REGENXBIO Inc.

Hello, and thank you for standing by. Welcome to the Q3 2022 Regenexx Bio, Inc. Earnings Call and Update on Altitude. At this time, all participants are in the listen-only mode. After the speaker presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 11 on your telephone. It is now my pleasure to introduce Chief Legal Officer Patrick Christmas.

Good morning, and thank you for joining us today. Earlier this morning, Regenexx Bio released financial and operating results for the third quarter ended September 30th, 2022, as well as new data from our altitude trial. The press releases 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 and our development of RGX 314, 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 discussions and analysis sections of Regenexx BIO's annual report on Form 10-K for the full year ended December 31st, 2021. And comparable risk factor 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 FCC's website. Any information we provide on this conference call is provided only as of the date of this call, November 3rd, 2022. 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 morning, everyone. Thanks for joining us. I'm pleased to begin today's call with a recap of the recent business highlights, as well as an update on our corporate goals. VIT FASISTA, our chief financial officer, will provide an overview of financial results for the third quarter ended September 30th, 2022. We're bi-coastal today. Steve Ficola, our chief medical officer, is on the West Coast, recently attending Retina Society. He'll provide an in-depth overview of the data that was presented yesterday at the Retina Society meeting from our Phase II altitude trial, evaluating RGX314 for the treatment of diabetic retinopathy, or DR, using suprachoroidal delivery. Towards the end of the call, we'll be joined by altitude investigator Leila Vasovich from Duke University and independent retina expert Dr. Peter Kaiser from the Cleveland Clinic. Leila and Peter will stay on with us as we open up the line for questions. At Regenexx Bio, our mission is to improve lives through the curative potential of gene therapy, focused on developing therapies for diseases that have significant unmet need. We continue to be a leader in gene therapy. There are thousands of patients who have been dosed with AAV therapeutics derived from our NAV technology platform and hundreds more receiving treatment every quarter. I'm very proud of how our company has been advancing our internal pipeline, and I believe our fundamentals have never been stronger. We put into place our five by 25 strategy to progress five AAV therapeutics from our internal pipeline and licensed programs into pivotal stage or commercial products by 2025. I'm now going to summarize some of the program highlights and operational updates from our announcements this morning. Our global eye care collaboration with AbbVie to develop and commercialize RGX314 for retina disease continues to advance. and is on track for a first BLA filing in 2024. Progress in trial enrollment and emerging clinical trial data also supports excellent progress in our suprachoroidal delivery program. At AAO, RGX314 subretinal delivery for the treatment of wet AMD was reported to be well tolerated with long-term durable treatment effects now observed up to four years. We expect this trial, along with the two ongoing pivotal trials, Atmosphere and Ascent, to support our planned VLA submission in 2024. In October, we also announced positive interim data from the Phase II AVA trial of RGX314 for the treatment of wet AMD using superchoroidal delivery. These data show that RGX314 was well-tolerated, with stable BCVA and a meaningful reduction in anti-VEGF treatment burden at all dose levels out to six months. We announced the expansion of this trial to further explore the third dose level in a sixth cohort with a short course of prophylactic ocular steroid following RGX314 administration in order to potentially prevent the observed incidence of mild to moderate intraocular inflammation. Yesterday, as I mentioned at the Retina Society meeting, new positive interim data was presented from our phase two altitude trial of RGX314 for the treatment of DR using suprachoroidal delivery and Steve will lead a review and discussion of these data in greater detail shortly. We've been working diligently to prepare on the initiation of our first in-human trial of RGX202 for the treatment of Duchenne and continue to expect to dose the first patient in the Affinity Duchenne trial in the first half of 2023. RGX202 is a potential one-time gene therapy for the treatment of Duchenne and being developed as a highly differentiated product designed to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-terminal domain, domains found naturally occurring in naturally occurring dystrophin. RGX202 is designed to support the delivery and targeted expression of genes throughout the skeletal and heart muscle using our NAV AAV8 vector. RGX121 is our candidate for the treatment of mucopolysaccharidosis type 2, also known as Hunter syndrome. This is currently being evaluated in an expanded pivotal phase program called Campsite. We are dosing patients in this pivotal program. Most recent positive interim data update from this study of this candidate reported that RGX121 was well-tolerated across all cohorts studied. Biomarker data from the patients in all three cohorts indicated encouraging dose-dependent reductions of cerebral spinal fluid GAGs. following one-time administration of RGX121. Improvements in neurodevelopmental function and caregiver-reported outcomes demonstrated CNS activity up to two years after RGX121 administration. The expanded pivotal phase of this program is expected to enroll up to 10 MPS II patients using commercial-scale GMP material to support a BLA filing in 2024 using the accelerated approval pathway. with the potential to enroll additional patients. This is our second active pivotal program and another opportunity for a BLA filing by 2024. Our ongoing phase 1-2 trial of RGX111 for the treatment of severe MPS1 or Hurler syndrome continues with plans to enroll additional patients in cohort 2 expansion R. Our manufacturing innovation center in GMP capacity Capability remains a key differentiator for Regenexx Bio and a key element of our strategy. Our in-house facility is cutting edge and allows us to move quickly from candidate selection to production of clinical grade material, which supports accelerating the early development of AAV therapeutics. Additionally, we believe our approach focuses early on product quality and process control, which lessens the need for changes during clinical development to enable efficient transition from clinical trials to commercial readiness. I would now just like to take this time to thank our entire Regenexx Bio team, all of our investigators and their site support staff, and the patient communities for their commitment to the continued development of our AAV therapeutics. We certainly believe that one-time gene therapy can address a whole range of unmet needs in both chronic and rare diseases, and we remain dedicated to these patients and their families. Reflecting on this quarter at this point in the year, I'm very proud of the progress we've made to advance our 5 by 25 strategy. And with that, I will now turn the call over to Vit for a review of our third quarter results and financial guidance.