Placebo Response Trips Up Harmony Biosciences’ Fragile X Drug in Late-Stage Phase 3 Trial
Harmony Biosciences; ZYN002; Fragile X syndrome; Phase 3 trial; placebo response; clinical trial failure; RECONNECT study; transdermal cannabidiol gel
Sparrow Pharmaceuticals Raises $95M Series B for Midstage Diabetes Study
Sparrow Pharmaceuticals; Series B fundraising; Type 2 diabetes; clofutriben; phase 2b trial; HSD-1 inhibition; cortisol; RA Capital Management; Forbion; venture capital
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
Acadia Halts Development of Nasal Prader-Willi Drug After Phase 3 Failure
Acadia Pharmaceuticals; carbetocin; Prader-Willi syndrome; Phase 3 trial; COMPASS PWS study; clinical trial failure; nasal spray; hyperphagia
Biogen Plans Resubmission After FDA Declines High-Dose Spinraza for SMA, Requests More Production Information
Biogen; Spinraza; nusinersen; high dose; FDA; Complete Response Letter; spinal muscular atrophy; SMA; supplemental New Drug Application; Chemistry Manufacturing and Controls; CMC; regulatory; resubmission
Sanofi Injects $625M Into Biotech Investment Arm to Accelerate Innovation
Sanofi; Sanofi Ventures; biotech investment; $625M cash infusion; digital health; immunology; rare diseases; neurology; venture capital; cell/gene therapy; portfolio expansion
FDA’s Move to Update GSK’s Leucovorin for Autism Raises Questions on Dose, Supply, and Data
FDA; GSK; leucovorin; Wellcovorin; autism; cerebral folate deficiency; drug relabeling; dose; supply; clinical data
FDA Issues Warning Letter to J&J Subsidiary Over Stopper Issues and Quality Reporting at Korean Plant
FDA warning letter; Johnson & Johnson subsidiary; Janssen Vaccines; Korean production plant; vial stopper defects; quality control violations; spotty complaint reporting; good manufacturing practices; Incheon; pharmaceutical manufacturing
Eli Lilly Picks Houston for $6.5 Billion API Manufacturing Facility
Eli Lilly; Houston; API manufacturing; $6.5 billion investment; Generation Park; small molecule medicines; pharmaceutical manufacturing; cardiometabolic; oncology; immunology; neuroscience
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