REGENXBIO Inc.

Good day and thank you for standing by. Welcome to the Regenexx BIO fourth quarter 2023 earnings conference call. At this time all participants are in a listen only mode. After the speaker's presentation there will be a question and answer session. To ask a question during the session you will need to press star 1 1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question please press star 1 1 again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to Patrick Christmas, Chief Legal Officer. Please go ahead.

Good afternoon, and thank you for joining us today. Earlier this afternoon, Regenix Bio released financial and operating results for the fourth quarter and full year ended December 31, 2023. The press release 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, 2023, and comparable risk factor sections of Regenexx BIO's quarterly report on Form 10-Q, which are on file with the Securities and Exchange Commission and available on the SEC's website. Any information we provide on this conference call is provided only as of the date of this call, February 27, 2024. and we undertake no obligation to update any forward-looking statements we may make on this call and account 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, CEO of Regenexx Bio.

Thank you very much, Patrick. Good afternoon, everyone, and thanks for joining us. I'm pleased to begin today's call with a recap of our recent business highlights, as well as an update on our corporate goals. Steve Piccola, our Chief Medical Officer, will then provide an update on clinical programs, and Vit Vasista, our Chief Financial Officer, will provide an overview of financial results for the fourth quarter and the full year ended December 31st, 2023. At the end of the call, we're going to open the line up for questions. Late last year, Regenexx Bio began work on a pipeline prioritization plan that enables us to focus our capabilities and resources on large commercial opportunities where product candidates are differentiated, can be expedited, and support meaningful value generation soon and for the long term. Our priority programs are ABVV, RGX314, program for the treatment of wet AMD and diabetic retinopathy being developed in collaboration with our partner, AbbVie, RGX202 for the treatment of Duchenne, and RGX121 for the treatment of MPS2 or Hunter syndrome. Early on in 2024, we've experienced exciting progress for each of these programs. We believe there's a multibillion-dollar potential for the 314, a single injection treatment to become a first-in-class gene therapy in wet AMD, and the standard of care to treat and prevent progression of diabetic retinopathy. Enrollment remains on track with our subretinal delivery program for wet AMD, currently in pivotal trials, with an expectation to support global regulatory submissions by the end of 2025 through the first half of 2026. We hosted calls recently with leading retina experts to review our new positive clinical data from our treatment for wet AMD and diabetic retinopathy using in-office supracoroidal delivery. These events were exciting for us, and today we're going to be talking about additional upcoming data and events that we're giving guidance on. Three weeks ago, we focused attention on our rare disease pipeline in two amazing milestones. In Duchenne, we announced the completion of enrollment in cohort two and additional positive interim data from our ongoing trial. We are thrilled to see RGX202 as demonstrating strong microdystrophin expression across a wide range of patients. And for MPS2, we achieved what I think is a major breakthrough with statistically significant results for our pivotal trial on which we are filing a BLA. Patients receiving this treatment have continued to show improvement in their neurodevelopmental skill acquisition up to four years, and we recently observed discontinuation standard of care intravenous enzyme therapy. Our recent milestones demonstrate how our new plan is supporting the acceleration and the development of gene therapies and the expansion of value for our shareholders. And today we're providing some new guidance, as I mentioned, on near-term milestones for Duchenne, and our treatments for wet AMD and diabetic retinopathy using in-office delivery. Next week, we have plans to share new Duchenne trial update at the Muscular Dystrophy Association Conference being held in Orlando, Florida. Also, we expect to share new program and data updates for the phase two altitude and AVA trials in Q2 and mid-2024, respectively. Overall, we're absolutely thrilled about our status and the progress the company's made And with the way things have started off in 2024, I'm going to circle back in a bit and talk more about next steps for us. But for now, I'm going to turn it over to Steve so he can do a deeper dive on the review of the clinical progress for our prioritized program.