KYTHERA ATX-101-10-16 trial for submental fat meets primary endpoints

KYTHERA Biopharmaceuticals, Inc. ("KYTHERA") today announced the presentation of initial trial results from Study ATX-101-10-16, the first of two pivotal European Phase III clinical trials with ATX-101, a facial injectable drug for the reduction of unwanted fat under the chin, or submental fat. V. Leroy Young, MD, FACS, presented the initial results at the American Society for Aesthetic Plastic Surgery (ASAPS) 45^th Annual Aesthetic Meeting in Vancouver, British Columbia, on May 4, 2012.

“As a physician, I'm excited about the potential for ATX-101, if approved, to expand our facial injectable treatment offerings to patients looking for facial aesthetic enhancement.”

The ATX-101-10-16 trial met its pre-specified primary endpoints based on clinician and patient assessments. At the 2 mg/cm² dose, ATX-101 resulted in a statistically significant reduction of submental fat, relative to placebo, as measured using a 5-point Clinician-Reported Submental Fat Rating Scale (CR-SMFRS) (mean of 0.90 vs. 0.22; p<0.001, week 24). Similarly, ATX-101 (2 mg/cm²) resulted in a statistically significant percentage of subjects, relative to placebo, achieving a pre-defined categorical change using a 7-point Subject Self Rating Scale (SSRS) (66.1 vs. 28.7; p<0.001, week 24).

"The positive results from this pivotal European trial demonstrate the potential of ATX-101 to be a well tolerated and effective treatment for patients who want to refine their appearance by removing fat below the chin," said Dr. Young, a board certified plastic surgeon in private practice in St. Louis, Missouri, who is currently an investigator in the ongoing U.S. pivotal Phase III REFINE trials. "As a physician, I'm excited about the potential for ATX-101, if approved, to expand our facial injectable treatment offerings to patients looking for facial aesthetic enhancement."

Importantly, Patient Reported Outcomes (PRO) specific to treatment with ATX-101 (2 mg/cm²) also resulted in statistically significant differences, relative to placebo, in the:

• reduction of submental fat using a 5-point Patient-Reported Submental Fat Rating Scale (PR-SMFRS) (mean of 0.98 vs. 0.32; p<0.001, week 24); and
• percentage of subjects expressing satisfaction with treatment using the Global Post-Treatment Satisfaction Scale (86% vs. 45%, p<0.001, week 24).

Additional Patient Reported Outcomes included a Patient-Reported Submental Fat Impact Scale (PR-SMFIS), which was developed to measure the visual and psychological impacts of submental fat, including whether patients perceived themselves to be happier, less bothered, less self-conscious, less embarrassed, younger or less overweight after treatment with ATX-101. Statistically significant differences were achieved, at 2 mg/cm² relative to placebo, for all PR-SMFIS measures (p<0.001, week 24).

ATX-101 (2 mg/cm²) also resulted in statistically significant reduction of submental fat thickness using calipers as an objective measure (p<0.001, week 24).

"These positive Phase III data are consistent with the encouraging results from previous trials, all of which have demonstrated that ATX-101 is well tolerated and provides meaningful aesthetic improvements according to multiple clinician, patient and objective measures," said Patricia S. Walker, M.D., Ph.D., KYTHERA's Chief Medical Officer. "We have taken a rigorous scientific approach in developing and evaluating ATX-101 for the aesthetic reduction of submental fat which includes a second European Phase III trial, Study 17, and our two ongoing multi-center pivotal U.S. and Canadian Phase III trials, REFINE-1 and REFINE-2."

Adverse events were mostly mild to moderate, and were transient. The most common adverse events were pain, swelling, numbness, bruising and induration. These adverse events were limited to the injection site; most were temporally associated with treatment. No systemic treatment-related adverse events were reported.

Results from the second pivotal European Phase III clinical trial, Study 17, will be presented at the 8^th Annual Vegas Cosmetic Surgery International Multispecialty Symposium in early June.