Neurocrine uses option to licence rights to Idorsia’s inhibitor for treatment of rare children epilepsy

Neurocrine Biosciences has exercised its option to license the global rights to Idorsia’s ACT-709478, as it 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, said he was pleased with the FDA’s acceptance of ACT-709478, a selective T-type calcium channel inhibitor, and that the company is looking forward to advancing this program into Phase 2 to potentially help children with a rare pediatric epilepsy. He said that this collaboration demonstrates Neurocrine Biosciences’ growing commitment in epilepsy and enhances its capabilities in precision medicine by targeting the underlying mechanism of disorders.

Martine Clozel, MD and Chief Scientific Officer of Idorsia, said that Idorsia’s drug discovery efforts have produced a diverse pipeline of innovative compounds, addressing different diseases where treatment options are either non-existent or unsatisfactory. Clozel said in the press release that, if the efficacy of Idorsia’s 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.

She said that when Idorsia discovers a new drug that shows promise in an indication where, it has the necessary expertise, the company will vigorously pursue the development. She said that sometimes more value can be created by working with a partner. Clozel said that this is one such example where Neurocrine Biosciences can bring their exceptional expertise in the development of innovative drugs for neurological disorders.

About the license and collaboration agreement
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 US 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.