REGENXBIO Inc.

Welcome everyone to the Q2 2024 RegenX Bioearnings Conference call. All lines have been placed on mute to prevent any background noise. After the speaker's remarks, there will be a question and answer session. If you would like to ask a question, simply press the star followed by the number one on your telephone keypad. If you would like to withdraw your question, press star one. At this time, I'd like to turn the conference over to Patrick Christmas, Chief Legal Officer of BeginXBio. Please go ahead.

Good afternoon, and thank you for joining us today. Earlier this afternoon, Regenexx Bio released financial and operating results for the second quarter ended June 30, 2024. 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 in the management's discussion and analysis section of Regenexx BIO's annual report on Form 10-K for the full year ended December 31st, 2023, and comparable risk factor sections 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, August 1st, 2024, 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 will now turn the call over to Kern Simpson, President and CEO of Regenexx Bio.

Thank you, Patrick. Good afternoon, everyone, and thank you for joining us. I'm pleased to be leading today's call, my first one as Regenexx Bio's Chief Executive Officer. Today we'll be sharing a number of exciting, positive updates and discuss the momentum happening across our pipeline of differentiated AV therapeutics. I'll begin with a recap of our business highlights, as well as an update of our corporate goals and key milestones that we have achieved. Dr. Steve Piccola, our Chief Medical Officer, will provide an update on our clinical programs, and then Vince Assista, our Chief Financial Officer, will provide an overview of financial results for the second quarter ended June 30, 2024. At the end of the call, we'll open up the line for questions. It's been a productive first half of the year for Regenexx Bio as we make significant progress advancing each of our programs toward pivotal stage clinical trials and future commercialization. Our priority programs are RGX202 for the treatment of Duchenne, AbbVie RGX314 program for the treatment of wet AMD and diabetic retinopathy, or DR, being developed in collaboration with AbbVie, and RGX-121 for the treatment of MPS II or Hunter syndrome. Our lead programs, specifically 202 and 314, represent large commercial opportunities where our product candidates are differentiated from current standard of care, can be expedited via accelerated approval due to significant unmet need, and support meaningful value generation soon and for the long term. Let me begin with RGX202, which represents the next generation of microdystrophin gene therapies and is poised to potentially be the second AAV-based product to reach the market. There are a number of exciting developments for RGX202. Steve will share more details about the positive data reported today, demonstrating consistent, robust microdystrophin expression across treated patients reflecting a broad range of ages. But I first want to highlight the differentiating factors that we believe will make RGX202 a best-in-class product and the excellent progress we are making to both expedite its development and maximize its commercial potential. RGX202 is a differentiated product candidate utilizing an advanced microdystrophin construct with potential for improved functional benefit, as shown in our preclinical data. It is the only microdystrophin product that includes the C-terminal domain, a key region of the naturally occurring dystrophin gene, which has been shown in preclinical studies to protect the muscle from contraction-induced stress and improve the ability of the muscle to repair itself. As I mentioned, RGX202 is demonstrating consistently high levels of microdystrophin expression across patients of all ages. But I want to note that it is in older ambulatory boys where we're seeing the highest levels of microdystrophin expression reported in older ambulatory patients, especially compared to other published data. And RGX202 has been well tolerated and no SAEs have been reported, which is a significant element of the overall risk-benefit analysis for patients, caregivers, and regulatory agencies, and a meaningful differentiator versus other Duchenne gene therapy trials. As I mentioned, our goal is to be the next approved gene therapy in Duchenne, and we are taking all of the necessary steps towards this goal. We recently completed a successful end-of-phase two meeting with the US FDA and walked away from this meeting confident in our plans to file a BLA using microdystrophin as the primary endpoint for accelerated approval. The meeting also involved discussion of our industry-leading NavXpress suspension-based commercial-ready manufacturing process used in this trial. At our in-house manufacturing facility, we have the capacity and yields to produce 2,500 doses of RGX202 per year. Given the differentiating characteristics of RGX202 and the significant ongoing unmet need in the Duchenne community, plus our manufacturing expertise, we are well positioned to advance this program towards commercialization. Turning to AbbVie RGX314, Our gene therapy being developed in chronic retinal diseases with our partner, AbbVie, we have made several advancements across the supracoroidal trials in diabetic retinopathy and wet AMD. First, with regard to the altitude trial of 314 for the treatment of DR using supracoroidal delivery, we are accelerating plans for our end of phase two meeting with the FDA. This meeting is now expected to take place in the fourth quarter of this year versus our initial guidance of first quarter 2025. The new timeline supports the rapid acceleration towards pivotal trials with initiation expected in the first half of 2025. Importantly, Regenexx Bio will be entitled to a $200 million milestone payment upon successful dosing of the first patient with AbbVie RGX 314 in DR, which again is anticipated in 2025. We are also excited to announce that working with our partners at AbbVie, we will be expanding the broad multi-indication global potential of 314 by initiating a new cohort in the altitude trial for patients with diabetic macular edema, DME, 314 is well positioned to become the standard of care to treat and progression of diabetic retinopathy. Broadening the altitude trial to include patients with DME further expands the global potential of 314. We have also made important progress on our 314 programs for wet AMD, as well as in our RGX121 program for MPS2, as we approach potential approval and becoming the first gene therapy for Hunter Syndrome. We remain on schedule to initiate a rolling BLA filing in the third quarter of 2024. Approval of the planned BLA could result in receipt of a priority review voucher in 2025. Overall, we are making excellent progress and have provided positive updates across all programs with a number of additional catalysts on track to be shared later this year. We remain excited by our progress as we continue on the strategic plan. We are accelerating the development of our pipeline and expanding their value for shareholders while bringing potentially life-changing therapies to patients facing great unmet needs. With that update, I'd like to now turn the call over to Steve for an update on our clinical programs. Steve?