ALBANY, N.Y. & SHANGHAI, China — Drug Farm today announced that preliminary immunologic and clinical results from its Phase 1b study of DF-003, a first-in-class oral ALPK1 inhibitor, will be presented in a podium session at IMMUNOLOGY2026, taking place April 15–19, 2026, in Boston, Massachusetts.
The presentation will highlight early evidence of clinical benefit and pharmacodynamic improvement in patients with ROSAH syndrome, a rare genetic autoinflammatory disease driven by disease-causing mutations in ALPK1.
Presentation Details
The presentation, titled “Preliminary immunologic and clinical Phase 1b study outcomes from patients with ROSAH syndrome treated with the first-in-class ALPK1 inhibitor DF-003,” will be delivered during the session “Interrogation of Chronic Immunity and Interventional Approaches.” It is scheduled for Friday, April 17, 2026, from 12:45 PM to 1:00 PM in Boston, Massachusetts.
Key Findings from Phase 1b Study
In this multicenter, open-label Phase 1b study (NCT06395285), six adult patients with genetically confirmed ROSAH syndrome associated with the ALPK1 T237M mutation received once-daily oral DF-003 treatment for four weeks.
Clinical improvements were observed across multiple assessments. Four of the six patients experienced reversal of anhidrosis, and improvements were also noted in arthralgia and overall quality of life as measured by a standardized EQ-5D questionnaire. Importantly, these clinical benefits were reversed upon treatment discontinuation, supporting a drug-related effect.
Pharmacodynamic activity was also demonstrated. In a patient with elevated baseline inflammation, treatment with DF-003 led to normalization of key inflammatory biomarkers, including interleukin-6 (IL-6), high-sensitivity C-reactive protein (hsCRP), interleukin-8 (IL-8), and CXCL10. These findings provide early evidence of ALPK1 pathway modulation in humans.
Evidence of organ-level impact was observed in one evaluable patient without prior splenectomy, who demonstrated an approximately 20% reduction in spleen volume after 28 days of treatment. This effect was reversed following treatment discontinuation.
DF-003 was well tolerated, with no serious adverse events or treatment-emergent adverse events reported during the 28-day dosing period. Renal and hepatic function remained stable across all participants, and pharmacokinetic data support once-daily oral dosing.
KOL Perspective
“These early clinical and immunologic findings are highly encouraging, particularly given the lack of targeted treatment options for patients with ROSAH syndrome,” said Professor John Grigg, Head of the Specialty of Clinical Ophthalmology and Eye Health, Save Sight Institute Faculty of Medicine and Health at The University of Sydney. “The observed improvements in anhidrosis, systemic inflammation, symptom burden, and spleen size—together with a favorable safety profile—support ALPK1 as a promising therapeutic target and warrant continued clinical development of DF-003.”
Company Statement
“These preliminary Phase 1b data provide the first clinical evidence that selective inhibition of ALPK1 can modulate inflammatory signaling and translate into clinical improvements in patients with ROSAH syndrome,” said Henri Lichenstein, PhD, Chief Executive Officer of Drug Farm. “The observed reversibility of both clinical and biomarker responses further supports a direct pharmacologic effect of DF-003, and we look forward to presenting additional clinical data on ophthalmological and headache improvement at a meeting later this year.”
Next Steps
Based on these findings, Drug Farm plans to advance DF-003 into a pivotal Phase 3 trial to further evaluate its efficacy, safety, and potential to address the underlying biology of ROSAH syndrome.
About DF-003
DF-003 is a proprietary, first-in-class drug developed by Drug Farm that inhibits the activity of ALPK1 and its disease-causing variants. It has therapeutic potential for ROSAH syndrome as well as heart and kidney diseases, based on efficacy observed in preclinical models. DF-003 has completed a Phase 1 clinical trial (NCT05997641) in healthy volunteers and is currently enrolling patients with ROSAH syndrome in an ongoing Phase 1b study (NCT06395285). DF-003 has received Fast Track, Orphan Drug, Rare Pediatric Disease, Rare Disease Evidence Principles designations from the FDA.
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. It 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.
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. Its proprietary IDInVivo platform integrates genetics and artificial intelligence to identify and validate novel drug targets directly in living systems. Drug Farm is advancing multiple first-in-class drug candidates into clinical development. For more information, please visit: https://www.drug-farm.com
Contacts
United States
Henri Lichenstein, Ph.D.
Chief Executive Officer
Email: [email protected]
China
Tony Xu, Ph.D.
Co-founder and Chief Operating Officer
Email: [email protected]