NEW YORK, NY. –(BUSINESS WIRE)– March 22, 2017 — Attune Pharmaceuticals, a biotechnology company focused on the discovery and development of novel oral small molecule therapeutics for treatment of rare diseases, announced today positive results from preclinical safety studies evaluating ATN-249, a novel orally administered plasma kallikrein inhibitor for the treatment of Hereditary Angioedema (HAE). The data was presented in an oral presentation at the 10th C1-INH Deficiency Workshop (Budapest, Hungary) and can be found on the company’s website here. The strong safety, high potency, and high selectivity results suggest a wide therapeutic window with once-daily dosing potential of ATN-249.
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 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.
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