The BioPharm Brief: A Survival Win, a Myeloma Filing, and Gene Therapy for Swallowing

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. We’re looking at a major survival result in head and neck cancer, a new filing in multiple myeloma, and early data for a gene therapy that targets a rare swallowing disorder.

First, Genmab’s petosemtamab plus pembrolizumab has cut the risk of death by 50% in a phase 3 trial in head and neck cancer. Petosemtamab is a bispecific antibody that targets two proteins on tumor cells, E-G-F-R and L-G-R-5. In the LiGeR-HN1 trial, patients with newly treated recurrent or metastatic PD-L1-positive disease received the combination or pembrolizumab alone. At the first interim analysis, the trial met both primary endpoints, overall survival and response rate, and the benefit held regardless of HPV status. Genmab has not yet released median survival figures and plans to present full results at a future medical meeting.

Next, AbbVie has submitted a Biologics License Application to the FDA for etentamig in adults with relapsed or refractory multiple myeloma who have received at least two prior lines of therapy. Etentamig is a bispecific T-cell engager that directs the immune system against B-C-M-A, a protein found on myeloma cells. In the phase 3 CERVINO trial of 393 patients, it reduced the risk of disease progression or death by 60% compared with standard therapies, and 74% of patients responded. It is given once a month after a single step-up dose. Cytokine release syndrome occurred in about 28% of patients, and all cases were mild to moderate.

And Benitec Biopharma has reported interim data for BB-301, a gene therapy for swallowing difficulties caused by oculopharyngeal muscular dystrophy, or OPMD. This rare inherited disorder weakens the throat muscles, and nearly all patients develop trouble swallowing. BB-301 is injected directly into the throat muscles, where it silences the faulty gene and supplies a working copy. In the phase 1b/2a study, all nine treated patients met the company’s criteria for response, and no serious side effects have been reported. The study has no control group, and Benitec plans a pivotal trial for mid-2027.

That’s today’s BioPharm Brief. For analysis and expert insights, please visit BioPharmInternational.com.

Key takeaways

• Head and neck cancer: Petosemtamab plus pembrolizumab halved the risk of death versus pembrolizumab alone in the phase 3 LiGeR-HN1 trial.
• Myeloma filing: AbbVie’s etentamig BLA is supported by a 60% reduction in the risk of progression or death in the phase 3 CERVINO trial.
• Rare disease gene therapy: All nine patients treated with BB-301 met response criteria for OPMD-related dysphagia in early data.