DaxibotulinumtoxinA for Injection for treatment for cervical dystonia met the primary and all secondary endpoints in the phase 3 ASPEN-1 study, according to topline results released today by Revance Therapeutics.
This agent has the potential to reduce the frequency of cervical dystonia injections by up to 50% annually, they note in a news release.
ASPEN-1 was a randomized, double-blind, placebo-controlled, parallel-group study of 301 patients with cervical dystonia from 60 sites in the United States, Canada, and Europe.
Participants were randomly allocated to receive a single injection of either 125 units or 250 units of daxibotulinumtoxinA or placebo and were followed for up to 36 weeks.
Treatment with daxibotulinumtoxinA led to clinically meaningful improvement in the signs and symptoms of cervical dystonia at weeks 4 and 6, on average, the company said.
Compared with placebo, patients treated with either 125 or 250 units had a statistically significant greater improvement from baseline on the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-Total score (12.7 and 10.9, respectively, vs 4.3; P < .0001 and P = .0006).
The median duration of effect was 24.0 weeks for the lower dose and 20.3 weeks for the higher dose, based on the median time to loss of 80% of peak treatment effect.
At weeks 4 and 6, results on the Clinical Global Impression of Change (CGIC) and Patients' Global Impression of Change (PGIC) scales, two secondary endpoints, were consistent and showed a statistically significant improvement greater than placebo (P < .001), the company said.
"I was delighted to see both the degree and duration of relief that DaxibotulinumtoxinA for Injection provided trial subjects in ASPEN-1," trial investigator Joseph Jankovic, MD, professor of neurology, Baylor College of Medicine, Houston, Texas, said in the release.
"Currently, most patients with cervical dystonia visit their physician 3 to 4 times a year for injections, which places a heavy burden on patients' time and schedule. Often, the treatment effect wears off between injections, significantly impacting the quality of their work and personal lives," Jankovic explained.
"If a treatment could offer longer duration of effect, thus requiring fewer trips each year for reinjection, I imagine patients would find this quite beneficial," he added.
DaxibotulinumtoxinA appeared to be generally safe and well tolerated through 36 weeks, with no serious treatment-related adverse events and no dose-dependent increase in adverse events.
Treatment-related adverse events were generally transient and mild to moderate in severity, with one case of neck pain reported as severe, which resolved 2 days after onset. The most common treatment-related adverse events were injection-site pain/erythema and headache.
The incidence of dysphagia and muscle weakness, considered adverse events of particular interest with botulinum toxin treatments for cervical dystonia, was "encouragingly low," the company said.
Dysphagia occurred in 1.6% of patients given the lower dose and 3.9% given the higher dose; muscular weakness occurred in 4.7% and 2.3%, respectively.
The company expects results from the companion ASPEN-OLS phase 3, open-label, long-term safety trial, which enrolled 354 patients, in 2021.
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Cite this: Positive Topline Results for New Cervical Dystonia Treatment - Medscape - Oct 14, 2020.
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