Acadia Pharmaceuticals Acquires Ex-North American Rights to...

Acadia Pharmaceuticals Acquires Ex-North American Rights to Trofinetide and Global Rights to Neuren’s NNZ-2591 in Rett Syndrome and Fragile X Syndrome

July 13, 2023

PDF Version

-- Expanded agreement follows Acadia’sApril 2023U.S. launch of DAYBUE™ (trofinetide) as the first and only drug approved for the treatment of Rett syndrome

-- Acadia provides DAYBUE launch update and announces second quarter preliminary net sales and guidance for third quarter

-- Company to host conference call and webcast today at4:30 p.m. Eastern Time

SAN DIEGO--(BUSINESS WIRE)--Jul. 13, 2023--Acadia Pharmaceuticals Inc.(NASDAQ: ACAD) today announced that it has expanded its current licensing agreement for trofinetide with Neuren Pharmaceuticals to acquire ex-North American rights to the drug as well as global rights in Rett syndrome and Fragile X syndrome to Neuren’s development candidate NNZ-2591. In April of this year, Acadia launched trofinetide inthe United Statesunder the brand name DAYBUE as the first and only drug approved for the treatment of Rett syndrome.

“This expanded worldwide agreement solidifies Acadia's position as the global leader in addressing the unmet needs of people with Rett syndrome,” saidSteve Davis, Acadia’s President and Chief Executive Officer. “We have successfully delivered DAYBUE, the first FDA-approved therapy that treats the core symptoms of Rett syndrome, and are deeply committed to broadening access to this important therapy for patients worldwide.”

In addition to expanding access to trofinetide outside ofNorth America, this agreement gives Acadia exclusive worldwide rights to NNZ-2591 in both Rett syndrome and Fragile X syndrome. NNZ-2591 is an investigational synthetic analogue of cyclo-glycyl-proline (cGP) which results from the breakdown of human insulin-like growth factor 1 (IGF-1). NNZ-2591 is currently under development by Neuren in four other rare neurodevelopmental syndromes.

Execution of this agreement advances Acadia’s corporate strategy to expand our rare disease business. This deal also enables Acadia to leverage insights from our successfulU.S.launch of DAYBUE in other global territories. In addition, this expansion will further advance the global potential of Acadia’s current development portfolio.

Acadia intends to submit a New Drug Submission (NDS) for trofinetide inCanadain the next 18 months with plans forEurope,Asiaand other regions to be announced at a later date.

Financial Terms

Under the terms of the expanded agreement, Neuren will receive an upfront payment of US$100 millionand is eligible to receive additional potential downstream milestone and royalty payments earned separately for trofinetide and NNZ-2591.

Outside of North America, Neuren is eligible to receive additional payments for trofinetide upon the achievement of specified revenue milestones as follows:

	First Commercial Sales Milestones	Total Sales Milestones(1)
Europe	$35M(Rett);$10M(2ndindication)	Up to$170M
Japan	$15M(Rett);$4M(2ndindication)	Up to$110M
Rest of World	-0-	Up to$83M

(1)

Each region’s sales milestones are divided into four distinct milestones based upon escalating annual net sales thresholds as defined in the agreement.

Neuren will also receive tiered royalties from the mid-teens to low-twenties percent of trofinetide net sales outside ofNorth America. InNorth America, all milestones and royalties for trofinetide remain unchanged from Acadia’s previously existing North American license agreement with Neuren. Potential future payments to Neuren related to NNZ-2591 in Rett syndrome and Fragile X syndrome are identical to the payments for trofinetide in each ofNorth Americaand outsideNorth America.

Preliminary Second Quarter Revenues and Updated Guidance

DAYBUE

• DAYBUE 2Q 2023 preliminary net sales:$21to$23 million.
• DAYBUE3Q2023 net sales guidance:$45to$55 million.

NUPLAZID

• NUPLAZID 2Q 2023 preliminary net sales:$140to$144 million.
• NUPLAZIDFull Year2023 net sales guidance:$530to$545 million.