Innovating Treatments for Rare Diseases

In the preclinical toxicology and safety pharmacology studies, ATN-249 was generally safe and well tolerated. In addition, pharmacokinetic studies indicated high 24-hour exposure and comprehensive drug recovery after repeat oral doses of ATN-249.

“This encouraging data, along with prior published potency and efficacy results, reinforce our belief that our lead drug candidate, ATN-249, has a favorable safety profile and once-a-day dosing regimen to address the unmet need for well-tolerated and safe oral therapies with improved patient quality of life and prophylactic efficacy,” said Dr. Andrew McDonald, CEO of Attune Pharmaceuticals. “These IND-enabling study results support commencing the Phase 1 clinical development of ATN-249 this summer.”

The oral presentation outlined the results of several well-established preclinical assays. Studies included evaluation of potency of ATN-249 compared to C1INH via inhibition of plasma kallikrein, selectivity of ATN-249 on biochemical inhibition of plasma kallikrein relative to other closely related serine proteases, and ATN-249’s pharmacokinetics, general toxicity, safety pharmacology, and genotoxicity profiles.