Regenxbio Pivotal Trial of RGX-121 for MPS II Achieves Primary Endpoint

 

• Results support BLA submission in 2024 using the accelerated approval pathway
• Primary endpoint of patients achieving reduction in CSF biomarker of MPS II disease was met with statistical significance (p value of 0.00016)
• Patients treated with RGX-121 have showed continued improvement in neurodevelopmental skill acquisition up to four years and discontinued intravenous enzyme therapy
• Company plans to discuss these results as part of a full rare disease program update on its conference call today, Wednesday, February 7, 4:30 p.m. ET

As part of a full rare disease program update, REGENXBIO will host a conference call today, Wednesday, February 7 at 4:30 p.m. ET and will be joined by Dr. Harmatz and Raymond Wang, M.D., Children's Hospital of Orange County to discuss the CAMPSIITE trial pivotal phase topline results and the expedited plan for filing a Biologics License Application using the accelerated approval pathway in 2024.

Listeners can register for the webcast via this link. Analysts wishing to participate in the question and answer session should use this link. A copy of the slides being presented will be available via the Company's investor website. Those who plan on participating are advised to join 15 minutes prior to the start time. A replay of the webcast will also be available via the Company's investor website approximately two hours after the call's conclusion.

https://www.prnewswire.com/news-releases/regenxbio-announces-pivotal-trial-of-rgx-121-for-the-treatment-of-mps-ii-achieves-primary-endpoint-302056283.html