ARYA-24 is a first-in-class small molecule designed in silico to target a novel transcription factor, demonstrating superior in vitro cytotoxicity against AML cells compared to current standard-of-care agents in NCI-60 testing. The program is advancing toward target confirmation with a robust patent estate and orphan drug eligibility.
ARYA-24 was designed in silico to target a novel transcription factor implicated in tumor cell metabolism and cell cycle regulation. Target engagement has not yet been confirmed by bioassay. In NCI-60 testing, ARYA-24 showed cytotoxicity where ibrutinib (BTK inhibitor) showed none, suggesting a differentiated mechanism from current AML standard of care.
In HL-60(TB) AML cells, ARYA-24 demonstrated 93% cytotoxicity — more than double the 43% observed with venetoclax, while ibrutinib showed no cytotoxic activity (0%) in head-to-head NCI-60 testing. ARYA-24 also shows broad activity across nine tumor types.
At cytotoxic concentrations for leukemia and HCC cells, ARYA-24 does not kill L6 skeletal muscle cells or 3T3-L1 adipocyte models. In fact, it drives beneficial metabolic effects in non-malignant cells.
A companion diagnostic patent application has been filed based on the computationally predicted target. If target engagement is confirmed by bioassay, target protein expression may enable patient stratification and a precision medicine approach.
Two USPTO provisional patent applications filed covering two lead scaffolds with broad composition-of-matter claims. Non-provisional filings planned to extend protection through 2046, augmented by seven-year Orphan Drug exclusivity.
ARYA-24 is eligible for Orphan Drug Designation in AML, ALL, and CML, and Fast Track designation to accelerate Phase I/II trials. Regulatory incentives include seven years of market exclusivity and reduced fees.