Kythera Biopharmaceuticals, Inc. recently reported encouraging data from two phase III studies on ATX-101. The pivotal studies, REFINE-1 (Study ATX-101-11-22) and REFINE-2 (Study ATX-101-11-23), were conducted in the U.S. and Canada. All primary as well as secondary endpoints were met. ATX-101 is being evaluated for the reduction of submental fat.
Kythera is focused on developing and commercializing novel prescription products catering the aesthetic medicine market. Upon approval, ATX-101 will enjoy the status of being the first-in-class submental contouring injectable drug.
The two identical multicenter, double-blind, randomized, placebo-controlled Phase III pivotal trials, REFINE-1 and REFINE-2, were conducted to evaluate the safety and efficacy of ATX-101 versus placebo. The enrolled patients had moderate to severe submental fat at baseline. The severity of the submental fat was decided on the basis of both validated clinician- and patient-rating scales.
Proportionate simultaneous improvement of at least one grade from baseline on the Clinician-Reported Submental Fat Rating Scale (CR-SMFRS) and the Patient-Reported Submental Fat Rating Scale (PR-SMFRS) was the first primary endpoint. Proportionate simultaneous improvement at least two grades from baseline on the CR-SMFRS and the PR-SMFRS was the second primary endpoint.
The REFINE-1 study result showed that while 70.3% patients on ATX-101 (versus 18.7% in the placebo arm) showed an improvement of at least one grade, 13.4% patients on ATX-101 (versus 0% in the placebo arm) improved two grades from the baseline.
The REFINE-2 study result showed that while 66.9% patients on ATX-101 (versus 22.4% in the placebo arm) showed an improvement of at least one grade, 18.7% patients on ATX-101 (versus 3.2% in the placebo arm) improved two grades from the baseline.
The studies also achieved secondary endpoints which included reduction in volume of the submental region as measured by magnetic resonance imaging (MRI), and improvement in appearance-related impacts of submental fat, assessed using the PR-SMFIS.
As far as the safety profile is concerned, there were no treatment-related serious adverse events.
Kythera currently carries a Zacks Rank #3 (Hold). Currently, companies which look attractive include Gilead Sciences Inc. (GILD - Analyst Report) and Actelion Ltd. (ALIOF - Snapshot Report) with a Zacks Rank #1 (Strong Buy), and Jazz Pharmaceuticals (JAZZ - Analyst Report) with a Zacks Rank #2 (Buy).