clemidsogene lanparvovec (RGX-121) / REGENXBIO, Nippon Shinyaku - LARVOL DELTA

RGX-121 (clemidsogene lanparvovec) : an investigational AAV Gene Therapy for the Treatment of Neuronopathic Mucopolysaccharidosis Type II (ASGCT 2025) - P2/3 | "We will discuss the outcome of CAMPSIITE™, a phase I/II/III, open-label trial involving 25 nMPS II patients treated with RGX-121 (NCT03566043). Having completed this initial clinical study, we are currently seeking commercial authorization using the accelerated approval pathway."

Gene therapy • CNS Disorders • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

REGENXBIO Announces FDA Acceptance and Priority Review of the BLA for RGX-121 for MPS II (GlobeNewswire) - "REGENXBIO Inc...today announced the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) seeking accelerated approval for clemidsogene lanparvovec (RGX-121) for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome....The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of November 9, 2025."

FDA filing • PDUFA • Priority review • Hunter Syndrome

REGENXBIO Announces Closing of Strategic Partnership with Nippon Shinyaku for MPS Diseases (PRNewswire) - "REGENXBIO Inc...today announced the closing of its previously announced strategic partnership with Nippon Shinyaku....Under the terms of the agreement, REGENXBIO and Nippon Shinyaku will develop and commercialize RGX-121 (clemidsogene lanparvovec) for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome in the United States and Asia."

Licensing / partnership • Hunter Syndrome

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) (clinicaltrials.gov) - P1/2 | N=6 | Completed | Sponsor: REGENXBIO Inc. | Active, not recruiting ➔ Completed | Trial completion date: Nov 2025 ➔ May 2024

Trial completion • Trial completion date • Gene Therapies • Hunter Syndrome

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) (clinicaltrials.gov) - P2/3 | N=48 | Active, not recruiting | Sponsor: REGENXBIO Inc. | Trial completion date: May 2025 ➔ Aug 2025

Trial completion date • Gene Therapies • Hunter Syndrome • Pediatrics

REGENXBIO and Nippon Shinyaku Announce Exclusive Partnership to Develop and Commercialize RGX-121 and RGX-111 for MPS Diseases (PRNewswire) - "REGENXBIO Inc...and Nippon Shinyaku Co., Ltd...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')....REGENXBIO retains rights to RGX-121 Priority Review Voucher (PRV) with potential accelerated approval expected in 2025; rolling BLA submission underway."

FDA approval • Licensing / partnership • Hunter Syndrome • Hurler Syndrome

CAMPSIITE™ PHASE I/II/III: AN INTERIM CLINICAL STUDY UPDATE OF RGX-121, AN INVESTIGATIONAL GENE THERAPY FOR THE TREATMENT OF NEURONOPATHIC MUCOPOLYSACCHARIDOSIS TYPE II (MPS II) (SSIEM 2024) - P2/3 | "RGX-121 has the potential to provide sustained CNS clinical outcomes and additional systemic effects in MPS II patients."

Clinical • Gene therapy • P1/2 data • Gene Therapies • Hunter Syndrome • Rare Diseases

CAMPSIITE™ PHASE I/II/III: AN INTERIM CLINICAL STUDY UPDATE OF RGX-121, AN INVESTIGATIONAL GENE THERAPY FOR THE TREATMENT OF NEURONOPATHIC MUCOPOLYSACCHARIDOSIS TYPE II (MPS II) (SSIEM 2024) - P2/3 | "RGX-121 has the potential to provide sustained CNS clinical outcomes and additional systemic effects in MPS II patients."

Clinical • Gene therapy • P1/2 data • Gene Therapies • Hunter Syndrome • Rare Diseases

CAMPSIITE™ Phase I/II/III: An Interim Clinical Study Update of RGX-121, an Investigational Gene Therapy for the Treatment of Neuronopathic Mucopolysaccharidosis Type II (MPS II) (ASGCT 2024) - P2/3 | "RGX-121 has the potential to provide sustained CNS clinical outcomes and additional systemic effects in MPS II patients."

Clinical • Gene therapy • P1/2 data • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

CAMPSIITE™ Phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II) (SIMD 2024) - P2/3 | "RGX-121 has the potential to provide sustained CNS clinical outcomes and additional systemic effects in MPS II patients."

Clinical • Gene therapy • P1/2 data • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice. (PubMed, Mol Ther Methods Clin Dev) - "In the brain, a dose of 1 × 1011 gc i.t. achieved the highest IDS activity levels and the greatest reduction in GAG content, and it prevented neurocognitive deficiency. We conclude that a dose of 1 × 1010 gc normalized metabolic and skeletal outcomes, while neurologic improvement required a dose of 1 × 1011 gc, thereby suggesting the prospect of a similar direct benefit in humans."

Journal • Preclinical • CNS Disorders • Gene Therapies • Hunter Syndrome • Rare Diseases

RGX-121: AN INVESTIGATIONAL GENE THERAPY FOR THE TREATMENT OF NEURONOPATHIC MUCOPOLYSACCHARIDOSIS TYPE II (MPS II): INTERIM ANALYSIS OF DATA FROM THE FIRST-IN-HUMAN STUDY (WRMC 2024) - P2/3 | "RGX-121 has the potential to provide sustained CNS clinical outcomes and additional systemic effects in MPS II patients."

Gene therapy • P1 data • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) (clinicaltrials.gov) - P2/3 | N=48 | Active, not recruiting | Sponsor: REGENXBIO Inc.

Gene therapy • Trial completion date • Trial primary completion date • Gene Therapies • Hunter Syndrome • Pediatrics

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) (clinicaltrials.gov) - P1/2 | N=6 | Active, not recruiting | Sponsor: REGENXBIO Inc.

Gene therapy • Trial completion date • Trial primary completion date • Gene Therapies • Hunter Syndrome

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) (clinicaltrials.gov) - P2/3 | N=48 | Active, not recruiting | Sponsor: REGENXBIO Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene therapy • Gene Therapies • Hunter Syndrome • Pediatrics

REGENXBIO Announces Presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023 (PRNewswire) - "REGENXBIO...announced presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023 in Jerusalem, Israel (August 29 – September 1). Investigators will deliver encore presentations of interim clinical trial data from REGENXBIO's pipeline of one-time investigational AAV Therapeutics for progressive, neurodegenerative lysosomal storage disorders, as well as initial interim data from the single-patient, investigator-initiated study of RGX-181 for the treatment of CLN2 disease."

Clinical data • CNS Disorders • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

RGX-121 An investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II), an interim analysis of data from the first-in-human study (SSIEM 2023) - P2/3 | "RGX-121 has the potential to provide sustained CNS clinical outcomes and additional systemic effects in MPS II patients."

Gene therapy • P1 data • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

Daily Living Skills on the Vineland Adaptive Behavioral Scale Version 2 (VABS-II) in Neuronopathic Mucopolysaccharidosis Type II (MPS II) (SSIEM 2023) - "RGX-121 has the potential to provide sustained skill acquisition in daily living domains that are applicable to young children with neuronopathic MPS II."

Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

RGX-121 Gene Therapy for the Treatment of Neuronopathic Mucopolysaccharidosis Type II (MPS II): Interim Analysis of Data from the First in Human Study (ASGCT 2023) - P2/3 | "Updated interim results from this trial will be presented. RGX-121 has the potential to provide sustained CNS clinical outcomes and additional systemic effects in MPS II patients."

Gene therapy • P1 data • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) (clinicaltrials.gov) - P1/2 | N=6 | Active, not recruiting | Sponsor: REGENXBIO Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Gene therapy • Gene Therapies • Hunter Syndrome

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) (clinicaltrials.gov) - P1/2 | N=6 | Recruiting | Sponsor: REGENXBIO Inc. | Trial primary completion date: Jun 2022 ➔ May 2023

Trial primary completion date • Gene Therapies • Hunter Syndrome

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) (clinicaltrials.gov) - P2/3 | N=48 | Recruiting | Sponsor: REGENXBIO Inc. | Phase classification: P1/2 ➔ P2/3 | N=18 ➔ 48 | Trial completion date: May 2024 ➔ May 2025 | Trial primary completion date: May 2022 ➔ May 2024

Enrollment change • Phase classification • Trial completion date • Trial primary completion date • Gene Therapies • Hunter Syndrome • Pediatrics

RGX-121 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II (MPS II): Interim Analysis of Data from the First in Human Study (ASGCT 2022) - P1/2 | "Updated interim results from this clinical trial will be presented. RGX-121 has the potential to provide sustained CNS clinical outcomes and additional systemic effects in MPS II patients."

P1 data • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) (clinicaltrials.gov) - P1/2 | N=6 | Recruiting | Sponsor: Regenxbio Inc. | Trial completion date: Sep 2023 ➔ Jan 2024 | Trial primary completion date: Mar 2022 ➔ Jun 2022

Trial completion date • Trial primary completion date • Gene Therapies • Hunter Syndrome

RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome) (clinicaltrials.gov) - P1/2; N=18; Recruiting; Sponsor: Regenxbio Inc.; N=12 ➔ 18; Trial completion date: Dec 2023 ➔ May 2024; Trial primary completion date: Dec 2023 ➔ May 2022

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Gene Therapies • Hunter Syndrome • Pediatrics