Entries by clickgiant

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 the company’s lead assets, SPL026 (DMT) and SPL028 (deuterated DMT). Additionally, the patent also covers novel injectable formulations of other known psychedelic […]

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) transfusions. Bluebird Bio presented a strong case for beti-cel in the treatment of beta-thal with emphasis on high transfusion-independent rate […]

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 the gene therapy. Initial concerns including malignancy with development of MDS, insertional oncogenesis, efficacy benchmarking, and long-term durability […]

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 meaningful reduction in the risk of relapse in adults with AQP4+ NMOSD compared to the external placebo arm from the pivotal Soliris PREVENT clinical trial. […]