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BioCentury Extra
As published Thursday, May 26, 2016 5:56 PM PST


  • Ionis hammered after GSK opts against TTR study

    Ionis Pharmaceuticals Inc. (NASDAQ:IONS) plummeted $13.90 (39%) to $21.36 on Thursday, shedding nearly $1.7 billion in market cap, after it said partner GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) will not start the Phase III CARDIO-TTR study of IONIS-TTRRx (GSK2998728) to treat transthyretin (TTR)-related amyloid cardiomyopathy.

    On a conference call Thursday, CEO Stanley Crooke said "less than a handful" of patients receiving IONIS-TTRRx in the separate Phase III NEURO-TTR trial to treat familial amyloid polyneuropathy (FAP) experienced a "serious" decline in platelet counts. Crooke also said an undisclosed number of patients receiving the company's volanesorsen (ISIS 304801) in a Phase III study to treat familial chylomicronemia syndrome experienced "severe" platelet declines after "relatively short dosing."

    FDA placed a clinical hold on the CARDIO-TTR study of IONIS-TTRRx last month. GSK has an exclusive option to license the transthyretin (TTR) antisense inhibitor under a 2010 deal (see BioCentury Extra, April 7).

    Crooke said the company was "confident" the platelet events did not signal a class effect of its second-generation antisense treatments, adding that there were "zero cases of meaningful platelet reductions" with any of Ionis' pipeline drugs beyond the IONIS-TTRRx and volanesorsen studies. Crooke declined to say if there were any patient deaths, and suggested higher doses or the presence of drug-independent anti-platelet antibodies may be linked to the events.

    Crooke said NEURO-TTR is continuing as planned, as is an ongoing Phase II, investigator-initiated study of IONIS-TTRRx to treat TTR-related amyloid cardiomyopathy. The company expects results from NEURO-TTR in 1H17, and from the Phase II investigator study in July. Crooke does not expect a clinical hold on the trial of volanesorsen, which is an apolipoprotein C-III (APOCIII; APOC3) antisense inhibitor.

    Alnylam Pharmaceutical Inc. (NASDAQ:ALNY) gained $7.02 (11%) to $70.57 on Thursday, adding about $600 million in market cap. Last month, VP of Clinical Research Jared Gollob told a conference call that Alnylam had observed "no clinically significant effects on platelet levels" with patisiran (ALN-TTR02), its own antisense therapy for FAP.

  • Flexion's Zilretta data sufficient for submission

    Flexion Therapeutics Inc. (NASDAQ:FLXN) gained $5.78 (50%) to $17.23 after it said FDA endorsed the company's plans to submit an NDA for osteoarthritis (OA) pain therapy Zilretta (FX006) based on existing data. In a letter to the company, Flexion said FDA wrote that data from Zilretta's registration program are "acceptable to support filing of an NDA submission." Flexion said a planned in-person pre-NDA meeting "has become unnecessary," and the company is on track to submit its NDA in 4Q16.

    Zilretta, a sustained-release intra-articular formulation of triamcinolone acetonide (TCA), has Fast Track designation to treat OA knee pain.

    In February, Flexion said the treatment met the primary endpoint of a Phase III trial to treat moderate to severe OA knee pain. Last September, Zilretta failed to significantly improve pain relief vs. placebo in a pivotal Phase IIb study, although a pre-specified analysis that accounted for patient dropouts showed a significant improvement (see BioCentury Extra, Feb. 16, 2016).

    Flexion shares remain well below their Sept. 8, 2015 close of $29, before it announced results of the Phase IIb study (see BioCentury Extra, Sept. 8, 2015).

  • Minerva gains on schizophrenia, depression data

    Minerva Neurosciences Inc. (NASDAQ:NERV) soared $8.26 (233%) to an all-time high close of $11.80 on Thursday after it said two candidates met the primary endpoints in Phase II studies to treat psychiatric disorders. The company's market cap rose to $329.7 million from $98.9 million at Wednesday's close.

    Minerva said MIN-101 significantly improved negative schizophrenia symptoms on a five-factor Positive and Negative Syndrome Scale (PANSS) model vs. placebo (p≤0.022 for a 32 mg dose and p≤0.003 for a 64 mg dose), meeting the primary endpoint of a 12-week, 244-patient Phase IIb study. Next quarter, the company expects to complete a 24-week extension study of the serotonin (5-HT2A) and sigma-2 receptor antagonist.

    Separately, Minerva said MIN-117 met the primary endpoint in a Phase IIa trial to treat major depressive disorder. A 2.5 mg dose of the candidate improved Montgomery-Asberg Depression Rating Scale (MADRS) scores vs. placebo by a magnitude of 0.33, while an 0.5 mg dose improved scores by a magnitude of 0.23. Minerva said the measurements were similar to marketed antidepressants' effects.

    CEO Remy Luthringer told BioCentury the 84-patient MIN-117 study was not powered to detect significant differences between the candidate and placebo, and said the company plans to meet with FDA to discuss a pivotal trial design. MIN-117 is an antagonist of serotonin (5-HT2A) and serotonin (5-HT) transporter (SLC6A4; SERT) receptors and an inhibitor of both serotonin and dopamine reuptake.

    Minerva has ex-Asia rights to both compounds from Mitsubishi Tanabe Pharma Corp. (Tokyo:4508).

  • Acucela's emixustat fails in AMD study

    Acucela Inc. (Tokyo:4589) dropped Y1,000 (17%) to Y4,790 after it said emixustat (formerly ACU-4429) missed the primary endpoint of the Phase IIb/III S.E.A.T.T.L.E. study to treat geographic atrophy (GA) in patients with age-related macular degeneration. Acucela said it is reviewing the data to determine next steps for the candidate.

    The company said none of the study's three emixustat doses significantly improved lesion growth rate over placebo in the 508-patient study. The treatment also failed to improve best corrected visual acuity from baseline over 24 months, a secondary endpoint.

    Acucela is developing the non-retinoid visual cycle isomerase inhibitor targeting retinal pigment epithelium-specific 65 kDa protein (RPE65) in partnership with Otsuka Pharmaceutical Co. Ltd. (Tokyo, Japan).

  • Reata surges after IPO

    Reata Pharmaceuticals Inc. (NASDAQ:RETA) gained $2.07 (19%) to $13.07 in its first day of trading Thursday after it raised $60.5 million through the sale of 5.5 million shares at $11 in an IPO underwritten by Citigroup, Cowen and Piper Jaffray. The IPO price valued Reata at $236.5 million.

    In January, Reata filed to raise up to $80 million. Earlier this month, it had hoped to sell 4 million shares at $14-$16. At $15, it would have raised $60 million and been valued at $300 million.

    Next half, Reata plans to begin enrolling patients in a Phase III trial of bardoxolone methyl (RTA 402) to treat pulmonary arterial hypertension associated with connective tissue disease (CTD-PAH). AbbVie Inc. (NYSE:ABBV) has rights to bardoxolone outside the U.S. and certain Asian markets.

    In 2012, Reata terminated a Phase III study of the therapy to treat chronic kidney disease (CKD) in Type II diabetics due to safety concerns. In registrational filings for the IPO, Reata said it has not seen any evidence of significant new safety concerns in more recent studies of the small molecule activator of nuclear factor (erythroid-derived 2)-like 2 (NFE2L2; NRF2) (see BioCentury, Oct. 29, 2012).

  • OvaScience sinks after follow-on

    OvaScience Inc. (NASDAQ:OVAS) fell $2.85 (29%) to $6.93 on Thursday after it raised $50.1 million through the sale of 7.2 million shares at $7 in a follow-on underwritten by Leerink Partners, Oppenheimer, H.C. Wainwright, Roth Capital Partners and JonesTrading Institutional Services. OvaScience proposed the offering after market hours Wednesday, when its shares closed at $9.78. The company markets the Augment in vitro fertilization product, and is developing the OvaPrime and OvaTure fertility products.

  • 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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