REGENXBIO today announced that it will delay the initiation of dosing in the Phase I/II AFFINITY DUCHENNETM trial studying RGX-202 in participants with Duchenne, due to an unexpected observation in the final stages of manufacturing at one of its third-party manufacturers. REGENXBIO expects to be in clinic and dosing their first participant in 2023. While the manufacturing issue is resolved they will continue readying clinical trial sites in anticipation of starting the trial next year.
RGX-202 is an investigational gene therapy product, utilizing a novel adeno-associated virus (AAV8) to transport a shortened version of the dystrophin gene (micro-dystrophin) that may provide benefit in place of the missing full-length dystrophin protein.
Read the Press Release from REGENXBIO:
REGENXBIO REPORTS FIRST QUARTER 2022 FINANCIAL RESULTS AND RECENT OPERATIONAL HIGHLIGHTS
May 4, 2022 at 4:05 PM EDT
- Continues to advance RGX-314 program for the treatment of wet AMD and diabetic retinopathy; remains on track for first BLA filing in 2024
- Enrollment ongoing in the pivotal ATMOSPHERE™ and ASCENT™ clinical trials of RGX-314 for the treatment of wet AMD using subretinal delivery
- Enrollment ongoing in AAVIATE® trial of RGX-314 for the treatment of wet AMD using suprachoroidal delivery
- Enrollment complete in ALTITUDE™ trial of RGX-314 for the treatment of diabetic retinopathy using suprachoroidal delivery
- Today announced update on initiation of AFFINITY DUCHENNE™ trial of RGX-202 for the treatment of Duchenne
- Continues plans for expanded enrollment of RGX-121 and RGX-111 clinical trials for the treatment of MPS II and MPS I, respectively
- In-house cGMP manufacturing facility is operational, allowing for high-yield manufacturing of AAV Therapeutics up to 2,000L scale
- $765 million in cash, cash equivalents and marketable securities as of March 31, 2022; operational runway into 2025
- Conference call Wednesday, May 4th at 4:30 p.m. ET
REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results for the first quarter ended March 31, 2022, and recent operational highlights.
“We are encouraged by our progress this year as we work to advance the development of our leading AAV Therapeutics pipeline,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “As we continue to enroll patients in two pivotal trials, RGX-314 continues to be on track for a 2024 BLA submission. We are collaborating with AbbVie on our subretinal and suprachoroidal trials for the treatment of wet AMD and diabetic retinopathy, as we aim to make a significant impact for the millions of patients suffering from vision loss associated with these retinal diseases.”
Mr. Mills continued: “We remain deeply committed to developing programs for rare diseases. Though we made the difficult decision to delay dosing patients in our RGX-202 trial, we continue to prepare for trial initiation. Plans for the expanded enrollment in our MPS II and MPS I clinical trials continue, and we look forward to sharing further updates on these trials in the second half of the year. Additionally, our state-of-the-art, in-house cGMP manufacturing facility is operational, and will be instrumental in advancing our programs. We look forward to leveraging our strong cash position to achieve our recently announced ‘5x’25’ goal of progressing five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.”
Program Highlights and Milestones
RGX-202: RGX-202 is an investigational one-time AAV Therapeutic for the treatment of Duchenne Muscular Dystrophy (Duchenne), using the NAV AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-Terminal (CT) domain as well as a muscle specific promoter to support a targeted therapy for improved resistance to muscle damage associated with Duchenne.
- REGENXBIO today announced that it has taken proactive measures that will result in the delayed dosing of the first patient in the Phase I/II AFFINITY DUCHENNE™ trial due to an unexpected observation in the final stages of manufacturing at one of its third-party manufacturers.
- Work preparing for the AFFINITY DUCHENNE trial initiation continues, including readying clinical trial sites and manufacturing additional clinical supply for the upcoming trial; REGENXBIO anticipates dosing the first patient in this trial in the first half of 2023.