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Auxilium Announces Top Line Phase IIb
XIAFLEX™ Peyronie's Results
XIAFLEX Meets Endpoint for Improvement in Change in Penile
Curvature and the Patient Reported Outcome (PRO) Endpoint for
Disease Bother in the Phase IIb Study for Peyronie's Disease;
Results From Phase IIb Trial Provide Support for Validation of
PRO; Auxilium to Host Conference Call and Webcast on Wednesday,
December 16th at 10:00 a.m. EST
MALVERN, PA, Dec 16, 2009 (MARKETWIRE via COMTEX) -- Auxilium
Pharmaceuticals, Inc. (NASDAQ: AUXL), a specialty
biopharmaceutical company, today announced top-line efficacy and
safety results for the phase IIb clinical trial for XIAFLEX™
(collagenase clostridium histolyticum) in the treatment of
Peyronie's disease. The phase IIb study was designed to measure
efficacy endpoints of improvement in penile curvature and
improvement in patients' sexual quality of life using the
Company's Peyronie's disease Patient Reported Outcome (PRO)
questionnaire. Overall, XIAFLEX demonstrated a statistically
significant change compared to placebo at 36 weeks in both
improvement in penile curvature (p=0.001) and the PRO Peyronie's
disease bother domain (p=0.046). XIAFLEX was well-tolerated and
the most common treatment related adverse events in the phase
IIb study were consistent with adverse events reported in
previous Peyronie's disease trials with XIAFLEX, which included
injection site bruising, edema and pain.
"We believe the results from this trial provide a meaningful
step forward to a better understanding of the treatment of
Peyronie's disease with XIAFLEX," said Armando Anido, Auxilium's
Chief Executive Officer and President. "We expect to meet with
the FDA in the second quarter of 2010 to discuss a proposed
phase III plan that potentially could be started in the second
half of 2010."
Phase IIb Efficacy Results
The XIAFLEX Peyronie's disease phase IIb trial is one of the
largest prospective, randomized, placebo-controlled studies
conducted in Peyronie's disease. A total of 145 patients
evaluable for efficacy enrolled in 12 clinical sites across the
U.S., with 109 patients receiving XIAFLEX as a series of
intralesional injections and 36 receiving placebo (3:1 ratio) in
the study. The treatment and placebo arms were also randomized
to test for a benefit with the addition of penile modeling
versus no modeling (1:1). Modeling refers to massaging of the
plaque and is intended to maximize the enzymatic effect of the
XIAFLEX injection in the plaque.
Overall results:
-- In patients who received XIAFLEX, a mean improvement of 29.7%
in
penile curvature from baseline to 36 weeks was seen (54.4
degrees to 38.2 degrees) vs. an
11.0% mean improvement in curvature seen in placebo patients
(50.6 degrees to
45.1 degrees); (p=0.001).
-- In patients who received XIAFLEX, 60.5% of patients achieved
the
endpoint of at least a 25% reduction in angle of curvature vs.
25.0% of
placebo patients achieving at least a 25% reduction in angle of
curvature.
-- In the PRO Peyronie's disease bother domain, the overall
XIAFLEX
treatment arm experienced a benefit in mean change in score from
baseline
to 36 weeks that was significantly better than the overall
placebo arm
benefit (p=0.046).
-- There was not a statistically significant change in mean
scores
between XIAFLEX and placebo in the PRO penile pain, intercourse
discomfort
or intercourse constraint domains.
Results with modeling:
-- XIAFLEX treated patients who received modeling experienced a
32.4%
mean improvement in penile curvature from baseline to 36 weeks
(54.7 degrees to
37.2 degrees) vs. a 2.5% mean worsening in curvature for the
placebo patients who
received modeling (51.9 degrees to 52.5 degrees); (p < 0.001).
-- In patients who received XIAFLEX, 64.8% of patients achieved
the
endpoint of at least a 25% reduction in angle of curvature vs.
10.0% of
placebo patients achieving at least a 25% reduction in angle of
curvature.
-- In the PRO Peyronie's disease bother domain, the XIAFLEX with
modeling
treatment arm experienced a benefit in mean change in score from
baseline
to 36 weeks that was significantly better than the placebo with
modeling
arm benefit (p=0.004).
Results with no modeling:
-- XIAFLEX treated patients who received no modeling experienced
a 27.1%
mean improvement in penile curvature from baseline to 36 weeks
(54.1 degrees to
39.1 degrees), which was not statistically different from the
27.9% mean
improvement for the placebo patients who received no modeling
(48.9 degrees to
35.9 degrees); (p=0.91).
-- In patients who received XIAFLEX, 56.4% of patients achieved
the
endpoint of at least a 25% reduction in angle of curvature vs.
43.8% of
placebo patients achieving at least a 25% reduction in angle of
curvature.
-- The mean change in score from baseline to 36 weeks did not
achieve
statistical significance in any of the four domains measured by
the PRO for
the XIAFLEX treatment arm with no modeling vs. the placebo arm
with no
modeling.
-- Based on a post-hoc sub-group analysis, the Company believes
that the
results from the non-modeling placebo group may have been biased
by a
higher relative percentage of patients with a shorter duration
of disease
(i.e. less than or equal to 18 months) and that the non-modeling
placebo
group may have had more spontaneous resolutions of disease. We
believe
that this imbalance may help to explain the higher than expected
placebo
response in the no modeling arm.
Validation of Peyronie's Disease PRO
-- The Company believes that the Peyronie's Disease PRO will be
validated
by data from 134 patients (100 XIAFLEX; 34 placebo) during this
phase IIb
trial; however, final determination will require further
discussion with
the U.S. Food and Drug Administration (FDA), which is expected
to take
place at the end of phase II meeting in the second quarter of
2010.
"XIAFLEX has shown a sustained benefit at 36 weeks versus
placebo for patients suffering from Peyronie's disease," said
Laurence A. Levine, MD, FACS, investigator and Professor,
Department of Urology, Rush University Medical Center. "The drug
had a statistically significant reduction in penile curvature
and demonstrated statistically significant improved quality of
life for patients as measured by the PRO domain for Peyronie's
disease bother. Getting these efficacy results without the risks
of surgical intervention would be an enormous advantage for
patients suffering from Peyronie's disease."
Adverse Events
The most common adverse events reported in the Peyronie's phase
IIb trial were injection site bruising, edema and pain. These
adverse events were consistent with previous trials. There have
been no drug related serious adverse events or systemic
hypersensitivity events reported in this trial or any of the
previous XIAFLEX Peyronie's disease clinical studies. Including
data from all XIAFLEX Peyronie's disease clinical studies, over
1,200 XIAFLEX injections have been administered to more than 240
Peyronie's patients.
Anticipated Next Steps
In the second quarter of 2010, Auxilium plans to have an end of
phase II meeting with the FDA to discuss the validation of the
PRO, phase IIb clinical results and phase III clinical trial
design for XIAFLEX for the treatment of Peyronie's disease.
Auxilium and its European partner Pfizer plan to request a
meeting to discuss phase IIb results with European health
authorities. Auxilium is responsible for executing the global
development program of XIAFLEX for the treatment of Peyronie's
disease. XIAFLEX is partnered with Pfizer in 46 European and
Eurasian countries for the treatment of Dupuytren's contracture
and Peyronie's disease.
Conference Call
Auxilium will hold a conference call today, December 16, 2009 at
10:00 a.m. EST, to discuss the top line results of the
Peyronie's phase IIb study. The conference call will be
simultaneously web cast on Auxilium's web site and archived for
future review until January 16, 2010.
Conference call details:
Date: Wednesday, December 16, 2009
Time: 10:00 a.m. EST
Dial-in (U.S.): 866-270-6057
Dial-in (International): 617-213-8891
Web cast:
http://www.auxilium.com
Passcode: Auxilium
To access an audio replay of the call:
Access number (U.S.): 888-286-8010
Access number (International): 617-801-6888
Replay Passcode #: 80240759
About Phase IIb Peyronie's Disease Trial
The phase IIb study is a randomized, double-blind,
placebo-controlled study that is designed to assess the safety
and efficacy of XIAFLEX, when administered two times a week
every six weeks for up to three treatment cycles (2 x 3), in
subjects with Peyronie's disease. The study was conducted at 12
sites throughout the U.S., and 145 patients were monitored for
36 weeks following the first injection.
The trial was designed to measure the improvement in penile
curvature and complete the validation of the Company's
proprietary Peyronie's disease PRO, which measured four domains
of patients' sexual quality of life over a period of 36 weeks:
penile pain, Peyronie's disease bother, intercourse discomfort
and intercourse constraint.
To qualify for the study, patients must have been diagnosed with
Peyronie's disease for longer than six months, have stable
disease, be able to maintain a rigid erection and have a penile
contracture between 30 and 90 degrees. Patients were stratified
by the degree of penile curvature (i.e. 30 degrees to 60 degrees
versus 60 to 90 degrees) and then randomized into four treatment
groups to receive either XIAFLEX or placebo with or without
modeling of the penile plaque. Modeling refers to massaging of
the plaque and is intended to maximize the enzymatic effect of
the XIAFLEX injection in the plaque. Patients were randomized in
a 3:1 ratio of XIAFLEX to placebo and a 1:1 ratio to receive
penile plaque modeling or no modeling.
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 often can prevent sexual intercourse.
In addition to difficulty with sexual intercourse, Peyronie's
disease may also be associated with emotional distress, loss of
self-esteem and depression. Peyronie's disease is a very
heterogeneous disease with an initial inflammatory component of
a poorly understood physiological disease course and occasional
spontaneous resolutions of 13% (3) to 50% (2). In certain
populations, the estimated number of men affected by Peyronie's
Disease may be as high as nine percent in men over 60 years of
age and three percent in men over 30.(1)
(1) L.A. Levine Peyronie's Disease: A Guide to Clinical
Management. Humana Press: 10-17, 2007.
(2) Williams JL, Thomas GG. The natural history of Peyronie's
disease. J Urol 1970;103:75-6.
(3) Gelbard MK, Dorey F, James K. The natural history of
Peyronie's disease. J Urol 1990; 144:1376-9.
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
five projects in clinical development. XIAFLEX™ (collagenase
clostridium histolyticum), formerly referred to as AA4500, has
completed phase III clinical trials for the treatment of
Dupuytren's contracture, and the biologics license application
is under review at the FDA for the treatment of Dupuytren's
contracture. XIAFLEX is in phase IIb 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's transmucosal film product candidate for
the treatment of overactive bladder (AA4010) and its fentanyl
pain product using its transmucosal delivery system are in phase
I of development. The Company is currently seeking a partner to
further develop these product candidates. Auxilium has rights to
additional pain products and products for hormone replacement
and urologic disease using its transmucosal film delivery
system. Auxilium also has options to all indications using
XIAFLEX for non-topical formulations. 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 interpretation of data; the
benefit to Peyronie's patients of XIAFLEX; the timing of the end
of phase II meeting with FDA to discuss the validation of the
Peyronie's disease PRO, results of the phase IIb trial of
XIAFLEX for the treatment of Peyronie's disease and phase III
clinical trial design for XIAFLEX for the treatment of
Peyronie's disease; the timing of the start of phase III trials
for XIAFLEX for the treatment of Peyronie's disease; the impact
of a higher relative percentage of patients with a shorter
duration of disease on the results from the non-modeling placebo
group; the extent to which there were more spontaneous
resolutions of disease in the non-modeling placebo group; the
validation of the Peyronie's disease PRO; the plan to request a
meeting to discuss the phase IIb results with European health
authorities; the number of patients with Peyronie's disease;
products in development for Dupuytren's contracture, Peyronie's
disease, Frozen Shoulder syndrome, overactive bladder, pain,
hormone replacement and urologic disease; 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 on Form 10-K for the year ended December 31, 2008,
in Auxilium's Quarterly Report on Form 10-Q for the period ended
June 30, 2009, and in Auxilium's Quarterly Report on Form 10-Q
for the period ended September 30, 2009 under the heading "Risk
Factors", 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
or
William Q. Sargent Jr.
Vice-President, Investor Relations and Corporate Communications
+1-484-321-5900
Source
Auxilium Pharmaceuticals, Inc.
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