Investors were jolted as Fulcrum Therapeutics, Inc. faced a sharp 61.1% decline in its stock value on Sept. 12. The tumble came on the heels of the company’s announcement of lackluster top-line data from the phase III REACH study, examining the efficacy of its drug candidate, losmapimod, for treating facioscapulohumeral muscular dystrophy (FSHD), a rare and debilitating ailment with no current approved therapies.
The study’s failure to meet its primary target – showing no improvement in relative surface area (RSA), crucial for reachable workspace (RWS), compared to a placebo after 48 weeks – sent shockwaves through the investor community.
Further compounding the disappointing news, the study fell short on secondary endpoints, leaving stakeholders with a bitter aftertaste. Notably, Fulcrum’s shares have plummeted by 49% year-to-date, significantly underperforming the industry.
Exploring Data from FULC’s REACH study
Details gleaned from the REACH study’s results revealed a marginal improvement of 0.013 in RSA for patients on losmapimod, compared to 0.010 in RWS for those on a placebo at the 48-week mark, precisely the primary goal of the study.
Moreover, patients receiving losmapimod experienced a 0.42% rise in muscle fat infiltration, eclipsed by the 0.57% observed in the placebo group. Importantly, losmapimod-treated patients exhibited a stellar 9.63% boost in abductor strength, dwarfing the 2.24% improvement in the placebo arm.
Unfortunately, the study showed no statistically significant variance in the secondary patient-reported outcomes, leading Fulcrum to halt further losmapimod development for FSHD.
Looking Ahead: Pipeline Development for FULC
Fulcrum now sets its sights on pociredir, another candidate in its roster. This early-stage asset targets sickle cell disease, an inherited blood disorder. Encouragingly, pociredir has received Fast Track and Orphan Drug designations from the FDA for SCD treatment.
Given the pressing medical need for SCD treatment, a successful development and subsequent approval of pociredir could serve as a beacon of hope, potentially reshaping Fulcrum’s future landscape.
With a healthy cash reserve of $273.8 million as of June 30, 2024, Fulcrum plans to harness these resources to advance pociredir’s clinical journey and bolster other pipeline initiatives.
Market Analysis & Future Considerations
Despite the recent setbacks, Fulcrum carries a Zacks Rank #1 (Strong Buy), reflecting confidence in its long-term potential.
Other notable players in the biotech realm include Illumina, Inc. and Krystal Biotech, Inc., both boasting a Zacks Rank #1 presently.
Market estimates for Illumina’s 2024 earnings per share have shown a positive trend, with substantial growth expected. Conversely, Krystal Biotech’s performance indicates a strong uptrend, positioning it as a noteworthy contender in the biotech sphere.
While Fulcrum navigates through the aftermath of the REACH study setback, all eyes are on its pipeline prospects and strategic moves as it charts a new course in the volatile biotech landscape.