CALABASAS, Calif., Oct. 7, 2013 (GLOBE NEWSWIRE) -- KYTHERA Biopharmaceuticals, Inc. (Nasdaq:KYTH) today announced additional patient satisfaction and efficacy data from REFINE-1 and REFINE-2, two pivotal Phase III clinical trials of ATX-101 for the reduction of submental fat, which commonly presents as a double chin. The new data demonstrates that the majority of ATX-101 subjects reported satisfaction with their treatment as well as improvements on visual and psychological impacts of their "chin fat." Additionally, the vast majority of subjects had a reduction in submental fat as assessed independently by clinicians and patients, and most adverse events were transient, local to the treatment area, and mild to moderate in severity.
KYTHERA's recent REFINE-1 and REFINE-2 top line data announcement showed that both pivotal trials met all their pre-defined primary and secondary endpoints and demonstrated high statistical significance across these measures (p < 0.001). The patient satisfaction data presented at ASDS supplement the top line data and demonstrate that 88.9 percent of REFINE-1 and 84.2 percent of REFINE-2 ATX-101 treated subjects reported satisfaction with treatment received in the trial, compared to 37.7 percent and 43.6 percent of placebo subjects, respectively (p < 0.001).
Further analysis of REFINE-1 presented at ASDS demonstrates that ATX-101 resulted in improvement in the visual and psychological impact of chin fat, using the 11-point Patient-Reported Submental Fat Impact Scale (PR-SMFIS). Statistical significance was achieved for each of the six individual components of the PR-SMFIS (all p < 0.001, ATX-101 vs. placebo).
ATX-101 treated subjects from REFINE-1 were:
- happier with the appearance of their chin fat (mean change from baseline in PR-SMFIS of 5.4 vs. 1.8 for ATX-101 vs. placebo, respectively),
- less bothered by the appearance of their chin fat (mean change from baseline in PR-SMFIS of 3.8 vs. 1.2 for ATX-101 vs. placebo, respectively),
- less embarrassed by the appearance of their chin fat (mean change from baseline in PR-SMFIS of 3.4 vs. 1.2 for ATX-101 vs. placebo, respectively),
- less self-conscious about the appearance of their chin fat (mean change from baseline in PR-SMFIS of 3.4 vs. 1.3 for ATX-101 vs. placebo, respectively),
and perceived themselves as:
- less overweight (mean change from baseline in PR-SMFIS of 3.0 vs. 0.6 for ATX-101 vs. placebo, respectively), and
- looking younger (mean change from baseline in PR-SMFIS of 2.9 vs. 0.7, for ATX-101 vs. placebo, respectively).
"As a physician, I see many patients who are dissatisfied with their appearance due to the presence of a double chin," said Jean D. Carruthers, M.D., FRCSC, FRC, REFINE-1 investigator and Clinical Professor, Department of Ophthalmology, University of British Columbia, Vancouver. "I am excited that this data demonstrated treatment with ATX-101 not only delivers meaningful results, but also leads to changes in patient self-perception. If approved, ATX-101 could offer physicians a much needed non-surgical option to both contour the area under the chin and deliver high patient satisfaction."
Additional efficacy results presented at ASDS demonstrate higher rates of clinical improvement on the independent clinician and patient ratings of submental fat than were observed for the trial's primary endpoints, which were based on a composite of clinician and patient ratings. Clinician ratings demonstrated that 79.5 percent of REFINE-1 and 78.3 percent of REFINE-2 subjects showed an improvement of at least one grade using the validated Clinician-Reported Submental Fat Rating Scale (CR-SMFRS) assessed 12 weeks after the last treatment vs. 36.4 and 34.6 percent, respectively, for placebo (both p < 0.001). Similar results were seen for patient ratings, where 82.4 percent of REFINE-1 and 78.9 percent of REFINE-2 subjects showed at least a one grade improvement on the validated Patient-Reported Submental Fat Rating Scale (PR-SMFRS) at 12 weeks after last treatment vs. 38.4 and 38.1 percent, respectively, for placebo (both p < 0.001). The composite endpoint derived from these independent clinician and patient ratings, which served as a primary endpoint of the study, required that subjects demonstrate at least a one grade improvement on both the clinician and patient ratings in order to be classified as a responder. This more stringent composite endpoint results in only modestly lower one-grade response rates for ATX-101 treated subjects (70.3 percent for REFINE-1 and 66.9 percent for REFINE-2, as previously reported), but results in substantially lower placebo response rates (18.7 percent for REFINE-1 and 22.4 percent for REFINE-2).
"We are pleased that these analyses further support the efficacy of ATX-101 for the reduction of submental fat," said Frederick Beddingfield, III, M.D., Ph.D., Chief Medical Officer, KYTHERA Biopharmaceuticals, Inc. "These new findings, along with the previously released data, reinforce that ATX-101 effectively reduces fat under the chin, improving the appearance of the submental profile. Importantly, this results in high patient satisfaction, which is critical in the field of aesthetic medicine."
Additional data on the safety of ATX-101 was also presented at ASDS. The REFINE-1 and REFINE-2 trials demonstrated ATX-101 was well tolerated by subjects and the most common adverse events, which included swelling, pain, bruising, numbness and redness, were predominantly transient, local to the treatment area, and mild to moderate in severity. In REFINE-1, 98.5 percent and in REFINE-2, 98.9 percent of adverse events were reported as mild or moderate. Additionally, there were no treatment-related serious adverse events in either study and less than 4 percent of subjects discontinued the studies due to adverse events.
About REFINE-1 and REFINE-2
REFINE-1 and REFINE-2 (studies ATX-101-11-22 and ATX-101-11-23) are two identical multicenter, double-blind, randomized, placebo-controlled Phase III pivotal trials conducted to compare the efficacy and safety of a 2 mg/cm2 dose of ATX-101 versus placebo for the reduction of submental fat. Patients received up to six treatments approximately 28 days apart. REFINE-1 and REFINE-2 were conducted at 70 centers in the U.S. and Canada and enrolled 506 and 516 subjects, respectively, with moderate to severe submental fat at baseline as determined through both validated clinician- and patient-rating scales. The two primary efficacy endpoints of the trial were: 1) proportion of patients with a simultaneous improvement of at least one grade from baseline on the Clinician-Reported Submental Fat Rating Scale (CR-SMFRS) and the Patient-Reported Submental Fat Rating Scale (PR-SMFRS); and 2) proportion of patients with a simultaneous improvement of at least two grades from baseline on the CR-SMFRS and the PR-SMFRS. Secondary endpoints included the reduction in volume of the submental region as measured using magnetic resonance imaging (MRI), and improvement in the appearance-related impacts of submental fat, assessed using the Patient-Reported Submental Fat Impact Scale (PR-SMFIS).
Top line results of REFINE-1 and REFINE-2 can be found here.
* ATX-101 Presentation Details
Presentations at the American Society for Dermatologic Surgery (ASDS) Annual Meeting in Chicago, IL, were:
Thursday, October 3rd "Neck Rejuvenation: Do We Have the Answer?" Rhoda S. Narins, M.D., PC, FAAD, REFINE-2 Investigator Saturday, October 5th "REFINE-1 and REFINE-2: Pivotal U.S. and Canadian Phase III Studies with ATX-101, an Injectable Drug for the Reduction of Submental Fat" Jean Carruthers, M.D., FRCSC, FRC, REFINE-1 Investigator
ATX-101 is currently in late stage clinical trials for the reduction of submental fat, which commonly presents as a double chin. It is a proprietary formulation of a purified synthetic version of deoxycholic acid, a naturally occurring molecule in the body that aids in the breakdown of dietary fat.1
The submental region, or the area under the chin, is visually important to patients, but is often undertreated. ATX-101 treatment contours the area under the chin by destroying fat cells while leaving surrounding tissue largely unaffected.
For the past six years, ATX-101 has been the focus of a global clinical development program that has enrolled more than 2,500 patients worldwide, of which more than 1,500 have been treated with ATX-101. ATX-101 has the potential to be a first-in-class submental contouring injectable drug if approved.
Bayer HealthCare holds development and commercialization rights to ATX-101 outside of the U.S. and Canada.
About KYTHERA Biopharmaceuticals, Inc.
KYTHERA Biopharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel prescription products for the aesthetic medicine market. KYTHERA's product candidate, ATX-101, is currently in late stage clinical trials for the reduction of submental fat, which commonly presents as a double chin, and is a potential first-in-class submental contouring injectable drug. KYTHERA also maintains an active research interest in hair and fat biology, pigmentation modulation and facial contouring. Find more information at http://www.kytherabiopharma.com.
To the extent that statements contained in this press release are not descriptions of historical facts regarding KYTHERA, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including that the topline results of the REFINE-1 and REFINE-2 trials for ATX-101 are indicative of the final results of the respective studies, the potential for ATX-101 to enter the market as the only FDA-approved drug for the reduction of submental fat and the commercial potential of ATX-101. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance, or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, the regulatory approval process, the development progress of our collaborative partners, our substantial dependence on ATX-101 and other matters that could affect the availability or commercial potential of our drug candidate. KYTHERA undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of the Company in general, see KYTHERA's reports filed with the Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended June 30, 2013 and our Annual Report on Form 10-K for the year ended December 31, 2012.
1Stryer L.Biosynthesis of membrane lipids and steroids. In: Biochemistry. New York, NY: WH Freeman and Co; 1995:691-707
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