Regenxbio, a leading US biopharmaceutical company, has entered into a significant collaboration with Japan’s Nippon Shinyaku to develop and commercialize novel gene therapies for rare genetic disorders. The two companies are focusing on RGX-121 and RGX-111, which target Mucopolysaccharidosis II (MPS II) and Mucopolysaccharidosis I (MPS I), respectively.
RGX-121 is a promising gene therapy designed to treat MPS II, commonly known as Hunter syndrome, in boys. RGX-111, on the other hand, utilizes the AAV9 vector to deliver the α-l-iduronidase (IDUA) gene to the central nervous system, offering a potential treatment for MPS I, also referred to as Hurler syndrome.
Key Terms of the Deal
Under the partnership agreement, Regenxbio will receive an upfront payment of $110 million, along with the opportunity to earn up to $700 million in milestone payments. These payments are divided into $40 million for potential development and regulatory milestones and $660 million for potential sales milestones. Additionally, Regenxbio will receive double-digit royalties on product sales within the US and Asia.
The agreement is expected to be finalized by the end of the first quarter of 2025, pending regulatory approval and customary closing conditions.
Strengthening Expertise in Gene Therapy
Regenxbio President and CEO Curran Simpson expressed excitement about the partnership, emphasizing the synergistic strengths of both companies. “This collaboration maximizes our collective capabilities and facilitates access to two potentially transformative gene therapies in key markets,” Simpson said. “RGX-121 is on track to become the first gene therapy for MPS II, with potential FDA approval as early as late 2025, while RGX-111 has shown promising results in Phase 1/2 trials.”
He further highlighted the importance of Regenxbio’s expertise in gene therapy manufacturing and the potential for capturing substantial future product revenues, alongside milestones.
Nippon Shinyaku’s Role in Commercialization
As part of the agreement, Nippon Shinyaku will be responsible for the commercialization of RGX-121 and RGX-111 in the US and Asia, which are designated as the licensed territories. Regenxbio will retain responsibility for future clinical development, manufacturing, and commercial supply in these regions. Moreover, Regenxbio maintains the right to develop and market the products outside the licensed territories.
Regenxbio will also retain all rights and proceeds from the sale of any Priority Review Voucher (PRV) granted to RGX-121 upon potential FDA approval.
A Transformative Opportunity for Patients
Nippon Shinyaku President and Representative Director Toru Nakai voiced confidence in the potential of RGX-121 and RGX-111, stating, “These one-time gene therapies have the potential to transform the treatment of MPS diseases. We are pleased to collaborate with Regenxbio, experts in gene therapy development and manufacturing, to bring these innovative therapies to patients in need.”
Both companies are optimistic that their combined efforts will bring significant benefits to individuals living with MPS II and MPS I, rare and serious diseases that currently have limited treatment options.
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