A Study to Evaluate the Safety, Tolerability and Efficacy of XEN1101 in Major Depressive Disorder

Summary

This is a multicenter, Phase 2, double-blind, randomized, parallel-arm, placebo-controlled clinical trial to evaluate the efficacy, safety, and tolerability of XEN1101 in subjects with Major Depressive Disorder.

Timeline

Start

2022-05-19

Primary completion

2023-09-18

Completion

2023-10-16

Outcome

Missed primary endpoint

paper not statistically significant (-3.04 points; 95% CI, -7.04 to 0.96 points; P = .14) at week 6 PMID 40423966 ↗

release “A clear dose response and a clinically meaningful, but not statistically significant, 3.04 difference between placebo and the XEN1101 20 mg group (p=0.135) was observed.” xenon-pharma.com ↗

release “A clear dose response and a clinically meaningful, but not statistically significant, 3.04 difference between placebo and the azetukalner 20 mg groups (P=0.135) was observed.” xenon-pharma.com ↗

release “The investigator-sponsored Phase 2 proof-of-concept study of azetukalner in MDD led by Icahn School of Medicine at Mount Sinai did not meet its primary neuroimaging endpoint.” xenon-pharma.com ↗

Publications

• Butterfield NN, Luzon Rosenblut C, Fava M, Correll CU, Rothschild AJ, Murrough JW, Mathew SJ, Beatch GN, Grayson C, Harden C, Qian J, McIntosh J, Namdari R, Kenney C. Azetukalner, a Novel KV7 Potassium Channel Opener, in Adults With Major Depressive Disorder: A Randomized Clinical Trial. JAMA Netw Open. 2025 May 1;8(5):e2514278. doi: 10.1001/jamanetworkopen.2025.14278.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Azetukalner	Small molecule	10 mg	Oral
Subject	Azetukalner	Small molecule	20 mg	Oral

Indications