Applied Genetic Technologies Corporation

Good morning and welcome to the AGTC financial results conference call for the first quarter of fiscal year 2022. Today's call is being recorded. Before we get started, I would like to remind everyone that during this conference call, AGTC may make forward-looking statements, including statements about the company's financial results, financial guidance, its future business strategies and operations, and its product development and regulatory progress including statements about its ongoing and planned clinical trials and preclinical programs. Actual results could differ materially from those discussed in these forward-looking statements due to a number of important factors, including uncertainty inherent in the clinical development and regulatory process, the extent and duration of the impact of the COVID-19 pandemic, and other risks described in the risk factor section of AGTC's most recently filed annual report on the Form 10-K, and other periodic reports filed with the SEC. AGTC undertakes no obligation to update any forward-looking statements after the date of this call. For introductions and opening remarks, I'd like to turn the call over to Sue Washer, Chief Executive Officer of AGTC. Please go ahead.

Good morning, and thank you all for joining us. During our last quarterly call in September, I had the pleasure of introducing John Lieber as our new Chief Financial Officer. Today, I am pleased to introduce Susan Schneider, our new Chief Medical Officer, who brings extensive expertise and a track record of success in ophthalmology drug development, including the approval and launch of Lucentis. Her experience and insights will be invaluable as we work to advance our XLRP and achromatopsia trials towards approval and as we expand our clinical portfolio with the advancement of our two lead preclinical programs towards IND smiling. Both Susan and John join me on today's call. Last week, we welcomed Sarah DeSalvatore as Vice President of Clinical Operations, and we expect that her strategic leadership and contributions to the FDA approval of multiple rare disease therapies further enhances our robust clinical development capabilities. Our ability to attract high-quality talent like Susan, John, and Sarah reflects the great potential of our pipeline and technology, as well as our amazing corporate culture, which is built on a shared commitment to improving patients' lives and values the contributions that each member of our team brings towards achieving that vision. Now turning to our ongoing clinical trials, our XLRP program is our nearest-turn opportunity to bring a potentially transformative therapy to patients living with a disease that today has no treatment options. As many of you are aware, we presented promising data from the ongoing Phase 1-2 trial earlier this year. This includes 12-month data from patients in the two highest dose groups in this trial, demonstrating a 50% response rate among patients who meet the inclusion criteria for the ongoing Skyline and VISTA trials, as well as improvements in visual acuity across a wide range of patients that provide supportive evidence of biological response. Additionally, data from a subset of patients in the high-dose group who are available for analysis at 24 months provide evidence of response durability and continued safety. As a reminder, We believe that our Skyline and VISTA trials will be successful if we are able to demonstrate the same 50% response rate as we did in patients in the ongoing Phase I-II trials that meet the inclusion criteria in assuming that we see a favorable safety profile. In September, our clinical collaborator, Dr. Paul Yang, Assistant Professor of Ophthalmology at the KCI Institute, presented data from a 12-month analysis of macular structure using optical coherence to evaluate patients in the Phase 1-2 XLRP trial. The data, which Susan will review in a few minutes, identify a correlation between macular structure and visual function that supports the treatment effect of our XLRP gene therapy candidates. These data add to the growing body of evidence supporting the industry-leading potential of this product candidate, and further increases our confidence in the possibility for positive results in both the Skyline and VISTA clinical trials. Based on the totality of the data generated to date, improvements in visual sensitivity, improvements in visual acuity, positive patient anecdotes, and Dr. Yang's observations of improvements in macular structure, we believe that our XLRP gene therapy may provide meaningful and durable benefits to patients. We are currently conducting the Skyline Phase I-II expansion trial and the VISTA Phase II-III trial, and we expect that data from both trials will support a potential filing of a Biological Assistance Application, or BLA. We anticipate multiple data readouts from our XLRP clinical trials between now and the end of 2022, which Susan will outline in a moment. Each of these readouts is an opportunity to further demonstrate the potential value of our XLRP product candidate, and we believe that collectively these trials will provide the most robust and differentiated set of data for any XLRP gene therapy currently in clinical development. Now let me turn to our chromatopsia clinical program. The data reported to date support the continued development of our chromatopsia clinical candidates and have also allowed us to identify a maximum tolerated dose in pediatric patients. As previously reported, we are moving forward with the clinical development of the B3 program and are preparing an end of phase two briefing document for submission to the FDA on which we expect their feedback in the first half of 2022. The path forward for our A3 product candidate will be determined after additional pediatric patient and preclinical data are available for evaluation. Before moving to our preclinical pipeline, I also wanted to remind everyone about our collaboration with Bionic Sight in optogenetics. Earlier in 2021, Bionic Sight announced promising initial Phase 1-2 data that showed that treated patients, all of whom are complete or near completely blind, can now see light and motion, and in two cases can detect the direction of motion. Our diversified preclinical pipeline provides multiple opportunities to expand into additional disease areas outside of inherited retinal diseases, including in central nervous system or CNS indications and otology indications, and to address large markets outside of the rare disease space, such as the dry form of age-related macular degeneration or dry AMD. As previously reported, we have prioritized our programs in dry AMD and frontal temporal dementia for the advancement to IND filing and are on track to initiate toxicology and biodistribution studies for both programs in 2022. As part of our strategic collaboration with autonomy, the otology program is moving forward with an IND application expected to be filed in the first half of 2023. Key to our ability to advance our clinical and preclinical candidates as quickly as possible is ensuring access to high-quality, scalable, and high-productivity manufacturing. Our leased, build-to-suit Current Good Manufacturing Process, or CGMP, manufacturing and quality control facility remains on track to become operational in the fourth quarter of 2022. We have already made several key hires to support the design and construction of the building and intend to continue to bring an additional staff to support the validation of the facility in the second half of 2022. Our goal for this custom manufacturing capacity is to enable an expedited BLA filing and commercial launch of our XLRP product candidates subject to FDA approval and support late stage development of our chromatopsia program. Our investment in and commitment to this facility reflects both our confidence in the clinical and commercial potential of our XRP and achromatopsia clinical programs and our commitment to supporting more rapid advancement of our entire product pipeline. Critically, this facility will also provide supply chain redundancy and reduce manufacturing risk. Now, I will turn the call over to Susan, who will provide additional detail on our XRP and achromatopsia clinical programs. Susan?

Thank you, Sue, for the warm welcome. I'm pleased to join my first call as AGTC's Chief Medical Officer, and I'm excited to have the opportunity to share several positive updates for both of our ongoing clinical programs. As Sue mentioned, Dr. Paul Yang presented promising new data from the ongoing Phase 1-2 XLRP clinical trial at the 14th International Symposium on Retinal Degeneration in September. The data he presented were from an analysis of macular structure in patients 12 months after receiving a single dose of our XLRP product candidate. A hallmark of XLRP is at the ellipsoid zone, or EZ, which is a defined region within the photoreceptor layer of the retina degenerates over time, and is eventually lost. The data Dr. Yang presented shows that intact retinal anatomy at baseline predicts potential for improvement. Of 20 patients evaluated in our XLRP Phase 1-2 clinical trial, 13 had intact baseline retinal anatomy, of which 9, or 69%, had either complete recovery or improvement in EZ at six months. By contrast, the seven patients who had end-stage retinal anatomy, as evidenced by absence of the EZ, showed no structural or functional improvements post-treatment. The data also showed that there was a significant association between improvements in visual sensitivity measured by Maya and improvements in retinal health measured by improvement in EZ in groups four to six. Among these patients, four of six with MYA improvement also had EZ improvement, and there was a statistically significant association between MYA improvement and EZ improvement with a p-value of 0.0212. We believe that these data further support a treatment effect from our XLRP product candidate and continue to differentiate the data set for our product candidate from competitive products in development. As Sue also mentioned, we are actively enrolling and dosing patients in both the Skyline and VISTA trials and expect to report multiple data sets from these trials in 2022, including three-month interim Skyline trial results in the first half of 2022, 24-month trial results from the ongoing Phase 1-2 clinical trial in the third quarter of 2022, 12-month Skyline trial results in the fourth quarter of 2022, and six-month interim VISTA trial results in the fourth quarter of 2022. Our clinical collaborator, Dr. Robert Sisk of Cincinnati Children's Hospital and University of Cincinnati College of Medicine, will also make an encore presentation of the 12-month data from the Phase 1-2 trial at the American Academy of Ophthalmology annual meeting taking place from November 12th to 15th, 2021. We're pleased to have this additional opportunity to help educate the ophthalmology community about the potential potential benefits of our XLRP candidate. With respect to the achromatopsia programs, we recently enrolled six pediatric ACHMB3 patients and five pediatric ACHMA3 patients in higher dose groups 5A and 6A and identified a maximum tolerated dose. As we have previously reported, to address suspected unexpected serious adverse reaction, or SUSAR, safety events in pediatric patients in the highest dose group, systemic and local steroid doses were increased, and patients are being monitored closely. I am pleased to say that the patients are doing well, and our investigators are tapering these patients to lower steroid doses. Importantly, we did not see comparable inflammation in the six pediatric patients across both trials at dose group 5A, nor in any of the adult patients, or lowest group 4 pediatric patients, on which we previously reported, and we plan to continue development of our achromatopsia product candidates. We expect to report interim three-month data for the pediatric patients in both achromatopsia trials before the end of 2021. For the B3 program, we are also developing an end of phase two briefing packet for submission to the FDA and expect to receive feedback from the agency in the first half of 2022. The agency's input will play a foundational role in our plans for advancing the achromatopathy of B3 candidate toward a pivotal trial. Now, I'll turn the call over to John for a review of our financial results.