REGENXBIO Inc.

To ask a question during the session, you will need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 11 again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your speaker 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 first quarter ended March 31, 2024. The press release is available on our website at www.regenexxbio.com. Today's conference call will include four 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 31st, 2023, and comparable risk factors 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 provided only as of the date of this call, May 8th, 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 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. 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. Today, Curran Simpson, our Chief Operating Officer, will be speaking for Steve Piccola, Chief Medical Officer, who's unable to join us today. Curran will provide an update on our clinical programs. Then, Vipa Sista, our Chief Financial Officer, will provide an overview of the financial results for the quarter ended March 31st, 2024. At the end of the call, we'll open up the line for questions. We have what I think are some exciting updates today, and so I want to get right into these topics. We started off the year making significant progress across our pipeline as we advance each of our programs toward pivotal stage clinical trials and commercialization. In 2024, we've put a focus on large commercial opportunities where our product candidates are differentiated, can be expedited, and support meaningful value generation soon and for the long term. Our priority programs are RGX202 for the treatment of Duchenne, ABBV RGX314 or 314 for the treatment of wet AMD and diabetic retinopathy or DR being developed in collaboration with AbbVie, and RGX121 for the treatment of MPS2 or Hunter Syndrome. All of our programs remain on track to meet our goals for this year. Program milestones over the past couple of months further demonstrate how our new plan is supporting the acceleration of the development of our gene therapies and the expansion of value for shareholders. Today, we're especially pleased to share new positive data and important updates about acceleration in our programs for Duchenne NDR using in-office delivery. We are providing some new updated guidance on near-term milestones for these programs, so let me explain. We're thrilled to see RGX202 is demonstrating strong microdystrophin expression across a wide range of patients, including in older boys, like those ages 8 to under 12. And importantly, we are seeing positive safety signals. No SAEs have been reported in patients who've received RGX202, which is a highly notable differentiator in Duchenne gene therapy trials. Today, we announced that we have selected our pivotal dose. Also, we have already begun a new expansion phase of our clinical development with the pivotal dose. This includes the enrollment of third and fourth boys already. Expansion using the pivotal dose will continue into the third quarter and can enroll up to a total of seven boys in the ages ranging from four to under 12. By early Q3, we expect to hold an end of phase two meeting with the FDA, which will support a final pivotal design. And pivotal trial initiation is anticipated in late Q3 to early Q4 of this year. RGX202 is a differentiated product candidate in the landscape for Duchenne, representing the closest thing to natural healthy dystrophin, and we believe has the potential to make a meaningful difference for a broad range of patients. Given these unique characteristics of RGX202, a significant ongoing unmet need in the Duchenne community, in conversations with the FDA, we're confident in our plans to file a BLA using the accelerated approval pathway. Separately, We believe there's a multibillion-dollar potential opportunity for ABVDRGX314 as a single injection treatment to become a first-in-class gene therapy for wet AMD and the standard of care to treat and prevent progression for diabetic retinopathy. Enrollment remains on track for our subretinal delivery program for wet AMD currently in pivotal trials. And today we announced that based on positive interim results to date from the phase two altitude trial in DR, the design and evaluation of two pivotal trials is ongoing, which would be the first pivotal trials for our in-office suprachoroidal delivery. These positive results and activities are in support of a planned discussion with the FDA at an end of phase two meeting anticipated in Q1 2025 that can enable the rapid acceleration toward pivotal development. We would expect to initiate the first two pivotal trials for supracroital delivery of diabetic retinopathy in the first half of 2025. As a one-time treatment, We believe that 314 is well-positioned to become the standard of care to treat and prevent progression of diabetic retinopathy, which is expected to impact over 20 million people globally in the next five years. Overall, we're thrilled about the current status and the way that 2024 can achieve meaningful value generation based on what we've seen in these first few months. With that, I'm going to turn the call over to Chief Operating Officer Curran Simpson, who will take us through a bit more of a detailed update on the Duchenne data and our plans for accelerating the pivotal trial initiation for Duchenne in the second half of this year and diabetic retinopathy in the first half of next year. Curran?