The BioPharm Brief: Kidney Approval, Retinal Cell Therapy, and a Breast Cancer Trial Shift

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. We’re looking at a new FDA approval for a progressive kidney disease, a major acquisition in retinal cell therapy, and a shift in strategy for a late-stage breast cancer trial.

First, Alexion’s Ultomiris has received FDA accelerated approval to reduce protein in the urine in adults with I-g-A nephropathy who are at risk of disease progression. It is the first drug of its kind, a C5 complement inhibitor, approved for this use. In the phase 3 I CAN trial, Ultomiris reduced urine protein levels by 46.6% at week 34, compared with 5.6% for placebo. It is given by infusion every eight weeks after a loading dose. Because the approval is accelerated, it has not yet been shown that Ultomiris slows the decline in kidney function, and the ongoing trial is designed to confirm that benefit.

Next, Alcon has acquired Neurotech Pharmaceuticals for $550 million. The deal brings Alcon Encelto, the only FDA-approved treatment for macular telangiectasia type 2, or MacTel, a disease that gradually destroys central vision. The therapy is a small capsule of engineered cells, implanted in the eye, that continuously releases a protein designed to protect light-sensing cells in the retina. In two phase 3 trials with 228 participants, the treatment slowed the loss of those cells by 54.8% and 30.6% compared with a sham procedure. About 25,000 people in the United States have been diagnosed with MacTel.

And BioNTech and Bristol Myers Squibb plan to pair their bispecific antibody pumitamig with Gilead’s Trodelvy in a phase 3 trial in first-line triple-negative breast cancer. Pending regulatory approval, the antibody-drug conjugate will replace chemotherapy in the trial’s combination arm for patients whose tumors have low or no PD-L1. The change reflects Trodelvy’s approval as a first-line treatment in June, after it extended median progression-free survival to 9.7 months, compared with 6.9 months for chemotherapy. The trial plans to enroll 558 patients, and the safety and efficacy of the new combination have not yet been established.

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

Key takeaways

• Insulin supply resilience: Public funding is now tied directly to production and stockpiling obligations for Sanofi’s new Frankfurt insulin plant.
• Bispecific vs. standard of care: EA2251 will test ivonescimab against immunotherapy-based regimens, not chemotherapy alone, in first-line biliary tract cancer.
• Longer dosing intervals: TMB-365’s host-directed mechanism and receptor occupancy data support its development as a backbone for long-acting HIV regimens.