Phase III study evaluating PEP005 Gel for treatment of AK meets primary clinical endpoint

LEO Pharma today announced that in a Phase III study, PEP005 (ingenol mebutate) Gel, the company's lead candidate for the topical treatment of actinic (solar) keratosis (AK) or pre-cancerous lesions, met its primary clinical endpoint of complete clearance of AK lesions in non-head locations.  Findings from the REGION Ib study in body AK support results from the earlier REGION-I trial, a study evaluating PEP005 for the treatment of AK on the head reported in December 2009.

AK is a pre-cancerous skin condition that if left untreated, can lead to the development of skin cancer.

"PEP005 is a recent addition to LEO Pharma's robust dermatology pipeline.  We could not be happier with the preliminary findings that suggest PEP005 may be a promising new treatment option for patients with this type condition, a pre-cursor to skin cancer," said Lars Olsen, Executive Vice President for Research & Development, LEO Pharma.  

Cryotherapy, a procedure in which the surface skin lesions are frozen, is currently the most common treatment alternative for AK. It is used as the sole approach in approximately 75% of treatments for AK lesions, and in combination with topical drugs in approximately 9% of treatments. The American Academy of Dermatology reports that 40 percent of cases of squamous cell carcinoma begin as AKs.

Approximately 250 patients were enrolled in the REGION Ib study, which evaluated the use of a 0.05% concentration of PEP005 Gel applied daily as monotherapy for two consecutive days to AK lesions on non-head locations.  

PEP005 Gel demonstrated a favorable safety profile and was well tolerated; side effects were consistent with those seen in Phase II studies. There were no drug-related serious adverse events reported. The trials were initiated and run by Peplin, Inc., acquired by LEO Pharma in September 2009.  

Results from REGION I and Ib will be presented at an upcoming meeting of the American Academy of Dermatology.

LEO Pharma expects to file for regulatory approval of the treatment in the U.S. and Europe in 2011.