Sanofi's SAR302503 meets in myelofibrosis
Sanofi (Euronext:SAN; NYSE:SNY) said once-daily 400 and 500 mg doses of oral SAR302503 each met the primary endpoint in the Phase III JAKARTA trial to treat primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF) and post-essential thrombocythemia myelofibrosis (PET-MF). Specifically, both doses improved the proportion of patients with a 35% or greater reduction in spleen volume from baseline to week 24 vs. placebo. The double-blind, international trial enrolled 289 patients with intermediate-2 or high-risk PMF, PPV-MF or PET-MF.
Novo reports Phase III data for hemophilia B compound
Novo Nordisk A/S (CSE:NVO; NYSE:NVO) reported data on Friday from the 74-patient Phase III paradigm 2 trial evaluating N9-GP -- a glyco-pegylated derivative of recombinant human Factor IX -- to treat and prevent bleeding episodes in patients with hemophilia B. Once-weekly 10 and 40 U/kg doses of N9-GP as prophylaxis for 12 months reduced median annualized bleeding rates compared to on-demand treatment with N9-GP for six months (2.9 and 1 episode per year, respectively, vs. 15.6 episodes per year). According to clinicaltrials.gov, the primary endpoint of the trial was incidence of inhibitory antibodies against Factor IX. Novo Nordisk declined to disclose details.
ThromboGenics' ocriplasmin misses in wet AMD trial
ThromboGenics N.V. (Euronext:THR) disclosed in a business update for the period ending April 30 that ocriplasmin missed the primary endpoint in the Phase IIa MIVI-5 trial to treat focal vitreomacular adhesion (VMA) associated with wet age-related macular degeneration (AMD). Specifically, a single injection of ocriplasmin plus anti-VEGF treatment missed the primary endpoint of a greater proportion of patients achieving complete resolution of their focal VMA at day 28 vs. sham control plus anti-VEGF treatment (24% vs. 12%, p=0.26). ThromboGenics said it is discussing future development plans for ocriplasmin in the indication with the Alcon Inc. ophthalmic unit of Novartis AG (NYSE:NVS; SIX:NOVN), which has ex-U.S. rights to commercialize the recombinant microplasmin, a truncated form of the natural human protein plasmin.
Bayer submits Japanese application for riociguat
Bayer AG (Xetra:BAYN) submitted a regulatory application in Japan for riociguat to treat chronic thromboembolic pulmonary hypertension (CTEPH). The oral soluble guanylyl cyclase (sGC) stimulator is under Priority Review in the U.S. to treat pulmonary arterial hypertension (PAH) and inoperable CTEPH or persistent/recurrent CTEPH after pulmonary endarterectomy (PEA), a surgical treatment. Bayer submitted the NDA in February. An eight-month Priority Review would place the PDUFA date in October; the specific date is not disclosed.
TaiGen submits nemonoxacin applications in Taiwan, China
TaiGen Biotechnology Co. Ltd. (Taipei, Taiwan) submitted a pair of NDAs in Taiwan and China for oral nemonoxacin to treat community-acquired pneumonia (CAP). The company, which said these are the first applications submitted worldwide, expects a decision in 1H14. An IV formulation of nemonoxacin is in Phase II testing to treat moderate to severe CAP, while TaiGen also has completed a Phase II trial of the oral formulation in diabetic foot infections. The company has exclusive, worldwide rights to the non-fluorinated quinolone antibiotic topoisomerase inhibitor from Warner Chilcott plc (NASDAQ:WCRX) under a 2011 deal.
G-BA rebuffs Pixuvri, Trajenta for second indication
Germany's Federal Joint Committee (G-BA) issued a final benefit assessment rebuffing diabetes drug Trajenta linagliptin from Boehringer Ingelheim GmbH (Ingelheim, Germany) as add-on therapy to insulin with or without metformin to treat Type II diabetes -- an indication approved by the European Commission last year. G-BA said the xanthine-based dipeptidyl peptidase-4 (DPP-4) as add-on therapy to insulin provides "no additional benefit" over human insulin and metformin, G-BA's requested comparator, because the company did not submit "necessary evidence." The decision is in line with a preliminary assessment G-BA issued in March (see BioCentury, March 18).
Kamada amends NASDAQ offering
Kamada Ltd. (Tel Aviv:KMDA) said it now plans to raise about $60 million through the sale and listing of 5.6 million shares on NASDAQ in a follow-on. The company also added RBC Capital Markets; Oppenheimer; and Chardan Capital Markets as underwriters. Morgan Stanley and Jefferies are lead underwriters. Kamada filed the offering, which it expects to price the week of May 27, in April.
POLITICS & POLICY
Sen. Coburn warns of 'IPAB-of-One'
A legal analysis released Thursday by Sen. Tom Coburn (R-Okla.) indicates that if members of Congress fail to confirm members of a controversial Medicare cost-containment board, HHS Secretary Kathleen Sebelius would be empowered to formulate and implement Medicare cuts. Coburn, a strong opponent of the Independent Payment Advisory Board (IPAB) and the Affordable Care Act that created it, said that under this scenario Sebelius would become an "IPAB-of-One."
Democratic Reps. reintroduce clinical transparency bill
A group of Democrats in the U.S. House of Representatives reintroduced a bill, H.R. 2031, that would impose stronger reporting requirements on the U.S. clinical trial registry data bank, clinicaltrials.gov. The Trial and Experimental Studies Transparency (TEST) Act would require all human trials to be registered before enrollment of the first subject and also would require publication of the trial protocol prior to the trial start, as well as the publication on clinicaltrials.gov within one year of trial completion. It also would require all foreign trials to meet the same registration and reporting requirements as U.S. trials if they are used to support a marketing application in the U.S.