KYTHERA
Biopharmaceuticals, Inc. KYTH today announced that its ATX-101
REFINE-1 and REFINE-2 Phase III trials met all primary and secondary
endpoints. These two pivotal Phase III trials were conducted in the U.S. and
Canada to compare the efficacy and safety of a 2 mg/cm^2 dose of ATX-101
versus placebo for the reduction of submental fat, which commonly presents as
a double chin. ATX-101 is a proprietary formulation of a purified synthetic
version of deoxycholic acid, a naturally occurring molecule in the body that
aids in the breakdown of dietary fat.^1 If approved, it will be a
first-in-class submental contouring injectable drug.
REFINE-1 and REFINE-2 are two identical multicenter, double-blind, randomized,
placebo-controlled trials that enrolled more than 1,000 subjects with moderate
to severe submental fat in 70 centers in the U.S. and Canada. Validated
clinician- and patient-rating scales were used to evaluate primary efficacy
endpoints, assessed 12 weeks after the last treatment.
Primary Endpoints
REFINE-1 (Study ATX-101-11-22)
* 70.3 percent of ATX-101 (2mg/cm^2) REFINE-1 subjects demonstrated a
simultaneous improvement of at least one grade from baseline on the
Clinician-Reported Submental Fat Rating Scale (CR-SMFRS) and
Patient-Reported Submental Fat Rating Scale (PR-SMFRS) vs. 18.7 percent in
placebo (p<0.001).
* 13.4 percent of ATX-101 (2mg/cm^2) REFINE-1 subjects demonstrated a
simultaneous improvement of at least two grades from baseline on the
Clinician-Reported Submental Fat Rating Scale (CR-SMFRS) and
Patient-Reported Submental Fat Rating Scale (PR-SMFRS) vs. 0 percent in
placebo (p<0.001).
REFINE-2 (Study ATX-101-11-23)
* 66.9 percent of ATX-101 (2mg/cm^2) REFINE-2 subjects demonstrated a
simultaneous improvement of at least one grade from baseline on the
Clinician-Reported Submental Fat Rating Scale (CR-SMFRS) and
Patient-Reported Submental Fat Rating Scale (PR-SMFRS) vs. 22.4 percent in
placebo (p<0.001).
* 18.7 percent of ATX-101 (2mg/cm^2) REFINE-2 subjects demonstrated a
simultaneous improvement of at least two grades from baseline on the
Clinician-Reported Submental Fat Rating Scale (CR-SMFRS) and
Patient-Reported Submental Fat Rating Scale (PR-SMFRS) vs. 3.2 percent in
placebo (p<0.001).
"The strong results from REFINE-1 and REFINE-2 are incredibly exciting and
consistent with the profile of ATX-101 based on previous data," said Frederick
Beddingfield, III, M.D., Ph.D., Chief Medical Officer, KYTHERA
Biopharmaceuticals, Inc. "We look forward to discussing submission plans with
U.S. and Canadian regulatory authorities in the near future. If approved,
ATX-101 will represent a significant innovation in the category of aesthetic
medicine."
Secondary Efficacy Endpoints
Additionally, an assessment of the trials' first secondary endpoint showed
46.6 percent of REFINE-1 and 40.0 percent of REFINE-2 patients achieved a
predefined, statistically significant reduction in the volume of their
submental region vs. 5.4 and 5.1 percent for placebo, respectively (both
p<0.001), as measured through magnetic resonance imaging (MRI).
Subjects also rated the visual and psychological impacts of submental fat
using the Patient-Reported Submental Fat Impact Scale (PR-SMFIS), which
assessed whether they perceived themselves to be happier, less bothered, less
self-conscious, less embarrassed, younger or less overweight after treatment
with ATX-101. Statistical significance was achieved for the change from
baseline in PR-SMFIS with 3.63 vs. 1.14, and 3.47 vs. 1.48, for ATX-101
(2mg/cm^2) and placebo for REFINE-1 and REFINE-2, respectively (p<0.001 for
both). Each individual component within the PR-SMFIS also demonstrated
statistical significance vs. placebo in both trials (p<0.001 for all PR-SMFIS
measures).
"The area under the chin is important to patients as it impacts overall facial
harmony, balance and attractiveness; however, an undesirable double chin is
often undertreated by aesthetic physicians as there is no proven non-surgical
option to effectively reduce submental fat," said Jean D. Carruthers, M.D.,
FRCSC, FRC, REFINE-1 investigator and Clinical Professor, Department of
Ophthalmology, University of British Columbia, Vancouver. "ATX-101 could
provide a solution that fulfills this unmet need and become an important
addition to the overall practice of aesthetics."
Safety
There were no treatment-related serious adverse events. The most common
adverse events, which were predominantly mild to moderate, were swelling,
pain, bruising, numbness and redness. Consistent with previous studies, these
adverse events were predominately transient and local to the treatment area.
Less than 4 percent of subjects discontinued the study due to adverse events.
Upcoming Scientific Presentation
Additional information from these studies will be reported at the upcoming
American Society for Dermatologic Surgery (ASDS) annual meeting, October 3-6
in Chicago.
Conference Call
KYTHERA will host an investor conference call and webcast to discuss the
results at 8:00 AM (Eastern Time) on September 17, 2013. Presenting from the
Company will be Keith Leonard, President and CEO, and Dr. Frederick
Beddingfield, III, Chief Medical Officer.
Individuals interested in participating in the call should dial (877) 344-3890
(U.S. and Canada) or (760) 666-3770 (international) using conference ID number
65266554. To access the webcast, please visit the Investors section of
KYTHERA's website at www.kytherabiopharma.com at least 15 minutes prior to the
start of the call to ensure adequate time for any software downloads that may
be required. A PDF of reference slides will be available during the call.
The call will be available for replay via telephone starting September 17,
2013 at approximately 11 a.m. PDT, running through 11:59 p.m. PDT on October
1, 2013. To listen to the replay, dial (855) 859-2056 (U.S. and Canada) or
(404) 537-3406 (international) using conference ID number 65266554. The
archived webcast will be available on KYTHERA's website for 14 days beginning
approximately one hour after the call has completed.
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