ZZ Biotech is a clinical stage biopharmaceutical company advancing new treatments for some of the biggest unmet needs in stroke, neurodegenerative disease and chronic wound healing. Our lead drug, 3K3A-APC, is a genetically engineered variant of human activated protein C (APC), a serine protease. 3K3A-APC was designed to enhance the cytoprotective and anti-inflammatory activities of APC while greatly reducing its anti-coagulant activity.
3K3A-APC is a genetically engineered variant of human activated protein C (APC), a serine protease with two biochemical pathways in the body. APC exerts anticoagulant activity by inactivation of Factor Va and VIIIa, and it exerts cytoprotective and anti-inflammatory activities primarily through interaction with the endothelial protein C receptor (EPCR) and protease-activated receptor-1 (PAR-1). However, at therapeutic doses, natural APC introduces an unacceptable risk for bleeding due to its anticoagulant activity. ZZ Biotech’s experimental drug 3K3A-APC was designed through elegant biochemistry and molecular biology to enhance the beneficial cytoprotective and anti-inflammatory activities of APC while greatly reducing its anti-coagulant activity to eliminate bleeding risk.
ZZ Biotech has completed RHAPSODY, a placebo-controlled multicenter Phase 2 clinical trial of 3K3A-APC in patients suffering from acute ischemic stroke. The study was supported and funded by the NIH’s National Institute of Neurological Disorder and Stroke (NINDS) through a pair of NeuroNEXT grants. The Phase 2 study evaluated safety, tolerability and vasculoprotective activity of ZZ Biotech’s experimental drug 3K3A-APC when given after tissue plasminogen activator (tPA), mechanical thrombectomy, or both, in patients who have experienced moderate to severe ischemic stroke. Four dose levels of 3K3A-APC were evaluated in 110 participants, ages 18 to 90, who were followed for 90 days. 3K3A-APC was deemed safe and well-tolerated at all dose levels tested. Furthermore, intracerebral bleeding was substantially reduced in patients treated with 3K3A-APC.
In June 2020 the FDA designated the investigation of the ZZ Biotech’s experimental drug 3K3A-APC for the treatment of acute ischemic stroke as a Fast Track development program. FDA Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier.
ZZ Biotech is planning a multi-national pivotal Phase 3 clinical trial in acute ischemic stroke patients to begin in early 2023.
ZZ Biotech also has active clinical programs in ALS and wound healing. 3K3A-APC’s unique multi-modal mechanism of action is well suited to address both of these areas of great unmet medical need. ZZ Biotech commenced a Phase 2 biomarker study in ALS in December 2021. The study is being conducted at Macquarie University, in Sydney, Australia, and seeks to investigate whether 3K3A-APC is safe and potentially effective in patients with ALS. The Firies Climb for Motor Neurone Disease provided an AU$1 million donation to support this clinical trial.