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news.Auxilium Pharmaceuticals announced positive results from a randomized, double-blind Phase 2a study of collagenase clostridium histolyticum (or CCH) for the treatment of edematous fibrosclerotic panniculopathy (EFP), commonly known as cellulite.
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In the Phase 2a trial, all three doses of CCH (low (0.06mg), mid (0.48mg) and high (0.84mg)) showed an improvement in the appearance of cellulite as measured by the trial endpoints of an investigator and a patient score on the Global Aesthetic Improvement Scale (GAIS), which was adapted by the Company for use in cellulite.
The mid and high dose groups demonstrated a statistically significant improvement in the appearance of cellulite, as measured by GAIS scores, with a p-value of <0.05 compared to placebo for both endpoints.
In the mid and high dose groups, 68 percent of patients reported being "Satisfied" or "Very Satisfied" with the results of their treatment, compared to only 34 percent of patients randomized to placebo.
Positive and consistent results were also demonstrated across the trial’s other endpoints, including Physician and Patient assessed Composite Responder Analyses.
CCH was well-tolerated by all dose groups with most adverse events (AEs) being mild to moderate and primarily limited to the local injection area.
Auxilium Pharmaceuticals chief executive officer and president Adrian Adams said: "We continue to be encouraged by the promising and positive results of our CCH program, specifically these Phase 2a data in the cellulite indication.
"We believe these data establish proof-of-concept for CCH as a potential treatment for cellulite and enable Auxilium to pursue our development and regulatory strategy to most efficiently and effectively advance this exciting program.
"We look forward to initiating a planned Phase 2b clinical trial in the second quarter of 2015."
The Phase 2a study enrolled 150 women between the ages of 18 and 45 in the US. Each subject received up to three treatment sessions of drug or placebo according to randomization (5 high-dose: 5 mid: 5 low: 3 placebo) and each treatment session was approximately 21 days apart. Up to 12 injections were administered into cellulite dimples during each session across an entire treatment quadrant – left or right buttock or left or right posterior thigh.
Only the dimples treated on Day 1 were able to be retreated on Day 22 (Treatment Session 2) and Day 43 (Treatment Session 3) if, in the opinion of the investigator, the dimple continued to be evident. A variable number of dimples were able to be treated within one treatment quadrant.
Key Phase 2a Trial Results Include:
Both the mid and high dose groups of CCH demonstrated a statistically significant improvement in investigators’ assessments of the appearance of cellulite, as measured by GAIS scores, with a p-value of <0.05 versus placebo
Both the mid and high dose groups of CCH demonstrated a statistically significant improvement in patients’ assessments of the appearance of cellulite, as measured by GAIS scores, with a p-value of <0.02 versus placebo
68 percent of both mid and high dose patients reported being "Satisfied" or "Very Satisfied" with their cellulite treatment, compared to only 34 percent of placebo patients
Both the mid and high dose groups of CCH demonstrated a statistically significant improvement in the post-hoc composite responder analysis, as measured by combined investigator and patient GAIS scores, with p-values of 0.021 and 0.004, respectively, versus placebo
While the trial was not designed to quantify changes in cellulite dimple measurements, 3-D photography was utilized to enable visualization of treatment effects.
CCH was well-tolerated by all dose groups with most AEs being mild to moderate and primarily limited to the local injection area; 86 percent of all related AEs resolved within 21 days and there was only one serious adverse event in the trial, determined to be unrelated to the treatment drug
Auxilium Pharmaceuticals chief medical officer James Tursi said: "These data have exceeded our expectations, demonstrating statistically significant improvements in cellulite as measured by both physicians and patients.
"These are challenging endpoints and ones that we believe are critical in the context of regulatory consideration.
"We are continuing to analyze these exciting data and will be gathering key opinion leader and regulatory input and look forward to publishing and presenting our findings in peer-reviewed venues in the future."