CORD I phase III clinical trial results of XIAFLEX published

Sep 3 2009

Auxilium Pharmaceuticals, Inc. (NASDAQ: AUXL), a specialty biopharmaceutical company, today announced that The New England Journal of Medicine (NEJM) has published in its September 3rd edition, the Company's pivotal CORD I Phase III clinical trial of XIAFLEX™ (collagenase clostridium histolyticum) a novel, first-in-class, biologic for the nonsurgical treatment of Dupuytren's contracture. The CORD I study is the largest prospective clinical trial ever conducted in the field of Dupuytren's contracture. Treatment with XIAFLEX significantly reduced the angle of contracture for patients with Dupuytren's contracture in both their metacarpophalangeal (MP) and proximal interphalangeal (PIP) joints, with clinically meaningful responses in both less severe and more severe contractures.

"The results for MP and PIP joints that were treated with XIAFLEX are compelling and compare favorably to surgery from both an efficacy and safety point of view," said Larry Hurst, M.D., study investigator and Professor and Chair, Department of Orthopaedics at SUNY Stony Brook. "As noted in the publication, the investigators believe that most patients with Dupuytren's contracture would be candidates for treatment with XIAFLEX and early intervention may be a prudent treatment approach. I believe that XIAFLEX, as a new non-surgical treatment, could potentially become the standard of care for Dupuytren's contracture."

Auxilium has previously announced top line results of the CORD I study, which indicate that 64.0% of all joints treated with XIAFLEX achieved the primary endpoint of 0° to 5° of full extension (normal or near normal straightening of the treated finger) at 30 days after the last injection compared to 6.8% of patients treated with placebo (P < 0.001). Today's publication includes stratified results of the study on a joint by joint basis and by severity of the diseased joint. Significant findings include:

"Dupuytren's cords may become disabling at an early stage of contracture and can significantly impact many aspects of patients' daily lives," said Dr. Tony DelConte, Chief Medical Officer for Auxilium. "We believe these new data will be well-received by the physician and patient community who have expressed a need for an alternative treatment option to the current standards of observation and, eventually, surgery."

The CORD I study was designed to assess the safety and efficacy of XIAFLEX therapy in Dupuytren's contracture with MP or PIP joint contractures greater than or equal to 20° at baseline. In the 3-month double-blind portion of the study, 308 patients with Dupuytren's contractures (stratified 2:1, MP joint to PIP joint) were initially randomized to receive up to 3 once-monthly doses of XIAFLEX (0.58 mg) or placebo. There were 203 patients randomized to receive XIAFLEX and 103 patients randomized to receive placebo that had primary joints able to be evaluated.

The most commonly reported adverse events were edema peripheral, contusion, injection-site hemorrhage, injection-site pain, upper-extremity pain, tenderness, ecchymosis, injection-site swelling, pruritus, skin laceration, lymph-node enlargement and tenderness on palpation. Most treatment-related adverse events were mild or moderate in intensity and the median resolution time for all adverse events was 10 days, with limited or no medical intervention. Three treatment-related serious adverse events were reported: two tendon ruptures and one complex regional pain syndrome. No deaths, clinically meaningful hypersensitivity events, nerve injuries, arterial injuries or significant changes in flexion or grip strength were observed.

Auxilium has filed a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for the use of XIAFLEX in the treatment of advanced Dupuytren's disease. The FDA's Division of Arthritis, Anesthesia and Rheumatoid Products has scheduled an Advisory Panel to review XIAFLEX on September 16, 2009 at the Holiday Inn in Gaithersberg, MD. If approved by the FDA, XIAFLEX is expected to be the first nonsurgical therapy indicated for Dupuytren's contracture.