Company created following Jazz Pharmaceuticals’ acquisition of Actio Biosciences Setara plans to advance clinical-stage small molecule TRPV4 inhibitor for treatment of Charcot-Marie-Tooth disease type 2C (CMT2C) and overactive bladder, along with other early-stage discovery programs Investor syndicate comprised of leading healthcare investors including Deerfield Management, Canaan, Droia Ventures, EcoR1, Euclidean Capital, and Regeneron Ventures, as well as Jazz Pharmaceuticals Setara Bioscienc

- Revuforj is the first and only menin inhibitor that is approved for adults and children one year and older with R/R NPM1m AML and KMT2A translocated acute leukemia - Oct. 30, 2026 -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today announced that Revuforj® (revumenib) has been recognized as the Best Product for Rare/Orphan Diseases of 2026 by The Galien Foundation. The Galien Foundation fosters, recognizes, and r

INOVIO delivers a new nonsurgical treatment option with a broad indication for adults living with RRP, a rare and debilitating disease that often requires surgery VOCERYQ™ is a plasmid DNA-based immunotherapy that targets the root cause of RRP: HPV-6 and HPV-11 INOVIO expects to make VOCERYQ available to order and ship in approximately 60 days, with full commercial launch in the first quarter of 2027 VOCERYQ represents the first U.S. FDA approved DNA medicine, validating INOVIO's novel platform

DEBRA Research and Topadur Pharma have announced a collaboration to explore a new treatment approach for dystrophic epidermolysis bullosa (DEB). DEB is one of the more severe forms of EB, characterised by extremely fragile skin and progressive, debilitating complications. The programme will evaluate Topadur’s clinical-stage compound TOP-N53 together with additional selected small molecules from its proprietary DualTOP platform. The compounds will be investigated in in vitro and in vivo models ad

The Phase III AIRWARD™ trial was published today on ClinicalTrials.gov (NCT07907991).1 AIRWARD will evaluate the efficacy and safety of verducatib, an investigational oral therapy for COPD.1 The Phase III trial will enroll adults with symptomatic COPD who continue to experience exacerbations and have blood eosinophil counts below 300 cells/µL.1 Boehringer Ingelheim today announced the registration of the Phase III AIRWARD™ trial evaluating its investigational oral therapy verducatib as a treatm

In adults with type 2 diabetes, retatrutide lowered A1C by an average of 2.2% vs. 1.8% with Ozempic 2 mg at 80 weeks Participants taking retatrutide lost an average of 44 lbs to 47 lbs (20% to 21%) vs. 21 lbs (9%) with Ozempic 2 mg at 80 weeks TRANSCEND-T2D-2 marks the sixth Phase 3 trial in which retatrutide has delivered powerful efficacy, reinforcing its potential to transform cardiometabolic care Oct 28, 2026 -- Eli Lilly and Company (NYSE: LLY), the maker of Mounjaro (tirzepatide) and Foun

The US Food and Drug Administration (FDA) has granted orphan drug designation to Octant’s OCT-980, an investigational oral small molecule, to treat retinitis pigmentosa (RP). The regulatory status specifically includes rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP), the indication for which the candidate is presently being evaluated. Orphan status is granted to support the development of therapies for rare diseases and conditions. This designation offers financial and re

New Cleveland Clinic research published in Cancer Cell shows that people with glioblastoma have more active platelets, which are cells involved in blood clotting. Platelets also communicate with other cells and can influence immune activity. The overactive platelets in people with glioblastoma slow the activity of cancer-fighting T cells, which can contribute to tumor growth and cancer progression. Researchers from the lab of Justin Lathia, PhD, continue to investigate the unknowns around sex d

Nxera has exercised its option to license lucerastat for Japan, South Korea, Taiwan and selected other Asia Pacific markets (excluding China), paying Idorsia CHF 7 million upfront and contributing to the global Phase 3 program. Lucerastat is an investigational oral therapy for Fabry disease with more than 400 patient-years of clinical experience, adding a late-stage rare disease asset to Nxera's portfolio in Japan and Asia Pacific. Idorsia today initiated FAB-KLEAR, a pivotal Phase 3 study, as

Initial award funds process development, manufacturing, and clinical evaluation of a single-dose vaccine candidate through strategic partnerships with Hilleman Laboratories, SK bioscience, and IDT Biologika October 28, 2026 / Today IAVI, a global nonprofit biomedical research organization, together with partners Hilleman Laboratories, SK bioscience, and IDT Biologika announced that IAVI has been awarded US $36 million of a contract valued up to US $99 million from the Administration for Strateg