Sample Alerts - inThought Research

Small Pharma Patents Novel Injectable Formulation of its DMT and Other Psychedelic Compounds

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…

Analysis

Astellas’ phase 1/2 FORTIS trial for late-onset Pompe disease placed on FDA clinical hold

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…

Analysis

FDA unanimously endorses beti-cel for the treatment of beta-thalassemia

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)…

Analysis

FDA endorses Bluebird Bio's eli-cel gene therapy for CALD

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…

Analysis

Ultomiris meets primary endpoint in phase 3 CHAMPION-NMOSD trial

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…

Analysis

Protalix's Phase III BALANCE trial of PRX-102 in Fabry meets endpoint and shows non-inferiority to Fabrazyme

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…

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