Neurogene Inc. has received FDA clearance of its IND application for NGN-401 for the treatment of Rett syndrome. The company plans to initiate a phase I/II trial in female pediatric patients with Rett syndrome this year.
Mirati Therapeutics Inc. has received FDA clearance of its IND application for MRTX-1133, a potent, oral small-molecule inhibitor of the KRAS G12D driver mutation.
Harbour Biomed Ltd. has received FDA clearance of its IND application to initiate clinical trials in the U.S. with HBM-1007, a fully human monoclonal antibody targeting CD73.
Exegenesis Bio (Hangzhou Exegenesis Biotechnology Co. Ltd.) has received FDA clearance of its IND application for EXG-102-031, a recombinant adeno-associated virus (rAAV)-based gene therapy that is being studied for the treatment of neovascular age-related macular degeneration (AMD).
The FDA has awarded rare pediatric disease designation to Orphagen Pharmaceuticals Inc.'s OR-449 for the treatment of pediatric adrenocortical carcinoma (ACC). OR-449 is a selective, first-in-class, potent and orally bioavailable small-molecule antagonist to steroidogenic factor-1 (SF-1; NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland.
Lantern Pharma Inc.'s LP-284 has been awarded orphan drug designation by the FDA for the treatment of mantle cell lymphoma (MCL). LP-284 is a novel small-molecule agent that preferentially damages DNA in cancer cells harboring mutations in DNA damage repair pathways.
SFA Therapeutics Inc. has received FDA clearance of its IND application to investigate SFA-001N in patients with nonalcoholic steatohepatitis (NASH) with or without fibrosis.
Hotspot Therapeutics Inc. has received FDA clearance of its IND application for HST-1011, the company's investigational small-molecule allosteric inhibitor of casitas B-lineage lymphoma-B (CBL-B), an E3 ubiquitin protein ligase critically involved in immune cell response.
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