Takeda Secures Global Rights to Rare Epilepsy Treatment from Ovid for $860 Million

Rare Daily Staff

Takeda Pharmaceutical entered into an exclusive agreement valued at $856 million to secure global rights from Ovid Pharmaceuticals for soticlestat, an experimental medicine for the treatment of developmental and epileptic encephalopathies, including Dravet syndrome and Lennox-Gastaut syndrome.

Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) are types of developmental and epileptic encephalopathies, a heterogeneous group of rare epilepsy syndromes. Dravet and Lennox-Gastaut syndrome typically become apparent during infancy or early childhood and are highly refractory to many antiseizure medications.

Dravet syndrome is most commonly caused by a genetic mutation in the SCN1A gene and affects approximately 1 in 15,000 to 1 in 21,000 people in the United States. Dravet syndrome is characterized by prolonged focal seizures that can evolve to convulsive tonic-clonic seizures. Children with Dravet syndrome experience developmental disabilities as seizures increase. Other common symptoms include changes in appetite, difficulty balancing and a crouched gait when walking.

Lennox-Gastaut syndrome is estimated to affect approximately 1 in 11,000 people in the United States. Lennox-Gastaut syndrome is a heterogeneous condition and characterized by several different types of seizures, most commonly atonic (drop), tonic, and atypical absence seizures. Children with Lennox-Gastaut syndrome may also develop cognitive dysfunction, delays in reaching developmental milestones and behavioral problems. Lennox-Gastaut syndrome can be caused by a variety of underlying conditions, but in some cases no cause can be identified.

Discovered by researchers at Takeda, soticlestat is a potent, highly selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H), with the potential to reduce seizure susceptibility and improve seizure control. Under a 2017 collaboration agreement, Ovid licensed the drug from Takeda, which received equity in Ovid and was eligible to receive up to $85 million in payments for regulatory milestones, including the initiation of phase 3 clinical trials. Ovid led global development of soticlestat through the successful demonstration of proof-of-concept in multiple rare epilepsies.

Takeda and Ovid reported results from the phase 2 ELEKTRA study in August 2020, in which soticlestat met its primary endpoint of reducing seizure frequency in pediatric patients with DS or LGS. Now Takeda is retaking the reins to further its development and commercialization.

“Soticlestat has emerged as an important late-stage molecule in our portfolio, which focuses predominantly on rare neurological and neuromuscular diseases with great unmet need,” said Sarah Sheikh, head of the Neuroscience Therapeutic Area Unit at Takeda.

Under the terms of the new agreement, Takeda will assume sole responsibility for further worldwide development and commercialization, and Ovid will no longer have any financial obligation to Takeda under the original collaboration agreement, including for milestone payments or any future development and commercialization costs.

Ovid will receive an upfront payment of $196 million at closing and is eligible to receive up to an additional $660 million upon achieving development, regulatory and sales milestones. In addition, Ovid will receive tiered royalties beginning in the low double-digits and up to 20 percent on sales of soticlestat, if approved and commercialized. The new agreement is expected to close by end of March 2021, subject to the satisfaction of customary closing conditions.

Takeda intends to initiate phase 3 studies of soticlestat in children and young adults with DS and LGS in the second quarter of 2021.

“This new agreement is a positive outcome for patients, for Ovid and for Takeda. Jointly, we have set the stage, optimized the program and enabled it to accelerate,” said Jeremy Levin, chairman and CEO of Ovid Therapeutics. “Ovid may benefit significantly, but without the obligation to commit the substantial capital needed over the coming years as soticlestat completes pivotal trials and, if successful, enters the global market. Importantly, with the resources this agreement delivers, Ovid is strategically and financially positioned well into the future. We will advance and enrich our pipeline while continuing to build a leading company in rare diseases of the brain.”

Photo: Jeremy Levin, chairman and CEO of Ovid Therapeutics