Regenxbio licenses gene therapies to Nippon Shinyaku Japan

Regenxbio, a well-known entity in the gene therapy industry, has entered into a significant agreement with Japan's Nippon Shinyaku. This collaboration grants Nippon Shinyaku certain rights to two of Regenxbio's experimental treatments. The deal is financially substantial, securing Regenxbio an initial payment of $110 million, with the potential for an additional $700 million in milestone fees based mainly on future sales performance. In exchange, Nippon Shinyaku gains the rights to market these treatments in both the United States and various Asian markets.

One of the promising treatments in this deal targets Hunter syndrome, a progressive disorder that impacts multiple bodily systems. This particular therapy could receive approval in the United States as early as the end of this year. If approved, Regenxbio would maintain the rights to any priority review voucher issued by the Food and Drug Administration, a valuable asset that acts as a regulatory fast track for future treatments.

Regenxbio has a rich history of collaboration within the gene therapy field. It has previously partnered with significant companies such as Eli Lilly, Novartis, and Astellas, licensing its innovative technology. In this latest arrangement with Nippon Shinyaku, Regenxbio is transferring partial responsibility for its most advanced therapy, RGX-121. The company is currently navigating the U.S. approval process for this Hunter syndrome treatment, anticipating completion within this quarter.

RGX-121, if approved, would be the pioneering gene therapy for Hunter syndrome, a condition known for its progressive nature and extensive symptomatology. Regenxbio has conducted multi-phase trials to evaluate RGX-121's impact, particularly focusing on a biomarker linked to brain disease activity. An approval could provide Regenxbio with a crucial priority review voucher, which can be sold for substantial sums, often around $100 million. As per the agreement with Nippon Shinyaku, Regenxbio retains full rights to this voucher and any proceeds from its sale.

The second treatment involved in this deal, RGX-111, is being developed for another variant of mucopolysaccharidosis known as Hurler syndrome. This treatment is currently in the earlier stages of clinical testing. Under the terms of their agreement, Regenxbio will continue to spearhead the clinical development for both RGX-121 and RGX-111. Additionally, Regenxbio is tasked with manufacturing these products for Nippon Shinyaku, although it retains the rights to sell them outside of the United States and Asia.

This agreement marks Regenxbio's first major deal since the transition in leadership, following the departure of longtime CEO Kenneth Mills. Curran Simpson, who took over as CEO on July 1, now oversees the company as it continues to navigate the complexities of the gene therapy landscape and seeks to expand its footprint in the market through strategic collaborations like the one with Nippon Shinyaku.