ATX-101

KYTHERA’s lead program, ATX-101 is a first-in-class injectable drug being studied for the reduction of localized fat. ATX-101 is a proprietary formulation of deoxycholate, a well-studied endogenous compound that is present in the body to promote the natural breakdown of dietary fat. ATX-101 reduces subcutaneous fat through localized fat cell membrane disruption followed by elimination from the treated area. This mechanism may enable a non-surgical alternative for the reduction of subcutaneous fat.

KYTHERA is conducting a robust pre-clinical and clinical development program with ATX-101 in the reduction of submental fat, or fat under the chin. In two randomized, double-blind, placebo-controlled, dose-ranging international studies, ATX-101 was found to be well-tolerated and demonstrated statistically significant efficacy compared to placebo in the reduction of submental fat as assessed by physicians and patients. Additionally, in a third multicenter randomized, double-blind, placebo-controlled trial conducted in the United States, ATX-101 was found to be well-tolerated and resulted in statistically significant clinical benefit from ATX-101 in the reduction of submental fat as assessed by three efficacy measures: a validated clinician scale, patient reported outcome (PRO) scale and Magnetic Resonance Imaging (MRI) measuring SMF volume and thickness. KYTHERA has also completed four Phase I (pharmacokinetic, histology, lipid and tolerability) clinical safety studies in the United States.

Potential uses

In the rapidly growing market of minimally-invasive, non-surgical facial rejuvenation, the reduction of lower facial fat to restore and reshape the jawline remains one of the largest unmet needs. While the initial application being studied for ATX-101 is the reduction of submental fat, over time KYTHERA intends to further develop ATX-101 in the reduction of other small, localized fat characteristic of lipodystrophic (undesirable presence or abnormal accumulation of localized fat) disorders.

Publications

Cytolytic effects of deoxycholate are attenuated by interactions with proteins
Knopp, K. et al.
American Academy of Dermatology, 2009

Removal of subcutaneous fat by deoxycholate in mice expressing luciferase selectivity in adipocytes
Thuangtong, R. et al.
American Academy of Dermatology, 2009

Phosphatidylcholine inhibits the adipolytic activity of deoxycholate in vitro and in vivo
Bentow, J. et al.
5-Continent Congress Lasers and Aesthetic Medicine, 2009