ZZ Biotech, LLC is a clinical stage company developing innovative biologic treatments for ischemic stroke, amyotrophic lateral sclerosis (ALS), other neurological diseases, and wound healing applications including the treatment of diabetic foot ulcers. ZZ Biotech was formed to focus on the development of 3K3A-APC, a novel second-generation variant of a naturally occurring human protein licensed from The Scripps Research Institute.
Eli Lilly received FDA approval in 2001 for wild-type activated protein C (wt-APC - Xigris) for the treatment of severe sepsis. Unfortunately, severe bleeding related to wt-APC’s anticoagulant activity led to poor market uptake and ultimately led to its withdrawal from the market.
ZZ Biotech’s 3K3A-APC is a genetically engineered variant of recombinant APC, a serine protease. APC has two biochemical pathways in the body. APC exerts anticoagulant activity by inactivation of Factor Va and VIIIa, and it exerts cytoprotective activity primarily through interaction with the endothelial protein C receptor (EPCR) and protease-activated receptor-1 (PAR-1). 3K3A-APC was designed through elegant biochemistry and molecular biology as a safer, second-generation version of Eli Lilly’s approved drug, with greatly reduced anti-coagulant activity.
ZZ Biotech has completed RHAPSODY, a Phase 2 clinical trial of 3K3A-APC in acute ischemic stroke and is initiating RHAPSODY-2, a Phase 3 registrational follow-up trial.
ZZ Biotech is undertaking a Phase 2a trial in ALS at Macquarie University Hospital in Sydney in 2021, based on outstanding animal data, and with the guidance and support of a number of the world's leading ALS researchers.
ZZ Biotech also acquired a complementary comprehensive suite of patents on the use of APC and engineered amino acid substitutions of APC in wound healing, wound scarring, burns, and inflammatory skin disorders, and is advancing 3K3A-APC through the requisite toxicology studies to commence a Phase 2 clinical trial in diabetic foot ulcer patients.
The company is funded by friends and family of Selim Zilkha. The scientific founder of the company was Dr. Berislav V. Zlokovic, Director, Zilkha Neurogenetic Institute at the Keck School of Medicine, University of Southern California. The name of the company, ZZ Biotech, is based on the names of these two founders—Zilkha and Zlokovic.