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DaxibotulinumtoxinA for Injection (DAXI) 125U and 250U, a novel botulinum toxin type A product with a proprietary peptide excipient, has shown to be safe and efficacious in reducing signs and symptoms associated with cervical dystonia in adults, according to study results presented at the International Congress of Parkinson’s Disease and Movement Disorders (MDS) Virtual Congress 2021, held from September 17 to 22, 2021.


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A previous phase 2 study found DAXI was beneficial in terms of efficacy and duration of effect in an adult population with cervical dystonia.

The authors of the current sought to evaluate the effect of duration and the safety profile of DAXI by comparing 2 doses of DAXI with placebo in patients with cervical dystonia.

In a phase 3, double-blind, placebo-controlled ASPEN-1 trial, the researchers evaluated the efficacy and safety of 2 doses of DAXI compared with placebo in the treatment of cervical dystonia. The study was conducted at 60 sites in the US, Canada, and European Union.

Adult patients with moderate to severe cervical dystonia were followed up with for up to 36 weeks after a single treatment. The Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) was used to assess the participants.

The average change from baseline in TWSTRS total score at weeks 4 and 6 was the primary efficacy endpoint; safety was assessed at all study visits.

A total of 301 participants (mean age, 57.7 years; 64.8% women; 95.3% White) were randomly assigned to receive placebo (n=46), DAXI 125U (n=125), or DAXI 250U (n=130). Mean baseline TWSTRS total score was 43.3.

Mean improvement from baseline in TWSTRS total score at the primary timepoint was 4.3±1.8 for placebo, 12.7±1.3 for DAXI 125U (P <.0001 vs placebo), and 10.9±1.2 for DAXI 250U (P =.0006 vs placebo). No statistical difference in TWSTRS total score reduction was observed between the DAXI dose groups.

TWSTRS severity, disability, and pain subscales demonstrated comparable improvement from baseline — 30% to 33% for DAXI 125U and 25% to 26% for DAXI 250U, compared with 11% to 12% for placebo. The Clinician Global Impression of Change (CGIC) and Patient Global Impression of Change (PGIC) showed improvement with DAXI regarding the primary endpoint (CGIC, 77%-78% for DAXI vs 46% for placebo; PGIC, 71%-73% for DAXI vs 41% for placebo).

The median duration of effect was 24.0 for DAXI 125U and 20.3 weeks for DAXI 250U, according to time to loss of 80% peak treatment benefit.

Treatment-related adverse events (TEAEs) were reported in 39.1% of participants who received placebo, 58.3% of those who received DAXI 125U, and 50% of those who received DAXI 250U. TEAEs included injection site pain, headache, injection site erythema, muscular weakness, and musculoskeletal pain. Dysphagia was reported in 1.6% of those who received DAXI 125U and 3.9% of those who received DAXI 250U.

“DAXI appeared to be generally safe and well tolerated with adverse event rates similar to, or lower than, other botulinum toxin products for the treatment of [cervical dystonia],” the researchers concluded.

Disclosure: This research was supported by Revance Therapeutics Inc. Please see the original reference for a full list of authors’ disclosures.

Reference

Jankovic J, Comella C, Hauser RA, et al. A phase 3 trial evaluating the efficacy, duration of effect, and safety of DaxibotulinumtoxinA for Injection in the treatment of cervical dystonia. Presented at: MDS Virtual Congress 2021; September 17-22, 2021. Poster 100.