A novel topical product that can help treat hundreds of thousands of patients globally with disfiguring infections of the skin
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).
“This is a unique opportunity to build on an extensive data set and bring a treatment that has already demonstrated Phase 3 efficacy to patients around the world. This is exactly the type of mission that Appili was built to take on and I look forward to applying our expertise, capabilities, and partner network to this effort.”
- Dr. Armand Balboni, CEO Appili Therapeutics
Cutaneous leishmaniasis is a disfiguring infection of the skin that affects hundreds of thousands of people around the world annually, characterized by the formation of lesions and ulcers that often lead to scarring, disfigurement, disability, and stigmatization of the infected. The disease is a serious impediment to socioeconomic development, especially for women, and a priority for governments and non-governmental organizations ("NGOs") around the world.
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 third 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, and ATI-2307, Appili’s novel clinical stage antifungal candidate
“The disease affects some of the poorest people and is associated with malnutrition, population displacement, poor housing, a weak immune system and lack of financial resources.”
Appili plans to meet with the FDA to discuss the previously generated Phase 3 data and agree on the necessary registration package to support a new drug application (“NDA”) submission, which the Company expects will include available nonclinical, manufacturing, and clinical data generated to date.
Appili expects to pursue non-dilutive funding and partnership opportunities with NGOs and government agencies which share the Company’s focus on tropical diseases to help complete remaining development work.
(1) Topical Paromomycin with or without Gentamicin for Cutaneous Leishmaniasis. Ben Salah A 2013 N Engl J Med 368: 524-532