Revance Therapeutics Initiates Phase 2 Clinical Study of Its Unique Botulinum Toxin Type A for Injection to Treat Cervical Dystonia
NEWARK, Calif., Sept. 29, 2015 (GLOBE NEWSWIRE) -- Revance Therapeutics, Inc. (NASDAQ: RVNC), a specialty biopharmaceutical company developing botulinum toxin products for use in aesthetic and therapeutic indications, announced today the initiation of patient dosing in a Phase 2 dose-escalating clinical study of RT002 investigational drug product candidate to treat cervical dystonia, a neurological muscle movement disorder. The Phase 2 study will evaluate safety, preliminary efficacy, and duration of effect of RT002 for injection in patients with moderate-to-severe isolated cervical dystonia symptoms of the neck. The company plans to release interim results in 2016.
"Cervical dystonia is a debilitating condition characterized by involuntary muscle contractions in the neck. This painful malady is the first therapeutic indication we're pursuing for our unique injectable neurotoxin," said Dan Browne, President and Chief Executive Officer at Revance. "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."
Phase 2 Study Design
Revance's Phase 2 trial is an open-label, sequential, dose-escalating study to evaluate the safety, preliminary efficacy and duration of effect of a single treatment of RT002 (RTT150 (Botulinum Toxin Type A) for Injection) for isolated cervical dystonia. Approximately 36 patients with at least moderate cervical dystonia are expected to be enrolled at multiple sites in the United States. 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 primary efficacy endpoint of the study is an improvement in dystonia symptoms as measured by change (reduction) from baseline in TWSTRS (Toronto Western Spasmodic Torticollis Rating Scale) total score at four weeks. TWSTRS is a composite scale that covers different features of the cervical dystonia condition. The first part of the scale is based on the physical findings and severity of dystonia, the second part rates the patient's perceived level of disability, and the third part rates the pain. Numerous secondary efficacy endpoints will be measured, including duration of effect and patient-rated quality of life as measured by change from baseline in CDIP (Cervical Dystonia Impact Profile).
All patients will be followed out to nine weeks post-injection. Patients with sustained improvement after this initial period of nine weeks will continue to be assessed for duration of efficacy and other measures until they return to baseline or for up to a total of 24 weeks after treatment.
About Cervical Dystonia
According to the Dystonia Medical Research Foundation, whose mission it is to advance research, promote awareness and education, and support the needs affected individuals, cervical dystonia is a painful condition in which the neck muscles contract involuntarily, causing abnormal movements and awkward posture of the head and neck. The movements may be sustained (tonic), jerky (clonic), or a combination. Cervical dystonia (also referred to as spasmodic torticollis) may be primary (meaning that it is the only apparent neurological disorder, with or without a family history) or may be brought about by secondary causes, such as physical trauma. It can result in considerable pain and discomfort.
Treatments for cervical dystonia include oral medications, botulinum toxin injections, surgery, and complementary therapies. Botulinum toxin can help block the communication between the nerve and the muscle and may alleviate abnormal movements and postures. Current botulinum toxin treatments for cervical dystonia have a duration of effect of approximately three months. Cervical dystonia can occur at any age, although most individuals first experience symptoms in middle age. It affects several hundred thousand adults and children in the U.S. alone. Revance estimates the global market for treating muscle movement disorders with botulinum toxins, including cervical dystonia, was approximately $900 million in 2014.
About RT002 for Injection
RT002 for Injection is a novel investigational drug product candidate that combines Revance's proprietary, pure 150kD botulinum toxin type A molecule, without any accessory proteins or animal derived components, with a patented TransMTS® peptide technology. RT002 is designed to offer targeted delivery to the intended treatment sites, while reducing its spread beyond the site of local injection. RT002 is in clinical development for the treatment of glabellar (frown) lines and for cervical dystonia, and has the potential to address additional therapeutic indications in movement disorders, pain management, urology, ophthalmology, and other potential uses where more targeted delivery is required or longer duration is desired.
In January 2015, Revance announced it had initiated its BELMONT Phase 2 active comparator trial RT002 for injection in the treatment of glabellar (frown) lines. This study is fully enrolled and will evaluate the safety, efficacy, and duration of effect of three doses of RT002, the labeled dose of the current market leader, BOTOX* Cosmetic, and a placebo control. Study results are expected in the fourth quarter of 2015.
In a previous Phase 1/2 open-label study of RT002 for the treatment of moderate to severe glabellar lines, RT002 met its efficacy endpoint, demonstrating high response rates across all doses. Further, RT002 was well-tolerated with no safety concerns and minimal adverse events, which included headache and itching or burning at the injection site; all events were considered mild or moderate and transient. There was no evidence of spread beyond the treatment site. In the final cohort, the only one where duration of effect was measured, RT002 achieved a median duration of 29.4 weeks, or 7 months, based on both investigator and patient assessments.
About Revance Therapeutics, Inc.
Revance is a specialty biopharmaceutical company focused on the development, manufacturing and commercialization of novel botulinum toxin products for multiple aesthetic and therapeutic indications. The company is leveraging its proprietary portfolio of botulinum toxin compounds combined with its patented TransMTS® peptide delivery system to address unmet needs in the large and growing neurotoxin markets. Revance's proprietary TransMTS technology enables delivery of botulinum toxin A through two novel drug product candidates, a needle-free topical form and an injectable form that may localize the drug to the site of injection resulting in a targeted and potentially long-lasting delivery. Revance is pursuing clinical development for drug product candidates RT001 topical gel and RT002 for injection in a broad spectrum of aesthetic and therapeutic indications. The company holds worldwide rights for all indications of RT001, RT002 and the TransMTS technology platform. More information on Revance Therapeutics can be found at www.revance.com.
"Revance Therapeutics", TransMTS® and the Revance logo are registered trademarks of Revance Therapeutics, Inc.
*BOTOX ® is a registered trademark of Allergan, Inc.
This press release contains forward-looking statements, including statements about our RT002 investigational drug product candidate, including but not limited to statements about our business strategy, goals and market for our anticipated products, plans and prospects and statements about potential benefits of our drug product candidates and our technologies.
Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from our expectations. These risks and uncertainties include, but are not limited to: the outcome, cost and timing of our product development activities and clinical trials; the uncertain clinical development process, including the risk that clinical trials may not have an effective design or generate positive results; our ability to obtain and maintain regulatory approval of our drug product candidates; our ability to obtain funding for our operations; our plans to research, develop and commercialize our drug product candidates; our ability to achieve market acceptance of our drug product candidates; unanticipated costs or delays in research, development and commercialization efforts; the applicability of clinical study results to actual outcomes; the size and growth potential of the markets for our drug product candidates; our ability to successfully commercialize our drug product candidates and the timing of commercialization activities; the rate and degree of market acceptance of our drug product candidates; our ability to develop sales and marketing capabilities; the accuracy of our estimates regarding expenses, future revenues, capital requirements and needs for financing; our ability to continue obtaining and maintaining intellectual property protection for our drug product candidates; and other risks. Detailed information regarding factors that may cause actual results to differ materially from the results expressed or implied by statements in this press release may be found in Revance's periodic filings with the Securities and Exchange Commission (the "SEC"), including factors described in the section entitled described in the "Risk Factors" section of our quarterly report on Form 10-Q filed August 7, 2015. These forward-looking statements speak only as of the date hereof. Revance disclaims any obligation to update these forward-looking statements.