In aged cynomolgus macaques with spontaneous type 2 diabetes, animals broadly comparable to humans over seventy-five, a single regimen was administered by two routes, intravenous and intrathecal. Dosing is ongoing.
Six efficacy domains were assessed: glucose control, liver function, muscle mass, neuromuscular activity, lipid metabolism, and cognition. In every one of them the between-group difference favored treatment, and in four of the six the control group moved in the opposite direction. Twelve of thirteen measured readouts moved favorably.
Glycated hemoglobin, the standard measure of long-term glycemic control, declined in treated animals to below the level targeted by standard of care. The principal target organ was unaffected: nucleic-acid drugs accumulate predominantly in the liver, yet liver enzymes declined in treated animals and rose in controls, moving monotonically at every time point in both groups. Creatinine, low at baseline in these aged animals, returned toward the reference range, accompanied by an increase in spontaneous activity. On a working-memory task, every treated animal evaluated performed below chance at baseline and above chance following treatment.
The single readout that moved in the unfavorable direction, uric acid, follows the purine pathway by which nucleic acids are metabolized, and remains under monitoring. No treatment-related adverse events, withdrawals, or dose adjustments occurred.
A single regimen produced readouts in four organ systems corresponding to four diseases: type 2 diabetes, NASH/NAFLD, sarcopenia, and Alzheimer's disease. The readouts are independent of one another, and all trace to the same upstream mechanism.
Domain 01Glucose control
Glycated hemoglobin, pre-prandial glucose, fasting glucose and insulin.
Favorable
Domain 02Liver function
Transaminases declined in treated animals and rose in controls.
Favorable
Domain 03Muscle mass
Creatinine returned from below range toward the reference interval.
Favorable
Domain 04Neuromuscular activity
Spontaneous activity increased in treated animals.
Favorable
Domain 05Lipid metabolism
Triglycerides and total cholesterol trended favorably versus controls.
Favorable
Domain 06Cognition
Working-memory performance moved from below chance to above chance.
Favorable
SafetySafety & tolerability
Six electrolytes were unremarkable, and no adverse events, withdrawals, or dose adjustments occurred.
Monitored
Counter-readoutUric acid
Follows the purine pathway of nucleic-acid metabolism; under continued monitoring.
Counter · Monitored