AN2 Therapeutics Announces Results of AN2-502998 for Chronic Chagas Disease

AN2 Therapeutics announced results from two studies evaluating the Company's oral CPSF3 inhibitor, AN2-502998, for the treatment of chronic Chagas disease caused by infection with the parasite T. cruzi. Key findings from the two studies: In NHPs with naturally acquired, chronic T. cruzi parasite infection, 28 days of AN2-502998 treatment resulted in 100% parasitic elimination at target exposures attainable in humans, through four months following the end of treatment. In the Phase 1 FIH study, AN2-502998 was generally well tolerated at exposure levels consistent with NHP efficacy thresholds, with no dose-limiting toxicities. "Results from these studies converge on the picture we were hoping to see: efficacy in NHPs at an exposure level achievable in humans, with an excellent safety profile as shown in the FIH study. Notably, parasites were eliminated after one month of treatment in NHPs with the same naturally acquired, chronic infection that we see in humans," said Eric Easom, CEO "Together, these data support our goal of making AN2-502998 the first FDA-approved therapy for chronic Chagas disease in adults. We believe an oral therapy capable of delivering high rates of parasitic cure after just one month of treatment could enable large-scale test-and-treat campaigns against this often lethal disease, which affects over 300,000 people in the U.S. and approximately 10 million globally. We look forward to initiating a Phase 2 proof-of-concept study late this year." The NHP efficacy study evaluated AN2-502998 administered for 28 days in macaques with naturally-acquired, chronic T. cruzi infection, contracted via triatomine vectors in their natural habitats, the same vector that transmits the parasite to humans. The Chagas disease vector is increasingly prevalent across the southern half of the U.S. The Company believes that efficacy in naturally infected NHPs is the most clinically relevant predictor of efficacy for human chronic Chagas disease. 100% of treated animals achieved parasite elimination at target exposures attainable in humans. Elimination of parasitemia in treated animals was durable and maintained through four months following the end of treatment. Parasitemia was evaluated using an enhanced PCR method with improved sensitivity and robustness. AN2-502998 was well tolerated with no drug-related adverse events. AN2-502998 is the only compound to have demonstrated curative activity in NHPs with long-term, naturally acquired T. cruzi infection. The Phase 1 FIH study evaluated single ascending oral doses of AN2-502998 and then multiple ascending doses administered over 10 days in healthy adult volunteers: AN2-502998 was generally well tolerated with no dose-limiting toxicities. Human PK was well characterized; plasma exposures at or above NHP efficacy thresholds were achieved.