REGENXBIO Inc.

Good day and thank you for standing by. Welcome to Regenexx BIO's third 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 touch-tone 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 your host today, Patrick Christmas, Chief Legal Officer. Please go ahead.

Good afternoon, and thank you for joining us today. Earlier this afternoon, Regenexx Bio released financial and operating results for the third quarter ended September 30, 2023. The press release 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 discussion and analysis section of Regenexx BIOS annual report on Form 10-K for the full year ended December 31st, 2022, and comparable risk factors in sections of Regenexx BIOS 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 being provided only as of the date of this call, November 8th, 2023, 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 in 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?

Thank you, Patrick. Good afternoon, everyone, and thanks for joining us. I'm pleased to begin today's call with an explanation of our updated strategic plans. Dr. Steve Pakola, our Chief Medical Officer, will then provide an update on our clinical programs, and Vita Sista, our Chief Financial Officer, will provide an overview of financial results for the third quarter ended September 30th, 2023. At the end of the call, we will be opening up the line for questions. At Regenexx Bio, our mission is to improve lives through the curative potential of gene therapy. Earlier today, we began work on a pipeline prioritization and corporate restructuring plan that will enable Regenexx Bio 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. I want to be clear about what this sharpened focus means moving forward. Our highest priority programs are ADBV-RGX314 program for the treatment of wet age-related macular degeneration and diabetic retinopathy being developed in collaboration with AbbVie, RGX202 for the treatment of Duchenne, and RGX121 for the treatment of MPS2 or Hunter syndrome. Now, in the last two months, we've experienced exciting progress from each of these programs. We've reported positive clinical data from investigational treatments from diabetic retinopathy and Duchenne, and we've held a very encouraging RMAT meeting with the FDA about expediting the BLA for the treatment for MPS II. These milestones demonstrate how our science is supporting avenues to accelerate the development of new gene therapies. And today, we're following that encouraging data and announcing updated strategic plans for Regenexx Bio. We believe that there's a multi-billion dollar potential for RGX314 as a single injection treatment to become the first in class gene therapy for wet AMD and the standard of care to treat and prevent the progression of diabetic retinopathy. Initial efficacy data from patients treated with RGX202 is enabling us to accelerate this program. Duchenne is a market where there is a large unmet need for new therapies, and that is capable of supporting multiple gene therapies, and we believe RGX202 has unique differentiating features that support its potential to be a best-in-class product. During a very constructive RMAT meeting with the FDA just recently in October, we received encouraging feedback and confirmed alignment with the FDA on key elements of an expedited DLA. So we remain on track to support a BLA filing in 2024 using the accelerated approval pathway. RGX121 would be the first gene therapy treatment for MPS2. In today's challenging market, the ability to create value quickly and efficiently is critical. Importantly, these restructuring decisions extends our cash runway much deeper into 2025. allowing us to progress our pipeline to a number of key inflection points. These would include initiating and dosing the first pivotal trials for 314 using supracroidal delivery, enrolling the pivotal program for our RGX202 program, and completing performance qualification lots to support a planned BLA for RGX202, and also achieving the BLA approval for RGX121 and MPS2. Now, it's worth noting that successfully achieving certain of these milestones between now and then would also trigger hundreds of millions of additional funds, such as milestone payments from our collaboration partner, AbbVie, for initiating superchoroidal pivotal trials, which these milestones represent a meaningful portion of the over $560 million in development milestones eligible through this partnership for us. and the potential receipt of a pediatric review voucher for approval of RGX121. Generally, we've been observing PRV sales are resulting in nearly $100 million to BLA sponsors at the time of receipt. So, our updated strategic plans are intended to generate significant value for shareholders as we ensure resources are allocated to our most valuable assets. To be able to accelerate the development of these assets, and to extend our operational runway in order to achieve even more milestones that can unlock value. These additional non-dilutive sources that I just highlighted, for instance, are not in our current runway guidance, and if received, would allow us to bridge to additional value-creating milestones, such as more product approvals and potentially to profitability. Now I'll turn the call over to Steve so he can review some of our clinical progress and guidance for the prioritized programs in greater detail. Steve?