Latest news with #)Pharmaceuticals
Business Insider
29-07-2025
- Health
- Business Insider
FDA approves Apellis' Empaveli for treatment of C3 glomerulopathy
Apellis (APLS) Pharmaceuticals announced that the U.S. Food and Drug Administration has approved EMPAVELI as the first treatment for C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis in patients 12 years of age and older, to reduce proteinuria. C3G and primary IC-MPGN are rare kidney diseases, affecting 5,000 people in the United States. 'I'm excited to now have a highly effective therapy for a broad range of patients living with C3G and primary IC-MPGN,' said Carla Nester, M.D., MSA, FASN, lead principal investigator for the VALIANT study, professor of internal medicine and pediatrics and director of pediatric nephrology, University of Iowa Stead Family Children's Hospital. 'With standard of care, patients living with these rare and severe diseases frequently progress to kidney failure, necessitating lifelong dialysis and/or a kidney transplant. Given the urgent need, particularly in children, the approval of EMPAVELI marks a pivotal moment in the treatment of rare kidney diseases.' In the Phase 3 VALIANT study, EMPAVELI demonstrated an unprecedented 68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of C3 deposits as measured by C3 staining, compared to placebo. The positive results were consistent across adolescent and adult patients with C3G and primary IC-MPGN, and in C3G patients with post-transplant disease recurrence.
Business Insider
14-07-2025
- Health
- Business Insider
Amylyx presents new analyses from Phase 2, Phase 2b trials of avexitide
Amylyx (AMLX) Pharmaceuticals announced the presentation of new exploratory analyses from the Phase 2 PREVENT and Phase 2b clinical trials of avexitide, an investigational, first-in-class glucagon-like peptide-1, GLP-1, receptor antagonist for the treatment of post-bariatric hypoglycemia, PBH, at the Endocrine Society's annual meeting, ENDO 2025. In the Phase 2b trial, avexitide 90 mg once daily, the dose being evaluated in the pivotal Phase 3 LUCIDITY trial, led to a 64% least-squares mean reduction vs. baseline in the composite rate of Level 2 and Level 3 hypoglycemic events in PBH, with more than half of the participants experiencing no events during the treatment period. The FDA-agreed-upon primary endpoint of LUCIDITY is reduction in the composite of Level 2 and Level 3 hypoglycemic events. Consistent reductions in composite rate of Level 2 and Level 3 hypoglycemic events also were seen with avexitide 45 mg twice daily studied in the Phase 2b trial and avexitide 30 mg twice daily and 60 mg once daily studied in the Phase 2 PREVENT trial. New pharmacokinetic and pharmacodynamic data were also presented demonstrating continuous pharmacologic activity of the 90 mg once daily dose regimen for a 24-hour period. Elevate Your Investing Strategy: Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Make smarter investment decisions with , delivered to your inbox every week.
Business Insider
14-06-2025
- Business
- Business Insider
KalVista says FDA will not meet PDUFA goal date for Sebetralstat NDA
KalVista (KALV) Pharmaceuticals nnounced that the U.S. Food and Drug Administration has notified the company that it will not meet the PDUFA goal date for the New Drug Application for sebetralstat, the company's investigational oral on-demand treatment for hereditary angioedema. The FDA notified the company on June 13, 2025, that the previously disclosed June 17, 2025 PDUFA goal date will not be met due to heavy workload and limited resources. The FDA indicated that it expects to deliver a decision within approximately four weeks. The FDA has not requested additional data or studies and has not raised any concerns regarding the safety, efficacy or approvability of sebetralstat. KalVista has addressed all prior information requests in a timely manner, and the company believes the only remaining item under FDA review is the finalization of the labelling. 'We are disappointed by this delay, most importantly because we know how much people living with HAE are looking forward to an oral on-demand option to treat their HAE attacks,' said Ben Palleiko, CEO of KalVista. 'At the same time, we remain confident in the near-term approval of sebetralstat. We are continuing to work closely with the FDA to support the completion of their review. Our commitment to bringing this important therapy to people living with HAE remains unwavering.'
Yahoo
13-06-2025
- Health
- Yahoo
Syndax reports publication of revumenib data from BEAT AML trial
Syndax (SNDX) Pharmaceuticals announced that data from the BEAT AML trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mutant NPM1 and KMT2A-rearranged acute myeloid leukemia patients were published in the Journal of Clinical Oncology and simultaneously presented in an oral session at the 30th European Hematology Association Annual Congress Meeting being held June 12-15, 2025, in Milan, Italy and virtually. The publication and EHA presentation report updated results from the Phase 1b BEAT AML trial evaluating the safety and clinical activity of revumenib in combination with venetoclax/azacitidine in newly diagnosed older adults with mNPM1 or KMT2Ar AML. The trial is being conducted as part of The Leukemia & Lymphoma Society's Beat AML Master Clinical Trial. As of September 2024, 43 patients were enrolled and treated in BEAT AML across two dose levels of revumenib in combination with venetoclax and azacitidine. Overall, 79% of patients had mNPM1 AML and 21% had KMT2Ar AML. Revumenib was generally well tolerated at both dose levels in combination with venetoclax and azacitidine without a maximal tolerated dose identified. The most common overall non-hematologic treatment-emergent adverse events (TEAEs) of any grade were nausea (60%), constipation (53%), QTc prolongation (44%), hypokalemia (44%), and vomiting (42%). Overall Grade greater than or equal to3 non-hematologic AEs were rare and similar between both dose levels. In the intent-to-treat population, the observed rate of complete remission was 67%, composite complete remission was 81%, and the overall response rate was 88%. Among 37 patients with measurable residual disease response assessment, 100% were MRD negative by centralized flow cytometry testing. The median duration of CRc was 12.0 months. 23% of patients had proceeded to hematopoietic stem cell transplantation as of the February 2025 data cut off. In an early analysis of survival from this single-arm trial, the median overall survival observed was 15.5 months. Subset analysis showed a CRc rate of 77% and an observed median OS of 15.5 months in mNPM1 patients with intermediate risk by ELN 2024, and a CRc rate of 89% and observed median OS of 18.0 months in KMT2Ar patients. In contrast, historical data from newly diagnosed mNPM1 patients with intermediate risk treated with venetoclax and azacitidine show a CRc of 57% and median OS of 9.9 months.1 In newly diagnosed KMT2Ar AML patients treated with venetoclax and hypomethylating agent therapy, a CRc rate of 43% and median OS of 2.5 months was observed in a retrospective analysis. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See today's best-performing stocks on TipRanks >> Read More on SNDX: Disclaimer & DisclosureReport an Issue Syndax announces new data from AUGMENT-101 trial of Revuforj Syndax Pharmaceuticals: Promising 2025 Outlook and Strategic Product Launches Drive Buy Rating Closing Bell Movers: slips 3% after results despite topping estimates Syndax exec buys $105.8K, director buys $91.1K in common stock Syndax Pharmaceuticals Appoints New Head of R&D Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Insider
02-06-2025
- Health
- Business Insider
Regeneron announces analyses from Phase 3 C-Post trial
Regeneron (REGN) Pharmaceuticals announced detailed analyses from the Phase 3 C-POST trial, which evaluated PD-1 inhibitor Libtayo in patients with high-risk cutaneous squamous cell carcinoma, CSCC, after surgery. The results, shared during an oral session at the 2025 American Society of Clinical Oncology, ASCO, Annual Meeting and simultaneously published in the New England Journal of Medicine, NEJM, include additional data for the primary endpoint of disease-free survival, DFS, and the first presentation of key secondary endpoint outcomes. With a median duration of follow-up of 24 months, efficacy results for Libtayo compared to placebo, were as follows: 68% reduction in the risk of disease recurrence or death, with median DFS not reached for Libtayo-treated patients; At two years, DFS was 87% with Libtayo versus 64% with placebo; 80% reduction in the risk of locoregional recurrence; 65% reduction in the risk of distant recurrence
