Takeda epilepsy drug fails in late-stage trial

Takeda, a pharmaceutical company based in Japan, has announced that its experimental pill, soticlestat, aimed at treating the rare epilepsies Dravet and Lennox-Gastaut syndromes, did not meet the main goals in two Phase 3 clinical trials. The trials were designed to evaluate the effectiveness of the drug in reducing seizure frequency over a period of four months, but the results were disappointing as the primary objectives were not achieved.

Despite the setback, Takeda reported that soticlestat did show some positive effects on secondary measures in Dravet syndrome and noted variability in patient responses across both disorders. The company plans to consult with the Food and Drug Administration (FDA) and other regulatory bodies to explore potential future steps for the therapy.

This development also impacts Ovid Therapeutics, a neuroscience-focused biotech company based in New York, which originally partnered with Takeda to conduct early- and mid-stage development of soticlestat. Following the trial results, Ovid's shares plummeted by over two-thirds, raising concerns about the company potentially missing out on substantial milestone payments.

Earlier research on soticlestat had shown promise, significantly reducing seizures in Dravet syndrome during mid-stage studies and indicating potential benefits for Lennox-Gastaut syndrome. These encouraging results led Takeda to reclaim full rights to the drug from Ovid for an upfront payment of $200 million, with the possibility of additional milestone payments totaling up to $856 million. However, the likelihood of achieving many of these milestones is now uncertain.

In response to the trial outcomes, Ovid emphasized that its research and development, as well as its financial strategy, do not solely depend on the success of soticlestat. CEO Jeremy Levin stated that the company had utilized the upfront payment to develop a diverse pipeline with innovative programs and assured stakeholders that Ovid had sufficient funds to continue operations through early 2026. As of March 31, the company reported having $90.3 million in cash, equivalents, and marketable securities. However, the significant drop in share value reduced Ovid's market capitalization to approximately $75 million.

Meanwhile, Takeda is undergoing a restructuring process aimed at enhancing its core operating profit by 1.5% to 2% annually, following a notable decline in earnings. As part of this initiative, Takeda has removed 18 early and mid-stage drugs from its pipeline. Despite the setback with soticlestat, the company maintains an interest in the neuroscience sector, with five drugs in Phase 2 trials and one in a Phase 1 trial remaining in its pipeline.

Dravet syndrome patients have seen more treatment options in recent years, including the FDA approval of the cannabinoid drug Epidiolex and Diacomit in 2018, and the weight loss drug fenfluramine, rebranded as Fintepla, in 2020. Both Epidiolex and Fintepla are also approved treatments for Lennox-Gastaut syndrome, providing additional therapeutic options for patients with these challenging conditions.