The ELEKTRA trial testing the investigational oral therapy soticlestat (OV935/TAK-935) in children with Lennox-Gastaut syndrome (LGS) and Dravet syndrome has completed patient enrollment, Ovid Therapeutics has announced.
“We completed enrollment significantly ahead of schedule in our placebo-controlled Phase 2 ELEKTRA trial in children with Dravet syndrome and Lennox-Gastaut syndrome, and as a result, we now expect data to be available in the third quarter of 2020,” Amit Rakhit, MD, president and chief medical officer of Ovid, said in a press release.
Soticlestat was developed by Takeda Pharmaceuticals in collaboration with Ovid Therapeutics for the treatment of rare developmental and epileptic encephalopathies, including Dravet and LGS.
The therapy is a highly-selective inhibitor of the enzyme cholesterol 24-hydroxylase, which has a major role in clearing cholesterol in the brain. By blocking this enzyme, soticlestat is thought to reduce the activation of a neuronal signaling pathway — glutamatergic signaling via NMDA receptors — potentially reducing seizure susceptibility and improving seizure control in patients with LGS.
ELEKTRA (NCT03650452) is a multicenter, placebo-controlled study to assess the effectiveness and safety of soticlestat treatment in chidren and adolescents, ages 2 to 17 years, who have experienced seizures associated with LGS (drop or atonic seizures) or Dravet (convulsive seizures).
The study includes a four- to six-week observation period to establish seizure frequency, followed by a 20-week treatment period that’s divided into an eight-week dose optimization period followed by 12 weeks of maintenance treatment. The primary outcome of the study is the percent change in the frequency of seizures for 28 days after soticlestat treatment, compared to a placebo.
Patients who complete the ELEKTRA study can enroll in the open-label, long-term Phase 2 ENDYMION (NCT03635073) trial.
Previous results from the first six adult patients enrolled in this ongoing trial, who had rolled over from a Phase 1b/2a clinical study (NCT03166215), showed that treatment with soticlestat was able to reduce the median seizure frequency by up to 90% in adults with LGS and other DEEs.
Importantly, the Phase 1b/2a trial found that soticlestat may have a drug-drug interaction with perampanel (sold under the brand Fycompa by Eisai), so the two therapies should not be taken together. Data from this earlier study and the ENDYMION study suggest that the potential therapy is otherwise safe and well-tolerated.
“We are also pleased to report that all patients who have completed the ELEKTRA and ARCADE [NCT03694275] trials have rolled over into our ENDYMION open-label extension study,” Rakhit said.
The U.S. Food and Drug Administration granted orphan drug designation to soticlestat for the treatment of both Dravet syndrome and LGS in December 2017.