The POLARIS-1 study enrolled 147 patients with uncontrolled, moderate-to-severe asthma, and the interim analysis was conducted on the first 98 patients. The analysis demonstrated a half-life of up to 75 days, with clinical effects consistent with twice-yearly dosing. WIN378 produced significant and sustained effects on FEV1 (up to 174 mL mean increase, placebo-adjusted mean increases up to +256 mL, p=0.033), FeNO and blood eosinophils, key biomarkers that correlate with asthma exacerbations. Th

With $116 million in new funding on tap, BrainChild Bio said Tuesday that it has initiated a pivotal study of the B7-H3-targeted autologous CAR-T cell therapy BCB-276, in the hopes of delivering a much-needed new treatment option for patients with diffuse intrinsic pontine glioma (DIPG). With the current standard-of-care treatment for DIPG limited to palliative focal radiation therapy, median overall survival for this rare and aggressive paediatric brainstem tumour is about 11 months from diagn

Having recently secured FDA approval for the first in a new class of immunomodulatory drugs for multiple myeloma, Bristol Myers Squibb continues to make advances in the field, announcing Tuesday positive data from a registrational trial of its GPRC5D-directed CAR-T cell therapy arlocabtagene autoleucel (arlo-cel). The results come from the Phase II QUINTESSENTIAL study, which investigated arlo-cel in patients with quadruple-class exposed relapsed and refractory multiple myeloma (RRMM). BMS note

Phase 3 study will evaluate safety and efficacy of an investigational once-daily formulation of trientine tetrahydrochloride compared to D-penicillamine as first line treatment in patients with Wilson disease. Sept. 8, 2026 -- Orphalan Inc. today announced the launch of the TRADITiONAL Study, a global, multicenter Phase 3 clinical trial evaluating an investigational once-daily formulation of trientine tetrahydrochloride for the first line treatment of individuals with Wilson disease. The study

Weight-loss giant Eli Lilly joined Moonwalk Biosciences' latest funding round, which will be used to advance the biotech's adipose tissue–directed siRNA therapy into first-in-human studies for obesity in late 2027. In addition to the US pharma, the financing saw participation from Gaorong Ventures, ARCH Venture Partners, Khosla Ventures and Future Ventures. The oversubscribed $70-million series B was co-led by Alpha Wave Ventures and YK Bioventures. The round comes more than two and a half yea

Italian company the Menarini Group has agreed to pay Chinese insulin maker Gan & Lee Pharmaceuticals €62 million ($72 million) up front to collaborate on the registration and commercialization of Gan & Lee’s GLP-1 bofanglutide. Gan & Lee will also be eligible to receive milestone payments of up to €664 million ($771 million), as well as double-digit royalties on sales, according to a release . Menarini will take over regulatory submissions and commercialization in a total of 39 countries, incl

Following 100% transfusion independence in Chinese TDT patients, new study confirms tBE is equally safe and effective for African SCD patients and TDT patients from South/Southeast Asia. Sept. 7, 2026 -- On September 7, 2026, Shanghai, Cell Stem Cell published online clinical research from CorrectSequence Therapeutics (Correctseq) in collaboration with multiple institutions in a paper titled "Clinical base editing for β-hemoglobinopathies across different genetic backgrounds", demonstrating tha

HARBOR did not meet its primary endpoint of vHOT in DM1, a progressive neuromuscular disease with high unmet need and no approved treatment options Del-desiran showed evidence of clinical activity in secondary endpoints and exploratory analyses; full HARBOR dataset to be analyzed and appropriate development path to be determined AOC pipeline continues to advance with FDA priority review granted for delpacibart zotadirsen in DMD44 and planned FDA meeting for delpacibart braxlosiran in FSHD, based

Inhibrx CEO Mark Lappe was particularly keen on INBRX-106’s performance in patients with HPV-positive disease and sees a nearly $4 billion near-term opportunity thanks to the Phase 2 results. Patients with head and neck cancer who took Inhibrx Biosciences’ OX40 receptor agonist in combination with Merck’s Keytruda responded at a higher rate than those who took Keytruda alone in a Phase 2 trial, clearing the way for the biotech to access what executives estimate is a $50 billion-plus market. IN

- This trial marks the fourth Phase I peptide study for Ascletis this year. - ASC36 oral tablets achieved absolute oral bioavailability of 6% to 8% at steady state in non-human primate (NHP) studies utilizing Ascletis' proprietary Peptide Oral Transport ENhancement Technology (POTENT). - ASC36 oral tablets reduced mean body weight up to 13.2% from baseline after once-daily dosing for seven days in NHPs. - ASC36 subcutaneous (SQ) injection demonstrated approximately 32% and 91% greater relative b