Aelis Farma’s Phase 2B clinical trial for AEF0117, a potential treatment for cannabis use disorder, did not meet its primary endpoint of reducing cannabis use to one day or less per week. Indivior, which had an option to license the global rights to AEF0117, has decided not to exercise its option due to the lack of separation from placebo in the trial results.
Windtree Therapeutics, Inc. (WINT) is seeing its stock rise after completing enrollment in its SEISMiC Extension Phase 2b study of istaroxime for early cardiogenic shock. This study builds upon positive results from a previous trial, focusing on longer dosing duration and exploring potential benefits of a tapering dosage regimen.
Biogen’s higher dose regimen of nusinersen, a drug for spinal muscular atrophy (SMA), demonstrated a statistically significant improvement in motor function in infants compared to a sham control group in a Phase 2/3 study. The results suggest that the higher dose regimen could be more effective in slowing neurodegeneration and provide greater clinical benefit for patients.
Athira Pharma’s experimental Alzheimer’s drug, fosgonimeton, failed to demonstrate a statistically significant improvement in cognition and function in a Phase 2/3 clinical trial. While the drug showed promising trends in certain patient subgroups, the overall results fell short of expectations, leading to a substantial drop in the company’s stock price.
Dyne Therapeutics announced positive clinical data from its Phase 1/2 DELIVER trial of DYNE-251 for Duchenne muscular dystrophy (DMD). The drug showed significant dystrophin expression and functional improvements in patients, exceeding levels observed in other DMD treatments. Dyne plans to initiate registrational cohorts in the trial and provide an update on the path to registration by the end of 2024.
Praxis Precision Medicines announced positive topline results from its EMBOLD Phase 2 study evaluating relutrigine in patients with SCN2A and SCN8A developmental and epileptic encephalopathy (DEE). The study showed a significant reduction in seizures, improvements in behavior and communication, and a favorable safety profile.
Recursion Pharmaceuticals’ stock took a hit despite announcing positive safety data from its Phase 2 trial for REC-994, an investigational treatment for Cerebral Cavernous Malformation (CCM). While the trial met its primary endpoint for safety and tolerability, the company reported no significant improvements in patient outcomes at the 12-month mark. However, secondary endpoints showed encouraging trends in reduced lesion volume and hemosiderin ring size in patients on the highest dose.
U.S. stock futures opened lower on Tuesday, with the Nasdaq futures leading the decline. Dyne Therapeutics shares fell sharply after positive clinical trial data, while other notable pre-market losers included Acadia Healthcare, United States Steel, Diversified Energy, and LG Display. Several factors, including potential strikes and downgraded ratings, contributed to the downward trend in some stocks.
Jazz Pharmaceuticals’ Epidiolex, a cannabidiol oral solution for treating rare forms of epilepsy, showed mixed results in a Phase 3 trial in Japan. While the trial did not achieve its primary goal of reducing seizure frequency, there were improvements in other areas, and no new safety concerns were identified. The company plans to continue collecting data and engage with Japanese regulatory authorities regarding a potential new drug application.
AN2 Therapeutics has discontinued its mid to late-stage study on epetraborole for patients with treatment-refractory mycobacterium avium complex (MAC) lung disease due to unsatisfactory efficacy results. The company is now focusing on restructuring and exploring other potential uses for epetraborole. AN2 Therapeutics is also moving forward with clinical trials for its other drug candidates.
