FibroGen (FGEN) Begins LELANTOS-2, a Second Phase 3 Clinical Study of Pamrevlumab for Treatment of DMD

March 16, 2021 7:08 AM EDT

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FibroGen, Inc. (NASDAQ: FGEN) announced the initiation of LELANTOS-2, a Phase 3, randomized, double-blind, placebo-controlled trial of pamrevlumab or placebo in combination with systemic corticosteroids in patients with ambulatory Duchenne muscular dystrophy (DMD).

The primary objective of this global study is to evaluate the effect of pamrevlumab on muscle function in patients with DMD. Approximately 70 patients ages 6 to 12 years will be randomized 1:1 to receive pamrevlumab plus systemic corticosteroids, or placebo plus systemic corticosteroids every two weeks, for up to 52 weeks. The primary efficacy endpoint is ambulatory function assessment, measured by the change in North Star Ambulatory Assessment (NSAA) from baseline to Week 52. Additional secondary endpoints will be assessed in the study. Subjects who complete the 52-week study will be eligible for rollover into an open-label extension study with pamrevlumab and systemic corticosteroids. For more information about LELANTOS-2 please visit www.clinicaltrials.gov (NCT04632940).

“Duchenne, the most common form of muscular dystrophy in children, is typically diagnosed by 2-6 years of age and progresses rapidly, resulting in the loss of independent motor function by the early teenage years,” said Elias Kouchakji, M.D., Senior Vice President, Clinical Development, Drug Safety, and Pharmacovigilance, FibroGen. “The initiation of LELANTOS-2, the second Phase 3 clinical trial in the pamrevlumab clinical development program, underscores our commitment to delivering a meaningful treatment for DMD, an area with high unmet medical need.”

Pamrevlumab is a first-in-class antibody developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Importantly, skeletal muscle from DMD patients shows elevated levels of CTGF, and a major manifestation of DMD is cardiac fibrosis.

“The initiation of our second Phase 3 study of pamrevlumab for DMD furthers our research on the clinical benefits of inhibiting connective tissue growth factor, an important biological mediator in fibrotic and proliferative disorders,” said Mark Eisner, M.D, M.P.H, Chief Medical Officer, FibroGen. “We are committed to advancing the science of CTGF biology and evaluating clinical benefit in diverse diseases with unmet medical need, including idiopathic pulmonary fibrosis, locally advanced unresectable pancreatic cancer and Duchenne muscular dystrophy.”

About PamrevlumabPamrevlumab is a first-in-class antibody developed by FibroGen that inhibits the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders. Pamrevlumab is in Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF), locally advanced unresectable pancreatic cancer (LAPC), and Duchenne muscular dystrophy (DMD). For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.

About Duchenne Muscular DystrophyDuchenne muscular dystrophy is a rare and debilitating neuromuscular disease that affects approximately 1 in every 5,000 newborn boys. The fatal disease is caused by a genetic mutation leading to the absence or defect of dystrophin, a protein necessary for normal muscle function. The absence of dystrophin results in muscle weakness, muscle loss, fibrosis, and inflammation. Patients with DMD are often wheelchair-bound before the age of 12, and their progressive muscle weakness may lead to serious medical problems relating to respiratory and cardiac muscle.



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