ATX-101

KYTHERA’s lead program, ATX-101, is a first-in-class injectable adipolytic agent intended for the reduction of small volumes of localized fat. The active ingredient in ATX-101 is a well-studied endogenous molecule that preferentially lyses fat cells while leaving surrounding tissue largely unaffected. The result is a minimally-invasive procedure done with little or no anesthetic that yields consistent and meaningful clinical results.

ATX-101 works by preferential cytolytic activity against adipocytes (adipolysis). In vitro and in vivo experiments indicate that while fat tissue is removed by ATX-101 treatment, non-fat tissues (e.g., skin, muscle) are unaffected.

KYTHERA has completed a robust pre-clinical toxicology program with ATX-101 and has regulatory investigational drug filings open in several countries worldwide. ATX-101 clinical research is focused on the reduction of localized fat deposits, including submental, or under the chin, fat. In two randomized, double-blind, placebo-controlled, dose-ranging international Phase 2 studies, ATX-101 was found to be safe, well-tolerated and demonstrated statistically significant efficacy compared to placebo in the reduction of submental fat as assessed by both physicians and patients. KYTHERA has also completed two Phase 1 clinical safety studies in the United States, which examined the pharmacokinetic and histological effects of ATX-101.

Potential uses

In the rapidly growing market of minimally-invasive, non-surgical facial rejuvenation, the reduction of facial fat deposits to restore and reshape the jawline remains one of the largest unmet needs. ATX-101 is currently being studied for the reduction of submental fat, a localized subcutaneous fat deposit located immediately beneath the chin and jawline. While the initial application is the treatment of submental fat, over time KYTHERA intends to further develop ATX-101 in the reduction of other small, localized fat deposits 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