Drug Farm Announces FDA Acceptance of DF-003 into Rare Disease Evidence Principles Process for ROSAH Syndrome

ALBANY, N.Y. & SHANGHAI--(BUSINESS WIRE)--Drug Farm today announced that the U.S. Food and Drug Administration (U.S. Food and Drug Administration, FDA) has accepted DF-003, the company’s investigational therapy for ROSAH syndrome, into the FDA Rare Disease Evidence Principles Process (RDEP).

The RDEP is an FDA initiative designed to facilitate early dialogue between the agency and sponsors developing therapies for rare diseases. Through the program, FDA works with selected sponsors to discuss regulatory strategies, clinical evidence development, and innovative approaches to generating the evidence needed to support potential regulatory submissions.

DF-003 is being developed as a potential treatment for ROSAH syndrome, a rare autoinflammatory disorder caused by gain-of-function mutations in the ALPK1 gene. The disease is characterized by retinal degeneration, optic nerve edema, systemic inflammation, and progressive vision loss. Currently, there are no approved therapies specifically indicated for ROSAH syndrome.

Participation in the RDEP program will allow Drug Farm to engage in structured discussions with the FDA regarding the development of DF-003, including potential approaches to clinical trial design, evidence generation in small patient populations, and strategies for demonstrating clinical benefit in this rare condition.

“Acceptance into the Rare Disease Evidence Principles Process provides an important opportunity for early collaboration with the FDA as we advance DF-003 for patients with ROSAH syndrome,” said Henri Lichenstein, Ph.D., Chief Executive Officer at Drug Farm. “Given the rarity of this disease and the lack of approved treatments, we believe engagement through RDEP will help inform efficient development strategies and accelerate progress toward potential therapies for patients.”

Drug Farm plans to initiate further clinical development activities for DF-003 following discussions with the FDA through the RDEP program.

About DF-003

DF-003 is a proprietary, first-in-class drug developed by Drug Farm that inhibits the activity of ALPK1 and variants of ALPK1 which cause ROSAH syndrome. DF-003 has therapeutic potential for ROSAH syndrome, as well as heart and kidney diseases, as the drug has shown efficacy in preclinical models of these indications. DF-003 has completed a Phase 1 clinical trial (NCT05997641) in normal healthy volunteers and is now accruing patients with ROSAH syndrome in a Phase 1b trial (NCT06395285).

About ROSAH Syndrome

ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache) syndrome is a rare, autosomal dominant autoinflammatory genetic disease caused by activating mutations in the ALPK1 gene. The disease is characterized by progressive visual loss, optic nerve and retinal pathology, and systemic inflammatory manifestations, including elevated pro-inflammatory cytokines. Symptoms often begin in childhood or early adulthood, and there are currently no approved disease-modifying therapies for ROSAH syndrome.

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 syndrome. Drug Farm’s unique IDInVivo platform combines breakthrough technologies in genetics and artificial intelligence to identify and validate novel drug targets directly in living animals with intact immune systems. Using this platform, Drug Farm is advancing multiple first-in-class drug candidates into clinical development.

For more information, please visit: https://www.drug-farm.com