REGENXBIO Inc.

Good day, ladies and gentlemen. Thank you for standing by. Welcome to the second quarter of 2022 Regenexx BIO Incorporated earnings conference call. At this time, all participants are on a listen-only mode. After the speaker's remarks, 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 keypad. At this time, I would like to turn the conference over to Mr. Vit Vasista, Chief Financial Officer, Regenexx BIO. Sir, please begin.

Good afternoon and thank you for joining us today. With us today are Ken Mills, Regenexx BIOS President and Chief Executive Officer, and Dr. Steve McCullough, our Chief Medical Officer. Earlier this afternoon, Regenexx Bio released financial and operating results for the second quarter and the June 30th, 2022. The press release reporting our financial results is 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 file's annual report on Form 10-K for the full year ended December 31st, 2021 and comparable risk factors sections in Regenexx FIO'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, August 3rd, 2022. And we undertake no obligations 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 in webcast. In addition, any unaudited or performant financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company. Natural results may differ materially. I would like to now turn the call over to Ken Mills. Ken?

Thank you, Bitt. 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 will then provide an update on our clinical programs and VIT will provide an overview of financial results for the second quarter ended June 30th, 2022. At the end of the call, we will open up the line for questions. Before Steve gets into the pipeline progress in detail, I'd like to provide some context on the gene therapy industry. Last quarter, I mentioned how encouraged I was about the future of the industry, and this past June, I believe we witnessed the impact of many effects, including with the FDA Advisory Committee unanimously endorsing two new gene therapies for the treatment of rare diseases. Clearly, this is great news for the gene therapy industry, and most importantly, for patients needing these potentially life-saving therapies. I believe the panel's approach to reviewing the risk-reward profile of these products and the Committee's acknowledgment for the product's construct and its role in both safety and efficacy highlights the willingness of these experts to take a more flexible approach when evaluating products for patients with large unmet needs. In addition, in the past couple of months, the European Commission has granted marketing authorization for one AAV therapeutic, and the EMA has also adopted a positive opinion recommending conditional market approval for another AAV therapeutic, both indicated for rare diseases. So overall, I find these endorsements encouraging for the industry as well as for Regenexx Bio as we advance our AAV therapeutics for patients with large unmet needs. Moving on to some of our internal highlights for the quarter, we continue to make excellent progress advancing RGX314 using both subretinal and suprachoroidal delivery. With RGX314 for the treatment of wet age-related macular degeneration using subretinal delivery, we remain on track for a BLA filing in 2024 based on two pivotal trials, Atmosphere and Ascent. We've also progressed our RGX314 trials using supracoroidal delivery for the treatment of wet AMD and diabetic retinopathy, or DR. And in May, we announced completion of enrollment of a Phase II altitude trial evaluating RGX314 for the treatment of DR. And more recently, in July, we announced completion of enrollment in Cohort 5 of our Phase II ABA trial evaluating RGX314 in the treatment of wet AMD. We plan to present additional super-choroidal data later this year, and we're pleased with the progress we've made across the entire RGX314 program this quarter. We continue to take full advantage also of our global partnership with AbbVie to bring RGX314 to market. Taking a moment to update on RGX202, our candidate for the treatment of Duchenne. Last quarter, we announced that we made the difficult decision to delay dosing of patients in our first in-human Duchenne clinical trial. This decision was due to an unexpected isolated observation in the final vial filling stage of the manufacturing process at a third-party contract manufacturer that didn't meet our quality criteria. The unexpected delay, among other things, reinforced the importance of investment toward in-house GMP manufacturing capabilities and the facility. This is why I would like to highlight that this past quarter we celebrated the opening of our new CGMP manufacturing facility called the Regenic Biomanufacturing Innovation Center. This 21,000 square foot facility is located in our headquarters building in Rockville, Maryland. This marks our successful expansion into a company with full end-to-end capabilities from research and development to commercial scale infrastructure. We are one of only a few gene therapy companies worldwide with a CGMP facility capable of production at scales up to 2,000 liters. The first runs in our Manufacturing Innovation Center support our clinical supply for the RGX202 program. New process improvements that we plan to use in our facility are expected to produce, for example in RGX202, an approximately five-fold improvement in vector yield per patch than previously used processes. I want to reiterate our strong commitment to the Duchenne community. Preparation for the initiation of our first in human trial with RGX202 continues, including ongoing manufacturing of additional clinical supply for the trial. We continue to anticipate dosing the first patient in this trial in the first half of 2023. Finally, our newest update comes from earlier this morning when we announced that the pivotal program in MPS II is now active and enrolling patients and our intention to file a BLA in 2024 using the accelerated approval pathway. MPS II or Hunter syndrome is a debilitating disease with a large unmet need that significantly impacts a child's daily life and life expectancy and is inadequately treated by any drugs on the market today. We are developing RGX121, a potential first-in-class one-time AAV therapeutic for the treatment of MPS II to treat the CNS manifestations of MPS II for which there are currently no available treatment options. Recent discussions with the FDA support this plan. The accelerated approval pathway was created to allow for expedited development of drugs that treat serious conditions and provide a meaningful advantage over available therapies based on a surrogate endpoint. I would note that following our announcement in November 2021 of the launch of the Pathway Development Consortium, co-founded by Regenic Bio and Solid Biosciences, which brings together key stakeholders, including the FDA, with the goal of expediting patient access to AAV therapeutics, we've seen an increased interest in the accelerated approval pathway across the gene therapy industry. This is an important advancement for rare disease communities, and especially those like the MPS II community. Our pivotal program will measure GAGs in the CSF, which we believe can be considered a surrogate biomarker that is reasonably likely to predict clinical benefit in MPS II disease under the accelerated approval pathway, as the buildup of GAGs or glycosaminoglycans in the central nervous system of MPS II patients leads to clinical manifestations, including neurodevelopmental deficits. We believe the accelerated approval pathway will allow us to advance RGX121 as quickly as possible with the aim of providing a much-needed new treatment option for the MPS II community. So with that, I will now turn the call over to Steve to talk in greater detail about the internal programs.