Regenxbio Inc. (NASDAQ:RGNX) Q4 2019 Earnings Conference Call - Final Transcript

Feb 26, 2020 • 04:30 pm ET

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Regenxbio Inc. (NASDAQ:RGNX) Q4 2019 Earnings Conference Call - Final Transcript

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Presentation
Executive
Ken Mills

pipeline across two treatment modalities, AAV-mediated antibody delivery and AAV-mediated monogenic gene replacement. Both modalities utilize our NAV technology platform to develop potential treatments for those suffering from both rare genetic diseases and potentially expanded treatment options for diseases broadly affecting public health.

In 2019, we made great strides broadening our internal gene therapy pipeline and advancing key programs. Last month, we announced updated data for our lead program RGX-314, which is for the treatment of wet age-related macular degeneration, six month data from cohort five of our Phase 1/2a trial. The data continues to demonstrate The data continues to demonstrate meaningful reductions in anti-VEGF, as well as improved visual acuity following a onetime administration of RGX-314. We remain strongly encouraged by the program's clinical results, and we look forward to reporting longer term data from all cohorts from the Phase 1/2a trial in 2020.

Further data regarding the durability and efficacy of RGX-314 will help us plan for the next steps in this program. Beyond RGX-314, we're excited to provide the recent interim data from the first three MPS II patients in our Phase 1/2 trial for RGX-121. Patients with MPS II continue to have significant neurocognitive difficulties despite availability of enzyme replacement therapy, which doesn't address manifestations of the disease in the central nervous system. The initial data from the first cohort showed that RGX-121 was well-tolerated following onetime administration of gene therapy using a relatively new delivery approach through the cisterna magna through direct to CNS approach.

Equally importantly, we observed consistent and sustained reduction in a key biomarker, as well as signs of neurocognitive stability. We've also completed dosing of patients in an expanded cohorts in our Phase 1/2 trial of RGX-501 for the treatment of Homozygous Familial Hypercholesterolemia or HoFH. And we plan to assess the LDL-C levels after patients have completed steroid prophylaxis treatment. This interim data should be available in the first half of this year.

Across REGENXBIO, the R&D team is continuing important research and preclinical work on potential treatment for hereditary angioedema based on antibodies directed at plasma kallikrein, key protein of the plasma contact pathway, which is left unregulated in patients with HAE. We also recently announced the expansion and exclusive collaboration with neuroimmune to design and develop vectorized antibody therapies targeting alpha-synuclein and tau. We expect to provide an update on these programs in the second half of 2020.

Elsewhere, the R&D came led by our Chief Science Officer, Olivier Danos, has also recognized the utility of our NAV Technology in treatments targeting muscle cells, specifically our AAV vector as evidenced in work by some of our partners like our dentists for the treatment of X-Linked Myotubular Myopathy. As a result, we've had ongoing work in our research pipeline focused on a number of neuromuscular indications. Some of this work is now starting to mature and so approaching a stage of candidate selection. We believe we have the opportunity and the potential to treat patients suffering from certain neuromuscular