AGTC Announces Completion of Enrollment in All Adult Dose Groups of its Ongoing Phase 1/2 Clinical Trials in Patients with Achromatopsia

Applied Genetic Technologies Corporation (AGTC) announced that it has completed the planned enrollment in all dose groups for adult patients (age 18 years or older), including the two higher dose groups, of its phase 1/2 clinical programs with achromatopsia due to mutation in the ACHM CNGB3 or ACHM CNGA3 genes. The company continues to enroll pediatric patients at the higher dose groups in both trials.

“We expect the information to be obtained from these adult dose groups, as well as the additional pediatric data, will help to reinforce the encouraging preliminary data generated to date and previously reported in September 2019 and January 2020,” Sue Washer, president and CEO of AGTC, said in a company news release. “We plan to report interim data from all adult dose groups in the second half of 2020 and to use the data to inform decision-making regarding readiness to move the product candidates to pivotal trials. In addition to being on track for our end-of-phase 2 submission for  our X-linked retinitis pigmentosa (XLRP) clinical study, we expect to have multiple achromatopsia data read-outs in 2020 that will build on the momentum we created in January.”

AGTC most recently reported interim 6-month data from the dose escalation cohorts of its ongoing ACHM phase 1/2 clinical trials in January 2020. Results from both studies demonstrated encouraging signs of biologic activity as shown by positive changes in light discomfort testing as well as encouraging patient anecdotes describing real-world improvements in visual function. A favorable safety profile with no dose-limiting inflammatory responses was observed.