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BioCentury Extra
As published Friday, March 27, 2015 6:50 PM PST

  • BioMarin rises on takeout rumors

    Rare disease company BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) jumped $13.23 (11%) to $128.78 on Friday, ending the day with a market cap of $20.5 billion, amid rumors that Shire plc (LSE:SHP; NASDAQ:SHPG) has approached it with a takeout bid. Both companies declined to comment.

    Shire CEO Flemming Ornskov said last month that the company remains well-positioned to do additional M&A deals after its $5.2 billion acquisition of NPS Pharmaceuticals Inc. (see BioCentury Extra, Feb. 12).

    BioMarin plans to complete its submission of a rolling NDA to FDA by the end of April for drisapersen (PRO051) to treat Duchenne muscular dystrophy. The antisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene has breakthrough therapy and Fast Track designations in the U.S. and Orphan Drug designations in the U.S. and EU. BioMarin gained the candidate in its acquisition of Prosensa Holding N.V. that completed in February (see BioCentury Extra, Dec. 8, 2014).

    BioMarin reported revenues of $751 million in 2014, including $334.4 million in sales of Naglazyme, a recombinant human N-acetylgalactosamine-4-sulfatase approved to treat mucopolysaccharidosis VI (MPS VI).

    In 2014, Shire acquired ViroPharma Inc., another rare disease company, for about $4.2 billion (BioCentury Nov. 18, 2013).

  • FDA denies Amgen's biosimilar Citizen's Petition

    FDA denied a Citizen's Petition from Amgen Inc. (NASDAQ:AMGN) asking the agency to require that biosimilar applications include a certification that the applicant will comply with the patent resolution protocol outlined in the Biologics Price Competition and Innovation Act of 2009 (BPCIA). Amgen submitted the petition in October after it filed a lawsuit against the Sandoz unit of Novartis AG (NYSE:NVS; SIX:NOVN) to prevent the launch of Zarxio filgrastim-sndz, a biosimilar version of Amgen's Neupogen filgrastim G-CSF (see BioCentury Extra, Nov. 3, 2014).

    Janet Woodcock, director of FDA's Center for Drug Evalation and Research, wrote in a letter to Amgen's attorneys that the BPCIA patent resolution protocol is "parallel to, but separate from, the FDA review process" and noted that the BPCIA does not require FDA "to impose a certification requirement as part of the biosimilar review process." The agency said interpretation of the BPCIA may be clarified by ongoing litigation.

    Last week, the U.S. District Court for the Northern District of California denied Amgen's motions for a preliminary injunction and ruled that Sandoz did not violate the BPCIA when it refused to provide its BLA to Amgen. The court also dismissed Amgen's claim that Sandoz violated the BPCIA when it gave Amgen its 180-day notice of intent to market Zarxio in advance of FDA approving the drug (see BioCentury Extra, March 19).

    On Wednesday, Amgen filed an appeal to the U.S. Court of Appeals for the Federal Circuit (CAFC). Amgen and Sandoz have agreed to request an expedited review of the appeal and an oral argument date as early as June (see BioCentury Extra, March 25).

    FDA approved Zarxio earlier this month, making it the first approved biosimilar product in the U.S. Sandoz has agreed not to launch Zarxio in the U.S. until the earlier of May 11 or a ruling by the CAFC on Amgen's application for an injunction pending appeal (see BioCentury Extra, March 6).

  • CHMP backs Eisai's Lenvima

    EMA's CHMP recommended marketing authorization for Lenvima lenvatinib from Eisai Co. Ltd. (Tokyo:4523) to treat adults with progressive, radioiodine-refractory, locally advanced or metastatic differentiated thyroid carcinoma. EMA reviewed Lenvima under its accelerated assessment program; it has Orphan Drug designation in the EU and the U.S.

    FDA approved the inhibitor of multiple VEGF receptor tyrosine kinases in February. SFJ Pharmaceuticals Inc. (Pleasanton, Calif.), which funded Phase III trials of Lenvima in thyroid cancer, was eligible for a milestone payment upon its U.S. approval. Eisai declined to disclose whether SFJ is eligible for a payment upon Lenvima's European approval (see BioCentury Extra, Feb. 13).

    Japan's Ministry of Health, Labor and Welfare (MHLW) approved the drug on Thursday (see BioCentury Extra, March 26).

  • Management tracks

    Bionor Pharma ASA (OSE:BIONOR) announced a trio of hires. Soren Keller is joining as SVP, COO and interim CFO. Keller was director and general counsel to the management at Zealand Pharma A/S (CSE:ZEAL). Kamilla Rolsted is joining Bionor as SVP and chief strategy and business officer. Rolsted was director of strategy and planning at Zealand. Finally, Bionor hired Barbara Ruskin as SVP, general counsel and chief patent officer

  • Genfit sinks on Phase II NASH data

    Genfit S.A. (Euronext:GNFT) plunged EUR 22.54 (44%) to a six-month low of EUR 28.31 on Friday after its GFT505 missed the primary endpoint in the Phase IIb GOLDEN-505 trial to treat non-alcoholic steatohepatitis (NASH) (see BioCentury Extra, March 26).

    Genfit said GFT505 met the primary endpoint of disease resolution after the company reanalyzed its data to account for baseline disease severity and trial site heterogeneity. The company plans to start a Phase III trial of the dual peroxisome proliferation activated receptor (PPAR) alpha and delta agonist by YE15.

    Genfit attributed the Phase IIb miss to an unexpectedly high incidence of disease resolution in the trial's placebo arm among patients with mild disease (NAFLD Activity Score=3). The company said its upcoming Phase III trial will exclude patients with a baseline NAS of 3.

  • Northwest gains on GBM survival data

    Northwest Biotherapeutics Inc. (NASDAQ:NWBO) reported data from glioblastoma multiforme (GBM) patients who were ineligible for enrollment in the company's ongoing Phase III trial of DCVax-Brain (DCVax-L), and instead were treated in a separate study arm. In that "informational arm," DCVax-Brain (DCVax-L) led to a median overall survival of 18.3 months in 51 GBM patients whose tumors showed evidence of regrowth following six weeks of radiation and chemotherapy.

    Northwest spokesperson Jane Searle declined to say when the company expects data from its Phase III trial of the autologous dendritic cells treated ex vivo with glioblastoma tumor fragments. Searle said data from the informational arm cannot be included in regulatory submissions.

    Northwest reported no serious adverse events in the informational arm. The company presented the findings at the Immunotherapy of Cancer Conference in Munich.

    Northwest added $0.38 to $7.67 on Friday.

  • Modest biotech rally ends bumpy week

    Biotech stocks recovered some of the week's losses Friday, ending a turbulent week that included a three-day sell-off in which some investors feared companies in the sector were overvalued (see BioCentury Extra, March 25).

    The BioCentury 100 gained 149.21 (2.1%) to 7,101.60 on Friday, with gainers outnumbering decliners 85 to 14 to finish the week off 5.6%. The index, which was essentially flat on Thursday, had lost 7.6% over the three days ending Wednesday.

    The BioCentury 100 is up 18% year-to-date, while the NASDAQ is up 3% and the DJIA is off 1%

  • PolyPid withdraws IPO

    PolyPid Ltd. (Petach Tikva, Israel) withdrew its proposed IPO on NASDAQ. CBO Asaf Bar said the company is confident it could have gone public under current market conditions, but anticipates undisclosed milestones in the next few months that could boost its valuation.

    In January, PolyPid said it was seeking to sell 1.8 million shares at $10-$12 in an offering underwritten by Aegis Capital; MLV; and Chardan Capital Markets. At $11, it would have raised $20 million and been valued at $106.3 million.

    PolyPid's lead candidate is BonyPid, a biodegradable bone filler combined with an antibiotic release platform designed to treat long-bone fractures and prevent bone infection. The company plans to start a confirmatory trial of BonyPid-1000 and a pilot program for dental counterpart BonyPid-500 in Europe in 2H15.

  • White House unveils antibiotics plan

    The White House outlined its five-year National Action Plan for Combating Antibiotic Resistant Bacteria, which includes measures to slow the emergence and spread of antibiotic resistant bacteria and advance the development of antibiotic drugs, vaccines and diagnostics. NIH, the Biomedical Advanced Research and Development Authority (BARDA) and the U.S. Department of Defense will allocate funding to projects aimed at developing rapid diagnostics and antibiotic drug candidates.

    Over the next five years, NIH and BARDA will establish a consortium of academic and industry partners that will aim to identify at least five targets for novel therapeutics, generate at least three preclinical antibiotic drug candidates and move at least two candidates through IND submission. The White House's timeline also calls for one NIH- or BARDA-funded diagnostic to be submitted to FDA for approval or clearance by 2020, and for government-funded genomic data, proteomic data and other datasets related to antibiotic resistance to be made publicly available in a way that preserves privacy.

    A government task force established by President Obama last September will monitor implementation of the plan. The Infectious Diseases Society of America (IDSA) said it welcomed the release of the National Action Plan and pushed for "new economic and regulatory incentives to stimulate the development of new antimicrobial drugs, diagnostics and vaccines."

    The President's Council of Advisors on Science and Technology (PCAST) held a meeting Friday focused on combating antibiotic resistance. PCAST issued a report last year recommending steps to improve surveillance of antibiotic resistance and development of new antibiotics, and optimize use of existing antibiotics (see BioCentury Extra, Sept. 18, 2014).

    In January, the U.S. House Energy & Commerce Committee issued a discussion draft of 21st Century Cures legislation that incorporates provisions of the Antibiotic Development to Advance Patient Treatment (ADAPT) Act, which would give FDA authority to approve antibiotics and antifungals for limited populations based on a smaller number of patients or alternative endpoints, as well as data from Phase II trials or pharmacologic or pathophysiologic data (see BioCentury Extra, Jan. 27).

    The White House included in its FY16 budget proposal earlier this year a request to nearly double spending on combating and preventing antibiotic resistance. The $1.2 billion request would include funding increases for NIH, FDA and BARDA to promote antibiotic drug and diagnostic development (see BioCentury Extra, Jan. 27).

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