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Kids Suffer Adverse Reactions In Applied Genetic's Color Blindness Gene Therapy Trial

After escalating the dose in its pediatric gene therapy trial, Applied Genetic Technologies Corporation (NASDAQ: AGTC) has run into safety problems. 

Benzinga · 09/24/2021 09:59
  • After escalating the dose in its pediatric gene therapy trial, Applied Genetic Technologies Corporation (NASDAQ:AGTC) has run into safety problems. 
  • The Company recently enrolled six pediatric achromatopsia (ACHM) with CNGB3 gene mutation and five pediatric ACHM patients with CNGA3 mutation in high dose groups 5a and 6a.
  • Three of the five pediatric patients who received the highest dose developed severe inflammation around one month after dosing. 
  • Investigators saw inflammation in both eye segments.
  • A fourth patient developed significant inflammation but didn’t meet the Suspected Unexpected Serious Adverse Reaction (SUSAR) definition.
  • To address the above safety events in pediatric patients, systemic and local steroid doses have been increased.
  • “These new data do not change our plans to continue development of the achromatopsia product candidates as the body of data gives us confidence in our plan to move forward at appropriate doses,” Sue Washer, President, and CEO said. 
  • The Company is drafting an End-of-Phase 2 briefing packet to submit to the FDA and expects feedback in 1H of 2022.
  • In May, AGTC announced an expansion of its manufacturing and analytics capabilities that will be operational by Q4 of 2022.
  • AGTC ended Q4 with cash, cash equivalents, and investments of $107.1 million, sufficient to allow the Company to generate data from its ongoing clinical programs and fund currently planned research and discovery programs. 
  • Related content: Benzinga's Full FDA Calendar.
  • Price Action: AGTC stock is down 1.17% at $3.38 during the market session on the last check Friday.