Disc Medicine Inc. CEO John Quisel said that top-line phase II findings from the study called Aurora with bitopertin in erythropoietic protoporphyria are “hard for us to interpret. This package of data is something that we’re going to have to sort through,” and the Watertown, Mass.-based firm expects to talk with the U.S. FDA about next steps in the second half of this year.
The possibility for expanding Bristol Myers Squibb Co.’s S1P modulator, Zeposia (ozanimod), into Crohn’s disease took a hit on disappointing data from the initial analysis of its Yellowstone study, the first of two phase III trials. Results showed the study failed to meet the primary endpoint, defined as clinical remission as measured by the Crohn’s Disease Activity Index at week 12.
Exact Sciences Corp. reported on data showing high sensitivity and specificity for the non-endoscopic Oncoguard Esophagus test that is currently in development for the detection of esophageal adenocarcinoma (EAC) and its precursors, including Barrett’s esophagus.
An increasingly popular target across varied cancer types is the immune system regulator V-domain Ig suppressor of T-cell activation (VISTA), where a number of developers have taken early stage aim – among them Sensei Biotherapeutics Inc., with SNS-101, which Wainwright analyst Edward White believes could be the first anti-VISTA monoclonal antibody approved as a therapeutic agent. But there’s plenty of work ahead.
Clarity Pharmaceuticals Ltd. raised AU$121 million (US$79.29 million) through a private placement that will fund the company’s radiopharma pipeline comprising targeted copper therapies out to early 2026.
The fast-shifting obesity space gained more clinical results as Viking Therapeutics Inc. shared data from its phase I, multiple ascending-dose trial with oral VK-2735, a dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors.
Updated results from Stoke Therapeutics Inc.’s studies of antisense oligonucleotide STK-001 showed a reduced convulsion seizure frequency in those ages 2 to 18 with Dravet syndrome. The new data, which also showed improved cognition and behavior in participants, substantially boosted the company’s stock value and caused analysts to applaud.
Despite numerous marketed anti-seizure medications in the U.S., patients with epilepsy continue to experience breakthrough seizures and tolerability issues compounded with drug interactions and side effects, leaving room for companies like Praxis Precision Medicines Inc. to fill treatment gaps. The Boston-based company presented phase IIa proof-of-concept data March 26 showing that in an evaluation of photo paroxysmal response, its once-daily oral small-molecule treatment, PRAX-628, resulted in a complete response in three epilepsy patients receiving 45 mg of the drug and four patients receiving 15 mg for a combined complete response rate of 88%.
China’s National Medical Products Administration has cleared China Grand Pharmaceutical and Healthcare Holdings Ltd. to advance radiopharmaceutical agent ITM-11 (177Lu-edotreotide) to phase III trials in gastroenteropancreatic-neuroendocrine tumors
An asset that some investors may have overlooked came through for Axsome Therapeutics Inc., which rolled out phase III data from the trial called Symphony with AXS-12 (reboxetine), which hit the primary endpoint by significantly reducing cataplexy attacks in narcolepsy patients vs. placebo.
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