Nippon Shinyaku and Atsena Therapeutics enter into an Exclusive Strategic Collaboration for ATSN-101 in the U.S. and Japan

Nippon Shinyaku Co., Ltd. and Atsena Therapeutics, Inc. have entered into an exclusive license agreement for the commercialization of ATSN-101 in the territory of the U.S. and for the development and commercialization of ATSN-101 in the territory of Japan for advancing Atsena’s first-in-class, investigational gene therapy ATSN-101 for Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1).

Under the terms of the licensing agreement, Nippon Shinyaku will receive exclusive commercial rights in the U.S. and Japan, and Atsena will retain commercial rights in the rest of the world. ATSN-101 will be marketed by NS Pharma, Inc. (New Jersey, USA, President: Yukiteru Sugiyama), a wholly owned subsidiary of Nippon Shinyaku in the U.S.

Atsena will receive an upfront payment, additional milestone payments, downstream royalties based on sales and will be reimbursed as it continues development work on ATSN-101, including an anticipated global pivotal trial.

ATSN-101 is a first-in-class, investigational gene therapy for the treatment of LCA1. Atsena has received Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for ATSN-101. In the event Atsena receives a Rare Pediatric Disease Priority Review Voucher (PRV) in connection with the approval of the Biologic License Application for ATSN-101, Atsena shall own and retain all rights, title and interest in such PRV.

“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.”

“This collaboration 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,” said Patrick Ritschel, CEO of Atsena Therapeutics. “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.”