TARRYTOWN, N.Y. — Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced encouraging data from the Phase 1/2 LINKER-MM4 trial evaluating Lynozyfic™ (linvoseltamab) in adults with newly diagnosed multiple myeloma (NDMM) who were transplant eligible or ineligible were shared in an oral presentation at the American Society of Hematology (ASH) Annual Meeting....
treatment News
Tarrytown, NY – Along with resolving site inspection issues that cost Regeneron a high-dose Eylea launch in June, the company has scored an FDA nod for Veopoz as the first treatment for the ultra-rare inherited immune condition Chaple disease. The disease, which is also known as CD55-deficient protein-losing enteropathy, has a global patient...
TARRYTOWN, N.Y. — Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the primary endpoint was met in the Phase 3 OPTIMA trial investigating garetosmab in adults with fibrodysplasia ossificans progressiva (FOP). At 56 weeks, both doses of garetosmab, 3 mg/kg and 10 mg/kg, were highly efficacious in reducing the number of new bone...
ROCKVILLE, Md., – Potential one-time gene therapy for the treatment of Duchenne includes a novel, optimized microdystrophin transgene and REGENXBIO’s proprietary NAV® AAV8 vector Innovative trial design, including comprehensive immunosuppressive regimen, to evaluate safety and optimal dose cGMP process material made at commercial-scale to be used throughout clinical development of RGX-202...
ROCKVILLE, Md. –– REGENXBIO Inc. (Nasdaq: RGNX) today announced the publication of results from the Phase I/IIa trial evaluating the safety and tolerability of a single dose of subretinal ABBV-RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). Two-year data were published in The Lancet in a paper titled...
ROCKVILLE, Md. — REGENXBIO Inc. (Nasdaq: RGNX) today announced topline results from the Phase I/II/III CAMPSIITE® trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, demonstrating that the pivotal phase of the trial met...
Takeda Korea said Wednesday that it received approval for Obizur, a treatment for bleeding in adult patients with acquired hemophilia A (AHA), from the Ministry of Food and Drug Safety. AHA is a disease characterized by the development of autoimmune antibodies against blood coagulation factor 8, resulting in bleeding-related complications....
PRINCETON, N.J. — Bristol Myers Squibb Company (NYSE: BMY) announced three regulatory acceptances from the U.S. Food and Drug Administration (FDA) and Japan’s Ministry of Health, Labour and Welfare (MHLW) for Breyanzi® (lisocabtagene maraleucel). In the U.S., the FDA has accepted the company’s two supplemental Biologics License Applications (sBLA) for...
SAN DIEGO, Calif. — Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs, today announced it has completed enrollment in the third cohort of patients in the Phase 1b MAD study of RGLS8429 for the treatment of ADPKD. “The completion of our...
SAN DIEGO, Calif. — Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs, today announced positive topline results from the second cohort of patients in its Phase 1b MAD study of RGLS8429 for the treatment of ADPKD. The Phase 1b...