Category: Dystonia: Clinical Trials and Therapy
Objective: To evaluate the pooled efficacy and safety of 2 doses of DaxibotulinumtoxinA-lanm for injection (DAXI) for the treatment of cervical dystonia (CD) in 2 Phase 3 studies.
Background: DAXI is the first long-acting neuromodulator product, a novel botulinum toxin (BoNT) type A, recently approved by the FDA for treating CD in adults. Its unique formulation includes a proprietary peptide excipient. The product has been studied in multicenter, double-blind, placebo-controlled Phase 3 trials: a main clinical trial from the USA and EU (ASPEN-1), and a similarly designed, smaller extension clinical trial conducted in China (ASPEN-1-CN). Here, we report pooled data from both clinical trials for the first time.
Method: Adults with moderate-to-severe CD were randomized (3:3:1) to receive DAXI 250U, DAXI 125U, or placebo and were followed for up to 36 weeks after a single treatment. Participants were evaluated using the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS). The primary efficacy endpoint was the change from baseline in TWSTRS total score averaged across Weeks 4 and 6. A key secondary endpoint was the duration of effect, which was defined as the time from treatment until loss of >80% of the peak improvement in average TWSTRS total score achieved at Weeks 4 and 6. Safety was evaluated at all study visits.
Results: A total of 357 participants were randomized to DAXI 250U (n=154), DAXI 125U (n=149), or placebo (n=54). DAXI 250U and 125U significantly improved the mean TWSTRS total score vs placebo as per least squares mean [SE] difference: DAXI 250U, -6.4 [1.8], p=0.0004; DAXI 125U, -7.0 [1.8], p=0.0001. The median (95% CI) duration of effect was 22.0 (20.1, 24.3) weeks for DAXI 250U and 24.2 (20.6, 29.1) weeks for DAXI 125U. Treatment-related rates for dysphagia were 4.5%/4.7%, and for muscular weakness were 4.5%/5.4%, in the DAXI 250U/125U cohorts, respectively.
Conclusion: This pooled analysis of Phase 3 trials from the USA/EU (ASPEN-1) and China (ASPEN-1-CN) demonstrates that DAXI is an effective, safe, and long-acting treatment for CD. Key adverse events occurred at rates lower than prior pivotal trials of BoNT products for this indication.
To cite this abstract in AMA style:
D. Truong, H. Xingyue, A. Ellenbogen, J. Lingjing, K. Dashtipour, A. Patel, T. Gross, R. Kazerooni, C. Gallagher, F. Xiaojin, S. Yunan, G. Lei, W. Xinhua, D. Hollander. Efficacy and Safety of DaxibotulinumtoxinA-lanm for Injection in Adults with Cervical Dystonia: Pooled Global Analysis of ASPEN-1 and ASPEN-1-CN [abstract]. Mov Disord. 2024; 39 (suppl 1). https://www.mdsabstracts.org/abstract/efficacy-and-safety-of-daxibotulinumtoxina-lanm-for-injection-in-adults-with-cervical-dystonia-pooled-global-analysis-of-aspen-1-and-aspen-1-cn/. Accessed June 15, 2025.« Back to 2024 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/efficacy-and-safety-of-daxibotulinumtoxina-lanm-for-injection-in-adults-with-cervical-dystonia-pooled-global-analysis-of-aspen-1-and-aspen-1-cn/