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FibroGen’s Duchenne drug misses mark in first large trial

2023-06-07

临床3期并购基因疗法临床结果

Dive Brief:

FibroGen once again has disappointing news to share from a clinical trial, this time from a study of an experimental treatment for Duchenne muscular dystrophy.

The Phase 3 trial included 99 people whose disease had progressed so much that they could no longer walk. Patients received treatment with corticosteroids and either a placebo or FibroGen’s drug, pamrevlumab. In the end, researchers found no significant benefit for pamrevlumab on the primary endpoint, a measure of upper limb function, FibroGen said Wednesday.

Still, the company said the drug was generally safe and well tolerated, with mostly mild or moderate side effects. That’s important because a different trial of pamrevlumab in patients who can still walk is continuing and due to report results in the third quarter.

Dive Insight:

While a study failure is never good news, expectations for this particular trial weren’t high. Past research on treatments for the debilitating condition suggests that interventions earlier in the progression of the disease have a better chance because muscles get weaker over time and less receptive to therapy.

Success with these late-stage patients — who were at least 12 years old — would have given FibroGen a significant foothold in the market. By contrast, a gene therapy developed by Sarepta Therapeutics could be initially approved later this month for patients 4 to 5 years of age, though use may be expanded if an ongoing study is successful.

FibroGen is trying to rally from a difficult few years. The company suffered a major blow in August 2021, when the Food and Drug Administration rejected an anemia pill called roxadustat. Afterward, FibroGen restructured and slashed jobs. Though the pill did win approval internationally, the company recently had another setback with a study failure in May.

The San Francisco biotech’s stock, which traded above $55 in early 2021, has never fully recovered. The company’s shares dropped 9%, below $17 apiece, early Wednesday.

Still, FibroGen has a chance to reverse its fortunes with the upcoming release of results from a Phase 3 study of pamrevlumab in idiopathic pulmonary fibrosis. Success treating that condition would open a market with “meaningfully higher value,” Stifel analyst Annabel Samimy wrote in a June 1 note to investors.

The company has also engaged in some dealmaking, last month licensing an experimental cancer drug from the startup Fortis Therapeutics along with an option to acquire the company altogether.

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