A large-scale study cross-referencing genomic data from multiple sources with primary care health records has identified genetic overlaps in 72 chronic diseases, opening the way for a more holistic approach to researching, treating and preventing multimorbidity.
Kura Oncology Inc. and Kyowa Kirin Co. Ltd.’s selective oral menin inhibitor, ziftomenib, met the primary endpoints in the phase II registrational Komet-001 trial in patients with relapsed/refractory NPM1-mutant acute myeloid leukemia (AML), and Kura expects to submit its NDA to the U.S. FDA in the second quarter of 2025.
Touting its novel approach for treating glaucoma, Qlaris Bio Inc. reported promising top-line data from two phase II studies testing QLS-111 in patients with primary open-angle glaucoma and ocular hypertension, showing the drug met all primary and secondary endpoints, reducing intraocular pressure with a clean safety profile that could encourage patients to remain on treatment.
Alterity Therapeutics Ltd. reported positive top-line phase II results for lead candidate ATH-434 for treating multiple system atrophy, a rare neurological disorder similar to Parkinson's disease.
George Medicines, a new spinout from Australia’s George Institute for Global Health, could offer patients better control of their blood pressure as well as fewer side effects thanks to an ultra-low-dose triple combination.
The first successful phase II trial in Duchenne muscular dystrophy (DMD) heart disease rolled out from Cumberland Pharmaceuticals Inc., which said top-line findings from the experiment called Fight DMD showed promising results in the indication that represents the main cause of death for such patients.
New and positive data for Maia Biotechnology Inc.’s lead candidate for a particularly deadly and advanced lung cancer propelled the stock modestly higher on Feb. 4. The pivotal phase II THIO-101 study of Thio, a telomere-targeting agent sequenced with Regeneron Inc.’s immune checkpoint inhibitor, Libtayo (cemiplimab), as a third-line treatment for advanced non-small-cell lung cancer (NSCLC) showed median overall survival of 16.9 months for the 22 NSCLC patients who received at least one dose of Thio in parts A and B.
To realize the promise of cell therapy for neurodegenerative disorders, S.Biomedics Co. Ltd. is looking to expand clinical trials of TED-A9, its stem cell therapy for Parkinson’s disease (PD), to the U.S., having reaped positive results from a domestic phase I/IIa trial in November 2024.
Major strides for the company on two fronts – psychedelic therapies and treatment-resistant depression (TRD) – came in the form of Dublin-based GH Research plc’s phase IIb data with GH-001, an inhalable mebufotenin (5-MeO-DMT) activator of serotonin 5-HT2A and 5-HT1A receptors that met not only its primary endpoint but all secondary goals in the TRD experiment. Shares of GH (NASDAQ:GHRS) closed Feb. 3 at $17.99, up $7.39, or 69.7%, on word that the fast-acting, short-duration compound led to a significant reduction from baseline of -15.2 points in Montgomery-Åsberg Depression Rating Scale total score on the eighth day of treatment compared with +0.3 points in the placebo group (a difference of -15.5 points, p<0.0001).
Alterity Therapeutics Ltd. reported positive top-line phase II results for lead candidate ATH-434 for treating multiple system atrophy, a rare neurological disorder similar to Parkinson's disease.
RSS
