AD101 (AmyriAD) is an innovative small molecule influencing the flow of calcium into the presynaptic neuron, which results in an enhanced release of the neurotransmitter acetylcholine. This unique mechanism leads to an upregulation of neurotransmission, ultimately benefiting learning and memory in animal models. AD101 has been evaluated extensively thus far, with 7 clinical trials involving over 500 participants with mild to moderate Alzheimer disease (AD). Encouragingly, data from these trials indicate that AD101 is well-tolerated and can be conveniently administered orally once daily (NCT00842816 and NCT00842673).¹

In a recent randomized, double-blind, placebo-controlled phase 2 study, AD101 displayed significant and statistically validated enhancements in global function and cognition when added to a stable dose of 10mg/day donepezil, as compared with donepezil alone. For context, donepezil has been part of the global standard of care for AD since 1996 and functions as an inhibitor of the enzyme acetylcholinesterase, safeguarding acetylcholine from degradation once it is released into the synapse.¹ Overall, AD101’s innovative approach of modulating calcium influx and acetylcholine release shows promise in improving cognitive function in patients with AD, offering new possibilities for treatment alongside existing medications like donepezil. AD101 has completed phase 2 clinical trials and is ready for phase 3, however the phase 3 trial has not been initiated.

In a recent interview with NeurologyLive®, Sharon L. Rogers, PhD, chief executive officer at AmyriAD Therapeutics, talked about the potential of AD101. She discussed how the therapy differs from the other drugs in the AD pipeline in its approach to treating the disease. Rogers, who previously served as a research chief at Eisai, also shared her reaction to the recent news on the traditional approval of lecanemab (Leqembi), the first traditionally approved agent in more than 20 years. In addition, she explained the ways that AD101 could potentially offer hope and benefits to patients with AD, their caregivers, and their providers.