Clinical Trials
New Confirmatory Study Suggests Progress in Applied Therapeutics–FDA Alignment for Rare Disease Drug
Applied Therapeutics; govorestat; CMT-SORD; rare disease; FDA; confirmatory study; clinical trial; regulatory alignment
Eli Lilly Withdraws $1.9B Phase 2b Obesity Trial Before Launching Enrollment
Eli Lilly; obesity drug; tirzepatide; bimagrumab; trial termination; phase 2b; Zepbound; type 2 diabetes; clinical trial; regulatory strategy
uniQure’s Gene Therapy AMT-130 Significantly Slows Huntington’s Progression in Pivotal Trial
Gene therapy; uniQure; AMT-130; Huntington’s disease; clinical trial; disease progression; FDA approval; huntingtin-lowering; neurodegeneration
BMS Breaks Clinical Losing Streak with Positive Phase 3 Myeloma Readout for Iberdomide
Bristol Myers Squibb; iberdomide; multiple myeloma; clinical trial; EXCALIBER-RRMM; minimal residual disease negativity; CELMoD; protein degradation; progression-free survival; phase 3; relapsed or refractory
FDA Rejects Lundbeck and Otsuka’s Rexulti for PTSD on Insufficient Efficacy Evidence
FDA; Rexulti; Lundbeck; Otsuka; PTSD; brexpiprazole; sertraline; Complete Response Letter; sNDA; clinical trials; drug approval
Fierce Biotech Fundraising Tracker ’25: Avenzo adds $60M Series B; Ollin debuts with $100M
Avenzo Therapeutics; Series B financing; Ollin Biosciences; biotech fundraising; oncology; ophthalmology; venture capital; pipeline development; bispecific antibody; clinical trials
Neurocrine Biosciences Reports Positive Phase 2 Results for Osavampator in Major Depressive Disorder
Neurocrine Biosciences; osavampator; Phase 2 results; major depressive disorder; AMPA-PAM; SAVITRI study; clinical trial; adjunctive treatment; depression; safety profile
Pfizer Acquires Metsera to Compete with Zepbound in Obesity Market
Pfizer; Metsera; obesity treatment; Zepbound; GLP-1 therapies; MET-233i; monthly dosing; amylin mimetic; MET-097i; acquisition; clinical trials
Big Pharma-Backed MapLight Plots IPO to Advance Neuro Pipeline
MapLight Therapeutics; IPO; neuro pipeline; Alzheimer’s disease psychosis; schizophrenia; M1/M4 muscarinic agonist; Series D financing; Morgan Stanley; biopharmaceutical; clinical trials
FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity, the First Treatment for Barth Syndrome
FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; mitochondrial disease; accelerated approval; rare disease; pediatric disease; clinical trial