Shares of Applied Therapeutics Inc. (NASDAQ:APLT) dropped 36% Jan. 5 on news that its oral aldose reductase inhibitor, AT-001 (caficrestat), failed to reach statistical significance in a phase III study in patients with diabetic cardiomyopathy. The New York-based firm, however, pointed to positive trends in the Arise-HF study that could offer a path forward, albeit via a partner.
Allogene Therapeutics Inc.’s decision to preferentially pursue first-line treatment of large B-cell lymphoma (LBCL) with CAR T cemacabtagene ansegedleucel (cema-cel, previously known as ALLO-501A) met mixed reviews on Wall Street.
Newron Pharmaceuticals SpA has reported what it claims are “exceptional” results in the 12-month analysis of a phase II open-label trial of evenamide in treatment-resistant schizophrenia. The glutamate modulator produced benefits “of a kind that have never been reported before,” the company said.
With positive initial phase I/II data in hand from two trials, Dyne Therapeutics Inc. plans to report more findings and start enrolling registrational cohorts in both studies by the end of this year for DYNE-101 in myotonic dystrophy type 1 (DM1) and a study called Deliver with DYNE-251 in Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
Anavex Life Sciences Corp.’s stock (NASDAQ:AVXL) sunk 35% on Jan. 2 after a phase II/III study failure in Rett syndrome but posted a next-day rebound of 8% on Jan. 3 with shares closing at $6.53 each. Top-line data from the randomized, double-blind, placebo-controlled study of the company’s lead asset, Anavex 2-73 (blarcamesine), showed improvement but did not reach statistical significance on the co-primary endpoint, the Rett Syndrome Behaviour Questionnaire.
Sciwind Biosciences Co. Ltd.’s injectable glucagon-like peptide-1 (GLP-1) analog, ecnoglutide (XW-003), achieved positive top-line results in a phase III trial in Chinese adults with type 2 diabetes. A long-acting, cAMP signaling biased GLP-1 analog, ecnoglutide is being developed for treating type 2 diabetes and obesity. GLP-1 receptor agonists are increasingly gaining attention in the obesity therapy area.
Starpharma Holdings Ltd.’s DEP docetaxel phase II trial met its primary endpoints, demonstrating antitumor activity in multiple advanced, metastatic cancers, including pancreatic, gastro-esophageal, non-small-cell lung cancer and cholangiocarcinoma.
Longboard Pharmaceuticals Inc.’s positive – and then some – phase Ib/IIa top-line data with 5-HT2C receptor superagonist bexicaserin (LP-352) in developmental and epileptic encephalopathies (DEEs) sparked Wall Street speculation about competitive odds as well as the shape of the firm’s upcoming phase III effort.
Clinical trial updates saw a 5.82% increase for the period of January to November 2023 compared to the same period last year. In November, BioWorld reported on 354 phase I-III trial updates. Although marking a decline from the 416 reported in October, the number of updates still surpasses the previous three months.
Cytokinetics Inc. received a slightly last minute Christmas gift in the form of positive top-line data from the pivotal phase III study of aficamten in treating symptomatic obstructive hypertrophic cardiomyopathy.
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