AGTC reports positive biologic signs in 12-month data for achromatopsia candidate
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AGTC plans to advance its achromatopsia B3 candidate to the next stage of development after 12-month phase 1/2 trial data showed positive biologic activity, according to a company press release.
Achromatopsia (ACHM) candidate B3 data for 25 patients with mutations in the ACHM B3 gene showed improvements in visual sensitivity measured by static perimetry and ocular photosensitivity analyzer, the release said. In addition to the positive biologics, patient anecdotes were also positive.
The B3 trial showed improved retinal sensitivity in the treated eye compared with the untreated eye in four of 11 patients receiving higher doses of the treatment and those in the pediatric study arms. Six of the 11 patients experienced improvement in light discomfort scores. In some patients, improved light discomfort in the untreated eye may suggest cortical adaptation in the brain, according to the release.
“We are incredibly pleased with these data, which further support AGTC as a leader in the field of ophthalmic gene therapy,” Sue Washer, president and CEO of AGTC, said in the release. “Our strong capabilities lead to differentiated products based on capsid and vector engineering, robust manufacturing and rigorous preclinical evaluation of our product candidates in validated models of retinal diseases.”
Patients in the ACHM A3 treatment arm did not show consistent evidence of biologic activity attributable to the therapy, the release said.
Both trials showed consistent safety profiles with earlier trial data for the company’s adeno-associated virus gene therapy platform. There were zero serious adverse events related to the therapy candidates. There was one serious adverse event relating to the surgical procedure and two relating to steroid use, the release said. All are resolved or improving.
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