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Auxilium Pharmaceuticals Announces Encouraging Top-line 30-Day Data from XIAFLEX® (CCH) Phase Ib Study in Cellulite



   Auxilium Pharmaceuticals Announces Encouraging Top-line 30-Day Data from
                  XIAFLEX® (CCH) Phase Ib Study in Cellulite

- Data Supports Progression into Phase II; All Doses Were Generally
Well-tolerated -

- Auxilium Plans to Initiate a CCH Phase IIa Cellulite Trial in 2H13 -

PR Newswire

MALVERN, Pa., Dec. 13, 2012

MALVERN, Pa., Dec. 13, 2012 /PRNewswire/ -- Auxilium Pharmaceuticals, Inc.
(NASDAQ: AUXL), a specialty biopharmaceutical company, today announced
top-line Day 30 data for collagenase clostridium histolyticum or CCH for the
potential treatment, if approved by the U.S. Food and Drug Administration
(FDA), of adult patients with edematous fibrosclerotic panniculopathy (EFP),
commonly known as cellulite.  The phase Ib study is a single site, open-label
dose-escalation study that enrolled 99 women between 21 and 60 years of age.
Study participants were assigned to one of 11 arms, each of which varied in
treatment dose, injection concentration and volume, to receive a single
injection of CCH, divided into 10 aliquots over a pre-defined 8x10cm template
around a target dimple.  The objectives of the study are to assess the safety
and effectiveness of a single injection of CCH for the treatment of EFP at 30,
60 and 90 days across multiple dosing arms.  Pharmacokinetic evaluations were
made as well.  Across all dosing arms, 60 patients (63%) who were treated
experienced some improvement in the volume of their target cellulite dimple at
Day 30.  Overall, 17% of patients had a greater than or equal to 30%
improvement in their target dimple at Day 30; however, multiple CCH dosing
arms had more than 40 percent of patients experience an improvement greater
than or equal to 30% in their target dimple at Day 30.  Cellulite has been
reported to occur in 85-98% of post-pubertal females and rarely in men; the
condition is believed to be prevalent in women of all races. ^1,3

(Photo: http://photos.prnewswire.com/prnh/20121213/PH28917-a)
(Photo: http://photos.prnewswire.com/prnh/20121213/PH28917-b)
(Photo: http://photos.prnewswire.com/prnh/20121213/PH28917-c)
(Photo: http://photos.prnewswire.com/prnh/20121213/PH28917-d)
(Logo: http://photos.prnewswire.com/prnh/20101202/MM10881LOGO)

"We are pleased with the safety profile and encouraging potential efficacy of
CCH, with initial phase Ib results demonstrating that some doses of CCH
exhibited potential improvement in dimple volume and were well tolerated with
no dose-limiting side effects," said Dr. James Tursi, Chief Medical Officer at
Auxilium. "We look forward to advancing the clinical development of CCH in
cellulite, which, if successful, we believe could lead to the first
FDA-approved, office-based biological treatment option for cellulite that is
supported by scientific results."

Treatment-related adverse events with CCH were mostly localized bruising,
injection site discomfort and swelling, and all such events resolved without
intervention, which are all consistent with XIAFLEX use in other indications. 
There were no new adverse events and no drug-related serious adverse events
reported. 

The following 3-D images represent baseline and Day 30 images of target
dimples from two subjects who participated in the study.  Red color identifies
the deepest areas within the target dimple, while blue color shows the areas
with shallower depth closest to the normal skin surface.  Following a single
injection of XIAFLEX, subject 1227 demonstrated a 30.6% improvement in target
dimple volume at Day 30 and subject 1057 demonstrated a 42.1% improvement in
target dimple volume at Day 30.  These results are not representative of all
subjects, but help demonstrate the extent of visible improvement across
patients.

(Photo:  http://photos.prnewswire.com/prnh/20121213/PH28917)

"Thirty-day data from our phase Ib cellulite clinical trial represents another
important development milestone for Auxilium as we advance a fourth potential
indication of CCH in development, further diversifying XIAFLEX's growing
pipeline," explained Adrian Adams, Chief Executive Officer and President of
Auxilium. "We believe cellulite represents a significantly undertreated
condition and that innovative approaches such as CCH may, if approved, one day
be a viable solution for treatment."

About Cellulite
Cellulite, also known medically as edematous fibrosclerotic panniculopathy or
EFP, describes a pathologic condition, in which lobules of subcutaneous
adipose tissue extend into the dermal layer.  Cellulite can involve the loss
of elasticity or shrinking of collagen cords, called septae, that attach the
skin to lower layers of muscle. When fat in cellulite prone areas swells and
expands, the septae tether the skin, which causes surface dimpling
characteristic of cellulite.  These changes can visibly affect the shape of
the epidermis and resemble an orange peel-like dimpling of the skin. ^1   CCH
treatment is intended to target and lyse, or break, those collagen tethers
with the goal of releasing the skin dimpling and potentially resulting in
smoothing of the skin.

In the normal subcutaneous fat layer directly under the skin, there are both
perpendicular columnar and net-like fibrous connective tissue called septae.
These fibrous septae, made of types I and III collagen, connect the epidermis
to the dermis and create a network of compartmentalized adipose deposits.
Women tend to have a higher proportion of columnar septae that are
perpendicular to the epidermis, while men tend to have more of the net-like
system.  In cellulite, the subcutaneous fat cells swell and push upwards. ^2
  As a result, the skin between the septae is pushed up and the perpendicular
septae act as an anchor to pull the epidermis downwards and form the classic
cellulite dimple. The surrounding adipose tissue forms small bulges under the
epidermis around the dimple that can give skin a "cottage cheese" texture 

Cellulite occurs mainly on the buttocks, lower limbs, and abdomen and has been
reported to occur in 85-98% of post-pubertal females and rarely in men.  The
condition is believed to be prevalent in women of all races. ^1,3 Cellulite is
different from generalized obesity. The fat cells found in generalized obesity
are not limited to the pelvis, thighs, and abdomen. Further, the fat cells
found in cellulite have different physiologic and biochemical property than
fat tissue located elsewhere. ^3 There is no definitive medical explanation
for the presentation and prevalence of cellulite and, despite multiple types
of therapeutic approaches for the attempted treatment of cellulite, there are
no approved medical treatments and little scientific evidence that any current
treatments are beneficial. ^4

About XIAFLEX
XIAFLEX (collagenase clostridium histolyticum or CCH) is a biologic approved
in the U.S., EU and Canada for the treatment of adult Dupuytren's contracture
patients with a palpable cord. XIAFLEX is a minimally invasive treatment for
this condition and consists of a highly purified combination of subtypes of
collagenase, derived from Clostridium histolyticum, in a specific proportion.
Together, the collagenase sub-types work synergistically to break the bonds of
the triple helix collagen structure more effectively than human collagenase.
XIAFLEX is currently under review by the FDA for the treatment of Peyronie's
disease.  CCH is also in phase IIa of development for the treatment of Frozen
Shoulder syndrome (adhesive capsulitis) and in phase Ib of development for the
treatment of cellulite (edematous fibrosclerotic panniculopathy).

About the Phase Ib Cellulite trial 
The phase Ib study is a single site, open-label dose-escalation study that
enrolled 99 women between 21 and 60 years of age. The objectives of the study
are to assess the safety and effectiveness of CCH for the treatment of EFP at
30, 60 and 90 days following a single dose, as well as evaluation of
pharmacokinetics, to determine if a path forward exists into a later phase of
development.  To qualify for the study participants must have had EFP in the
posterolateral thighs and/or buttocks for at least 12 months prior to a
screening visit. Following screening and determination of eligibility, study
participants were assigned to one of eleven groups that varied in treatment
dose, injection concentration and volume. Subjects received 10 concurrent
injections (0.1 or 0.5 mL per injection) of CCH via a standardized template
over a targeted area (8 cm x 10 cm) of EFP.  The template included a targeted
centralized dimple.  The total dose of CCH that was administered into the
targeted area ranged between 0.0029 mg and 0.464 mg; these doses represent
between 0.5% and 80% of the dose used in a single injection for Dupuytren's
contracture (0.58 mg). Safety will be evaluated through the collection of
adverse events, as well as a targeted assessment of local reactions to the
treatment through 90 days. The treatment effectiveness will be evaluated by
investigator and patient assessments, as well as 3-D photographic imaging
techniques.

About Auxilium
Auxilium Pharmaceuticals, Inc. is a specialty biopharmaceutical company with a
focus on developing and marketing products to predominantly specialist
audiences. Auxilium markets Testim® 1% (testosterone gel) for the topical
treatment of hypogonadism and XIAFLEX® (collagenase clostridium histolyticum)
for the treatment of adult Dupuytren's contracture patients with a palpable
cord in the U.S. GSK co-promotes Testim with Auxilium 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.  Pfizer has marketing rights for
XIAPEX® (the EU tradename for collagenase clostridium histolyticum) in 46
countries in Eurasia through April 24, 2013; 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.  The sBLA for XIAFLEX for the treatment
of Peyronie's disease was submitted to the FDA in November 2012. 
Additionally, collagenase clostridium histolyticum (CCH) is in phase IIa of
development for the treatment of Frozen Shoulder syndrome (adhesive
capsulitis) and phase Ib of development for the treatment of cellulite
(edematous fibrosclerotic panniculopathy).  Auxilium also has rights to pursue
additional indications for CCH. For additional information, visit
http://www.auxilium.com

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 potential for CCH to treat edematous fibrosclerotic
panniculopathy, commonly known as cellulite; the continued evaluation of
subjects in the phase Ib study of CCH for the treatment of cellulite; the
potential progression of CCH for the treatment of cellulite into Phase II and
the timing and design of any subsequent study(ies); the number of people with
cellulite and the market opportunity represented by that number; potential
approval of CCH for the treatment of cellulite; and products in development
for Peyronie's disease, Frozen Shoulder syndrome and cellulite; and all other
statements containing projections, statements of future performance or
expectations, our beliefs or statements of plans or objectives for future
operations (including statements of assumption underlying or relating to any
of the foregoing). Forward-looking statements can generally be identified by
words such as "believe," "appears," "may," "could," "will," "estimate,"
"continue," "anticipate," "intend," "should," "plan," "expect," and other
words and terms of similar meaning in connection with any discussion of
projections, future performance or expectations, beliefs, plans or objectives
for future operations (including statements of assumption underlying or
relating to any of the foregoing). 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,
decisions by regulatory authorities as to whether and when to approve drug
applications, and general financial, economic, regulatory and political
conditions affecting the biotechnology and pharmaceutical industries and those
discussed in Auxilium's Annual Report under the heading "Risk Factors" on Form
10-K for the year ended December 31, 2011 and Form 10-Q for the quarter ended
September 30, 2012, which are 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
Auxilium's home page on the Internet at http://www.Auxilium.com under the
heading "For Investors -- SEC Filings." 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. 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 Auxilium's expectations, plans
or forecasts of future events and views as of the date of this release.
Auxilium anticipates that subsequent events and developments will cause
Auxilium's assessments to change. However, while Auxilium may elect to update
these forward-looking statements at some point in the future, Auxilium
specifically disclaims any obligation to do so. These forward-looking
statements should not be relied upon as representing Auxilium's assessments as
of any date subsequent to the date of this release.

CONTACT:
James E. Fickenscher
Chief Financial Officer, Auxilium Pharmaceuticals, Inc.
+1-484-321-5900
jfickenscher@auxilium.com 
or 
William Q. Sargent Jr.
Vice-President, Investor Relations and Corporate Communications
+1-484-321-5900
wsargent@auxilium.com

References

 1. Avram, Cellulite: a review of its physiology and treatment, Journal of
    Cosmetic Laser Therapy 2004; 6: 181–185
 2. Querleux, Anatomy and physiology of subcutaneous adipose tissue by in vivo
    MRI and spectroscopy: Relationship with sex and presence of cellulite,
    Skin Research and Technology; 8: 118-124
 3. Khan MH et al. Treatment of cellulite: Part I. Pathophysiology. J Am Acad
    Dermatol. 2010 Mar;62(3):361-70.
 4. Wanner M et al. An evidence-based assessment of treatments for cellulite.
    J Drugs Dermatol. 2008 Apr;7(4):341-5.

SOURCE Auxilium Pharmaceuticals, Inc.

Website: http://www.auxilium.com
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