Neurocrine Biosciences Exercises Option to License Idorsia’s Novel Treatment for Rare Pediatric Epilepsy

Rare Daily Staff

Neurocrine Biosciences said it has exercised its option to license global rights to Idorsia’s experimental therapy for the treatment of a rare pediatric epilepsy.

The license exercise follows U.S. Food and Drug Administration acceptance of Idorsia’s New Drug application for ACT-709478, a selective, orally active, brain penetrating T-type calcium channel blocker, for the treatment of a rare pediatric epilepsy. Neurocrine plans to initiate a phase 2 study in the second half of 2020.

“This collaboration demonstrates Neurocrine Biosciences’ growing commitment in epilepsy and enhances our capabilities in precision medicine by targeting the underlying mechanism of disorders,” said Kevin Gorman, CEO of Neurocrine Biosciences.

In 2019, Neurocrine Biosciences paid a $5 million upfront fee to Idorsia for the option rights to ACT-709478 and entered into a preclinical research collaboration. Exercise of the option triggers an upfront payment of $45 million in cash to Idorsia. In addition, Neurocrine Biosciences will provide an incremental $7 million in funding to Idorsia as part of the research collaboration to discover, identify and develop additional novel T-type calcium channel blockers.

Idorsia is also entitled to receive up to $365 million in development and regulatory milestone payments, one-time commercial payments based on sales thresholds, plus royalties.

The companies will also work together to identify novel T-type channel blockers and explore their use in potential new disease states for which Idorsia may be entitled to additional development, regulatory and commercial milestones as well as tiered royalties on annual sales for each product included in the research collaboration.

“Our drug discovery efforts have produced a diverse pipeline of innovative compounds, addressing different diseases where treatment options are either non-existent or unsatisfactory. If the efficacy of our selective T-type calcium channel blocker seen in preclinical models is confirmed in children with rare pediatric epilepsy, it could transform the life of children with this disease, said Martine Clozel, chief scientific officer of Idorsia.

Idorsia has received Rare Pediatric Disease designation from the FDA for ACT-709478 for the treatment of a rare pediatric epilepsy. The FDA grants Rare Pediatric Disease designation for diseases that primarily affect children ages 18 years or younger and fewer than 200,000 persons in the United States.

Photo: Kevin Gorman, CEO of Neurocrine Biosciences