REGENXBIO and Nippon Shinyaku have formed a strategic partnership for the development and commercialization of RGX-121 for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome.
Under the terms of the agreement, REGENXBIO will receive $110 million at closing and up to an additional $700 million if certain milestones are achieved, consisting of $40 million in potential development and regulatory milestones and $660 million in potential sales milestones. REGENXBIO will also receive meaningful double-digit royalties on net sales in the U.S. and Asia (the “Licensed Territory”).
“This partnership with Nippon Shinyaku is exciting in that it maximizes our collective strengths and enables access of two potentially transformational medicines to key markets,” said Curran M. Simpson, President and Chief Executive Officer, REGENXBIO. “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.”
“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,” said Toru Nakai, President and Representative Director of Nippon Shinyaku. “We are confident these therapies can bring tremendous value to those living with MPS II and I.”
Per the agreement, Nippon Shinyaku will commercialize both products in the Licensed Territory and future clinical development of RGX-121 and RGX-111 will be led by REGENXBIO. REGENXBIO retains all rights to, and 100 percent of any proceeds related to the sale of, the Priority Review Voucher (PRV) for RGX-121 received upon potential approval.
REGENXBIO will lead the manufacturing of both products for clinical and commercial supply in the Licensed Territory. REGENXBIO reserves the right to develop and commercialize these products in countries outside of the Licensed Territory.
