ATI-1801 is Appili’s topical paromomycin product with demonstrated Phase 3 efficacy used to treat cutaneous leishmaniasis (CL), a disfiguring infection of the skin that that affects hundreds of thousands of people around the world annually. It is an infection characterized by the formation of lesions and ulcers often leading to scarring, disfigurement, and stigmatization for those infected.
Licensed from the US Department of Defense (“DOD”) via the US Army Medical Materiel Development Activity (“USAMMDA”), ATI-1801 is novel topical product with demonstrated safety and efficacy across multiple Phase 2 and Phase 3 studies.
Current treatments are often invasive, toxic and/or require hospitalization, greatly limiting access to communities in need. Further development of ATI-1801 has the potential to significantly reduce the burden of CL by providing patients with a safe and effective topical therapy that can be used in the outpatient setting.
Appili holds the full clinical dossier for ATI-1801, including the results of a randomized, double-blind, vehicle-controlled Phase 3 study which evaluated the safety and efficacy of ATI-1801 for the treatment of cutaneous leishmaniasis in Tunisia. The study met its primary endpoint, with ATI-1801 administered topically once daily for 20 days demonstrating a significant improvement in the rate of clinical cure of the index lesion compared to vehicle (82% vs 58%; p-value < 0.001).
Leishmania affects hundreds of thousands of people around the world and treatments are invasive and limiting. Due to this high unmet need, we hope to work with the FDA on the necessary registration package to support a new drug application
– Dr. Don Cilla, President and CEO, Appili Therapeutics
Due to this high unmet need, Appili believes that ATI-1801 may be eligible for a PRV if approved, by the FDA. The PRV program was developed to incentivize drug development in US government priority areas including tropical diseases such as leishmaniasis. Once issued, a PRV can be used by its holder to accelerate the review of a subsequent drug submission. PRVs are transferrable and the secondary market for PRVs is well established with recent transactions often exceeding US$100 million.
The Company is actively evaluating the eligibility of ATI-1801 for a priority review voucher (“PRV”) which, if confirmed, would make ATI-1801 the second PRV eligible program at Appili, joining ATI-1701, a leading and potential first-in-class vaccine to prevent infection by top priority biothreat Francisella tularensis.