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.”
Kala Pharmaceuticals Inc.’s anticipated win with Eysuvis (loteprednol etabonate ophthalmic suspension) 0.25% for the short-term (up to two weeks) treatment of the signs and symptoms of dry eye disease (DED) didn’t help shares (NASDAQ:KALA), which closed Oct. 27 at $6.28, down $1.44, or 18.7%, on word of the FDA approval.
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.
CEO Eric Dube said Retrophin Inc. will “share more [about clinical development plans] once the deal has closed” in the fourth quarter of this year and Orphan Technologies Ltd. belongs to his firm, which has seen only animal data so far with OT-58, an enzyme replacement therapy (ERT) for classical homocystinuria (HCU).
The large-scale failure of a handful of drugs repurposed for COVID-19 hasn’t slowed efforts to find existing – and new – therapies as well as vaccines that can fight the pandemic, with research updates continuing to roll out.
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.
Cambridge, U.K.-based Bicycle Therapeutics plc’s phase I dosing about a month ago of its first patient with nectin-4-targeting BT-8009 put the company on the road to hoped-for success by pursuing the same mechanism of action as Padcev (enfortumab vedotin-ejfv), the antibody-drug conjugate (ADC) from Astellas Pharma Inc. and Seagen Inc.
The large-scale failure of a handful of drugs repurposed for COVID-19 hasn’t slowed efforts to find existing – and new – therapies as well as vaccines that can fight the pandemic, with research updates continuing to roll out.
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.”
