SEOUL, South Korea and HYDERABAD, India — The International Vaccine Institute (IVI), an international organization with a mission to discover, develop, and deliver safe, effective, and affordable vaccines for global health, today announced that it has commenced a technology transfer of simplified Oral Cholera Vaccine (OCV-S) to Biological E. Limited...
treatment News
PHILADELPHIA – Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, announced today that the data from the Phase 3 PROPEL pivotal trial, assessing the efficacy, safety and tolerability of AT-GAA in adults with late-onset Pompe disease compared to the standard of...
Relacorilant plus nab-paclitaxel improves progression-free and overall survival in patients with platinum-resistant ovarian cancer, with no need for biomarker selection Addition of relacorilant did not increase side-effects, compared to nab-paclitaxel monotherapy Results presented in late-breaking podium presentation at ASCO 2025 with simultaneous publication in The Lancet REDWOOD CITY, Calif. — Corcept...
Marks the First-Ever Global Pediatric Trial for Menin Inhibitors The PedAL (Pediatric Acute Leukemia) Master Trial is at the heart of LLS’s Dare to Dream Project to transform treatment and care for kids with blood cancer WASHINGTON, DC — The Leukemia & Lymphoma Society (LLS) announced today the first...
WASHINGTON, DC — The Melanoma Research Alliance (MRA), the largest non-profit funder of melanoma research worldwide, welcomes the U.S. Food and Drug Administration (FDA) decision to approve Iovance Biotherapeutics’ AMTAGVI™ (lifileucel) for the treatment of patients with advanced melanoma in the second line treatment setting. AMTAGVI is the first individualized...
STOCKHOLM, Sweden and WALTHAM, Mass, USA – Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that The New England Journal of Medicine (NEJM) published results from the phase 3 PEGASUS study, which demonstrated the superiority of pegcetacoplan, an investigational targeted C3 therapy, in improving...
SAN DIEGO, Calif. — Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the final results from the completed Phase 1/2 MARINA® trial of delpacibart etedesiran (del-desiran) in people living with myotonic dystrophy type 1...
SOUTH SAN FRANCISCO, Calif. — Denali Therapeutics Inc. (Nasdaq: DNLI) today announced the publication of results from the open-label Phase 1/2 clinical trial of its investigational, next-generation enzyme replacement therapy (ERT), tividenofusp alfa (DNL310), for the treatment of Hunter syndrome (mucopolysaccharidosis type II, or MPS II) in the January 1,...
NEWARK, Calif. — CymaBay Therapeutics Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, today announced that The New England Journal of Medicine (NEJM) has published detailed results from the RESPONSE Phase 3 trial evaluating seladelpar, an investigational agent, and the...
WALTHAM, Mass. — Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that The New England Journal of Medicine (NEJM) published positive results from the Phase 3 VALIANT study investigating EMPAVELI® (pegcetacoplan) for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), which are severe and rare kidney diseases. “The positive data published in...