Neurocrine Biosciences and Idorsia Amend Option Agreement to License Novel Treatment for Rare Pediatric Epilepsy

Publisher: The Insight Partners

Neurocrine Biosciences, Inc. (NASDAQ: NBIX) and Idorsia Ltd. (SIX: IDIA) announced an amendment to the agreement that was originally signed in 2019 granting Neurocrine Biosciences an option to license ACT-709478, a potent, selective, orally-active, and brain penetrating T-type calcium channel blocker, in clinical development for the treatment of a rare pediatric epilepsy. The option also includes a research collaboration to discover, identify and develop additional novel T-type calcium channel blockers.

"We are excited to leverage the scientific expertise of Idorsia in T-type calcium channel inhibition to potentially advance a Phase II ready compound to help people suffering from epilepsy. In addition to the treatment of epilepsy, the modulation of the calcium channel may be useful for the treatment of other disorders such as essential tremor and pain," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "This option agreement enhances our commitment to utilizing precision medicine and differentiated mechanisms of action to develop a product portfolio capable of treating patients impacted by different forms of epilepsy."

Option and License Agreement Financial Terms
In 2019, Neurocrine Biosciences paid a non-refundable $5 million upfront fee to Idorsia for the option rights to ACT-709478 and a preclinical research collaboration. Furthermore, Neurocrine has agreed to cover additional costs as part of the IND application. The agreement is subject to the following terms upon Neurocrine Biosciences exercise of the option:

• Option Exercise: Upon Investigational New Drug (IND) application acceptance by the U.S. Food and Drug Administration (FDA), expected in mid-2020, Neurocrine Biosciences will have 30 days to exercise the option to license ACT-709478. If the option is exercised by Neurocrine Biosciences, Idorsia will receive an upfront payment of $45 million in cash. In addition, Neurocrine Biosciences will provide an incremental $7 million in funding to Idorsia as part of the research collaboration.
• 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 U.S. and a tiered royalty at slightly lower rates outside the U.S. 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.