HIGH IMPACT - Small Pharma announced that the United States Patent and Trademark Office (USPTO) will be issuing a patent for its application pertaining to novel injectable formulations of N,N-dimethyltryptamine (DMT) based compounds including…
https://inthought.com/wp-content/uploads/2022/03/blank-transparent.png720720clickgianthttps://inthought.com/wp-content/uploads/2019/03/inThought-Logo-Transparent-3.7.pngclickgiant2022-07-28 00:00:212022-08-05 00:51:51Small Pharma Patents Novel Injectable Formulation of its DMT and Other Psychedelic Compounds
MODERATE IMPACT - The FDA placed a clinical hold on the FORTIS phase 1/2 trial of AT845 following a case of peripheral sensory neuropathy in one trial participant. FORTIS is evaluating AT845, an investigational AAV8-based gene replacement…
https://inthought.com/wp-content/uploads/2022/03/blank-transparent.png720720clickgianthttps://inthought.com/wp-content/uploads/2019/03/inThought-Logo-Transparent-3.7.pngclickgiant2022-06-27 00:00:452022-07-07 17:49:28Astellas’ phase 1/2 FORTIS trial for late-onset Pompe disease placed on FDA clinical hold
MODERATE IMPACT - After a second day of presentations and discussions, the FDA has voted unanimously to endorse betibeglogene autotemcel (beti-cel) for the treatment of patients with beta-thalassemia who require regular red blood cell (RBC)…
https://inthought.com/wp-content/uploads/2022/03/blank-transparent.png720720clickgianthttps://inthought.com/wp-content/uploads/2019/03/inThought-Logo-Transparent-3.7.pngclickgiant2022-06-10 00:00:042022-07-07 17:44:13FDA unanimously endorses beti-cel for the treatment of beta-thalassemia
HIGH IMPACT - After a full day of presentation and debates around the risk vs benefit of Bluebird Bio's elivaldogene autotemcel (eli-cel) for the treatment of early active cerebral adrenoleukodystrophy (CALD), the FDA unanimously voted to endorse…
MODERATE IMPACT - Although no detailed data were provided, Alexion/AstraZeneca showed positive topline results for the phase 3 open-label CHAMPION-NMOSD trial for Ultomiris. Ultomiris achieved a statistically significant and clinically…
HIGH IMPACT - Protalix and Chiesi announce topline results of Phase III BALANCE trial evaluating the safety and efficacy of PRX-102 in Fabry. Study: Phase III BALANCE study is a 12-month, randomized, double-blind, study of 1 mg/kg…
https://inthought.com/wp-content/uploads/2022/03/blank-transparent.png720720clickgianthttps://inthought.com/wp-content/uploads/2019/03/inThought-Logo-Transparent-3.7.pngclickgiant2022-04-04 00:00:372022-05-06 14:55:07Protalix's Phase III BALANCE trial of PRX-102 in Fabry meets endpoint and shows non-inferiority to Fabrazyme