PTG-300, AN HEPCIDIN MIMETIC PEPTIDE FOR TREATMENT OF INEFFECTIVE ERYTHROPOIESIS AND CHRONIC ANEMIA IN HEMOGLOBINOPATHY DISEASES
Ashok Bhandari
Ashok, Protagonist Therapeutics
Hepcidin is a key regulator of Iron Hemeostasis. Hepcidin binds to ferroportin, a major iron regulator. Reduced or absent of Hepcidin lead to iron imbalance in various diseases. Protagonist used its proprietary technology platform to identify a novel peptidic scaffold that matches key pharmacophores of hepcidin leading to PTG-300 as clinical development candidate. PTG-300 reduces cell surface expression of human ferroportin with an EC50 of 80% sustained reduction of serum iron over 48 hr after a single subcutaneous dose of 1 mg/kg. PTG-300 ameliorates ineffective erythropoiesis as a treatment for chronic anemia in a mouse model of β-thalassemia. Protagonist successfully completed Ph-1 clinical trials of PTG-300. De novo discovery, preclinical and Ph-1 clinical data will be presented
Dr. Ashok Bhandari is Vice President and Head of Chemistry at Protagonist. He has as over 20 years of experience in biotech industry and brings a unique blend of expertise in Peptide, Medicinal, Combinatorial Chemistry. His current research interests are discovery and early clinical development of orally stable peptides and CMC. Dr. Bhandari has broader knowledge and expertise in peptide drug lead optimizations leading to new chemical entities. He did his Post-doctoral research at University of Santa Barbara, California and he received his Ph.D. in chemistry from Indian Institute of Chemical Technology, India.