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BioCentury Extra
As published Friday, October 24, 2014 5:56 PM PST

  • Shire sets its peer group

    With a $1.6 billion breakup fee in hand from erstwhile merger partner AbbVie Inc. (NYSE:ABBV), Shire plc (LSE:SHP; NASDAQ:SHPG) on Friday reiterated its prospects as an independent company focused on rare and specialty diseases, comparing its business development strategy to the paths taken by Gilead Sciences Inc. (NASDAQ:GILD), Biogen Idec Inc. (NASDAQ:BIIB) and Celgene Corp. (NASDAQ:CELG).

    "We are confident in our strong prospects as a standalone company," CEO Flemming Ornskov said on the company's 3Q14 earnings call. He said the company believes it has "all the elements" required to deliver $10 billion in sales by 2020, of which $7 billion is slated to come from its current products and $3 billion from its pipeline.

    "Investors should hold us to the numbers," Ornskov told BioCentury. "The plan is aggressive but not unachievable."

    He told the conference call the numbers do not include two deals made in May, the $260 million acquisition of orphan disease company Lumena Pharmaceuticals Inc. and a $75 million buyout of renal specialist Fibrotech Therapeutics Pty. Ltd.

    Ornskov noted Gilead, Biogen and Celgene validated the strategy of specialization in targeted areas of unmet need, and said Shire was confident it, too, would achieve lasting scale as a "domain expert."

    He added: "We have all the necessary resources required to continue to pursue our business development strategy," including the addition of products and candidates.

    Shire set out its "10x20" revenue building plan in June as news surfaced about AbbVie's original unsolicited acquisition offers (see BioCentury, June 30).

    On Friday, the company raised its earnings guidance for 2014. Growth of non-GAAP earnings per ADS is now forecast in the high 30% range, up from the low- to mid-30s. The company reported non-GAAP operating income of $717 million ($2.93 per ADS) in 3Q14, up 60% over 3Q13. Product sales climbed 33% to $1.6 billion, led by 66% growth of its rare disease portfolio to $645 million. Shire bolstered that segment with the addition of complement 1 (C1) esterase inhibitor Cinryze to treat hereditary angioedema (HAE) via the acquisition of ViroPharma Inc. in January.

    On the call, Shire deferred questions about its pipeline plans to its R&D day on Dec. 10.

    Shire's ADSs gained $9.51 to $194.49 on NASDAQ. In London, the stock was up 155p to 4,050p.

  • Unlike FDA panel, CHMP backs AZ's olaparib

    EMA's CHMP recommended marketing authorization of Lynparza olaparib from AstraZeneca plc (LSE:AZN; NYSE:AZN) as maintenance treatment of platinum-sensitive relapsed ovarian, fallopian tube or peritoneal cancer in patients with germline breast cancer early onset (BRCA) mutations who are responding to platinum-based chemotherapy. If approved, the therapy would be the first poly(ADP-ribose) polymerase (PARP) inhibitor available in the indication.

    CHMP based its recommendation on the results of the Phase II Study 19, which showed that olaparib increased progression free survival (PFS) by 7.1 months compared to placebo.

    Looking at the same data in June, FDA's Oncologic Drugs Advisory Committee voted 11-2 that the data did not support accelerated approval in the same indication. Concerns about the validity and clinical significance of the PFS data led the panel to conclude it could not put the therapy's risks in context (see BioCentury, June 30).

    In July, the agency pushed back its PDUFA date to Jan. 3 from Oct. 3 to review additional trial data.

    In other PARP news, Myriad Genetics Inc. (NASDAQ:MYGN) Friday said it has established a European lab for its companion diagnostic Tumor BRACAnalysis CDx. The test identifies patients with BRAC1 and BRAC2 mutations who may benefit from treatment with PARP inhibitors such as Lynparza. Myriad was off $1.04 to $36.96 on Friday.

  • CHMP wants more warnings for Ariad's Iclusig

    EMA's CHMP recommended stronger warnings concerning risks of blood clots and arterial blockage with use of leukemia drug Iclusig ponatinib from Ariad Pharmaceuticals Inc. (NASDAQ:ARIA). CHMP's opinion endorses a similar recommendation from EMA's Pharmacovigilance Risk Assessment Committee (PRAC) earlier this month.

    CHMP said the risk is "likely dose-related," but stopped short of recommending lower doses due to insufficient data and a concern that efficacy would be lost.

    CHMP said Ariad would update product information to reflect risks, inform healthcare professionals about the effects and risks of dosage changes, and perform an additional study to clarify whether lower doses would still offer benefits with reduced risk of clots or blockages.

    The committee also recommended that treatment be stopped after three months if a complete response has not occurred.

    Last November, CHMP made several recommendations to minimize the risk of occlusive vascular events in patients using Iclusig, but did not recommend its removal from the market (see BioCentury Extra, Nov. 22, 2013).

    Iclusig, a pan-BCR-ABL tyrosine kinase inhibitor (TKI), is approved in the U.S. and EU to treat chronic myelogenous leukemia (CML) and acute lymphoblastic leukemia (ALL).

    Ariad relaunched Iclusig in the U.S. in January after FDA approved a REMS and revised label limiting the drug's use. The agency had asked the biotech to suspend marketing and distribution of Iclusig last October due to the risk of arterial thrombotic events.

    Ariad said it expects the European Commission's decision on Iclusig warnings in December.

  • CHMP recommends Clinuvel's Scenesse

    EMA's CHMP recommended marketing authorization under exceptional circumstances for Scenesse afamelanotide from Clinuvel Pharmaceuticals Ltd. (ASX:CUV; Xetra:UR9) to treat phototoxicity in patients with erythropoietic protoporphyria (EPP), a disorder characterized by extreme sensitivity to sunlight.

    Scenesse is the first compound in EMA's pilot project to involve patients in CHMP's assessment of benefits and risks. The committee said its recommendation was based in part on reports from patients that Scenesse improved their quality of life, although the compound's benefits were difficult to assess because placebo-controlled trials are difficult to conduct. There is no other approved treatment (see BioCentury Extra, Nov. 11, 2013).

    CHMP recommended approval on the condition Clinuvel puts in place a risk management plan that includes a patient registry.

    The committee extended its review of Scenesse to examine data from the U.S. Phase III trial CUV039, in which the compound missed its primary endpoint of increasing the median total duration of pain-free direct sunlight exposure over an eight-hour period. Scenesse, a sustained-release subcutaneous implant of synthetic alpha melanocyte stimulating hormone (MSH), has Orphan Drug designation for EPP in the U.S., EU, Australia and Switzerland.

    CHMP said marketing authorization will include a pharmacovigilance plan.

    Clinuvel trading was halted Friday in Australia in advance of the announcement. It was up A$0.30 (14%) to A$2.62 on Thursday.

  • Baxter's Rixubis gets CHMP nod for hemophilia B

    EMA's CHMP recommended marketing authorization of Rixubis (BAX 326) from Baxter International Inc. (NYSE:BAX) to treat and prevent bleeding in patients with hemophilia B. Approval will include a pharmacovigilance plan.

    FDA approved the recombinant Factor IX (rFIX) protein last year for use in adults with hemophilia B. Last month, FDA approved an sBLA for Rixubis to control and prevent bleeding episodes, for perioperative management and for routine prophylactic use in children with hemophilia B.

  • Venture Tracks

    PureTech Ventures (Boston, Mass.) named Robert Horvitz, Joi Ito and Raju Kucherlapati as senior partners.

    Horvitz, a Nobel Laureate, is a professor at Massachusetts Institute of Technology and a co-founder of Epizyme Inc. (NASDAQ:EPZM) and Idun Pharmaceuticals Inc., which now is part of Pfizer Inc. (NYSE:PFE).

    Ito is director of the MIT Media Lab and was an early investor in technology startups including Twitter Inc. and Kickstarter.

    Kucherlapati co-founded Millennium Pharmaceuticals Inc., now a subsidiary of Takeda Pharmaceutical Co. Ltd. (Tokyo:4502), and Abgenix Inc., which Amgen Inc. (NASDAQ:AMGN) acquired in 2006. He also is a professor at Harvard Medical School and a member of President Obama's Commission for the Study of Bioethical Issues.

  • Coherus sets IPO terms

    Biosimilars developer Coherus Biosciences Inc. (Redwood City, Calif.) set terms for its IPO on NASDAQ and plans to sell 6.3 million shares at $12-$15. At $13.50 Coherus would raise $85 million and be valued at $432.7 million. JPMorgan; Credit Suisse; and Cowen are underwriters.

    Coherus' pipeline includes versions of anti-tumor necrosis factor (TNF) drugs Enbrel etanercept from Amgen Inc. (NASDAQ:AMGN) and Humira adalimumab from AbbVie Inc. (NYSE:ABBV).

  • Angion withdraws IPO

    Angion Biomedica Corp. (Uniondale, N.Y.) withdrew its proposed IPO on NASDAQ, citing market conditions. In June, the company amended its IPO to sell 2.7 million shares at $10-$12. At $11, the company would have raised $30 million and been valued at $73.1 million. In April, Angion filed to raise up to $34.5 million. Aegis Capital was the sole underwriter.

    Angion in August said it would use the proceeds to continue development of BB3, a hepatocyte growth factor/scatter factor (HGF/SF) mimetic. It planned to start a Phase III trial in renal transplantation by 3Q15, resume a Phase II trial in acute myocardial infarction (MI) that had been halted, and start a Phase II trial in acute kidney injury (AKI) by 2Q15. The company did not respond to inquiries about its plans now that the IPO has been withdrawn.

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