Benzinga | May 3, 2021 08:42AM EDT

AGTC Clinical Investigators To Present Data From Ongoing XLRP And Achromatopsia Phase 1/2 Trials At

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 diseases, today announced an encore presentation of previously reported data from the Company's ongoing clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM) at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Virtual Annual Meeting. Paul Yang, MD, PhD, Assistant Professor at Casey Eye Institute, Oregon Health & Science University, will review the six-month data from the Phase 1/2 XLRP trial, on which he is a clinical investigator today in a session scheduled to be held at 4:30-6:00 PM EDT; Mark Pennesi, MD, PhD, Assistant Professor at Casey Eye Institute, Oregon Health & Science University, will provide an overview of the Phase 1/2 ACHM clinical trials including examples of patients who the Company previously defined as responders on Thursday, May 6, 5:15-6:45 PM EDT.

"We are pleased to have the opportunity to share these data with members of the eye health community in this peer-reviewed format, and are especially pleased to demonstrate that the XLRP trial and both ACHM trials show favorable safety profiles out to 12 months," said Sue Washer, President and CEO of AGTC. "We believe that the XLRP data, which includes six-month data from all patients dosed and 12-month data for Groups 2 and 4, are best-in-class, and are excited for the multiple additional XLRP milestones that we anticipate in 2021 and 2022. The 12-month ACHM data are encouraging and have given us important insight that we are considering as we move this program forward."

With respect to anticipated upcoming XLRP milestones, AGTC remains on track to report 12-month data for Groups 5 and 6 from the XLRP Phase 1/2 trial in the second quarter of 2021; 3-month masked interim Skyline data in the fourth quarter of 2021; and 12-month Skyline and 6-month masked interim Vista data in the third quarter of 2022. The Company also remains on track to report additional 12-month data from both ACHM trials in the second quarter of 2021, and preliminary 3-month data from the pediatric cohorts of both ACHM trials in the fourth quarter of 2021.

Phase 1/2 XLRP Data Presented at ARVO

The data from all 28 patients in the XLRP trial across six dose groups, which Dr. Yang will present today and which were previously reported in November 2020, demonstrate a favorable safety profile with no dose-limiting inflammatory responses observed. Dr. Yang also will review efficacy data from centrally dosed patients. At six months, the data showed increased mean sensitivity relative to baseline as assessed by microperimetry, and these results were durable through month 12. We also believe that the best corrected visual acuity (BCVA) assessments in 20 centrally dosed patients at six months provides supportive evidence of improved visual acuity across dose groups, with additional supportive evidence of sustained improvement in eight of nine patients who were available for assessment at month 12. We are not aware of similar findings in clinical trials of competitors developing XLRP gene therapies.

Further a combined analysis of visual sensitivity data from all 19 evaluable centrally dosed patients showed that 10 of 15 evaluable patients in Groups 2, 4, 5 and 6 that meet inclusion criteria for the planned Vista Phase 2/3 trial show robust and durable signs of improvements in visual sensitivity through month six for Groups 5 and 6 and month twelve for Groups 2 and 4. The Company based improvement of visual sensitivity on multiple measures including on a change from baseline in visual sensitivity of at least 7 decibels in at least 5 loci or a statistically meaningful improvement in sensitivity improvement profile between the treated and untreated eyes.

Phase 1/2 ACHM Data Presented at ARVO

AGTC previously reported interim data from its ongoing Phase 1/2 trials in patients with mutations in the ACHMB3 or ACHMA3 gene in January 2021. On May 6, 2021, Dr. Pennesi will review case studies from two patients in the ACHMB3 trial and one patient in the ACHMA3 trial. Data from these case studies and the previously presented data from these trials demonstrate that subretinal injection of AGTC's ACHM candidates has been safe and well tolerated out to 12 months. Results also demonstrate potential improvements in visual sensitivity and photosensitivity in these patients that were identified to have positive results

As previously announced, AGTC is focusing on completing enrollment of pediatric patients in the two highest dose groups in the ACHMB3 and ACHMA3 trials and plans to follow all patients through 12 months. The company has amended the study protocol for these trials to allow enrollment of patients as young as four years of age and to include both functional magnetic resonance imaging (fMRI) and improved color brightness tests. AGTC also plans to work with sites to obtain the best quality multi-focal ERG data possible. The Company is hopeful that these changes, combined with longer follow-up times, will add to the developing body of evidence and supportive anecdotal patient-reported outcomes.