Drug Farm Gets FDA IND Clearance to Start Phase 1b for DF-003 in ROSAH Syndrome

Drug Farm, a biotechnology company based in Guilford, Conn. and Shanghai, has announced that the U.S. Food and Drug Administration (FDA) has given clearance for the Investigational New Drug (IND) application of DF-003. This drug is a pioneering oral medication specifically designed to inhibit alpha-kinase 1 (ALPK1) and is set for clinical evaluation in patients suffering from ROSAH syndrome. The upcoming clinical trials will examine the safety, pharmacokinetics, and efficacy of DF-003 in treating ROSAH syndrome.

DF-003 stands out due to its potency and high selectivity as an inhibitor targeting the ALPK1 mutation, which plays a crucial role in causing ROSAH syndrome. The company's research has shown promising preclinical activity of DF-003 in a mouse model mimicking ROSAH disease. The FDA has also granted DF-003 a Rare Pediatric Designation, emphasizing the drug's potential significance. Moreover, Phase 1 trials involving healthy subjects have already been completed, indicating that the drug is safe and supports a once-daily oral dosing schedule for the forthcoming trials involving ROSAH patients.

Dr. Lloyd Williams, Director of Global Ophthalmology at Duke University, expressed enthusiasm about the potential impact of DF-003. He highlighted that existing treatments for ROSAH syndrome have only been able to address the symptoms rather than the underlying genetic cause. DF-003, by inhibiting the mutant ALPK1, aims to halt the progression of the disease, providing a more targeted and effective treatment option.

Dr. Jeysen Yogaratnam, Chief Medical Officer at Drug Farm, emphasized the significance of developing a precision drug that targets the root cause of ROSAH syndrome. He praised the rapid progress of the DF-003 program, aligning with the company's goal to create a safe and targeted therapy for ROSAH patients. Dr. Henri Lichenstein, CEO of Drug Farm, added that preclinical data support the potential use of DF-003 not only for ROSAH but also for cardio-renal diseases, marking the drug as a uniquely differentiated candidate for broader applications.

Drug Farm is a privately-owned biotechnology company focusing on innovative treatments targeting innate immunity. These treatments span various diseases, including hepatitis B, heart and kidney diseases, and ROSAH syndrome (characterized by retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache). Drug Farm employs its proprietary IDInVivo platform, which combines advancements in genetics and artificial intelligence (AI) to discover new treatments. The IDInVivo technology facilitates the direct assessment of gene targets in living animals with intact immune systems, enabling the identification of novel pathways and targets in innate immunity. Currently, Drug Farm is advancing multiple first-in-class drug candidates into clinical development stages, driven by the insights gained from the IDInVivo platform.

As Drug Farm continues to develop DF-003 and other innovative treatments, the company's efforts signify a promising advancement in the medical field, particularly for patients suffering from rare and currently inadequately treated conditions like ROSAH syndrome.