FibroGen’s DMD drug fails phase 3 trial, adding to clinical troubles

FibroGen continues to face challenges in its clinical endeavors, now extending into a second month of setbacks. After the phase 3 failure of Evrenzo, their oral anemia drug, which shattered hopes of entering the U.S. market, the biotech company has now encountered another blow as its Duchenne muscular dystrophy (DMD) candidate disappoints in a late-stage study.

FibroGen is recognized as a biotechnology firm renowned for its dedication to researching and developing cutting-edge treatments. Their primary objective is to tackle medical challenges that have yet to be adequately addressed, aiming to enhance the well-being of patients grappling with intricate diseases. FibroGen remains steadfast in progressing its portfolio by conducting ongoing investigations into conditions like idiopathic pulmonary fibrosis and pancreatic cancer. The company actively seeks out fresh prospects through acquisitions and the advancement of preclinical candidates. Despite encountering obstacles along the way, FibroGen persists in its pursuit of transformative therapies and advancements in the field of medicine.

Known as the LELANTOS-1 trial, the study involved 99 participants aged 12 years and older with non-ambulatory DMD. These individuals were randomly assigned to receive either pamrevlumab, the experimental drug, or a placebo in combination with systemic corticosteroids. Regrettably, the study failed to meet its primary endpoint, as assessed by a scoring system measuring the functionality of patients’ arms at Week 52.

In a relatively brief press release, FibroGen CEO Enrique Conterno expressed disappointment with the results but mentioned the company’s intention to share the data at a future medical conference, hoping to contribute to the understanding of this devastating disease. However, no mention was made regarding the potential impact of this failure on the broader pamrevlumab program. It is worth noting that FibroGen has another phase 3 trial for DMD, the LELANTOS-2 study, which involves 70 participants and is expected to release results in the third quarter of this year.

Apart from DMD, FibroGen has been exploring the use of pamrevlumab in idiopathic pulmonary fibrosis (IPF). In 2017, the drug was considered a potential blockbuster after phase 2 data showed promise as an anti-connective tissue growth factor antibody for the lucrative IPF market. At present, FibroGen is conducting two phase 3 studies, with anticipated outcomes for one expected in the third quarter of this year and the other in mid-2024.

Apart from their investigations into DMD and IPF, FibroGen is also exploring the use of pamrevlumab in the treatment of locally advanced unresectable pancreatic cancer. A phase 3 study specifically targeting this indication is expected to release results during the first half of the upcoming year.

Unfortunately, the recent news signifies that both of FibroGen’s leading assets have struggled to demonstrate their worth. While Evrenzo failed to convince U.S. regulators, it has gained approval in China. 

Last month, FibroGen acquired another clinical contender for its portfolio: a phase 1 antibody-drug conjugate for prostate cancer and multiple myeloma, obtained from Fortis. Looking ahead, FibroGen plans to seek FDA permission to initiate trials for two preclinical candidates—a promising anti-Gal9 antibody and an anti-CCR8 antibody—before the end of this year. Despite the numerous clinical avenues being explored, FibroGen faces significant challenges in validating the efficacy of its key assets in recent times.

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