Biopharma Leaders Join Trump and Thunberg in Davos

No. 3989 January 31, 2020

again a key theme at Davos, and many biopharma executives are joining the con- versation this year. These include Werner Baumann, CEO of Bayer, who spoke on a panel on 21 Janu- ary about ‘When Humankind Overrides Evolution,’ focusing on gene-editing technologies for use in agriculture, food and medicine. Meanwhile Takeda CEO Chris- tophe Weber joins a discussion on shap- ing the future of health and healthcare systems on 22 January. Speaking on the gene-editing panel, Baumann said there was “less and less trust in society for the advances of technology,” adding that “the only way to get beyond it is that we do a better job in terms of explaining what we are doing.” Bayer is working with CRISPR Thera- peutics on CRISPR/Cas9 gene-editing in hemophilia, ophthalmology and autoim- Biopharma Leaders Join Trump mune diseases, but also has a major stake in genetically modified seeds via its Mon- And Thunberg In Davos santo division. He called for absolute transparency ANDREW MCCONAGHIE [email protected] from corporations and governments on gene-editing technology, but also decried fter focusing on the year ahead tive to plant 1 trillion trees, an effort aimed the previous “insane dominance” that anti- in biopharma business at the J.P. at soaking up rising levels of carbon diox- GM crop campaigners have been allowed AMorgan conference in San Fran- ide in the atmosphere. to wield. cisco last week, the sector’s attention has 17-year-old environmental campaigner Others taking part include Stéphane switched to the World Economic Forum in Greta Thunberg made her own address to Bancel, Moderna Therapeutics’ chief exec- Davos, Switzerland from 21-24 January. the meeting shortly afterwards, and berat- utive, and Julie Gerberding, EVP and chief The annual “big picture” summit brings ed world leaders for a lack of action, and patient officer for MSD, who will be on a together politicians, economists, academ- said planting trees was not enough. panel looking at breakthroughs in can- ics and business leaders, and this year She called on corporations, banks and cer care on 24 January, while Lars Rebien has been kicked off with a typically pro- governments to halt fossil fuel investment Sørensen, chairman of the Novo Nordisk vocative address by US President Don- and extraction. “Planting trees is good, Foundation, chaired a debate on one of ald Trump, who contradicted growing of course, but it’s nowhere near enough,” the biggest risks to future healthcare, anti- concern about climate change, decrying Thunberg said. “We don’t need to lower microbial resistance (AMR), on 21 January. what he called the “prophets of doom” on emissions….Emissions need to stop.” (Also see “Novo Executive Calls For ‘Real AMR global warming. Action’ In Davos “ - Scrip, 20 Jan, 2020.) Trump is withdrawing the US from the BIOPHARMA’S BIG THINKERS Novartis’s chief executive Vas Nara- Kyoto climate agreement, but neverthe- The future of healthcare, and the role of simhan will join a panel hosted by Fast less did pledge to join a new global initia- business and new technologies are once CONTINUED ON PAGE 4

FOR THE LATEST BUSINESS INSIGHT ON THE BIOPHARMA INDUSTRY VISIT: SCRIP.PHARMAINTELLIGENCE.INFORMA.COM

UK Biotech Sector J&J’s Gorsky On Immunologicals Stockwatch Financing holds up despite Q4 results season gets underway Coronavirus and earnings Brexit and Woodford (p5) with J&J pushing immunology (p8) under scrutiny (p21) IN THIS ISSUE

from the editor [email protected]

Last week, Johnson & Johnson fulfilled its time-hon- depression, Spravato, which was approved in the US in ored duty of kicking off the annual financial results March then the EU in December. Hailed as an impor- reporting season, as we report on p8. The company has tant new product, its cost and complexity of adminis- managed to shore up its all-important immunology tration may be hampering its uptake. franchise against biosimilar erosion: while sales of its The US Institute for Clinical and Economic Review one-time mainstay blockbuster Remicade slipped 17% (ICER) questioned its long-term benefits and risks and in the third full year since the US launch of a biosimi- calculated that its $32,400 US list price offered “low lar, it succeeded in growing its overall immune drug value for money” and should be discounted by 25-52%. portfolio. Partly this was down to impressive growth It also warned that even with the called-for price reduc- outside the US, but even US sales grew, thanks to J&J’s tion, treating more than 20-30% of eligible patients success in launching and expanding the indications would have budgetary impacts that could displace for newer products in a highly competitive field. On- other needed services or push up insurance costs. Over cology, which is driving much of the biopharma in- in Europe, Spravato has just been knocked back for re- dustry, also served the group well. imbursement in England in draft guidance from NICE. But there was little detail on the launch performance For more on this, check out online coverage in Scrip and of the esketamine nasal spray for treatment-resistant sister publication Pink Sheet.

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2 | Scrip | January 31, 2020 © Informa UK Ltd 2020 Amgen’s JV Buy Out AZ’s Dobber On AZ And Lokelma Plans Clovis Prostate Cancer Race

20 6 10 16

exclusive online content inside: COVER / Biopharma Leaders Join Trump And Thunberg In Davos Market Snapshot: Migraine Prevention Therapies’ Slow Road To Blockbuster Status 4 Novo Executive Calls For ‘Real AMR Action’ In Davos MANDY JACKSON [email protected] 5 UK Biotech Financing Holds Up Despite Woodford and Brexit

6 Amgen To Buy Out Japan JV With Astellas

7 Piramal Profits As It Sheds DRG To Clarivate For $950m

8 J&J’s Gorsky Hails Strong Performance By Immunology Therapies Tremfya, Stelara

10 AstraZeneca’s Dobber On Ramping In Renal, Expanding In CV And Business Development

12 Roche Gears Up For Potential Neuroscience Breakthroughs The launches in 2018 of three closely watched calcitonin gene-related peptide (CGRP) inhibitors as preventive ther- 13 New CEO Brett Monia Takes The Lead As Ionis Prioritizes apies and the anticipated launches of three more products Non-Partnered Programs against the same target have brought new attention to migraine decades after the launch of triptans – the last big class of drugs to treat the disease’s debilitating headaches 16 Horizon Sees Blockbuster Future For Tepezza After and other symptoms. But while the migraine market is US Approval large, the first round of CGRP inhibitors have yet to achieve blockbuster status. 16 AZ’s Lynparza Gets Prostate Cancer Priority Review Amgen Inc./Novartis AG’s Aimovig (erenumab) targets the CGRP receptor while Eli Lilly & Co.’s Emgality (galcan- 17 Genentech’s SMA Type 1 Data Strengthen Case Backing ezumab) and Teva Pharmaceutical Industries Ltd.’s Ajovy Risdiplam Approval (fremanezumab) both target the CGRP ligand; all three are approved to prevent headaches in patients diagnosed 20 Health Sector On High Alert As Wuhan Coronavirus Spreads with chronic migraines (15 or more headaches per month) and episodic migraines (14 or less). The drug makers es- 21 Stockwatch: Earnings Season Catches An Early Cold timated that 6 million people in the US alone are eligible As Coronavirus Sweeps In for preventive treatment with one of these new biologics. Aimovig, Emgality and Ajovy have shown similar efficacy 22 Pipeline Watch in clinical trials, cutting the number of headache days that many migraine patients experienced by half or more. And since doctors have seen many people respond well to these 23 Appointments treatments in the real world, it would seem that growth is guaranteed given the need for new medicines in this space. Published online 23 January 2020 @PharmaScrip /scripintelligence To read the rest of this story go to: https://bit.ly/3aN0UcT

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scrip.pharmaintelligence.informa.com January 31, 2020 | Scrip | 3 HEADLINE NEWS/DAVOS MEETING

CONTINUED FROM PAGE 1 Company to explore the role of CEO as Novo Executive Calls For “disruptor in chief,” something he has been pursuing with the “Unbossed” cul- tural shift in the company. ‘Real AMR Action’ In Davos The conference is not simply focused STEN STOVALL [email protected] on political and corporate leaders making well-intentioned statements on trade and ime is fast slipping away for finding workable solutions to address antimicrobial global co-operation, however. resistance, and the World Economic Forum’s annual meeting this week offers a rare Two years ago President Trump met T opportunity to spur action, says Lars Rebien Sørensen, chairman of the Novo Nor- Bayer’s Baumann and Novartis’s Narasim- disk Foundation. The foundation is sponsoring a panel debate on AMR on the first day of han and other corporate leaders at a pri- the elite gathering in Davos, Switzerland, in hopes of spurring real action on the issue. vate dinner. The annual World Economic Forum from 21-24 January will be attended by heads of state, global business leaders and others who have great influence on setting policy, both nationally in their own countries, and potentially worldwide. “Hopefully with this discussion on AMR we can promote understanding and discussion about finding solutions in Davos and hopefully take this effort further,” Sørensen told Scrip in advance of the Tuesday panel. The backdrop is well known - with estimates suggesting 700,000 people a year die as a result of AMR, big pharma having largely exited the space, and no viable reward system in place for supporting novel antibiotics, there is a void where potential pandemics could thrive, said Sørensen, a former CEO of Danish diabetes specialist Novo Nordisk AS. “The market for new antibiotics is broken. We need action on AMR from the responsible players in national health systems, from pharmaceuticals manufacturers, research-based organizations, generics makers. We need to get them together to come up with solutions, otherwise we will soon have a major problem on our hands,” the executive said. Novo Holdings has tried to make a difference by establishing an alternative funding entity Bayer CEO Werner Baumann called REPAIR (Replenishing and Enabling the Pipeline for Anti-Infective Resistance) Impact This time around, the US presidential Fund which invests in start-ups, early-stage companies and corporate spin-outs across Eu- election on 3 November is drawing closer, rope and North America involved in discovering and early-stage development of therapies and despite impeachment proceedings to combat AMR. “We decided 18 months ago to set up REPAIR with the aim to try to kick against Trump, many predict he could be start a movement that would put AMR higher on the agenda because this is an underap- returned to office for a second term. preciated risk that society is facing,” Sørensen explained. Biopharma leaders are sure to want to “The whole infrastructure of continuing the development of these new product ideas impress upon the president their views has deteriorated. The whole pipeline of developing new antibiotics has been deteriorat- on US healthcare reform, including ing. Meanwhile many former pharma producers have left the antibiotics space, and many Trump’s own suggestion of international of the research-based entities have collapsed. The situation is therefore actually worse reference pricing. This is something the than it was a year and a half ago, in terms of bringing antibiotics forward,” he added. sector fears almost as much as Democrat Speakers headlining the Davos AMR panel on Tuesday will be Novo Holding’s Sørensen, candidate proposals for “Medicare for Jayasree Iyer who heads the Access to Medicine Foundation, the director general of phar- All” and US government price negotia- ma manufacturing trade body IFPMA Thomas Cueni, and Wellcome Trust CEO Jeremy Far- tions with the industry. rar. The panel’s chairman will be Kevin Outterson, who heads the Boston-based CARB-X China’s vice-premier Han Zheng also non-profit partnership for accelerating antibacterial research. gave a speech in Davos on 21 January, The discussion will have as a starting point the view that government intervention and and defended globalisation while making policy change is essential and demand both long-term and short-term solutions to battle veiled criticism of Trump-led US market AMR, including market entry rewards, perhaps designed as prescription models along the protectionism. lines of pilots underway in the UK and Sweden. China is now firmly established as “By conducting their own pilots, Sweden and the UK are acknowledging that this is a the biopharma’s second biggest mar- huge problem. But it is not nearly enough to just have two countries engaged in this ef- ket – but is less open to discussions fort to find a workable reward model for antibiotics,” Sørensen said. with industry on political policy-making “Pharmaceuticals is a global business. It calls for a much larger market and much more than the US. The sector has just agreed investment. The pharmaceutical companies have the capabilities; they have the manufac- to slash prices of scores of medicines turing sites, some of them are research-based AstraZeneca Pledges To in China by more than 60% in order to and they therefore have the research capabil- Become Carbon-Negative secure inclusion on the state-run insur- ities. Some are generics manufacturers and By 2030: ance scheme. have the manufacturing capabilities.” https://bit.ly/2O3ay1l Published online 22 January 2020 Published online 21 Jan 2020

4 | Scrip | January 31, 2020 © Informa UK Ltd 2020 BIOTECH FINANCING

UK Biotech Financing Holds Up Despite Woodford and Brexit ANDREW MCCONAGHIE [email protected]

ew figures show that 2019 was raised over £1bn across all financing was a series B round, the £100m raised by another strong year for the UK bio- routes. The year’s total of £1.33bn was, T-cell therapy focused Achilles Therapeu- Ntech sector, with a total of £1.3bn however, a sharp decline compared to the tics in September. ($1.71bn) raised by companies, underlin- previous year’s tally of £2.20bn, though Overall, the figures confirmed once ing its status as the leading cluster in Eu- this may prove to be an outlier fuelled by again the UK’s lead in Europe in terms of rope. 2018’s exceptional number of IPOs, such venture capital raised, accounting for a This was a strong showing despite as those from Autolus Ltd. and Orchard quarter (26%) of the continent’s total. global economic uncertainty, plus the Therapeutics Ltd. The BIA has reiterated that the UK is the more local problems of the implosion of Data published by the UK BioIndustry third global life sciences cluster behind Neil Woodford’s investment funds, and Association (BIA) and Informa Pharma the twin US hubs of Cambridge, MA and the UK’s long-running political paralysis Intelligence (the publisher of Scrip) in the San Francisco bay area. caused by Brexit. the report show that venture capital was The biotech association’s chief execu- Much of this Brexit uncertainty is now responsible for the lion’s share of the to- tive Steve Bates has just returned from over, as the UK’s exit is set to be confirmed tal in 2019 (£679m) with a much smaller the J.P. Morgan investor conference in San within days, but Boris Johnson’s policy of amount (£64m) raised via IPOs – most of Francisco, where the BIA worked along- ‘non-alignment’ with the European Union this from the sector’s single US NASDAQ side the government to promote the UK could undermine UK biotech’s long-term launch, from Bicycle Therapeutics. as a destination for biotech investment. growth trajectory. The remaining sum of £596m came This included an eye-catching invest- Despite these problems, 2019 was the from all other public financings. The year’s ment by Novartis in a large-scale clinical fifth year in a row that the sector had single biggest fundraising in the sector TURN TO PAGE 6

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scrip.pharmaintelligence.informa.com January 31, 2020 | Scrip | 5 BIOTECH FINANCING/DEALS

CONTINUED FROM PAGE 5 UKUK biotech biote financech n araisingnce r2015–2019aising 2015-2019 trial and market access deal with NHS After a record-breaking 2018, totals raised by UK biotechs fell back to £1.33bn ($1.74bn) in 2019 England for its late-stage cardiovascular 2400 drug inclisiran. 2200 Bates said: “The UK biotech sector continues to chart an ambitious global path. 2000 With five consecutive years of raising over 1800 £432m £1bn and a 400% increase in investments 1600 £307m since 2012, the sector is in a very strong 1400 1200

position heading into a new decade. £m £658m “It’s clear that UK biotech companies 1000 £234m remain an attractive investment opportu- 800 £775m £344m £596m nity for global investors, meaning there’s 600 a greater diversity of capital than we saw £452m 400 five years ago.” 200 However the sector has a big Brexit £795m £681m £515m £1113m £679m cloud over it. After nearly four years of un- 0 2015 2016 2017 2018 2019 certainty the UK is set to confirm its exit Venture Capital All other public nancings IPO from the EU at the end of January, but life Source: BIA: Global and growing: UK biotech nancing in 2019 science leaders have been alarmed by sig- nals from the UK government that it does not intend to align with EU regulations. “While we welcome overseas invest- for small companies. “In the UK public mar- The sector has long warned that such ment, diversifying the domestic life sci- kets, it has been a much more difficult year a move would severely disadvantage the ences investor base is critical to capturing with few IPOs and a handful of follow-on UK, forcing it to set up its own regulatory the full benefits of this key sector of the UK offerings. Barring a couple of exceptions, arrangements separate from the Europe- economy,” he said. stock performance has in general been an Medicines Agency (EMA), and making He called on the government to lever- disappointing for life sciences companies.” it a less attractive market in the process. age new scale-up capital through the Companies such as Autolus, AdaptIm- The BIA is working hard with UK gov- British Business Bank and the UK pension mune and Bicycle Therapeutics saw their ernment agencies such as the Office For funds industry, with more grant funding share prices decline in 2019, either due Life Sciences (OLS) to introduce new in- for early-stage companies through the ex- to underwhelming clinical development centives and streamlined regulatory pro- isting Biomedical Catalyst fund. news, links to the Woodford debacle, or cesses to mitigate any such impact, but Dan Mahony, partner, Polar Capital, market adjustments during the year. The still hopes to persuade the government to commented in the report that it was al- long term success of the sector will be remain aligned as closely as possible with ways going to be hard to follow the great heavily dependent on how well the com- the EU. success of 2018, but said VC funding re- panies can convert their promising science Beyond Brexit, Bates is calling for extra mained ”reasonably robust” in 2019. Nev- into commercial products, though access measures from the UK government to ertheless he noted that Brexit had been to continued funding will also be a crucial stimulate investment in the UK. the chief cause for a drying up of funding factor. Published online 23 Jan 2020 Amgen To Buy Out Japan JV With Astellas IAN HAYDOCK [email protected]

mgen Inc. has confirmed it will products to the Japanese market. Astellas purchase the 49% of shares in its initially used its development and mar- AJapanese joint venture Amgen keting capabilities to get the products Astellas BioPharma KK currently held by to market, while participating financially, Astellas Pharma Inc., effective 1 April, con- while Amgen benefited from a strength- verting the Tokyo-based operation into a ening commercial presence. wholly owned affiliate. Since being set up, Amgen Astellas Bio- The move is as envisioned in the origi- Pharma has now grown into an organiza- nal strategic alliance contract signed be- tion with over 600 employees and fully tween Amgen and Astellas in 2013, under integrated operations that have enabled which the JV started operations that year it to become a marketing authorization to bring a portfolio of five novel Amgen holder for its products in Japan.

6 | Scrip | January 31, 2020 © Informa UK Ltd 2020 DEALS

The venture has already launched Repatha (evolocumab) for and Immunology Business Unit. The drug is approved globally familial hypercholesterolemia or hypercholesterolemia in patients in various indications including psoriasis. Japan is seen as one of with high cardiovascular event risk who do not adequately respond the key growth markets for the PDE4 inhibitor. (Also see “Amgen’s to statins, Blincyto (blinatumomab) for patients with relapsed or $13.4bn Otezla Buy Helps Bristol/Celgene Merger Close By Year- refractory B-cell acute lymphoblastic leukemia, and Evenity (romo- End” - Scrip, 26 Aug, 2019.) sozumab) for osteoporosis patients at high risk of fracture. No financial details of the joint venture buyout transaction were The new post-buyout renamed Amgen KK Japanese subsidiary, provided. No upfront or milestone payments were involved in the which will remain in Tokyo but at a different site, will continue to original agreement, as part of which the JV’s sales were booked by co-promote these three products with Astellas, which will remain Astellas, with Amgen to receive royalties and costs and profits to responsible for distribution and sales beyond 2020. be split equally. The Otezla (apremilast) business acquired from Celgene for Amgen Astellas head Steve Sugino will continue to serve as $13.4bn last November has already been incorporated into Am- president of Amgen KK. gen KK’s structure with the establishment of a new Inflammation Published online 23 January 2020 Piramal Profits As It Sheds DRG To Clarivate For $950m VIBHA RAVI [email protected]

ust over a decade after the sale of the Piramal group’s formu- DEAL STRENGTHENS FINANCES lations business to Abbott Laboratories Inc., chairman Ajay PEL had invested the sales proceeds in setting up a financial ser- JPiramal has stitched up another profitable deal. Piramal En- vices business and acquiring a diverse set of companies and as- terprises Ltd. (PEL) is selling its stake in fully owned Decision Re- sets. The returns have been mixed. sources Group (DRG), a healthcare research and consulting com- While the investment in Vodafone paid off handsomely in 2014, pany, to Clarivate Analytics Plc for $950m. with PEL making a reported 52% return on its 11% stake sale in The divestment gives PEL $900m in cash and an undefined Vodafone India Ltd to a subsidiary of Vodafone Group Plc, the bet stake in Clarivate worth $50m post a year of the deal closure. PEL in financial services hasn’t worked to plan. realized 2.3 times its initial equity investment on Indian rupee This business, which accounts for over half of PEL’s revenues, ran terms after acquiring DRG from private equity firm Providence into trouble due to exposure of its loan book to the real estate Equity Partners in 2012 for $650m, of which $260m was infused sector and small and medium-sized manufacturing companies, as equity. which were hit by a business slowdown. While borrowers found “We are pleased to have grown DRG’s market leadership over it difficult to pay back their loans, a large proportion of short-term the last few years and believe that to further accelerate DRG’s borrowings meant PEL faced an imbalance in its funds flow. growth trajectory, it requires scale and size. Clarivate Analytics To improve liquidity and its debt-equity ratio, PEL has been is well positioned to provide the required platform for the next raising equity funds, apart from reducing short-term borrowings phase of growth,” said Ajay Piramal. and diversifying its loan book. As part of a plan to bring in around “This is a milestone acquisition which doubles the size of our INR100bn in PEL via equity, a INR17.5bn preferential allotment life sciences business...(and) is accretive to our 2020 earnings,” said of compulsory convertible debentures was made to Canadian Jerre Stead, Clarivate Analytics executive chairman and CEO. Clari- institutional investor, Caisse de dépôt et placement du Québec vate said it expects to achieve cost synergies of approximately (CDPQ), and a rights issue of INR36.5bn to existing shareholders is $30m within the first 18 months of the transaction closing. currently underway, set to close 21 January. While the PEL board has approved the stake sale, it needs share- “Company is also subsidiarizing its pharma business and work- holders’ nod at a meeting to be held on 13 February. The deal is ing towards raising additional around 20% equity capital in the expected to be completed by 28 February. pharma business to tap organic and inorganic growth opportuni- DRG, referred to as PEL’s healthcare insights and analytics busi- ties. Along with the ongoing equity capital raise in PEL, this trans- ness, contributed INR13.30bn ($187.2m) or 10% to PEL’s revenues action not only further strengthens the company’s balance sheet for the financial year 2019 but reported a net loss of INR2.18bn. but also marks another step towards significantly unlocking value During the first half of fiscal year 2020, it had revenues of in future,” said Ajay Piramal. (Also see “Piramal Eyes 20% Pharma INR6.52bn, a 14% increase over the same period in the previous Stake Sale” - Generics Bulletin, 8 Jan, 2020.) fiscal year. PEL is looking at monetizing more of its investments in the Shri- PEL’s pharmaceutical business, which brought in 36% of its ram group of companies too. In June 2019, Piramal Enterprises FY2019 revenues, has also been growing steadily and the com- sold its stake in Chennai-based Shriram Group’s Shriram Transport pany is expected to introduce several products in 2020 as a non- Finance for INR23bn. It is also looking to exit Shriram Capital Ltd, compete clause with Abbott has ended. in which it holds a 20% stake. Once the stake sales are through, In 2010, PEL, then known as Piramal Healthcare Ltd, had sold Piramal Enterprises can increase its focus on building the pharma- its formulation business to Abbott for $3.7bn, making a sizeable ceutical business into the formidable entity it used to be. profit in the process. Published online 20 January 2020

scrip.pharmaintelligence.informa.com January 31, 2020 | Scrip | 7 Q4 RESULTS

J&J’s Gorsky Hails Strong Performance By Immunology Therapies Tremfya, Stelara JOSEPH HAAS [email protected]

ohnson & Johnson has made a major with US sales of $2.52bn up 6.8% and ex- als. (Also see “J&J Targets Tremfya Growth push with its immunology portfolio US revenue just over $1bn up 1.9%. For the In PsA” - Scrip, 13 Nov, 2019.) Jeven as its mature Remicade (inflix- year, immunology revenue of $13.95bn Now J&J’s top-selling pharmaceutical imab) franchise began to wane and faced was up 6.3%, mirroring a 6.3% uptick in US product, Stelara (ustekinumab) posted biosimilar competition. CEO Alex Gorsky sales to $9.64bn and a 65% increase ex-US 18.6% worldwide growth during the quar- cited the immunology sales performance to $4.31bn. ter to $1.7bn. The US figure of $1.19bn as a particular factor in the company’s Gorsky pointed out that for the full was up 18.4%, while international sales fourth quarter and full year 2019 success. year, J&J pharmaceuticals posted 5.8% of $506m were up 19.1%. For the full year, Gorsky told the 22 January earnings sales growth not including foreign ex- Stelara totaled $6.36bn, a 25.2% increase, call he was “extremely proud” particularly change, which outpaced the company’s with US sales of $4.34bn up 25.3%. At the of the immunology group’s sales perfor- peers. This performance “more than J.P. Morgan Healthcare Conference on mance. “The way that they were able to offset the loss of exclusivity due to bio- 17 January, J&J’s worldwide chairman of manage the biosimilar impact with Remi- similar competition and generic erosion pharmaceuticals Jennifer Taubert predict- cade while simultaneously launching mul- as well as new competitive entrants and ed that J&J’s sales momentum would con- tiple new indications for compounds like other market pressures,” he said. “Now, it’s tinue with 10 launches – including label Stelara, launching Tremfya,” he said. The important to note that our robust growth expansions for already approved drugs – interleukin-23 inhibitor Tremfya (gusel- can be attributed to volume, not price, expected between 2019 and 2023. (Also kumab) moved into blockbuster territory and our sales growth is a reflection of see “J&J To Power Ahead With 10 Launches during the third quarter, which the com- the increased number of patients we’re In Four Years “ - Scrip, 17 Jan, 2020.) pany attributed in part to head-to-head reaching with our transformational med- The oncology franchise also produced data against Novartis AG’s IL-17 blocker icines for unmet needs.” a strong quarter with worldwide sales of Cosentyx (secukinumab). (Also see “J&J’s $2.72bn up 10.6%. Darzalex (daratumum- Tremfya Gets Its Blockbuster Wings “ - Scrip, TREMFYA, STELARA POST ab) and Imbruvica (ibrutinib) were the top 15 Oct, 2019.) DOUBLE-DIGIT GROWTH sellers, with the former yielding $830m Remicade brought in $1.04bn globally Indicated for psoriasis, Tremfya global globally, up 44.5% year-over-year, and the during the fourth quarter, down 16% from sales tallied $270m during the fourth latter, partnered with AbbVie, bringing in $1.24bn one year earlier, due to biosimilar quarter, with the US total of $199m a $875m, up 26.6%. Overall, cancer therapy competition and increased discounts. In 39.7% year-over-year increase. Outside brought in $10.69bn in 2019 for J&J, an the US, quarterly sales of $755m meant a the US, where Tremfya has been avail- 11.9% uptick over 2018. 10.4% decline. For the full year, Remicade able for less than a year, sales reached J&J still is not breaking out individual sales of $4.38bn decreased 16.8% from $71m. The overall increase for the quar- sales numbers for major depressive disor- $5.33bn in 2018, with US sales down 16% ter compared to Q4 2018 was 55.1% For der drug Spravato (esketamine), launched to $3.08bn. Remicade sales have been the full year, the product realized world- last March. In September, the firm filed dropping for years, as Pfizer Inc. and Cell- wide sales of $1.01bn, with US sales of a supplemental new drug application trion Inc. launched Inflectra (infliximab- $764m up 68.5%. (sNDA) to add treatment of MDD with dyyb) in November 2016. But J&J has held The traction is notable given the crowd- suicidal ideation to the product’s US label. it off to a large degree due in part to J&J ed competitive field for Tremfya, where (Also see “J&J Looks To Expand Spravato signing exclusive contracts with payers to it contends with the IL-17 blockers in- Label To Suicidal Ideation” - Scrip, 10 Sep, cover Remicade. (Also see “Exclusive Remi- cluding Cosentyx and Eli Lilly & Co.’s Taltz 2019.) Vice president of investor relations cade Contracts Are Slowing Biosimilar Up- (ixekizumab), as well as the second-to- Christopher DelOrifice told the call that take” - Scrip, 1 Aug, 2017.) market IL-23 blocker, AbbVie Inc.’s Skyrizi “patient demand continues to build [for J&J reported 4.4% global sales growth (risankizumab), which was approved last Spravato], and the unmet need remains for its pharmaceuticals division in the April. (Also see “AbbVie’s Humira Succes- very high. New patient starts continue to fourth quarter, with full-year sales growth sion Plan Begins Taking Shape With Skyrizi steadily increase each month with over for the entire company of 2.8%, not count- US Approval” - Scrip, 24 Apr, 2019.) 3,500 patients being treated to date.” ing the impact of foreign exchange rates. J&J hopes to grow Tremfya’s sales fur- In a same-day note, Morningstar ana- Gorsky noted this was J&J’s 36th consecu- ther this year with supplemental approval lyst Damien Conover pointed out that tive year posting overall revenue growth. applications for psoriatic arthritis pend- pharmaceuticals continues to be J&J’s Immunology tallied $3.52bn worldwide ing in the US and Europe backed by the strongest-performing unit, compared to on the quarter, up 5.4% year-over-year, Phase III DISCOVER-1 and DISCOVER-2 tri- consumer health and medical devices,

8 | Scrip | January 31, 2020 © Informa UK Ltd 2020 Q4 RESULTS

despite the impact of generic and biosimilar competition. “Gener- ciple,” chief financial officer Joseph Wolk said. “We remain, I would ics to cancer drugs Zytiga (abiraterone) and Velcade (bortezomib) say, cautiously optimistic that that’s progressing very well. We’re as well as biosimilar pressure to immunology drug Remicade highly engaged to the extent we can be to finalize that agreement weighed on the overall drug sales,” he wrote. “However, newer in principle, and we hope to hear more over the coming months drugs launched in those indications offset the generic competi- from the lead negotiators representing not just the states, but as tion. We expect continued strong growth from newer drugs, in- you know, the counties and municipalities.” cluding cancer drugs Darzalex and Erleada (apalutamide) as well The company added that it expects its cash flows to decrease as immunology drug Tremfya.” by about 10% in 2020 due to the opioid settlement. Bernstein Erleada, which is approved to treat non-metastatic, castration- analyst Ronny Gal estimated this could mean a cash flow de- resistant prostate cancer, brought in $116m during the fourth cline of as much as $2bn for J&J, but added in a 22 January quarter and $332m for full-year 2019. (Also see “J&J’s Erleada Ap- note that the company’s cautious optimism that a settlement proved By FDA In New Prostate Cancer Indication “ - Scrip, 19 Sep, is near was a good sign for all of the companies affected by the 2019.) Zytiga worldwide revenue of $677m during the fourth settlement process. quarter was a 12.9% decrease, while Velcade’s $115.2m meant a Gorsky indicated that J&J’s approach to business develop- 55.2% decline. ment would continue to focus on bolt-on type acquisitions rather than larger deals, noting that across all three of its busi- AWAITING OPIOID SETTLEMENT FINALIZATION; ness areas, the company paid more than $7bn in 2019 for 11 STAYING THE COURSE IN M&A acquisitions and six licensing agreements. “We have contin- J&J really had nothing new to report about its expected settle- ued, I think, our pattern of tuck-in acquisitions where we get ment in the ongoing opioid lawsuits – the company has reserved new technologies, as you saw that we did with both XBiotech $4bn for damages in the pending cases. (Also see “J&J Oklahoma Inc. and other opportunities in our pharmaceutical business,” Judgement May Set Benchmark For Massive Opioid Resolution” - the exec said, adding that J&J will continue looking at oppor- Pink Sheet, 26 Aug, 2019.) tunities “across all three of our segments.” “With respect to the opioid settlement and agreement in prin- In December, J&J paid $750m up front to license worldwide ciple that was announced shortly after our Q3 earnings, where we rights to XBiotech’s interleukin-1 alpha antagonist bermekimab set aside $4bn, we continue to work with the negotiating commit- in Phase II for atopic dermatitis and hidradenitis suppurativa. tee of the state attorney generals to finalize the agreement in prin- Published online 23 January 2020

Scrip Awards Winner 2019

Best Contract Research Organization – Full-Service Providers

ICON contributed to the development of 21 new drugs in 2018/19 and exceeded sponsor expectations, beating industry medians through strong collaboration and Winner: ICON detailed planning during start-up, careful site selection and monitoring progress of enrolment and in-depth global regulatory expertise.

“ICON is honoured to have been named as the Best Contract Research Organization – Full-Service Providers at the Scrip 2019 Awards. This achievement completes a strong year for ICON, with over 12 high proﬁ le industry awards. We are very proud to be recognised for our notable developments in patient, site and data solutions, innovation in trial design and real world evidence,” Steve Cutler, Chief Executive Oﬃ cer, ICON

JN3086 Scrip Awards 2019 Winner Advert B.indd 1 2020/01/28 17:11 scrip.pharmaintelligence.informa.com January 31, 2020 | Scrip | 9 J.P.MORGAN MEETING

AstraZeneca’s Dobber On Ramping In Renal, Expanding In CV And Business Development JESSICA MERRILL [email protected]

his year AstraZeneca PLC Biophar- Both launches will be challenging as maceuticals President Ruud Dob- AstraZeneca ramps up in a new space T ber is focused on executing on the that presents specific market access chal- company’s expansion into kidney disease lenges, both around commercial payer with the launch of Lokelma (sodium zirco- dynamics and Medicare restrictions for nium cyclosilicate) underway for hyper- drugs administered in dialysis centers un- kalemia and a second potential approval, der the end-stage renal disease bundled roxadustat, anticipated later in 2020. payment system. Dobber, who oversees AstraZeneca’s “From a market access perspective this global drug portfolio outside of oncology, is a tough area,” Dobber acknowledged. talked with Scrip at the J.P. Morgan Health- The company has established a renal care conference in San Francisco on 14 commercial sales force for the launch of January. He has been leading the biophar- Lokelma, which will likely be expanded maceuticals business for a year, having to support the launch of roxadustat, previously run AstraZeneca’s North Ameri- Dobber said. can commercial operations. He discussed With the launch of Lokelma, AstraZen- the ongoing expansion in renal disease, eca is competing against Vifor Pharma the company’s commitment to cardio- Group’s Veltassa (patiromer), which was vascular disease, including the move into “From a market access approved by the FDA in 2015, but carries heart failure, and business development. warnings about constipation and can in- The commercial oncology business is perspective this is a teract with other oral drugs. overseen by David Fredrickson, who sep- tough area.” “We are extremely pleased because arately talked to Scrip about the launch within the six months after the commer- of Enhertu (trastuzumab deruxtecan) in – Ruud Dobber cial launch we are equivalent or even HER2-positive breast cancer. (Also see higher in what is called new-to-brand pre- “J.P. Morgan Notebook Day 2: Bourla Feels scriptions, which is an incredible achieve- Pfizer’s Underappreciated, GSK Prepares ment of the medical and commercial team For Myeloma First And More” - Scrip, 15 so quickly,” Dobber said. Jan, 2020.) There is significant underdiagnosis in Among the launches on the biophar- the space because of lack of awareness on maceutical side of the business are two the part of general practitioners and cardi- in renal disease: Lokelma, which was ap- ologists and to a smaller extent nephrolo- proved by the US Food and Drug Adminis- 2019.)(Editor’s note: this story has been gists, the exec explained. tration (FDA) for hyperkalemia in 2018 but updated to reflect that AstraZeneca ex- Hyperkalemia is high serum potas- didn’t launch until mid-2019, and roxadu- pects FDA approval, but not necessarily the sium concentration in the blood that stat for the treatment of patients with ane- launch, of roxadustat in 2020). can be asymptomatic but can cause mia from chronic kidney disease. long-term damage. It is often caused by Dobber confirmed that AstraZeneca BUILDING MARKET CKD or other metabolic deficiencies. The filed an application for roxadustat with ACCESS IN “A TOUGH AREA” standard of care has been a restrictive the FDA before the end of the year. The Both launches are core elements of As- diet or an old generic medicine, sodium drug, developed with partner FibroGen traZeneca’s strategy to expand in kidney polystyrene sulfonate, that carries a lot Inc., is a first-in-class HIF-PH inhibitor disease beyond diabetes, where it has of side effects. There are roughly 3 mil- that has shown improved safety and effi- long had a presence. In 2018, AstraZen- lion patients in the US with hyperkale- cacy in clinical trials over the standard of eca renamed its CVMD (cardiovascular/ mia, according to AstraZeneca. care, erythropoiesis-stimulating agents, metabolic disease) therapy area to CVRM “Hyperkalemia is not always seen by and which AstraZeneca hopes will be ap- (cardiovascular/renal medicine) to reflect payers as an area of high unmet medical proved for both CKD patients on dialysis the growing emphasis on renal disease need, so we need to do a lot of education,” and those not on dialysis. (Also see “Clear outside of diabetes. (Also see “Next Up For Dobber said. “Our access is getting better Path For AstraZeneca’s Roxadustat After AstraZeneca: Building Out In Renal Disease every day and the expectation clearly is Reassuring Safety Data” - Scrip, 11 Nov, “ - Scrip, 24 Oct, 2018.) that we will exceed Veltassa for 2020 from

10 | Scrip | January 31, 2020 © Informa UK Ltd 2020 J.P.MORGAN MEETING

a prescription standpoint.” Senior VP for SGLT2 inhibitor outside of diabetics. Farx- vascular space can be. The company an- market access Rick Suarez also talked iga was already approved to reduce the nounced on 13 January the discontinua- to Scrip about the launch. “We are very risk of hospitalizations for heart failure in tion of the cardiovascular outcomes trial pleased with payers’ willingness to add type 2 diabetics with cardiovascular risk testing Epanova in patients with high it to formulary and preferred positions factors in October, the first SGLT2 to win triglycerides who are at increased risk at that,” Suarez said. “Some of the largest the indication. of cardiovascular disease due to a low Medicare payers in the country have done “It will open up a completely new pa- likelihood of success. (Also see “AZ Halts so, and we are also seeing very good up- tient population,” Dobber said. “We have Epanova Study As High Placebo Effect Kills take in hospitals.” high expectations of course for this op- Acasti’s Omega-3 TRILOGY-1 Trial “ - Scrip, As for the anticipated launch of roxadu- portunity.” The expanded filing was based 14 Jan, 2020.) stat, Dobber said it is a complex product on the Phase III DAPA-HF trial that showed The drug was first approved by the FDA with two separate segments of the market the drug reduced the composite endpoint in 2014 for the treatment of high triglyc- to target, dialysis and non-dialysis. of cardiovascular death or worsening erides, but a positive cardiovascular out- “We will have specific people detailing heart failure by 26% when given on top comes trial would have opened the drug in the dialysis centers versus [to] nephrol- of standard of care, with benefits seen in up to a potentially broader cardiovascular ogists working in hospitals and clinics,” he both diabetics and non-diabetics. (Also risk reduction claim. A rival drug, Amarin said. “We have a very strong field force al- see “Farxiga Data Change Heart Failure Corp. PLC’s Vascepa (icosapent ethyl), has ready but probably it is reasonable to say Treatment Outlook” - Scrip, 2 Sep, 2019.) succeeded where Epanova failed, result- that we will expand it a little bit more.” He said AstraZeneca will remain coming in mega-blockbuster sales expecta- As for the challenging reimbursement mitted to the diabetes space, but that tions from some analysts. (Also see “Sales environment for bundled payments in di- within diabetes, the focus will move Already Growing As Vascepa Secures Car- alysis centers under Medicare, Suarez said more toward cardiovascular disease and dio Approval” - Scrip, 16 Dec, 2019.) the company is prepared. comorbidities of diabetic patients. That “This is a very hard place because the “The bundles create their own chal- comes after another major diabetes bar is very high,” Dobber said of the cardio- lenges because the legislative environ- player, Sanofi, announced in Decem- vascular therapeutic area. “But that makes ment can change at any time, but we feel ber that it will end diabetes research it also very attractive if you find a molecule we have a good grasp of how it is currently altogether. (Also see “Sanofi, Long-Time that can further reduce [the risks].” reimbursed and how our product will be Leader In Diabetes, Is Exiting Diabetes Re- As for building in cardiovascular disease treated in the bundle,” Suarez said. “Time search“ - Scrip, 10 Dec, 2019.) through business development, Dob- will tell.” ber said it is challenging to find a break- A HIGH BAR FOR through asset. “As a large company, we are MOVING BEYOND DIABETES CARDIOVASCULAR DISEASE scanning every day potential opportuni- WITH FARXIGA At a time when there is some renewed in- ties,” he stated. He said CEO Pascal Soriot In addition to the two new launches, As- terest in the cardiovascular disease thera- and chief financial officer Marc Donoyer traZeneca also has a potential large com- py among some big pharma players, Dob- would take a critical view of any assets he mercial opportunity coming in the first ber reiterated AstraZeneca’s commitment might bring to their attention because of half of the year from the approval of the to the space. the high valuations. But, he added, “if it diabetes pill Farxiga (dapagliflozin) for “There is a lot of attention of oncology makes sense, there is a clear willingness the treatment of heart failure. The FDA and the breakthroughs in oncology, but to in-license or buy those new assets, but granted a priority review for Farxiga for we need to understand the number one there are not too many,” he said. the reduction of cardiovascular death or killer in the world is still cardiovascular dis- Two areas AstraZeneca has identified worsening of heart failure in adults with ease by far,” he said. AstraZeneca is looking as new areas for expansion are gene reduced ejection fraction (HFrEF) both increasingly at inflammation as a trigger therapy and cell therapy, he added. with and without type 2 diabetes, with ac- for atherosclerotic plaques and heart at- “It is fair to say we are not one of the tion expected in the second quarter. (Also tacks, with work ongoing in R&D. frontrunners at the moment, but clearly see “AZ’s Farxiga Gets FDA Priority Review But it is a challenging space. He point- there is a huge commitment from our For Heart Failure” - Scrip, 6 Jan, 2020.) ed to AstraZeneca’s experience with Ep- R&D colleagues in order to have a close The filing represents a significant op- anova (omega-3-carboxylic acids) as an look,” Dobber noted. portunity for AstraZeneca to expand the example of just how tough the cardio- Published online 23 January 2020

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scrip.pharmaintelligence.informa.com January 31, 2020 | Scrip | 11 J.P. MORGAN MEETING

Roche Gears Up For Potential Neuroscience Breakthroughs

ANDREW MCCONAGHIE [email protected]

ne of the most eye-catching re- Anderson hinted last week that the Graham said this same approach was marks made at last week’s J.P. company intends to undercut its rivals on now being taken in the neuroscience OMorgan conference in San Fran- price, with the aim of maximising its share therapy areas it is preparing to enter. cisco was a bold prediction by Roche’s of the market, as it looks unlikely to show “We’re spending a lot of time getting pharma division head Bill Anderson. clinical superiority against its competitors. to know the [physician] community to He told investors: “We think that neu- Meanwhile, Roche and Ionis Pharma- really figure out what they need, and roscience has the potential to be in the ceuticals Inc.’s first-in-class Huntington’s what their patients need. We’re trying to ‘20s what oncology has been in the last disease therapy HTT-ASO (RG6042) could take a very customer-centric approach in decade…and we have a very strong sci- be filed and approved by the end of 2020. all of our sales and marketing activities entific and development effort there.” The drug entered a pivotal Phase III trial, going forward.” This is a bold statement – as oncol- GENERATION-HD1 last year, with a pri- She noted that many of the patient ogy looks unlikely to be knocked off the mary completion date of March 2022, but needs in these disease area differed great- number one spot in terms of biopharma the urgency of unmet need for patients ly from cancer – they are more chronic revenues – but neuroscience could never- means regulators are likely to accept ear- conditions, and physicians can be more theless prove to be one of the decade’s big lier data for filing. safety adverse, with fears of disability a growth drivers. Roche is expected to seek approval leading concern. In terms of sheer numbers of patients, based on open-label extension study “That does require you to have a dif- within neuroscience it is of course Al- which is due this year, as well as on interim ferent approach. I think we have cut our zheimer’s disease where the greatest un- biomarker data from Generation-HD1 and teeth in MS with Ocrevus, and there are met need is, and 2020 may see an historic results from a natural history study. a lot of lesson learned that we are able (and most likely controversial) approval of All of which means HTT-ASO could be to apply.” Biogen’s aducanumab by the US Food and approved by the end of 2020. Drug Administration. Speaking to Scrip at the J.P. Morgan SAREPTA GENE THERAPY Roche is one of the many other compa- conference, Roche’s head of global prod- PARTNERSHIP nies with a stake in Alzheimer’s research uct strategy, Teresa Graham, said the com- Perhaps Roche’s most remarkable prog- (its gantenerumab is in Phase III), but the pany was gearing up for significant ex- ress in the field last year came with the field is littered with failures, and real clini- pansion in its commercial operations for unveiling of a deal with Sarepta, in which cal progress against this disease may still neuroscience therapies. the big pharma company would commer- be some years away. The company is already undergoing a cialise the specialist company’s gene ther- However it is the runaway success of major reorganisation of its commercial apy for Duchenne muscular dystrophy. It Roche’s multiple sclerosis drug Ocrevus operations, with its US arm Genentech signed a deal worth $1.15bn which would (ocrelizumab) which has convinced the leading a shift away from a product-fo- see it take charge of marketing the drug Swiss pharma company it can build an cused marketing structure to a geographi- in all global markets outside the US. Read industry-leading neuroscience portfolio. cal approach aimed at reflecting the prior- the full article here The company has a number of key clini- ities of local healthcare systems, including The gene therapy candidate, SRP-9001, cal trial readouts and milestones in neuro- market access and reimbursement issues. is expected to produce pivotal data in science to watch out for in 2020 and 2021, reflecting a broader increase in activity Roche’s neuroscience pipeline across the broad therapy area. DRUG INDICATION CATALYST DATE

A FIRST IN HUNTINGTON’S Spinal muscular atrophy US FDA approval Risdiplam 24 May 2020 DISEASE? types 1,2,3 (expected) The first significant milestone of the year Follow up from for Roche in neuroscience is the antici- HTT-ASO (RG 6942) Huntington’s Disease Phase I/II and H1 2020 pated FDA approval of its spinal muscular potential filing atrophy (SMA) candidate risdiplam. Neuromyelitis optica US FDA approval Q3 2020 Satrolizumab This is the first oral treatment for the spectrum disorder (NMOSD) (expected) (estimated) condition (being developed for types 1,2, Balovaptan Pediatric autism Phase II data H2 2020 and 3 of the disease), and will challenge Duchenne Muscular Biogen’s Spinraza (nusinersen) and Novar- SRP-9001* Phase I/II Early 2021 Dystrophy tis’s gene therapy Zolgensma (onasemno- gene abeparvovec). * Ex-US commercial deal with Sarepta

12 | Scrip | January 31, 2020 © Informa UK Ltd 2020 J.P. MORGAN MEETING

early 2021. Phase I data suggest it could is perfectly synergistic with the portfolio this is an increasingly common precaution be the first true breakthrough in halting or that we already have. This is the first drug taken in gene therapy. reversing the disease, raising microdystro- that’s really shown any kind of benefit [in Neuroscience differs considerably to phin expression levels to 74-96% of nor- Duchenne] so I think it could be transfor- oncology and other therapy areas better mal levels three months after treatment in mational for those kids.” known to Roche, so the company is al- four patients. Sarepta and Roche will face competi- ready laying the groundwork on clinical “There’s still definitely a lot of work to tion from Pfizer and Solid Biosciences rival infrastructure (e.g. readiness for intrathe- do, I’m really excited about that data,” Duchenne gene therapy, SGT-001. This cal injections for Huntington’s disease) said Graham. product was placed on clinical hold by the and market access (including novel pay- “It is very rare for us to do an ex-US FDA in November after a patient experi- ment methods) in these therapy areas. deal like that, but the overlap with SMA enced a serious adverse event, however Published online 21 January 2020 New CEO Brett Monia Takes The Lead As Ionis Prioritizes Non-Partnered Programs

MANDY JACKSON [email protected]

onis Pharmaceuticals Inc. has monetized its expertise in an- Some things at Ionis will stay the same as the company enters tisense drug discovery by linking up with bigger biopharma- a new leadership era, including its commitments to advance Iceutical companies to lead late-stage development and com- the science of RNA-targeting medicines and to its existing part- mercialization. But going forward, new CEO Brett Monia said, the ners. However, Monia said, “the company is in a different place company will be more selective about entering into new collabo- today than before. We’re in a very strong financial position. rations and prioritize Ionis-owned drug candidates. That gives us leverage to invest … in technologies to expand Scrip spoke with Monia about Ionis’s future in an interview at the scope of antisense.” the J.P. Morgan Healthcare Conference earlier this month in San “In addition, we will begin to prioritize the Ionis-owned pipe- Francisco. Monia, a founding scientist of Ionis who most recently line,” he continued. “We’ve done a lot of partnerships over the years very successfully, and although we’re much more careful and much more selective in the partnerships we do today … we’ll continue those, but we’ll also build out the Ionis-owned pipeline and build commercial capabilities internally to maximize the commercial value of drugs that we develop and bring to approval our- selves. That’s a significant change.” The company’s three marketed drugs include Tegsedi (inoter- sen), approved in the US and Europe for polyneuropathy associated with hereditary transthyretin-mediated (TTR) amyloidosis, and Waylivra (volanesorsen), approved in the EU for familial chy- lomicronemia syndrome (FCS); both are commercialized by Akcea Therapeutics Inc., an Ionis affiliate majority owned by the parent company. (Also see “Akcea C-Suite Upheaval’s Reasons Unclear, Firm Set To Take In More Ionis Programs” - Scrip, 23 Sep, 2019.) Its third commercial product is the first-to-market spinal muscular atrophy (SMA) drug Spinraza (nusinersen), which is approved in the US and EU, and marketed by Biogen – Ionis’s most frequent collaborator. Biogen paid $1bn up front in 2018 to enter into its sixth agreement with the company; the 10-year partnership is focused on drugs for neurological and ophthalmic indications. (Also Brett Monia was a see “Biogen Re-Ups With Ionis In Search Of A Neuroscience ‘Innova- founding scientist at Ionis

Source: Ionis Pharmaceuticals Inc. Ionis Pharmaceuticals Source: tion Engine’” - Scrip, 20 Apr, 2018.) Ionis had $2.2bn in cash at the end of the third quarter of 2019, served as chief operating officer, took the helm from longtime but expected a significant increase by the end of the year from CEO Stanley Crooke in a planned leadership transition at the start product sales, royalty revenue and partnership fees announced of 2020. The change comes as the company’s pipeline is set to early in the fourth quarter. Pfizer Inc. agreed in October to pay Ak- deliver regulatory filings for 10 or more partnered and wholly- cea $250m up front – half of which will go to Ionis – to collaborate owned drugs over the next three to five years. on the development of AKCEA-ANGPTL2-LRx, which is designed

scrip.pharmaintelligence.informa.com January 31, 2020 | Scrip | 13 J.P. MORGAN MEETING

to reduce the production of angiopoietin- like3 (ANGPTL3) protein for the treatment Ionis By The Numbers cardiovascular diseases. (Also see “Pfizer Reaffirms CV Disease Commitment With ● Three approved drugs: Spinraza, Tegsedi, Waylivra Deal For Akcea’s ANGPTL3 Drug” - Scrip, 7 ● Five Phase III programs: IONIS-HTT-Rx (RG6042) in Huntington’s disease with Oct, 2019.) Roche, tofersen in SOD1 ALS with Biogen, AKCEA-APO(a)-LRx in cardiovascular Also, Bayer AG was required to pay Ionis disease with Akcea and Novartis, AKCEA-TTR-LRx in hATTR polyneuropathy with a $10m milestone fee based on the Ger- Akcea and AKCEA-TTR-LRx in hATTR in cardiomyopathy man big pharma’s decision in October to ● Ten or more new Phase II trial starts in 2020 take IONIS-FXI-LRx into Phase II; the anti- ● Five or more new Phase III starts in 2020-2021 thrombotic antisense drug candidate that reduces production of factor XI. (Also see ● Ten or more new drug application submissions by 2025 “Bayer/Ionis Advance Next-Generation An- tithrombotic After Positive Clinical Results” - Scrip, 10 Oct, 2019.) quarter and generated $82m in royalty tral nervous system … I think we’ve only Ionis’s commitment to bringing in revenue for Ionis in the July-to-September scratched the surface in CNS and there’s a outside technology will be another big period. (Also see “Novartis’s Zolgensma lot more coming.” change for the company, which tradi- Finds Commercial Legs In A First For Gene tionally has been a seller, not a buyer, of Therapy” - Scrip, 22 Oct, 2019.) MORE ALSO COMING IN novel science. Other progress in the company’s neu- CARDIO-METABOLIC, RARE “Now, with our financial position and rology pipeline in 2019 and early 2020 DISEASES where the technology is – it’s validated, includes enrollment of the first patient The CEO said Ionis’s cardio-metabolic it’s delivering medicines, we expect 10 by Roche in a pivotal trial for IONIS-HTT- pipeline also has a lot more programs or more new potential filing applica- Rx (RG6042) in Huntington’s disease, coming, including drug candidates that tions for approval over the next three Biogen’s initiation of a Phase III trial for may take the company’s antisense tech- to five years – we’re really running with tofersen in the reduction of superoxide nology into large population diseases. a lot of momentum these days,” Monia dismutase 1 (SOD1) in patients with amy- “This now gets us into very large popu- said. “It gives us the opportunity to in- otrophic lateral sclerosis (ALS), and initia- lations of patients – tens of millions of pa- vest in new technologies that do things tion of Phase III trials in TTR amyloidosis tients – with cardiovascular disease due to that antisense maybe doesn’t do so well patients who experience polyneuropa- risk factors that haven’t been treatable or today, like reaching new organ systems thy and in patients who have cardiomy- addressable with other platforms,” Monia and new cell types, and genomics invest- opathy with AKCEA-TTR-LRx. said. “A drug like our [lipoprotein(a), or ments that allow us to select new targets AKCEA-TTR-LRx was developed us- Lp(a)] program where Lp(a) is a risk factor so that we continue to populate our ing Ionis’s Ligand Conjugated Antisense that can be thought of like cholesterol is a pipeline for years to come.” (LICA) technology platform, which is de- risk factor. If you have really high Lp(a) lev- signed for selective delivery to the target- els – it’s genetically determined – you’re at NEUROLOGY PIPELINE ed cell type, allowing for greater potency, high risk of cardiovascular disease.” CONTINUES TO EXPAND less frequent dosing, new routes of de- This program, AKCEA-APO(a)-LRx, is be- “Our two largest and most productive livery for antisense drugs and delivery to ing tested in patients with cardiovascular franchises are cardio-metabolic and neu- new organ systems. disease in a Phase III trial initiated by No- rodegenerative diseases,” Monia noted. The company has six drugs in clinical vartis. Akcea and Ionis split a $150m li- In the ongoing commercialization of development for neurological indica- cense fee for the asset in early 2019 when Spinraza, the drug gained a new com- tions with more coming, since it plans Novartis opted in to the development petitor last year with US Food and Drug to move six Ionis-owned programs into program. (Also see “Deal Watch: Novartis Administration approval of the Novartis the clinic in 2020, including a treatment Opts In On Lipoprotein A Candidate From AG gene therapy Zolgensma (onasem- for Angelman syndrome. Also, Biogen Akcea” - Scrip, 25 Feb, 2019.) nogene abeparvovec) and could have selected four additional neurological More recently, Ionis and Akcea an- a second competitor with US FDA ap- disease targets in 2019 to move into nounced positive top-line Phase II results proval of Roche’s risdiplam, which is drug discovery. on 22 January for AKCEA-APOCIII-LRx, expected in May. (Also see “Genentech’s “So many other companies have ex- which is designed with the LICA tech- SMA Type 1 Data Strengthen Case Backing ited neuro or tried to slow down or tried nology to inhibit production of apolipo- Risdiplam Approval” - Scrip, 23 Jan, 2020.) to acquire from other companies, largely protein-CIII in patients who are at risk of Zolgensma generated $160m in sales because they have failed, whereas we cardiovascular disease due to high triglyc- during the third quarter – its first full believe we’ve cracked the code in neuro- eride levels. The drug lowered triglyceride quarter on the market – while Biogen re- degenerative diseases,” Monia said. “That levels in a dose-dependent manner with ported $547m in third quarter Spinraza was based on 10 years of investing in the highest monthly dose of 50mg cutting sales, which was up 12% from the second research to deliver our drugs to the cen- fasting triglycerides to 150mg/dL or less

in more than 90% of patients versus less 2018.) Ionis and Akcea still intend to pursue He noted that most of Ionis’s drugs than 5% of patients in the placebo group. approval of Waylivra in the US while ad- are administered subcutaneously with a “We believe monthly dosing is a criti- vancing AKCEA-APOCIII-LRx for FCS. self-administered pen or locally, but the cal advantage that may ultimately allow company believes it has figured out how these drugs to treat larger populations, INFECTIOUS DISEASES, to deliver its drugs orally as well and will particularly ones with asymptomatic con- ONCOLOGY AND BEYOND have data in 2020 on its first oral therapy. ditions,” Needham analyst Chad Messer “Where are we going beyond this in “It may sound a bit audacious, but I re- said in a same-day note suggesting that the future? We, of course, have other ar- ally believe Ionis has the potential to be the the AKCEA-APOCIII-LRx data validate Io- eas that we’re working in – in infectious best company in the biotech industry, be- nis’s LICA technology. diseases and an HBV program; oncology cause of the size of our pipeline, the perfor- The companies plan to move AKCEA- is a growing area for us and other disease mance of our technology and where I see APOCIII-LRx into a Phase III trial in patients areas as well,” Monia said. it going,” Monia said. “There’s a reason why with FCS – a disease in which patients GlaxoSmithKline PLC opted in to devel- so many companies want to partner with are unable to break down fats, leading to op a pair of drug candidates for the hepa- us – they want access to this technology, pancreatitis – and they intend to explore titis B virus (HBV) in August. because they see what it’s delivering.” further development of the drug for other “One area that we’re moving into, where Published online 24 January 2020 rare and common diseases associated I think we’ve optimized the technology with high triglycerides. enough to deliver, is in pulmonary diseases, William Blair analyst Myles Minter point- where we’re actually delivering our drugs by ed out in a 22 January note on the AKCEA- aerosol delivery to the lung and we’re mov- APOCIII-LRx top-line data that the candi- ing into cystic fibrosis, idiopathic pulmonary LET’S GET date is a follow-on to Waylivra for which the fibrosis, COPD, chronic bronchitis, severe FDA issued a complete response letter in asthma,” Monia said. “We haven’t proven it SOCIAL 2018. (Also see “Keeping Track: Busy August yet, but I’m hopeful toward the end of this @PharmaScrip Ends With Approval For Doravirine, CRLs For year we’ll have enough clinical data to con- Dasotraline And Waylivra “ - Pink Sheet, 3 Sep, clude that we’ve validated the approach.”

Scrip Awards Winner 2019 Best Partnership Alliance (sponsored by CMIC)

This multimillion pound strategic partnership brought together Cancer Research UK, the medical charity LifeArc and Ono Pharmaceutical Cancer Immunotherapy Alliance in a unique alliance that relies on the complementary expertise of each partner to progress Winner: Cancer Research UK, LifeArc and Ono research into new immuno-oncology drug targets. It provides a clear path for the development of drug Pharmaceutical Cancer Immunotherapy Alliance targets identiﬁ ed by the research community supported by investment from Ono Pharmaceutical Cancer Immunotherapy Alliance.

“We’re thrilled to receive this celebrated award for a truly unique and ambitious partnership between LifeArc, Ono Pharmaceutical and Cancer Research UK. A collaboration like ours, which draws together diﬀ erent areas of expertise and promotes team science, will take drug discovery to a diﬀ erent level, enabling promising new immunotherapies to reach people with cancer.” Dr Hamish Ryder, Director, Cancer Research UK’s Therapeutic Discovery Laboratories

JN3086 Scrip Awards 2019 Winner Advert A_2.indd 1 2020/01/28 17:03 scrip.pharmaintelligence.informa.com January 31, 2020 | Scrip | 15 APPROVALS

Horizon Sees Blockbuster Future For Tepezza After US Approval ANDREW MCCONAGHIE [email protected]

orizon Therapeutics PLC has per vial, or approximately $200,000 per in our evolution to an innovation-focused gained approval for its thyroid patient per year. This combined with a US biopharma company, developing new Heye disease treatment Tepezza market of 15,000 to 20,000 patients means medicines for debilitating diseases with (teprotumumab-trbw) from the US Food the company expects to hit peak sales in few or no treatment options.” and Drug Administration, six weeks excess of $1bn a year. The FDA approval was based on re- ahead of schedule. The drug is a targeted inhibitor of insu- sults from a Phase II study and a Phase The Dublin, Ireland-domiciled com- lin-like growth factor-1 receptor (IGF-1R), III confirmatory OPTIC study. The OPTIC pany has gained notoriety in recent years, administered to patients once every three study found that significantly more pa- thanks to its practice of buying up estab- weeks for a total of eight infusions. tients treated with Tepezza (82.9%) had lished drugs and hiking its prices, and was Analysts at Jefferies note that the FDA a meaningful improvement in proptosis among 22 companies ejected from US in- has granted Tepezza a broad label of (≥2mm) as compared with placebo pa- dustry association PhRMA in 2017 for not “treatment of thyroid eye disease” with- tients without deterioration in the fellow investing enough in innovative medicines. out any limitations or mention of active or eye at week 24. But since then, chairman, president inactive disease, and therefore conclude Additional secondary endpoints were and CEO Timothy Walbert has steered the that this should help it achieve rapid up- also met, including a change from base- company towards more novel medicines take. The analysts also believe the com- line of at least one grade in diplopia in development, and the approval of Tepe- pany has prepared the market well for 67.9% of patients receiving Tepezza com- zza, which had secured FDA breakthrough launch, establishing relations with leading pared with 28.6% of patients receiving therapy designation, is seen as a turning ophthalmologists and endocrinologists. placebo at week 24. point by the company’s leadership. “Today is a great day for people living Horizon must conduct a post-marketing Tepezza is the first FDA-approved treat- with thyroid eye disease, a rare, vision- safety study in a larger patient population ment for thyroid eye disease (TED), an threatening disease that previously had as was agreed at the Dermatologic and autoimmune disease associated with no FDA-approved treatment options,” said Ophthalmic Drugs Advisory Committee proptosis (eye bulging), diplopia (double Timothy Walbert. (DODAC) FDA Advisory Committee meet- vision), blurred vision, pain, inflammation Walbert also noted that the approval ing on 13 December. The study will also and facial disfigurement. was the company’s first biologic. Acknowl- evaluate retreatment rates relative to how The company has announced that Te- edging its former notoriety and efforts to long patients receive the medicine. pezza will be priced at around $14,900 change, he called the approval a “key step Published online 22 January 2020 AZ’s Lynparza Gets Prostate Cancer Priority Review KEVIN GROGAN [email protected]

straZeneca PLC’s hopes of having a fourth tumor type ap- The UK major and partner Merck & Co. Inc. have announced that proved for Lynparza have received a big boost with US a supplemental new drug application for Lynparza (olaparib) has Aregulators agreeing to a quicker evaluation of the firm’s been granted a priority review by the US Food and Drug Admin- PARP inhibitor for prostate cancer. stration for patients with metastatic castration-resistant prostate cancer (mCRPC). This will speed up the review period from the AZ and Clovis neck standard 10 months to six months and AstraZeneca noted that and neck in the Prescription Drug User Fee Act (PDUFA) date is set for the sec- prostate cancer ond quarter of 2020. The FDA’s decision is based on results from the Phase III PRO- found trial, which were presented during the European Society of Medical Oncology congress in Barcelona last year. They showed that Lynparza significantly reduced the risk of disease progression or death by 66% (7.4 months vs. 3.6 months) compared with Johnson & Johnson’s Zytiga (abiraterone) or Pfizer Inc./Astellas Pharma Inc.’s Xtandi (enzalutamide) in patients with BRCA1/2 or ATM-mutated mCRPC, the primary endpoint of the trial.

The trial also showed that Lynparza reduced the risk of disease Beating AstraZeneca and Merck to a first approval in CRPC progression or death by 51% (5.8 months vs 3.5 months) compared would help Clovis start to catch up on its big pharma rivals and with Zytiga and Xtandi – which are androgen deprivation thera- could lead to preferential use as clinicians gain familiarity with pies – in the overall trial population of patients with homologous PARP in this patient population. Rubraca is currently approved for recombination repair (HRR)-mutated mCRPC. AstraZeneca noted ovarian cancer but sales have been underwhelming in compari- that PROfound was the first positive Phase III trial testing a targeted son to Lynparza. treatment in biomarker-selected prostate cancer patients. At last week’s J.P. Morgan Healthcare conference in San Fran- If all goes smoothly at the FDA, AstraZeneca and Merck will be cisco, Clovis discussed its unaudited revenues for 2019 which re- entering a large market. Prostate is the second most common vealed full-year Rubraca sales of $142-$143m. The company was cancer in men, with an estimated 1.3 million new cases diagnosed positive about its performance in Europe, following successful worldwide in 2018. AstraZeneca stated that approximately 10- launches in the UK and Italy (with launches in France and Spain 20% of men with advanced prostate cancer will develop CRPC coming next month) but SVB Leerink analyst Andrew Berens is- within five years, and at least 84% of these will have metastases at sued a note on 14 January claiming that management’s com- the time of diagnosis. ments on Europe “suggest that US sales may be flat to down.” Lynparza has quickly become a key drug for AstraZeneca, with Lynparza and Rubraca are both ahead of the other PARPs in pros- sales in the first nine months of 2019 hitting $847m, up 93% on tate cancer. In October last year, the FDA granted breakthrough the like, year-earlier period. It is marketed for ovarian cancer and therapy designation to J&J’s Zejula (niraparib) in BRCA-mutated BRCA-mutated metastatic breast cancer, and at the end of 2019, mCRPC cancer patients after androgen receptor therapy and tax- the FDA approved the drug for use in a third tumor type, pan- ane chemotherapy on the back of positive Phase II data. GlaxoS- creatic cancer, less than two weeks after its advisory panel gave mithKline PLC acquired Zejula in its $5.2bn purchase of Tesaro Inc. a narrow nod to the new indication. (Also see “Lynparza Gets US last year; the drug is approved for ovarian cancer but J&J holds the OK For Pancreatic Cancer “ - Scrip, 30 Dec, 2019.) (Also see “US FDA prostate cancer rights to the PARP. Approval Of Lynparza For Pancreatic Cancer Opens Door For PFS Endpoint In Disease” - Pink Sheet, 7 Jan, 2020.) ORPHAN DRUG STATUS FOR IMFINZI The Lynparza announcement was not the only piece of good news PRIORITY REVIEW FOR RUBRACA TOO for AstraZeneca’s oncology franchise on 20 January. The company The news will have been closely noted by Clovis Oncology Inc. es- noted that its PD-L1 inhibitor Imfinzi (durvalumab) and the anti- pecially since its PARP inhibitor Rubraca (rucaparib) was granted a CTLA4 antibody tremelimumab have been granted orphan drug priority review for mCRPC just last week (15 January). The PDUFA designations by the FDA for liver cancer. date for the US firm’s drug is 15 May, so the race to be the first ap- The agency has granted orphan drug status on the basis of proved targeted therapy for prostate cancer is set to be a close one. the ongoing Phase III HIMALAYA trial, testing Imfinzi as a mono- Clovis’s filing is based on the results from the TRITON trials therapy and in combination with tremelimumab in patients with program, in particular the TRITON2 study which showed a 44% unresectable, advanced hepatocellular carcinoma. Results from response rate with Rubraca in BRCA-mutated CRPC, and a 52% the study are scheduled for later this year. prostate-specific antigen (PSA) response. Published online 21 January 2020 Genentech’s SMA Type 1 Data Strengthen Case Backing Risdiplam Approval

JOSEPH HAAS [email protected]

ith top-line, pivotal data show- for at least five seconds, which the com- THE FOUR TYPES OF SMA ing efficacy in infants with pany called a medically meaningful motor ● Type 1 onsets from birth to six months Wtype 1 spinal muscular atrophy milestone improvement. The top-line data of age with a lifespan of two years; mor- (SMA), Genentech Inc. has bolstered the were from part two of the pivotal FIREFISH tality usually associated with pulmo- case for approval of risdiplam, a survival study; part one of that study investigat- nary complications motor neuron-2 (SMN-2) splicing modi- ing risdiplam’s safety and optimal dosing ● Type 2 occurs between the ages of fier under review at the US Food and Drug were included in the new drug application six and 18 months, with life expec- Administration with a 24 May action date. at the FDA, along with safety and efficacy tancy around 30 years and respira- On 22 January, the Roche affiliate re- data from the SUNFISH study in patients tory impairment ported that risdiplam demonstrated sta- with type 2 or type 3 SMA. (Also see “Keep- ● Type 3 occurs between ages two and tistical significance in infant patients with ing Track Of Thanksgiving Leftovers: Ris- 17, lifespan near-normal or normal but type 1 SMA aged one to seven months diplam, Artesunate Highlight Glut Of Sub- roughly 50% end up wheelchair-bound for the ability to sit up without support missions/Filings” - Pink Sheet, 5 Dec, 2019.) TURN TO PAGE 18

scrip.pharmaintelligence.informa.com January 31, 2020 | Scrip | 17 RESEARCH & DEVELOPMENT

CONTINUED FROM PAGE 17 due to preclinical findings of dorsal root In a 23 January note, Deutsche Bank ● Type 4 is adult onset after age 20, nor- ganglia (DRG) mononuclear cell inflam- analyst Richard Parkes said the FIREFISH mal life expectancy but progressive mation. (Also see “US FDA Puts IT Zolgens- part two data, including no reported muscle weakening ma Studies On Partial Clinical Hold” - Scrip, treatment-related safety findings, offers (Previously known as the CARE Awards) Roche/Genentech are seeking a broad la- 30 Oct, 2019.) further de-risking of risdiplam behind bel in SMA, which is characterized by four At the J.P. Morgan Healthcare Confer- the confirmatory data from SUNFISH. types of the disease with type 1 being the ence on 13 January, David Lennon, CEO “We continue to see sales of CHF2bn- earliest to onset and having the most se- of Novartis’s AveXis Inc. subsidiary, which 3bn [about $2.06bn-$3.1bn] as realistic vere mortality. (Also see “The SMA Market: developed the gene therapy, said the given its oral administration and likely Assessing The Unknowns” - In Vivo, 26 Nov, company is working to reply to the FDA’s approval in a broad age range of pa- 2019.) Although the data included in the safety concerns this quarter in hopes of tients, making it a major contributor to NDA do not include efficacy data for type getting the Phase II STRONG study under- Roche’s growth through near-term pres- Book your table 1 SMA, it is possible that given the unmet way again. sures,” he said. medical need in SMA, the FDA may ap- “The important thing is to recognize Because the SUNFISH and FIREFISH prove a broad label including type 1 pa- that was an important safety finding re- studies have enrolled patients up to 25 tients while awaiting confirmatory data. ported to the FDA. They wanted to un- years of age, approval of risdiplam likely Roche previously has said it anticipates derstand better what that safety finding would mean a much larger addressable two-year efficacy data from FIREFISH in was,” Lennon said. “What we saw was a patient base than applies to Zolgensma, Citeline late 2023. (Also see “Roche Makes Case minimal to marked inflammation and the analyst added. “If, as we suspect, For Its Oral SMA Drug Risdiplam As Filings degeneration of some dorsal root gan- detailed data confirm that risdiplam’s Beckon” - Scrip, 6 Feb, 2019.) glia,” which are sensory neurons that af- efficacy is competitive with Biogen’s In an email exchange with Scrip, the fect tactical function, usually to painful intrathecally administered Spinraza company indicated it continues to hope or hot/cold stimuli. and Novartis’ Zolgensma, we believe its for a fairly broad indication. “Th[e] body “Now, we haven’t seen any impact of advantageous oral administration and Awards 2020 of data submitted in the NDA includes re- that in the clinical trials overall,” he con- broad label could open up a very large sults from risdiplam treatment in people tinued, “and when you actually look at the opportunity,” Parkes said. living with SMA Types 1, 2 and 3,” it said. preclinical data that we had, while the in- Datamonitor Healthcare analyst So- “We are in ongoing discussions with the dividual cellular impact can be described phie Ng offered Scrip a similar assess- FDA, and sharing new data on risdiplam as as we said, it’s not every DRG that gets ment, saying “we believe that risdiplam it becomes available, including FIREFISH impacted. It’s not every cell in the DRG will have a large opportunity to shift Thursday, April 30, 2020 Part 2. … Our current focus is to closely that are impacted that show that degen- the market share in Roche’s favor once work with the FDA to explore broad ac- eration and there were no neurological it has gained approval, especially with Hyatt Regency Boston, Boston, MA cess to risdiplam for all individuals in the findings in the non-human primates that the completion of FIREFISH study rein- community who might benefit.” were studied.” forcing the positive safety profile of ris- Risdiplam would be the third drug to diplam,” as well as the demonstration of www.clinicalresearchexcellence.com reach market for SMA, following Biogen STILL ROOM FOR RISDIPLAM efficacy in type 1-3 patients. Inc.’s blockbuster Spinraza (nusinersen), While the market is gaining more options, Considering the data from Biogen’s approved by the FDA in 2016, and Novar- Genentech noted that a majority of SMA perspective, Credit Suisse analyst Evan tis AG’s gene therapy Zolgensma (onase- patients are untreated. Seigerman speculated that Roche might mnogene abeparvovec-xioi), approved The company thinks, however, that the price risdiplam lower than Spinraza to en- in May 2019. However, as an orally ad- systemic impact of an oral therapy may courage use, especially in type 2 and type SPONSORSHIP AND TABLE BOOKING ENQUIRIES: ministered liquid risdiplam would be the offer a broader benefit than the targeted 3 SMA patients. He wrote on 23 January Christopher Keeling first at-home administered option, which therapies from Biogen and Novartis. “In- that the FIREFISH part two data “further T: +44 (0) 20 3377 3183 would be given daily. (Also see “Rapid US creasing evidence suggests that the loss highlight the threat” to Spinraza from FDA Review For Roche’s SMA Contender” - of SMN protein may affect many tissues risdiplam. Spinraza has a first-to-market E: [email protected] Scrip, 25 Nov, 2019.) and cells throughout the body and that advantage and had been dosed in more Spinraza is administered three times SMA is a multisystem disorder,” Genentech than 9,300 patients as of the end of Sep- annually as an injection into the patient’s told Scrip. “Risdiplam distributes systemi- tember, he added. GENERAL ENQUIRIES: spinal cord, while Zolgensma, which cur- cally and preclinical data show that it in- Price does offer another avenue Jo Kirkpatrick rently is approved only for patients two- creases SMN protein in multiple tissues. of differentiation in SMA. Novartis years-old and younger (type 1), is an in- This ability to increase SMN protein in the grabbed headlines last May when Zol- T: +44 (0) 20 7017 7180 travenous therapy approved for one-time CNS and peripheral tissues in the body gensma became the world’s highest- E: [email protected] use. The company is working to expand is the key advantage of daily oral admin- price drug therapy, with a price tag use into the larger type 2 and 3 popula- istration. Risdiplam may therefore have above $2m. (Also see “It’s Official: No- tions with an intrathecal formulation of potential benefits beyond motor neurons vartis SMA Gene Therapy Zolgensma Is Zolgensma, but the FDA placed the pro- which could be important for achieving World’s Most Expensive Drug” - Scrip, 24 Sponsored by gram on a partial clinical hold in October optimal clinical outcomes.” TURN TO PAGE 20

JN2783 Citeline Awards 2020 Book your Table Advert A4.indd 1 2019/12/17 10:42 HEADLINE NEWS

(Previously known as the CARE Awards)

Book your table Citeline Awards 2020

Thursday, April 30, 2020 Hyatt Regency Boston, Boston, MA www.clinicalresearchexcellence.com

SPONSORSHIP AND TABLE BOOKING ENQUIRIES: Christopher Keeling T: +44 (0) 20 3377 3183 E: [email protected]

GENERAL ENQUIRIES: Jo Kirkpatrick T: +44 (0) 20 7017 7180 E: [email protected]