Shares of Cambridge, Mass.-based Scholar Rock Holding Corp. closed Oct. 27 at $30.02, up $16.30, or 119%, on positive six-month interim analysis results from the Topaz phase II trial with inhibitor of myostatin activator SRK-015 in type 2 and type 3 spinal muscular atrophy, and CEO Tony Kingsley pointed to “a rich cascade of data ahead of us.”
Two complete response letters for group members dispatched this month by the FDA was enough to contribute to a flat performance in October for the BioWorld Neurological Diseases index. Overall, the price-weighted index has remained underwater for most of the year with its value down about 5% during this period.
The fatty acid amide hydrolase (FAAH) inhibitor space may seem, in the words of one analyst, “historically a graveyard,” but that didn’t keep Jazz Pharmaceuticals plc from buying Springworks Therapeutics Inc.’s program with a plan to take aim first at post-traumatic stress disorder (PTSD) and associated symptoms.
With a Nov. 6 FDA adcom meeting on Biogen Inc.'s Alzheimer's candidate, aducanumab, creeping ever closer, the candidate's prospects stole the show in its third-quarter earnings report, even outshining attention to the cloud of generics raining on the company's years-long Tecfidera (dimethyl fumarate) parade. FDA acceptance for aducanumab's BLA lines the candidate up for a priority review and regulatory action by March 7, the company said. Furthermore, global progress remains underway, with an EU marketing application now made and one in Japan on deck.
PERTH, Australia – Sydney-based Actinogen Medical Ltd. will advance its lead compound, Xanamem, into two proof-of-concept phase II studies in Alzheimer’s disease and fragile X syndrome.
Aptinyx Inc. CEO Norbert Riedel said the design is unclear of the pivotal trial due next year with the N-methyl-D-aspartate (NMDA) receptor modulator NYX-783 in post-traumatic stress disorder (PTSD), as fine points must be hashed out with the FDA.
Eli Lilly and Co.’s definitive agreement to acquire privately held Disarm Therapeutics Inc. brings Cambridge, Mass.-based Disarm $135 million up front and as much as $1.225 billion in additional future payments for potential development, regulatory and commercial milestones if Lilly successfully develops and commercializes therapies tied to the agreement.
CEO Christopher Missling said New York-based Anavex Life Sciences Corp. is still deciding on the dose for the pivotal study with Anavex2-73 (blarcamesine) in Parkinson’s disease dementia (PDD), and called the phase II findings “just the first data run,” about which the firm “wanted to immediately inform shareholders.”
Privately held Dyno Therapeutics Inc., an early stage biotech company applying artificial intelligence to gene therapy, entered a collaboration and license agreement with Spark Therapeutics Inc. that could bring Dyno milestone payments exceeding $1.8 billion.
Cyclerion Therapeutics Inc.’s phase II blowup with sickle cell disease (SCD) candidate olinciguat ended its development, and attention turned to the Cambridge, Mass.-based firm’s earlier-stage effort with IW-6463, a drug in the same class for central nervous system (CNS) disorders.
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