Atsena Therapeutics and Nippon Shinyaku enter exclusive license agreement for ATSN-101

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Atsena Therapeutics Inc. and Nippon Shinyaku Co., Ltd. have entered into an exclusive license agreement for the commercialization of ATSN-101 in the United States and for the development and commercialization of ATSN-101 in Japan. This collaboration aims to advance ATSN-101, an investigational gene therapy for Leber congenital amaurosis (LCA) caused by biallelic mutations in the GUCY2D gene (LCA1).

Under the terms of the agreement, Nippon Shinyaku will hold exclusive commercial rights for ATSN-101 in both the US and Japan. Atsena will retain commercial rights in all other regions worldwide. In the U.S., ATSN-101 will be marketed by NS Pharma Inc., a wholly owned subsidiary of Nippon Shinyaku.1

According to Atsena, it will receive an upfront payment, milestone payments, and downstream royalties based on sales. Additionally, the company will be reimbursed for its continued development efforts, including a planned global pivotal trial of ATSN-101.

ATSN-101 is a first-in-class gene therapy designed for the treatment of LCA1. Atsena has received several designations from the U.S. Food and Drug Administration (FDA), including Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy (RMAT) Designation, and Orphan Drug Designation for ATSN-101.1

Should Atsena receive a Rare Pediatric Disease Priority Review Voucher (PRV) following the approval of ATSN-101’s Biologics License Application (BLA), the company will retain all rights, title, and interest in the PRV.

This partnership marks a significant step forward in the development and potential commercialization of ATSN-101, bringing hope for a new therapeutic option to patients with LCA1.

Patrick Ritschel, CEO of Atsena Therapeutics, said the agreement creates a path to accelerate the development of ATSN-101 and validates Atsena’s pioneering technology and development capabilities.

“We anticipate this will be the first of many ocular gene therapy treatments from our clinical portfolio to come,” Ritschel said. “We look forward to working with Nippon Shinyaku as we advance ATSN-101 into a pivotal trial and potential approval to provide an innovative solution to patients and families affected by LCA1 around the world.”

“ATSN-101 provides a potential, innovative treatment in an area where no approved solutions currently exist,” said Nippon Shinyaku President Toru Nakai. “We are excited by the opportunity of this novel ocular gene therapy and our collaboration with Atsena and its groundbreaking science.”

Reference

1. Haserick B. Atsena Therapeutics – Nippon Shinyaku and Atsena Therapeutics Enter into an Exclusive Strategic Collaboration for ATSN-101 in the U.S. and Japan. Atsena Therapeutics. Published November 13, 2024. Accessed November 13, 2024. https://atsenatx.com/press-release/nippon-shinyaku-and-atsena-therapeutics-enter-into-an-exclusive-strategic-collaboration-for-atsn-101-in-the-us-and-japan/