US-based biopharmaceutical firm Revance Therapeutics has started dosing in a Phase II dose-escalating clinical trial of an investigational drug product candidate, RT002, to treat cervical dystonia, a neurological muscle movement disorder.
The company is focused on developing botulinum toxin products for use in aesthetic and therapeutic indications.
The trial is designed to evaluate the safety, preliminary efficacy and duration of effect of a single treatment of RT002 (RTT150 (Botulinum Toxin Type A) for injection) in patients with moderate-to-severe isolated cervical dystonia symptoms of the neck.
RT002 for injection combines the company’s pure 150kD botulinum toxin type A molecule, without any accessory proteins or animal derived components, with a patented TransMTS peptide technology.
Interim results from the open-label, sequential, dose-escalating Phase II trial are expected to be released next year.
Revance president and chief executive officer Dan Browne said: "Cervical dystonia is a debilitating condition characterised by involuntary muscle contractions in the neck. This painful malady is the first therapeutic indication we’re pursuing for our unique injectable neurotoxin.
"RT002 employs our patented TransMTS delivery technology and is designed to offer a long-lasting, targeted delivery of botulinum toxin with reduced spread beyond the site of local injection.
"This could be extremely valuable in treating cervical dystonia, since current treatments have potential adverse events, such as muscle weakness and difficulty swallowing if the toxin migrates beyond the targeted neck muscles.
"We believe the prospects of an improved safety profile from less spread, along with a long-lasting duration of effect, might make RT002 an important future treatment option for a variety of involuntary muscle movement disorders."
Approximately 36 patients, with at least moderate cervical dystonia, will be enrolled at multiple sites in the US.
In the trial, there will be three treatment groups of approximately 12 patients each, who will be treated with one of three doses of RT002 for injection.
The trial’s primary efficacy endpoint is an improvement in dystonia symptoms as measured by change from baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) total score at four weeks.
Following completion of the trial, all patients will be followed out to nine weeks post-injection.
Currently, RT002 is in clinical development to treat glabellar (frown) lines and for cervical dystonia. It also has the potential to address additional therapeutic indications in movement disorders, pain management, urology, ophthalmology, as well as other potential uses where more targeted delivery is required or longer duration is desired.
Image: A person with medication induced dystonia. Photo: courtesy of James Heilman, MD.