Neurocrine Biosciences exercises option to license Idorsia's novel treatment for rare pediatric epilepsy

• Neurocrine Biosciences to develop and commercialize ACT-709478, a clinical stage selective T-type calcium channel blocker for the treatment of a rare pediatric epilepsy – Phase 2 study planned for the second half of 2020.
• Idorsia receives a $45 million upfront payment in cash.
• Idorsia will also be entitled to potential development and regulatory milestone payments up to $365 million and tiered royalties on net sales.

SAN DIEGO, US and ALLSCHWIL, Switzerland – May 12, 2020
Neurocrine Biosciences, Inc. (Nasdaq: NBIX) and Idorsia Ltd (SIX: IDIA) today announced that following Investigational New Drug (IND) application acceptance by the US Food and Drug Administration (FDA), Neurocrine Biosciences has exercised its option to license the global rights to Idorsia’s ACT-709478. Neurocrine Biosciences plans to initiate a Phase 2 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.

Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences, commented:
“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 2 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.”

Martine Clozel, MD and Chief Scientific Officer of Idorsia, commented:
“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."