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agtc AGTC Announces Financial Results and Business Update for the Quarter Ended September 30 , 2021 November 9 , 2021 -Company on track to provide multiple data readouts for XLRP and ACHM clinical programs in 2021 and 2022 -Company to host management update and webcast today at 8:00 a.m. ET GAINESVILLE , Fla . and CAMBRIDGE , Mass . , Nov. 09 , 2021 ( GLOBE NEWSWIRE ) -- Applied Genetic Technologies Corporation ( Nasdaq : AGTC ) , a biotechnology company conducting human clinical trials of adeno - associated virus ( AAV ) -based gene therapies for the treatment of rare inherited retinal diseases ( IRDs ) , today announced financial results for the quarter ended September 30 , 2021 . " We have significant momentum toward achieving multiple clinical and regulatory milestones in the year ahead . We believe that the data we have generated to date in the Phase 1/2 XLRP clinical trial point to a higher likelihood of success in achieving similar results in both the Skyline and Vista trials and look forward to sharing that data in 2022 , " said Sue Washer , President and CEO of AGTC . " In order to execute on these opportunities , we have strengthened our management team with individuals who we believe will provide additional expertise to navigate the clinical development and regulatory pathways for both our clinical programs and the exciting preclinical product candidates we are advancing towards potential IND filings . We are confident that we will be able to generate value for patients , clinicians and stockholders . " Recent Highlights X - linked Retinitis Pigmentosa ( XLRP ) The Company is currently conducting the Skyline expanded Phase 1/2 XLRP and Vista Phase 2/3 XLRP clinical trials to support a potential filing of a Biologics License Application ( BLA ) . Earlier in the year , the Company reported positive 12 - month data from the highest dose groups in the ongoing XLRP Phase 1/2 clinical trial , including 12 - month data from Groups 5 and 6 reflecting a 50 % response rate among patients who meet the inclusion criteria for the Skyline and Vista trials . In addition , the Company reported 24 - month data from two Group 4 patients that provides preliminary evidence of response durability . These data together with the statistically significant improvements in visual acuity and data presented in September 2021 indicating a correlation between improvements in macular structure and visual function as well as continued evidence of a favorable safety profile add to the growing body of evidence suggesting the significant benefit possible for patients receiving AGTC's XLRP product candidate . Over the next 12 months , the Company expects to achieve the following milestones in the XLRP development program : • Presentation of 12 - month trial results from the ongoing Phase 1/2 clinical trial at the American Academy of Ophthalmology Annual Meeting being held from November 12- 15 , 2021 ; • Release of 3 - month interim Skyline trial results in 1H 2022 ; • Release of 24 - month results from the ongoing Phase 1/2 clinical trial in 3Q 2022 ; • Release of 12 - month Skyline trial results in 4Q 2022 ; and • Release of 6 - month interim Vista trial results in 4Q 2022 . Achromatopsia ( ACHM ) In June , the Company reported 12 - month data from its ongoing Phase 1/2 ACHM clinical trials , including data from all adult and low - dose pediatric patients . In the ACHMB3 trial , the product candidate demonstrated biologic activity based on improvements in visual sensitivity and light discomfort , both of which were supported by anecdotal patient reports . In addition , the safety profile of the product candidate remained favorable . Based on these data , AGTC intends to advance the ACHMB3 program to the next stage of clinical development . AGTC recently enrolled six pediatric ACHMB3 patients and five pediatric ACHMA3 patients in higher dose groups 5a and 6a and has achieved a key objective of the trial - the identification of a maximum tolerated dose . To address the previously reported Suspected Unexpected Serious Adverse Reactions , or SUSARS , in three pediatric patients in the highest dose group 6a , systemic and local steroid doses were increased . In addition , patient monitoring also increased and the investigators are now tapering these patients to lower doses of steroids and all patients are improving . Over the next 12 months , the Company expects to achieve the following milestones in the ACHM development program : • Release of 3 - month data from the pediatric patients in both trials in 4Q 2021 ; and • Submission of an End - of - Phase 2 briefing packet to the U.S. Food and Drug Administration ( FDA ) and receipt of feedback in 1H 2022 . Bionic Sight Collaboration Earlier in 2021 the Company's collaborator , Bionic Sight , announced promising initial Phase 1/2 data in a retinitis pigmentosa optogenetics study that showed that treated patients , all of whom have complete or near - complete blindness , can now see light and motion , and , in two cases , can detect the direction of motion . Manufacturing Facility In May , the Company announced an expansion of its manufacturing and analytics capabilities by leasing a 21,250 square foot build - to - suit current