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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.
Sanofi said it plans to submit regulatory applications for SAR302503 in the indication, but said it is "premature to comment" on a potential time frame. The pharma, which gained the selective oral Janus kinase-2 (JAK-2) inhibitor through its 2010 acquisition of TargeGen Inc., has an SPA from FDA for the JAKARTA trial.
Incyte Corp. (NASDAQ:INCY), which markets an oral JAK-1 and JAK-2 inhibitor to treat myelofibrosis, was down $0.13 to $23.04 on Friday. The company's ruxolitinib is approved in the U.S. as Jakafi and in the EU as Jakavi. Novartis AG (NYSE:NVS; SIX:NOVN) has exclusive ex-U.S. rights to the product from Incyte under a 2009 deal.
Separately, Sanofi said it will explore three options, including the establishment of local start-ups, to transform its research facility in Toulouse, France, over the next five years under a restructuring of its French operations announced last year (see BioCentury, April 29).
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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.
The company plans to complete the remaining two Phase III trials of N9-GP in the paradigm program in pediatric patients and in patients undergoing surgery within the next 12 months. Novo Nordisk plans to submit regulatory applications for the product in 2015.
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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.
Ocriplasmin is approved in the EU to treat vitreomacular traction, including when associated with a macular hole of 400 µm or less in diameter; and in the U.S. to treat symptomatic VMA, which causes vitreomacular traction. ThromboGenics, which launched the drug in the U.S. in mid-January, also reported U.S. sales of over $10 million through the end of April for Jetrea. Novartis launched ocriplasmin in the EU this quarter.
On Friday, ThromboGenics was off EUR 2.81 to EUR 34.35.
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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.
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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.
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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).
Boehringer and partner Eli Lilly and Co. (NYSE:LLY) do not market Trajenta in Germany.
Separately, G-BA issued a final benefit assessment saying that cancer drug Pixuvri pixantrone from Cell Therapeutics Inc. (NASDAQ:CTIC; Milan:CTIC) provides "no additional benefit" over patient-individualized care, G-BA's requested comparator, because the company did not submit suitable data. In March, Germany's Institute for Quality and Efficiency in Health Care (IQWiG) said in a preliminary benefit assessment that Pixuvri provides "no additional benefit" (see BioCentury Extra, March 1).
Drugs that do not have an additional benefit are added to the reference pricing system, which gives a similar base price to all comparable drugs. If there is no reference, the company will negotiate a price no higher than that of the comparator. Cell Therapeutics was up a penny to $1.16 on Friday.
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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.
Kamada develops and markets plasma-derived protein therapeutics, including Glassia, an IV formulation of alpha-1 antitrypsin (AAT) that partner Baxter International Inc. (NYSE:BAX) markets in the U.S. to treat AAT deficiency. The Tel Aviv Stock Exchange was closed Friday.
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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."
The Affordable Care Act specifies that 12 of IPAB's 15 members are to be appointed based on consultations with the Speaker of the U.S. House of Representatives and the minority leader, and the Senate majority and minority leaders. The remaining three members are appointed by the President. All members must be confirmed by the Senate.
Earlier this month, House Speaker John Boehner (R-Ohio) and Senate Minority Leader Mitch McConnell (R-Ky.) sent President Obama a letter declining to appoint IPAB members (see BioCentury Extra, May 10).
Starting in FY15, IPAB is required to submit a proposal to Congress in any fiscal year in which Medicare costs exceed a threshold established by the Affordable Care Act. If IPAB doesn't submit a proposal for any reason -- including failure of the Senate to confirm members -- the HHS Secretary must submit her own proposal, according to the legal analysis. The recommendations -- whether from IPAB or the HHS Secretary -- would automatically go into effect unless Congress enacted equivalent cuts.
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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.
The same group of representatives introduced a version of the bill during the last Congressional session, but that bill, H.R. 6272, was never voted out of the Energy and Commerce Committee's health subcommittee.
In February, GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) became the first pharma to publicly support the AllTrials campaign, which is calling for the disclosure of clinical trial results and clinical study reports -- which are formal reports that provide details on the design, methods and results of trials and may also form the basis of regulatory applications -- to increase trial transparency (see BioCentury This Week, April 14).