Drug Farm Announces IND Clearance by U.S. FDA Enabling Phase 1b Initiation for ALPK1 Selective Kinase Inhibitor, DF-003 in ROSAH Syndrome Patients
17 Mayo 2024 - 4:34AM
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:
https://www.drug-farm.com.
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Henri Lichenstein, Ph.D. Chief Executive Officer Email:
hlichens@drug-farm.com