REGENXBIO Inc.

Thank you for joining Q4 2021 RegenXBio, Inc. Earnings Conference Call. At this time, your lines have been placed on mute. Later in the call, there will be a Q&A session and instructions will be given at that time. If you need operator assistance, please press star zero. As a reminder, today's call is being recorded. And if you wish to ask a question, please press star one. I'll now turn to call over to your host today, Patrick Christmas, Chief Legal Officer with GenX Bio. Sir, please go ahead.

Good afternoon, and thank you for joining us today. With us today are Ken Mills, for GenX Bio's President and Chief Executive Officer, Dr. Steve Bacola, our Chief Medical Officer, and Vipa Sista, our Chief Financial Officer. Earlier this afternoon, Regenexx Bio released financial and operating results for the fourth quarter and full year ended December 31, 2021. 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 discussion and analysis sections of Regenexx BIO's annual report on Form 10-K for the full year ended December 31, 2021, 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, March 1, 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 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. Ken?

Thank you, Patrick. Good afternoon, everyone, and thanks again for joining us. I'm pleased to begin today's call with a Quick recap of business highlights as well as an update on our corporate goals. Steve will then provide an update on our clinical programs and Vint will provide an overview of financial results for the fourth quarter and year ended 2021. At the end of the call, we will open up the line for questions. I want to start off by saying how pleased I am with our performance as an organization over this past quarter and this past year. Against the challenging backdrop in 2021, Regenexx Bio has continued to perform at a very high level. And I'd really like to take this time to thank our Regenexx Bio team, our investigators, and the patient communities for their commitment to the development of our innovative AAV therapeutics. One of the more important events for our company this past year was the signing and recent closing of our eye care collaboration agreement with AbbVie for the development and commercialization of RGX314. We're really pleased to be working with AbbVie, an industry leader in eye care, as we advance RGX314, a potential one-time treatment, in clinical trials for the treatment of wet AMD and diabetic retinopathy, and consider development in other chronic eye disorders. Aside from AbbVie's significant financial investment that we believe validates our innovative gene therapies and provides significant cash runway, the collaboration allows us to leverage AbbVie's leading development and commercial expertise. We plan to take full advantage of this collaboration as we work together to advance RGX314 in multiple billion-dollar eye care markets. Yesterday, at Regenexx BIO, we recognized Rare Disease Day as we brought focus to this event and sought to generate change for the more than 300 million people worldwide living with a rare disease, their families, and caregivers by participating in a number of awareness and stakeholder engagement exercises. Our 2021 momentum has continued in this new year with a number of recent announcements that we believe raise the profile of our pipeline of AAV therapeutic for rare diseases. For instance, we made significant progress advancing RGX202, our gene therapy for the treatment of Duchenne. Importantly, RGX202 is our first homegrown program leveraging our proprietary NAV AAV8 vector to deliver a transgene for a novel microdystrophin that includes the functional elements of the C-terminal domain of dystrophin. We believe that RGX202 has the potential to address the limitations of other treatment approaches for patients suffering from this severe degenerative disease. With the recent clearance of our investigational new drug application, we're excited to get our clinical trial started, which we expect to take place in the first half of this year. Today, we also announced that the FDA has granted rare pediatric disease designation for RGX202, which makes this program eligible for obtaining a priority review voucher in the future. Last month, we presented positive interim data at the World Symposium for RGX121 being developed for the treatment of Hunter syndrome and RGX111 for the treatment of Hurler syndrome. We believe the data updates highlighted the potential of both programs to alter the course of these debilitating neurodegenerative diseases and deliver improved patient outcomes. As you may know, we have moved into and begun utilizing our new headquarters building in Rockville, Maryland that has our internal GMP facility located within our headquarters. This facility is on track to be fully operational in the first half of 2022 and is expected to allow for the production of NAV vectors that scales up to 2,000 liters. Importantly, with the new headquarters and CGMP manufacturing facility, we are continuing to expand our capabilities from basic research and development to commercial scale infrastructure. As a result of this and all of our achievements, we're excited to be introducing a new arm of the Regenexx Biostrategy called 5x25. that will be a clear and definable plan to advance AAV therapeutics. With a number of license and collaboration agreements in place, as well as a growing internal pipeline, the purpose of 5x25 is to identify the key programs that treat very high unmet needs in areas where we, our partners and licensees, can use our NAV technology platform to develop and potentially commercialize AAV therapeutics as soon as possible. We believe this will help us focus as an organization on key value creating assets where we have the expertise to develop and enable the development of these programs in an efficient way. Specifically, we intend to progress five AAV therapeutics from our internal pipeline and licensed programs into pivotal stage or commercial products by 2025. The leading candidates to meet our five by 25 strategy include our internal programs such as RGX314 for the treatment of wet AMD and diabetic retinopathy, RGX202 for the treatment of Duchenne, and RGX121 for the treatment of Hunter syndrome. But in addition, we will draw from other programs that we plan to advance internally into development and programs being developed through our NAV technology licensees to achieve this strategic goal. What this signifies for me is that we're steering Regenexx Bio in the direction of supporting more late-stage development and commercialization of AAV therapeutics. I view this as a natural evolution for the company that has been foundational in the progress in the field of AAV therapeutics and has made over 10 plus years of foundational commitment. With that, I'd like to turn the call over to Steve. He's going to go into greater detail about our internal programs. Steve?