- REGENXBIO to receive $110 million upfront, potential milestone payments of up to $700 million and meaningful double-digit royalties on net sales, and lead manufacturing
- Nippon Shinyaku to lead commercialization of first potential gene therapies for Mucopolysaccharidosis II (MPS II) and Mucopolysaccharidosis I (MPS I) in U.S. and Asia
- REGENXBIO retains rights to RGX-121 Priority Review Voucher (PRV) with potential accelerated approval expected in 2025; rolling BLA submission underway
ROCKVILLE, MD, USA I January 14, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) and Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) today announced 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 (collectively, 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.
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.
SOURCE: REGENXBIO
Post Views: 140
- REGENXBIO to receive $110 million upfront, potential milestone payments of up to $700 million and meaningful double-digit royalties on net sales, and lead manufacturing
- Nippon Shinyaku to lead commercialization of first potential gene therapies for Mucopolysaccharidosis II (MPS II) and Mucopolysaccharidosis I (MPS I) in U.S. and Asia
- REGENXBIO retains rights to RGX-121 Priority Review Voucher (PRV) with potential accelerated approval expected in 2025; rolling BLA submission underway
ROCKVILLE, MD, USA I January 14, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) and Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) today announced 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 (collectively, 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.
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.
SOURCE: REGENXBIO
Post Views: 140
