US-based drugmaker Regenxbio and Japan’s Nippon Shinyaku have entered a strategic partnership for the development and commercialisation of RGX-121 and RGX-111.
RGX-121 is a potential one-time AAV therapeutic for the treatment of boys with Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.
RGX-111 leverages the AAV9 vector to deliver the α-l-iduronidase (IDUA) gene to the central nervous system (CNS) to treat Mucopolysaccharidosis I (MPS I), or Hurler syndrome.
Under the terms of the deal, Regenxbio will receive a $110m in upfront payment, and up to $700m in milestone payments.
The milestone payments include $40m in potential development and regulatory milestones and $660m in potential sales milestones.
In addition, Regenxbio will receive double-digit royalties on sales in the US and Asia.
The proposed acquisition is expected to be completed by the end of the first quarter of 2025, subject to customary conditions, including applicable regulatory approvals.
Regenxbio president and CEO Curran Simpson said: “This partnership with Nippon Shinyaku is exciting in that it maximises our collective strengths and enables access of two potentially transformational medicines to key markets.
“The structure of the agreement allows us to leverage our expertise in gene therapy manufacturing while also capturing milestones and a meaningful share of future product revenues.
“RGX-121 is poised to be the first gene therapy for MPS II with potential FDA approval as early as late 2025, and RGX-111 has demonstrated very promising results in Phase 1/2 study.
“With Nippon Shinyaku’s expertise in rare disease and strong commercial capabilities, we look forward to working together to get both of these promising candidates across the finish line for patients.”
As per the agreement, Nippon Shinyaku will commercialise both RGX-121 and RGX-111 in the US and Asia (licensed territory).
Regenxbio will be responsible for the future clinical development and manufacturing of the two products, along with their commercial supply in the licensed territory.
The US drugmaker also reserves the right to develop and commercialise these products in countries outside of the Licensed Territory.
Furthermore, Regenxbio will retain all 100% rights and any proceeds related to the sale of the Priority Review Voucher (PRV) for RGX-121, received upon potential approval.
Nippon Shinyaku president and representative director Toru Nakai said: “RGX-121 and RGX-111 represent one-time gene therapies that can potentially change the course of MPS disease, and we are very pleased to be partnering with Regenxbio, experts in gene therapy development and manufacturing.
“We are confident these therapies can bring tremendous value to those living with MPS II and I.”
