- Efficacy observed with both 800 mg
once-daily and 400 mg twice-daily XP23829 dosing
- XP23829 safe and generally
well-tolerated with low incidence of flushing similar to
placebo
- Low incidence/severity of
lymphopenia seen with XP23829
- First clinical demonstration of
efficacy of a MMF prodrug other than DMF
XenoPort, Inc. (Nasdaq:XNPT) announced today positive
preliminary top-line results from its Phase 2 clinical trial of
XP23829 as a potential treatment for moderate-to-severe chronic
plaque-type psoriasis. XP23829 met its primary endpoint in both 800
mg once daily and 400 mg twice daily doses, demonstrating
statistically significant improvements in percent change from
baseline to week 12 in Psoriasis Area and Severity Index (PASI)
score. XP23829 is a patented prodrug of monomethyl fumarate (MMF)
in a novel oral formulation that was designed to potentially offer
physicians and patients an effective, better tolerated and easier
to use therapeutic option to currently available fumarate
products.
Richard Kim, M.D., chief medical officer of XenoPort, stated,
"We believe these clinical data demonstrate for the first time that
a MMF prodrug other than dimethyl fumarate (DMF) can be effective
in reducing lesions in psoriatic patients. The magnitude of
XP23829’s effect on the primary efficacy endpoint met our
expectations for this relatively short duration trial and we are
particularly encouraged by the results with 800 mg once-daily
dosing. Based on what is known about fumarates, we believe that the
efficacy of XP23829 is likely to improve with a more extended
duration of treatment beyond 12 weeks. We are also pleased with the
safety and tolerability profile of XP23829 emerging from this
study. We believe that this demonstration of efficacy, safety and
tolerability of XP23829 could lead to a differentiated product in
psoriasis. We also believe that there is potential for the
observations from this study to read through to other potential
indications such as multiple sclerosis (MS).”
Description of the Trial
This randomized, double-blind, placebo-controlled Phase 2
clinical trial of XP23829 was conducted in 33 sites in the United
States in subjects with moderate-to-severe chronic plaque-type
psoriasis. Two hundred eligible subjects were randomized to placebo
or one of three treatment arms of XP23829: 400 mg or 800 mg once
daily (QD) or 400 mg twice daily (BID). The 12-week treatment
period included a three-week titration period followed by nine
weeks of treatment at the targeted dose. There was a washout phase
of up to four weeks prior to randomization for subjects who were
previously taking systemic agents for the treatment of psoriasis.
Treatment assignment was stratified based on prior biologic use and
approximately 35% of randomized subjects had previous experience
with biological treatments for their psoriasis.
The primary endpoint of the study was the percent change from
baseline to week 12 in PASI score. Least squares mean percent
reductions in PASI score at 12 weeks were as follows:
400 mg QD
800 mg QD 400 mg BID Placebo
Least squares mean -38.1 -48.2
-50.7 -25.0 p-value 0.066
0.001 <0.001
XP23829 was safe and generally well tolerated. There were no
deaths or life-threatening adverse events. No subjects met the
safety discontinuation criteria and the majority of treatment
emergent adverse events were non-serious and mild or moderate in
severity. Diarrhea adverse event rates were consistent with other
drugs in the fumaric acid ester class ranging from 22% to 40% in
the XP23829 treatment groups compared with 15% for placebo. Other
treatment emergent adverse events occurring at an incident rate of
greater than or equal to 10% were nausea, abdominal pain, vomiting
and headache. The incidence of flushing in the XP23829 dose groups
was similar to placebo. Gastrointestinal events were the most
frequent adverse event leading to withdrawal during XP23829
treatment. There were two treatment emergent serious adverse events
assessed as possibly related to treatment with XP23829: acute
cholecystitis and enterocolitis. Both subjects recovered.
No subjects experienced Grade 3 or Grade 4 lymphopenia. Less
than 5% of subjects in any XP23829 treatment group reached Grade 2
lymphopenia and less than 15% reached Grade 1 at any visit.
Lymphocyte levels in all subjects experiencing lymphopenia returned
to within normal limits after treatment.
“I’m excited to have participated in this phase 2 study with
positive efficacy data that we believe justifies further
development of XP23829 into moderate-to-severe chronic plaque-type
psoriasis,” stated Alice Bendix Gottlieb, M.D., Ph.D.,
Dermatologist-in-Chief; Harvey B. Ansell Professor of Dermatology,
Tufts University School of Medicine and Lead Investigator for the
XP23829 trial. “Despite the wide availability of biologics, there
still remains a significant unmet medical need for a more
effective, safe, well-tolerated, and convenient oral treatment for
patients with psoriasis. Fumarates have been the leading treatment
for psoriasis in Germany for more than 2 decades. With this
long-term real-world fumarate experience and these data in
consideration, I look forward to the potential of seeing XP23829 in
Phase 3 development for moderate-to-severe chronic plaque-type
psoriasis.”
“We are extremely pleased by the preliminary top-line results
from this Phase 2 study. I want to thank the clinical investigators
and the psoriasis sufferers who participated in the study. We look
forward to getting the complete data set for this trial later this
month and to presenting more comprehensive results at future
medical conferences and in publications,” stated Ronald W. Barrett,
Ph.D., chief executive officer of XenoPort.
Dr. Barrett continued, “In the near future, we intend to share
these data with psoriasis and multiple sclerosis experts, speak
with regulatory authorities regarding next steps and explore
potential partnerships that could accelerate the development of
XP23829 globally. We recently completed non-clinical development
studies and manufacturing activities necessary to support Phase 3
development and we believe we will be ready to potentially initiate
Phase 3 studies in 2016."
Conference Call
XenoPort will host a webcast presentation today at 8:30 a.m.
Eastern Time to discuss the preliminary top-line XP23829 Phase 2
clinical trial results. The live presentation may be accessed via
the Calendar of Events page in the Investors section of the
XenoPort website at www.XenoPort.com. A replay of the presentation
will be available via the Internet for one month following the live
presentation and can be accessed after 1:30 p.m. Eastern Time
today.
The presentation may be accessed via phone by dialing
1-888-275-3514. International callers may access the live call by
dialing 706-679-1417. The reference number to enter the call is
41274020. The replay of the call will be available for one week and
may be accessed after 11:30 a.m. Eastern Time today via phone at
1-855-859-2056 for domestic callers, or 404-537-3406 for
international callers. The reference number to enter the replay of
the call is 41274020.
About XP23829
XP23829, an investigational drug discovered and currently under
development by XenoPort, is a fumaric acid ester compound that is a
prodrug of MMF. Fumaric acid ester compounds have shown
immuno-modulatory and neuroprotective effects in cell-based systems
and preclinical models of disease. TECFIDERA, which is approved for
relapsing forms of MS in the United States and relapsing-remitting
MS in the European Union and FUMADERM, which is approved in Germany
for psoriasis, are based on another MMF prodrug known as DMF.
XP23829 is protected by a U.S. composition-of-matter patent that
currently has an expiration date of 2029. In addition XenoPort
holds 48 issued patents worldwide, including those providing
coverage for XP23829 and other MMF prodrug compositions and their
uses, crystalline forms of XP23829, methods of treatment with MMF
prodrugs and oral dosage forms.
About Psoriasis
Psoriasis is a chronic, systemic, inflammatory disease that
manifests in the skin and/or joints. It typically manifests as
thick scaling red plaques, with variable morphology and
distribution, resulting from an unusually high rate of skin cell
growth. There is no cure for psoriasis, and treatment often
requires complex medical intervention. The main cause of psoriasis
is uncertain, but it is thought to be caused by autoimmunity,
genetic predisposition and environmental factors.
Psoriasis is the most prevalent autoimmune disease in the United
States with as many as 7.5 million Americans suffering from the
condition. It is estimated that approximately 1.5 million adults in
the United States are considered to have moderate-to-severe
psoriasis and between 150,000 and 260,000 new cases of psoriasis
are diagnosed each year.
About XenoPort
XenoPort, Inc. is a biopharmaceutical company focused on
developing and commercializing a portfolio of internally discovered
product candidates for the potential treatment of neurological
disorders. XenoPort is currently commercializing HORIZANT®
(gabapentin enacarbil) Extended-Release Tablets in the United
States and developing its novel fumaric acid ester product
candidate, XP23829, as a potential treatment for patients with
moderate-to-severe chronic plaque-type psoriasis and potentially
for relapsing forms of multiple sclerosis. REGNITE® (gabapentin
enacarbil) Extended-Release Tablets is being marketed in Japan by
Astellas Pharma Inc. XenoPort has entered into a clinical trial
agreement with the National Institute on Alcohol Abuse and
Alcoholism (NIAAA) under which the NIAAA has initiated a clinical
trial evaluating gabapentin enacarbil as a potential treatment for
alcohol use disorder, and XenoPort has granted exclusive world-wide
rights for the development and commercialization of its
clinical-stage oral product candidate, arbaclofen placarbil, to
Indivior PLC for all indications. XenoPort's pipeline of product
candidates also includes a potential treatment for patients with
idiopathic Parkinson's disease.
To learn more about XenoPort, please visit the website at
www.XenoPort.com.
Forward-Looking Statements
This press release contains “forward-looking” statements,
including, without limitation, all statements related to furthering
the development of XP23829, including XenoPort’s belief that it
will be ready to initiate potential Phase 3 studies of XP23829 in
2016; the suitability of XP23829 as a potential treatment for
moderate-to-severe chronic plaque-type psoriasis and/or relapsing
forms of MS; XenoPort’s belief that the efficacy of XP23829 is
likely to improve with a more extended duration of treatment beyond
12 weeks; XenoPort’s intent to speak with regulatory authorities
regarding next steps for XP23829 and to explore potential
partnerships that could accelerate the development of XP23829
globally; and the therapeutic and commercial potential of XP23829,
including XenoPort’s beliefs that XP23829 could lead to a
differentiated product in psoriasis and that there is potential for
the observations from the Phase 2 clinical trial to read through to
other potential indications such as MS. Any statements contained in
this press release that are not statements of historical fact may
be deemed to be forward-looking statements. Words such as
“believe,” “can,” “could,” “intend,” “forward,” “further,”
“likely,” “possibly,” “potential,” “will” and similar expressions
are intended to identify forward-looking statements. These
forward-looking statements are based upon XenoPort's current
expectations. Forward-looking statements involve risks and
uncertainties. XenoPort's actual results and the timing of events
could differ materially from those anticipated in such
forward-looking statements as a result of these risks and
uncertainties, which include, without limitation, the difficulty
and uncertainty of pharmaceutical product development and the
uncertain results and timing of clinical trials and other studies,
including the risk that success in preclinical testing and early
clinical trials do not ensure that later clinical trials, such as
Phase 3 clinical trials, will be successful, and that the results
of clinical trials by other parties may not be indicative of the
results in trials that XenoPort may conduct; the risk that the
final data from the Phase 2 clinical trial of XP23829 may be
materially different from the preliminary data that XenoPort has
reported, particularly as additional and updated patient data
becomes available; XenoPort’s ability to successfully advance
XP23829 development and to conduct clinical trials in the
anticipated timeframes, or at all; the risk that the completion of
clinical trials for XP23829 may be delayed or terminated as a
result of many factors, including delays in patient enrollment; the
risk that XenoPort’s belief regarding potential distinguishing
attributes of XP23829 may not be observed or validated in future
Phase 3 or other clinical testing; that XP23829 will require
significant additional clinical testing prior to any possible
regulatory approvals and failure could occur at any stage of its
development; the uncertainty of the FDA’s review process and other
regulatory requirements that must be satisfied in order to further
XP23829 development; the uncertainty of protecting and expanding
XenoPort’s intellectual property rights, including the risk that
patent rights may not provide XenoPort with sufficient protection
against competitive products or otherwise cover commercially
valuable products or processes; XenoPort’s dependence on potential
future collaborative partners; the availability of resources to
develop XP23829 and to support XenoPort’s operations; and the
uncertain therapeutic and commercial value of XP23829. These and
other risk factors are discussed under the heading “Risk Factors”
in XenoPort’s Quarterly Report on Form 10-Q for the quarter ended
June 30, 2015 filed with the Securities and Exchange Commission on
August 6, 2015. XenoPort expressly disclaims any obligation or
undertaking to release publicly any updates or revisions to any
forward-looking statements contained herein to reflect any change
in the company's expectations with regard thereto or any change in
events, conditions or circumstances on which any such statements
are based.
HORIZANT, REGNITE and XENOPORT are registered trademarks of
XenoPort, Inc.
Source code: XNPT2C
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version on businesswire.com: http://www.businesswire.com/news/home/20150915005703/en/
XenoPort, Inc.Jackie Cossmon, 408-616-7220ir@XenoPort.com
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