Drug Farm Partners with NIH to Develop ROSAH Syndrome Treatment

GUILFORD, Conn. & ROCKVILLE, Md.--Drug Farm, a biopharmaceutical company at the forefront of clinical-stage development, has announced a significant collaboration with the U.S. National Institutes of Health's National Institute of Allergy and Infectious Diseases (NIAID). This partnership entails a Cooperative Research and Development Agreement (CRADA) to advance DF-003, a pioneering drug aimed at treating ROSAH syndrome, a rare and complex genetic disorder.

DF-003 is a highly selective ALPK1 inhibitor with exceptional potency at the nanomolar level. The drug is capable of crossing the blood-retina barrier, a crucial feature for treating conditions affecting the eye. Preclinical studies have already demonstrated its efficacy in a transgenic mouse model specific to ROSAH syndrome. Having successfully concluded Phase 1 trials in healthy individuals, DF-003 is set to be tested in patients suffering from ROSAH syndrome to evaluate its safety, pharmacokinetics, and efficacy.

The partnership with NIAID will support a clinical trial (NCT06395285) sponsored by Drug Farm. The aim of this trial is to further investigate DF-003's capabilities in addressing the needs of those affected by ROSAH syndrome. Dr. Henri Lichenstein, CEO of Drug Farm, expressed enthusiasm about this collaboration, citing NIAID's leadership in ROSAH research as a critical component in potentially delivering a targeted therapeutic solution to patients.

ROSAH syndrome, known for its characteristic symptoms—retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headaches—is a rare genetic disorder with an autosomal dominant inheritance pattern. Mutations in the ALPK1 gene are responsible for the condition. The disorder typically manifests as a gradual decline in vision, often identifiable before individuals reach 20 years of age. Affected patients frequently exhibit optic disc elevation, uveitis, and retinal nerve degeneration. Beyond ocular symptoms, many patients experience systemic inflammatory signs such as low-grade fever, joint pain, and persistent elevation of inflammatory cytokines, including TNFα, IL-6, and IL-1β.

DF-003, developed by Drug Farm, represents a novel class of drugs targeting the ALPK1 mutations that cause ROSAH syndrome. The drug has completed initial clinical evaluations in healthy subjects, setting the stage for further trials in patients with the disorder.

Drug Farm itself is a private biotechnology company dedicated to developing groundbreaking treatments that focus on innate immunity. The company's research spans various conditions, including hepatitis B, as well as heart and kidney diseases, alongside ROSAH syndrome. A distinctive feature of Drug Farm's approach is the IDInVivo platform, which merges cutting-edge genetic and AI technologies. This platform is instrumental in identifying and validating new therapeutic targets by allowing researchers to assess gene targets in living organisms with functioning immune systems.

Through the IDInVivo platform, Drug Farm has unearthed novel pathways and targets within innate immunity, propelling multiple first-in-class drug candidates into clinical development. The alliance with NIAID and the ongoing development of DF-003 underscore Drug Farm's commitment to advancing medical science and providing innovative solutions for challenging health conditions like ROSAH syndrome.