ALPK1

GUILFORD, Conn. & SHANGHAI--(BUSINESS WIRE)-- Drug Farm announced today the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application of DF-003, a first-in-class, oral, potent, highly selective alpha-kinase 1 (ALPK1) inhibitor for clinical evaluation in ROSAH syndrome patients. The trial will assess the safety, pharmacokinetics and efficacy of DF-003 in ROSAH patients. DF-003 is a first-in-class, potent, highly selective inhibitor of the gain-of-function ALPK1 mutation responsible for causing ROSAH syndrome. Drug Farm has demonstrated significant preclinical activity for DF-003 in a ROSAH mouse model of disease and has received a Rare Pediatric Designation from FDA. Furthermore, DF-003 has completed Phase 1 evaluation in healthy subjects with safety and pharmacokinetics supporting a once-a-day oral dosing regimen in the upcoming ROSAH patient trial. “We are delighted to offer a promising new therapeutic with the potential to improve ocular and systemic outcomes in ROSAH syndrome,” said Lloyd Williams, M.D., Ph.D., Director of Global Ophthalmology, Duke University. “Until now, treatments for ROSAH have addressed the symptoms, but not the genetic root cause of disease. We have developed a precision drug that inhibits mutant ALPK1 and may stop progression of disease in patients afflicted with ROSAH,” said Dr. Jeysen Yogaratnam, Chief Medical Officer, Drug Farm. “We are thrilled with the rapidity that Drug Farm has executed on the DF-003 program as our mission is to develop a safe, targeted therapy for ROSAH patients. Drug Farm preclinical data also supports the use of DF-003 as a uniquely differentiated candidate for cardio-renal disease and we are excited about the prospects of our drug in this patient population as well,” said Dr. Henri Lichenstein, CEO, Drug Farm. 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 (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome. Drug Farm's unique IDInVivo platform combines breakthrough technologies in genetics and AI to discover new treatments. IDInVivo technology allows the direct assessment of gene targets in living animals with intact immune systems. Using the IDInVivo platform, Drug Farm has identified novel innate immunity pathways and targets and is now rapidly advancing multiple first-in-class drug candidates into clinical development. For more information please visit: . View source version on businesswire.com: Contacts Henri Lichenstein, Ph.D. Chief Executive Officer Email: hlichens@drug-farm.com Source: Drug Farm View this news release online at:

GUILFORD, Conn.--(BUSINESS WIRE)-- Drug Farm, a private biotechnology company utilizing genetics and artificial intelligence technologies to discover and develop innovative, immune-modulating therapies, today announced that the chronic hepatitis B patient data from their Phase 1b trial (ACTRN12621000592842) was accepted as a late breaking poster presentation at the 2024 European Association for the Study of the Liver (EASL) conference that will be held in Milan, Italy between 05 June 2024 to 08 June 2024. Poster #LB46 Title: A phase 1, double-blinded, randomized, placebo-controlled, multicenter global study evaluating the safety, tolerability, and antiviral activity of DF-006 in chronic hepatitis B, virologically-suppressed patients. Date: 05 June 2024 Time: 8.30 am – 5.00 pm CET DF-006 is a first-in-class, orally administered ALPK1 agonist immunomodulator that potently stimulates local, innate immunity in the liver1. DF-006 has successfully completed single and multiple ascending dose (MAD) evaluation in healthy volunteers, and MAD evaluation in virologically suppressed HBeAg negative chronic hepatitis B (CHB) patients. “There remains no effective cure for CHB. DF-006 has a novel mechanism of action that is capable of stimulating the body’s own immune system to help clear the infection. As an oral drug administered once weekly, DF-006 has the potential to be the immunomodulator of choice in combination HBV cure regimens”, said Dr. Ed Gane, Professor of Medicine at the University of Auckland, New Zealand and Chief Hepatologist, Transplant Physician, and Deputy Director of the New Zealand Liver Transplant Unit at Auckland City Hospital. “DF-006 has demonstrated potent antiviral responses in preclinical studies, including inhibition of cccDNA and recruitment of T-cells. The data from this trial study now provides clinical support for a drug with a novel mechanism of action that could be used to help cure CHB patients”, said Drug Farm’s Chief Medical Officer, Dr. Jeysen Yogaratnam. About Drug Farm. Drug Farm is a private biotechnology Company developing innovative treatments targeting innate immunity for hepatitis B, cancer, and autoimmune diseases. Drug Farm's unique IDInVivo platform combines breakthrough technologies in genetics and AI to discover new treatments. IDInVivo technology allows the direct assessment of gene targets in living animals with intact immune systems. Using the IDInVivo platform, Drug Farm has identified novel innate immunity pathways and targets and is now rapidly advancing multiple first-in-class drug candidates into clinical development. For more information please visit: Reference: Xu C, et al. Hepatology. 2023 Jan 1;77(1):275-289. doi: 10.1002/hep.32614. Epub 2022 Sep 17. View source version on businesswire.com: Contacts United States: Henri Lichenstein, Ph.D. Chief Executive Officer Email: hlichens@drug-farm.com China: Tony Xu, Ph.D. Co-founder and Chief Operating Officer Email: tony.xu@drugfarminc.com Source: Drug Farm View this news release online at:

ROSAH syndrome is caused by mutations in the ALPK1 gene. Credit: Gorodenkoff via Shutterstock. Drug Farm has been granted a rare paediatric disease (RPD) designation from the US Food and Drug Administration (FDA) for DF-003 to treat retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache (ROSAH) syndrome. DF-003 is an alpha-kinase 1 (ALPK1) indicated for the treatment of the rare autosomal dominant disorder ROSAH syndrome. The condition is caused by mutations in the ALPK1 gene, characterised by the symptoms listed in its name. ROSAH syndrome currently has no approved treatments. Drug Farm received FDA clearance of its investigational new drug (IND) application to initiate Phase I studies for the candidate in June 2023, after pre-clinical studies in ROSAH syndrome transgenic mouse models showed significant activity and favourable drug-like properties. If a new drug application for DF-003 is approved by the FDA, the company may be eligible to receive a priority review voucher (PRV) that can be redeemed to receive for any later marketing application. The double-blind, placebo-controlled Phase I trial (NCT05997641) is evaluating the safety, tolerability, and pharmacokinetics of DF-003 on 96 healthy subjects.  The study is expected to conclude in Q1 2025. The company has other candidates in the pipeline, including another ALPK1 agonist DF-006, which is under investigation in the third part of a Phase I study for the treatment of chronic hepatitis B infection. The trial is assessing the levels of DF-006 and its related compounds in the blood following doses of the formulation. In May 2023, Drug Farm generated $27m in a Series C funding round led by Betta Capital. The company, which has offices in both the US and China, also raised $56m in February 2021 in a Series A lead by BVCF. In the announcement accompanying the designation, Drug Farm’s chief medical officer Jeysen Yogaratnam said: “Obtaining Rare Pediatric Disease Designation recognizes the serious and debilitating complications of this rare disease and upholds our goal to provide DF-003 as the first targeted drug for potential treatment in patients afflicted with ROSAH syndrome.”