Auxilium Pharmaceuticals, Inc. Announces First Patient Dosed in Phase 2b Frozen Shoulder Syndrome Study of Collagenase

   Auxilium Pharmaceuticals, Inc. Announces First Patient Dosed in Phase 2b
    Frozen Shoulder Syndrome Study of Collagenase Clostridium Histolyticum

PR Newswire

CHESTERBROOK, Pa., Dec. 13, 2013

CHESTERBROOK, Pa., Dec. 13, 2013 /PRNewswire/ -- Auxilium Pharmaceuticals,
Inc. (NASDAQ: AUXL), a fully integrated specialty biopharmaceutical company,
today announced that the first patient was dosed in its Phase 2b study of
collagenase clostridium histolyticum (CCH) for the treatment of adult patients
with adhesive capsulitis, commonly known as Frozen Shoulder syndrome (FSS).
FSS can be a prolonged, painful condition of the shoulder in which increased
capsular collagen thickening and subsequent capsular contraction in the
shoulder joint is thought to cause a loss of range of motion in the shoulder
and pain. The condition is believed to affect approximately two to five
percent of the general adult population and the full duration of the disease
can last from approximately one year to up to 3.5 years.^[i] Some cases never
completely resolve. The majority of cases are currently managed conservatively
with prolonged physical therapy and occasionally with steroid injections. It
is estimated that approximately 300,000 cases of FSS are diagnosed annually
and approximately 10 percent are treated with an invasive intervention such as
surgery or manipulation under anesthesia on an annual basis.^[ii]

CCH is a biologic approved in the U.S., EU, Canada and Australia under the
trade name XIAFLEX^® for the treatment of adult Dupuytren's contracture (DC)
patients with a palpable cord and in the U.S. as the first Food and Drug
Administration approved non-surgical treatment of adult men with Peyronie's
disease (PD) with a palpable plaque. XIAFLEX consists of a combination of two
subtypes of collagenase, derived from Clostridium histolyticum. Together, the
collagenase sub-types are thought to work synergistically to break the bonds
of the triple helix collagen structure. XIAFLEX has been granted Orphan Drug
Designation in the U.S. by the FDA for DC and PD.

^"The initiation of our Phase 2b Frozen Shoulder syndrome study represents
another development milestone for Auxilium as we further evolve our pipeline
and advance a fourth potential indication for CCH," explained Adrian Adams,
Chief Executive Officer and President of Auxilium. "We believe that our
continued measured investments in research and development, together with the
upcoming product launches of XIAFLEX in Peyronie's disease and STENDRA™ in
erectile dysfunction, position the Company well for growth and shareholder
value creation."

The Phase 2b study is a double-blind, placebo-controlled study of the safety
and efficacy of CCH for the treatment of Stage 2 (frozen stage) unilateral
idiopathic FSS. The study will enroll approximately 300 adult men and women at
approximately 35 sites in the U.S. and Australia. Subjects will be randomized
3:1 to receive CCH or placebo and will receive up to three ultrasound-guided
injections of study drug. Each injection will be separated by a minimum of 21
days. All subjects will also perform home shoulder exercises after the first
injection.

In a prior Auxilium Phase 2a FSS study, both the 0.58mg (1mL) and 0.58mg (2mL)
dosing arms showed statistically significant improvement (p<0.05) from
baseline in forward flexion versus the exercise-only group. The 0.58mg (1mL)
dosing arm also showed statistically significant improvement (p<0.05) from
baseline in shoulder abduction versus the exercise-only group. For this Phase
2b study, the dose selected will be 0.58 mg (1mL).

The Phase 2b study's primary endpoint is the change (degrees) from baseline to
the Day 95 follow-up visit in active forward flexion in the affected shoulder
compared to placebo. Patients will also be assessed using the American
Shoulder and Elbow Surgeons (ASES) Scale for function and pain as well as
additional patient reported outcome measures. Safety assessments will be made
during all study visits and immunogenicity testing will be performed at
screening and at the end of the study.

"We are very excited to be moving into this next phase of development for CCH
in the potential treatment of Frozen Shoulder," said Dr. James Tursi, Chief
Medical Officer of Auxilium. "FSS is a condition with no approved therapies
and limited treatment options. Innovative treatment approaches such as CCH, if
approved by the FDA, may allow patients quicker relief from limits in range of
motion and associated pain."

About Frozen Shoulder Syndrome

FSS, also known as adhesive capsulitis, can be a prolonged, and painful
disorder of diminished shoulder motion. The full duration of the frozen stage
(Stage 2) of disease can last from 1 to 3.5 years with a mean of 30 months.
Increased capsular collagen thickening and subsequent capsular contraction in
the glenohumeral joint is thought to cause restriction in both active and
passive range of motion in the shoulder.^[iii] As the condition progresses,
pain can become more severe and is accompanied by stiffness and decreased
range of motion. The stiffening may increase to the point where the natural
arm swing that accompanies normal gait is lost.^[iv] The most common
treatments for FSS are long-term intensive physical therapy, corticosteroids,
manipulation under anesthesia and/or arthroscopic release and each treatment
has differing associated risks. Drugs are often used to manage associated
pain, but none have been demonstrated to have an impact on the course of FSS.

FSS is estimated to affect approximately two to five percent of the general
adult population^i and has an increased incidence among patients with
diabetes,^[v] Dupuytren's contracture, and thyroid disease. It tends to occur
in a patient's fourth to sixth decade of life. The condition occurs slightly
more often in women than in men, often presents bilaterally and commonly
affects the contralateral side years after onset of symptoms in the first
shoulder, but it does not typically affect the same shoulder twice.[vi]

About XIAFLEX

XIAFLEX (collagenase clostridium histolyticum, or CCH) is a biologic approved
in the U.S., EU, Canada and Australia for the treatment of adult Dupuytren's
contracture (DC) patients with a palpable cord and, in the U.S. for the
treatment of adult men with Peyronie's disease (PD) with a palpable plaque and
curvature deformity of at least 30 degrees at the start of therapy. XIAFLEX
consists of a combination of two subtypes of collagenase, derived from
Clostridium histolyticum. Together, the collagenase sub-types are thought to
work synergistically to break the bonds of the triple helix collagen
structure. XIAFLEX has been granted Orphan status in the U.S. by the FDA for
DC and PD. Additionally, CCH is currently being studied in clinical trials in
the following collagen-related conditions: frozen shoulder syndrome (Phase 2),
cellulite (Phase 2), and lipoma (Phase 2).

About Auxilium

Auxilium Pharmaceuticals, Inc. is a fully integrated specialty
biopharmaceutical company with a focus on developing and commercializing
products to predominantly specialist audiences. Auxilium markets Testim
(testosterone gel) for the topical treatment of hypogonadism in the U.S. and
XIAFLEX (collagenase clostridium histolyticum (CCH) for the treatment of adult
men with Peyronie's disease with a palpable plaque and curvature deformity of
at least 30 degrees at the start of therapy in the U.S., and XIAFLEX for the
treatment of adult Dupuytren's contracture patients with a palpable cord in
the U.S. Ferring International Center S.A. markets Testim in certain countries
of the EU and Paladin Labs Inc. markets Testim in Canada. Swedish Orphan
Biovitrium AB has marketing rights for XIAPEX (the EU tradename for CCH) in 71
Eurasian and African countries. Asahi Kasei Pharma Corporation has development
and commercial rights for XIAFLEX in Japan and Actelion Pharmaceuticals Ltd
has development and commercial rights for XIAFLEX in Canada, Australia, Brazil
and Mexico. Auxilium also markets TESTOPEL®, a long-acting implantable
testosterone replacement therapy, Edex®, the leading branded non-oral drug for
erectile dysfunction, Striant®, a buccal system for testosterone delivery,
Osbon ErecAid®, the leading device for aiding erectile dysfunction, and also
has a non-promoted respiratory franchise, including Theo-24® and Semprex®-D,
along with other non-promoted products, in the U.S. Auxilium has exclusive
marketing rights in the U.S. and Canada for STENDRA™, an oral erectile
dysfunction therapy. Auxilium has two projects in clinical development. CCH is
in Phase 2 of development for the treatment of Frozen Shoulder syndrome
(adhesive capsulitis) and Phase 2 of development for the treatment of
cellulite (edematous fibrosclerotic panniculopathy). Auxilium also has rights
to pursue additional indications for XIAFLEX. For additional information,
visit http://www.auxilium.com.

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

This news release contains forward-looking statements as defined by the
Private Securities Litigation Reform Act of 1995, including statements made
with respect to: the design, efficacy, safety and timing of the Phase 2b study
for CCH for the potential treatment of FSS; whether CCH will be approved by
the FDA and, if approved, whether it will be an effective or successful
treatment option for FSS; if approved, whether CCH for the potential treatment
of FSS will allow quicker relief for patients; whether Auxilium is well
positioned for growth and increased shareholder value; the timing of
Auxilium's launches of XIALFLEX for the treatment of Peyronie's disease and of
STENDRA; Auxilium's other product candidates; and other statements regarding
matters that are not historical facts, and involve predictions. These
statements involve known and unknown risks, uncertainties and other factors
that may cause actual results, performance, achievements or prospects to be
materially different from any future results, performance, achievements or
prospects expressed in or implied by such forward-looking statements. In some
cases you can identify forward-looking statements by terminology such as
''may'', ''will'', ''should'', ''would'', ''expect'', ''intend'', ''plan'',
''anticipate'', ''believe'', ''estimate'', ''predict'', ''potential'',
''seem'', ''seek'', ''future'', ''continue'', or ''appear'' or the negative of
these terms or similar expressions, although not all forward-looking
statements contain these identifying words. Although forward-looking
statements are based on Auxilium's current plans or assessments that are
believed to be reasonable as of the date of this press release, they
inherently involve certain risks and uncertainties. These forward-looking
statements are subject to a number of risks and uncertainties, including those
discussed under ''Risk Factors'' in Auxilium's Annual Report on Form 10-K for
the year ended December 31, 2012 and in other public filings with the SEC,
including, without limitation, as such Form 10-K was updated in Item 8.01 of
the Current Report on Form 8-K filed on April 29, 2013 and Auxilium's
Quarterly Reports for 2013. While Auxilium may elect to update the
forward-looking statements made in this news release in the future, Auxilium
specifically disclaims any obligation to do so. Auxilium's SEC filings may be
accessed electronically by means of the SEC's home page on the Internet at
http://www.sec.gov. There may be additional risks that Auxilium does not
presently know or that Auxilium currently believes are immaterial which could
also cause actual results to differ from those contained in the
forward-looking statements.

Auxilium Contacts:

Keri P. Mattox / SVP, IR &     Nichol L. Ochsner / Senior Director, IR &
Corporate Communications      Corporate Communications
Auxilium Pharmaceuticals, Inc. Auxilium Pharmaceuticals, Inc.
(484) 321-5900                (484) 321-5900
Kmattox@auxilium.com          Nochsner@auxilium.com

^[i] Hsu JE, Anakwenze OA, Warrender WJ, Abboud JA. Current review of adhesive
capsulitis. J Shoulder Elbow Surg. 2011 Mar;20(3):502-14.
^[ii] SDI claims data.
^[iii] Pal B, Anderson J, Dick WC, Griffiths D. Limitation of joint mobility
and shoulder capsulitis in insulin and non-insulin dependent diabetes
mellitus.Br J Rheumatol. 1986;25:147-51.
^[iv] Rizk TE, Pinals RS. Frozen shoulder. Semin Arthritis Rheum. 1982
May;11(4):440–452.
^[v] Pal B, Anderson J, Dick WC, Griffiths D. Limitation of joint mobility and
shoulder capsulitis in insulin and non-insulin dependent diabetes mellitus.Br
J Rheumatol. 1986;25:147-51.
^[vi] Hsu JE, Anakwenze OA, Warrender WJ, Abboud JA. Current review of
adhesive capsulitis. J Shoulder Elbow Surg. 2011 Mar; 20(3):502-14.

SOURCE Auxilium Pharmaceuticals, Inc.

Website: http://www.auxilium.com