Drug Farm announced that the FDA has granted Fast Track designation to DF-003, its investigational, first-in-class ALPK1 inhibitor for the treatment of ROSAH syndrome. ROSAH syndrome is a rare, autosomal dominant autoinflammatory disease with no approved therapies.

The FDA’s Fast Track designation is intended to accelerate the development and review of drugs that address serious or life-threatening conditions with unmet medical needs. The designation provides opportunities for more frequent interactions with the FDA and allows eligibility for rolling review and, if applicable criteria are met, priority review.

DF-003 is a structurally novel, orally administered small-molecule inhibitor designed to selectively and potently inhibit disease-causing mutant ALPK1, including the ROSAH-associated ALPK1 T237M variant. In preclinical studies, DF-003 demonstrated the ability to cross the blood–retina and blood–brain barriers and significantly suppress inflammatory cytokines and disease-associated phenotypes in a mouse model of ROSAH syndrome.¹ These data supported the advancement of DF-003 into clinical development.

“We have designed DF-003 to directly target the genetic root cause of ROSAH syndrome,” said Henri Lichenstein, PhD, Chief Executive Officer of Drug Farm. “The FDA’s Fast Track designation recognizes the serious nature of this disease and the urgent need for targeted, disease-modifying therapies, and it supports our efforts to accelerate development of DF-003 for patients.”

DF-003 is the first ALPK1 inhibitor to successfully complete a first-in-human phase 1 clinical study and has now entered a phase 1b trial in patients with ROSAH syndrome (ClinicalTrials.gov Identifier: NCT06395285). The ongoing study is evaluating the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of DF-003. Clinical data from the trial are expected to be presented at scientific meetings in 2026.

Reference

1. Fan J, et al. Discovery of a selective alpha-kinase 1 inhibitor for the rare genetic disease ROSAH syndrome. Nature Communications. 2025 Sep 9;16(1):8251. doi:10.1038/s41467-025-63731-5. PMID: 40925900.