Lilly granted one person extraordinary access to its next-gen drug

Eli Lilly and the FDA have allowed one person to receive the pharma company's highly promising obesity candidate through the agency's “compassionate use” program, my colleague Lizzy Lawrence exclusively reports.

This person was a 79-year-old man at the time the request was made in April, three sources familiar with the matter said. STAT could not determine the identity of the person, but sources said the application drew the interest of top health officials, suggesting the person was well connected.

The treatment, retatrutide, has shown potential to lead to bariatric-surgery levels of weight loss. It's been dubbed the “triple-G” drug since it targets three hormones — GLP-1, GIP and glucagon.

The case is notable since the compassionate use program is typically used by patients with immediately life-threatening issues to access last-resort treatments. Experts also questioned why Lilly would grant this access to a single person when obesity is such a widespread condition and drugmakers often establish compassionate use programs for large cohorts.

gene therapy

All in a day's work: a licensing, a financing, and a reverse merger

We have another exclusive story — a new startup, Serapha Bio, launched today to develop a gene-editing therapy for a liver and lung disease called Alpha-1 Antitrypsin Deficiency, or AATD.

To create the company, founding investors RA Capital and RTW Investments went through multiple hoops — licensing the drug from a Chinese company, arranging $230 million in funding, and executing a reverse merger with Boundless Bio.

It was like “landing a whole bunch of planes at the same time,” one of the investors said.

Read more from STAT's Allison DeAngelis.

oncology

Pfizer's lung cancer drug disappoints

Pfizer's experimental drug for one the most common forms of lung cancer fell short in a clinical trial, serving a big blow to the company, which had positioned the treatment as a key growth driver.

In the Phase 3 study, the drug, called sigvotatug vedotin, did not lead to a statistically significant improvement in overall survival over a chemotherapy called docetaxel.

Pfizer acquired the therapy when it bought Seagen for $43 billion in 2023.

regulation

FDA launches pilot aimed at speeding up early trials

Officials yesterday announced a pilot program designed to speed up early-stage clinical trials and reduce development timelines by six to 12 months. The hope is to incentivize U.S.-based trials and combat Chinese competition.

The FDA will issue guidance reaffirming that a single, high-quality Phase 3 trial supported by confirmatory evidence is enough to allow drug approval. The agency also plans to release guidance on dose selection, support for program participants aimed at accelerating first-in-human studies, and clarity on approval pathways.

Read more from STAT's Chelsea Cirruzzo and Lizzy Lawrence.

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