Aurigene licenses small molecules to Agios | BioMarin's Batten disease drug wins FDA nod | Silvergate gets FDA nod for pediatric pJIA, ALL drug
April 28, 2017
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Aurigene licenses small molecules to Agios
Agios Pharmaceuticals has been granted exclusive global rights to Aurigene Discovery Technologies' portfolio of small molecules targeting an undisclosed cancer metabolism. Under the terms of the deal, Aurigene gets $3 million upfront and is entitled to milestone payments of $17 million per licensed product, plus royalties, while Agios will fund development, regulatory and marketing activities.
BioCentury (4/28) 
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Health Care & Policy
BioMarin's Batten disease drug wins FDA nod
BioMarin Pharmaceutical has received FDA approval for its Brineura, or cerliponase alfa, as a treatment for symptomatic pediatric patients with late infantile neuronal ceroid lipofuscinosis type 2, a type of Batten disease that is also called tripeptidyl peptidase 1 deficiency. The drug is expected to gain marketing approval for Europe in June.
Seeking Alpha (free registration) (4/27) 
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Silvergate gets FDA nod for pediatric pJIA, ALL drug
The FDA has approved Silvergate's Xatmep, or methotrexate, oral solution indicated for treatment of acute lymphoblastic leukemia as part of multiphase combination chemotherapy maintenance in pediatric patients. Xatmep is also approved for the management of polyarticular juvenile idiopathic arthritis in patients who cannot use first-line therapies because they did not respond adequately to or cannot tolerate the treatment. (4/26) 
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Sanofi, Regeneron's BLA for Kevzara accepted for FDA review
The FDA has accepted for review Regeneron Pharmaceuticals and Sanofi's resubmitted biologics license application for Kevzara, or sarilumab, with an action date of May 22. A final decision on an EU marketing authorization application for the drug is expected from the European Commission in the coming months.
Reuters (4/28) 
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Flex's IND for ALS drug wins FDA nod
Flex Pharma's investigational new drug application for FLX-787 has received FDA approval for a midstage trial involving patients with amyotrophic lateral sclerosis. The company is also exploring use of the drug for treatment of the Charcot-Marie-Tooth group of neuropathies.
Seeking Alpha (free registration) (4/26) 
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EMA grants PRIME status to uniQure's hemophilia B drug
The European Medicines Agency has granted priority medicines designation to uniQure's AMT-060, an investigational gene therapy for patients with hemophilia B. The designation was based on early-stage trial data showing patients with severe disease were nearly cured of spontaneous bleeding episodes with up to a year of follow-up, and they experienced significantly increased levels of Factor IX and substantially reduced need for Factor IX replacement.
PharmaTimes (U.K.) (4/26) 
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Company & Financial News
Heavy hitters fund development of monoclonal antibody combo
The Bill & Melinda Gates Foundation, GV (formerly Google Ventures) and Alexandria Venture Investments led a Series D financing round that raised $45.5 million for Arsanis, which is moving an experimental treatment for Staphylococcus aureus pneumonia into Phase II testing. The drug, ASN100, combines two monoclonal antibodies, and the company said it will use the fresh funds to study other monoclonal antibodies for multi-drug resistant Escherichia coli and Klebsiella pneumoniae, Streptococcus pneumoniae and respiratory syncytial virus.
Xconomy (4/26) 
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Clovis, Myriad sign deal for Rubraca companion diagnostic
Clovis Oncology will partner with Myriad Genetics on a companion diagnostic test for germline BRCA mutations to fulfill a postapproval requirement for Rubraca, or rucaparib, with the FDA. The deal calls for Myriad to submit a supplementary premarket approval to the FDA under the existing PMA for BRACAnalysis CDx to add Rubraca.
Seeking Alpha (free registration) (4/27) 
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Food & Agriculture
Opinion: Biotech crops beneficial for environment, people
Biotech crops reduce the use of chemicals that permeate the soil and harm lungs, with a study in PLOS ONE showing the adoption of agricultural biotechnology cut pesticide use worldwide by 37% and led to a 22% rise in crop yields, writes former USDA Secretary Tom Vilsack. Eliminating the use of biotech crops, on the other hand, would reduce corn, soybean and cotton yields by 11.2%, 5.2% and 18.6%, respectively, according to a Purdue University study.
The Hill (4/27) 
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Industrial & Environmental
CVR's cost of RFS compliance credits negative in first quarter
A proposal by CVR Energy's Carl Icahn to shift the blending point of obligation from refiners to fuel terminals is partly responsible for a sharp drop in the cost of compliance credits required under the Renewable Fuel Standard. CVR Energy said biofuels compliance expenses for its refining unit were negative during the first quarter of 2017.
Reuters (4/27) 
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The news summaries appearing in BIO SmartBrief are based on original information from news organizations and are produced by SmartBrief, Inc., an independent e-mail newsletter publisher. The information is not compiled or summarized by BIO. Questions and comments should be directed to SmartBrief at
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