REGENXBIO Announces Positive Data from Pivotal Dose Level of RGX-121 Demonstrating Long-Term Systemic Effect
REGENXBIO (Nasdaq: RGNX) announced positive results from the Phase I/II/III CAMPSIITE® trial of RGX-121 for treating Mucopolysaccharidosis Type II (MPS II), or Hunter syndrome. The pivotal dose level demonstrated an 85% median reduction in cerebrospinal fluid levels of heparan sulfate D2S6, a key biomarker of brain disease activity, sustained for up to two years. Notably, 80% of patients at the pivotal dose level were enzyme replacement therapy (ERT)-free at the last time point, up to 18 months post-dosing.
The trial met its primary endpoint with statistical significance, and patients exceeded expectations in neurodevelopmental function compared to natural history data. REGENXBIO plans to initiate a rolling Biologics License Application submission using the accelerated approval pathway in Q3 2024, potentially leading to a Priority Review Voucher in 2025.
REGENXBIO (Nasdaq: RGNX) ha annunciato risultati positivi dallo studio clinico di fase I/II/III CAMPSIITE® relativo a RGX-121 per il trattamento della Mucopolisaccaridosi di tipo II (MPS II), nota anche come sindrome di Hunter. Il livello di dose chiave ha dimostrato una riduzione mediana dell'85% nei livelli di heparan sulfate D2S6 nel liquido cerebrospinale, un biomarker cruciale dell'attività della malattia cerebrale, mantenuta fino a due anni. È degno di nota che l'80% dei pazienti al livello di dose fondamentale erano liberi da terapia enzimatica sostitutiva (ERT) all'ultimo punto di misurazione, fino a 18 mesi dopo la somministrazione.
Lo studio ha raggiunto il suo obiettivo primario con significatività statistica, e i pazienti hanno superato le aspettative in termini di funzione neuroevolutiva rispetto ai dati della storia naturale. REGENXBIO prevede di avviare una presentazione di domanda di licenza biologica in modo progressivo utilizzando il percorso di approvazione accelerata nel Q3 2024, con la possibilità di ottenere un Voucher per Revisione Prioritaria nel 2025.
REGENXBIO (Nasdaq: RGNX) anunció resultados positivos del ensayo clínico de fase I/II/III CAMPSIITE® sobre RGX-121 para tratar la Mucopolisacaridosis Tipo II (MPS II), o síndrome de Hunter. El nivel de dosis pivotal mostró una reducción mediana del 85% en los niveles de heparan sulfate D2S6 en el líquido cefalorraquídeo, un biomarcador clave de la actividad de la enfermedad cerebral, mantenida durante hasta dos años. Notablemente, el 80% de los pacientes en el nivel de dosis pivotal estaban libres de terapia de reemplazo enzimático (ERT) en el último punto de tiempo, hasta 18 meses después de la dosificación.
El ensayo cumplió su objetivo principal con significancia estadística, y los pacientes superaron las expectativas en función neurodesarrollo en comparación con los datos de la historia natural. REGENXBIO planea iniciar la presentación de una solicitud de licencia biológica en forma continua utilizando el camino de aprobación acelerada en Q3 2024, lo que podría llevar a un Voucher de Revisión Prioritaria en 2025.
REGENXBIO (Nasdaq: RGNX)는 Mucopolysaccharidosis Type II (MPS II), 즉 Hunter 증후군 치료를 위한 RGX-121의 1/2/3상 CAMPSIITE® 임상 시험 결과가 긍정적이라고 발표했습니다. 주요 용량 수준에서는 뇌 질환 활성의 주요 바이오마커인 heparan sulfate D2S6의 뇌척수액에서 85%의 중앙 감소가 나타났으며, 이는 최대 2년 동안 지속되었습니다. 특히, 80%의 환자가 마지막 측정 시점에서 치료 효소 대체 요법 (ERT) 없이 18개월까지 유지되었습니다.
이 시험은 통계적 의미를 갖는 주요 목표를 달성했으며, 환자들의 신경 발달 기능이 자연 이력 데이터와 비교했을 때 기대를 초과했습니다. REGENXBIO는 2024년 3분기에 가속화 승인을 통한 생물학적 라이센스 신청을 시작할 계획이며, 2025년에는 우선 심사 바우처 가능성이 있습니다.
REGENXBIO (Nasdaq: RGNX) a annoncé des résultats positifs de l'essai clinique de Phase I/II/III CAMPSIITE® de RGX-121 pour le traitement de la Mucopolysaccharidose de type II (MPS II), ou syndrome de Hunter. Le niveau de dose clé a démontré une réduction médiane de 85% des niveaux de sulfate d'héparane D2S6 dans le liquide céphalo-rachidien, un biomarqueur essentiel de l'activité de la maladie cérébrale, maintenue jusqu'à deux ans. Il est notable que 80% des patients au niveau de dose clé étaient sans thérapie de remplacement enzymatique (ERT) au dernier point de mesure, jusqu'à 18 mois après la dose.
L'essai a atteint son objectif principal avec une signification statistique, et les patients ont dépassé les attentes en matière de fonction neurodéveloppementale par rapport aux données de l'histoire naturelle. REGENXBIO prévoit de commencer une soumission de demande de licence biologique progressive en utilisant la voie d'approbation accélérée au T3 2024, ce qui pourrait conduire à un Voucher de Révision Prioritaire en 2025.
REGENXBIO (Nasdaq: RGNX) hat positive Ergebnisse aus der Phase I/II/III-Studie CAMPSIITE® zu RGX-121 zur Behandlung der Mukopolisaccharidose Typ II (MPS II), auch bekannt als Hunter-Syndrom, bekannt gegeben. Die entscheidende Dosisstufe zeigte eine medianen Reduktion von 85% der Heparansulfat D2S6-Spiegel im Liquor cerebrospinalis, einem wichtigen Biomarker für die Aktivität der Gehirnerkrankung, die bis zu zwei Jahre anhielt. Erwähnenswert ist, dass 80% der Patienten bei der entscheidenden Dosisstufe zum letzten Zeitpunkt bis zu 18 Monate nach der Dosierung keine Enzymersatztherapie (ERT) benötigten.
Die Studie erreichte ihren primären Endpunkt mit statistischer Signifikanz, und die Patienten übertrafen die Erwartungen in Bezug auf die neuroentwicklungsfunktion im Vergleich zu Daten aus der natürlichen Geschichte. REGENXBIO plant, im Q3 2024 eine laufende Einreichung eines Antrags auf biologische Lizenz über den beschleunigten Genehmigungsweg zu starten, was möglicherweise zu einem Priority Review Voucher im Jahr 2025 führen könnte.
- 85% median reduction in CSF levels of HS D2S6, a key biomarker of brain disease activity, sustained for up to two years
- 80% of patients at pivotal dose level were ERT-free at last time point, up to 18 months post-dosing
- Trial met primary endpoint with statistical significance
- Patients exceeded expectations in neurodevelopmental function compared to natural history data
- On track for rolling BLA submission in Q3 2024 using accelerated approval pathway
- Potential to receive Priority Review Voucher in 2025
- None.
Insights
The data from REGENXBIO's CAMPSIITE trial for RGX-121 in MPS II (Hunter syndrome) is highly promising. The 85% median reduction in CSF levels of HS D2S6, a key biomarker of brain disease activity, approaching normal levels and sustained for up to two years, is particularly significant. This suggests a long-term systemic effect that could potentially alter the course of this devastating disease.
Moreover, the fact that 80% of patients at the pivotal dose level were able to discontinue or remain naïve to enzyme replacement therapy (ERT) is a game-changer. This indicates that RGX-121 might not only address the neurocognitive decline associated with MPS II but also potentially replace the current standard of care.
The consistent safety profile across 25 patients is reassuring, but ongoing monitoring will be important as the therapy progresses towards potential approval.
REGENXBIO's announcement is a significant milestone that could substantially impact its market position. The positive data from the pivotal dose level of RGX-121 positions the company to potentially capture a share of the MPS II treatment market, estimated to be worth over
The planned BLA submission in Q3 2024 using the accelerated approval pathway could lead to faster market entry. If approved, RGX-121 could become the first gene therapy for MPS II, potentially commanding premium pricing. The possibility of receiving a Priority Review Voucher in 2025 is an additional value driver, as these vouchers can be sold for
Investors should monitor the BLA submission process and any updates on pricing and reimbursement strategies, as these will be critical for commercial success.
- Data from pivotal dose level demonstrates long-term, sustained reductions in CSF levels of HS D2S6, a key biomarker of brain disease in MPS II
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- Submission of a rolling BLA using the accelerated approval pathway on track for Q3 2024
The totality of evidence from the CAMPSIITE trial continues to support RGX-121 as the potential first gene therapy and one-time treatment for MPS II. In
"As we quickly approach the BLA filing for RGX-121, we are very pleased with the data presented at SSIEM demonstrating encouraging evidence of systemic activity and long-term reductions of CSF D2S6," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "The data continue to support that by restoring the gene missing in boys with Hunter syndrome, RGX-121 changes the course of disease and has the potential to significantly improve both vital brain function and the systemic manifestations of this devastating disease."
Data Summary
In new, long-term data from the Phase I/II/III CAMPSIITE® trial, patients receiving RGX-121 at the pivotal dose level demonstrated an
In the dose-finding part of the trial, investigators chose to discontinue standard-of-care intravenous enzyme replacement therapy (ERT) or to remain ERT-naïve for a majority of patients. At the pivotal dose level (dose level 3),
As of January 5, 2024, RGX-121 continues to be well tolerated in 25 patients dosed across all phases of the CAMPSIITE trial.
"A potential one-time treatment that can allow these boys to exceed the natural history of this disease in their neurocognitive development, as well as the ability to remain off enzyme replacement therapy for multiple years represents a meaningful option for patients and their families," said Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA,
REGENXBIO is on track to initiate a rolling Biologics License Application (BLA) submission using the accelerated approval pathway in the third quarter of 2024 using CSF D2S6 as a surrogate endpoint reasonable likely to predict clinical benefit. Approval of the planned BLA could result in receipt of a Priority Review Voucher in 2025.
Data presented is available on the "Publications" section of the REGENXBIO website at WWW.REGENXBIO.COM.
About the CAMPSIITE® Trial
CAMPSIITE is a Phase I/II/III multicenter, open-label trial for boys aged four months up to five years with neuronopathic MPS II. The primary endpoint of the trial is measurement of CSF GAGs. Accurate and sensitive measurements of CSF GAGs, such as HS D2S6, have the potential to be considered a surrogate endpoint that is reasonably likely to predict clinical benefit in MPS II disease under the accelerated approval pathway, as buildup of GAGs in the CSF of MPS II patients correlates with clinical manifestations including neurodevelopmental deficits.
The pivotal program is using commercial-scale cGMP material from REGENXBIO's proprietary, high-yielding suspension-based manufacturing process, named NAVXpress™. In addition to measuring GAGs in the CSF, the trial will continue to collect neurodevelopmental data and caregiver-reported outcomes.
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. RGX-21 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
About Mucopolysaccharidosis Type II (MPS II)
MPS II, or Hunter Syndrome, is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase (I2S) leading to an accumulation of glycosaminoglycans (GAGs), including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction, including in the central nervous system (CNS). MPS II is estimated to occur in 1 in 100,000 to 170,000 births. In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Specific treatment to address the neurological manifestations of MPS II remains a significant unmet medical need. Key biomarkers of I2S enzymatic activity in MPS II patients include its substrate HS D2S6, which has been shown to correlate with neurocognitive manifestations of the disorder.
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.
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 and clinical trials. 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 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
Zolgensma® is a registered trademark of Novartis Gene Therapies. All other trademarks referenced herein are registered trademarks of REGENXBIO.
Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
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SOURCE REGENXBIO Inc.
FAQ
What is the efficacy of RGX-121 in reducing CSF levels of HS D2S6 in MPS II patients?
How many patients treated with RGX-121 were able to discontinue enzyme replacement therapy (ERT)?
When does REGENXBIO (RGNX) plan to submit a Biologics License Application (BLA) for RGX-121?
What potential regulatory benefits could RGX-121 receive if approved?
