Phase 3 trial of pamrevlumab misses mark for patients unable to walk

Data expected later this year from other trial in DMD patients who can walk

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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The LELANTOS-1 Phase 3 clinical trial, assessing pamrevlumab in combination with corticosteroids in people with Duchenne muscular dystrophy (DMD) who are no longer able to walk, did not reach its main goal.

FibroGen, the therapy’s developer, announced the top-line results from the trial, whose main objective was to assess changes in upper limb function after 52 weeks (one year) compared to the study’s start.

Pamrevlumab was generally safe and well-tolerated. Most treatment-related emergent adverse events were mild or moderate.

FibroGen plans to present full results at upcoming meetings and publish them in scientific journals.

“While disappointed with these results, we look forward to sharing the data at a future medical conference to contribute towards the understanding of this devastating disease,” Enrique Conterno, CEO of FibroGen, said in a company press release. “FibroGen would like to thank the patients, caregivers and clinical trial investigators for their dedication in participating in this study.”

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The LELANTOS-1 trial (NCT04371666), which began enrolling in 2020, randomly assigned 99 patients to receive pamrevlumab or a placebo, along with oral corticosteroids. Pamrevlumab was delivered by infusion into the bloodstream every two weeks for up to one year. The therapy was tested at a dose of 35 mg/kg.

Changes in motor function were assessed using the total score of the performance of upper limb (PUL) 2.0, a measure of upper limb function initially developed for DMD. Additional goals included assessments of lung and heart health.

A second Phase 3 trial, called LELANTOS-2 study (NCT04632940) is testing pamrevlumab in combination with corticosteroids in DMD patients who have maintained their ability to walk. The trial is underway at multiple clinical sites across the U.S., Europe, Australia, Canada, and China.

Top-line results from LELANTOS-2 are expected some time from July to September.

Pamrevlumab is designed to target the connective tissue growth factor (CTGF), a pro-inflammatory and pro-fibrotic (scarring) signaling protein. CTGF is found at unusually high levels in the muscles of people with DMD. Abnormal scarring leads to muscle weakness and organ impairment.

The therapy was granted fast track, orphan drug, and rare pediatric disease designations by the U.S. Food and Drug Administration to support its clinical development.