Atsena Therapeutics, Inc. has agreed to an exclusive license agreement with Nippon Shinyaku Co., Ltd. for the development and commercialization of ATSN-101, Atsena’s investigational gene therapy for Leber congenital amaurosis (LCA1), in the United States and Japan.
Start with Nippon Shinyaku.
Based in Japan, the pharmaceutical company is involved in developing and manufacturing “ethical pharmaceuticals and functional foods,” with a focus on four key therapeutic areas:
- Urology
- Hematology
- Intractable and rare diseases
- This is key
- Gynecology
Take a look at its recent product developments.
Next up: Atsena.
The North Carolina-based clinical-stage gene therapy company is developing a pipeline of novel, next-generation therapeutics to treat three forms of inherited blindness:
- X-linked retinoschisis (XLRS)
- GUCY2D-associated LCA1
- MYO7A-associated Usher syndrome (USH1B)
Its science: Involves adeno-associated virus (AAV) technologies featuring novel, laterally-spreading capsids, dual vectors, and intravitreal capsids—all comprising a “dual vector technology.”
- About this tech: These “dual vectors” are designed to deliver larger payloads for genetic mutations that are typically too large to treat with just one AAV vector.
Check out that mechanism of action below.
Moving on to this asset …
ATSN-101 was developed to introduce the functional human copy of the GUCY2D gene to photoreceptors—potentially treating LCA1.
About the asset: Administered via a single subretinal injection, the therapeutic is an AAV5 capsid and human rhodopsin (RHO) promoter.
- Its clinical status: The FDA previously granted the following designations:
- Orphan Drug designation
- Rare Pediatric Disease (RPD) designation
- Regenerative Medicine Advanced Therapy (RMAT) designation
And the clinical data on it so far?
The therapeutic is currently under clinical investigation in an ongoing phase 1/2 clinical trial (NCT03920007) of LCA1 patients treated with three ascending doses of ATSN-101.
The data: In September 2024, 12-month safety and efficacy data reported that ATSN-101 “demonstrated durable, clinically significant improvements in vision at the high dose” and was also well-tolerated post-treatment among patients with LCA10 caused by biallelic mutations in GUCY2D.
See how this compares to 6-month data, reported in April 2023.
Now about this license agreement … what does it entail?
- Nippon Shinyaku will receive exclusive commercial rights to ATSN-101 in the U.S. and Japan
- In the U.S., ATSN-101 will be marketed by NS Pharma, Inc., the wholly-owned U.S. subsidiary of Nippon Shinyaku.
- Atsena will retain commercial rights across the globe (excluding the U.S. and Japan)
Any money to talk about?
Alas, no specific amount was given. However, per the agreement, Atsena will receive the following:
- Upfront payment
- Additional milestone payments
- Downstream royalties based on sales
Plus: The company will be reimbursed if it continues development work on ATSN-10—“including an anticipated global pivotal trial,” according to Atsena.
Nice! Anything else the company will receive?
Potentially …
Pending the FDA approving a Biologic License Application (BLA) for ATSN-101, Atsena may also receive an RPD priority review voucher (RPV) as well as “own and retain all rights, title and interest in such PRV.”
Keep in mind: A PRV would (potentially, keep in mind) allow Atsena to receive an expedited 6-month priority review for any marketing applications that follow—i.e., an investigational new drug (IND) or new drug application (NDA)—or that could be sold or transferred to other companies (to generate additional revenue).
- Regulatory update: As of Dec. 20, 2024, the FDA is adjusting its PRV program to specific criteria. Check it out here.