Ionis and Otsuka announced on 22 September that intrathecal ulefnersen met the prespecified primary endpoint in the placebo-controlled FUSION trial for FUS-mutated amyotrophic lateral sclerosis. A joint rank of death or permanent ventilation, rescue…
Johnson & Johnson released additional analyses of the randomized MajesTEC-3 trial in relapsed or refractory multiple myeloma after one to three prior treatment lines. At 36 months, overall survival was 83.3 percent with teclistamab…
Roche and Ionis reported that the 459-patient randomized IMAgINATION Phase 3 trial met its prespecified 37-week proteinuria endpoint in primary IgA nephropathy. Monthly subcutaneous sefaxersen significantly reduced 24-hour urine protein-to-creatinine ratio versus placebo. The…
Roche reported that once weekly enicepatide met both primary endpoints in the randomized Phase 2 CT 388 104 study. At 24 mg, mean HbA1c fell 2.65 percentage points and body weight fell 15.5 percent…
Shanghai RAAS disclosed on 17 September that SR604 entered a single arm multicenter Phase 3 study for prophylaxis in hemophilia A or B patients with or without inhibitors. The registered dose is 0.4 mg…
Vertex reported Phase 2b AMPLIFIED data in 41 people with APOL1 mediated kidney disease. Inaxaplin 45 mg once daily on top of standard care reduced urine albumin to creatinine ratio by 42.7 percent at…
Viking reported that participants switching from weekly VK2735 induction to every other week dosing maintained an average 90 percent of prior weight loss over 12 weeks, while monthly dosing maintained 85 percent versus 61…
Amgen reported that the approximately 621-participant OASIZ 301 trial met its Week 48 primary endpoint in adults with moderate-to-severe systemic Sjogren disease. Dazodalibep produced a statistically significant and company-characterized clinically meaningful improvement in ESSDAI,…
Compass disclosed that FDA recommended a trial demonstrating overall survival benefit before a biologics license application for tovecimig in previously treated advanced biliary tract cancer. Compass disagrees that a new trial is warranted and…
Pharming reported positive topline data from 13 participants with genetically defined primary immunodeficiencies linked to PI3K signaling. In the open label within patient dose escalation study, leniolisib produced a mean 26.4 percent spleen volume…
