Shares of Fulcrum Therapeutics, Inc. (FULC) took a nosedive on September 12th, plummeting by a staggering 61.1%. This dramatic drop was triggered by disappointing top-line data from the company’s phase III REACH study, which evaluated its drug candidate, losmapimod, for treating patients with facioscapulohumeral muscular dystrophy (FSHD), a rare and debilitating disease. The study’s primary endpoint, demonstrating a change from baseline in relative surface area (RSA), a measure of reachable workspace (RWS), compared to placebo at week 48, was not met. Furthermore, the study failed to achieve statistical significance on its secondary endpoints. The disappointment is amplified by the fact that there are currently no approved treatments for FSHD. This news significantly impacted Fulcrum’s overall performance, with shares plunging 49% year to date, significantly exceeding the industry’s decline of 0.8%.
The REACH study showed a marginal improvement in RSA with losmapimod treatment compared to placebo, but this difference was not statistically significant. Additionally, while patients receiving losmapimod demonstrated improvement in muscle strength, the results were not statistically significant compared to the placebo group. This ultimately led Fulcrum to halt further development of losmapimod for FSHD.
However, Fulcrum is not giving up on its pipeline development. The company is currently focusing its efforts on another promising candidate, pociredir. Pociredir is being evaluated in an early-stage study (phase Ib PIONEER study) for treating patients with sickle cell disease (SCD), a genetic blood disorder. Pociredir has already received a Fast Track designation and Orphan Drug designation from the FDA for treating SCD, highlighting its potential significance in addressing this critical unmet medical need. If successful, pociredir could become a game-changer for Fulcrum, boosting its prospects in the coming years.
Fulcrum is in a strong financial position, holding $273.8 million in cash, cash equivalents, and marketable securities as of June 30, 2024. This financial cushion will allow the company to advance pociredir in the treatment of SCD and further invest in other pipeline development activities.
Despite the setback with losmapimod, Fulcrum remains a compelling stock to watch. The company’s strong financial position and focus on addressing a significant unmet medical need with pociredir make it a potential investment opportunity. It’s important to note that investing in any company, particularly in the biotech sector, comes with inherent risks, and conducting thorough research is crucial before making any investment decisions.
