DaxibotulinumtoxinA Has a 24-Week Effect for Treatment of Cervical Dystonia
Phase 2 clinical trial study data for daxibotulinumtoxinA (RT002; Revance, Newark, CA) for treatment of cervical dystonia (CD) were published in the journal Movement Disorders. The data show that daxibotulinumtoxinA provides clinically significant improvement in CD signs and symptoms as measured by the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS). DaxibotulinumtoxinA is an investigational compound comprised of botunlinumtoxinA molecule and a proprietary peptide designed as a long-lasting injectable neuromodulator.
Patients received 100 to 240 units (n = 21) or 300 to 450 units (n = 16) of daxibotulinumtoxinA. At 4 weeks after injection both groups had a significant reduction in TWSTRS score of 37% and 39% respectively; 94% of subjects had a ³20% reduction in their TWSTRS score at week 6 and 68% maintained this through week 24. The median duration of effect (defined as ³20% of effect seen 4 weeks after injection) was 25.3 weeks, over twice as long as the period of effect for currently available formulations of botunlinumtoxinA.
The most common treatment-related adverse events were dysphagia (14%) and injection site erythema (8%). These were generally characterized as mild to moderate and were transient, with one case of neck pain reported as severe (Day 10 onset; duration 2 days).
Trial investigator Joseph Jankovic, MD, Professor of Neurology and Distinguished Chair in Movement Disorders, Baylor College of Medicine said the data, “. . .indicated that this new formulation of botulinum toxin serotype A may significantly improve symptoms of cervical dystonia and have an impressive duration of benefit of 24 weeks, which is almost twice as long as the toxins currently available.”
Revance President and Chief Executive Officer Dan Browne noted that the company “. . .looks forward to additional data from the upcoming ASPEN Phase 3 trials to further characterize the safety, efficacy and duration of effect of RT002 in this important orphan disease indication.” That phase 3 trial is expected to begin in the second quarter of 2018 as a single pivotal trial with
approximately 300 patients; enrollment information is available at www.clinicaltrials.gov.