Sodium Phenylbutyrate/Tauroursodeoxycholic acid (Amylyx Pharmaceuticals)

BrainStorm Cell Therapeutics has submitted an IND amendment to the FDA for NurOwn, its autologous mesenchymal stem cell therapy for ALS, setting the stage for a pivotal Phase 3b clinical trial.

Amylyx reported positive topline data from its Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome, showing improvement or stabilization across key disease measures.

Amylyx Pharmaceuticals' AMX0035 demonstrated sustained improvements in pancreatic function in adults with Wolfram syndrome in a Phase 2 trial.

Aldeyra Therapeutics' stock plummeted after the FDA identified substantive review issues with its NDA for reproxalap for dry eye disease, potentially delaying approval.

A comprehensive review of ALS drug development reveals 53 new drug candidates in clinical trials between 2020-2022, with 13 compounds advancing to Phase 3 trials and showing promising therapeutic potential.