Takeda and Ovid’s Epilepsy Drug Sees Positive Results in Phase II Trial
Takeda Pharmaceutical and Ovid Therapeutics announced on Tuesday that they have gathered positive data from their Phase II ELEKTRA study of soticlestat in children with Dravet Syndrome (DS) or Lennox-Gastaut Syndrome (LGS).
Soticlestat is a highly selective, oral inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H), and is currently being investigated by Takeda and Ovid for the treatment of rare developmental and epileptic encephalopathies.
The ELEKTRA study reached its primary endpoint with high statistical significance. There was a 27.8% median reduction from baseline in convulsive seizure and drop seizure frequency, compared to a 3.1% median increase in patients receiving a placebo. Additionally, DS and LGS patients who were treated with soticlestat showed a 29.8% median reduction in convulsive seizure and drop seizure frequency, compared to a 0.0% change in median seizure frequency in subjects taking a placebo. This data was recorded over the course of 20 weeks.
Soticlestat was overall well-tolerated in the ELEKTRA study and demonstrated a safety profile that was consistent with those of previous studies. All of the subjects who completed the ELEKTRA study opted to enroll in the ENDYMION open-label extension study.
“We are extremely encouraged by these results, which show a clear statistically significant reduction of seizures in Dravet syndrome patients treated with soticlestat, as well as a trend for seizure reduction in Lennox-Gastaut patients,” said Amit Rakhit, M.D., MBA, President and Chief Medical Officer of Ovid. “We look forward to continuing our collaboration with Takeda to initiate a Phase III registrational program for soticlestat in patients with DS, while continuing to analyze the data from patients with LGS in the ELEKTRA and ENDYMION studies to define potential next steps.”
At the beginning of August, GW Pharmaceuticals reported positive news from the U.S. Food and Drug Administration (FDA) regarding Epidiolex, its oral solution designed to treat seizures. The FDA ultimately approved of Epidiolex to address seizures in patients with tuberous sclerosis complex (TSC) in patients one year of age and older. The age range was expanded to include patients one year of age and older who experience seizures associated with LGS or DS.
“FDA approval of this new indication is exciting news for those with refractory seizures due to tuberous sclerosis complex,” said Justin Gover, GW’s Chief Executive Officer. “Since Epidiolex is already available to patients by physician’s prescription, patients with TSC can immediately access the medication. This label expansion, including the expansion of the age range in all approved indications, further demonstrates that the FDA process can continue to enable broader patient access to appropriately tested regulatory approved cannabinoid medicines. It also provides hope for these patients and their families and is yet another important milestone for Epidiolex as a first-in-class antiepileptic drug.”
Phase III study data has already demonstrated the safety and efficacy of Epidiolex. The most common adverse effects in those receiving the product were diarrhea, transaminase elevations, decreased appetite, somnolence, pyrexia and vomiting.
In the Phase III trial, Epidiolex reached its primary endpoint, which was the reduction in seizure frequency compared to a placebo. Epidiolex appeared to reduce seizures by 48% when patients received 25mg/day, compared to 24% who received a placebo. All secondary endpoints were supportive of the effects of the primary endpoint.
“Based on previous positive trial results in TSC patients, Epidiolex may become an important treatment option for patients. It is a new tool in the toolbox for physicians and could meet a significant unmet need," said Elizabeth Thiele, M.D., Ph.D., Director of the Herscot Center for Tuberous Sclerosis Complex at Massachusetts General Hospital, Professor of Neurology at Harvard Medical School and clinical investigator.