Researchers from the University Paris Descartes have discovered that erythrocyte precursors in bone marrow depended on serotonin signaling for their maturation into mature red blood cells. Read More
The FDA issued the draft guidance, "Rare Diseases: Natural History Studies for Drug Development," intended to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological products for rare diseases. Read More
Liquidia Technologies Inc., of Research Triangle Park, N.C., said it priced an underwritten public offering of 3 million shares at $11.50 each, for total gross proceeds of $34.5 million. The underwriters have been granted a 30-day option to purchase up to an additional 450,000 shares of common stock at the public offering price, less the underwriting discount and commissions. Read More
Alzprotect, of Lille, France, engaged in the development of drugs for the treatment of Alzheimer's disease (AD), said that Parexel Biotech, a new division of Parexel International Corp., of Waltham, Mass., will perform the clinical phase IIa development of AZP-2006 for the treatment of progressive supranuclear palsy, a rare degenerative disease of the brain. Read More
Innovation is on a roll with the sector establishing a record of 59 new molecular entities (NMEs) gaining U.S. approval last year, a 22 percent increase over the 46 medicines approved in 2017, and six more than the previous industry high-water mark for the number of NMEs approved in one year in the U.S. established back in 1996. In addition, for the first time ever, the FDA approved more orphan drugs than non-orphans and the number of big pharma approvals fell. Read More
LONDON – The industry must continue with contingency planning and preparations for a crash-out Brexit, according to an agreement on Friday to postpone the March 29 departure date in a bid to stop the U.K. leaving without a deal. Read More
Next-gen bispecific antibodies (BsAbs) are on the move. The blockbuster potential of the class has galvanized biopharma, with deals and assets racing to capture the pole position as the entire field gains speed. Read More
Sorrento Therapeutics Inc.'s majority owned subsidiary Scilex Pharmaceuticals Inc. will merge with Semnur Pharmaceuticals, creating a new company, Scilex Holding Co., focusing on non-opioid pain management. Scilex's lead product is Ztlido, a lidocaine topical system, and Semnur's lead compound is a non-opioid corticosteroid gel that's in phase III pivotal studies. Sorrento said the merger positions Scilex Holding to become "a global leader in non-opioid pain management." Read More
Another mid-stage trial failure for emricasan, the caspase inhibitor at the heart of a multimillion-dollar NASH deal between developer Conatus Pharmaceuticals Inc. and Novartis AG, appears to have shaken investor confidence in the program's future, sending Conatus shares (NASDAQ:CNAT) down 56 percent to $1.28 on Friday. A shortfall on ENCORE-NF's primary endpoint, improvement in a measure of liver scarring, is the second setback in the collaboration following the drug's failure to meet the primary endpoint of a separate portal hypertension study. Read More
Sherlock Biosciences has arrayed a team of all-star academics and innovators in pursuit of next-gen molecular diagnostics. Among its nine co-founders are leading researchers in CRISPR, synthetic biology and genomics pulled from top institutions such as Massachusetts Institute of Technology (MIT), the Broad Institute and the Wyss Institute at Harvard University. Read More
On the PDUFA date for Zynquista (sotagliflozin), the FDA delivered a complete response letter (CRL) to Lexicon Pharmaceuticals Inc. and partner Sanofi SA related to the NDA for the type 1 diabetes therapy, an oral dual inhibitor of sodium glucose transporter (SGLT) 1 and 2 that was previously known as LX-4211. Recro Pharma Inc. also found a CRL in its mailbox, related to the NDA for intravenous (I.V.) meloxicam for the management of moderate to severe pain. Read More
