REGENXBIO and Nippon Shinyaku Announce Exclusive Partnership to Develop and Commercialize RGX-121 and RGX-111 for MPS Diseases
REGENXBIO and Nippon Shinyaku have announced a strategic partnership for developing and commercializing RGX-121 and RGX-111, gene therapies for MPS II (Hunter syndrome) and MPS I (Hurler syndrome) respectively. The deal includes a $110 million upfront payment to REGENXBIO, with potential milestone payments of up to $700 million ($40M in development/regulatory milestones and $660M in sales milestones).
REGENXBIO will receive double-digit royalties on net sales in the U.S. and Asia, lead manufacturing operations, and retain rights to the RGX-121 Priority Review Voucher. Nippon Shinyaku will lead commercialization efforts in these territories. RGX-121's FDA approval is anticipated by late 2025, with a rolling BLA submission currently underway. The transaction is expected to close by Q1 2025.
REGENXBIO e Nippon Shinyaku hanno annunciato una partnership strategica per lo sviluppo e la commercializzazione di RGX-121 e RGX-111, terapie geniche per la MPS II (sindrome di Hunter) e la MPS I (sindrome di Hurler) rispettivamente. L'accordo prevede un pagamento iniziale di 110 milioni di dollari a REGENXBIO, con potenziali pagamenti legati a traguardi fino a 700 milioni di dollari (40 milioni di dollari in traguardi di sviluppo/regolamentazione e 660 milioni di dollari in traguardi di vendita).
REGENXBIO riceverà royalties a doppia cifra sulle vendite nette negli Stati Uniti e in Asia, guiderà le operazioni di produzione e manterrà i diritti sul Voucher di Priorità di Revisione per RGX-121. Nippon Shinyaku guiderà gli sforzi di commercializzazione in questi territori. Si prevede che l'approvazione della FDA per RGX-121 arrivi entro la fine del 2025, con una presentazione della BLA già in corso. La transazione dovrebbe chiudersi entro il primo trimestre del 2025.
REGENXBIO y Nippon Shinyaku han anunciado una asociación estratégica para desarrollar y comercializar RGX-121 y RGX-111, terapias génicas para la MPS II (síndrome de Hunter) y la MPS I (síndrome de Hurler) respectivamente. El acuerdo incluye un pago inicial de 110 millones de dólares a REGENXBIO, con pagos por hitos potenciales de hasta 700 millones de dólares (40 millones en hitos de desarrollo/regulatorios y 660 millones en hitos de ventas).
REGENXBIO recibirá regalías de dos dígitos sobre las ventas netas en EE. UU. y Asia, liderará las operaciones de fabricación y retendrá los derechos sobre el Voucher de Revisión Prioritaria de RGX-121. Nippon Shinyaku liderará los esfuerzos de comercialización en estos territorios. Se anticipa que la aprobación de la FDA para RGX-121 llegará a finales de 2025, con una presentación de BLA en curso. Se espera que la transacción se complete para el primer trimestre de 2025.
REGENXBIO와 Nippon Shinyaku는 MPS II(헌터 증후군) 및 MPS I(허를 증후군)을 위한 유전자 요법인 RGX-121 및 RGX-111의 개발 및 상용화를 위한 전략적 파트너십을 발표했습니다. 이 계약에는 REGENXBIO에 대한 1억 1천만 달러의 선불 지급이 포함되며, 최대 7억 달러의 이정표 지급이 가능합니다(개발/규제 이정표 4천만 달러와 판매 이정표 6억 6천만 달러).
REGENXBIO는 미국과 아시아의 순매출에 대해 두 자릿수 로열티를 받고, 제조 운영을 주도하며, RGX-121 우선 리뷰 바우처에 대한 권리를 유지합니다. Nippon Shinyaku는 이러한 지역에서 상용화 노력을 주도할 것입니다. RGX-121의 FDA 승인은 2025년 말로 예상되며, 현재 롤링 BLA 제출이 진행 중입니다. 거래는 2025년 1분기까지 완료될 예정입니다.
REGENXBIO et Nippon Shinyaku ont annoncé un partenariat stratégique pour le développement et la commercialisation de RGX-121 et RGX-111, des thérapies géniques pour la MPS II (syndrome de Hunter) et la MPS I (syndrome de Hurler) respectivement. L'accord comprend un paiement initial de 110 millions de dollars à REGENXBIO, avec des paiements d'étape potentiels pouvant atteindre 700 millions de dollars (40 millions de dollars en étapes de développement/réglementaires et 660 millions de dollars en étapes de vente).
REGENXBIO recevra des redevances à deux chiffres sur les ventes nettes aux États-Unis et en Asie, dirigera les opérations de fabrication et conservera les droits sur le Bon de Révision Prioritaire RGX-121. Nippon Shinyaku dirigera les efforts de commercialisation dans ces territoires. L'approbation de la FDA pour RGX-121 est attendue d'ici la fin de 2025, avec une soumission de BLA en cours. La transaction devrait être finalisée d'ici le premier trimestre de 2025.
REGENXBIO und Nippon Shinyaku haben eine strategische Partnerschaft zur Entwicklung und Kommerzialisierung von RGX-121 und RGX-111, Gentherapien für MPS II (Hunter-Syndrom) und MPS I (Hurler-Syndrom), bekannt gegeben. Der Deal umfasst eine Vorauszahlung von 110 Millionen Dollar an REGENXBIO, mit potenziellen Meilensteinzahlungen von bis zu 700 Millionen Dollar (40 Millionen Dollar in Entwicklungs-/Regulierungsmeilensteinen und 660 Millionen Dollar in Verkaufsmeilensteinen).
REGENXBIO erhält zweistellige Lizenzgebühren auf die Nettoumsätze in den USA und Asien, wird die Produktionsoperationen leiten und die Rechte am RGX-121 Priority Review Voucher behalten. Nippon Shinyaku wird die Kommerzialisierungsbemühungen in diesen Regionen leiten. Die FDA-Zulassung für RGX-121 wird bis Ende 2025 erwartet, wobei derzeit eine rollierende BLA-Einreichung im Gange ist. Der Abschluss der Transaktion wird bis zum ersten Quartal 2025 erwartet.
- Substantial upfront payment of $110 million
- Potential for additional $700 million in milestone payments
- Meaningful double-digit royalties on future sales
- Retention of valuable Priority Review Voucher rights
- Expected FDA approval for RGX-121 by late 2025
- Strategic partnership reduces commercialization costs while maintaining manufacturing control
- Giving up commercialization rights in key markets of U.S. and Asia
- Deal completion subject to regulatory approvals and conditions
Insights
This partnership represents a major financial boost for REGENXBIO, with an immediate
The retention of the Priority Review Voucher (PRV) rights is a strategic win, as PRVs typically sell for
This partnership has profound implications for the rare disease treatment landscape. RGX-121 is positioned to potentially become the first-ever gene therapy for MPS II, targeting a devastating lysosomal storage disorder that currently lacks curative options. The rolling BLA submission and potential 2025 approval timeline suggests strong clinical data.
The deal's structure is particularly clever from a technical perspective - REGENXBIO maintains control over the complex manufacturing process, which is important for gene therapy success, while leveraging Nippon Shinyaku's rare disease commercial infrastructure. Think of it as keeping the "engine" (manufacturing) in-house while partnering on the "delivery system" (commercialization).
This deal transforms REGENXBIO's market position in the rare disease space. The MPS II market alone is estimated at
The timing is strategic - entering 2025 with a strong cash position and potential near-term catalysts (FDA approval, PRV sale) could drive significant value creation. The deal provides immediate validation of REGENXBIO's platform while de-risking commercialization through an experienced partner. For investors, this creates a clearer path to market with multiple value-generating milestones ahead.
- REGENXBIO to receive
$110 million $700 million - Nippon Shinyaku to lead commercialization of first potential gene therapies for Mucopolysaccharidosis II (MPS II) and Mucopolysaccharidosis I (MPS I) in
U.S. andAsia - REGENXBIO retains rights to RGX-121 Priority Review Voucher (PRV) with potential accelerated approval expected in 2025; rolling BLA submission underway
Under the terms of the agreement, REGENXBIO will receive
"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.
The transaction is expected to close by the end of the first quarter of 2025, subject to the customary conditions, including applicable regulatory approvals.
About RGX-121
RGX-121 is a potential one-time AAV therapeutic for the treatment of boys with MPS II. RGX-121 expressed protein is structurally identical to normal I2S. Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted I2S beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS.
RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the U.S. Food and Drug Administration and advanced therapy medicinal products (ATMP) classification from the European Medicines Agency.
About RGX-111
RGX-111 is designed to use the AAV9 vector to deliver the α-l-iduronidase (IDUA) gene to the central nervous system (CNS). By providing rapid IDUA delivery to the brain, RGX-111 could potentially help prevent the progression of cognitive deficits that otherwise occurs in MPS I patients. Positive interim data from a Phase I/II trial of RGX-111 were reported in February 2023. RGX-111 has received orphan drug product, rare pediatric disease and Fast Track designations from the U.S. Food and Drug Administration (FDA).
ABOUT REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.regenxbio.com.
ABOUT NIPPON SHINYAKU
Based on Nippon Shinyaku's business philosophy, "Helping people lead healthier, happier lives," we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness. Please visit our website (www.nippon-shinyaku.co.jp/english/) for products or detailed information.
FORWARD-LOOKING STATEMENTS
This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, clinical trials, and regulatory plans. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the anticipated completion of REGENXBIO's proposed transaction with Nippon Shinyaku, the outcome of REGENXBIO's proposed collaboration with Nippon Shinyaku, whether the milestones contemplated by the proposed transaction will be achieved, the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2023, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the
Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
George E. MacDougall
Investor Relations
IR@regenxbio.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-and-nippon-shinyaku-announce-exclusive-partnership-to-develop-and-commercialize-rgx-121-and-rgx-111-for-mps-diseases-302350270.html
SOURCE REGENXBIO Inc.
FAQ
What is the total value of RGNX's partnership deal with Nippon Shinyaku?
When is RGX-121's expected FDA approval date for MPS II treatment?
What territories are included in RGNX's partnership with Nippon Shinyaku?
Who will handle manufacturing for RGX-121 and RGX-111 under the partnership?
What are the milestone payment components in the RGNX-Nippon Shinyaku deal?
