Celgene Announces Positive Top Line Data from Randomized Controlled Phase II Study of Apremilast in Psoriatic Arthritis
Study met primary objective using 20 mg of apremilast twice per day with superior ACR20 of 43.5% (p<0.0001) compared to placebo (11.8%) after 12 weeks of oral treatment
Impressive safety and tolerability profile
Efficacy and adverse event profile supports initiation of pivotal phase III studies in the first half of 2010
SUMMIT, N.J.--(BUSINESS WIRE)--Jun 15, 2009 - Celgene Corporation (NASDAQ: CELG) announced the preliminary results of a phase II, multi-center, randomized, double-blind, placebo-controlled, three-arm study of apremilast - a novel, orally available small molecule that exhibits anti- inflammatory activities through the suppression of multiple pro-inflammatory mediators and cytokines - in adult patients with psoriatic arthritis (CC-10004-PSA-001). The study met its primary objective of assessment of ACR20 at 12 weeks. ACR20 is defined as the percentage of patients achieving a 20% or better improvement according to the American College of Rheumatology (ACR) criteria. ACR20 is the primary assessment utilized by the U.S. Food and Drug Administration for assessment of efficacy in psoriatic arthritis, as well as rheumatoid arthritis.
The study sought to determine the efficacy and safety of apremilast in 204 patients at two different dose regimens - 20mg twice per day and 40mg once per day - compared to placebo after 12 weeks. In the study, both apremilast treatment arms had a significant improvement in their ACR20 outcome versus placebo: 43.5% of patients in the 20 mg twice daily arm and 35.8% of patients in the 40 mg once daily arm achieved an ACR20 compared to 11.8% of patients in the placebo arm.
In addition, the study measured secondary 12-week endpoints including ACR50 and ACR70, defined as the percentage of patients achieving 50% and 70% improvements respectively according to ACR criteria. These measures are utilized to demonstrate clinical benefit for patients in addition to the primary regulatory measure of ACR20. The 12-week ACR50 was 17.4% in the 20mg twice daily arm, 13.4% in the 40 mg once daily arm, and 2.9% in the placebo arm. The 12-week ACR70 was 5.8% in the 20 mg twice daily arm, 7.5% in the 40 mg once daily arm, and 1.5% in the placebo arm.
The five most common adverse events reported in the study were nausea, diarrhea, headache, nasopharyngitis and fatigue. Additionally, there was not a significant difference in infections between apremilast and placebo. In the study 9, 6 and 3 percent of patients discontinued treatment due to adverse events while 9, 0 and 18 percent discontinued due to lack of efficacy in the 20 mg twice daily, 40 mg once daily and placebo groups, respectively.
“The positive data from this phase II study are encouraging indicators of the potential of apremilast as an innovative oral treatment that may fill a significant unmet need in this debilitating disease,” said Randall M. Stevens, MD, Vice President and Head of Immunology and Inflammation Clinical Development for Celgene. “The activity of apremilast has now been demonstrated in studies of both psoriasis and psoriatic arthritis including a favorable safety and tolerability profile. Based on these results, we plan to actively pursue registration studies in psoriatic arthritis as a part of our larger development plan in inflammatory diseases.”
Based on our evaluation of other ongoing trials, Celgene plans to initiate pivotal phase III studies that include doses of 20 mg twice a day, as well as 30 mg twice a day to optimize the therapeutic potential of apremilast. This dosing schedule is also being investigated in a phase IIb study initiated in February 2008 in moderate-to-severe plaque-type psoriasis with results expected in the first half of 2010.
In addition to the planned psoriatic arthritis pivotal program, a phase III study of apremilast in moderate-to-severe plaque-type psoriasis is anticipated to begin in 2010, pending the results of the phase IIb trial. Apremilast is also currently being investigated in a phase II study in recalcitrant plaque-type psoriasis with data expected in the first quarter of 2010. A number of investigator-initiated trials are ongoing in cutaneous lupus, prurigo nodularis, ankylosing spondylitis, erosive osteoarthritis, cutaneous sarcoidosis and a number of other inflammatory conditions.
Apremilast is a novel, orally available small molecule compound that exhibits anti-inflammatory activities through the suppression of multiple pro-inflammatory mediators including, TNF-alpha, interleukins 6, 17 & 23, and interferon-gamma among others. Apremilast is the lead investigational anti-inflammatory compound in the Celgene Inflammation Franchise, and is in phase II clinical development for the treatment of psoriasis, psoriatic arthritis and in proof of concept trials in other inflammatory diseases.
About Psoriatic Arthritis
Psoriatic arthritis is a type of inflammatory arthritis that affects more than a million people in the U.S. and Europe. This debilitating condition causes pain, stiffness and swelling in and around the joints, as well as joint destruction. This data positions apremilast as an innovative oral treatment, with the potential to fill a significant unmet need in this disease category.
About Celgene Corporation
Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of novel therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit the company's Web site at www.celgene.com.
This release contains certain forward-looking statements which involve known and unknown risks, delays, uncertainties and other factors not under the Company's control, which may cause actual results, performance or achievements of the Company to be materially different from the results, performance or other expectations implied by these forward-looking statements. These factors include results of current or pending research and development activities, actions by the FDA and other regulatory authorities, and those factors detailed in the Company's filings with the Securities and Exchange Commission such as Form 10-K, 10-Q and 8-K reports.
Contact: Celgene Corporation
David Gryska, 908-673-9059
Sr. Vice President and
Chief Financial Officer
Brian P. Gill, 908-673-9530
Posted: June 2009