Extend A Hand more patients,” Miller said. The WAC of Pralulent is $14,600 a year, though the companies have been offer- On Praluent, Express Scripts Takes It ing rebates that some analysts estimate JESSICA MERRILL [email protected] have reduced the costs to about $8,000 per year. After releasing the FOURIER car- anofi and Regeneron Pharmaceuti- Scripts’ national formulary only through a diovascular outcomes trial for Repatha in cals Inc. said in March they were will- medical exception pathway. 2017, concluded the benefit sup- Sing to make a deal on the cost of the Reducing the access restrictions for ported a net price in the range of $7,700 PCSK9 inhibitor Praluent () in ex- Praluent is an important win for Sanofi/ to $11,200 per year. (Also see “Amgen Says change for improved access. Now, Express Regeneron. Prescription rejection rates Repatha Outcomes Trial Backs Up Its Pricing Scripts Holding Co. has taken them up on have been as high as 75% for PCSK9 Math” - Pink Sheet, 19 Mar, 2017.) the offer. drugs, according to Sanofi. Amgen has Sanofi/Regeneron announced they The companies announced on May 1 presented data showing as many as 80% were willing to lower the price of Praluent that Express Scripts will grant Praluent pre- of PSCK9 prescriptions initially are reject- in line with ICER’s recommendations for ferred access on its national formulary over ed. (Also see “Will Physician Demand For Re- insurers that removed access restrictions Amgen Inc.’s Repatha () and patha Put Pressure On Payer Restrictions?” - in March, at the same time the partners remove some of the burdensome access Pink Sheet, 19 Mar, 2017.) released positive outcomes data from the restrictions in exchange for a higher rebate “It takes [physicians] days and some- long-term ODYSSEY trial, showing a sig- on the wholesale acquisition cost (WAC) of times weeks to get approvals, and there- nificant reduction in cardiac events and a benefit on mortality for patients taking Praluent. Express Scripts national formulary fore, in the end, it is the patient that re- Praluent. Sanofi/Regeneron’s decision to includes 20 million individuals. Repatha will ally suffers, waiting a month, or sometimes now be available to patients on Express more, to get a prescription,” Sanofi Senior CONTINUED ON PAGE 7

BROUGHT TO YOU BY THE EDITORS OF PHARMASIA NEWS, START-UP AND SCRIP INTELLIGENCE

Chinese Biogenerics Return Of The Mega-Merger? Q1 Results Limited uptake perplexes (p16) One big deal could do it, Sanofi, Roche, Amgen, Shire, says PwC (p18) Boehringer and Sun report (p4-6, 8-11) IN THIS ISSUE

from the editor [email protected]

Many of our readers will be aware of the annual Scrip don Hilton on Park Lane, Mayfair, on November 28. Awards, which recognize outstanding achievements in Check out those entry criteria and good luck with your the pharmaceutical, biotech and allied industries. It’s submissions. The entry deadline is June 1. my great pleasure to inform you that the 2018 Scrip Back to the here and now, this issue’s coverage Awards are now open for entries. I know from our daily is heavy on details gleaned from first-quarter big reporting how much there is to celebrate in this indus- pharma financial reporting, from Roche’s executives try, and I encourage all of you to get nominating. talking about their vision for their electronic health With a new award for best use of real-world evidence record specialist acquisition Flatiron Health (p8) to having been added this year, there are now 16 trophies Boehringer Ingelheim’s expectations for the impact up for grabs, to be judged by our independent panel of of impending patent US patent expiries on its big- 17 senior industry experts. For details of the individual gest revenue generator, Spiriva (p11). Sanofi, Amgen, award categories and how to enter, visit scripawards. Shire and GlaxoSmithKline are among the other ma- com. Every year our prestigious ceremony gets bigger jor firms whose results we cover in the issue; go to our and better, and I look forward to welcoming even more website for additional reporting on companies includ- of you when we gather to bestow the awards in the Lon- ing Biogen and Gilead.

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EDITORS IN CHIEF John Hodgson EDITORIAL OFFICE Eleanor Malone (Europe) Ian Schofield Christchurch Court Denise Peterson (US) Vibha Sharma 10-15 Newgate Street Ian Haydock (Asia) Joanne Shorthouse London, EC1A 7AZ Sten Stovall CUSTOMER SERVICES EXECUTIVE EDITORS US Tel: +44 (0)20 7017 5540 COMMERCIAL Michael Cipriano or (US) Toll Free: 1 800 997 3892 Alexandra Shimmings (Europe) Derrick Gingery Email: clientservices@ Mary Jo Laffler (US) Joseph Haas pharma.informa.com Emily Hayes POLICY AND REGULATORY Mandy Jackson TO SUBSCRIBE, VISIT Maureen Kenny (Europe) Cathy Kelly scrip.pharmaintelligence.informa.com Nielsen Hobbs (US) Jessica Merrill TO ADVERTISE, CONTACT Brenda Sandburg [email protected] Bridget Silverman EUROPE Sue Sutter Neena Brizmohun Francesca Bruce ASIA John Davis Anju Ghangurde All stock images in this publication Lucie Ellis Jung Won Shin courtesy of www.shutterstock.com Kevin Grogan Brian Yang unless otherwise stated

Scrip is published by Informa UK Limited. ©Informa UK Ltd 2018: All rights reserved. ISSN 0143 7690.

2 | Scrip | 4 May 2018 © Informa UK Ltd 2018 Lilly’s Baricitinib Limited

Exit Zerhouni, Enter Reed

GSK In Artifical Intelligence 20 6 12 21

exclusive online content inside: Alnylam Set For Transformation As Patisiran Nears COVER / Sanofi/Regeneron Extend A Hand On Praluent, Market With Data Upgrade Express Scripts Takes It https://bit.ly/2I3evRD 4 Dupixent Dip Dogs Sanofi Efforts To Deal With Alnylam has outlined further data and analyses from the Phase Diabetes Decline III APOLLO study supporting the use of its selective interfering RNA agent, patisiran, as it nears the market for the treatment of 5 As Amgen Looks To Aimovig’s Launch, It May Learn hATTR amyloidosis. From Repatha’s Past

Brakes On Sanofi’s Dengvaxia Prospects Amid WHO- 6 Zerhouni Retires, Sanofi Hires John Reed SAGE Screening Advice? https://bit.ly/2HJieQS 8 ‘Watch This Space’ Roche Execs Say, Outlining RWE Rationale For Flatiron Buy WHO’s recommendation around a pre-vaccination screening strategy for Sanofi’s Dengvaxia could potentially dull the 9 Keeping Mum On Takeda, Shire Outlines Two-Division prospects of the product further, especially in emerging markets Performance with stretched healthcare resources. One industry expert suggests that governments should probably consider introducing the 10 Sun Rises For GSK’s Trelegy And Shingrix, Sets For Advair vaccine only after “better tests and tools” become available. 11 Boehringer Breathes Easy Over Spiriva Patent Expiry In US Biogen’s Spinraza Sales Plateau; The Challenge Is Keeping Up With The Successful Launch 12 Lilly May Need To Reassess Baricitinib Market After https://bit.ly/2HGqC7Z FDA Advisory Committee Sales of Biogen’s blockbuster rare disease drug moderated in the 13 Bayer Exercises Option To Up Stake In JV With Zydus first quarter as the company worked through the bolus of urgent infant and pediatric patients awaiting treatment in the US. 14 Glenmark Eyes First In-House Biologic Blockbuster

HCV Sales Drive AbbVie’s Great Quarter, But Gains 15 Toujeo, Perjeta Biosimilars On Biocon-Mylan Menu Won’t Last But Humira Version Iffy https://bit.ly/2Ks8MTO Q1 earnings far outpaced expectations due to the successful 16 Biologics Kill Biosimilars? Limited Originator Uptake launch for new hepatitis C drug Mavyret, but with the HCV Perplexes China Biogenerics Market market overall in decline those gains won’t last and won’t make 18 Q1 Was Slow For Deals, But That Could Change With up for the looming loss of Humira revenue. One Big Transaction – PwC

GW CEO: Cannabinoid Platform Is Growth Engine 19 Mereo Halts Plans For US Listing Beyond Epidiolex https://bit.ly/2raBJM6 20 Crescendo In Largest European Biotech Series B Of Year Aside from epilepsy in children, GW Pharma also looking to other therapeutic spaces for testing its cannabinoid product 21 GSK’s Head Of Artificial Intelligence Unveils R&D Goals candidates, including autism and brain cancer. 22 Pipeline Watch

Finance Watch: Former Alexion Execs Raise $37m To 23 Appointments Launch Rare-Disease Focused RallyBio https://bit.ly/2FwJqAs Private Company Edition: Martin Mackay and two other former Alexion executives co-founded the rare disease start-up in January with a $37m Series A to kick things off. Also, Innovent @PharmaScrip /scripintelligence raises $150m in venture cash, Tmunity adds $35m and Third Rock launches Cedilla. /scripintelligence /scripintelligence

scrip.pharmaintelligence.informa.com 4 May 2018 | Scrip | 3 Q1 RESULTS

Dupixent Dip Dogs Sanofi Efforts To Deal With Diabetes Decline KEVIN GROGAN [email protected]

anofi is hoping for a stronger second therapy” both in atopic dermatitis and mod- Factor IX) for hemophilia B contributed half of 2018 after posting a first-quar- erate-to-severe asthma. Filings for the latter €43m (+27%) and €21m (+12%) respec- Ster sales decrease due to unfavorable indication have been made on both sides of tively to Sanofi’s coffers. exchange rates and weaker sales of the the Atlantic and Japan. Brandicourt stressed that Sanofi had atopic dermatitis drug Dupixent (dupilum- Regarding the weaker sales for Dupixent, done extensive work to analyze the hemo- ab), a drug the French group hopes will be Deutsche Bank issued a note saying that it philia market before buying Bioverativ and instrumental in offsetting the decline of its backed up AG’s claim that it had believes that factor replacement therapy diabetes franchise. similar inventory issues in the first quarter will remain the standard of care for many years to come. Some observers have ques- tioned that view, citing the arrival of Roche’s Sanofi is hoping that Dupixent will help fill the gap Hemlibra (emicizumab) to the market. in revenues that is growing with the continuing Speaking on the conference call, Biovera- tiv’s CEO John Cox said that safety questions decline of its off-patent diabetes blockbuster Lantus remained about Hemlibra, which is cur- rently restricted to patients with factor VII inhibitors, and physicians will closely weigh Dupixent sales came in at €107m (€95m for its skin drug, the psoriasis blockbuster up that risk relative to its benefit. However, in the US), some way off consensus esti- Cosentyx (secukinumab). However, analysts Eloctate has an excellent safety profile with mates of €144m and down from €118m in at Jefferies called Dupixent “a significant very low bleed rates, he added, saying that the fourth quarter. The fall was due to inven- miss,” and although destocking and higher doctors could also adjust the treatment ac- tory reductions in the US and to higher con- start of year contributions to patient assis- cording to the patient’s lifestyle. tributions to patient assistance programs, tance programs may have accounted for Eyebrows have been raised over Biovera- hitting revenues by €30m. €30m of the shortfall, “even after adjusting tiv’s $11.6bn price tag but Brandicourt said On a conference call, CEO Olivier Brandi- for this, it is still 5% light of consensus ex- “we feel very confident that we are getting court claimed that the decline was not a pectations.” a very strong return” even factoring in the major issue, saying that in the US, the total Sanofi is hoping that Dupixent will help likelihood of Hemlibra getting some non- number of prescriptions for Dupixent re- fill the gap in revenues that is growing with inhibitor market share. mained strong, climbing 25% compared the continuing decline of its off-patent dia- The acquisition of Bioverativ and the with last quarter, adding 500 new patients betes blockbuster Lantus ( glargine). soon-to-be completed deal to buy Bel- a week. Bill Sibold, head of Sanofi Genzyme, The latter brought in €911m, down 17.7%, gium’s Ablynx NV will see Sanofi focusing added that of the €30m reduction, two- while Lantus sales sank 31.1% to €413m in on integrating the two companies in the thirds was an inventory issue and some the US, battered by lower prices and the loss short term and “that’s going to require a lot inter-quarter variability was to be expected, of Medicare Part D business. of attention,” said Brandicourt. However, the but the higher contributions to patient as- Brandicourt highlighted that compared shopping may not stop there. sistance programs, a common phenome- with the same quarter two years ago, US When asked about the possibility of more non post-launch for specialty care products, sales of Lantus have more than halved and M&A, he spoke about a “€20bn envelope are already diminishing. now represent only 30% of global diabetes we wanted to dedicate to inorganic growth He went on to say the launch of Dupixent sales as compared with 50% of a much big- [and] we basically have spent about €13bn was going well and while initially most use ger franchise in the first quarter of 2016. The of that.” Therefore there is still €7bn to spend had been in severe eczema sufferers, now impact of the product’s decline is waning “if we find attractive bolt-on opportunities.” more moderate patients were also getting and “we expect this headwind to diminish As for divesting assets, Brandicourt noted access to the drug as Sanofi expanded its in the coming quarter.” that following the €21.8bn business swap educational efforts and a direct-to-consum- The CEO said that “I’m confident in de- last year that saw Sanofi exchange its ani- er campaign in the US promoting disease livering a return to growth in the second mal health division Merial for Boehringer awareness rolled out. half,” and much of that confidence comes Ingelheim GMBH’s consumer healthcare Brandicourt insisted that the lower Q1 from the performance of recently acquired business, and the proposed €1.9bn sale of sales of the interleukin-4 and -13 inhibitor Bioverativ, sales from which were only con- its European generics business to private were “not at all representative of the under- solidated from March 9. Still, in just three equity group Advent International, no ad- lying dynamics of the launch,” adding that it weeks, Eloctate (recombinant Factor VIII) ditional divestitures are planned. had the potential to be “a practice-changing for hemophilia A and Alprolix (recombinant Published online 28 April 2018

4 | Scrip | 4 May 2018 © Informa UK Ltd 2018 Q1 RESULTS

As Amgen Looks To Aimovig’s Launch, It May Learn From Repatha’s Past MANDY JACKSON [email protected]

ince Amgen Inc. and Novartis AG are quarter, including $84m in the US and $39m PAYER NEGOTIATIONS REMAIN likely to have the first CGRP inhibitor in the rest of the world. UNDER WRAPS Son the market with Aimovig (ere- “Over the past few months we’ve seen However, none of the companies develop- numab), the partners will have an important access to Repatha continue to improve and ing CGRP inhibitors has given explicit guid- opportunity to set the pricing and reim- we remain committed to ensuring access ance about planned pricing or payer nego- bursement tone for the class of and affordability for high-risk cardiovascular tiations, and Amgen executives offered few therapies, which ultimately will determine patients. We’ve been negotiating with sev- specifics during its earnings call. whether Aimovig lives up to the hype or be- eral payers for months to expand patient “Novartis has a rich history of presence in comes a PCSK9-like disappointment. access to Repatha and offering significant the neuroscience market both in terms of Amgen CEO Bob Bradway said on the discounts with multiple offers pending,” Ex- the sales force and an outstanding medical company’s April 24 first quarter earnings call ecutive Vice President-Global Commercial organization. We are complementing it with that the company expects to launch Aimovig Operations Anthony Hooper said during the both teams calling on specialists as well as for the prevention of migraine headaches April 24 earnings call. some of the primary care physicians who during the second quarter, assuming the have a propensity to look after patients with drug wins US FDA approval by its May 17 NEUROSCIENCE, NEGOTIATING severe headaches or ,” Hooper said. user fee date. EXPERTISE FOR AIMOVIG “From a timing perspective, we clearly are Amgen and Novartis are poised to have Amgen carefully chose Novartis as its part- in the lead; we look forward to launching the first-to-market advantage, but will be ner for the co-development of multiple first,” he continued. “Unlike the PCSK9 situa- closely followed by Eli Lilly & Co.’s galca- neurology drug candidates and Aimovig tion where we had to follow, we will actually nezumab, which is likely to be the second will be the first product approved under be setting the price ourselves. And this is CGRP inhibitor approved in the US after a that 2016 agreement. (Also see “Amgen, No- clearly a market where patients have huge setback for Teva Pharmaceutical Indus- vartis Trade Rights For Migraine, Alzheimer’s symptoms and actually know when they’re tries Ltd.’s fremanezumab. (Also see “Bad Drugs” - Scrip, 2 Sep, 2015.) not being properly treated, so we look for- News Teva Really Doesn’t Need: A Potential In addition to its neuroscience exper- ward to a large bolus of patients who want Delay For Fremanezumab” - Scrip, 8 Feb, tise, Novartis also is one of the biopharma to come on this drug as quickly as possible.” 2018.) Setting the precedent for pricing and industry’s most active negotiators of out- Hooper took some issue with a recent reimbursement will be a critical aspect for comes-based contracts that offer payers Institute for Clinical and Economic Review the drugs, which may be informed by the discounts or refunds when the company’s (ICER) report assessing CGRP inhibitor pric- path to reimbursement for Amgen’s Re- drugs don’t work. In fact, Novartis indicated ing, because he said it didn’t assess the patha (evolocumab). last year that value-based contracts may be absenteeism that occurs when migraine negotiated for Aimovig. sufferers miss work. However, he indicated LESSONS LEARNED Being first to market will be a big win that the range of prices suggested by ICER FROM PCSK9 for Amgen and Novartis, since efficacy ($8,500) and a separate Amgen-funded The PCSK9 inhibitors, including Repatha, among CGRP inhibitors has been simi- assessment by academic researchers were expected to become quick block- lar across clinical trials, often cutting the published earlier this month ($14,238 to busters due to their impressive LDL choles- number of patients’ days with migraine $23,998) are within the range of prices that terol-lowering abilities. However, with the headaches in half. are being considered for Aimovig. availability of generic oral statins, the in- Physicians and patients have been ea- Jefferies analyst Michael Yee said in an jectables Repatha and Praluent (alirocum- gerly awaiting the first new migraine thera- April 24 note that he estimates Aimovig’s ab) from Sanofi and Regeneron Phar- pies in more than a decade, but pricing and net price after discounts and rebates will maceuticals Inc. have struggled to find a reimbursement agreements that payers be $5,000-$7,000. The drug will be “a key price that convinces payers to reimburse consider reasonable will help drug makers revenue upside driver, but over [the] longer- term since payers will likely try to restrict us- the biologics’ costs. win market share for products that will cost age in [the] first year,” Yee wrote. But Repatha finally is making significant thousands of dollars per year with millions Hooper was asked about a recent me- headway after the FDA cleared a claim for of patients eligible for treatment. dia report that Express Scripts Holding reduction of cardiovascular risk in Decem- But as with the PCSK9 inhibitors compet- Co. wants to see outcomes-based con- ber. (Also see “Outcomes Claim May Help ing with less effective statins, payers may tracts and other pricing concession for Amgen Make Case For PCSK9 Inhibitor Re- steer migraine patients away from brand- CGRP inhibitors. “We’re delighted to hear patha” - Scrip, 1 Dec, 2017.) Sales jumped name, injectable CGRP inhibitors to oral, ge- 151% year-over-year to $123m in the first neric to limit use of the new drugs. CONTINUED ON PAGE 6

scrip.pharmaintelligence.informa.com 4 May 2018 | Scrip | 5 Q1 RESULTS/COMPANIES

CONTINUED FROM PAGE 5 global presence,” he said. “As for business de- that [Express Scripts] are looking at value- velopment, we continue to look for innova- Zerhouni Retires, based prices and look forward to them tive opportunities that are consistent with Sanofi Hires opening up access in those situations to al- our areas of strategic focus while remaining low access to appropriate patients,” he said. disciplined about the path to earning a re- John Reed “We will continue to come forward with turn for our shareholders.” prices that are responsible, that take into ac- Bradway’s comments suggested that Sanofi is bringing in an outsider to count the co-pay requirements as best we Amgen is more comfortable investing in spark the company’s R&D engines: the can. We have a number of risk-based contracts its current assets than in overpaying to add former Global Head of Pharma Re- on the table with Repatha, and we’re quite products to its portfolio, although he said search & Early Development (pRED) prepared to talk to payers about risk-based biopharma company valuations are begin- at Roche. Sanofi announced April 24 contracts with Aimovig,” Hooper added. ning to come down. that John Reed will join Sanofi as the Amgen’s 2% year-over-year revenue head of global R&D effective July 1, SEARCHING FOR growth in the first quarter, including a 3% succeeding the company’s long-time R&D Chief Elias Zerhouni. DIFFERENTIATION rise in product sales to $5.34bn, was driven Zerhouni joined Sanofi in 2009 as a Hooper asked Amgen Executive Vice Pres- by double-digit gains for newer products, scientific officer to the CEO, a consult- ident-Research and Development Sean reinforcing the company’s ongoing invest- ant of sorts, and was appointed to head Harper to discuss Aimovig’s differentiating ments in its pipeline. R&D in January 2011. The passing of the features, which include the fact that it targets Repatha’s 151% jump was by far the torch appears to come at an appropriate the CGRP receptor rather than CGRP itself. biggest gainer, but the bispecific T-cell en- turning point for Sanofi, as the company “We’re the only receptor antagonist in the gager (BiTE) Blincyto (blinatumomab) for looks to redefine its R&D strategy in an clinic, and we chose that path for a number certain leukemia patients saw a 44% spike increasingly innovative drug industry. of reasons, but one of them was potency,” to $49m; Sensipar/Mimpara (cinacalcet) for Sanofi is building a new hematology Harper said. “We seem to be the only prod- secondary hyperparathyroidism rose 18% specialty, powered by the recent acquisi- uct that doesn’t require loading doses or to $497m; and the multiple myeloma drug tions of the blood products specialist intravenous administration, which can be Kyprolis (carfilzomib) grew 17% to $222m. Bioverativ Inc. and the nanobody quite an awkward thing for patients and However, several of the company’s legacy platform company Ablynx NV, and providers, in general.” products had significant declines due to the company’s long-stand antibody de- He also pointed out that new data in competition and pricing pressures, including velopment agreement with Regeneron migraine patients who have failed as a 6% drop for the blockbuster TNF inhibitor Pharmaceuticals Inc. is winding down. many as four prior prophylactic treat- Enbrel for inflammatory diseases (etanercept) Reed has led the research and early de- ments appear to be different from results to $1.1bn; a 5% decline for the neutropenia velopment activities at Roche Pharma- for other CGRP inhibitors, but Harper therapy Neulasta (pegfilgrastim) to $1.16bn; ceuticals for the last five years, respon- expected the novel CGRP receptor-tar- a 10% fall for the anemia drug Epogen (epo- sible for activities through Phase IIb geting mechanism to be Aimovig’s most etin alfa) to $244m; and a 30% plunge for proof-of concept across all therapeutic distinguishing feature. the shorter-acting neutropenia treatment areas, including oncology, immunology, He noted that when physicians have to Neupogen (filgrastim) to $103m. rare diseases, neuroscience, ophthalmol- switch patients from a CGRP inhibitor due Neupogen and Epogen already are facing ogy and infectious disease. He joined to adverse reactions or a lack of efficacy, biosimilar competition, but Amgen has so Roche after a long career in academia they’re more likely to choose a CGRP recep- far delayed biosimilars for Neulasta, Enbrel at the prestigious Burnham Institute, tor-targeting product than another CGRP and Sensipar. where he was eventually appointed pres- ligand-sequestering antibody. “Amgen beat on total product sales of ident and CEO of the Sanford-Burnham $5.3bn versus consensus of $5.2bn, but the Medical Research Institute. NEW DRUGS MOST LIKELY majority of this was from Sensipar at $497m Roche already announced in March AVENUE versus consensus of $405m,” Yee wrote. that Reed would be leaving the com- While CEO Bradway noted that Amgen con- “Good news is other products like Repatha pany for personal reasons and be tinues to invest in innovation that will drive … was a slight beat (and a notable ‘pick up’ in succeeded by William Pao, the global growth, he stuck to previous assertions that acceleration from Q4, which could be a good head of the oncology discovery and the company would be conservative in sign if this continues) and Enbrel was in line translational area for pRED. At Sanofi, terms of the price it will pay to acquire other (always a sensitive spot of focus for investors).” Reed will report directly to CEO Olivier biopharma firms. (Also see “Amgen Invests In Amgen revised its earnings guidance Brandicourt and will join the executive Deals, Share Buybacks And Manufacturing As slightly from a range of $21.8bn-$22.8bn in committee. He will join Sanofi on April Sales Dip” - Scrip, 1 Feb, 2018.) 2018 revenue to a new range of $21.9bn to 30 to help ensure a smooth transition. “Our priorities for the use of cash con- $22.8bn. Earnings per share (EPS) guidance Zerhouni has been a steady hand at tinue to be investment in innovation and changed from $12.60-$13.70 to a new range Sanofi amid some tumultuous leader- supporting the launches of our growth of $12.80-$13.70. ship transitions, notably when Chris- products while continuing to build out our Published online 25 April 2018 topher Viehbacher was pushed out

6 | Scrip | 4 May 2018 © Informa UK Ltd 2018 COMPANIES/HEADLINE NEWS of the company in 2014 and eventu- CONTINUED FROM COVER do something special with Praluent that ally replaced by Brandicourt in 2015. work with ICER on the review and willing- we think will be a trend in the marketplace,” Zerhouni’s focus was on revamping ness to publicly accept the outcome was Miller said. Sanofi’s R&D machine as the compa- a first for the industry, representing an in- Beginning on July 1, physicians will only ny’s top-selling revenue generator – the teresting turning point with a drug maker need to submit an attestation form con- insulin Lantus – came under pressure. agreeing to lower the price of a drug de- firming Praluent is appropriate for patients He focused on pruning Sanofi’s pipe- spite positive new data. based on the FDA-approved indication and line and improving R&D productivity. It also highlighted how the PCSK9 patient history versus more burdensome re- In an interview with Scrip last year, manufacturers miscalculated when they quirements. The form is more of a check box Zerhouni talked about his track record initially set the price of the drugs. Sanofi physicians can use to assert patients have at the company, pointing to 13 drug acknowledged that the company had met certain requirements for treatment, launches between 2012 and 2017 versus learned some valuable lessons through rather than having to provide layers of doc- three drug lunches between 2008 and the process. “I would hope that others will umentation, like laboratory results, patient 2012. He steered the company’s port- learn from what Sanofi/Regeneron have histories and proof of prior statin failure. folio toward biologics, with the help of done, to be very public with this,” Koenig Regeneron, a partnership that resulted said. “We want to show that we are really REBATES AT POINT OF SALE in drugs like Praluent (alirocumab) for committed to improving access.” Another important element of the contract high cholesterol, Kevzara (sarilumab) Whether or not payers would take the is that Express Scripts will pass a portion for rheumatoid arthritis and Dupixent manufacturers up on the offer remained of the rebate for Praluent onto eligible pa- (dupilumab) for atopic dermatitis. a big question, since payers have been tients. The decision could reduce out-of- Although the antibody development quite effective blocking access to the pocket costs for patients and thus improve deal with Regeneron is winding down, expensive biologics. Praluent generated the affordability of Praluent. Miller said the two companies are continuing to only €49m ($59m) in the first quarter, about one-third of the rebate would be collaborate in an immuno-oncology €26m ($31m) of which came from the US, passed onto patients. alliance that was signed in 2015. well below the blockbuster level sales the While Express Scripts has distributed Despite some R&D successes during drug was expected to generate when it some rebates at the point of sale before, Zerhouni’s tenure at Sanofi, the com- launched in 2015. The situation has been Miller said it is the first time they have done pany has also experienced R&D disap- similar for the rival drug Repatha, but the point-of-sale rebates for a specific drug. pointments and misses. Even some clini- addition of an outcomes claim in labeling “That’s going to be a game-changer that cal and regulatory wins like Praluent and has helped. ( (Also see “Outcomes Claim we hope we can take forward with other Kevzara have failed to become commer- May Help Amgen Make Case For PCSK9 drugs in the future,” he said. cial wins. Sanofi’s attempts to extend the Inhibitor Repatha” - Scrip, 1 Dec, 2017.)) The move is part of a broader debate life of its franchise with Repatha sales jumped 151% year-over- in the industry over how rebates should the launch of Toujeo only made up some year to $123m in the first quarter, includ- be distributed to patients. The drug off- of the ground lost to competition. ing $84m in the US and $39m in the rest sets traditionally have gone directly to Most notable perhaps were the of the world. insurance plans, which say they are dis- company’s missteps in cancer, as Sanofi Miller said the decision by Sanofi/Re- tributed indirectly to patients through failed to transition its portfolio from generon to work closely with ICER on the lower premiums. But affordability has chemotherapy to targeted cancer drugs value assessment for Praluent was notable become a growing issue for patients and then missed out altogether on the and put the onus on payers to respond. as high-deductible health plans have early push into immuno-oncology. The Express Scripts has been a proponent of grown. UnitedHealthCare, for exam- company experienced two high-profile ICER and other similar third-party organi- ple, announced in March that it would late stage failures in cancer R&D in zations, he said. launch a point-of-sale rebate program in 2013 in areas where others have had “With that combination of great clinical its fully-insured commercial group plans success: the PARP inhibitor iniparib for data, a company willing to voluntarily look in 2019. Aetna Inc. followed with a simi- breast and lung cancer and the JAK2 at fair price and bring it down to a level of lar announcement. inhibitor fedratinib for myelofibrosis. affordability, it gave us the opportunity to Published online 1 May 2018 There was also the downright dismal launch of Zaltrap (aflibercept) for meta- static colorectal cancer in 2012. Whether Sanofi can compete in increasingly innovative areas of drug LET’S GET SOCIAL development like immuno-oncology We are tweeting, liking and sharing the latest industry news and and gene therapy – where some of its insights from our global team of editors and analysts, join us! rivals are finding success – will now fall to the next R&D leader. @PharmaScrip [email protected], 24 April 2018 scrip.pharmaintelligence.informa.com 4 May 2018 | Scrip | 7 Q1 RESULTS

‘Watch This Space’ Roche Execs Say, Outlining RWE Rationale For Flatiron Buy STEN STOVALL [email protected]

rilled by journalists and analysts during Roche’s quarterly have done with Flatiron,” Schwan said. Roche’s pharmaceuticals head, sales update about the purchase of Flatiron Health Inc., Daniel O’Day noted that Roche had had a long relationship with Flatiron Gthe Swiss group’s CEO and pharma chief explained in detail before deciding in February to buy it. Roche already held a 12.6% stake how acquiring the oncology-focused electronic health record com- in Flatiron before announcing that month that it was paying $1.9bn for pany will give valuable access to real-world evidence (RWE) data that the remainder. The deal is to close during the first half of 2018. Roche aims to use to better design clinical trials and outcomes, im- “Flatiron as an independent company was already on track as be- prove chances for drug reimbursement, and thus reinforce its place ing successful. We only want to encourage and accelerate that, as a as a leader in oncology therapeutics. standalone company that provides services to oncology customers, Answering questions on April 26 while outlining its first-quarter providers and life sciences companies, “ O’Day told the analyst call. sales performance – which saw revenue from new drugs offset erosion of sales from generic and biosimilar competition - Severin ‘Increasingly, healthcare data is getting Schwan, Roche’s Austrian CEO said buying Flatiron Health offered digitized and as such there’s a new space the world’s biggest developer of cancer drugs research-quality elec- tronic medical records (EMR) data that will be used to leverage and of innovation opening up for us that we are increase oncology synergies and help accelerate funding. very interested and very active in’ FLATIRON’S INDEPENDENCE ‘KEY’ Roche also stressed that it will not block competitors from using Flat- iron Health. Rather, Roche will encourage rival pharma companies CHANGING TIMES and other life sciences groups to use the digital record generator’s “What we get out of it and what the industry gets out of it is a world- services, activating firewalls to preserve client relationships, and leading, real-world database that allows us to do things very differ- thereby encourage success. ently than the way we do them today in oncology; everything from “There’s a new space opening up here. Our industry probably has looking at different treatment regimens to get reimbursement ap- been a bit behind in terms of this digitization compared to other proval and markets,” O’Day said. industries such as the media or retail industries – but now increas- “You can’t possibly, in a Phase III , have every dif- ingly healthcare data is getting digitized and as such there’s a new ferent treatment regimen that might be appropriate for a reim- space of innovation opening up for us that we are very interested bursement authority around the world,” he explained. “By using and very active in,” Schwan told Scrip during a media call on the the robustness of this data, we’ve been able to get reimburse- day. Success would depend on Flatiron remaining an independent ment faster and I think some of the real benefit will also be in ac- operator, he added. celerating clinical trial hypotheses, clinical trial design by bringing “We normally work through partnerships. In the case of Flatiron, the clinical trial to the patient or in the community oncology set- however, we felt we could drive the synergies of the market leader in ting, and also potentially supplementing or replacing or speed- oncology on the one hand and the market leader in oncology EMR ing up clinical trials by using a real-world data control arm,” said [electronic medical records] data with Flatiron on the other even bet- O’Day, adding that regulators were also keen to see what can be ter, and make even faster progress – including funding – and this done with the approach. benefit is also available to competitors because we’ve put a lot of “Every time a sponsor company has a new medicine, they have to emphasis on keeping the Flatiron operations separate.” replicate a standard-of-care control arm. We, and the FDA to a large Doing that is the only way Flatiron could remain credible as a extent, believe there’s a better way to do things out there.” neutral partner for other stakeholders, including competitors of He added: “The only way that we can be successful leveraging this Roche, Schwan said . is if all companies are successful by leveraging this, because you have “The acquisition is not about blocking out competitors at all – on an unbiased and a standard created out there with the regulatory the contrary, we want to make the offering, by leveraging our syn- authorities.” ergies, even more attractive for other stakeholders, including other As a result, Roche needs to keep Flatiron at arm’s length and inde- pharma companies, and as the market leader in oncology medicines pendent. “So we are doing the contrary to blocking companies’ [ac- Roche has a special interest in being at the very forefront of this,” cess to Flatiron]; we are creating firewalls so that providers and other Schwan told analysts on an afternoon conference call. life science companies can be very confident in the primacy of their “We have a lot of partnerships in this space but from time to time data and to make sure that this business model continues. We know it makes sense to either build up internal know-how and expertise to that this business model can work overall,” O’Day concluded. start with or strengthen that by means of acquisition. That’s what we Published online 26 April 2018

8 | Scrip | 4 May 2018 © Informa UK Ltd 2018 Q1 RESULTS

Keeping Mum On Takeda, Shire Outlines Two-Division Performance JOSEPH HAAS [email protected]

he possible acquisition by Takeda Pharmaceutical Co. tive feedback on the Takeda/Shire merger is coming mainly from Ltd. was top of mind for everyone involved in Shire PLC’s first analyst and media coverage. Tquarter 2018 earnings call April 26, but under UK takeover law “I think a lot of that feedback has also siphoned into the public do- the company could not discuss the situation, although that didn’t main through analyst reports and other people that had comment- stop one analyst from asking CEO Flemming Ornskov how share- ed, so I’m not going to comment more on that,” Ornskov said. “But holders feel about the possible buyout. getting the right feedback, hearing that feedback, making sure that’s Since first publicly disclosing its interest in acquiring Shire on March an important part of any decision-making, I can assure you I have a 28, Takeda has made five offers, increasing incrementally from £44 board of directors that puts significant emphasis on that and have an to £49 per share (about $64.3bn in aggregate) as of April 24, which independent channel through our brokers to get that information.” Shire said it would take to its shareholders. This outcome extended the deadline for the transaction from an original date of April 25 to PAIR OF OUTLIERS May 8, although investors on both sides of the deal reportedly op- Shire’s apparent goal on the call was to refocus attention on solid pose the merger. earnings performance and success in its efforts to position itself as Overall, Shire reported a solid first quarter with 7% year-over-year mainly a rare disease company. Ornskov noted that about 70% of sales growth, led by 10% growth within its rare disease franchise. The Shire’s revenue comes from rare disease therapies, which mostly are specialty pharma said in January that it would begin presenting its fi- biologics. The pipeline, likewise, is increasingly focused on rare dis- nancial performance as two separate business units – rare diseases and eases, with approximately 70% of the 40-odd clinical candidates ad- neuroscience – with a decision to come later in the year on whether it dressing such indications. would seek to spin out the neuroscience unit in some manner. Ornskov pointed to lanadelumab, a for the The neuroscience business – comprised of ADHD drugs Vyvanse prevention of hereditary angioedema (HAE) now being reviewed for (lisdexamfetamine dimesylate), Adderall XR (amphetamine, dextro- potential approval in the US, Canada and Europe, as one of Shire’s amphetamine mixed salts) and Mydayis (mixed salts of single-entity more promising rare disease assets. The drug could shake up the treat- amphetamine product) as well as ulcerative colitis products Lialda ment paradigm in HAE, he asserted. Shire’s existing three-product HAE and Pentasa (different formulations of mesalamine) – brought in portfolio brought in $369m in first quarter sales, up 1% year-over-year. $918m during the quarter. Firazyr () – for the acute treatment of HAE attacks – That represents a 2% decline year-over-year, but Chief Financial Of- posted solid growth, bringing in $206m globally, compared with ficer Thomas Dittrich noted that the unit saw a 14% increase if the $119m a year earlier. However, prophylactic agent Cinryze (C1 sharp decline in Lialda, which has faced generic competition since esterase inhibitor) fell off sharply, from $226m globally in first mid-2017, was excluded. Worldwide Lialda sales totaled $62m for the quarter 2017 to $147m in the recent quarter. Shire attributed this quarter, down 65% from the first quarter of 2017. Non-Lialda growth decline both to inventory and stocking issues, as well competi- could be attributed mainly to Vyvanse and also international sales tion from CSL Ltd.’s Haegarda, a self-administered, subcutane- growth, Dittrich added. ous formulation of C1 esterase inhibitor. Shire is counting on lanadelumab, which has an Aug. 26 PDUFA NEUROSCIENCE OFFERS HIGH-MARGIN BUSINESS date at US FDA, to change that dynamic. The company noted that Two analysts noted that despite its sales decline, the neuroscience unit lanadelumab 300 mg dosed every two weeks reduced the inci- could offer a potential acquirer high profitability, as it produced a seg- dence of HAE attacks by 83%-93% across patient subtypes in its ment contribution margin of 84%, compared to 48% for rare diseases. Phase III program. These subgroups differentiated based on how Shire defines segment contribution margin as total revenue minus cost frequently patients experienced attacks and whether they used pre- of sales, direct R&D expenses, and direct sales and marketing expense. ventative therapy previously. Calling neuroscience “a highly profitable division” in an April 26 “If lanadelumab is approved in the US and outside the US, that’s, of note, Jefferies analyst Peter Wellford ascribed a $13bn net present course, going to be the key product for us with this franchise,” Orns- value to the unit with potential for $4bn in peak worldwide sales, kov said, noting efficacy and quality-of-life data for the candidate and prior to Vyvanse losing patent protection in 2022. its less-frequent dosing. Despite statements at the start of the call that Shire could not “We feel very confident that this will be a potential paradigm shift comment on the pending transaction with Takeda, one market ana- in the way hereditary angioedema patients are being treated and lyst thought perhaps he’d found a clever way around this limitation, their attacks are being prevented,” he added. He said it was too early asking Ornskov how he interacts with Shire shareholders and what to speculate about price, but indicated that Shire expects lanade- feedback he is getting about the potential deal. lumab, as an antibody, to be a higher-margin product compared to Ornskov replied that he, his executive team and the board all make the company’s existing HAE therapies. efforts to hear out the shareholders. He also implied that the nega- Published online 26 April 2018 scrip.pharmaintelligence.informa.com 4 May 2018 | Scrip | 9 Q1 RESULTS

Sun Rises For GSK’s Trelegy And Shingrix, Sets For Advair JESSICA MERRILL [email protected]

laxoSmithKline PLC is investing on the efficacy. Now, GSK appears poised to dered if the evidence was strong enough behind several important drug take over the big market for a shingles vac- to support stepping up to a single triple Glaunches as it builds a new genera- cines, a target population of about 100m therapy in clinical practice. tion of blockbusters to make up for lost sales patients, according to the company. But GSK is confident the data support from its aging stalwart, Advair. Management In fact, Pharmaceuticals President Luke broader commercialization of Trelegy. “This tried to keep investors’ attention on the po- Miels said the latest market share data data, which is answering for the first time tential of new drugs during the company’s shows Shingrix holds a 99% share of the important questions in COPD management, first quarter sales and earnings call on April pharmacy market. He pointed out that the supports the expanded label that was ap- 25, even while sales of Advair (fluticasone early sales were driven by filling the pipeline, proved by the FDA, and will now enable us proprionate/salmeterol) fell 25%. and that only about one-third of the doses to ramp up our promotional activities and As for any big strategy updates coming sold have been used to vaccinate patients. expand our reach beyond the initial target from the new leadership team of CEO Emma universe to the primary care physician base,” Walmsley and President-R&D Hal Barron, that DATA HAVE A BIG IMPACT Miels said. “This really is the key to the lon- will have to wait. Walmsley said Barron will be New respiratory launches, including Trelegy ger-term success for Trelegy.” ready to deliver an update on R&D priorities and the biologic therapy Nucala (mepoli- GSK was initially targeting 8,500 pulmo- during the second quarter conference call. zumab) for patients with severe eosinophilic nologists in the US with its promotional The company announced the appoint- asthma, could face more challenges getting strategy, but plans to expand outreach to ment of Genentech Inc.’s oncology business off the ground, particularly when it comes primary care physicians as well. Miels said development head Kevin Sin to lead world- to convincing payers to reimburse the med- the company is now shifting commercial wide business development on April 18, sug- icines. Trelegy is made up of the inhaled spending away from Advair to drugs like gesting big changes could be coming on the corticosteroid fluticasone, the long-acting Trelegy, Breo and Nucala (mepolizumab). business development front as the company muscarinic antagonist (LAMA) umeclidini- Nucala was the first in a wave of new looks to ramp up in cancer. But Barron was um and the long-acting beta2-adrenergic biologics for a severe eosinophilic asthma tight-lipped about what the appointment agonist (LABA) vilanterol, dosed once-daily when it was approved in 2015, but Astra- could mean for GSK during the call. in the Ellipta dry powder inhaler. Zeneca PLC launched a new rival Fasenra “Kevin has got a lot of business develop- GSK has been working to build a case that (benralizumab) last year, which could put ment experience in oncology, although he’s the triple combination provides a synergistic pressure on the launch. GSK said it is fo- got a lot of experience in other areas, includ- effect that improves outcomes for patients, cusing on the consistent efficacy seen ing technology,” Barron said when pressed including a lower rate of exacerbations and with Nucala as it competes against a new by an analyst. hospitalizations versus dual therapy. Posi- entry. The company has filed a supple- In the near-term GSK is in the midst of tive data from the IMPACT study were pub- mental biologic license application (sBLA) launching several new drugs, including the lished April 19 in the New England Journal of for an expanded indication in COPD and shingles vaccine Shingrix (zoster vaccine Medicine, showing that Trelegy was superior is awaiting action from the FDA later this recombinant, adjuvanted), the first triple to two of GSK’s dual therapies, Breo Ellipta year. Nucala generated £104m ($144.8m) combination therapy for chronic obstruc- (fluticasone/vilanterol) andAnoro Ellipta in the first quarter. tive pulmonary disease Trelegy (fluticasone (umeclidinium/vilanterol). Nucala, Trelegy and other respiratory furoate/umeclidinium/vilanterol), and a The data supported FDA approval of medicines like Breo and Anoro are expected new dual regimen for HIV, Juluca (dolute- an expanded indication for Trelegy that to fill the gap from lost sales of Advair, which gravir and rilpivirine). opens the door to a broader patient pop- has come under increased pricing pressure All three launches are just getting off the ulation with COPD. The new indication in the US and is facing generic competition ground, with the first quarter representing is for the long-term, once-daily mainte- in Europe. The first generic version of Advair the first full quarter on the market for each. nance treatment of airflow obstruction in the US could launch later this year from Already, Shingrix appears to be on a solid in patients with COPD. It is also indicated Mylan NV, which has an ANDA pending at launch trajectory, generating revenue of to reduce exacerbations in COPD pa- the FDA with a June action date. £110m ($153.2m) in the first quarter.Trelegy tients with a history of exacerbations. The Pricing pressure on Advair in the US was and Juluca generated £11m ($15m) and original approval last September was for worse than expected in the first quarter, Miels £10m ($14m), respectively. maintenance treatment of COPD patients said. Sales of Advair declined 25% to £566m Last year, Shingrix cleared a big hurdle in who are receiving Breo and require addi- ($788.3m) worldwide. In the US, sales fell the US, when the Centers for Disease Con- tional bronchodilation. 32% to £229m ($318.9m). Sales of products trol and Prevention’s Advisory Committee Not everyone has been sure about what delivered on the Ellipta inhaler, meanwhile, on Immunization Practices (ACIP) recom- the data will mean for clinical practices; an nearly eclipsed Advair in the US with £207m mended Shingrix over a rival shingles vac- accompanying editorial in the NEJM ques- ($288.3m) in revenues, growth of 16%. cine, Zostavax, from Merck & Co. Inc., based tioned the robustness of the data and won- Published online 26 April 2018

10 | Scrip | 4 May 2018 © Informa UK Ltd 2018 Q1 RESULTS

Boehringer Breathes Easy Over Spiriva Patent Expiry In US KEVIN GROGAN [email protected]

eports of the death of Boehringer ing patients to its Respimat inhaler from in many countries it is the number one Ingelheim GMBH’s Spiriva (tiotro- the older Handihaler device. He added SGLT-2 inhibitor not in the market as a Rpium) are greatly exaggerated, and that tiotropium “is not an easy compound whole – it competes with AstraZeneca even though its US patents start to expire for one thing,” plus he doubts very much PLC’s Farxiga/Forxiga (dapagliflozin) and in the summer, the German group does not if generics companies will come up with a Johnson & Johnson’s Invokana (cana- expect to see any competition across the soft mist inhaler like Respimat, making the gliflozin) – but in terms of new-to-brand Atlantic to its chronic obstructive pulmo- pathway to US approval for copies even prescriptions. nary disease (COPD) blockbuster for quite more difficult. The company is reaping the benefits of a while yet. a label update based on the 7,000-patient Boehringer’s financials for 2017 show that ‘We are not forecasting it to EMPA-REG cardiovascular trial, in which em- Spiriva was once again comfortably the pagliflozin demonstrated a 38% reduced family-owned firm’s biggest earner, with grow; it would be a bit crazy risk of CV death, a 35% reduction in the risk sales reaching €2.83bn. That represents a if we did, quite frankly’ of hospitalization for chronic heart failure decrease of 3.9% (currency adjusted) due to and cut the risk of incidence or worsening the increased level of generic competition nephropathy by 39%. Hillgrove noted that in Europe and while the decline in the lat- about half of diabetic patients die of CV dis- ter market is significant, CEO Hubertus Von He noted that the combination prod- ease so having “a 38% risk reduction is pretty Baumbach told Scrip at Boehringer’s annual uct Spiolto (tiotropium/olodaterol) was significant” and has impressed physicians press conference in Ingelheim that physi- making good progress from a market and regulators. cians were still preferring to use the original share point of view as well for sales and Boehringer is now looking at whether a in some countries. confirmed that Boehringer was not look- similar reduction in the risk of heart failure Allan Hillgrove, head of Boehringer’s ing at a triple combo such as GSK’s Trel- (HF) and kidney disease would be seen in human pharma business, echoed that egy Ellipta (fluticasone furoate/umecli- the general population, ie patients with and point, saying that penetration of generic dinium/vilanterol). Hillgrove said that the without diabetes. It began the EMPEROR Spiriva varies from country to country in company looked at the possibility of a clinical trial program for HF last year and Europe but the impact on sales was not triple a number of years ago, but decided earlier this month launched EMPA-KIDNEY very strong. The reality is that the cop- to put its resources elsewhere, believing in partnership with Lilly, Oxford University, ies will take a greater share over time in that a triple combo would only be used Duke University and others to study the Europe, “due to a combination of a price as an end-stage rather than regular ther- drug in patients with chronic kidney dis- impact and a value issue,” he acknowl- apy. (Also see “Experts Challenge GSK Triple ease. The trials are expected to read out be- edged, and “we are not forecasting it to Inhaled COPD Therapy Claims” - Scrip, 19 tween 2019 and 2020. grow; it would be a bit crazy if we did, Apr, 2018.) Some of the aforementioned Hillgrove said “the potential for Jard- quite frankly,” but as for the US, the pic- resources will be going on expanding in- iance is very large,” noting that more car- ture is still a rosy one. dications of Ofev (nintedanib). The drug, diologists are increasingly interested in SGLT-2 inhibitors. He added that “we are The first of the patents on Spiriva in the which is approved for idiopathic pulmo- very bullish about the class,” saying he will US will start to run out from July this year nary fibrosis, has become market leader be looking out with interest for the forth- but Hillgrove told Scrip that “we have got a in the US and had 2017 sales of €915m coming data from the PIONEER 2 trial pit- few more years to go” and generics are not (+52.3%); Boehringer is conducting two ting Novo Nordisk AS’s investigational expected in the short term. His confidence Phase III trials which are exploring Ofev in oral version of its GLP-1 agonist Ozempic is based on the case of GlaxoSmithKline systemic sclerosis and progressive fibros- () head-to-head with Jard- PLC’s rival COPD blockbuster Advair (flutica- ing interstitial lung disease. iance. (Also see “Novo Nordisk Already sone propionate/salmeterol). Plans A ‘Next Generation’ Oral Semaglutide Although the US patents on Advair JARDIANCE BOOSTED BY CV - CEO” - Scrip, 8 Mar, 2018.) ran out a while ago, there are still no ap- LABEL CHANGE Published online 26 April 2018 proved generics as the FDA has rejected Away from respiratory, Hillgrove told a number of copycat versions that have Scrip he was particularly pleased with been submitted, due in part to concerns the performance of its Eli Lilly & Co.- Boehringer Bigs Up US and about the inhalers used to deliver the partnered type 2 diabetes blockbuster China As Europe Sales Slip: drug. Hillgrove sees similarities in the case Jardiance (empagliflozin). The drug had https://bit.ly/2jleeLO of Spiriva and Boehringer has been shift- sales of €1.01bn (+135.7%) in 2017 and

scrip.pharmaintelligence.informa.com 4 May 2018 | Scrip | 11 COMPANIES

Lilly May Need To Reassess Baricitinib Market After FDA Advisory Committee MANDY JACKSON [email protected]

li Lilly & Co. is seeking US FDA approv- was 10-5 in favor of the 2 mg dose but 10-5 Lilly cited findings from the Sentinel safe- al for baricitinib to treat adults with against the 4 mg dose, because commit- ty database to show that the rate of venous Emoderate to severe rheumatoid arthri- tee members did not believe there were thromboembolism (VTE) in its studies was tis (RA) who have inadequately responded enough data to rule out a thrombosis risk. consistent with the general population of to or cannot tolerate methotrexate, but “Bulls may argue that at least the 2 mg DMARD-treated moderate to severe RA pa- members of the agency’s Arthritis Advisory has likely made it through,” Bernstein ana- tients, but the agency did not agree that the Committee suggested on April 23 that the lyst Timothy Anderson noted in a report is- Sentinel data reduced concerns about VTE JAK1/2 inhibitor’s risk-benefit profile could sued after the advisory committee meeting, risks for baricitinib. limit the drug’s market. while “bears may argue that without the 4 Donald Miller, a pharmacy professor at Multiple advisory committee panelists mg dose, the perception and commercial North Dakota State University, said he voted recommended baricitinib’s indication be profile of baricitinib is wounded (in the US).” “yes” in favor of the safety data supporting limited to moderate to severe RA patients Lilly closed up 1.4% at $80.20 per share the 2 mg dose, because he believes the who have failed or can’t tolerate treatment on April 23, but fell 1.2% in after-hours trad- population for which Lilly is seeking ap- with methotrexate and other disease-mod- ing to $79.28. Lilly issued a statement that proval are “patients who will tolerate a little ifying anti-rheumatic drugs (DMARDs), in- although it was “disappointed” in the panel’s higher risk.” cluding biologics – which would give Lilly’s assessment of the 4 mg dose, it is “confident Patient representative Diane Aronson drug a narrower label than the only other in the positive benefit-risk profile of both voted the opposite, however, noting approved JAK inhibitor, Inc.’s Xel- the 2 mg and the 4 mg doses” and will con- that the safety profile of the 2 mg dose janz (tofacitinib). But since the potential for tinue working with the FDA. represented too high a risk for RA pa- thrombosis risk can’t be ruled out without Anderson noted that “nothing is final until tients who have failed or can’t tolerate larger studies of baricitinib, the committee FDA weighs in with its decision, but history methotrexate. was more inclined to limit use to later lines shows FDA usually follows the advice of the However, patient advocate Jennifer Ho- of treatment or approving only the lower panels it convenes. Recall that with com- ronjeff voted in favor of the 2 mg dose’s dose than to support Lilly’s proposed indi- petitor product Xeljanz, the lower dose was safety profile, but said she was a little un- cation and dual dosing. the only one approved, too, a decision [that] comfortable with the vote because the Lilly is developing baricitinib under a li- came as a surprise to most investors.” indication Lilly is seeking is for patients censing agreement with Incyte Corp. dat- Xeljanz is approved for RA in the US at a who’ve failed methotrexate, not metho- ing back to 2009. The drug is approved with dose of 5 mg twice-daily with an extended- trexate as well as DMARDs. Horonjeff is a a once-daily dose of 2 mg or 4 mg in more release formulation, Xeljanz XR, dosed at 11 founder and patient advocate at the Savvy than 40 countries and is marketed as Olumi- mg once-daily. However, Xeljanz is limited Cooperative as well as a patient outcomes ant in the EU, where it’s approved for mod- to 5 mg daily for patients with moderate to and quality consultant in the rheumatolo- erate to severe RA in patients who don’t re- severe renal impairment and moderate he- gy division at Columbia University Medical spond to or can’t tolerate methotrexate. patic impairment, but Xeljanz XR is not rec- Center in Sunnyside, NY. Lilly is seeking approval for 2 mg as the ommended at any dose for those patients. recommended baricitinib dose with 4 mg re- NOT ENOUGH DATA TO SATISFY served for patients whose RA has progressed THROMBOSIS SAFETY IS TOP Beth Jonas, rheumatology division chief at after treatment with more than one DMARD. BARICITINIB CONCERN University of North Carolina School of Medi- FDA’s Arthritis Advisory Committee voted The FDA was particularly concerned in its cine in Chapel Hill, NC, said she came to the 14-1 that the data show efficacy for the 2 review of the baricitinib new drug applica- difficult decision of determining that safety mg once-daily baricitinib dose and 15-0 in tion (NDA) about thrombotic events, which data does not support the 2 mg dose be- support of the 4 mg once-daily dose’s effi- occurred more frequently in Lilly’s trials than cause “we just don’t have enough data to cacy. But when it came to the question of in Pfizer’s studies for Xeljanz, including post- say we have enough data.” safety data being adequate to support bar- marketing studies. The agency’s reviewers University of Pennsylvania biostatistician icitinib’s approval, the committee voted 9-6 concluded that there was not enough data Warren Bilker voted in favor of the 2 mg dose’s for the 2 mg dose and 10-5 against the 4 mg on the 2 mg dose to determine it was suf- safety, noting that the completed studies dose (one committee member changed ficiently safe and effective to merit approval, were not sized to prove baricitinib does not their vote to “no” after voting “yes” by mis- and said that a 1 mg dose tested in an early carry a thrombosis risk, but that data could take, bringing the 4 mg dose tally to 11-4). dose-finding study may have been effective be collected in post-marketing studies. Asked whether the benefit-risk profile enough to treat patients and a safer alterna- As to safety for the 4 mg baricitinib dose, was adequate to support approval, the vote tive to the 2 mg and 4 mg doses. Alyce Oliver of the rheumatology division at

12 | Scrip | 4 May 2018 © Informa UK Ltd 2018 COMPANIES

the Medical College of Georgia at August Horonjeff also voted in favor of both the that included four completed Phase III stud- University voted in favor of the drug’s safety 2 mg and the 4 mg dose’s benefit-risk being ies enrolling 3,492 patients. Baricitinib stum- to be consistent with her “yes” vote on the sufficient for approval, but repeated her pre- bled on its way to a committee hearing with 2 mg dose. However, Oliver said she hopes vious caveat that she wished the requested the FDA initially rejecting Lilly’s NDA. An FDA that baricitinib will be used to treat patients indication was for failure after DMARDs, in- approval decision is expected in June. with multiple failures of methotrexate and cluding biologics. The baricitinib advisory committee’s DMARDs. Soko Setoguchi of the Institute for Health, reluctance to endorse the drug’s higher Many of the panelists unconvinced by the Health Care Policy and Aging Research at dose and the member’s questions about 4 mg dose’s safety commented that there Rutgers University also voted in favor of the safety do not bode well for a competing wasn’t enough data available, but Jon Rus- lower dose’s benefit-risk profile, but said drug from AbbVie Inc. The company plans sell, medical director of fibrosis and consult- post-marketing studies should be required to submit it JAK1 inhibitor upadacitinib for ing at the Arthritis and Osteoporosis Center to confirm baricitinib’s safety. Her vote was FDA approval in the second half of 2018, but of South Texas, voted in favor of the higher the opposite for the 4 mg dose, however, along with positive efficacy in RA, AbbVie’s dose regardless of any safety risk. noting that the thrombosis risk should be candidate has raised similar questions about Russell said Lilly’s studies showed the 4 more certain before baricitinib is approved cardiovascular safety. (Also see “AbbVie’s Upa- mg baricitinib dose’s efficacy against RA at the higher dose. dacitinib Safety Appears Improved In Largest, and noted that “it’s war” when treating the Thomas Ortell, Duke University Medi- Longest RA Study” - Scrip, 9 Apr, 2018.) debilitating disease, so potent medicines cal Center, also voted against approval of Published online 24 April 2018 are needed. the 4 mg dose, saying that additional data On the questions about baricitinib’s ben- are needed to show whether thrombotic Lilly Rides To Solid efit-risk profile, Miller voted in favor of the 2 events seen in the baricitinib studies was First Quarter On Diabetes, mg dose, because, he said, “if I was going to due to the drug or other factors. Newer Products: be a patient on this drug, I am going to take The advisory committee’s votes were https://bit.ly/2HEH3BH the 2 mg dose.” based on a global development program

Bayer Exercises Option To Up Stake In JV With Zydus ANJU GHANGURDE [email protected]

artners Bayer and Cadila Healthcare Bayer Zydus Pharma Pvt. Ltd (BZPPL) con- TRANSITIONING ALLIANCE? Ltd. are tweaking the holding struc- tinues to exist and Zydus has not moved While it’s not immediately clear if there’s Pture of Bayer Zydus Pharma Private out of the JV,” Zydus emphasized. more to the seemingly “transitioning” ven- Limited, their long-running 50:50 sales and In response to specific queries from ture, some industry watchers say that Bayer marketing joint venture for pharmaceutical Scrip, Zydus also maintained that the joint AG may be keen to have a bigger say in the products in India. venture company independently markets alliance as it builds on its play in the Asia Cadila Healthcare, the flagship company products and so products such as Xarelto, Pacific - a region where senior Bayer execu- of the Zydus Cadila group, said that it was Yaz, Yasmin and Nexavar will continue to tives have, in the past, underscored an in- divesting 12,500,001 equity shares “at a be marketed by the JV. “Zydus’ women’s tent to give “special attention”. value determined in terms of their joint ven- healthcare products, diagnostic imaging The changing alliance contours perhaps ture agreement” to Bayer. business would also continue to remain the also need to be seen in the backdrop of Zy- The transaction would take Bayer’s hold- products of BZPPL and are not being trans- dus Cadila’s own growing global ambitions. ing in the JV up to 75%, leaving the Zydus ferred back to Zydus,” it added. “But with just 25% in an alliance, it could group with a 25% stake – a move that has led Bayer did not respond to specific queries certainly limit the interest of the minor- to speculation that a definitive transitioning from Scrip on the reasons for realigning its ity partner (Zydus),” an industry expert told of the venture was underway. Both Cadila holding in the Indian joint venture. The Ger- Scrip. Staid numbers – Cadila’s annual report Healthcare and Bayer held 25 million shares man multinational said: “We can confirm the for 2016-17 attributes a loss of INR117m to each in Bayer Zydus Pharma Private Limited. transaction with Cadila Healthcare, which the JV - won’t have helped either though the Zydus Cadila, however, told Scrip that was executed as per our joint venture agree- latest earnings/revenues position of the alli- the current divestment of “25% of the joint ment for Bayer Zydus Pharma.” ance could not be immediately ascertained. venture’s shareholding plus one share is The Bayer-Zydus JV operates in the female Zydus is the fourth-largest drug firm in In- exactly as per the terms of the joint ven- healthcare, metabolic disorders, diagnostics, dia with market share of 4.2%, while it’s the ture agreement”, which gives an option to CVS, anti-diabetics and oncology segments ninth largest US generics player based on Bayer to buy 25% plus one share at the end in India; it essentially leverages the strengths prescriptions, details in a company presen- of seven years of forming the JV as per the of Bayer’s optimized product portfolio and tation in March indicated. agreement. “The joint venture company, Zydus’ marketing and distribution capabilities. Published online 30 April 2018

scrip.pharmaintelligence.informa.com 4 May 2018 | Scrip | 13 COMPANIES

Glenmark Eyes First In-House Biologic Blockbuster PENELOPE MACRAE

ndian drug firm Glenmark Pharmaceuticals Ltd. says GBR 830, a signals of disease activity, measurable changes in skin thickening and novel investigational drug candidate to treat moderate-to-severe clinical improvement in AD symptoms,” said dermatologist Emma Iatopic dermatitis, could be a “blockbuster.” The drug is the first bio- Guttman-Yassky, who teaches at Mount Sinai’s Icahn School of Medi- logic that Glenmark has developed in-house and is part of the com- cine in New York. pany’s quest to be more innovation-led and shift from away produc- In addition to dermatitis, Glenmark is evaluating possibilities for ing plain-vanilla generics. conducting studies with GBR 830 for treating other inflammatory The company completed a Phase IIa trial last year of GBR 830 that autoimmune conditions where dysregulation of OX40 overexpres- indicated “clinically meaningful and sustained” improvement in pa- sion is implicated in disease activity. Preparations for a clinical trial tients with moderate-to-severe atopic dermatitis, also known as assessing GBR 830 for treating systemic lupus erythematosus (SLE) atopic eczema. Atopic dermatitis is a skin inflammation often associ- are underway. ated with other atopic disorders like allergic rhinitis and asthma and represents a multi-billion-dollar treatment market globally. GLENMARK MAKING STRATEGIC TRANSITION Now, Glenmark has said it is beginning a Phase IIb clinical trial of Development of the biologic drug is part of Glenmark’s game-plan the medicine, marking what company president Fred Grossman to remake itself from being a pure generics-driven company to be- called an “exciting step.” GBR 830 is “Glenmark’s first new biologic en- ing an organization built on three pillars -- global generics, specialty tity developed in-house,” he said, adding the company is moving “to products and innovative products. Saldanha, who took over Glen- rapidly advance the GBR 830 development program.” mark nearly two decades ago from his father, was one of the early Company chairman Glenn Saldanha, whose father founded the movers in India in the drug-discovery business but the company has company in 1977 and named it after his two sons, has said the global so far met with limited success. market size for atopic dermatitis “is estimated at being $8-$9bn.” He Now Glenmark’s making a concerted strategic transition in the told India’s CNBC TV18 network last year following the successful face of increased buyer consolidation and a more crowded gener- Phase 2a trial he believed GBR 830 “could be a blockbuster” drug. He ics space in the US. Other Indian drug firms, like market leader Sun said the company would seek an out-licensing deal with a “signifi- Pharmaceutical, are making the same strategy pivot to come up with cant upfront payment” and “milestones as the drug progresses.” more complex drugs that offer bigger margins and face less com- The Mumbai-headquartered company expects it will take at least petition even though these medicines are also more costly, time- till fiscal year 2022 to get GBR 830 to market because of “multiple consuming and risky to develop. studies” required, he said. On the novel biologics side, Glenmark added it’s making prog- ress on GBR 1342, a bi-specific antibody targeting multiple my- APAC SHOWING HIGHEST DERMATITIS GROWTH eloma by initiating phase 1 trials. In addition, the company plans Anti-inflammatory agents are viewed as a major revenue-generating to submit an NDA for its first specialty product, a nasal spray segment as they’re normally used as a first-line therapy after failure called Ryaltris to treat seasonal allergic rhinitis, with the US FDA of moisturizers. Analysts expect Asia Pacific to show the highest rate by the end of June. of market growth, eclipsing North America, as atopic dermatitis treat- The company has completed Phase III clinical trials in the US for ment awareness in the region grows. But the atopic dermatitis treat- the nasal spray, containing a fixed-dose combination of 25 mcg mo- ment market also is fiercely competitive with a multitude of players metasone furoate and 665 mcg olopatadine hydrochloride. including AbbVie Inc, F Hoffmann-La Roche AG, Glaxosmithkline Even though it has operations in over 50 countries, Glenmark is a plc., Novartis AG, Pfizer Inc. and Sanofi SA. small drug player -- it’s ranked just 13th in India’s fragmented market Glenmark aims to start enrolling volunteers in the trial in two with annual sales of INR28bn ($421.4m). But the company has big months on GBR 830 which is designed to inhibit OX40, a costimula- aspirations and it’s working to discover new molecules -- both new tory immune checkpoint receptor expressed on activated T-cells and chemical entities (NCEs) and new biological entities (NBEs). As part memory T-cells. Inhibiting OX40 potentially reduces inflammation of its strategy, the company is also looking at a bigger presence in linked with atopic dermatitis symptoms. Costimulatory signals are complex generics. essential for T-cell activity, and binding between OX40 and OX40L is By 2025, Glenmark expects specialty and innovative drugs to con- a biomarker for severity of autoimmune diseases. tribute 30% to total sales. “Our pipeline of speciality products, to be The Phase 2b, double-blind, placebo-controlled multicenter trial rolled out over the next three to four years, is expected to act as a will randomize some 392 patients across four dosing arms of GBR defence against generics price erosion and increase in competition, 830 and a placebo. The launch of the trial is a “meaningful milestone,” and boost profitable growth,” Saldanha said in the company’s annual said Kurt Stoeckli, Glenmark’s chief scientific officer. report. The OTC segment will be another growth lever. In the 12-week Phase 2a study which involved 64 patients with moderate-to-severe atopic dermatitis, data showed GBR 830 had an SHARES RALLY ON MORE POSITIVE NEWS FLOW effect on AD-related disease biomarkers and there was clinical im- The company got a sharp reminder of the need to generate new rev- provement in the Eczema Area and Severity Index (EASI) scores in enue streams when third-quarter-to-December net profit plunged 17 out of 23 patients. “We observed consistency between molecular 78% to INR1.04bn ($15.6m) from a year earlier on the back of a 40.2%

14 | Scrip | 4 May 2018 © Informa UK Ltd 2018 COMPANIES

slide in US revenues. Performance was hit by loss of exclusivity in the Glenmark’s pipeline contains oncology, respiratory disease and US market for its generic version of anti-cholesterol drug Zetia and dermatology compounds in various stages of clinical development falling generics prices. The company has said it believes the fourth- along with molecules primarily focused on inflammation -- asthma quarter should be better, even though it expects the US market busi- and COPD, rheumatoid arthritis, neuropathic pain and inflammatory ness “to remain very challenging.” pain and metabolic disorders like diabetes and obesity. The company Glenmark’s shares have gained around 10% in the past month has said it will continue with its approach of out-licensing its mol- on positive news that has included US regulatory clearance for its ecules, a business model that involves licensing original molecules Baddi plant, which produces oral solids and liquid doses, and makes to foreign drug companies which conduct clinical trials, especially up about 10% of company sales to the US market. The plant was hit Phase III trials. by a Form 483 last November. Glenmark’s stock was up nearly 2% Even Indian drug companies with the big balance sheets find it dif- at INR581.80 on April 23, but that was still less than half its peak of ficult to finance expensive large-scale programs. INR1,226.80 reached in mid-2015. Published online 24 April 2018

Toujeo, Perjeta Biosimilars On Biocon-Mylan Menu But Humira Version Iffy ANJU GHANGURDE [email protected]

iocon Ltd. and Mylan NV are expanding their long-running Ltd. (FKB) for its biosimilar adalimumab. FKB’s biosimilar Humira could biosimilars collaboration to include two new next-generation potentially obtain approval in Europe in the second half of 2018. Bbiosimilar programs - insulin glargine 300 units/mL (Sanofi’s To an analyst’s query on Biocon’s plans with its own Mylan-part- Toujeo) and pertuzumab (Roche’s Perjeta) – but the Indian firm’s nered adalimumab in markets such as the US and Europe, Biocon management was non-committal on the outlook for the duo’s own CEO and Joint MD, Dr Arun Chandavarkar, said that while the ar- partnered Humira (adalimumab) biosimilar. rangement with FKB is currently for Europe, there are “options to ex- Biocon said that the new programs would bolster their existing tend that to the US or other jurisdictions.” global biosimilars portfolio comprising antibodies and insulin ana- “At this stage, based on what we have seen and you are aware logs, though no specifics on plans around the programs were pro- of the potential launch timelines/market formation timelines in vided. The duo have, so far, had a fairly effective collaboration that the US, we feel that there is time to take a decision on which op- cross-leverages development and commercialization capabilities in tion to pursue in the US and other markets. We have not ruled in a risk and reward share model. or ruled out any option, outside of Europe at this stage.” Last month Biocon and Mylan’s Semglee (insulin glargine) was ap- proved by the European Commission (EC), following a positive rec- MARKET COMES FIRST ommendation earlier by the EMA’s Committee for Medicinal Products Mylan president Rajiv Malik had at the time of the FKB deal main- for Human Use (CHMP). In the US, the duo’s insulin glargine under the tained that “nothing is wrong with the Biocon partnership” but that NDA pathway is under review by the FDA; their biosimilar version of the market comes first. (Also see “Mylan Looks To Expedite Biosimilar Roche’s breast cancer drug Herceptin (trastuzumab) was approved by Humira in EU Through Kyowa Deal “ - Scrip, 12 Apr, 2018.) the FDA in December last year. “And when we realized that we will not be in time for Europe for market formation with our biosimilar to Humira, we had to make EARNINGS CALL the call in favour of the FKB product. If we don’t have a product Semglee is expected to be launched by Mylan in Australia and Eu- and market needs it, we’ll go and find it,” Malik had explained. rope in the second half of 2018, Biocon chair, Kiran Mazumdar-Shaw Biocon had, at the time, said that it retained “its economic inter- said on the company’s fourth-quarter earnings call April 27. The est” in the FKB arrangement vis-a-vis Mylan “in line with its existing product is also expected to be commercialized by Biocon’s local part- global collaboration with Mylan for monoclonal antibodies.” ner in South Korea later this year. “We participate in whatever costs and profits Mylan has as Near-term opportunities are currently being addressed by “our part of the deal [with FKB]; we participate in our share of that very successful” global partnership with Mylan, Mazumdar-Shaw not- as per our global arrangement,” Chandavarkar explained on the ed. This, in the backdrop that Biocon is straddling another biosimilars earnings call. collaboration – with Sandoz International GMBH – for developing Biocon reported a growth of 27% in revenues to INR12.37bn a set of next-generation biosimilar products, opportunities for which ($186m) in the fourth quarter ended March 2018 led by the biolog- are expected to open up in the next decade. ics and research services businesses, which grew 47% and 45%, re- But the Biocon management were less forthcoming on how com- spectively. Its traditional small molecules and branded formulations mercialization priorities could pan out for its partnered Humira bio- businesses also turned in a positive performance in the quarter. Net similar with Mylan currently in phase III, in the backdrop of Mylan’s profits for the quarter grew 2% to INR1.30bn. recent in-licensing deal with Fujifilm Kyowa Kirin Biologics Co. Published online 27 April 2018

scrip.pharmaintelligence.informa.com 4 May 2018 | Scrip | 15 BIOSIMILARS

Biologics Kill Biosimilars? Limited Originator Uptake Perplexes China Biogenerics Market Scrip Awards 2018 BRIAN YANG [email protected]

rmed with four biosimilars that are poised to gain regula- to what biologics companies have been doing – will be necessary tory approvals, China’s most prominent biosimilar developer but will also add cost. “We must inform physicians about our prod- AHenlius Biotech Co. Ltd still finds it difficult to develop ucts similar to originators’ products so they fully understand the such products alone, and sees it necessary to have an innovative clinical data, which are obtained under international standards,” biologics pipeline. Liu stressed. Open for Entries The Shanghai-based firm, which is majority controlled byFosun A premium associated with being the first anticancer biologic to International Ltd., has high hopes to launch its first biosimilar prod- be launched in China could also mean the price won’t be low. Fur- uct to China soon. But it seems not to be holding its breath for a thermore, China in the past has resorted to negotiations to lower spectacular market debut. costly cancer drug prices, and the last round of such reductions was Founded in 2009, Henlius has managed to enter four biosimilars over 50% for some targeted therapies. into Phase III development, among which HLX01 (rituximab) is pend- Outside China, biosimilars are priced at a discount of roughly 30% The 14th Annual ing approval at the new China State Drug Administration (CSDA) af- to originator biologics. In China, biologics are deemed to be high- ter an NDA filed last October. priced treatments, partially because there is no reimbursement. The follow-on product to Roche’s anticancer MabThera could be the first biosimilar to be launched in China under a new regulatory LIMITED ORIGINATOR UPTAKE pathway for these products issued in 2015. The company is going Interchangeability is another key issue. Although China has a regula- through manufacturing inspections by the regulatory agency, and tory approval pathway, there are no formal rules on whether origina- an approval is expected within 2018. Scrip Awards tors can be substituted with biosimilars, and no rules on indication Aside from rituximab, Henlius also has HLX02 (trastuzumab), extrapolation and how biosimilars will be named. HLX03 (adalimumab) and HLX04 () in late-stage In 2015, China issued its first regulatory pathway for biosimilars, development. largely adopted from the regulations of the European Medicines Biosimilars are expected to grow rapidly in China given the latest Agency. Many believe the naming of biosimilars and interchange- Entry deadline: 1 June 2018 strong policy bush; the country now has only a very low market pen- ability will be similar to what the EU has in place. etration of biologics, below 5%, partially due to the high costs associ- Meanwhile, cancer drug makers including Roche have puts much ated with them. effort on market access programs in China, providing patient assis- But there are many hurdles facing biosimilars in China, said industry tance including free product after certain treatment cycles. experts attending the BioCon China conference, held on April 23-24 Still, the lack of insurance coverage has led to the limited uptake in Shanghai. These include: a lack of awareness among physicians and of original biologics in China, where Roche’s global bestselling an- patients; no reimbursement; head-on competition with established 2018 www.scripawards.com ticancer Avastin (bevacizumab) sales are merely 16% of their sales multinational originator companies such as Roche and AbbVie; un- in Japan, despite China having a much larger population. certain pricing; and China’s feverish activity to develop innovative biologic therapies from gene-editing to immuno-oncology drugs to Despite the high hopes for biosimilars in China, several devel- CAR-T treatments. opers are aggressively looking at overseas markets, or develop- 28 November 2018 | The Hilton on Park Lane, London, UK Biosimilar and conventional generic drugs are expected to play a ing their own other innovative biologic therapies given the gen- bigger role in the country, where the government has recently voiced eral environment. its strongest-ever support for generics in a bid to greatly enhance ac- Just three years ago, biosimilars seemed to be a sure area for bio- cess to some life-saving but costly drugs, many in the cancer area. tech investment in China, but now many companies are switching to immuno-oncology and cell therapies, fueled by the deep pockets of HOW AFFORDABLE? venture funds that are eager to leap into the foray and grab a share Entry and General Enquiries: Affordable innovation is exactly what Henlius is banking on to grab of a potentially huge market. Natalie Cornwell biologic market share in China, and the company’s motto is literally One of these, Innovent Biologics Inc., founded by US returnee “affordable innovation”. Michael Yu and which started as a developer of biosimilars, is now is Tel: +44 (0) 7827 993 776 | Email: [email protected] The company has yet to disclose its pricing for biosimilar rituximab, steaming ahead as an immuno-oncology developer. Billed as Chi- however, this may not be as low as some hope, cautioned Scott Liu, na’s largest biotech unicorn, Innovent recently received a fast-track CEO and co-founder of the Shanghai-based company. review designation from the CSDA for its PD-1 checkpoint inhibitor Sponsorship and Table Booking Enquiries: “The pricing must let physicians and patients feel the differ- to treat cancer. Christopher Keeling ence compared to the originator product,” Liu told participants But Innovent suffered a setback for its anti-PD-1 agent, IBI308, de- at the conference. “However, the price can’t be that low, because veloped jointly with Eli Lilly, after the Suzhou firm withdraw its ap- Tel: +44 (0) 20 3377 3183 | Email: [email protected] we must consider the pricing of the originator product, and leave proval application a month ago, but it seems the company is back enough room for further [downward] adjustment.” Liu emphasized on track. that marketing efforts centered on academic promotion – similar Published online 29 April 2018 Sponsored by Headline Sponsor

16 | Scrip | 4 May 2018 © Informa UK Ltd 2018

JN1031 Scrip Awards 2018 Insights Advert A4_2.indd 1 2018/04/16 16:14 HEADLINE NEWS

Scrip Awards 2018

Open for Entries The 14th Annual Scrip Awards Entry deadline: 1 June 2018

2018 www.scripawards.com

28 November 2018 | The Hilton on Park Lane, London, UK

Entry and General Enquiries: Natalie Cornwell Tel: +44 (0) 7827 993 776 | Email: [email protected]

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scrip.pharmaintelligence.informa.com 4 May 2018 | Scrip | 17

JN1031 Scrip Awards 2018 Insights Advert A4_2.indd 1 2018/04/16 16:14 DEALS

Q1 Was Slow For Deals, But That Could Change With One Big Transaction – PwC JOSEPH HAAS [email protected]

y volume, life sciences deal-making de- for deals is plentiful both because of rela- payer arena should drive a cycle of large- clined during the first quarter of 2018, tively inexpensive financing, as well as the scale M&A in biopharma, the report says, Balthough four fairly large deals meant overseas cash freed up for US companies by pointing specifically to Pfizer Inc. as a cata- that the aggregate value of such deals rose last year’s tax reform legislation. lyst. (Also see “Pfizer, Poised For A Tax Reform compared to the fourth quarter, PwC’s deal “Pent up demand and surplus cash are Windfall, Talks About Ways To Reinvest” - Scrip, practice notes in a new report. But the group expected to drive greater activity going for- 30 Jan, 2018.) Noting the New York pharma’s thinks completion of one big transaction ward,” he wrote. “As a result, we believe the thwarted M&A bids in the past five years, could open the floodgates, so to speak. pharmaceutical and biotech sub-sectors the report predicts CEO Ian Read will con- There has been a lot of speculation about will be active due to potential transforma- tinue “to stress the inevitability of upcom- an increase in deals with the US corporate tional deals combined with smaller, bolt-on ing consolidation and will seek to leverage tax reform that passed late last year, and acquisitions by larger companies,” he added. the company’s core competency in large- Takeda Pharmaceutical Co. Ltd.’s pursuit “For the generics and specialty pharmaceu- scale acquisitions.” of Shire PLC – currently valued at about tical sub-sectors where scale is critical, mid- “PharmaVitae expects such a deal to spark $64.3bn, which would make it one of the tier companies are expected to consolidate, a trail of subsequent combinations, with the largest biopharma deals ever – shows that driving additional activity.” industry still largely fragmented,” it contin- mega-mergers are back on the table. (Also Hunzinger also predicted an increasing ues. “This will be buoyed by the potential see “Takeda Closes In On Shire With Revised rate of corporate divestitures, driven partly of the technology industry’s prospective £49/Share Offer” - Scrip, 24 Apr, 2018.) by private equity buyers, although this incursion, as biopharmaceutical companies PwC’s Global Pharma & Life Sciences Deals may occur mainly in medical device sec- aim to stake a claim in a future that will be Insights Q1 2018 report indicates that if one tor. “Divestitures will likely continue to be a largely swayed by data analytics and artifi- large-scale transaction goes through, it major focus of big pharma as they seek to cial intelligence capabilities.” could unleash pent up demand on the part extract value and reshape their portfolios,” of other possible acquirers. (Also see “Shire’s the report states. Overall, PwC anticipates RETURN OF THE MEGA-MERGER Suitors: Takeda, Pfizer Seen As Likely To Bid; increased deal activity during the second In March, Leerink Partners advised the Amgen Could Enter Fray” - Scrip, 6 Apr, 2018.) quarter of 2018. market to expect the return of the mega- In a foreword, PwC’s US Pharma and Life merger, listing Pfizer, Merck & Co. Inc. and Sciences Deals Leader Glenn Hunzinger said CONSENSUS FORMING Roche as the most likely acquirers of large the fundamentals for deal-making – based PharmaVitae perceived a similar environ- assets in the biopharma sector. on both asset demand and the availability ment in its quarterly M&A Analysis report, During the first quarter, life sciences deal- of funds – have “never been better.” Funding published April 24. Consolidation in the making totaled $45.8bn in aggregate esti- mated value, PwC’s report states, up 141% Life Sciences Deals, Aggregate Value, 2016-2018 over fourth quarter 2017 but down 15%

90 120 from the first quarter of last year. However, the total of 47 deals declined from both pri- 80 or time points, down 29% sequentially and 96 100 37% year-over-year. 70 98 PwC notes that the decline in deal vol- ) s n

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63.5 53.1 32.6 54.1 77.7 30.0 19.0 45.8 deals (all in January) with estimated values of - $5bn or more. These were a pair of acquisi- Q2 2016 Q3 2016 Q4 2016 Q1 2017 Q2 2017 Q3 2017 Q4 2017 Q1 2018 tions each by Sanofi and Celgene Corp. Total Value Total Volume The French pharma’s $11.7bn acquisition of

18 | Scrip | 4 May 2018 © Informa UK Ltd 2018 DEALS/FINANCING

External Investment In US Assets Increases INBOUND DEALS Inbound deals comprised the second 90 largest segment of life sciences deals by 80 aggregate value during the first quarter,

70 PwC adds, although the Sanofi purchase of Bioverativ drove a significant portion 60 of that. (See table.) Still, the group expects 50 the impact of tax reform to drive a contin- ued strong pace of inbound deals. 40 “While inbound deals continue to repre- 30 sent a relatively small share of total value, we expect tax reform to drive a renewed 20 focus for foreign companies looking to 10 expand operations in the US in coming - years,” PwC predicted. Tax reform also will Q2 2017 Q3 2017 Q4 2017 Q1 2018 drive an increase in acquisitions by US US Outbound Inbound Foreign firms, the report said. PharmaVitae also concluded that US M&A prospects received “a major boost” thanks Bioverativ Inc. on Jan. 22 was the largest TAX REFORM MAY DRIVE to the tax reform enacted last December, life sciences deal during the quarter, while FOREIGN INTEREST which lowered the federal corporate in- it bought Ablynx NV for nearly $5.5bn on PwC also pointed out that deal-making come tax rate from 35% to 21%, which it said Jan. 29. Meanwhile, Celgene’s $9.3bn buy- between US-based companies comprised will make deals more accretive. Likewise, out of Juno Therapeutics Inc. on Jan. 22 the largest share by aggregate value dur- the reduction of tax on overseas profits to and its $7.0bn takeout of Impact Biomedi- ing the first quarter, compared to deals 15.5% should be beneficial to deal-making. cines on Jan. 7 were the second and third between non-US firms and so-called “in- “Repatriation and the lowering of corporate largest deals of the quarter. bound” deals in which a foreign company tax have stimulated the biopharmaceutical “Q1 2018 had four deals larger than invests in a US asset and “outbound” deals industry’s appetite for US-based M&A,” the $5.0bn, which suggests that megadeals are in which a US company invests in an ex- report states. expected to continue in future quarters, US interest. During the fourth quarter, a Published online 30 April 2018 similar to the trends exhibited over the two majority portion of deal value featured years prior to the second half of 2017,” the non-US companies as both parties to the (Images: PwC Deals: Global Pharma & Life report notes. transaction, the report says. Sciences Deals Insights Q1 2018)

Mereo Halts Plans For US Listing LUCIE ELLIS [email protected]

K-based Mereo BioPharma Group Despite the uncertainty, a spokesperson post admission” in the US. The biopharma, PLC has postponed its plans for an for the company said, “Mereo’s share price which is developing therapeutics for rare U$80m Nasdaq listing because of increased by 12% during the listing process and specialty diseases, had a net cash, challenging market conditions – a decision and remains above the price at the start of short-term deposits and short-term in- that has knocked its UK stock price. the process.” vestment balance of £52.5m as of Dec. 31, Mereo’s stock, traded already on the The spokesperson also told Scrip, “The 2017. It noted that this funding would pro- London Stock Exchange, had spiked on the Nasdaq Biotech Index’s (NBI) decline during vide a strong cash runway to deliver on its news the company would float on Nasdaq. 2018 has created challenging market condi- next key clinical program milestones and However, its price has tumbled again after tions in the US and there is no doubt that corporate objectives. the company announced it was halting the markets have been very volatile, leading Mereo’s CEO said in a statement: “We re- this move. both to nervousness and a very price-con- main well funded and are confident in the Mereo announced on April 9 that the com- scious environment for IPOs and follow-ons.” future development and potential value of pany would list American Depositary Shares These conditions led Mereo’s board to take our pipeline.” on the Nasdaq Global Market to raise around the decision to withdraw the global offering The company told Scrip it was consider- $80m. But in an April 26 statement, Mereo and postpone the proposed listing. ing what its next steps would be and that it said “current market conditions are not con- Additionally, the spokesperson noted “will update the market once we have for- ducive for an offering on terms that would be that “many of the recent development mulated our plans.” in the best interests of its shareholders.” stage biotech IPOs have not performed CONTINUED ON PAGE 20

scrip.pharmaintelligence.informa.com 4 May 2018 | Scrip | 19 FINANCING

CONTINUED FROM PAGE 19 full quantitative results in Q4 2018 to further as an oral first-line therapy for patients with Mereo expects to report data this year for elucidate the aromatase inhibitor’s clinical acute exacerbation of chronic obstructive two of its key pipeline products. The com- profile,” they noted. pulmonary disease. Mereo’s spokesperson pany expects to see topline results from BGS-649 is differentiated by its mecha- said the company continues to seek a part- the Phase II ASTEROID trial of BPS-804 in nism of action as an aromatase inhibitor, ner for this compound. In late-2017, Mereo osteogenesis imperfecta between May and which is unique compared to the majority of reported mixed topline Phase II data for September 2017. testosterone replacement therapies on the BCT-197 from the AETHER trial. Mereo said it was currently preparing market and in the pipeline. Biomedtracker Biomedtracker analysts said at the time to move forward with a pivotal study of analysts noted that “since available therapies that while the primary endpoint was met BPS-804, a novel antibody for osteogen- may adversely impact male fertility through in the AETHER trial (reduction in FEV1 from esis imperfecta, with fracture rate as the LH and FSH [luteinizing and follicle stimulat- baseline to day seven) for both the high and primary endpoint. ing hormone] suppression, there is need for low dose of BCT-197, statistical significance Furthermore, the company expects data treatments without these issues.” was not achieved for a standard COPD end- from the Phase IIb safety extension study of point comparing FEV1 reduction in the pla- BGS-649 in hypogonadotropic hypogonad- A NEUTROPHIL ELASTASE cebo and BCT-197 arms. ism (HH) in the fourth quarter of 2018. INHIBITOR “This suggests that the reduction in FEV1 In March this year, the company reported This year will also see Mereo initiate a Phase was only marginally larger in the BCT-197 positive topline data from its Phase IIb dose- II proof-of-concept clinical trial with AZD- arms than in the placebo arm and could ranging clinical trial for BGS-649 in obese 9668, a reversible inhibitor of human neu- have been due to chance,” the analysts said. men suffering from HH. Biomedtracker ana- trophil elastase, in patients with severe al- Updated results from the AETHER study lysts said at the time that preliminary results pha-1 antitrypsin deficiency. Mereo licensed will be presented next month during the showed the Phase IIb trial had encouraging- AZD-9668 from AstraZeneca PLC in Oct. late-breaking session at the American Tho- ly met all primary, secondary, and explorato- 2017 for an initial upfront payment of $5m. racic Society (ATS) 2018 International Con- ry endpoints with a dose response relation- Mereo’s final pipeline program is for BCT- ference on May 21. ship demonstrated. “We await release of the 197, a MAP kinase inhibitor in development Published online 27 April 2018

Crescendo In Largest European Biotech Series B Of Year LUCIE ELLIS [email protected]

K-based Crescendo Biologics Ltd. Pack also highlighted that the company castration-resistant prostate cancer and is has raised $70m (€57m) through a had managed to attract investment from expected to enter human clinical trials with- Useries B financing to progress its lead different sources, including existing and in 18 to 20 months. program CB307 into clinical studies – mark- new European investors and a Chinese/US The series B financing will see Crescendo ing the largest European series B financing establishment. through proof-of-concept trials with its lead of 2018 so far. The series B round was led by Andera asset and allow the company to grow its Crescendo is developing multi-function- Partners (formerly Edmond de Rothschild pipeline, exploring the use of Humabody al biologics in oncology, known as Huma- Investment Partners), which holds Europe’s drugs in other cancer indications. bodies, that are focused on targeted T-cell largest life science fund, Biodiscovery V. Oth- As Crescendo approaches the clinic with engagers. The company has a pipeline of er participants included Quan Capital, with its first drug, Pack said it had added develop- multiple preclinical programs, including its life sciences fund Quan Venture Fund I, ment expertise to its team. He also said the one asset partnered with Takeda Pharma- and existing investors Sofinnova Partners, IP company, over the last few years, had been ceutical Co. Ltd. Group, EMBL and Takeda Ventures. slowly shifting away from discovery to focus Peter Pack, CEO of Crescendo, told Scrip the “We have attracted diverse funds… It is on drug development. company was able to secure substantial series important for us that we have attracted the Last year Crescendo named Philip Bland- B funding because it had a novel way of tar- largest life science financier in Europe, An- Ward chief scientific officer and Edward geting and activating specific T-cells. “This is dera Partners, and that we have kept our Stewart chief business officer. Bland-Ward a huge topic in the immuno-oncology space former investors. We have a really fascinat- came to Crescendo from Kymab Ltd., and we have developed a novel mechanism ing mix of investors, it will be interesting to where he was responsible for leading its to do this, which we think is safer and more see how the dynamics work,” Pack said. most advanced development program; and specific than current approaches,” he said. The company’s lead asset CB307, a novel Stewart joined the company from Merri- The financing round represents the larg- bispecific T-cell engager for the selective ac- mack Pharmaceuticals Inc., where he pre- est disclosed series B biotech financing in tivation of tumor-specific T-cells exclusively viously managed the company’s business Europe in 2018. Crescendo previously raised within the tumor microenvironment, is clos- development strategy. $28m in a series A round in December 2013. est to the clinic. This drug is being tested in Published online 30 April 2018

20 | Scrip | 4 May 2018 © Informa UK Ltd 2018 EXECUTIVE INTERVIEW

GSK’s Head Of Artificial Intelligence Unveils R&D Goals LUCIE ELLIS [email protected]

laxoSmithKline PLC’s John Baldoni stepped down from stepped down from the company. a senior role within the big pharma’s R&D executive com- To get this AI experiment started, GSK has identified a biological Gmittee in 2017 to take charge of the company’s use of pathway it wants to explore – the big pharma is particularly inter- artificial intelligence for and development. In an ested in “targets of difficult diseases of the brain,” Baldoni said. How- exclusive interview with Scrip, he sheds light on the UK drug ma- ever, the company is yet to publicly announce the specific diseases jor’s internal AI unit and the diseases it hopes to target through it will target. machine-driven R&D. The company expects to work with four different collaborators and two academic advisers who are interested in learning how an AI approach would be used to develop drugs for this particular disease. GSK believes the pathway it is targeting through this drug discovery experiment plays a role in several diseases that have been difficult to discover drugs for. “We see this as a confluence of science and tech- nology in the brain; artificial intelligence methodologies being ap- plied to these very intractable diseases that have plagued society for- ever and we just think that this is a good combination,” Baldoni said. GSK’s AI leader added that the disease GSK has targeted was not an easy one to use as a test case. However, he would “rather prove the approach on something that is meaningful rather than easy.”

AI PARTNERSHIPS The AI drug discovery unit at GSK has only six members, currently, but the group is working with external partners. For example, in July

Shutterstock: agsandrew Shutterstock: 2017, GSK signed a drug discovery collaboration with Exscientia Ltd., a UK-based company that automates drug design with its AI-based platform. Under the agreement, Exscientia will receive research pay- ‘There was skepticism around ments from GSK that could reach more than $42m. The pair will use GSK’s pharma know-how and Exscientia’s AI-enabled platform to dis- me but Patrick Vallance and the cover small molecules to treat up to 10 targets chosen by GSK. decision-makers at the senior level Baldoni added that the big pharma has “a number of other deals that are in the making right now.” He said the company intends “high- felt this was an opportunity’ ly leveraging outside experts in this space.” Striking an AI deal was tricky to start with though, Baldoni noted. “It Baldoni highlighted that roughly 30% of the cost of drug develop- was so new that the finance people, the legal people and the busi- ment was generated in the phase between target identification and ness development people, they were learning as things progressed,” proof-of-pharmacology. “On average, it usually takes about six and a he said. half years and one third of the cost of drug development to get from Also, to complete a deal in this domain, GSK had to give third par- the idea to the molecule, and our goal is to significantly reduce that,” ties access to its data prior to an agreement. “That’s something that he said. GSK hadn’t done before,” Baldoni said. GSK has previously granted ex- Baldoni approached GSK’s top management internally to influ- ternal groups access to large quantities of data for molecules it had ence the use of AI within the company’s R&D operations, after fol- no commercial interest in. But AI partnerships require the company lowing the machine reading and learning technology trend in other to reveal “millions of compounds where we have deep interest in the sectors, such as the financial sector. He is also a leader for the ATOM molecules,” he said. initiative (Accelerating Therapeutics for Opportunities in Medicine), a public-private consortium that integrates high-performance com- EXPERIMENT TIMELINE puting, shared biological data from public and industry sources, and GSK’s AI team has completed a first iteration of designing molecules emerging biotechnologies to accelerate the discovery of effective using its new machine-enabled approach; those molecules are now cancer therapies. being tested in human relevant assays of the disease. The response to Baldoni’s proposal was varied, he said. “There was “One of the components of our approach is that we want to really skepticism around me but Patrick Vallance and the decision-makers go into human-like test systems,” Baldoni said. “We don’t want to do at the senior level felt this was an opportunity – and a relatively in- very simple biochemical assays to demonstrate that the molecules expensive experiment to do in our environment – to see whether AI work. We want to see how these molecules work in a very complex will play and create value for the sector,” Baldoni said. Vallance, who micro-environment.” The team has also recently launched a second at the time was Baldoni’s boss as GSK’s president of R&D, has since CONTINUED ON PAGE 23

scrip.pharmaintelligence.informa.com 4 May 2018 | Scrip | 21 PIPELINE WATCH

Scrip’s weekly Pipeline Watch tabulates the most recently reported late-stage clinical trial and regulatory developments from the more Click here for the entire pipeline than 10,000 drug candidates currently under active research worldwide. with added commentary: http://bit.ly/2mx4jY3

Selected clinical trial developments for the week 20–26 April 2018

LEAD COMPANY/PARTNER COMPOUND INDICATION COMMENTS PHASE III INTERIM/TOP-LINE RESULTS relebactam plus carbapenem-resistant RESTORE-IM1; favorable efficacy and side Merck & Co. Inc. imipenem/cilastatin infections effect responses. ALXN1210, every paroxysmal nocturnal Alexion Pharmaceuticals Inc. Switching endpoints non-inferior to Soliris. eight weeks hemoglobinuria spinal muscular atrophy, AveXis Inc. AVXS-101, gene therapy STRIVE; signs of efficacy. type 1 Epidiolex (cannabidiol) Lennox-Gastaut and Dravet GW Pharmaceuticals PLC GWPCARE5; clinical improvements. oral solution syndromes SELECT-SUNRISE; met primary endpoint, AbbVie Inc. upadacitinib rheumatoid arthritis in Japanese patients. Imfinzi (durvalumab) with non-small cell lung cancer ARCTIC; combination missed endpoints, AstraZeneca PLC or without tremelimumab (NSCLC), third-line monotherapy encouraging. UPDATED PHASE III RESULTS Biogen Inc. Spinraza (nusinersen) spinal muscular dystrophy SHINE; benefits sustained. PROMISE1; increased migraine-free intervals, Alder Biopharmaceuticals Inc. episodic migraine quality of life. Teva Pharmaceutical Industries Ltd. fremanezumab chronic migraine HALO CM, EM; efficacy confirmed. Biohaven Pharmaceuticals Holding migraine Durability of clinical effects. Co. Ltd. Neurocrine Biosciences Inc. Ingrezza (valbenazine) tardive dyskinesia Kinect 3, 4; improved symptoms. Zilretta (triamcinolone Flexion Therapeutics Inc. osteoarthritis Robust treatment responses. acetonide) Inj ZX008 (low-dose Zogenix Inc. Dravet syndrome Reduced seizures, well tolerated. fenfluramine) Celgene Corp. ozanimod multiple sclerosis RADIANCE, SUNBEAM; reduced relapse rates. hATTR amyloidosis Ionis Pharmaceuticals Inc. inotersen Benefits observed, sustained response. polyneuropathy community-acquired OPTIC, OASIS 2; high response rate, non-inferior Paratek Pharmaceuticals Inc. omadacycline bacterial pneumonia to moxifloxacin. Achaogen Inc. plazomicin urinary tract infections EPIC, CARE; high response rates, well tolerated. Vabomere (meropenem Melinta Therapeutics Inc. bacterial infections TANGO II, efficacy in patients with co-morbidities. and vaborbactam) PHASE III INITIATED VX-445, tezacaftor Vertex Pharmaceuticals Inc. cystic fibrosis A triple regimen. and ivacaftor Aldeyra Therapeutics Inc. reproxalap conjunctivitis, allergic Topical ocular administration. pre-symptomatic AveXis Inc. AVXS-101, gene therapy spinal muscular atrophy SPRINT; a one-time dose. types 1,2,3 PHASE III ANNOUNCED Clostridium difficile Synthetic Biologics Inc. ribaxamase (SYN-004) In the US. associated diarrhea Source: Biomedtracker

22 | Scrip | 4 May 2018 © Informa UK Ltd 2018 EXECUTIVE INTERVIEW/APPOINTMENTS

CONTINUED FROM PAGE 21 agement recognized the benefits of speeding up molecule creation, project, which will see this first tranche of molecules tested against a and potentially getting to drug targets with greater relevance. second disease target. Baldoni said there were three races a pharmaceutical company A DESERVED TEST must win to excel in drug development: “I think society deserves this from big pharma,” Baldoni said, when • the race to the molecule; asked why he wanted to lead AI use at GSK. He is also motivated by • the race to the clinic; the use of AI in other sectors, such as the financial sector, where ma- • and the race to the market. chine learning is used to detect fraud or predict growth areas. For a company to be successful it must win in each one of Baldoni noted that while a lot of companies were testing comput- those races. er-driven technologies in aspects of drug development, they were “The race to the molecule is probably the place where you can cre- not using AI in all areas. GSK will take a different route, pulling all its AI ate the most value for a company,” Baldoni said. “Pick the right targets, experiments together under one business unit. make molecules that will modulate that disease, and quickly deter- “The idea that you’re going to segment this into six or seven differ- mine whether or not your hypothesis is correct. This is where there ent workflows and not integrate them, I don’t think is going to create is a lot of failure in the industry. A huge amount of failure,” he said. the best flow of molecules to treat those diseases,” Baldoni said. He noted that roughly 5,000 molecules were generated for every Integration is possible at GSK because in 2015 the company started candidate that made it into human studies, and even then, the failure to consolidate all its data into one system. GSK spent time and money rate was roughly 85% to 90%. to have 2,500 databases consolidated into one platform that was “There’s a lot of failure there. If you can reduce that failure by just a searchable with good quality, curated data in it, Baldoni noted. “This little bit you’re creating a lot of value and you help that organization created a great data source for artificial intelligence use, machine win the race to the clinic.,” Baldoni said, adding that GSK’s senior man- learning requires good quality data.” Published online 30 April 2018

Dr. Gerrit Hauck has been appointed as chief technology officer at Eurofins in Brussels, and before that, he was a regional business and member of the management committee at Basilea Pharma- director at Becton Dickinson, for Russia, Turkey, the Middle East and ceutica Ltd., effective May 1. He will succeed Dr. Günter Ditzinger Africa. Kohli also held a number of leadership roles at Sanofi. who will take on new responsibilities at Basilea. Hauck joins Basilea from Sanofi, where he was cluster head synthetic molecules and a Recursion Pharmaceuticals, a biotechnology company that com- member of Sanofi’s research stage gate committee. Basilea has also bines artificial intelligence, experimental biology, and automation announced that Dr. Josef Künzle, head of global quality manage- to discover drugs at scale, has hired Dr. Kevin Lynch, as chief busi- ment and a member of the extended management committee, will ness officer. Lynch joins Recursion after 22 years at AbbVie, where retire on Oct. 31. He will be succeeded by Dr. Anne Stehlin, who he most recently served as vice president of search and evaluation joins Basilea from Novartis Pharma AG where she was global head and before that as director of licensing and business development. of product quality lifecycle management. She will initially serve as Sonde Health Inc., an affiliate of PureTech Health developing a Basilea’s deputy head of global quality management until Oct. 31. voice-based technology platform for monitoring and diagnosing Joanna Shields has been appointed group CEO at the private mental and physical medical conditions, has appointed Thai Lee, artificial intelligence companyBenevolentAI , with immediate ef- president and chief executive officer of SHI International Corporation, fect. Shields most recently served as the UK Minister for Internet to its board. Lee is the co-founder of SHI International Corporation, Security & Safety, a special advisor to the UK government on the where she currently serves as president and chief executive officer. digital economy, and chair & CEO of TechCityUK. Prior to her work in Jon Neal has taken up the position of managing director, UK and government, Shields spent 25 years in senior leadership positions Ireland for Takeda UK Ltd. He was previously the oncology busi- at companies including Facebook, Bebo/AOL, Google, Decru/Ne- ness unit director, sitting on the UK senior leadership team. He suc- tApp, RealNetworks, Veon and EFI. ceeds Adam Zaeske, who has moved to a new position as Takeda’s OMEICOS Therapeutics, a Berlin-based biopharmaceutical com- regional lead for gastroenterology across Europe and Canada. Neal pany developing first-in-class small molecule therapeutics for the will be succeeded as UK oncology business unit director by Emma prevention and treatment of cardiovascular and ophthalmic dis- Roffe who previously led the oncology medical affairs team. eases, has appointed Dr. Alexander Gebauer to its management board. Gebauer will lead the company’s clinical development ef- Therapeutics has appointed Dr. Claudia D’Augusta to its forts. He has also been appointed chief executive officer and board as a non-executive director. D’Augusta has more than 20 years’ chairman of the recently founded US-based subsidiary, OMEICOS experience in corporate finance, capital markets and M&A. She is cur- Ophthalmics. Gebauer has more than 25 years of R&D experience rently chief financial officer at TiGenix N.V. and is part of the manage- within the biotechnology and pharmaceutical industry. ment team at TiGenix, which Takeda has announced its intention to acquire. Prior to TiGenix, D’Augusta held various other senior financial Pharnext SA has appointed Amit Kohli chief operating officer positions across a number of international public and private com- responsible for leading Pharnext’s corporate strategy and opera- panies. She replaces Wim Goemaere, formerly chief financial officer, tions. Kohli was previously general manager of clinical diagnostics who will step down as a non-executive director. scrip.pharmaintelligence.informa.com 4 May 2018 | Scrip | 23 969_A4_crop.pdf 1 2018/03/09 2:39:47 PM

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