FibroGen’s Tough Luck Persists: Phase III DMD Trial Disappointment Strikes Again

“FibroGen’s Bumpy Ride Continues: Setback in Phase III DMD Trial Sends Stocks Tumbling”

In a disheartening blow, FibroGen unveiled disappointing results on Tuesday, revealing that their highly anticipated investigational antibody fell short of its primary efficacy goal in the Phase III LELANTOS-2 trial for Duchenne muscular dystrophy (DMD). This unexpected stumble triggered an after-hours plunge in the company’s stock, reflecting the market’s swift reaction to the news.

LELANTOS-2, a trial cloaked in randomization and double-blinds, enrolled 73 boys grappling with ambulatory Duchenne muscular dystrophy (DMD). These participants were split into two groups, either receiving the promising pamrevlumab or a placebo, all while undergoing background corticosteroid treatment. The study’s focal point was the change in the North Star Ambulatory Assessment (NSAA) score over a span of 52 weeks, a metric gauging functional motor abilities in DMD-afflicted children.

Alas, the anticipated uplift in the NSAA score didn’t materialize, as pamrevlumab failed to demonstrate a statistically significant improvement compared to the placebo group. Regrettably, the setbacks extended beyond the primary endpoint, with pamrevlumab also missing the mark on secondary endpoints such as stair climb velocity, walk/run tests, standing time, and time to loss of ambulation.

A glimmer of solace could be found in the safety realm, with LELANTOS-2 deeming pamrevlumab generally well-tolerated. Most side effects were mild or moderate in severity. However, a slightly higher frequency of treatment-emergent serious adverse events emerged in the pamrevlumab group compared to the placebo arm.

As FibroGen confronts this unexpected twist, the company’s course of action hinges on the forthcoming analysis of LELANTOS-2’s data. Additionally, the full study results are poised to make their debut at an upcoming medical congress, adding an element of anticipation to the horizon.

This Phase III setback arrives on the heels of a challenging second quarter for FibroGen. The blows commenced in June 2023, as pamrevlumab faced consecutive defeats, first in DMD and subsequently in idiopathic pulmonary fibrosis.

Notably, pamrevlumab was once heralded by CEO Thane Wettig as a cornerstone of FibroGen’s strategic framework, intended to optimize value for stakeholders. Back then, the antibody was on the cusp of three pivotal late-stage readouts. Now, the spotlight narrows down to two trials focused on pancreatic cancer: the Phase III LAPIS study and the Phase II/III Precision Promise Platform Study.

Amidst the volatility surrounding pamrevlumab, FibroGen’s other key contender roxadustat, a pioneering small molecule inhibitor targeting the HIF-PH protein, is carving its own path. Despite facing a rocky road with the FDA, roxadustat’s strides in China shone bright, generating a 44% surge in revenue to $76.4 million during the second quarter.

FibroGen’s journey isn’t limited to these two stars, as the company harnesses its early-stage oncology pipeline and robust financial standing to navigate the currents well into 2026. The evolving saga of this biotech enterprise unfolds with a mix of challenges and aspirations, showcasing the resilience that defines the realm of medical innovation.

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