REGENXBIO Inc.

Good afternoon and welcome to the Region X BIO fourth quarter and full year 2020 earnings conference call. At this time, all participants are in a listen-only mode. Later, we will conduct a question and answer session and instructions will be given at the time. As a reminder, this conference call is being recorded. I would now like to turn the call over to Mr. Patrick Christmas, Chief Legal Officer for Region X BIO. You may begin.

Good afternoon, and thank you for joining us today. With us are Ken Mills, Regenexx Bio's President and Chief Executive Officer, Dr. Steve Piccola, our Chief Medical Officer, Dr. Olivier Danos, our Chief Scientific Officer, and Vit Basista, our Chief Financial Officer. Earlier this afternoon, Regenexx Bio released financial and operating results for the fourth quarter and full year ended December 31, 2020. The press release reporting our financial results is available on our website at www.regenixbio.com. Today's conference call will include forward-looking statements regarding our financial outlook in addition to regulatory and product development plans. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted. and can be identified by words such as expect, plan, will, may, anticipate, believe, should, intend, and other words of similar meaning. Any such forward-looking statements are not guarantees of future performance and involve certain risks and uncertainties. These risks are described in the risk factors and the management's discussion and analysis sections of Regenexx Bio's annual report on Form 10-K for the full year ended December 31, 2020, and comparable risk factor sections of Regenexx BIOS quarterly reports on Form 10Q, which are on file with the Securities and Exchange Commission and available on the SDGs website. Any information we provide on this conference call is provided only as of the date of this call, March 1, 2021, and we undertake no obligation to update any forward-looking statements we may make on this call on account of new information, future events, or otherwise. Please be advised that today's call is being recorded and webcast. In addition, any unaudited or pro forma financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company. Actual results may differ materially. I would now like to turn the call over to Ken Mills. Ken?

Thank you, Patrick. Good afternoon, everyone, and thanks for joining us. I hope that everyone is staying safe and healthy. Thank you. On today's conference call, we will provide a recap of our recent progress, advancing and expanding our NAV technology platform, and discuss expected future milestones for the company. Steve and Olivier join us to discuss our pipeline, and VIT will provide an update on financial results for the fourth quarter and the full year 2020. We'll then open up the call for questions. Throughout 2020 and already in 2021, I believe we're making great strides towards achieving our goal of realizing the curative potential of gene therapy. We have advanced and broadened our internal pipeline of programs in several important ways. I'll touch on just a few pipeline highlights before turning the call over to the team. Starting our first pivotal program is an important and exciting milestone that we're proud to achieve. This program is to evaluate our gene therapy candidate for wet age-related macular degeneration, a retinal disease that is a leading cause of total and partial vision loss, affecting more than 2 million people in the United States. Our treatment, called RGX314, is a novel one-time gene therapy that is designed to work by delivering therapeutic genes to the retina of a patient in order to interrupt pathway of the disease. The subretinal delivery technique that we are using in our Pivotal program is an established and targeted route of delivery for gene therapy for retinal disease. The Pivotal program builds on data that we have seen from our ongoing Phase 1-2 study, where we have observed that RGX314 has been generally well tolerated, and durable effects of the one-time gene therapy are now out to three years after dosing patients in the trial. In 2020, we also initiated new clinical trials evaluating RGX314 using a different delivery approach. These trials are the first to evaluate the delivery of any gene therapy to the suprachoroidal space of the eye. This form of delivery is a non-surgical approach that can be administered in an office setting and potentially allow for targeted delivery to the retina. Overall, our RGX314 programs are evaluating these two routes of administration because we believe we can provide these one-time treatments to the largest population of patients with wet AMD and diseases of similar origins like diabetic retinopathy by providing access to gene therapy in all settings of care. Our RGX121 candidate is being studied as a single administration treatment for neurodegeneration associated with Hunter syndrome. This treatment is designed to work by delivering a new gene to a patient's cells in the brain and central nervous system RGX121 is administered directly into the cerebrospinal fluid. We've shown evidence of consistent biomarker reductions and continued neurocognitive development from patients in the RGX121 trial. And we're also encouraged by new data providing evidence that after treatment is delivered to the cerebrospinal fluid, it may be able to address disease in patients in other parts of their body. This includes data from patients who were never treated with the standard of care enzyme replacement therapy prior to enrolling in our trial. And this is exciting to us because we know that Hunter syndrome is a disease that affects nearly every organ. We also recently announced development of RGX202, a potential single administration gene therapy for the treatment of Duchenne muscular dystrophy. Olivier has been spearheading the development of this program, which is based on a novel advanced microdystrophin construct. He'll provide further details, but we're excited to bring this program forward this year. In addition to our pipeline progress and potential, we're well capitalized with the resources to achieve our next planned program milestones. We have completed two recent transactions that have added over $400 million to our balance sheet, and this enables us to continue to advance our programs as well as support investment in our commercial-ready manufacturing platform and the build-out of our own manufacturing facility. Finally, before turning it over to the team, I want to take a moment to acknowledge what's going on in the wonderful background worldwide events that have taken place and messages that have emerged in support of Rare Disease Day, observed yesterday, February 28th, and celebrated across many days recently. On February 26th, last Friday, Regenexx Bio hosted an event that allowed our teams a special opportunity to hear directly from families, caregivers, and advocates in order to continue to enhance our awareness of the needs of those living with rare diseases. Overall, I wish to express my sincere appreciation, especially this year, from me and the Regenexx Bio leadership team to our dedicated employees, clinical investigators, partners, and most importantly, patient communities, for their unwavering support as we work hard to advance our mission. With that, I'll turn the call over to Steve for a more detailed update on the active clinical programs.