MALVERN, Pa., Nov. 20 /PRNewswire-FirstCall/ -- Auxilium Pharmaceuticals, Inc. today announced that the first patients have been dosed in the Company's second U.S. Phase III pivotal trial and the first Phase III study outside the U.S. for AA4500 for the treatment of Dupuytren's contracture, a disabling and recurring condition in which the joints in the hand contract, impairing patients' ability to straighten and move their fingers.
"We are tremendously excited about achieving these important development milestones for AA4500, our lead pipeline candidate," said Mr. Armando Anido, Chief Executive Officer and President. "AA4500 is a company-transforming product opportunity that has the potential to be the first, effective, non-surgical treatment for patients with Dupuytren's contracture."
The U.S. study is a double-blind, randomized, placebo controlled study of AA4500 involving more than 200 patients at up to 15 sites throughout the U.S. Patients in the study will be randomized on a 2:1 basis in favor of AA4500 treatment. To qualify for the study, patients must have at least 20 degrees of contracture. The primary endpoint of the study is to determine if AA4500 can reduce the contracture angle to within 0 to 5 degrees of normal as measured by digital goniometry.
Upon completion of the study, all patients will be enrolled in a separate open label extension study in which the patient will receive active drug if they were initially given placebo and/or they have the opportunity to have other affected joints treated. Patients from the pivotal study and the open label extension study will form the basis of 12-month follow-up data to be used in the Biologics License Application for marketing approval with the U.S. Food and Drug Administration.
The ex-U.S. study is being conducted in Australia and Switzerland. It also is a double-blind, randomized, placebo-controlled study involving up to 60 patients and follows a protocol similar to that of the U.S. pivotal Phase III trial and its open label extension.
"As AA4500 is ultimately targeted for worldwide markets, we believe that it is very important to perform well controlled clinical trials in sites in and outside of the U.S.," said Dr. Jyrki Mattila M.D., Ph.D., Executive Vice President of R&D, Business Development and Technical Operations. "The fact that individuals of Northern European ancestry seem to be pre-disposed to Dupuytren's contracture provides a significant opportunity for us to generate clinical data in Europe, and we also are eager to raise physician and patient awareness of the potential benefits of AA4500 overseas."
An earlier pivotal Phase III study, sponsored and monitored by BioSpecifics Technologies Corp., licensor of AA4500, with follow up and data analysis conducted by Auxilium, showed promising results. The trial involved a total of 35 patients, 23 of whom were randomized to receive up to three injections of AA4500 and 12 patients who were randomized to receive placebo. AA4500 achieved a 91% success rate for the primary endpoint of less than five degrees of contracture in treated joints, including both Proximal Intra-Phalangeal (PIP) joints and Metacarpal Phalangeal (MP) joints, after up to three injections. The placebo group had a 0% response rate (p < 0.001). 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 (contracture reduced to five degrees or less); no patients responded to placebo (p < 0.001). These Phase III results were consistent with those from a Phase II study published in The Journal of Hand Surgery (2002:27A:788-798).
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 180-person sales and marketing team. Auxilium has four projects in clinical development. Auxilium believes that AA4500, an injectable 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 timing and outcome of the clinical studies for AA4500 for the treatment of Dupuytren's contracture, the benefit to patients of AA4500 for the treatment of Dupuytren's contracture, the timing for the commencement of various clinical trials for Auxilium's product candidates, including AA4500, during 2006. 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 the Company's Quarterly Report on Form 10-Q for the quarterly period ended June 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.
