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BioCentury Extra
As published Friday, May 27, 2016 6:05 PM PST


  • FDA rejects AZ's ZS-9

    AstraZeneca plc (LSE:AZN; NYSE:AZN) received a complete response letter (CRL) from FDA for hyperkalemia treatment ZS-9 sodium zirconium cyclosilicate. AZ gained the candidate via its $2.7 billion acquisition of ZS Pharma Inc. in 4Q15 (see BioCentury Extra, Nov. 6, 2015).

    AZ said the CRL cited "observations arising from a pre-approval manufacturing inspection." It said FDA has "acknowledged receipt of recently-submitted data which it has yet to review," and noted that the CRL does not require additional clinical data. AZ spokesperson Michele Meixell told BioCentury that FDA identified undisclosed issues during a March inspection.

    When AZ acquired ZS Pharma, CEO Pascal Soriot predicted $1 billion in peak annual sales of ZS-9, an inorganic crystal form of zirconium silicate designed to trap potassium ions over the ions throughout the GI tract.

    Relypsa Inc. (NASDAQ:RLYP) gained $1.46 to $18.06 on Friday's news. The company markets hyperkalemia treatment Veltassa patiromer, a high-capacity oral potassium binder. FDA approved Veltassa last October with a black box warning that recommends against taking it within six hours of other oral medications (see BioCentury Extra, Oct. 22, 2015).

    On Wednesday, Relpysa submitted an sNDA to FDA requesting an updated label for Veltassa that would reflect results of Phase I drug-drug interaction studies, which showed absorption of 12 tested drugs were not affected when taken within three hours of Veltassa (see BioCentury Extra, Jan. 25).

  • FDA approves Zinbryta for MS

    FDA approved Zinbryta daclizumab from Biogen Inc. (NASDAQ:BIIB) and AbbVie Inc. (NASDAQ:ABBV) to treat relapsing forms of multiple sclerosis in adults. The drug is a humanized IgG1 mAb against IL-2 receptor alpha chain (CD25).

    Distribution of Zinbryta will be limited by a REMS, and its label will have a black box warning about risks of severe liver injury and immune disorders including non-infectious colitis, skin reactions and lymphadenopathy.

    Biogen spokesperson Lindsey Smith told BioCentury the treatment will be available next quarter, and will be priced "generally in line" with other disease-modifying MS therapies.

  • EC approves GSK's Strimvelis

    GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) said the European Commission approved gene therapy Strimvelis (GSK2696273) to treat patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) for whom no suitable HLA-matched related stem cell donor is available. GSK said the approval is the first granted to a corrective gene therapy for children.

    Strimvelis is a retroviral vector delivering ADA for ex vivo hematopoietic stem cell therapy. The EC based its approval on data from 18 patients, including 12 participants in a pivotal Phase I/II study. GSK said all 18 patients remain alive after an average follow-up period of seven years. GSK said ADA-SCID affects 15 patients annually in Europe (see BioCentury Extra, May 5, 2015).

    ADA-SCID patients will be able to receive Strimvelis at Milan's Ospedale San Raffaele. GSK did not respond to inquiries about Strimvelis' price or launch date.

    GSK has exclusive rights to Strimvelis from Fondazione Telethon (Milan, Italy) and Fondazione San Raffaele (Milan, Italy) under a 2010 deal.

  • CHMP recommends HCV combos, spurns Ninlaro

    EMA's CHMP recommended approval of HCV therapies from Gilead Sciences Inc. (NASDAQ:GILD) and Merck & Co Inc. (NYSE:MRK), but recommended against approving cancer compound Ninlaro ixazomib from Takeda Pharmaceutical Co. Ltd. (Tokyo:4502).

    CHMP backed approval of Epclusa sofosbuvir/velpatasvir, Gilead's pan-genotypic HCV treatment. The combination therapy is a fixed-dose combination of pan-genotypic HCV NS5A inhibitor velpatasvir (GS-5816) and nucleotide analog HCV NS5B polymerase inhibitor Sovaldi sofosbuvir. Gilead markets Sovaldi to treat HCV genotypes 1, 2, 3 and 4. The pan-genotypic combo is under FDA review, with a June 28 PDUFA date.

    The committee also recommended Merck's Zepatier grazoprevir/elbasvir to treat chronic HCV infection in adults. FDA has approved Zepatier to treat genotype 1 and 4 infection. It is a fixed-dose oral combination of the HCV NS3/4A protease complex inhibitor grazoprevir (MK-5172) and HCV NS5A protein inhibitor elbasvir (MK-8742).

    The CHMP recommended the European Commission refuse to approve Takeda's Ninlaro to treat multiple myeloma (MM), saying that Ninlaro's possible benefit to relapsed or refractory MM patients was insufficient to outweigh the drug's risks. The oral proteasome inhibitor is approved in the U.S. in combination with Revlimid lenalidomide from Celgene Corp. (NASDAQ:CELG) and dexamethasone to treat MM in patients who have received at least one prior therapy. Takeda said it would appeal CHMP's opinion.

  • 5AM closes $285M fund

    5AM Ventures disclosed to BioCentury the firm closed its 5AM Ventures V fund at $285 million. The firm will continue to focus on early stage companies developing therapeutics, tools and drug delivery technologies. 5AM now has about $1 billion under management.

  • Aclaris raises $20M in private placement

    Aclaris Therapeutics Inc. (NASDAQ:ACRS) raised $20 million through the sale of 1.1 million shares at $18.50 in a private placement led by new investor Aisling Capital. Undisclosed new and existing investors also participated. William Blair and Guggenheim Securities were the placement agents. The price is a 6% discount to Aclaris' close of $19.70 on Thursday, before the deal was announced. Aclaris gained $0.30 to $20 on Friday.

    Aclaris' share price has slipped since reaching an intraday high of $33.88 on Dec. 17, 2015. In October, the company priced its IPO at $11, raising $55 million (see BioCentury Extra, Oct. 7, 2015).

    In January, the company began Phase III testing of A-101, a high-concentration hydrogen peroxide topical solution to treat seborrheic keratosis, with an NDA submission slated for 4Q16. The company also intends to submit an IND next half for ATI-50001 and ATI-50002, which are oral and topical formulations of a selective Janus kinase-1 (JAK-1) and JAK-3 inhibitor to treat alopecia. Aclaris obtained exclusive, worldwide rights to the JAK program in September from Rigel Pharmaceuticals Inc. (NASDAQ:RIGL) (see BioCentury Extra, Sept. 9, 2015).

  • Sanders, House Dems press USTR on Colombia license

    In separate letters to U.S. Trade Representative Michael Froman, a group of U.S. House Democrats and Sens. Bernie Sanders (I-Vt.) and Sherrod Brown (D-Ohio) expressed concern over press reports that U.S. officials had discouraged the Colombian government from issuing a compulsory license for Glivec imatinib from Novartis AG (NYSE:NVS; SIX:NOVN).

    In a letter dated Thursday, Sanders and Brown said a meeting with congressional staff left Colombian officials "under the impression" that the country would lose U.S. funding for its Paz Colombia initiative if it issued a compulsory license for Glivec, and said it would be "unconscionable" to threaten to remove the funding. The Senators called on Froman to clarify publicly that compulsory licenses are permitted under international trade agreements and "a legitimate means of promoting access to medicines."

    The Representatives' letter, dated Wednesday, says Glivec's annual price in Colombia is "almost twice the average annual income per person" in the country. The letter, signed by House Committee on Ways and Means Ranking Member Rep. Sander Levin (D-Mich.), also asserts that "we should not seek to limit the existing, agreed upon flexibilities public health authorities have to address" high drug prices.

    The office of the USTR said it is reviewing the Representatives' letter and does not yet have a position.

    Novartis markets imatinib in the U.S. as Gleevec.

  • EMA to review first-in-human guidelines

    EMA said it has begun a review of its guidelines for first-in-human clinical trials following a patient death and several hospitalizations that occurred in January during a French Phase I study (see BioCentury Extra, Jan. 15).

    One agency group is evaluating preclinical data that are required to begin human testing, while another will study the design of first-in-human trials. The agency plans to produce a concept paper by July that will identify areas for change to minimize risk in future studies. It intends to hold a public consultation on the paper this year.

    In March, the Temporary Specialist Scientific Committee (TSSC) of the French National Agency for Medicines and Health Products Safety (ANSM) recommended that a therapy show preclinical pharmacological activity that is "comparative whenever possible" and "reasonably predictive" of therapeutic efficacy before human testing begins, and that "more reasonable and careful practices" be established for dose escalation in Phase I trials (see BioCentury Extra, March 10).

  • NIH awards Mayo Clinic $142M for PMI biobank

    NIH will grant $142 million over five years to the Mayo Clinic to create a repository of biological samples for the White House's Precision Medicine Initiative. The project, launched in January 2015, is recruiting a cohort of 1 million volunteers for a longitudinal research study that aims to improve health and identify new disease treatments (see BioCentury Innovations, Feb. 12, 2015).

  • inVentiv Health Consulting Summit Highlights Key Challenges and Best Practices for Medical Affairs Leaders

    inVentiv Health Consulting (formerly Campbell Alliance) brought together biopharma Medical Affairs (MA) leaders to share ideas at the fifth annual Medical Affairs Leadership Summit, a signature event capping a year-long cycle of collecting and analyzing data about MA leaders' most pressing issues. Download your copy of the white paper now!


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