REGENXBIO Inc.

Good day and thank you for standing by. Welcome to the Q1 2022 Regenexx Bioincorporated 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 on your telephone. If you require any further assistance, please press star 0. I would now like to hand the conference over to our speaker today, Mr. Patrick Christmas, Chief Legal Officer. Please go ahead.

Good afternoon, and thank you for joining us today. With us are Ken Mills, Regenexx Bio's President and Chief Executive Officer, Dr. Steve Piccola, our Chief Medical Officer, and Vid Vasista, our Chief Financial Officer. Earlier this afternoon, Regenexx Bio released financial and operating results for the first quarter ended March 31, 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 and involves certain risks and uncertainties. These risks are described in the risk factors and the management's discussion and analysis sections of Regenexx Bio's annual report on Form 10-K for the full year ended December 31st, 2021, and the comparable risk factors sections of Regenexx Bio's quarterly reports on Form 10-Q, which will be 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, May 4, 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 Mills. Ken?

Thank you, Patrick. 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 first quarter ended March 31, 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 would like to provide some context on the gene therapy industry. Despite the recent market performance of the sector, I cannot help but be optimistic for the future of gene therapy. Over the years, we've seen a high level of investment in people, quality, and safety monitoring, and have developed a deeper understanding of the science across the industry. The recent challenges experienced across the industry are not, as history tells us, as a guide uncommon for novel modalities like gene therapy. Challenges create opportunities. In a relatively new field like gene therapy, the recent challenges have led to greater collaboration and involvement with key leaders, including regulatory agencies, that I believe will better define the path towards getting gene therapies to patients with unmet needs as efficiently as possible. While this past year has led to a difficult decision for a number of gene therapy companies, I'm encouraged as Regenexx Bio and the field of gene therapy head deeper into 2022 and beyond. To this end, I'm incredibly proud of how our company has been advancing our pipeline during these times, and I believe our fundamentals have never been stronger. This is why we felt this past quarter was the right time to announce our 5x25 strategy to progress five AAV therapeutics from our internal pipeline and licensed programs into pivotal stage or commercial products by 2025. The goal of the 5 by 25 strategy was to lay out a clear and definable plan to advance key programs that have the potential to treat very high unmet need in areas where we and our partners can use our NAV technology platform to develop and potentially commercialize AAV therapeutics as soon as possible. One of the leading candidates to meet our 5x25 strategy is RGX314 for the treatment of wet AMD and diabetic retinopathy. As you know, we have a global partnership with AbbVie to bring RGX314 to market, for which we have received a $370 million upfront payment and are eligible to receive up to $1.3 billion in additional milestones. We believe this deal not only validates Regenexx Bio as a leading player in the gene therapy space, but also validates RGX314 and its broad opportunity in ophthalmology. While the landscape for therapies being developed in both wet AMD and in diabetic retinopathies has changed over the past few months, With the new product launches and also high-profile setbacks in the field, we believe the opportunity for RGX314 has improved based on the encouraging interim safety and efficacy data presented for both wet AMD and diabetic retinopathy. Overall, we plan to take full advantage of AbbVie's leadership in eye care and its commercial strength as we work together to advance RGX314 for both the treatment of wet age-related macular degeneration and diabetic retinopathy. In our 5 by 25 strategy, we also expect to include candidates from our other internal programs, such as RGX202 for the treatment of Duchenne and RGX121 for the treatment of Hunter syndrome. This quarter, we presented positive interim data at the World Symposium for our neurodegenerative lysosomal storage disorder program, RGX121 for the treatment of Hunter syndrome, and RGX111 for the treatment of Hurler syndrome. We believe the data updates highlight the potential of both programs to alter the course of these debilitating diseases and deliver improved patient outcomes. Moving to RGX202, today we announced that we made a difficult decision to delay dosing of patients in our first in-human Duchenne clinical trial. This was a proactive step we elected to take in consideration of patient safety. This decision was due to an unexpected and isolated observation in the final vial-filling stage of the manufacturing process. It didn't meet our quality criteria. We've informed all key stakeholders and are investigating this situation. Based on this update, our current expectation is to be able to dose patients in the first half of 2023. Work preparing for trial initiation continues, including readying clinical trial sites, and manufacturing additional clinical supply. Importantly, this recent and unexpected event highlights the importance of having our own in-house manufacturing capabilities. I view this as a key to our longer-term success. While this will delay our current development plan, it certainly does not impact our strong commitment to developing our GX202 for patients with Duchenne, which continues to be a key element of our 5x25 strategy. Beyond our internal programs, 5x25 may also include programs developed by our NAV technology licensees. Our NAV technology platform is driving the field of AAV gene therapy forward, with over 60 clinical trials utilizing NAV vectors registered in the National Institutes of Health Clinical Trials Database since 2015. Our NAV technology is also the basis for Zolgensma, one of two FDA-approved gene therapies in the United States today. With a number of additional AAV therapeutics being developed over a broad range of therapeutic areas and disease indications by our NAV technology licensees, we believe these programs will also contribute key gene therapy products to meet our 5x25 strategy. With that, I'd like to turn the call over to Steve now to talk in greater detail about the internal programs.