WEEK OF SEPTEMBER 9, 2013 BioCentury THE BERNSTEIN REPORT ON BIOBUSINESS Volume 21 • Number 34 • Page A1 of 22 Regulation Breakthrough encore By Steve Usdin BioCentury This Week Washington Editor Friends of Cancer Research, the Cover Story which are prescribed off label for the condi- advocacy organization that conceived of tion./A13 and persuaded Congress to enact FDA’s Breakthrough Encore — Friends of breakthrough therapies program, last week Cancer Research is trying to create a Regulation unveiled a proposal to expedite develop- breakthrough diagnostics program to ensure ment of companion diagnostics that are companion Dx are ready when breakthrough Better Decisions Post-Avandia — Three intended for use with a breakthrough therapies are approved. While FDA agrees in cardiologists have asked FDA to ease its CV drug. principle, it warns money could be an issue. requirements on diabetes drugs in the aftermath In contrast to the breakthrough thera- of Avandia. But the bigger question is how FDA pies program, which was incorporated Strategy will react to the next post-market crisis./A14 into the FDA Safety and Innovation Act, FOCR’s new proposal focuses on steps Setting Amgen’s Targets — Acquiring Onyx Finance the agency and diagnostics companies can will more than double the bellwether’s portfolio take to streamline development and re- of marketed targeted cancer therapies, and puts Amgen’s Poker Face — SEC filings show view within existing law. it into a tie with GSK for second place behind Amgen shaved more than $400 million off the Senior agency officials, including Jef- Roche/Genentech. But it has to execute./A5 sticker price for Onyx after Kyprolis failed to frey Shuren, director of the Center for meet the early stopping criteria in the Phase III Devices and Radiological Health (CDRH), Product Discovery & Development FOCUS trial during negotiations./A16 and Janet Woodcock, director of the Cen- Ebb & Flow — Argos takes E round in the ter for Drug Evaluation and Research Checkpoint Complementarity — hand vs. IPO in the bush. What BVF sees in (CDER), made it clear they support the Amplimmune adds a new set of immune Evotec’s business model. The Astex chronicles. proposal in principle at a Sept. 6 meeting checkpoint targets to MedImmune’s cancer Also: Ariad; Agenus; ArQule; Cytokinetics; sponsored by FOCR. portfolio, giving it more potential combina- Rockwell Medical; Transgene; Ironwood; Pozen; See next page tions to test in cancer./A8 Vivus, et al./A17 Viral Persistence — TCN-032 missed the primary endpoint in a Phase IIa influenza trial, Featured links this week/A12 but Theraclone says there’s enough evidence of Stock charts & tables/A22 a treatment effect to move forward with Phase SAVING DIAGNOSTICS II trials in hospitalized patients./A11 Company index/A10 Breakthroughs Needed TM Emerging Company Profile BioCentury 100 Indicators www.biocenturytv.com Week ended 9/6/13 Lasting Longer — Ixchelsis’ oxytocin recep- PRICES VOLUME Please See Program Notes on A2 tor antagonist could help treat premature ejacu- 4205.98 555.6M shrs lation more safely than antidepressant SSRIs, up 4% dn 14% NewsMakers in Sight Point/Counter-Point The 20th Annual NewsMakers in the Biotech Get Ready for Vienna Think tank experts draw a roadmap for Industry convenes in New York on Sept. 27. Online partnering for the 19th Annual BIO- epigenetics, and their peers respond at the Online registration is brisk. Don’t miss the Europe starts on Sept. 23. SciBX Summit on Innovation in Drug Discovery chance to see the NewsMakers Class of 2013. See announcement following A22. & Development. Early bird registration still open. Details follow A22. Details follow A22. BioCentury, THE BERNSTEIN REPORT ON BIOBUSINESS SEPTEMBER 9, 2013 PAGE A2 OF 22 Regulation, biopharma industry has been making in Woodcock added that she hopes to from previous page creating targeted therapies, according to see targeting in antibiotic drug develop- Woodcock (see BioCentury, June 24). ment based on improved diagnostic tech- Shuren suggested, however, that sup- At the same time, the rapid develop- nology. porting the breakthrough diagnostics pro- ment of diagnostics is a prerequisite to Despite this central role in targeted gram may require additional funding. speeding the deployment of these break- medicine, diagnostics were not explicitly CDRH wasn’t at the table when the through therapies, said Michael Pacanows- included in the breakthrough therapy leg- breakthrough therapies program was de- ki, associate director for genomics and islation. veloped, and the center has not received targeted therapy in the Office of Clinical “There is pressing need to clarify the any additional funds to help it speed the Pharmacology at CDER. pathways to make both [drugs and com- review of breakthrough companion diag- Indeed, about two-thirds of the appli- panion diagnostics] come to market in a nostics applications, he noted. cations FDA has accepted for its break- timely fashion,” Pacanowski told the meet- Shuren said the center has been able to through therapies program include a com- ing. handle the added workload, but “resources panion diagnostic. In the absence of a process for expedit- are a challenge.” In addition, he said Woodcock noted the use of targeting ing development of companion diagnos- budget sequestration “has thrown a ter- strategies to enhance efficacy in products tics, “in many cases the co-development of rible monkey wrench” into CDRH’s fi- that have breakthrough designations is a diagnostic may lag behind” development nances. not limited to cancer and monogenic he- of a breakthrough therapy, warned Howard All or most of the breakthrough diag- reditary diseases, but also include “other Scher, chief of the genitourinary oncology nostics proposals could be implemented diseases where the biomarkers are more service at Memorial Sloan-Kettering without legislation. Many of them echo fuzzy.” Cancer Center. steps CDRH has already implemented on See next page an informal basis and plans to codify in formal guidance documents, Shuren said. Rational exuberance Drug companies, patients and inves- tors have responded enthusiastically to The future of precision medicine requires innovative diagnostics. So the breakthrough therapies program: as of what’s holding them up? Aug. 23, CDER had received 82 requests for breakthrough designation. It had University researchers, diagnostics and insurance companies, and granted 25, and 25 were pending. patient groups point to a “vicious cycle” of inconsistent regulation, poor The program is intended to speed the reimbursement and under-investment in R&D. development of drugs when there is dra- matic clinical evidence of efficacy. The newest edition of BioCentury This Week television explores the The first approval of a breakthrough vicious cycle, and what must be done to break it. Featuring: drug is likely to come this fall, according to • Dr. Jeff Allen, Executive Director of Friends of Cancer Research, FDA officials. which spearheaded what is now FDA’s Breakthrough Therapies Marketing applications are pending for program and now is building the case for a breakthrough diagnostics at least three breakthrough therapies. pathway. Obinutuzumab to treat previously un- treated chronic lymphocytic leukemia • Kate Claessens, R.N., Senior Director of Health Policy and Reim- (CLL), from Roche and its Genentech bursement at Roche Diagnostics. unit, has a Dec. 20 PDUFA date. Ibrutinib from Pharmacyclics Inc. and Johnson • Dr. Robert McCormack, Head of Technology Innovation, Janssen & Johnson has a Feb. 28, 2014, PDUFA Diagnostics. date for previously treated mantle cell • Dr. Sheila Taube of ST-Consulting, former Associate Director of the lymphoma (MCL) and previously treated NCI Cancer Diagnosis Program. CLL/small lymphocytic lymphoma (SLL). The PDUFA date is not disclosed for Key opinion leaders; sophisticated questions serelaxin from Novartis AG to treat Always on BioCentury This Week television acute heart failure (AHF). In its January earnings announcement, the pharma said Watch Anytime on the Web it expected a U.S. submission in 2Q13 but it has not disclosed precisely when the www.biocenturytv.com application was submitted. FDA frequently beats PDUFA goals for New weekly shows go online at 9:00 a.m. EDT on Sundays. cancer drugs that, like obinutuzumab and Prior shows available 24/7 in the online Program Archive. ibrutinib, could provide dramatic improve- BioCentury This Week also is broadcast on Sunday in Washington, D.C. ments over standard care. Watch it on WUSA Channel 9 at 8:30 a.m. EDT The flood of breakthrough designa- tions is a reflection of the progress the BioCentury This Week is brought to you by BIO and Biogen Idec BioCentury, THE BERNSTEIN REPORT ON BIOBUSINESS SEPTEMBER 9, 2013 PAGE A3 OF 22 Regulation, can start offering a companion diagnostic from previous page “The set of proposals is as soon as it is approved, but it can take incredibly on point, incred- months for other labs to complete the FOCR’s proposal necessary training, establish standard op- ibly helpful; it echoes a lot erating procedures and verify test meth- In an interview on BioCentury This ods, said Christine Gathers, senior direc- Week television, FOCR Executive Direc- of things we have been tor for regulatory affairs for diagnostics at tor Jeff Allen said the group and its col- thinking about internally or Eli Lilly and Co. laborators has been working to identify According to FOCR, CDRH has au- “particular strategies for keeping the de- have implemented.” thority