ATX-101 for reduction of submental fat: A phase III randomized controlled trial

• Published in: Journal of the American Academy of Dermatology Vol. 75; no. 4; pp. 788 - 797.e7
• Main Authors: Humphrey, Shannon, MD, Sykes, Jonathan, MD, Kantor, Jonathan, MD, MSCE, MA, Bertucci, Vince, MD, Walker, Patricia, MD, PhD, Lee, Daniel R., MS, Lizzul, Paul F., MD, PhD, MPH, MBA, Gross, Todd M., PhD, Beddingfield, Frederick C., MD, PhD
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01-10-2016
• Subjects: Adipocytes - drug effects; Adipocytes - metabolism; Adult; aesthetics; Aged; ATX-101; Canada; contouring; Cosmetic Techniques; deoxycholic acid; Deoxycholic Acid - pharmacology; Dermatology; Dose-Response Relationship, Drug; Double-Blind Method; Drug Administration Schedule; efficacy; Esthetics; Humans; injectable; Injections, Subcutaneous; Magnetic Resonance Imaging - methods; Middle Aged; minimally invasive; nonsurgical; Patient Safety; Patient Satisfaction - statistics & numerical data; safety; Subcutaneous Fat - diagnostic imaging; Subcutaneous Fat - drug effects; submental fat; United States; nonsurgical; magnetic resonance imaging; Patient-Reported Submental Fat Impact Scale; AE; MRI; adverse event; deoxycholic acid; minimally invasive; aesthetics; SMF; efficacy; MMN; marginal mandibular nerve; injectable; ATX-101; safety; PR-SMFIS; submental fat; contouring
• ISSN: 0190-9622; 1097-6787
• DOI: 10.1016/j.jaad.2016.04.028

Background ATX-101, an injectable form of deoxycholic acid, causes adipocytolysis when injected subcutaneously into fat. Objective We sought to evaluate the efficacy and safety of ATX-101. Methods In this phase III trial (REFINE-2), adults dissatisfied with their moderate or severe submental fat (SMF) were randomized to ATX-101 or placebo. Coprimary end points, evaluated at 12 weeks after last treatment, were composite improvements of 1 or more grades and 2 or more grades in SMF observed on both the validated Clinician- and Patient-Reported SMF Rating Scales. Other end points included magnetic resonance imaging–based assessment of submental volume, assessment of psychological impact of SMF, and additional patient-reported outcomes. Results Among those treated with ATX-101 or placebo (n = 258/treatment group), 66.5% versus 22.2%, respectively, achieved a composite improvement of 1 or more grades (Mantel-Haenszel risk ratio 2.98; 95% confidence interval 2.31-3.85) and 18.6% versus 3.0% achieved a composite improvement of 2 or more grades in SMF (Mantel-Haenszel risk ratio 6.27; 95% confidence interval 2.91-13.52; P  < .001 for both). Those treated with ATX-101 were more likely to achieve submental volume reduction confirmed by magnetic resonance imaging, greater reduction in psychological impact of SMF, and satisfaction with treatment ( P  < .001 for all). Overall, 85.7% of adverse events in the ATX-101 group and 76.9% in the placebo group were localized to the injection site. Limitations Follow-up was limited to 44 weeks. Conclusion ATX-101 is an alternative treatment for SMF reduction.