FibroGen Initiates LELANTOS-2 – Second Phase 3 Clinical Study of Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy

Rhea-AI Summary

FibroGen initiated LELANTOS-2, a Phase 3 clinical trial of pamrevlumab for Duchenne muscular dystrophy (DMD). This global study will assess pamrevlumab's impact on muscle function in approximately 70 patients aged 6 to 12 over 52 weeks, comparing it to a placebo. The primary endpoint is measured using the North Star Ambulatory Assessment. The trial highlights FibroGen's commitment to addressing the unmet medical needs in DMD, a condition characterized by rapid progression and severe complications.

Positive

• Initiation of LELANTOS-2 Phase 3 trial demonstrates commitment to DMD treatment.
• Focus on muscle function assessment could provide valuable data for future therapies.

Negative

• None.

SAN FRANCISCO, March 16, 2021 (GLOBE NEWSWIRE) -- 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 Pamrevlumab
Pamrevlumab 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 Dystrophy
Duchenne 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.

About FibroGen
FibroGen, Inc. is a biopharmaceutical company committed to discovering, developing, and commercializing a pipeline of first-in-class therapeutics. The Company applies its pioneering expertise in hypoxia-inducible factor (HIF) and connective tissue growth factor (CTGF) biology to advance innovative medicines for the treatment of unmet needs. The Company is currently developing and commercializing roxadustat, an oral small molecule inhibitor of HIF prolyl hydroxylase activity, for anemia associated with chronic kidney disease (CKD). Roxadustat is also in clinical development for anemia associated with myelodysplastic syndromes (MDS) and for chemotherapy-induced anemia (CIA). Pamrevlumab, an anti-CTGF human monoclonal antibody, is in clinical development for the treatment of locally advanced unresectable pancreatic cancer (LAPC), Duchenne muscular dystrophy (DMD), and idiopathic pulmonary fibrosis (IPF). For more information, please visit www.fibrogen.com.

Forward-Looking Statements
This release contains forward-looking statements regarding our strategy, future plans and prospects, including statements regarding the development and commercialization of the company’s product candidates, the potential safety and efficacy profile of our product candidates, our clinical programs and regulatory events, and those of our partners. These forward-looking statements include, but are not limited to, statements about our plans, objectives, representations and contentions and are not historical facts and typically are identified by use of terms such as “may,” “will”, “should,” “on track,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue” and similar words, although some forward-looking statements are expressed differently. Our actual results may differ materially from those indicated in these forward-looking statements due to risks and uncertainties related to the continued progress and timing of our various programs, including the enrollment and results from ongoing and potential future clinical trials, and other matters that are described in our Annual Report on Form 10-K for the fiscal year ended December 31, 2020 filed with the Securities and Exchange Commission (SEC), including the risk factors set forth therein. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and we undertake no obligation to update any forward-looking statement in this press release, except as required by law.

Contacts:
FibroGen, Inc.

Investors:
Michael Tung, M.D.
Corporate Strategy / Investor Relations
1.415.978.1434
mtung@fibrogen.com

Media:
Jennifer Harrington
1.610.574.9196
Jennifer.Harrington@gcihealth.com 

FAQ

What is the purpose of the LELANTOS-2 trial?

The LELANTOS-2 trial aims to evaluate pamrevlumab's effect on muscle function in patients with Duchenne muscular dystrophy.

How many patients are involved in the LELANTOS-2 trial?

Approximately 70 patients aged 6 to 12 will participate in the LELANTOS-2 trial.

What are the main endpoints of the LELANTOS-2 trial?

The primary efficacy endpoint is the change in North Star Ambulatory Assessment (NSAA) from baseline to Week 52.

What is pamrevlumab?

Pamrevlumab is a first-in-class antibody developed by FibroGen designed to inhibit connective tissue growth factor (CTGF), which is involved in fibrotic disorders.

When was the LELANTOS-2 trial initiated?

The LELANTOS-2 trial was announced on March 16, 2021.