Applied Genetic Technologies Suffers Setback in Eye Study

Health and Healthcare

Published: December 13, 2018 9:50 am

Last Updated: January 11, 2020 1:46 pm

Applied Genetic Technologies Corp. (NASDAQ: AGTC) shares dropped on Thursday after the company announced that its midstage eye study did not live up to expectations.

Specifically, the firm reported top-line interim six-month data from its Phase 1/2 clinical trial of rAAV2tYF-CB-hRS1, an investigational adeno-associated virus-based gene therapy delivered via intravitreal injection for X-linked retinoschisis (XLRS) due to mutations in the RS1 gene.

Results from the study show that rAAV2tYF-CB-hRS1 is generally safe and well-tolerated, but no signs of clinical activity were observed at six months. As per the study protocol, the company will continue to monitor enrolled patients at scheduled visits through the end of the study.

The company will complete patient monitoring activities on the XLRS program according to the clinical protocol but will not further develop the product. Applied Genetic Technologies plans to advance the XLRP program as previously disclosed, and will determine next steps for the three discovery programs over the next several months.

Sue Washer, president and CEO of Applied Genetic Technologies, commented:

The data from the XLRS trial support the general safety and tolerability of our gene delivery platform and further reinforce our confidence in our ongoing Phase 1/2 trials in achromatopsia (ACHM) and XLRP. We are extremely grateful for the commitment of the patients, families, patient advocacy organizations and our clinical collaborators, which was crucial to completing enrollment of this Phase 1/2 trial. Through this study, we have deepened our understanding of the challenges that patients with inherited ophthalmic diseases face every day. It has also provided us with valuable information and insights in designing and analyzing clinical trials for inherited retinal diseases. We remain dedicated to leveraging our gene delivery platform to improve the care and outcomes for patients with ACHM, XLRP and other ophthalmic diseases.

Shares of Applied Genetic Technologies traded down about 42% early Thursday at $3.81, in a 52-week range of $3.25 to $7.50.