REGENXBIO Inc. and Nippon Shinyaku
Type: License agreement
In-Licensee: Nippon Shinyaku Co., Ltd.
Out-Licensor: REGENXBIO Inc.
Focus: RGX-121, RGX-121
Upfront Payment: $110 million
Milestone payments: $700 million
Financial impact: REGENXBIO Inc. (revenue is expected to grow)
REGENXBIO Inc. has entered into a licensing agreement with Nippon Shinyaku Co., Ltd. for the development and commercialization of two gene therapies: RGX-121 for Mucopolysaccharidosis II (MPS II or Hunter syndrome) and RGX-111 for Mucopolysaccharidosis I (MPS I or Hurler syndrome). Under the terms of the agreement, REGENXBIO will receive an upfront payment of $110 million and is eligible for up to $700 million in additional milestone payments. These include $40 million tied to development and regulatory achievements and up to $660 million based on sales milestones. Additionally, REGENXBIO will earn significant double-digit royalties on net sales of both therapies across the U.S. and Asia, which make up the designated licensed territory. It is anticipated that Nippon Shinyaku's revenue will increase following their partnership with REGENXBIO for the development and commercialization of RGX-121 and RGX-111. This is because Nippon Shinyaku will be responsible for commercializing the therapies in the United States and Asia, leading to potential sales and revenue growth.
Mechanism of Clemidsogene lanparvovec (RGX-121) in MPSII treatment
Clemidsogene lanparvovec (formerly RGX-121) is a personalized gene therapy developed by REGENXBIO (formerly ReGenX Biosciences) for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. The therapy uses an AAV9 viral vector to deliver the human iduronate-2-sulfatase (IDS) gene directly to cells in the central nervous system (CNS). Deficiency of IDS is the underlying cause of MPS II.
Once delivered, the IDS gene enables transduced cells to produce and secrete the functional enzyme. This enzyme can potentially be taken up by neighboring, non-transduced cells—a process known as cross-correction—thereby restoring enzyme activity across the CNS. Clemidsogene lanparvovec is currently under regulatory review in the United States, with ongoing clinical development in the U.S., Canada, and Brazil.
RGX 111Gene therapy Overview
RGX-111 is a personalized gene therapy developed by REGENXBIO (formerly ReGenX Biosciences) for the treatment of mucopolysaccharidosis type I (MPS I), a rare genetic disorder caused by a deficiency of the enzyme α-L-iduronidase (IDUA). The therapy utilizes an AAV9 vector to deliver the IDUA gene directly to the central nervous system (CNS), aiming to restore the enzyme needed to break down glycosaminoglycans such as heparan sulfate and dermatan sulfate within lysosomes. Clinical trials for RGX-111 are currently ongoing in the United States, Israel, and Brazil.
Five years' market revenue of Nippon Shinyaku
Before its partnership with REGENXBIO, Nippon Shinyaku had an established focus on rare and intractable diseases as a core area of research and development. This included a combination of proprietary drug discovery and in-licensing strategies aimed at expanding its pipeline in these specialized therapeutic areas. The company experienced steady revenue growth through 2021; however, a downward trend began post-2021. In the second quarter of fiscal year 2022 (ending September 30, 2022), revenue declined by 0.6% to 71,136 million yen. This was accompanied by a 12.1% decrease in operating profit and an 11.9% decline in profit before tax, reflecting broader market and operational challenges.
Five years market revenue of Nippon Shinyaku (2020-2024)
Fig 9: Market revenue of Nippon Shinyaku
In 2023, revenue continued to decline, largely due to reduced pricing across several products and market headwinds, particularly in Asia. Despite these setbacks, the company reported positive growth in certain segments, including its automotive coatings business in Japan and the Americas.
In FY2024, revenue was further impacted by the expiration of key product patents and an unfavorable shift in product mix. Notably, the loss of exclusivity for Uptravi—a treatment for pulmonary arterial hypertension—in Europe in October 2024, and the anticipated expiration in Japan in 2026, led to a significant reduction in royalty income. Although global sales volumes increased, this was offset by price erosion, especially in the U.S. market. The company also faced rising personnel costs and increased import expenses, exacerbated by the depreciation of the yen.
Market Impact of This Partnership
Nippon Shinyaku’s revenue is expected to grow following its licensing agreement with REGENXBIO for the development and commercialization of RGX-121 and RGX-111. As part of the agreement, Nippon Shinyaku will lead the commercialization efforts in the United States and Asia, key markets that could drive significant sales and contribute to future revenue expansion. At the same time, the partnership allows REGENXBIO to expand its market reach through a well-established commercial partner, while benefiting from significant financial incentives, including upfront payments and milestone-based fees. RGX-121 is positioned to become the first approved gene therapy for MPS II, with potential FDA approval anticipated as early as late 2025. Meanwhile, RGX-111 has shown encouraging results in its Phase 1/2 clinical trial, underscoring its promise as a treatment for MPS I.
“RGX-121 could become the first approved gene therapy for MPS II by late 2025, while RGX-111 shows promise for treating MPS I.”