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Ersodetug missed its phase 3 primary endpoint in congenital HI, but FDA is reviewing CGM data showing 50%+ hypoglycemia reductions.

Amgen and AstraZeneca announced that the Phase 3 DeLLphi-305 trial met its primary endpoint, with Imdelltra (tarlatamab) plus Imfinzi (durvalumab) significantly improving overall survival versus durvalumab alone as first-line maintenance therapy for extensive-stage small cell lung cancer — the first phase 3 study of a bispecific T-cell engager to show a survival benefit in this earlier-line setting.

Del-desiran missed its primary endpoint in a phase 3 DM1 trial, though secondary measures showed signs of clinical activity.

HHS announced four senior FDA leadership selections, including Karim Mikhail as permanent Director of the Center for Biologics Evaluation and Research and Michael Davis as Director of the Center for Drug Evaluation and Research, capping more than a year of turnover at both centers.

BrainChild Bio, Moonwalk Biosciences, and Aptar Pharma are advancing new approaches to CAR-T therapy, RNAi for obesity, and inhaled antisense oligonucleotide delivery.

Aptar Pharma's Nanopharm business will lead inhaled formulation development and device assessment for ACT-101, Aceso Therapeutics' antisense oligonucleotide candidate designed to correct CFTR protein function in cystic fibrosis patients carrying the F508del mutation.

BrainChild Bio's BCB-276 extended survival to 19.8 months from diagnosis in phase 1, backing its $116 million-funded pivotal DIPG trial.

Moonwalk's $70 million new funding advances MW101, an adipose-targeted siRNA aiming to cut fat while preserving muscle in obesity patients.

Bristol Myers Squibb reported positive topline phase 2 results for arlocabtagene autoleucel, a potential first-in-class GPRC5D-directed CAR T cell therapy, in patients with quadruple-class exposed relapsed and refractory multiple myeloma who had already received a prior BCMA-targeted therapy.

Experts Harpreet Singh and Steven Quay discuss FDA expedited regulatory pathways for biologics, including Fast Track, Breakthrough Therapy, RMAT, Real-Time Oncology Review, and the National Priority Voucher Pilot Program, and explain how these mechanisms can accelerate development without lowering evidentiary standards.

Acepodia's ACE723 delivers 2 cytotoxic payloads to GPC3-positive tumors, clearing FDA review to begin trials in liver cancer.

Cipla's US subsidiary secured exclusive rights to commercialize QL2107, Qilu Pharmaceutical's phase 3 biosimilar candidate to Keytruda, ahead of the reference biologic's anticipated 2028 patent expiration.

Medicus Pharma secured worldwide rights to PF-08046031 (CD228V), an antibody-drug conjugate targeting melanotransferrin that Pfizer discontinued earlier this year following its $43 billion Seagen acquisition, in a co-development and license agreement that could pay Pfizer more than $1 billion in milestones.