Neurocrine Biosciences Exercises Option to License Rare Pediatric Epilepsy Treatment

Neurocrine Biosciences and Idorsia announced, following Investigational New Drug (IND) application acceptance by the U.S. Food and Drug Administration (FDA), Neurocrine Biosciences has exercised its option to license the global rights of Idorsia's ACT-709478. Neurocrine Biosciences plans to initiate a Phase II study with this potent, selective, orally-active and brain penetrating T-type calcium channel blocker for the treatment of a rare pediatric epilepsy in the second half of 2020.

"We are pleased with the FDA's acceptance of ACT-709478, a selective T-type calcium channel inhibitor, and we look forward to advancing this program into Phase II to potentially help children with a rare pediatric epilepsy. This collaboration demonstrates Neurocrine Biosciences' growing commitment in epilepsy and enhances our capabilities in precision medicine by targeting the underlying mechanism of disorders," Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences, said.

"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. When we discover a new drug that shows promise in an indication where we have the necessary expertise, we will vigorously pursue the development ourselves. Sometimes we believe more value can be created by working with a partner. This is one such example where Neurocrine Biosciences can bring their exceptional expertise in the development of innovative drugs for neurological disorders," Martine Clozel, MD and Chief Scientific Officer of Idorsia, said.

In 2019, Neurocrine Biosciences paid a $5 million upfront fee to Idorsia for the option rights to ACT-709478 and a preclinical research collaboration. In May 2020, upon Investigational New Drug (IND) application acceptance by the U.S. Food and Drug Administration (FDA), Neurocrine Biosciences exercised the option to license.

ACT-709478. The exercise of the option triggered an upfront payment of $45 million in cash from Neurocrine Biosciences 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.

The agreement is subject to the following terms:

ACT-709478 milestones: In addition to the up-front payment, Idorsia may also receive up to $365 million in additional development and regulatory milestone payments. Furthermore, Idorsia may also be entitled to one-time commercial payments based on sales thresholds.

ACT-709478 royalties: Idorsia will have the right to receive a tiered royalty ranging from the low double-digits to upper teen percentage in the US and a tiered royalty at slightly lower rates outside the US based upon aggregate global net sales.

Preclinical research collaboration: The parties will work together to identify novel T-type channel blockers and explore their use in potential new disease states. 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.

ACT-709478 is a potent, selective, orally-active and brain penetrating T-type calcium channel blocker in development for epilepsy. A Phase I clinical trial was completed in healthy adult subjects in 2019. The IND application was accepted by the FDA on April 30, 2020. A Phase 2 study in a rare pediatric epilepsy is planned for the second half of 2020.

Idorsia has received Rare Pediatric Disease designation from the U.S. 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 U.S.