Pamrevlumab Misses Endpoints in Duchenne Muscular Dystrophy Study

Topline results were announced from a phase 3 study evaluating the efficacy and safety of pamrevlumab for the treatment of ambulatory patients with Duchenne muscular dystrophy (DMD) who are taking background corticosteroids.

Pamrevlumab is a first-in-class fully human antibody that works by inhibiting the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders.

The randomized, double-blind, LELANTOS-2 trial (ClinicalTrials.gov Identifier: NCT04632940) included 73 boys aged 6 to less than 12 years with ambulatory DMD. Study participants were randomly assigned to receive either pamrevlumab or placebo, in combination with systemic corticosteroids. 

Results showed that treatment with pamrevlumab did not meet the primary endpoint of change in the North Star Ambulatory Assessment (NSAA) total score from baseline to week 52 (placebo-corrected mean difference, -0.528 points; 95% CI, -2.308, 1.251; P =.5553). Moreover, the study missed secondary endpoints, including change from baseline to week 52 in 4-stair climb velocity, 10-meter walk/run test, time to stand, time to loss of ambulation, and proportion of patients with greater than 10 seconds in the 10-meter walk/run test.

The Company will determine next steps for the pamrevlumab program after evaluating the totality of the study data. Full study results from LELANTOS-2 will be shared at an upcoming medical forum.

“We are deeply disappointed that the LELANTOS-2 study did not meet its primary endpoint,” said Thane Wettig, Interim CEO, FibroGen. “We are grateful for the courageous efforts of patients, their caregivers, the advocacy community, and the trial investigators who have contributed to this important clinical study. We are committed to sharing all learnings from this trial with the Duchenne community and hope that there are insights that may help future efforts to develop treatments for this devastating disease.”

The FDA previously granted Fast Track and Orphan Drug designations to pamrevlumab for this indication.

This article originally appeared on MPR