Auxilium Pharmaceuticals, Inc. Completes Enrollment of XIAFLEX® Phase III Double-Blind Studies for Peyronie's Disease

MALVERN, Pa., March 9, 2011 /PRNewswire/ -- Auxilium Pharmaceuticals, Inc. (Nasdaq: AUXL), a specialty biopharmaceutical company, today announced it has reached the target enrollment for the double-blind placebo-controlled phase III program of XIAFLEX^® for the treatment of Peyronie's disease.  In accordance with the study design, all enrolled patients will have received their first injection of either XIAFLEX or placebo by the end of March 2011.  The Company will exceed its enrollment target of 600 patients.  Peyronie's disease is the development of a collagen plaque on the penis that can cause the penis to curve during erection, often interfering with or preventing intercourse and resulting in psychological distress or bother for the patient.

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"We are very pleased to achieve this important development milestone in Peyronie's disease and advance what can potentially be a second indication for XIAFLEX," said Armando Anido, Chief Executive Officer and President of Auxilium. "We believe completing the enrollment of over 600 patients at sites in the U.S. and Australia in less than six months speaks to the enthusiasm in the urology community and their prospective patients for a non-surgical treatment alternative. We anticipate reporting top-line results in the first half of 2012."  

The late stage global development plan for XIAFLEX for Peyronie's disease consists of four clinical studies and is known by the acronym IMPRESS – The Investigation for Maximal Peyronie's Reduction Efficacy and Safety Studies.  The two randomized, double-blind, placebo-controlled phase III studies enrolled a total of over 600 patients at approximately 70 sites in the U.S. and Australia, with a 2:1 ratio of XIAFLEX to placebo.  There is also one open label study, which is expected to enroll approximately 250 patients, at approximately 30 sites in the U.S., EU and New Zealand, and one pharmacokinetic study, which should enroll approximately 16 patients in the U.S.  XIAFLEX will be administered two times a week every six weeks for up to four treatment cycles (2 x 4).  Each treatment cycle will be followed by a penile modeling procedure.  Patients will be followed for 52 weeks post-first injection in the double-blind studies and for 36 weeks in the open label trial.  

The trials' co-primary endpoints are the mean percent improvement from baseline in penile curvature compared to placebo and the mean change from baseline in the Peyronie's disease bother domain of the Peyronie's Disease Questionnaire (PDQ) compared to placebo.  The PDQ is a proprietary questionnaire that the Company developed with the FDA following our phase IIb studies as a valid tool for measuring Peyronie's disease bother.  The PDQ also measures severity of psychological and physical symptoms of Peyronie's disease and penile pain.  Safety measurements include adverse event monitoring, immunogenicity testing and clinical labs.

"We are encouraged by the clinical profile of XIAFLEX, which emerged from our earlier phase II and IIb clinical trials in Peyronie's disease," said James P. Tursi, M.D., Auxilium's Vice President of Clinical Research & Development.  "We believe that our phase III double-blind studies with XIAFLEX in Peyronie's disease should provide pivotal evidence that XIAFLEX is well-tolerated and produces clinically and statistically significant reductions in both penile curvature and disease bother."  

About Peyronie's Disease

Peyronie's disease is the development of collagen plaque, or scar tissue, on the shaft of the penis that hardens and reduces flexibility, thus causing pain and forcing the penis to bend or arc during erection. This may prevent intercourse. In addition to difficulty with sexual intercourse, Peyronie's disease may also be associated with emotional distress, bother, loss of self-esteem and depression. Prevalence of Peyronie's disease is estimated to be approximately 5% in adult men, but actual prevalence may be higher, based on autopsies. (1,2,3,4)

1. Bella  A. Peyronie's Disease J Sex Med 2007;4:1527–1538
2. Lue TF, et al. Summary of the recommendations on sexual dysfunctions in men. J Sex Med 2004;1:6–23.
3. Mulhall JP, et al. Subjective and objective analysis of the prevalence of Peyronie's disease in a population of men presenting for prostate cancer screening. J Urol 2004;171:2350–3.
4. Smith BH. Am J Clin Pathol. 1966;45:670-678.

About Auxilium

Auxilium Pharmaceuticals, Inc. is a specialty biopharmaceutical company with a focus on developing and marketing products to predominantly specialist audiences, such as urologists, endocrinologists, certain targeted primary care physicians, hand surgeons, subsets of orthopedic, general, and plastic surgeons who focus on the hand, and rheumatologists.  Auxilium markets XIAFLEX® (collagenase clostridium histolyticum) for the treatment of adult Dupuytren's contracture patients with a palpable cord and Testim® 1%, a testosterone gel, for the topical treatment of hypogonadism.  Auxilium has two projects in clinical development.  XIAFLEX is in phase III of development for the treatment of Peyronie's disease and is in phase II of development for treatment of Frozen Shoulder syndrome (Adhesive Capsulitis).  Auxilium also has rights to pursue additional indications for XIAFLEX.  For additional information, visit http://www.auxilium.com.

AUXILIUM SAFE HARBOR STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995

This release contains "forward-looking-statements" within the meaning of The Private Securities Litigation Reform Act of 1995, including statements regarding the scope, enrollment, timing, methodology, endpoints, safety, execution and results of the phase III studies for XIAFLEX for the treatment of Peyronie's disease; the level of interest in XIAFLEX as a treatment for Peyronie's disease among the urology community and prospective patients; the timing of reporting of results from the phase III trial for XIAFLEX for the treatment of Peyronie's disease; the potential for a second indication for XIAFLEX; the interpretation of data from earlier phase II clinical trials for Peyronie's disease; the number of men affected by Peyronie's disease; and products in development for Dupuytren's contracture and Frozen Shoulder syndrome.  All statements other than statements of historical facts contained in this release, including but not limited to, statements regarding future expectations, plans and prospects for the Company, statements regarding forward-looking financial information and other statements containing the words "believe," "appears," "may," "could," "will," "estimate," "continue," "anticipate," "intend," "should," "plan," "expect," and similar expressions, as they relate to the Company, constitute forward-looking statements. Actual results may differ materially from those reflected in these forward-looking statements due to various factors, including further evaluation of clinical data, results of clinical trials, the performance of third-party service providers, decisions by regulatory authorities regarding whether or when to proceed to the next phase of clinical trials, general financial, economic, regulatory and political conditions affecting the biotechnology and pharmaceutical industries and those discussed in the Company's Annual Report on Form 10-K for the year ended December 31, 2010, which is on file with the Securities and Exchange Commission (the "SEC") and may be accessed electronically by means of the SEC's home page on the Internet at http://www.sec.gov or by means of the Company's home page on the Internet at http://www.auxilium.com under the heading "For Investors -- SEC Filings." There may be additional risks that the Company does not presently know or that the Company currently believes are immaterial which could also cause actual results to differ from those contained in forward-looking statements. Given these risks and uncertainties, any or all of the forward-looking statements contained in this release may prove to be incorrect. Therefore, you should not rely on any such factors or forward-looking statements.

In addition, forward-looking statements provide the Company's expectations, plans or forecasts of future events and views as of the date of this release. The Company anticipates that subsequent events and developments will cause the Company's assessments to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's assessments as of any date subsequent to the date of this release.

SOURCE Auxilium Pharmaceuticals, Inc.