August 19, 2025 – Talk Bio

FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful

4 days ago talkbio0Tagged Clinical Trials, concerns, Effectiveness, factor IX, Failure (biologic function), Friedreich 's ataxia, Rare Diseases, Rejection, Response process, United States Food and Drug Administration, vatiquinone

PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure

Stealth BioTherapeutics Resubmits Third FDA Application for Rare Disease Drug

4 days ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, access program, Drug Approval, Elamipretide, Infrequent, resubmission, Stealth BioTherapeutics, United States Food and Drug Administration

Stealth BioTherapeutics; FDA resubmission; rare disease; Barth syndrome; elamipretide; drug approval; expanded access program

Battle Intensifies in the Obesity Drug Market as Novo Nordisk and Eli Lilly Vie for Dominance

4 days ago talkbio0Tagged Benefit, Clinical Trials, competition, Compounders, Direct-to-Consumer, eli lilly, Glucagon-Like Peptide 1, manager, Market, Nordisk, Obesity, Pharmaceutical Services, Wegovy, Zepbound

obesity drugs; GLP-1; Wegovy; Zepbound; Novo Nordisk; Eli Lilly; market competition; direct-to-consumer; pharmacy benefit managers; compounders; clinical trials

Shankar Ramaswamy’s Kriya Therapeutics Raises $313M for Gene Therapy Advancements

4 days ago talkbio0Tagged Biotechnology, gene therapy, Kriya, Shankar Ramaswamy, venture

Kriya Therapeutics; Shankar Ramaswamy; gene therapy; biotech funding; fundraising; biotechnology; venture capital; 2025

VantAI and Halda Strike $1B+ AI-Driven Partnership on ‘Hold-and-Kill’ Drugs

4 days ago talkbio0Tagged Aortic Valve Insufficiency, cellular targeting, Chimera organism, Drug Discovery, Halda Therapeutics, hold-and-kill, Immunology, Induce (action), Neoplasms, Proximity, RIPTAC, target-effector pairs, VantAI

VantAI; Halda Therapeutics; RIPTAC drugs; hold-and-kill; AI drug discovery; target-effector pairs; oncology; immunology; regulated induced proximity targeting chimeras; $1 billion deal

FDA Extends PDUFA Date for Regenxbio’s Hunter Syndrome Gene Therapy RGX-121

4 days ago talkbio0Tagged clemidsogene lanparvovec, Deferred, gene therapy, IDS gene, Infrequent, Mucopolysaccharidosis II, PDUFA, Regenxbio, RGX-121, United States Food and Drug Administration

FDA delay; PDUFA extension; Regenxbio; Hunter syndrome; clemidsogene lanparvovec; RGX-121; gene therapy; Mucopolysaccharidosis II; IDS gene; rare disease

Merck KGaA and Skyhawk Therapeutics Launch $2B RNA-Targeting Neurology Collaboration

4 days ago talkbio0Tagged Billion Collaboration, Drug Development, Merck KGaA, milestone payments, nervous system disorder, RNA-targeting, royalties, Skyhawk, SkySTAR platform

Merck KGaA; Skyhawk Therapeutics; RNA-targeting; SkySTAR platform; neurological disorders; $2 billion collaboration; drug development; milestone payments; royalties

Reunion Psychedelic Therapy Advances to Pivotal Trials after Postpartum Depression Success

4 days ago talkbio0Tagged Clinical Trials, Depression, Postpartum, Montgomery-Åsberg Depression Rating Scale, Montgomery-Asberg Depression Rating Scale Questionnaire, Neuroscience discipline, Phase 2 Clinical Trials, Phase 3 Clinical Trials, psychedelic therapy, RE104, RECONNECT, remission rates, Reunion

Reunion Neuroscience; RE104; psychedelic therapy; postpartum depression (PPD); Phase II trial; Phase III trial; RECONNECT trial; clinical trial results; Montgomery-Åsberg Depression Rating Scale (MADRS); remission rates

Federal Appeals Court Overturns FDA Rejection of Vanda’s Hetlioz for Jet Lag Disorder

4 days ago talkbio0Tagged Clinical Trials, Evidence, Federal Appeals Court, Hetlioz, jet lag disorder, New Drug Application, regulatory law, sNDA, United States Food and Drug Administration, Vanda Pharmaceuticals

Vanda Pharmaceuticals; Hetlioz; FDA; jet lag disorder; Federal Appeals Court; supplemental New Drug Application (sNDA); regulatory law; clinical trial evidence