Drug Farm Announces Positive Phase 1 Safety and PK Results for DF-003, a First-in-Class Treatment for ROSAH Syndrome and Cardio-Renal Disease

Drug Farm, a private biotechnology company leveraging genetics and artificial intelligence, recently announced positive results from a Phase 1 first-in-human trial of DF-003, a first-in-class immune-modulating alpha-kinase 1 (ALPK1) inhibitor, in healthy volunteers. The findings were presented at the American Society for Clinical Pharmacology & Therapeutics (ASCPT) meeting in Washington, DC. The Phase 1 study, which was randomized, placebo-controlled, and double-blinded, aimed to evaluate the safety, tolerability, and pharmacokinetics of DF-003. It included forty-eight healthy volunteers divided into two portions. The first portion involved single ascending doses ranging from 3 mg to 150 mg, administered orally across five cohorts. The second portion involved 50 mg of DF-003 being administered daily for fourteen days in one cohort. Each cohort was randomized 3:1 (DF-003: placebo). The results of the trial were promising. DF-003 was deemed safe across all doses tested, with no serious adverse events (SAEs) reported. The rates of treatment-emergent adverse events (TEAEs) were similar between DF-003 and placebo groups, and none of the TEAEs necessitated dose modification or interruption. The drug's pharmacokinetic profile was largely dose-proportional, indicating that it could be effectively administered as a once-daily oral medication. According to Dr. Neil Solomons, Head of Clinical Development at Drug Farm, the safety data and pharmacokinetic profile of DF-003 are encouraging. "We are happy to share the results from our Phase 1 study that demonstrate the excellent safety of DF-003," he stated. "Additionally, the pharmacokinetic data show trough concentrations in blood that are consistent with efficacy in preclinical models of ROSAH syndrome and cardio-renal disease. This supports our plan to move forward with proof-of-concept trials in both patient populations." Dr. Henri Lichenstein, CEO of Drug Farm, emphasized the significance of these findings. "This is a major inflection point for our company," he said. "DF-003 is a groundbreaking, immune-modulating drug that specifically targets the disease-causing mutations of ROSAH syndrome and the excessive activation of the ALPK1 pathway in cardio-renal disease. With no approved drugs for ROSAH syndrome, DF-003 has the potential to significantly improve the lives of patients suffering from this devastating condition." ROSAH syndrome is a rare, autosomal dominant autoinflammatory genetic disease characterized by a combination of retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache. Mutations in ALPK1 are known to cause this syndrome. Symptoms often include progressive vision loss, starting before age 20, along with optic disc elevation, uveitis, and retinal nerve degeneration. Many patients also experience inflammatory symptoms such as non-infectious low-grade fevers, joint pain, and headaches, accompanied by elevated levels of inflammatory cytokines like TNFα, IL-6, and IL-1β. Preclinical studies have shown that inhibiting ALPK1 can lead to significant improvements in these conditions, making DF-003 a promising candidate. Following the positive Phase 1 results, Drug Farm is now enrolling patients with ROSAH syndrome in a Phase 1b trial (NCT06395285) to further assess the drug's safety and efficacy. Drug Farm's innovative approach to drug discovery and development is rooted in its IDInVivo platform, which integrates genetics and artificial intelligence to identify and validate new therapeutic targets directly in living animals with intact immune systems. This platform has enabled the company to uncover novel innate immunity pathways and is driving the rapid advancement of several first-in-class drug candidates. The positive outcomes of the Phase 1 trial of DF-003 mark a crucial step forward in the development of a treatment for ROSAH syndrome, a condition with no current FDA-approved therapy. Industry insiders laud Drug Farm's progress, noting the potential of DF-003 to address a significant unmet medical need. The company's pioneering use of AI and genetics-based platforms sets it apart in the field of rare disease research, offering hope for effective treatments where none currently exist.