Applied Genetic Technologies Corporation (NASDAQ: AGTC) has reported interim three-month paediatric data and further adult safety findings to almost 24 months from the ongoing Phase1/2 dos3e escalation trial of AGTC-401.
AGTC-401 is an AAV gene therapy for achromatopsia
AGTC-401 is a recombinant AAV viral vector-based gene therapy for achromatopsia (ACHMB3) patients with mutations in the CNGB3 gene. The findings from seven young patients back up those from adults treated with AGTC-401, who showed promising biologic ability and safety and tolerability profile. Interestingly no new Suspected Unexpected Serious Adverse Reactions (SUSARs) were reported, and the inflammation in the three previously reported SUSARs is improving with steroid therapy. The company expects to pursue the clinical development of AGTC-401 pursuant to discussion with the US FDA at an End-of-Phase 2 (EOP2) conference in the 1H 2022, depending on the totality of the ACHMB3 data gathered to far from 31 subjects over as long as 24 months.
CEO Sue Washer said, “The three-month pediatric findings provide further evidence for the strong potential of our product candidate for patients with ACHMB3, and we look forward to continuing our discussions with the regulatory agencies to determine the best path forward to bring this important therapy to patients. We understand the challenges that patients with achromatopsia face in their daily lives and our team remains dedicated to advancing this program toward commercialization.”
AGTC released findings of a parallel AGTC-402 study
The company also released updated data from a parallel trial of AGTC-402 addressing CNGA3 mutations in achromatopsia individuals, which are consistent with prior adult results, show no clinical benefit, and do not warrant continued clinical advancement. The clinical data will be vital in the next phase of development. The majority of individuals with CNGA3 mutations exhibit a mutant peptide that is not commonly observed in patients with CNGA3 alterations, which the AGTC believes could well have influenced the outcomes seen in patients who received AGTC-402. The ACHMA3 patients will be followed by AGTC for long-term safety monitoring.