Nippon Shinyaku and Atsena Therapeutics Form Exclusive U.S.-Japan Pact for ATSN-101

On November 13, 2024, Nippon Shinyaku Co., Ltd., headquartered in Kyoto and led by President Toru Nakai, announced an exclusive licensing agreement with Atsena Therapeutics, Inc., based in Durham, North Carolina, USA, with Patrick Ritschel as CEO. This agreement focuses on the commercial rights for ATSN-101, an investigational gene therapy developed by Atsena for treating Leber congenital amaurosis (LCA1) caused by biallelic mutations in the GUCY2D gene. Nippon Shinyaku will hold exclusive commercialization rights in the U.S. and Japan, with Atsena retaining rights for other global markets. In the U.S., ATSN-101 will be marketed by NS Pharma, Inc., a subsidiary of Nippon Shinyaku.

As part of the agreement, Atsena will receive an upfront payment from Nippon Shinyaku, along with additional milestone payments, royalties based on sales, and reimbursement for ongoing development work, including a planned global pivotal trial. ATSN-101, a first-in-class gene therapy, has garnered Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy Designation, and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). Should Atsena obtain a Rare Pediatric Disease Priority Review Voucher (PRV) upon the Biologic License Application approval for ATSN-101, the company will maintain all rights to the PRV.

According to Toru Nakai, President of Nippon Shinyaku, ATSN-101 represents a groundbreaking treatment option for a condition that currently lacks approved therapies. Nakai expressed enthusiasm for the potential of this novel ocular gene therapy and the collaboration with Atsena. Patrick Ritschel, CEO of Atsena Therapeutics, echoed this sentiment, highlighting the partnership as a means to accelerate ATSN-101's development, validate Atsena's innovative technology, and pave the way for future ocular gene therapy treatments from their clinical portfolio.

LCA1 is a genetic eye disorder that disrupts retinal function, leading to severe vision impairment or blindness from an early age. It is caused by mutations in the GUCY2D gene and is one of the most prevalent forms of LCA. ATSN-101 is an investigational gene therapy administered subretinally using AAV5 vectors, currently undergoing a Phase I/II clinical trial. Results after 12 months post-treatment have shown significant, durable improvements in vision and good tolerability, with findings published in The Lancet.

The FDA's Rare Pediatric Disease Designation aims to promote drug development for rare pediatric diseases affecting fewer than 200,000 individuals in the U.S. It offers the potential to receive a Priority Review Voucher, expediting the review of another program. The Regenerative Medicine Advanced Therapy (RMAT) Designation, based on the 21st Century Cures Act, is granted to therapies showing substantial clinical promise for serious conditions, allowing priority review and accelerated approval. The Orphan Drug Designation supports the development of treatments for rare diseases affecting fewer than 200,000 people in the U.S., offering benefits such as market exclusivity, tax credits, and other incentives.

Atsena Therapeutics is focused on developing gene therapies for inherited retinal diseases to prevent or reverse blindness. The company is currently evaluating ATSN-101 in a Phase I/II trial for LCA1 and ATSN-201 for X-linked retinoschisis (XLRS), a genetic disorder affecting males. Atsena's innovative pipeline leverages advanced adeno-associated virus (AAV) technology to address challenges in treating inherited retinal diseases. The company, founded by leaders in ocular gene therapy, is committed to meeting the needs of patients experiencing vision loss.