ZZ Biotech Announces First Patients Dosed in Phase 2 Clinical Trial of 3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS)

ZZ Biotech, a clinical stage biopharmaceutical company developing the experimental drug 3K3A-APC for some of the biggest unmet needs in stroke, neurodegenerative disease and chronic wound healing, today announced that the first patients have been dosed in a Phase 2 clinical trial evaluating 3K3A-APC for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. 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.

The Phase 2 open label trial (NCT05039268) will enroll a total of 16 patients with ALS into two dose cohorts. The primary study outcomes are to ensure the safety and tolerability of 3K3A-APC in ALS patients and to determine whether 3K3A-APC is able to reduce the pathological changes that are thought to cause ALS. The study will evaluate biomarkers of microglial and monocyte activation and should provide evidence as to whether microglial activation is a major pathogenic contributor to ALS.

ZZ Biotech has completed a Phase 2 clinical trial of 3K3A-APC in acute ischemic stroke patients called RHAPSODY. 3K3A-APC has received Fast Track designation from the U.S. FDA for the treatment of stroke. The beneficial cytoprotective activity of 3K3A-APC on multiple cell types in the brain holds great promise for ALS as well as stroke.

"We have been developing 3K3A-APC for some time now, and we are making great strides in studying it for stroke," said Kent Pryor, Chief Executive Officer of ZZ Biotech. "The extraordinary generosity of the Firies Climb for Motor Neurone Disease and their donors has allowed us to initiate a clinical program in ALS with 3K3A-APC, another area of great unmet medical need."

The trial is being led by neurology Professor Dominic Rowe, M.D., PhD, at the Macquarie University Centre for Motor Neuron Disease Research, the largest motor neuron research center in Australia. The Macquarie University Centre for Motor Neuron Disease Research is one of the most active centers in Australia for ALS clinical research.

"3K3A-APC has shown potent anti-inflammatory effects, along with repairing the blood-brain barrier, both of which are challenges in ALS," said Professor Dominic Rowe, M.D., PhD. "I am extremely excited to test this interesting experimental drug for the first time in ALS patients. Currently, we can't cure ALS, and we can't reverse it. But with early diagnosis, and if we can dramatically slow the progression of disease, I firmly believe we can turn this disease around."

About 3K3A-APC

ZZ Biotech's experimental drug 3K3A-APC is a genetically engineered variant of human activated protein C (APC), a serine protease with two biochemical pathways in the body. 3K3A-APC was designed to enhance the beneficial cytoprotective and anti-inflammatory activities of APC while greatly reducing its anti-coagulant activity.

About ZZ Biotech

Headquartered in Houston, Texas, ZZ Biotech, LLC, is a clinical stage company developing 3K3A-APC for ischemic stroke, other neurological diseases and wound healing applications including the treatment of diabetic foot ulcers. ZZ Biotech has completed a successful National Institute of Neurological Disorder and Stroke-funded Phase 2 clinical study, RHAPSODY, in acute ischemic stroke patients. In June 2020, the U.S. FDA designated 3K3A-APC for the treatment of acute ischemic stroke as a Fast Track development program.

About Macquarie University

Macquarie ? ranked among the top one per cent of universities in the world ? is recognised for producing graduates who are among the most sought-after professionals in the world. Since our foundation 55 years ago, we have aspired to be a different type of university: one focused on fostering collaboration between students, academics, industry and society. We are uniquely located in the heart of Australia's largest high-tech precinct, and bring together more than 40,000 students and 3000 staff in one thriving hub of discovery. More than 300 leading companies are located on or around the Macquarie campus, giving our students the opportunity to tap into industry connections that give them an edge in their future careers.