REGENXBIO Inc.

Good day, and thank you for standing by. Welcome to the Regenexx BIO Incorporated's first quarter 2023 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'll need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised. To remove yourself from the queue, please press star 11 again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker today, Patrick Christmas, Executive Vice President and Chief Legal Officer. Please go ahead, sir.

Good afternoon, and thank you for joining us today. Earlier this afternoon, Regenexx Bio released financial and operating results for the first quarter ended March 31, 2023. The press release and data presentation are available on our website at www.regenexxbio.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, 2022, and comparable risk factors section of Regenexx BIO's quarterly reports 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, May 3, 2023. 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. Ken? Ken?

Thank you, Patrick. Excuse me. Good afternoon again, everyone. Thanks for joining us. I'm pleased to begin today's call or continue today's call with a recap of our recent business highlights as well as an update on our corporate goals. Dr. Steve Ficola, our Chief Medical Officer, will then provide an update on our clinical programs, and Vipa Sista, our Chief Financial Officer, will provide an overview of financial results for the first quarter ended March 31st, 2023. At the end of the call, we will open up the line for questions. At Regenexx Bio, our mission is to improve lives through the curative potential of gene therapy, and we're focused on developing therapies for diseases that have significant unmet need. We continue to be a leader in gene therapy with thousands of patients who have been dosed with AAV therapeutics derived from our NAV technology platform. There's been meaningful progress in the gene therapy industry lately. including growing understanding of gene therapy pricing models and the FDA's validation of surrogate markers as viable clinical endpoints. Regenexx Bio continues to work closely with our regulatory partners to help advance and accelerate the development of gene therapies, particularly for rare diseases. Today, I'm pleased to discuss how Regenexx Bio remains a leader in gene therapy as we share with you how our 5x25 strategy is on track for advancing 5 AAV therapeutics from our internal pipeline and licensed programs into pivotal stage or commercial products by 2025. Our end-to-end capabilities continue to set us apart as a leader, with our in-house manufacturing innovation center running scalable, commercial-ready batches of our AAV therapeutics, and our research and early development experts continuing to advance what's possible in gene therapy. In fact, this week, we announced a summary of around 15 presentations at the upcoming ASGCT conference, which reflect leadership in areas such as engineering novel NAV capsids for better targeting of AAV therapeutics to clinically relevant tissues and organs, enhancing AAV therapeutics, tissue and cell type specificity and expression, including optimizing enhancer and promoter combinations, optimizing devices and routes of delivery for AAV therapeutics, and other proof of concept research that informs next steps in our pipeline strategy. Today, we also announced significant update and progress in our global eye care collaboration with AbbVie. including the transfer of INDs for all ongoing clinical trials to AbbVie, as originally outlined in the collaboration agreement, and the global expansion of pivotal trials, Atmosphere and Ascent, for the treatment of wet age-related macular degeneration, or wet AMD, using subretinal delivery. Our investigational one-time gene therapy formerly known as RGX 314, also has been renamed ABBV RGX 314. And from now on, by the way, I'll refer to it as 314. The new global site plans and expanded enrollment targets for atmosphere and ascent involve updates to our U.S. plans and new guidance for global regulatory milestones. These trials are now expected to support regulatory submissions with the FDA and the EMA in late 2025 through the first half of 2026. Steve will share additional clinical details, but first, I want to frame our view of how today's update of the global expansion further optimizes the value and enhances the robustness of the 314 program. I want to take the opportunity to make clear where we are today, what is our clinical experience with 314, and how we view the global commercial opportunity for RGX314. In major retinal vascular diseases such as wet AMD, the current standard of care anti-VEGF treatments require patients to receive injections in every eye every four to 12 weeks for the duration of disease. First-generation anti-VEGF treatments are not good, and emerging second-generation anti-VEGF treatments are making an impact for some patients with longer incremental acting mechanisms. However, real-world evidence consistently shows us that vision improvements lag behind the controlled clinical trial evidence. Patients cannot be treated with the required frequency of both the first and second generation anti-VEGF treatments, and they do not control disease well enough between doses. Patients, doctors, and clinics continue to struggle with the impact of the limitations of the current and the emerging anti-VEGF standard of care. and under-treatment is still leading to disease progression, damage to the retina tissue, and lost vision. Together with AbbVie, we're developing 314 to be the first one-time option in major retinal vascular diseases to address the significant unmet need in the treatment of wet AMD and diseases like diabetic retinopathy and other chronic retinal conditions. A main goal in our partnering our gene therapy leadership with AbbVie's global infrastructure and leadership in eye care was to expand beyond our US footprint, to bring 314 to patients worldwide. And we're pleased to see this vision coming to life with today's announcement and the forthcoming global expansion. Where are we today? We think we're in a great position to optimize the opportunity for creating value with 314 for patients worldwide. We have the largest global clinical program for one-time treatment option in major retinal vascular diseases. This involves seven ongoing clinical trials, including two pivotal trials, global, three phase two trials, and we're studying two delivery devices, subretinal and supracoroidal delivery, in two different lead indications, wet AMD and diabetic retinopathy, with opportunities to expand further into several other adjacent retinal diseases. Additionally, as I mentioned before, Regenexx Bio's Manufacturing Innovation Center, which is here in Maryland, is a fully operational, state-of-the-art GMP gene therapy manufacturing facility designed to meet global clinical and commercial regulatory standards. Our NavXpress platform process is already being used in the pivotal trials for 314. And the Regenexx Manufacturing Innovation Center is on track to produce U.S. commercial supply operating at 500 liter level with bioreactors to support commercialization and with an option to expand up to 2,000 liters as needed. In 2023, We plan to use the Manufacturing Innovation Center to produce that commercial-scale GMP material for the entire 314 clinical program, as well as performance qualification lots to support planned regulatory filings. To summarize our clinical experience, in total, more than 600 patients have been dosed in the 314 program, representing over 400 patient years of exposure across seven trials. The totality of the clinical evidence shared to date shows the treatment of RGX314 using both subretinal and suprachoroidal delivery methods is generally well tolerated. Patients are generally responding to one-time treatment of 314. Long-term follow-up in our subretinal studies has shown durability over four years, with stable to improved BCVA and meaningful reduction in anti-VEGF burden, with the majority of patients injection-free. Now, real-world evidence shows that patients with wet AMD are severely undertreated due to the unsustainable burden of these frequent injections. We believe the profile of 314 as a potential one-time treatment addresses this high unmet need, as a majority of patients currently on high interval standard of care could transition to a one-time gene therapy. And there's opportunity to capitalize on additional market share from standard of care agents, biosimilars, and emerging longer interval treatments. Together with AbbVie, we're developing 314 to be the first one-time therapeutic option in major retinal vascular diseases to address significant unmet need for patients. This is a worldwide opportunity with a potential to reach over 30 million patients with high unmet need in what is already calculated to be over $14 billion global anti-VEGF market. Additionally, today, Frankly, the utilization of anti-VEGF to treat diseases like early diabetic retinopathy is low. The treatment burden is high. Less than 1% of patients with early disease are treated with intravitreal injections. We believe that there's a potential for 314 as a single in-office injection to become a new standard of care for early diabetic retinopathy treatment to prevent vision loss. An estimated 6 million patients have early DR in the U.S. alone, and DR is estimated to be a potential $15 billion global market in the next decade, supported with the right treatment profile, such as a one-time treatment. So with that, I'm going to turn the call over to Steve so he can get into greater depth on our 314 clinical progress and also discuss our rare disease pipeline and progress and updates. Steve?