Takeda’s enzyme therapy soticlestat, which aims to balance brain cholesterol and reduce seizures, failed two Phase 3 trials in certain forms of epilepsy.
The double whammy throws cold water on potential approvability, as the Japanese drugmaker had planned to submit soticlestat to regulators by the end of this fiscal year.
The flops also impact Ovid Therapeutics, a struggling biotech that had received nearly $200 million upfront in 2021 for the drug and about another $660 million on the line in biobucks. Ovid’s shares $OVID were down by as much as 59% before Monday’s opening bell. Takeda’s shares $TAK were down less than 1%.
In a separate press release, Ovid said its “R&D and financial strategy is independent of soticlestat’s outcome.”
Takeda said the drug “narrowly missed,” at a p-value of 0.06, the primary endpoint of reducing convulsive seizure frequency versus placebo in patients with refractory Dravet syndrome. Meanwhile, in a trial of patients with refractory Lennox-Gastaut syndrome, the drug failed to reduce the so-called major motor drop seizure frequency versus placebo.
In the two studies, dubbed SKYLINE and SKYWAY, respectively, Takeda said it saw “nominally significant treatment effects” on caregiver and clinician assessments of improvement and seizure intensity, among other endpoints.
The drugmaker said the experimental medicine was “generally well-tolerated.” Despite the misses, Takeda said it will discuss the full data with regulatory authorities to figure out the program’s next steps. Results from both trials will be presented at undisclosed scientific conferences as well.
Sarah Sheikh“Even with currently available therapies, we know that many patients with developmental encephalopathies like DS and LGS still experience persistent unmet need across multiple dimensions, such as seizure burden and treatment tolerability,” Sarah Sheikh, head of Takeda’s neuroscience therapeutic area, said in a release. “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.”
Takeda is calculating the financial impacts of the results, including impairment loss, for the first quarter, which ends June 30.