Kythera Biopharmaceuticals, Inc. (KYTH) 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) and Actelion Ltd. (ALIOF) with a Zacks Rank #1 (Strong Buy), and Jazz Pharmaceuticals (JAZZ) with a Zacks Rank #2 (Buy).