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REGENXBIO Provides Update on Progress of Clinical Programs for Rare Genetic Neurodegenerative Diseases
- RGX-111 and RGX-121 continue to be well-tolerated in patients with MPS I and MPS II following one-time intracisternal administration - Company completed
About this update from Regenxbio Inc.
[{"type":"text","content":"- RGX-111 and RGX-121 continue to be well-tolerated in patients with MPS I and MPS II following one-time intracisternal administration\n - Company completed dosing of three patients in Cohort 2 of Phase I/II trial of RGX-121 for the treatment of MPS II; additional interim data expected in second half of 2020\n - Data from a single-patient investigator-initiated IND of RGX-111 for the treatment of MPS I demonstrates encouraging biomarker activity and continued progression in neurocognitive development\n\n\nROCKVILLE, Md., July 8, 2020 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced that it has completed dosing of three patients in Cohort 2 of the Company's Phase I/II study of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II) and reported encouraging data under a single-patient investigator-initiated Investigational New Drug (IND) application for RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I) conducted at CHOC Children's. \n\"We are pleased to have completed the dosing of three patients with MPS II at the second dose level of our RGX-121 Phase I/II study, which included involvement from new leading centers at UCSF Benioff Children's Hospital Oakland and Hospital de Clínicas de Porto Alegre in Brazil. We expect to provide an additional interim data update from the RGX-121 program later this year,\" said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. \"Further, we are encouraged by the initial data from the first patient dosed with RGX-111, and we look forward to advancing our RGX-111 Phase I/II study.\" \nEnrollment in Cohort 2 of the Phase I/II study of RGX-121 is now complete, with three patients with MPS II dosed intracisternally with 6.5x1010 genome copies per gram (GC/g) of brain mass. As of June 24, 2020, RGX-121 is reported to be well-tolerated in patients across two dose levels, with no drug-related serious adverse events (SAEs). Additional data from both cohorts in this study and a program update will be available in the second half of 2020.\nUnder a single-patient investigator-initiated IND for RGX-111, Raymond Wang, M.D., a biochemical genetics specialist at CHOC Children's, dosed a patient wit...