Ovid Therapeutics presented data from several studies that support the clinical development of the investigational therapy TAK-935/OV935 as a potential treatment for developmental and epileptic encephalopathies.
These results are particularly significant for Angelman syndrome patients, 85 percent of whom suffer from some form of epilepsy.
The findings from two Phase 1 clinical trials, two preclinical studies, and a dosing model were recently presented at the 71st Annual Meeting of the American Epilepsy Society, held Dec. 1-5 in Washington, D.C.
Earlier this year, Takeda and Ovid established a clinical collaboration to develop TAK-935/OV935 for adults with developmental and epileptic encephalopathies. The drug is a strong and highly selective blocker of the cholesterol 24-hydroxylase (CH24H) enzyme, which is involved in over-activation of a brain signaling pathway that may lead to epilepsy.
“We are pleased with the results of these studies, which validate CH24H inhibition as a novel target for epilepsy treatment and also form a strong foundation supporting our ongoing clinical development and biomarker strategy for TAK-935/OV935,” Dr. Emiliangelo Ratti, head of Takeda’s Central Nervous System Therapeutic Area Unit, said in a press release.
“Not only do these results reinforce the safety and tolerability of this potential therapy, but they also provide vital information for the design and dosing of future clinical trials.”
In two Phase 1 clinical trials with healthy participants (NCT02201056 and NCT02539134), researchers investigated the safety and tolerability profile of single and multiple ascending doses of TAK-935/OV935, respectively.
The results showed the drug candidate was safe and well tolerated when administrated as a single 1,350 mg dose (the highest single dose tested), and when given as once-daily doses of up to 400 mg for 14 days.
“These results support our broader development strategy for TAK-935/OV935, which includes development in younger patient populations since developmental and epileptic encephalopathies are often diagnosed early in life. We believe providing an early intervention may alter the course of the disease,” said Amit Rakhit, MD, MBA, chief medical and portfolio management officer of Ovid Therapeutics.
“We are excited about these data and the potential of TAK-935/OV935 to be an impactful treatment for people with developmental and epileptic encephalopathies who currently do not have sufficient therapeutic options,” he added.
The company is conducting a Phase 2 study (NCT03166215) to evaluate the multiple-dose safety and tolerability of TAK-935/OV935 in adult patients with developmental or epileptic encephalopathies. The study is currently recruiting participants.
“We are looking forward to the results of our ongoing Phase 1b/2a clinical trial of TAK-935/OV935 in adults with developmental and epileptic encephalopathies, which are expected in 2018,” Rakhit added.