Dr. Cheng-Lung (Andy) Chen presented a scientific poster in “2022 Annual Meeting of Taiwan Neurological Society” titled “A Phase 1, Randomized, Double Blind, Placebo Controlled Study, To Evaluate the Safety, Tolerability and Pharmacokinetics of Single and Multiple Ascending Doses (SAD-MAD) of AJ201 in Healthy Volunteers”, in which pharmacological safety and pharmacokinetics (PK) of AJ201 in human, were reported (ClinicalTrials.gov Identifier: NCT04392830).

A total of 72 healthy volunteers were enrolled and randomized to receive either AJ201 (54 subjects) or Placebo (18 subjects). AJ201 showed an excellent safety profile with no serious adverse events reported and no adverse events leading to AJ201 discontinuation or subject withdrawal.

AJ201 showed favorable PK features, including a dose dependent increase in systemic exposure in both single and repeated administration. The absorption of AJ201 appeared to approach saturation at 600 mg/day. There was no apparent accumulation of systemic exposure after repeated dosing. Moreover, consumption of food did not significantly affect the PK profile of AJ201. For details, please download and refer to the PDF file below.

AJ201 is a first-in-class pleiotropic small molecule under development by AnnJi for the treatment of Kennedy’s disease, a rare neurological disorder. AJ201 has been granted with three Orphan Drug Designations (ODD) by the U.S. FDA for the indications of Kennedy’s disease, Huntington’s disease and Spinocerebellar ataxia (SCA). Patient enrollment of a Phase 2a trial for AJ201 will be initiated in the US by the end of 2022.