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 reported encouraging interim six-month data from the dose-escalation cohorts of its ongoing Phase 1/2 clinical programs in patients with achromatopsia due to mutation in the ACHM CNGB3 or ACHM CNGA3 genes. The interim results from both studies demonstrate encouraging signs of biologic activity and a favorable safety profile. The company plans to report data from additional dose groups, age groups and time-points in the second half of 2020.
“We are pleased that both of our achromatopsia clinical candidates are demonstrating preliminary signs of biologic activity for patients who have no current treatment options for a disease that significantly compromises their vision and quality of life. We are further encouraged by many patients’ real-world positive anecdotes. We are continuing dose escalation in order to determine the highest safe dose with the potential to provide maximal benefit,” said Sue Washer, President and CEO of AGTC. “Combined with the positive six-month data presented earlier this month from our ongoing Phase 1/2 clinical trial in patients with X-linked retinitis pigmentosa, the results presented today further validate our AAV vector technology, clinical development expertise and ability to innovate and advance product candidates that have the potential to transform patient care.”
The data reported today are from 22 patients who have been dosed in the initial groups of the trials, comprising 13 patients in the ACHM CNGB3 trial and 9 patients in the ACHM CNGA3 trial. Data from both trials continue to demonstrate a favorable safety profile with no dose-limiting inflammatory responses observed.
These data also demonstrate encouraging signs of biologic activity, as shown by positive changes in light discomfort testing and encouraging patient anecdotes describing real-world improvements in visual function. The company is currently dosing two higher dose groups in adults and three groups of pediatric patients at the three highest dose groups in both trials. The company will use the additional data, which is expected to be available in the second half of 2020, to inform decision-making regarding readiness to move the product candidates to pivotal trials.