- Ovid Therapeutics and its big pharma partner Takeda revealed on Tuesday new data for an experimental seizure drug that they claim are positive enough to push it into the final stage of clinical testing.
- The drug, soticlestat, hit the main goal of a 139-person clinical trial focused on two rare types of epilepsy known as Dravet syndrome and Lennox-Gastaut syndrome. However, only the Dravet cohort of patients who took soticlestat showed statistically significant reductions in seizures when compared to placebo.
- Takeda and Ovid said they plan to meet with regulators to hash out the details of a registrational program for Dravet syndrome, and to further analyze the Lennox-Gastaut data to determine next steps. They aim to see if soticlestat can compete with a growing field of Dravet medicines, three of which have been approved since 2018.
Many of the world's most powerful pharmaceutical companies have backed away from neuroscience drug development after finding the research too challenging or expensive. Those exits then created a void that small biotechs are now filling.
New York-based Ovid, for instance, formed in 2014 with the aim of advancing new treatments for rare neurological conditions. To accomplish its mission, Ovid has primarily looked externally. The biotech's most advanced candidate, now called OV-101, is in clinical testing for two genetic diseases. But previously, it had been investigated by Lundbeck and Merck & Co. as a sleeping pill. Ovid licensed it from Lundbeck in 2015.
With soticlestat, Ovid reached an agreement with Takeda in early 2017 to split rights to the drug. By August 2018, the companies had kicked off a mid-stage study of the drug in Dravet and Lennox-Gastaut patients.
Patients were first randomized to the drug or placebo group. Then they went through eight weeks of dose optimization before 12 weeks of maintenance. The trial enrolled 141 participants, with 126 completing all 20 weeks.
According to the companies, the drug met its primary goal by leading to a 28% median reduction in seizures when considering all participants who received soticlestat over the maintenance period. Patients in the placebo group, conversely, had a 3% median increase in seizures. Takeda and Ovid said these results were based on an analysis of 120 patients who had seizure data from the 12-week period.
But when broken down over the full 20 weeks of the study, the results show that Dravet patients had a statistically significant reduction in seizures, compared to placebo patients, while Lennox-Gastaut participants didn't.
In Dravet, the drug posted a placebo-adjusted decrease in seizure frequency that RBC Capital Markets analyst Brian Abrahams noted "compares well" to recently approved medicines from Zogenix and GW Pharmaceuticals. That result points to "a clear path forward for a pivotal trial and potential approval in Dravet," Abrahams wrote.
That purported benefit wasn't as stark for LGS patients. On a conference call Tuesday, Ovid executives suggested the differing results may have to do with enrollment issues and, in particular, the diversity of baseline characteristics for Lennox-Gastaut participants. In the screening period before the trial started, those patients showed a vast range of seizure frequencies, ranging from as low as four to as high as 5,188. They also appear more likely to have been on several anti-epileptic drugs.
Ovid, though, said it's not yet giving up on that indication.
"We do think that there are possibilities to move forward with LGS, we just want to make sure we have a good handle on all the data," Amit Rakhit, the company's chief medical officer, said on the conference call. Ovid will present full data from the trial at upcoming medical meetings, according to Rakhit.
In the meantime, Ovid and Takeda are already working on the late-stage Dravet program for soticlestat. Rakhit said the partners expect some interaction with regulators by the end of the year, while Ovid CEO Jeremy Levin said his company's balance sheet is in a good position to support further testing.
Ovid estimates there are around 30,000 Lennox-Gastaut patients and between 15,500 and 22,000 Dravet patients in the U.S. Epidiolex, a seizure medication from GW Pharmaceuticals, is approved to treat both diseases and had net sales of $118 million in the second quarter. Zogenix's Fintepla was cleared for Dravet patients in June.
Alongside the data release, Ovid announced an underwritten offering of 6.25 million shares of common stock priced at $8 apiece. The offering is expected to close on Aug. 27.