Evaluating 3K3A-APC in Patients With ALS: Kent Pryor, PhD

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The CEO of ZZ Biotech outlined the design of the phase 2 trial of 3KA-APC, which will enroll 16 patients with amyotrophic lateral sclerosis. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

“There are basically 2 broad categories of approaching ALS clinical research right now. One is going after genetic forms of the disease—looking at a particular slice of patients and going after it that way. But what 3K3A-APC does, it addresses the underlying pathologies of the disease, pretty much regardless of the genetic aspects of the disease.”

A phase 2 clinical trial (NCT05039268) of an investigational treatment from ZZ Biotech, 3K3A-APC, recently began dosing patients with amyotrophic lateral sclerosis (ALS). The experimental drug, which previously saw success in acute ischemic stroke, is a genetically engineered variant of human active protein C (APC) that investigators hope will both protect motor neurons and attenuate microglial cells in patients with ALS, regardless of the genetic basis of the disease.

NeurologyLive^® recently sat down with the CEO of ZZ Biotech, Kent Pryor, PhD, to discuss the trial’s design and end points, as well as the potential of the treatment if successful in this patient population. Pryor outlined the trial's design, which is set to enroll 16 patients, divided into 2 cohorts to receive 5 doses of either 15-mg or 30-mg 3K3A-APC, given intravenously 12 hours apart. The trial will be conducted at Macquarie University in Sydney, Australia.

Pryor spoke on the unique aspects of the phase 2 trial, noting that investigators are still working to identify the optimal dosing regimen in patients with ALS. The regimen was also used in the phase 1 study and the phase 2 RHAPSODY trial (NCT02222714) in stroke, Pryor said, with the current trial helping to give investigators a better idea of the treatment’s ability to move different biomarkers and impact the ALS disease state.