Takeda Fails Key Epilepsy Trials, Considers Market Path for Ovid Partnered Drug

Takeda's late-phase epilepsy program recently faced significant setbacks, with pivotal trials in two key indications failing to meet their primary endpoints. Despite these challenges, the Japanese pharmaceutical giant remains committed to seeking regulatory guidance based on the comprehensive data gathered from the trials. The drug at the center of this effort is soticlestat, a cholesterol 24-hydroxylase inhibitor developed in partnership with Ovid Therapeutics.

Soticlestat is crucial to Takeda's strategic plans, particularly because the company's neuroscience division currently lacks a product ready for market launch. With few other phase 3 trial results expected soon, successfully bringing soticlestat to market is seen as vital for jumpstarting the company's sales. Takeda had previously invested $196 million in Ovid in 2021 to support the development of this candidate.

However, the phase 3 trials did not deliver the definitive success Takeda had hoped for. One trial focused on patients with refractory Lennox-Gastaut syndrome, a severe epilepsy type, failed to meet its primary endpoint. This endpoint measured the change in Major Motor Drop seizure frequency from baseline compared to a placebo.

Similarly, a phase 3 trial involving patients with refractory Dravet syndrome, another form of epilepsy, also missed its primary endpoint. The trial aimed to assess the reduction in convulsive seizure frequency from baseline compared to placebo. While this study did not meet the primary endpoint, with a p-value of 0.06, it did achieve several secondary objectives. These included significant changes in the responder rate, caregiver and clinician global impression of improvement, and seizure intensity and duration scales over the 16-week treatment period.

Sarah Sheikh, head of Takeda’s neuroscience therapeutic area unit, highlighted the unmet medical needs in these epilepsy indications and discussed the future steps for soticlestat. “While we would have wished for more declarative results on the primary endpoints, we are encouraged by positive outcomes seen in the totality of the data and are looking forward to engaging health authorities to determine the best path forward,” she stated.

Ovid Therapeutics also released a statement in response to the trial results. The company underscored how the upfront payment from Takeda had supported the development of its pipeline, which includes two promising clinical candidates. Ovid emphasized its commitment to pursuing its programs with financial discipline and noted that its cash reserves are projected to last until the first half of 2026. The biotech firm aims to achieve several clinical milestones before its funding runs out.

Despite the optimistic outlook from both companies, investors reacted negatively. Shares in Ovid plummeted by more than 60%, reaching $1.20 in premarket trading. The original deal between Ovid and Takeda included up to $660 million in milestone payments contingent on the successful development, authorization, and commercialization of soticlestat.

In summary, while the recent phase 3 trial results for soticlestat were disappointing, Takeda and Ovid are focusing on the broader dataset and preparing to discuss the next steps with regulators. The outcome of these discussions will be crucial in determining the future of soticlestat and its potential impact on Takeda’s neuroscience portfolio.