The BioPharm Brief: Weight Loss Head-to-Head, Precision Cell Therapy, and Individualized Gene Editing

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. Today, we’re looking at a head-to-head win in type 2 diabetes, a more targeted path for cell therapy in lung cancer, and new FDA guidance on gene editing therapies built for individual patients.
First, Eli Lilly’s retatrutide has more than doubled the weight loss of Ozempic in the first phase 3 trial to compare it with another incretin medicine. Retatrutide acts on three hormone receptors involved in blood sugar and appetite: GIP, GLP-1, and glucagon. In the 80-week TRANSCEND-T2D-2 trial of nearly 1,300 adults with type 2 diabetes, participants on retatrutide lost up to 20.9% of their body weight, compared with 9.4% on Ozempic, and saw greater reductions in blood sugar. More participants on retatrutide stopped treatment because of side effects. Retatrutide is not yet approved.

Next, Iovance’s lifileucel has reached a 52% response rate in a biomarker-selected group of patients with advanced lung cancer. Lifileucel is a cell therapy made from immune cells taken from a patient’s own tumor, expanded in the lab, and infused back. In the phase 2 IOV-LUN-202 trial, about a quarter of all patients responded. But among those whose tumors lacked mutations in the KRAS and HER2 genes, 52% responded. Iovance plans a new biomarker-selected cohort starting in November and a regulatory filing in 2027. The subgroup findings are retrospective and based on small numbers.

And FDA has issued draft guidance on licensing individualized genome editing products for rare genetic diseases. These therapies can be tailored to a patient’s specific mutation. The guidance describes how a single Biologics License Application could cover multiple patient-specific versions of a product, within predefined boundaries. After approval, new versions that fall within those boundaries could be added based largely on laboratory data rather than new clinical trials. FDA is accepting public comments through November 27.

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

Key takeaways

• Head-to-head win: Retatrutide produced up to 20.9% weight loss vs 9.4% with Ozempic in adults with type 2 diabetes.
• Precision cell therapy: Lifileucel’s response rate rose to 52% in patients with KRAS and HER2 wild-type lung cancer.
• Individualized editing: FDA’s draft guidance outlines how one BLA could cover multiple patient-specific genome editing products.