This marks the second program Ovid has advanced into Phase 2 clinical development this year.
Takeda and Ovid formed a global collaboration focused on the clinical development and commercialization of TAK-935/OV935, a potent and highly selective cholesterol 24-hydroxylase (CH24H) inhibitor, being investigated in adults with developmental and/or epileptic encephalopathies in January 2017.
“We are excited to achieve this important milestone as this is the first clinical trial in our collaboration with Ovid and TAK-935/OV935 is the first CH24H inhibitor in clinical development,” said Dr. Emiliangelo Ratti, head of Takeda’s Central Nervous System Therapeutic Area Unit.
“While there are many treatments available for epilepsy, few therapeutic options exist for patients with developmental and/or epileptic encephalopathies, which cause intractable seizures that are often associated with cognitive, neurologic and behavioral symptoms. Our collaboration with Ovid is geared to leverage each company’s strengths to jointly develop this investigational medicine for people living with rare epilepsies.”
“We believe that TAK-935/OV935 has the potential to target the increased pro-epileptic signaling that occurs in developmental and/or epileptic encephalopathies and provide a treatment for people with rare epilepsies who currently do not have sufficient options,” said Amit Rakhit, M.D., MBA, chief medical and portfolio management officer of Ovid Therapeutics.
“This trial is the beginning of a planned broader development program for TAK-935/OV935. We intend to investigate TAK-935/OV935 in younger populations because rare epilepsies, such as Dravet syndrome, Lennox-Gastaut syndrome and Tuberous Sclerosis Complex, are diagnosed early in life and this may provide an opportunity to intervene early in the disease course.”
Data from the Phase 1b/2a trial in adults with developmental and/or epileptic encephalopathies, which are types of rare epilepsies that share similar clinical manifestations, is anticipated in 2018.
As part of the broader development program, Ovid and Takeda also plan to study the role of 24-S-hydroxycholesterol (24HC) as a peripheral biomarker that can inform future clinical trial designs and help clinicians individualize the use of this therapy.
TAK-935/OV935 has the potential to become a first-in-class CH24H inhibitor and is believed to modulate the N-Methyl-D-Aspartate (NMDA) receptor, which has been implicated in several neurologic disorders, including rare epilepsies. TAK-935/OV935 has been tested in four Phase 1 clinical trials involving 86 healthy volunteers and has been found to be well tolerated without clinically significant safety findings. Data from multiple preclinical models indicate that TAK-935/OV935 may affect both seizure intensity and frequency.
The randomized, double-blind, placebo-controlled, dose-escalation Phase 1b/2a clinical trial will enroll approximately 20 adult patients with developmental and/or epileptic encephalopathies, including Dravet Syndrome, Lennox-Gastaut Syndrome and Tuberous Sclerosis Complex.
The multicenter trial will include an initial one-month baseline period followed by a one-month double-blind, dose-escalation phase. Participants will have the option to continue for an additional two-month open-label extension phase.
The primary endpoint of the study is to characterize the safety and tolerability of TAK-935/OV935. Secondary endpoints include assessment of standard safety laboratory values and evaluation of pharmacokinetics.