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BioCentury Extra
As published Friday, August 01, 2014 6:12 PM PST

  • L300M in new investments for Genomics England

    U.K. Prime Minister David Cameron announced over L300 million ($509.4 million) in new investments for Genomics England Ltd. (London, U.K.), which is sequencing and analyzing the genomes of 100,000 NHS patients by 2017. The investments include about L162 million ($275.1 million) from Illumina Inc. (NASDAQ:ILMN), the partner for the sequencing element of the project. In turn, Genomics England will pay Illumina about L78 million ($132.4 million) for its services.

    Other new investments include L27 million ($45.8 million) from the Wellcome Trust for a sequencing hub at its genome campus in Cambridge; L24 million ($40.7 million) from the Medical Research Council for data analysis and interpretation; and up to L20 million ($34 million) from NHS for the establishment of patient sequencing centers.

    The U.K.'s Department of Health launched Genomics England last year. The company is creating a database with genomic information for cancer patients and those with rare or infectious diseases. The data will be linked to patients' medical records. The project is in its pilot phase, with the main phase slated to begin next year.

    In December 2012, the U.K. allocated L100 million ($152.2 million) for the project (see BioCentury Extra, Dec. 11, 2012).

  • Biota's laninamivir octanoate misses in Phase II influenza

    Biota Pharmaceuticals Inc. (NASDAQ:BOTA) said laninamivir octanoate delivered via TwinCaps dry-powder inhaler missed the primary endpoint of improving median time to alleviation of influenza symptoms vs. placebo in the intent-to-treat (ITT) population of the Phase II IGLOO trial to treat influenza infection. The double-blind trial enrolled 248 patients with PCR-confirmed influenza A or B infection. Median time to alleviation of influenza symptoms was 102.3 hours for the 40 mg laninamivir octanoate cohort (p=0.248) and 103.2 hours in the 80 mg cohort (p=0.776) vs. 104.1 hours for placebo.

    Biota will report full data from IGLOO in September. The company said it does not plan to independently advance the development of laninamivir octanoate to treat influenza infection and will evaluate next steps for the program outside of Japan with partner Daiichi Sankyo Co. Ltd. (Tokyo:4568). Daiichi markets the second-generation long-acting neuraminidase inhibitor (LANI) in Japan as Inavir to prevent influenza A and B infections.

    In June, Biota said it will reduce headcount by 67% after HHS's Office of the Assistant Secretary for Preparedness and Response (ASPR) and Biomedical Advanced Research and Development Authority (BARDA) terminated a 2011 contract worth up to $231 million to develop laninamivir octanoate (see BioCentury Extra, May 8).

    Biota was off $0.79 (25%) to $2.42 on Friday.

  • NIH planning Phase I Ebola vaccine trial

    NIH plans to start a Phase I trial in September to evaluate an Ebola virus vaccine, with data expected by January 2015. The plans come in the wake of a worsening outbreak of Ebola in West Africa. According to the World Health Organization, at least 729 deaths this year have been attributed to the virus, including 57 deaths reported July 24-27. NIH said it hopes to give the vaccine to health workers in affected African countries "as early as 2015" if it proves safe and effective. The vaccine -- a non-replicating chimpanzee adenovirus vector containing two Ebola genes -- was designed by researchers at the Vaccine Research Center at NIH's National Institute of Allergy and Infectious Diseases.

    News of the increased severity of the outbreak sent the stock price of Tekmira Pharmaceuticals Corp. (TSX:TKM; NASDAQ:TKMR), which has an Ebola therapy in the clinic, up $1.45 (11%) to $14.26 on NASDAQ and up C$1.57 (11%) to C$15.55 in Toronto on Friday. Last month, FDA placed a full clinical hold on a Phase I trial evaluating the company's TKM-Ebola on safety concerns. The company is preparing its response to FDA and anticipates resolution by 4Q14. The product is a combination of short interfering RNAs (siRNAs) targeting Zaire Ebola L polymerase, Zaire Ebola membrane-associated protein (VP24) and Zaire Ebola polymerase complex protein (VP35) formulated with Tekmira's lipid nanoparticle (LNP) technology.

    Other companies developing therapies for Ebola were largely unchanged on Friday.

  • FDA approves Lumizyme for infantile Pompe's, says comparable to Myozyme

    FDA expanded the label of Lumizyme alglucosidase alfa from the Genzyme Corp. unit of Sanofi (Euronext:SAN; NYSE:SNY) to include treatment of all patients with Pompe's disease. The agency also lifted a REMS that ensured the drug was used only in its previously approved population -- patients ages 8 and up who do not have evidence of cardiac hypertrophy. The product is recombinant human acid alpha glucosidase enzyme (rhGAA).

    Additionally, FDA determined that Lumizyme and Myozyme -- alglucosidase alfa produced from the same cell line as Lumizyme at a different production scale -- are "chemically and biochemically comparable." Lumizyme is produced at Genzyme's 4,000L manufacturing plant, while Myozyme is produced at the 160L scale. The determination comes after the agency's 2008 decision that the larger-scale product would require a separate BLA for approval, citing differences in the carbohydrate structure of the molecules produced in the larger bioreactors (see BioCentury, April 28, 2008).

    In the U.S., Myozyme has been reserved for patients with infantile-onset disease or those 8 years or younger with late-onset disease. In the EU and elsewhere, regulators have not made a distinction between the two products, and the 4,000L product is marketed for both infantile and late-onset patients as Myozyme.

  • FDA approves Boehringer's Jardiance

    FDA approved Jardiance empagliflozin from Boehringer Ingelheim GmbH (Ingelheim, Germany) to treat Type II diabetes. The company said it plans to launch the sodium-glucose cotransporter 2 (SGLT2) inhibitor in the U.S. as soon as possible. Boehringer resubmitted an NDA for Jardiance in June after receiving a complete response letter for the product in March. According to Boehringer and partner Eli Lilly and Co. (NYSE:LLY), FDA had cited "previously observed deficiencies" at a manufacturing facility in the complete response. The European Commission approved Jardiance in May.

  • Kyowa submits application in Japan for antithrombin product

    Kyowa Hakko Kirin Co. Ltd. (Tokyo:4151) submitted an application to Japan's Ministry of Health, Labor and Welfare (MHLW) for KW-3357 to treat thrombophilia due to congenital antithrombin deficiency and disseminated intravascular coagulation accompanied by a reduction in antithrombin. This is the first submission for the recombinant antithrombin.

  • Goolsbee steps down as Sarepta chair

    Sarepta Therapeutics Inc. (NASDAQ:SRPT) said William Goolsbee stepped down as chairman but will remain a director. Director John Hodgman is serving as interim chair. Goolsbee has been a Sarepta director since October 2007 and became chair in June 2010. Hodgman -- EVP of finance and CFO of InterMune Inc. (NASDAQ:ITMN) -- has been on Sarepta's board since March 2004. Both Goolsbee and Hodgman are part of the board's audit and compensation committees. The only non-independent director on Sarepta's seven-member board is President and CEO Chris Garabedian.

    The chairman change comes about a week after Sarepta terminated Arthur Krieg as SVP and CSO. Earlier this week, the Wall Street Journal also reported that the company had moved to limit Garabedian's role in discussions with FDA for eteplirsen (AVI-4658). Sarepta plans to submit an NDA to FDA seeking accelerated approval of the compound to treat Duchenne muscular dystrophy (DMD) by year end (see BioCentury Extra, July 24).

    Sarepta said its board "fully supports" Garabedian and said additional information will come during a conference call to discuss its 2Q14 financial results, which are slated to be released on Aug. 7. The company was up $0.63 to $21.97 on Friday.

  • Loxo raises $68.4 million in IPO

    Loxo Oncology Inc. (NASDAQ:LOXO) raised $68.4 million through the sale of 5.3 million shares at $13 in a bumped-up IPO. The price values the company at $207.2 million. Last month, the company amended the offering to sell 4.4 million shares at $12-$14. At $13, the company would have raised $57 million and been valued at $195.8 million. Existing investor New Enterprise Associates purchased 230,769 shares for about $3 million in a concurrent private placement. Loxo's LOXO-101 is in Phase I testing to treat advanced solid tumors. Data are expected early next year. Loxo has exclusive, worldwide rights to the neurotrophic tyrosine kinase receptor 1 (NTRK1;TrkA) inhibitor from Array BioPharma Inc. (NASDAQ:ARRY). Cowen; Stifel; Oppenheimer; and JMP Securities are underwriters.

    Loxo was unchanged at $13 on its first day of trading Friday.

  • MacroCure raises $53.5 million in IPO

    Israeli biotech regenerative medicine company MacroCure Ltd. (NASDAQ:MCUR) raised $53.5 million through the sale of 5.4 million shares at $10 in an IPO underwritten by Credit Suisse; Jefferies; Nomura; and Oppenheimer. The IPO priced valued MacroCure at $161.8 million. Last month, the company said it planned to sell 5.4 million shares at $13-$15.

    MacroCure's CureXcell is in Phase III testing to treat diabetic foot ulcers and venous leg ulcers, with data slated for 2H15. The suspension containing activated allogeneic white blood cells isolated from the peripheral blood of healthy donors is already marketed in Israel.

    MacroCure was off $2.75 (28%) to $7.25 on its first day of trading Thursday. On Friday, the stock was up $0.24 to $7.49.

  • Gen9 raises $25 million in undisclosed venture round

    Synthetic biology company Gen9 Inc. (Cambridge, Mass.) raised $25 million from undisclosed investors in an undisclosed venture round. CEO Kevin Munnelly confirmed that the $25 million is in addition to a $21 million series C round raised from tool company Agilent Technologies Inc. (NYSE:A) in April 2013 (see BioCentury Extra, April, 24, 2013).

    Gen9's BioFab platform comprises gene synthesis technologies that the company said enables high throughput DNA synthesis at lower cost and higher accuracy compared to other methods. Gen9 synthesizes and markets gene fragments it calls GeneBits that are up to about 1,000 base pairs long. The company is also launching GeneBytes, or fragments that are 1,000-3,000 base pairs long.

  • Senators want HHS to release biosimilars guidance

    Five Republican senators sent a letter to HHS Secretary Sylvia Burwell asking the agency to release an FDA draft guidance on biosimilar naming that is awaiting HHS clearance before it can be released for stakeholder comment. In the letter, the senators -- Lamar Alexander (R-Tenn.), Richard Burr (R-N.C.), Mike Enzi (R-Wyo.), Orrin Hatch (R-Utah) and Pat Roberts (R-Kan.) -- also questioned whether FDA intends to approve the first biosimilar before policies on "key scientific questions" are released.

    FDA has previously said it plans to issue guidance this year on labeling for biosimilar products, as well as interchangeability. Late last month, the Sandoz unit of Novartis AG (NYSE:NVS; SIX:NOVN) said the agency accepted a BLA for a biosimilar version of Neupogen filgrastim G-CSF from Amgen Inc. (NASDAQ:AMGN) -- the first publicly disclosed FDA acceptance of a biosimilars application (see BioCentury, July 28).

    FDA directed questions to HHS, which could not be reached.

  • Brookings releases framework for compassionate use

    The Engelberg Center for Health Care Reform at the Brookings Institution released a paper that lays out principles for compassionate access to experimental medicines. The paper outlines an industry-wide structure addressing how patients, healthcare providers, and FDA should navigate the process.

    First, companies should anticipate the need for expanded access as part of their drug development plan, the paper recommends. It also stressed avenues for making drugs accessible to patients who qualify, and said companies need to be accountable for reviewing requests for expanded access in a timely manner. In addition, companies should release timely analysis of data from patients with expanded access as well as outcomes from expanded access.

    Brookings' Darshak Sanghavi discussed the role of social media to gain access to compassionate-use drugs during the April 13 edition of BioCentury This Week television, part of BioCentury's continuing coverage of the issue (see BioCentury This Week, April 13).

  • Pfizer and Roche join ADDPLAN DF consortium

    Pfizer Inc. (NYSE:PFE) and Roche (SIX:ROG; OTCQX:RHHBY) joined the ADDPLAN DF consortium, which is seeking to develop new statistical methods for designing dose-finding trials. Aptiv Solutions (Reston, Va.), Eli Lilly and Co. (NYSE:LLY), Johnson & Johnson (NYSE:JNJ), and Novartis AG (NYSE:NVS; SIX:NOVN) formed the consortium to develop new statistical methods for the design of dose-finding trials. In February, the group said it would start by developing an adaptive version of existing multiple comparison procedure modeling (MCP-Mod) software for optimizing dose selection for Phase III testing (see BioCentury, Feb. 24).

  • Campbell Alliance's Dealmakers' Intentions study available for download

    Campbell Alliance's Dealmakers' Intentions Study is the only forward-looking measure of dealmaking activity in the biopharma industry. Now in its sixth year, the study provides insight into what will likely drive the industry's partnering and M&A efforts moving forward -- the results of which were released at last month's BIO International Convention. Download a copy of the full white paper here.

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