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Takeda drug for rare types of epilepsy misses goal in late-stage trial

Dive Brief:

  • An experimental pill developed by Takeda to treat the rare epilepsies Dravet and Lennox-Gastaut syndromes missed the main goals of two Phase 3 trials, failing to reduce the frequency of seizures after four months of treatment, the company said Monday.
  • Takeda said the drug, called soticlestat, helped on some secondary effectiveness measures in Dravet syndrome, and in both disorders some groups of patients fared better than others. The Japan-based drugmaker plans on discussing data from the trials with the Food and Drug Administration and other regulators to determine if there is a path forward for the therapy.
  • The data were also negative for neuroscience-focused biotech Ovid Therapeutics, which conducted early- and mid-stage development on soticlestat in partnership with Takeda. Shares in the New York-based company fell by more than two-thirds in Monday morning trading as the company could potentially miss out on hundreds of millions of dollars of milestone payments.

Dive Insight:

Soticlestat looked promising in mid-stage studies, helping to significantly reduce seizures in Dravet syndrome and showing signs it could help in Lennox-Gastaut. The data were enough to persuade Takeda to take full rights to the drug — discovered in its own laboratories — back from Ovid for $200 million upfront and as much as $856 million more in milestone payouts.

Many of those milestones now look in doubt. In its own announcement of the news, Ovid said it had used the upfront payment to build a “differentiated pipeline with novel programs,” and had sufficient cash to continue operations through early 2026.

“Our R&D and financial strategy is independent of soticlestat’s outcome,” said Jeremy Levin, Ovid’s CEO, in a statement.

The company had $90.3 million in cash, equivalents and marketable securities as of March 31. With Monday’s share losses, the company’s market capitalization fell to about $75 million.

Takeda, meanwhile, is in the midst of a restructuring that aims to improve its core operating profit by 1.5% to 2% a year, following a steep decline in earnings. Among other initiatives, company has cut 18 early and mid-stage drugs from its pipeline.

Soticlestat was the only Phase 3 neuroscience drug in Takeda’s pipeline, but it appears the company retains interest in the sector. It still lists five Phase 2 drugs and one in a Phase 1 trial.

Dravet syndrome patients have seen an increased number of specialized drug treatments in recent years, with the FDA approval of the cannabinoid drug Epidiolex and another product called Diacomit in 2018, and a version of the weight loss drug fenfluramine, called Fintepla, in 2020. Both Epidiolex and Fintepla are also approved for Lennox-Gastaut syndrome.