Jul 22, 2020

AGTC Planning Phase 2/3 Clinical Trial for XLRP Gene Therapy

Eye On the Cure Research News

The company is also expanding its Phase ½ trial for the emerging treatment

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Applied Genetics Technology Corporation (AGTC), a developer of gene therapies for rare diseases, is planning to launch a Phase 2/3 clinical trial in the first quarter of 2021 for its emerging X-linked retinitis pigmentosa (XLRP) gene therapy for people with mutations in the gene RPGR.

AGTC is also expanding its Phase 1/2 XLRP gene therapy trial. In the fourth quarter of 2020, the company plans to dose approximately 20 additional patients in the Phase 1/2 trial to collect more functional data. A mobility test is being added as a supplemental endpoint.

In January 2020, AGTC reported a favorable safety profile and evidence of efficacy for the XLRP gene therapy in the Phase 1/2 trial.

The US Food & Drug Administration provided comprehensive written feedback to AGTC regarding the design and execution of the Phase 2/3 XLRP trial and future regulatory submissions. The company continues to move forward with manufacturing, clinical site preparation, and other activities to enable initiation of the studies as planned.

AGTC is not currently recruiting participants for the expanded Phase 1/2 or Phase 2/3 XLRP clinical trials, but will make an announcement when recruitment is open.