Bristol Results Show Promise in Rare Cancer, Lung Disease

Bristol Myers Squibb Co.’s experimental drugs for treating a rare endocrine cancer and a deadly form of lung disease showed promise in late-stage studies, a potential boon for the pharmaceutical company’s new product pipeline.

Wall Street has been eagerly anticipating both results, as the drugs use novel approaches for hard-to-treat conditions that impair patients’ quality of life and are eventually fatal. They could also help Bristol offset revenue losses from looming patent expirations for some of its most important medicines.

The company said patients with idiopathic pulmonary fibrosis, a chronic condition that scars the lungs and makes it difficult to get oxygen into the bloodstream, had their breathing significantly improve after getting a lower dose of the company’s pill, called admilparant. Patients given a higher dose didn’t fare definitively better than those on placebo in the study from the third and final phase of testing required for a new drug approval.

A planned interim analysis of a late-stage study in patients with gastroenteropancreatic neuroendocrine tumors, a rare type of cancer that starts in the endocrine cells of the gastrointestinal tract or the pancreas, found the company’s RYZ101 significantly slowed progression of the disease. Bristol bought the medicine’s developer, RayzeBio, for $3.7 billion in 2024.

Bristol Myers shares gained 2% at 9:31 a.m. Monday in New York, while the S&P 500 Index was little changed.

The lung disease pill admilparant works in a new and highly anticipated area of drug discovery, though its mixed results with a difference in response between the low and high doses requires further analysis. It blocks the receptor for lysophosphatidic acid, which is linked to inflammation and scarring. Bristol has said it could be the first in its class to treat pulmonary fibrosis, a chronic disease that scars the lungs and can lead to death in three to five years.

“The trial meeting its primary endpoint could be sufficient to support admilparant regulatory approval, but the lack of dose response raises questions about its profile,” wrote David Risinger, an analyst at Leerink Partners. “We await detailed results.”

Bristol plans to discuss the trial data with regulators who would consider approval of the drug, and will present the findings at a future medical meeting, the company said. The medicine is also in the final stage of study for treating progressive pulmonary fibrosis, a related condition, with initial results expected next year.

“IPF remains one of the most complex and challenging lung diseases to treat, with unpredictable progression, irreversible scarring and a profound impact on patients’ daily lives,” said Fernando Martinez, a professor of medicine at UMass Chan Medical School, who led the steering committee for Bristol’s admilparant program known as ALOFT. He called the results “important advancement” for treatment of the condition.

The cancer study found RYZ101, an advanced form of targeted radiation that’s delivered directly to tumors, slowed progression of the disease in patients who had worsened after previously getting a different form of radiation. The US Food and Drug Administration granted the medicine an orphan drug designation in 2026 based on the unmet medical need for patients with the rare tumors.