REGENXBIO and Nippon Shinyaku Partner to Develop Gene Therapies for MPS Diseases
Wednesday, January 15, 2025
REGENXBIO and Nippon Shinyaku have formed an exclusive partnership to advance the development and commercialisation of RGX-121 and RGX-111, gene therapies targeting Mucopolysaccharidosis II (MPS II or Hunter syndrome) and Mucopolysaccharidosis I (MPS I or Hurler syndrome), respectively.
Under the agreement, REGENXBIO will receive an upfront payment of $110 million and may earn up to $700 million in milestone payments.
This includes $40 million tied to development and regulatory milestones and $660 million linked to sales milestones.
Additionally, the company will benefit from double-digit royalties on net sales in the United States and Asia, referred to as the Licensed Territory.
The collaboration combines REGENXBIO’s gene therapy manufacturing capabilities with Nippon Shinyaku’s expertise in rare diseases and commercial operations.
The agreement specifies that Nippon Shinyaku will handle commercialisation in the Licensed Territory, while REGENXBIO will lead clinical development and manufacturing for both therapies.
REGENXBIO retains rights outside the Licensed Territory and will have sole control over proceeds from the Priority Review Voucher (PRV) associated with RGX-121 upon potential approval.
RGX-121 is an investigational gene therapy designed for boys with MPS II. By delivering the IDS gene to cells in the central nervous system (CNS), it aims to provide a long-term source of I2S protein, overcoming the blood-brain barrier and promoting cell correction throughout the CNS.
The therapy has received multiple regulatory designations, including Orphan Drug Product, Rare Pediatric Disease, and Fast Track status from the U.S. Food and Drug Administration (FDA), as well as advanced therapy medicinal product (ATMP) classification from the European Medicines Agency.
RGX-111 utilises an AAV9 vector to deliver the IDUA gene to the CNS, enabling rapid delivery of the enzyme to the brain. It is intended to help prevent cognitive decline in patients with MPS I.
Positive interim results from a Phase I/II trial were reported in early 2023. The therapy has also received Orphan Drug Product, Rare Pediatric Disease, and Fast Track designations from the FDA.
The transaction is expected to close by the end of the first quarter of 2025, subject to regulatory approvals.
Source: regenxbio.com
