Amylyx Pharmaceuticals' ALS drug, Relyvrio, has failed in a Phase III trial, showing no efficacy against the primary and secondary endpoints. The drug, a combination of sodium phenylbutyrate and taurursodiol, had previously been approved by the FDA in September 2022 after a second advisory committee meeting. Patient advocacy groups played a role in pressuring the FDA to reverse its initial decision. The drug had a good safety profile but did not provide any therapeutic benefit to ALS patients. Amylyx will announce its plans for the drug in the coming weeks. The failure of Relyvrio highlights the importance of efficacy and safety in drug approvals, even in the face of unmet medical need [1a97b4e9].
In other news, Axsome Therapeutics announced positive results from its Phase 3 SYMPHONY trial, showing that its drug AXS-12 significantly reduced narcolepsy symptoms compared to a placebo. The trial enrolled 90 patients diagnosed with narcolepsy, who received either AXS-12 or a placebo over five weeks. AXS-12 met the primary endpoint, demonstrating a statistically significant reduction in weekly cataplexy attacks and achieving remission of cataplexy in 33% of patients. The drug also improved excessive daytime sleepiness and cognitive function. The most common adverse events were dry mouth, nausea, and constipation. AXS-12 has been granted Orphan Drug Designation by the FDA for the treatment of narcolepsy and is protected by patents until at least 2039 [3aeca2f6].
Following the positive Phase 3 narcolepsy data, Citi has maintained a Buy rating on Axsome Therapeutics and increased the price target to $127.00 from $125.00. The adjustment came after Axsome reported positive Phase 3 narcolepsy data. The stock experienced a 4% decline in Monday's trading due to a high placebo rate in the trial. Citi believes that if AXS-12 is approved, Axsome could utilize its existing Sunosi sales force for commercialization. The financial firm has increased the probability of success for AXS-12 to 80% from 50% and estimates the drug's potential U.S. peak sales at approximately $310 million [1fa76b55].
Axsome Therapeutics has launched a Phase 3 clinical trial named ENGAGE to evaluate the efficacy and safety of solriamfetol, a drug candidate for the treatment of binge eating disorder (BED) in adults. The trial will involve approximately 450 participants who will receive either solriamfetol or a placebo over a 12-week period. The primary goal of the trial is to observe changes in the frequency of binge eating episodes among patients. Solriamfetol has not yet received approval from the U.S. Food and Drug Administration (FDA) for the treatment of BED. BED is recognized as the most prevalent eating disorder in the United States, affecting an estimated 2.8% of adults, which translates to roughly 7 million people. Axsome Therapeutics is focused on the development of novel therapies for central nervous system (CNS) conditions that currently have limited treatment options [a295025a].