Biopharma News Round Up 7–11th September
This week's biopharma news was led by oncology readouts, regulatory momentum in rare disease, and a handful of notable pipeline setbacks.
BY RXCLARITY
Oncology: lung cancer and cell therapy in focus
Lung cancer remained especially active. Bayer secured FDA approval for Hyrnuo (sevabertinib) in first-line HER2-mutant NSCLC, creating direct competition with Boehringer’s Hernexeos. Cullinan Therapeutics presented interim Phase 3 data for zipalertinib plus chemotherapy in first-line EGFR exon20ins NSCLC, while Amgen reported improved overall survival for tarlatamab combined with a checkpoint inhibitor in advanced lung cancer.
Elsewhere, Bristol Myers Squibb reported positive Phase 2 data for its GPRC5D CAR-T, arlocabtagene autoleucel, in relapsed/refractory multiple myeloma. Disc Medicine also reported encouraging Phase 2 results for DISC-3405 in polycythemia vera, including reduced phlebotomy and improved hematocrit control.
Not all oncology news was positive. Tyra Biosciences’ Phase 2 bladder-cancer results reportedly missed remission-rate expectations. BridgeBio Oncology also narrowed its KRAS pipeline to focus on its lead inhibitor, reducing the breadth of its development portfolio.
Immunology: lupus progress, atopic dermatitis setback
In systemic lupus erythematosus, Beeline Medicines said afimetoran met its Phase 2 primary endpoint, with significantly improved Week-48 SRI-4 responses versus placebo. Spyre Therapeutics’ SPY003 also met its Week-12 primary endpoint, completing proof of concept for all three components of its IBD combination strategy.
Conversely, Evommune’s MRGPRX2 antagonist failed a Phase 2b study in atopic dermatitis - a material setback in a competitive immunology market. Sobi received FDA Fast Track designation for pacritinib in VEXAS syndrome.
Rare disease: oral HAE prevention and achondroplasia advances
Pharvaris reported pivotal Phase 3 success for extended-release deucrictibant in hereditary angioedema prevention, describing “injectable-like efficacy.” Intellia’s lonvo-z BLA was accepted for Priority Review, with a PDUFA target date of March 10, 2027.
In achondroplasia, Ascendis reported positive Phase 2 infant data for once-weekly navepegritide, while BioMarin submitted an FDA filing to expand Voxzogo following Phase 3 success.
Neurology: progress alongside clinical and regulatory challenges
COMPASS Pathways reported positive one-year follow-up for COMP360 psilocybin in treatment-resistant depression. Quantum BioPharma received FDA clearance to begin Phase 2 testing of Lucid-MS in multiple sclerosis, and Teitur Trophics reported positive Phase 1 safety and tolerability findings for Parkinson’s candidate TT-P34.
However, Shionogi’s redasemtide missed its primary endpoint in acute ischemic stroke, and Biohaven’s BHV-7000/opakalim epilepsy program entered a partial FDA clinical hold pending additional preclinical safety information.
Other notable developments
AirNexis/Haisco’s inhaled dual PDE3/4 inhibitor AN01 met its Phase 2b COPD primary endpoint in China. Abbott received FDA clearance for its TactiFlex catheter for atrial fibrillation. In pain, Adneuris presented data suggesting cebranopadol may have lower abuse liability than Schedule II and IV pain medicines.
Across the week, the clearest theme was continued separation between programs moving on meaningful clinical or regulatory milestones and those facing efficacy or safety-driven delays. Oncology, immunology, rare disease, and neurology all saw both sides of that divide.
Source
Curated news from RxClarity.