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REFINE-1, a Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial With ATX-101, an Injectable Drug for Submental Fat Reduction

Jones, Derek H. MD*; Carruthers, Jean MD, FRCSC, FRC (OPHTH); Joseph, John H. MD; Callender, Valerie D. MD§; Walker, Patricia MD, PhD; Lee, Daniel R. MS; Subramanian, Meenakshi PhD; Lizzul, Paul F. MD, PhD, MPH, MBA, FAAD; Gross, Todd M. PhD; Beddingfield, Frederick C. III MD, PhD*,‖

doi: 10.1097/DSS.0000000000000578
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BACKGROUND ATX-101, an injectable form of deoxycholic acid, is approved in the United States and Canada for submental fat (SMF) reduction.

OBJECTIVE To report results of REFINE-1, a randomized, double-blind, placebo-controlled, Phase 3 trial investigating the efficacy and safety of ATX-101.

METHODS Subjects dissatisfied with their moderate or severe SMF received ATX-101 (2 mg/cm2) or placebo. Coprimary outcome measures were composite ≥1-grade and ≥2-grade improvements in clinician-assessed and subject-assessed SMF severity using validated scales at 12 weeks after last treatment. Magnetic resonance imaging (MRI) provided an objective measure of submental volume reduction. Patient-reported outcomes were assessed.

RESULTS Among 256 ATX-101–treated and 250 placebo-treated subjects, a ≥1-grade composite response was achieved in 70.0% and 18.6%, and a ≥2-grade composite response in 13.4% and 0%, respectively (p < .001 for both). The proportion of MRI responders was more than 8 times higher with ATX-101 than placebo (46.3% vs 5.3%; p < .001). ATX-101–treated subjects reported improvement in the psychological impact of SMF and satisfaction with treatment (p < .001 for all assessments vs placebo). Of note, 55% and 75% of ATX-101–treated subjects reported 1-grade improvement in clinician-assessed SMF after 2 and 4 treatments, respectively. Adverse events (primarily localized to the injection site) were mostly mild or moderate, and transient. Marginal mandibular nerve paresis reported in 4.3% of ATX-101–treated subjects (1.0% of all ATX-101 treatment sessions) was mostly mild, transient, and resolved without sequelae.

CONCLUSION ATX-101 is a safe and efficacious, first-in-class, injectable drug for SMF reduction.

*Dermatology Division, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California;

Department of Ophthalmology and Visual Sciences, University of British Columbia, Vancouver, Canada;

Department of Head and Neck Surgery, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, California;

§Department ofDermatology, Howard University College of Medicine, Washington, DC;

Kythera Biopharmaceuticals, Inc., Westlake Village, California (now an affiliate of Allergan plc, Irvine, California);

Evidence Scientific Solutions, Philadelphia, Pennsylvania

Address correspondence and reprint requests to: Derek H. Jones, MD, 9201 W. Sunset Boulevard Suite 602, Los Angeles, CA 90069, or e-mail: derekjonesmd@gmail.com

Supplemental digital content is available for this article. Direct URL citations appear in the printed text and are provided in the full text and PDF versions of this article on the journal's Web site (www.dermatologicsurgery.org).

D. H. Jones, J. Carruthers, J. H. Joseph, and V. D. Callender all received honoraria, payment, or other compensation for work on this trial. J. H. Joseph purchased stock after completion of the trial. P. Walker, D. R. Lee, P. F. Lizzul, T. M. Gross, and F. C. Beddingfield were employees of Kythera Biopharmaceuticals, Inc. (now an affiliate of Allergan plc), during the execution of this trial. M. Subramanian is an employee of Evidence Scientific Solutions, Philadelphia, PA and provided medical writing assistance that was supported by Kythera Biopharmaceuticals, Inc.

© 2016 by the American Society for Dermatologic Surgery, Inc. Published by Wolters Kluwer Health, Inc. All rights reserved.
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