Drug Farm Announces IND Clearance by U.S. FDA Enabling Phase 1 Initiation for First-in-Class Oral ALPK1 Selective Kinase Inhibitor, DF-003

IND demonstrates Drug Farm’s leadership in ALPK1, representing the Company's second ALPK1 program to enter clinical development

GUILFORD, Conn. & SHANGHAI, China--(BUSINESS WIRE)--Drug Farm announced today the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application of DF-003, a first-in-class, oral, potent, highly selective ALPK1 inhibitor for clinical evaluation.

ALPK1 has been linked to heart and kidney disease, and a mutated form of ALPK1 is the driver for the rare genetic disease, ROSAH, that leads to blindness. DF-003 thus has therapeutic promise to treat hundreds of millions of patients worldwide living with heart and chronic kidney disease and offer the first precision targeted drug for ROSAH syndrome.

DF-003 is a highly potent investigational inhibitor of ALPK1 and the ALPK1 T237M driver mutation that causes disease in ROSAH patients. DF-003 has demonstrated significant preclinical activity in heart and kidney disease in vivo models, as well as in ROSAH transgenic mouse models. In addition, DF-003 has favorable drug-like properties including a low risk for off-target activity and drug interactions, and a predicted human half-life consistent with once-a-day dosing.

“The FDA clearance to initiate clinical evaluation of DF-003, the second program in our ALPK1 franchise to enter human trials, is illustrative of the innovative approach by Drug Farm for drug discovery/development and exemplifies the best-in-class capabilities of our team. With the potency and selectivity shown by DF-003, and its ability to cross the blood-eye barrier, we are confident that our drug will inhibit ALPK1 in key target tissues, including heart, kidney, and retina," said Jeysen Yogaratnam, MB.BCh, MRCSEd, Ph.D., MBA, Chief Medical Officer, Drug Farm. "We are thrilled to advance our first-in-class drug, DF-003, into clinical development as a promising treatment for heart and kidney disease patients, as well as those afflicted with ROSAH syndrome."

The Phase 1 clinical trial will launch shortly with plans for evaluation of single and multiple ascending doses of DF-003 in normal healthy volunteers.

About Drug Farm

Drug Farm is a private biotechnology Company developing innovative treatments targeting innate immunity for hepatitis B, heart and kidney diseases, and ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome. Drug Farm's unique IDInVivo platform combines breakthrough technologies in genetics and AI to discover new treatments. IDInVivo technology allows the direct assessment of gene targets in living animals with intact immune systems. Using the IDInVivo platform, Drug Farm has identified novel innate immunity pathways and targets and is now rapidly advancing multiple first-in-class drug candidates into clinical development. For more information please visit: www.drug-farm.com