Appili Therapeutics Inc. has announced that the U.S. Food and Drug Administration (FDA) has provided positive feedback on its development strategy for ATI-1801, a novel topical formulation of paromomycin (15% w/w) intended for the treatment of cutaneous leishmaniasis. The FDA's agreement covers the necessary registration package to support a New Drug Application (NDA). This decision simplifies the regulatory pathway for ATI-1801, potentially expediting its availability to patients suffering from this disfiguring skin infection.
Streamlined Development Path
The FDA's response to Appili's Type B meeting request included an agreement with the company's strategy to establish a scientific bridge between previous clinical trial material and new drug product batches. This involves developing a validated in-vitro release test (IVRT) method and manufacturing a new reference standard for IVRT studies. According to Appili, this approach will allow for completion and submission of an NDA much sooner than if additional clinical data were required.
"This positive response from the FDA simplifies and de-risks our development program for ATI-1801," said Don Cilla, President and CEO of Appili Therapeutics. "The agency's agreement with our proposed strategy enables Appili to leverage key results from the clinical dossier for ATI-1801 licensed from U.S. Army Medical Materiel Development Activity (USAMMDA), including the successful results of a Phase 3 study, and provides a clear path towards an NDA submission."
Clinical Background and Potential Benefits
ATI-1801 has already demonstrated safety and efficacy across multiple Phase 2 and Phase 3 studies. Cutaneous leishmaniasis affects hundreds of thousands of people globally, and current treatments can be invasive and require hospitalization. ATI-1801 offers the potential for a safe and effective topical therapy that can be used in an outpatient setting, significantly reducing patient suffering.
Regulatory and Financial Considerations
ATI-1801 has received Orphan Drug Designation from the FDA for certain forms of cutaneous leishmaniasis. Appili is also evaluating ATI-1801’s eligibility for a priority review voucher (PRV). Subject to securing the requisite funding Appili expects to implement the agreed-upon strategy and continue to advance ATI-1801 topical paromomycin cream (15% w/w) program to NDA submission.
Aditxt Transaction
Appili also provided an update on its arrangement agreement with Aditxt Inc., where Aditxt, through its subsidiary Adivir, Inc., will acquire all of Appili's Class A common shares. Shareholders are encouraged to vote on this transaction at the upcoming special meeting on November 6, 2024.
