Auxilium Announces Results from Phase III Trial that Show AA4500 Successful in Treating Dupuytren's ContractureLong-Term Study Shows Company's Lead Injectable Collagenase Enzyme Safe and Effective in Treating Patients with Multiple Contracted Hand Joints
MALVERN, Pa., February 20, 2007 /PRNewswire-FirstCall/ -- Auxilium Pharmaceuticals, Inc. today announced the positive final results of an early Phase III study of its lead development product AA4500 in the treatment of Dupuytren's contracture, a disabling and recurring hand condition in which the joints contract, impairing patients' ability to straighten and move their fingers. The study was comprised of a randomized, placebo-controlled, double- blind phase, as well as an open-label phase. As previously reported, data from the double-blind phase showed that injecting the collagenase enzyme into study patients' affected joints led to a 91 percent success rate in reducing joint contracture to within five degrees of normal, compared to the placebo arm in which no patients achieved success. In the open-label phase, in which patients had additional joints treated with AA4500, results showed that 88 percent of Metacarpophalangeal (MP) joints and 68 percent of Proximal Interphalangeal (PIP) joints were fully corrected.
The data from this study were presented at the 74th annual meeting of the American Academy of Orthopedic Surgeons (AAOS) in San Diego, CA by Lawrence Hurst, M.D., lead investigator and Professor and Chair of Department of Orthopaedics and Chief of the Division of Hand Surgery at SUNY at Stony Brook, NY.
"These statistically significant study results exceeded our expectations and show the potential for AA4500 to become an effective, first line therapy for patients suffering from Dupuytren's contracture, a condition currently treated with complex hand surgery," said Dr. Hurst. "PIP joint contractures are the most difficult to correct with surgery. Even in these joints, AA4500 demonstrated results that are at least equal to surgery if not better. AA4500 was well tolerated. It was reassuring that we were able to give patients multiple injections in multiple joints with no immunological side effects."
Dr. Hurst added, "The patients' willingness and desire to continue being treated demonstrated their satisfaction with this non-surgical treatment of Dupuytren's contracture."
The Company is also studying AA4500 as a treatment for other disabling conditions, including Peyronie's disease and frozen shoulder syndrome.
"The compelling results of this study support our belief that AA4500 may fundamentally change the treatment paradigm for Dupuytren's contracture," said Jyrki Mattila, M.D., Ph.D., Executive Vice President, Business Development, R&D, and Technical Operations of Auxilium Pharmaceuticals. "We consider these results a significant milestone for the Company's AA4500 clinical development program and importantly for patients, as this product appears to offer them an effective, non-surgical treatment for Dupuytren's contracture. We have completed market research that leads us to believe that the annual opportunity for Dupuytren's and Peyronie's in the U.S. and Europe is in excess of $1 billion."
Study Design and Key Findings
This was a randomized, double-blind, placebo-controlled phase III clinical trial. After finishing this pivotal efficacy study, patients from both AA4500 and placebo groups were permitted to receive additional AA4500 injections for either unsuccessfully treated joints or additional untreated joints during an open-label, extended treatment period. This open-label phase provided further support for the long-term safety and efficacy of AA4500 injections in the treatment of Dupuytren's contracture.
In the double-blind study, 35 patients with Dupuytren's contracture and fixed-extension deformity of at least 20 degrees in a single finger were enrolled and randomly assigned in a 2:1 ratio to receive either AA4500 treatment or placebo, with the goal of reaching therapeutic success. Therapeutic success was defined as reducing the contracture of the affected joint to within five degrees of normal, essentially allowing the hand to be flat when placed on a table. AA4500 achieved a 91 percent success rate for the primary endpoint of reduced contracture to within five degrees of normal in treated joints, both PIP and MP joints, after up to three injections, compared to a 0% response rate in the placebo group (p<0.001). When stratified by joint type, 100 percent of PIP joints and 86 percent of MP joints achieved therapeutic success. The mean number of injections per joint was 1.4. The results observed after a single injection of AA4500 showed that 70% of subjects achieved therapeutic success; no patients responded to placebo (p<0.001). When stratified by joint type, 67 percent of PIP joints and 71 percent of MP joints achieved therapeutic success after a single injection.
These results were consistent with those from a Phase II study published in The Journal of Hand Surgery (2002:27A:788-798).
A total of 19 patients enrolled in the open-label extension phase of the study and received up to five injections of AA4500. This treatment period was a continuation of the protocol in the double-blind study. These results demonstrated 88 percent of MP joints and 68 percent of PIP joints achieved therapeutic success after receiving an average of 1.4 injections of AA4500.
Safety data in both studies showed that the most commonly reported adverse events were pain and swelling (edema) of the hand at the injection site, and post-injection temporary swelling of a modest nature in the lymph node area of the armpit. Adverse events were mild to moderate in nature and resolved without treatment within 30 days. There were no serious adverse events reported which were judged by the investigator to be related to treatment with AA4500. BioSpecifics Technologies Corp., licensor of AA4500, sponsored and monitored the trials.
About Dupuytren's contracture
Dupuytren's contracture is a condition that involves contracture of joints in the hand that impairs patients' ability to straighten and move their fingers due to a thickening and shortening of the normal ligaments of the palm and fingers. As the disease progresses, functionality of the hand is severely impaired. The incidence of Dupuytren's contracture is highest in Caucasians, historically those of Northern European descent. Most cases of Dupuytren's contracture occur in patients older than 50 years.(1)
The most frequently affected joints associated with Dupuytren's contracture are the joints called the Metacarpal Phalangeal Joint, or MP joint, which is the joint closest to the palm of the hand and the Proximal Intra-Phalangeal Joint, or the PIP joint, which is the middle joint in the finger. The little finger and ring finger are most frequently involved.
(1) Badalamente, M. A., Hurst, L. C. et al., Collagen as a Clinical Target: Nonoperative Treatment of Dupuytren's Disease, The Journal of Hand Surgery, (2002;27A:788-798) About Auxilium
Auxilium Pharmaceuticals, Inc. is a specialty biopharmaceutical company with a focus on developing and marketing to urologists, endocrinologists, orthopedists and select primary care physicians. Auxilium markets Testim(R) 1%, a topical testosterone gel, for the treatment of hypogonadism through its approximately 190-person sales and marketing team. Auxilium has four projects in clinical development. Auxilium believes that AA4500, an injectable collagenase enzyme, is in phase III of development for the treatment of Dupuytren's contracture and is in phase II of development for the treatment of Peyronie's disease and Frozen Shoulder syndrome (Adhesive Capsulitis). Auxilium's transmucosal film product candidate for the treatment of overactive bladder (AA4010) is in phase I of development. The Company is currently seeking a partner to further develop this product candidate. Auxilium has two pain products using its transmucosal film delivery system in pre-clinical development. Auxilium has rights to six additional pain products and products for hormone replacement and urologic disease using its transmucosal film delivery system, and options to all indications using AA4500 for non-topical formulations. For additional information, visit http://www.auxilium.com.
Safe Harbor Statement
This release contains "forward-looking-statements" within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the interpretation of clinical results presented at the 74th annual meeting of the American Academy of Orthopedic Surgeons in San Diego, CA, the impact of AA4500 on the treatment paradigm for Dupuytren's contracture, and products in development. 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, financial guidance and other statements containing the words "believe," "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 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 period ended December 31, 2005 and in the Company's Quarterly Report on Form 10-Q for the period ended September 30, 2006 under the heading "Risk Factors," 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 "Investor Relations - 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 the forward-looking statements. Given these risks and uncertainties, any or all of these forward-looking statements 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.
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Posted: February 2007