CAMBRIDGE, Mass. - Fulcrum (LON:FMETF) Therapeutics, Inc. (NASDAQ:FULC), a clinical-stage biopharmaceutical company, announced today that it is suspending the development of its drug losmapimod, following the failure to meet primary and secondary endpoints in a Phase 3 trial. The REACH trial, which evaluated the efficacy of losmapimod in patients with facioscapulohumeral muscular dystrophy (FSHD), did not show significant improvement in patients as opposed to those who received a placebo.
The trial, which involved 260 patients, aimed to measure the change from baseline in reachable workspace (RWS (LON:RWS)), muscle fat infiltration (MFI), shoulder abductor strength, and patient-reported outcomes after 48 weeks of treatment. Despite the drug's safety and tolerability profile being consistent with previous studies, losmapimod did not achieve statistical significance in improving these measures.
Alex C. Sapir, President and CEO of Fulcrum, expressed disappointment at the trial's outcome, thanking patients, families, and the FSHD community for their support. The company plans a full evaluation of the data received this week and intends to present the findings at an upcoming medical meeting.
Fulcrum's financial position remains robust, with approximately $273.8 million in cash, cash equivalents, and marketable securities as of June 30, 2024. The company intends to reallocate resources to advance other programs, including pociredir for the treatment of sickle cell disease (SCD) and agents for Diamond-Blackfan Anemia (DBA), among other early discovery programs.
FSHD is a rare and progressive disease affecting skeletal muscles, leading to muscle wasting and functional decline. There are currently no approved treatments for FSHD, which affects an estimated 30,000 people in the United States.
Losmapimod, a selective p38α/β mitogen-activated protein kinase inhibitor, had been granted Fast Track designation and Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of FSHD. Although it had not been previously explored for muscular dystrophies, losmapimod had been evaluated in more than 3,600 subjects across various clinical trials for other indications.
This news is based on a press release statement from Fulcrum Therapeutics.
In other recent news, Fulcrum Therapeutics has seen a series of significant developments. BofA Securities upgraded the biotechnology company's stock from Underperform to Neutral, doubling the price target to $10.00. This revision comes as Fulcrum Therapeutics approaches a critical phase 3 readout for its drug candidate losmapimod, developed to treat facioscapulohumeral muscular dystrophy (FSHD). RBC Capital also maintained its Outperform rating on the company, increasing its price target from $14.00 to $15.00 based on anticipated results from the phase III REACH study of losmapimod.
Fulcrum Therapeutics has also made operational strides, including an exchange of common stock for pre-funded warrants with two institutional stockholders, RA Capital Healthcare Fund, L.P., and an unnamed entity. The company appointed Isabel Kalofonos as Chief Commercial Officer and Heather Faulds as Chief Regulatory Affairs & Quality Assurance Officer, roles expected to contribute to the submission of a New Drug Application for losmapimod.
Finally, Fulcrum Therapeutics is progressing with its Phase 3 REACH trial for losmapimod and anticipates reporting top-line data by the end of October 2024. In collaboration with Sanofi (EPA:SASY) (NASDAQ:SNY), the company is preparing for regulatory filings and the commercial launch of losmapimod outside the U.S. These recent developments highlight Fulcrum Therapeutics' commitment to advancing its clinical trials and preparing for the commercialization of losmapimod.
InvestingPro Insights
In the wake of the recent news regarding Fulcrum Therapeutics' suspension of its losmapimod drug development, the company's financials and market performance provide a broader context for investors. According to InvestingPro data, Fulcrum Therapeutics holds a market capitalization of $552.25 million, which is a significant indicator of the company's size and investor valuation. Despite the setback, analysts have revised their earnings upwards for the upcoming period, indicating a potential rebound or confidence in the company's other programs.
Moreover, Fulcrum's cash position appears solid, with the company holding more cash than debt on its balance sheet, which is reassuring for stakeholders considering the costs associated with halted drug development. This financial stability is further reflected in the company's liquidity, with liquid assets surpassing short-term obligations, suggesting Fulcrum is well-prepared to navigate the current challenge.
However, it's important to note that analysts do not expect Fulcrum to be profitable this year, and the stock price has shown considerable volatility. The company's revenue has seen an impressive growth rate of 2582.55% over the last twelve months as of Q2 2024, but this has not yet translated into profitability, as indicated by the negative operating income margin of -40.15% for the same period.
For investors looking for more in-depth analysis, there are additional InvestingPro Tips available for Fulcrum Therapeutics, which can provide further guidance on the company's prospects. These tips can be accessed through the dedicated InvestingPro platform, which offers a comprehensive suite of tools and insights for informed investment decisions.
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