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BioCentury Extra
As published Wednesday, April 01, 2015 7:06 PM PST

  • Sarepta CEO resigns ahead of FDA meeting

    Sarepta Therapeutics Inc. (NASDAQ:SRPT) gained $1.01 to $14.29 on Wednesday on news that Christopher Garabedian resigned as president, CEO and director after a Tuesday meeting with the board. CMO Edward Kaye will become interim CEO. The company said it remains on track to meet with FDA this quarter and to submit an NDA in mid-2015 for eteplirsen (AVI-4658) to treat Duchenne muscular dystrophy (DMD).

    Kaye told a conference call he believes he has a "difference in style" compared to Garabedian, and said Sarepta plans to give FDA "everything they've asked for" to make a decision.

    Sarepta's shares plummeted in October after it said it would delay the eteplirsen NDA submission from YE14 to mid-2015. The company said FDA requested additional data following a pre-NDA meeting in September (see BioCentury Extra, Oct. 27, 2014).

    FDA subsequently issued a rare statement on a compound still in development, and maintained it had been consisent in the advice it had provided to Sarepta on the development and regulatory pathway for the phosphorodiamidate morpholino oligomer (PMO) targeting exon 51 (see BioCentury Extra, Oct. 31, 2014).

    On Wednesday's conference call, interim Chairman John Hodgman said the company expects Kaye will remain interim CEO through the NDA submission for eteplirsen, which has Orphan Drug designation in the U.S. and EU and Fast Track designation in the U.S. for DMD. Hodgman said the company will conduct "a complete search which will include [Kaye]" for a permanent replacement.

    Kaye told BioCentury he thinks the board selected him in part for his regulatory experience. He said he has had a "collegial" relationship with FDA. Kaye is a former group VP of clinical development at Genzyme Corp., where he worked on the development of rare disease drugs Myozyme/Lumizyme alglucosidase alfa and Fabrazyme agalsidase beta.

    John Porter, CEO of the not-for-profit advocacy group Parent Project Muscular Dystrophy, released a statement supporting Kaye's appointment. Kaye told BioCentury the work of advocacy groups has "opened up doors" for companies developing DMD therapies by giving FDA perspective on the risks patients are willing to take to gain access to new therapies.

  • Shire launches Natpara in the U.S.

    Shire plc (LSE:SHP; NASDAQ:SHPG) announced the U.S. launch of Natpara as an adjunct to calcium and vitamin D to control hypocalcemia in patients with hypoparathyroidism. The wholesale acquisition cost (WAC) of the recombinant human parathyroid hormone 1-84 (PTH) is $7,916.67 per 28-day package.

    The chronic therapy is the first hormone replacement approved for the indication (see BioCentury Extra, Jan. 23).

    Natpara's label carries a boxed warning for increased risk of osteosarcoma, and is available under a REMS. Shire said physicians and pharmacies must be certified to prescribe and dispense the drug. Shire spokesperson Scott Santiamo said Natpara is available through two specialty pharmacies, with the company in the process of contracting a third.

    Santiamo said Shire estimates about 50,000 people in the U.S. have hypoparathyroidism.

    Shire gained the Orphan drug this year in its $5.2 billion acquisition of NPS Pharmaceuticals Inc. An MAA for Natpara is under EMA review (see BioCentury Extra, Jan. 12).

    Shire lost $6.48 to $232.81 on NASDAQ and 105p to 5,260p in London on Wednesday.

  • Calico in-licenses cognitive decline assets from UCSF

    Calico LLC (South San Francisco, Calif.) received an exclusive license from the University of California San Francisco to technology covering modulators of the integrated stress response to treat cognitive decline.

    The parties said the integrated stress response can contribute to memory decline. The UCSF technology was developed in the lab of Peter Walter, professor of biochemistry and biophysics. Carmela Sidrauski, now a scientist at Calico, led the studies while she was at UCSF.

    Calico will be responsible for research, development and commercialization of the modulators. UCSF will receive an undisclosed upfront payment and is eligible for milestones and royalties.

    Last month, Calico partnered with the California Institute for Quantitative Biosciences (QB3) and the Broad Institute of MIT and Harvard to investigate aging and age-related diseases (see BioCentury Extra, March 24).

  • Dr. Reddy's buys UCB's India established products business

    Dr. Reddy's Laboratories Ltd. (NYSE:RDY) acquired the established dermatology, respiratory and pediatrics business in India of UCB Group (Euronext:UCB) for Rs8 billion ($128 million). Combined sales of drugs in the portfolio were Rs1.5 billion ($24 million) in 2014.

    UCB said the sale allows it to focus on its neurology portfolio in India.

    Dr. Reddy's said the deal also includes UCB's established products business in Nepal, Sri Lanka and the Maldives. UCB will transfer 350 employees to Dr. Reddy's, which expects the transaction close this quarter.

  • Management tracks

    Antibody-drug conjugate company ImmunoGen Inc. (NASDAQ:IMGN) hired Anna Berkenblit as VP and CMO. Berkenblit was SVP of clinical development at H3 Biomedicine Inc. (Cambridge, Mass.).

    Ear disease company Otonomy Inc. (NASDAQ:OTIC) named Dean Hakanson CMO. Hakanson was VP and head of health economics and outcomes research for U.S. medical and drug regulatory affairs at Novartis AG (NYSE:NVS; SIX:NOVN).

    Oncology company Immunocore Ltd. (Abingdon, U.K.) named Christina Coughlin as CMO. Coughlin most recently was executive director of clinical research in early oncology development at Novartis.

    Cancer and autoimmune play 4SC AG (Xetra:VSC) hired Susanne Danhauser-Riedl as CMO. Danhauser-Riedel was medical head of hematology and oncology in Germany at GlaxoSmithKline plc (LSE:GSK; NYSE:GSK).

    Spinal product developer NuVasive Inc. (NASDAQ:NUVA) named Greg Lucier as chairman and interim CEO, succeeding Alex Lukianov. NuVasive said Lukianov resigned as CEO and a director after an investigation revealed he had not complied with "expense reimbursement and personnel policies." Lucier has been a NuVasive board member since 2013; he was chairman and CEO of Life Technologies Corp., which was acquired by Thermo Fisher Scientific (NYSE:TMO).

    Celgene Corp. (NASDAQ:CELG) named Gerald Masoudi EVP, general counsel and corporate secretary, effective June 1. Masoudi will succeed Lawrence Stein, who is retiring. Masoudi is a partner and co-chair of the food and drug practice group at Covington & Burling LLP. He was chief counsel at FDA from 2007 to 2008.

  • Dyax market cap tops $3.5B on DX-2930 news

    Dyax Corp. (NASDAQ:DYAX) gained $9 (54%) to $25.75 on Wednesday following postmarket data Tuesday that showed DX-2930 significantly reduced hereditary angioedema (HAE) attacks in a Phase Ib trial and received Fast Track designation from FDA. Dyax ended Wednesday with a market cap of $3.5 billion.

    Dyax said Tuesday it plans to meet with FDA to discuss the "minimum pathway forward" for DX-2930, a human mAb against plasma kallikrein (see BioCentury Extra, March 31).

  • Index raises EUR 650M for growth fund

    Index Ventures raised EUR 650 million ($706.2 million) for a new fund, Index Ventures Growth III, according to a Wednesday SEC filing. Details were not disclosed. The firm invests in tech and life science companies.

  • Coherus raises $120M in follow-on

    Coherus BioSciences Inc. (NASDAQ:CHRS) raised $120 million through the sale of 4.1 million shares at $29 in a follow-on underwritten by JPMorgan; Credit Suisse; and Cowen. Coherus proposed the offering after market Monday, when its shares closed at $30.02. Coherus lost $3.30 (11%) to $27.28 on Wednesday.

    The company expects to spend about $85 million to fund development of its preclinical biosimilar candidates and $7 million to fund a Phase II study it began last month of INT-131, a non-thiazolidinedione (TZD) peroxisome proliferation activated receptor (PPAR) gamma selective modulator, to treat multiple sclerosis. Coherus acquired INT-131 when it bought InteKrin Therapeutics Inc. last year.

    Coherus' pipeline includes CHS-0214, a biosimilar of Enbrel etanercept from Amgen Inc. (NASDAQ:AMGN) and Pfizer Inc. (NYSE:PFE) that is in Phase III testing to treat rheumatoid arthritis (RA) and psoriasis; and CHS-1420, a biosimilar of Humira adalimumab from AbbVie Inc. (NYSE:ABBV) that is expected to enter Phase III testing this half to treat psoriasis. The company expects it can complete ongoing studies with its newly raised funds, existing cash and expected funding from license agreements, including a deal giving Baxter International Inc. (NYSE:BAX) and Daiichi Sankyo Co. Ltd. (Tokyo:4568) ex-U.S. rights to CHS-0214.

    Coherus raised $91.8 million, including the overallotment, in an IPO last November (see BioCentury Extra, Nov. 6, 2014).

  • FDA to continue approving opioids without abuse deterrence

    FDA released final guidance on development and labeling of abuse-deterrent opioids that clarifies how abuse deterrence claims will be reflected on a drug's label. The agency also reiterated that it will continue to approve opioids that do not include abuse-deterrent properties.

    Douglas Throckmorton, deputy director for regulatory programs at FDA's Center for Drug Evaluation and Research, said during a press conference Wednesday that the agency scrapped its proposed four-tier scheme for describing abuse-deterrent properties on drug labels because stakeholders found the tiers "confusing." Instead, the final guidance specifies that abuse-deterrent label claims should be supported by data from three categories of premarket studies: in vitro, pharmacokinetic and clinical abuse potential studies. Data from postmarket studies should be added to labels when they become available.

    Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, told BioCentury This Week television in November 2014 that FDA would not require that all new opioids incorporate abuse deterrence features and that it will not remove opioids that lack abuse deterrence features from the market soon because the technology is in its "infancy" (see BioCentury This Week, Nov. 9, 2014).

    FDA spokesperson Eric Pahon told BioCentury on Wednesday that a class-wide requirement "is not feasible or in the interests of public health," in part because some patients rely on non-abuse-deterrent formulations. Patients in hospice who have difficulty swallowing or are on a feeding tube need oral solutions or pills that can be crushed for immediate-release, he said.

    FDA's Patrick Raulerson, senior regulatory counsel for the Office of Regulatory Policy at CDER, told the press conference the agency intends to release guidance on generic versions of abuse-deterrent opioids. Pahon told BioCentury that abuse deterrence would be required for a generic version to be automatically substitutable for the branded drug.

    FDA released the final guidance, called "Guidance for Industry: Abuse-Deterrent Opioids - Evaluation and Labeling," ahead of the June 30 deadline imposed by Congress as part of the Consolidated and Further Continuing Appropriations Act, 2015 (see BioCentury Extra, Dec. 15, 2014).

    The agency published draft guidance in January 2013 (see BioCentury Extra, Jan. 9, 2013).

  • FDA announces PRO compendium

    At an FDA workshop to discuss clinical outcome assessments, the agency said it plans to publish an online compendium of clinically acceptable patient-reported outcomes (PROs). The compendium will include PROs used by companies to support a label claim for drugs approved in 2003 and beyond, as well as PROs that have gone through the FDA's qualification process.

    The agency said the compendium will help inform industry of available PROs that could be used in clinical trials, and that companies should then discuss the PRO choices with the agency before using the measures in new clinical trials.

    The announcement came at Wednesday's workshop to discuss the opportunities and challenges of clinical outcomes assessments as part of FDA's PDUFA V requirement to incorporate patient perspectives into the review process.

    At the meeting, industry, patient groups and academics voiced their support for the compendium and also discussed additional efforts the stakeholders could undertake to improve the development of PROs based on patient input earlier in the clinical trial process. Suggested efforts included a precompetitive public-private consortium to develop a framework for disease or symptom-specific PROs as well as guidance from FDA on what level of interaction industry can have with patient groups to develop PROs that wouldn't be mischaracterized as marketing of an unapproved drug.

    FDA said it will issue a notification of the compendium in the Federal Register by Sept. 30, the end of its FY15, and seek public comments on the draft. At the workshop, the agency also said it plans to conduct more than its previously planned 20 patient-focused drug development meetings before the end of FY17, but didn't disclose a specific number.

  • Japan opens early access pathway

    Japan began accepting NDAs under a new early access pathway on April 1, the first day of its fiscal year. Tatsuya Kondo, chief executive of Japan's Pharmaceuticals and Medical Devices Agency, likened it to FDA's breakthrough therapy designation and the EU's pilot project on adaptive licensing in a presentation at the BIO Asia conference in Tokyo on March 24.

    The aim of the pathway is to speed approval of Japan-discovered drugs, said Kondo. The program is called "Forerunner Review Assignment" in English or sakigake in Japanese, meaning pioneer or explorer.

    To qualify, therapies must show "prominent effectiveness," which PMDA defines as a radical improvement compared to existing therapies.

    As with FDA's breakthrough therapy designation, regulators would accept Phase I/II data and would allow a rolling review while Phase III trials are ongoing.

    The move is part of PMDA's five-year plan that includes shortening approval times, enhancing safety and globalization of Japan's pharmaceutical industry.

  • CDER seeking permanent OPQ director

    FDA's Center for Drug Evaluation and Research is seeking a permanent director for its Office of Pharmaceutical Quality. CDER's job description says the director "provides executive leadership and technical direction in all matters related to the regulation of pharmaceutical quality within CDER."

    CDER Director Janet Woodcock has been serving as OPQ's acting director since the office was created in January (see BioCentury Extra, Jan. 12).

    In a memo to CDER staff, Woodcock described OPQ as a "new super-office" that is "expected to provide better alignment among all drug quality functions in CDER." The job posting closes April 14.

  • Lowy becomes NCI's acting director

    As previously announced, Douglas Lowy on Wednesday became acting director of NIH's National Cancer Institute. Harold Varmus, who had held the position since 2010, stepped down March 31. Lowy was NCI's deputy director (see BioCentury Extra, March 4).

  • Raising orphans: How pharma can capture value while treating rare diseases

    In a challenging environment for pharmaceuticals, the orphan sector offers the prospect of strong growth and attractive profit margins--along with the opportunity to improve the lives of patients with often debilitating conditions. However, commercial success is not guaranteed and the regulatory and access bars are rising. L.E.K. Consulting looks at the changing dynamics of the orphan market and identifies winning strategies designed to capture value within this sector. Click here to download the whitepaper.

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