THE WOODLANDS, Texas,
Aug. 9, 2011 /PRNewswire/ -- Lexicon
Pharmaceuticals, Inc. (Nasdaq: LXRX), a biopharmaceutical company
focused on discovering breakthrough treatments for human disease,
announced positive, top-line proof-of-concept data from its
recently completed Phase 2 study in carcinoid syndrome with LX1032,
telotristat etiprate. Carcinoid syndrome is a chronic condition
caused by neuroendocrine tumors that usually originate from the
gastrointestinal tract. It is characterized by severe diarrhea and
flushing episodes with long-term consequences including
malnutrition, heart disease, and death. Symptoms of carcinoid
syndrome have been linked to excess production of serotonin by
metastatic tumor cells. Telotristat etiprate is designed to reduce
serotonin production.
"Telotristat etiprate is our third drug candidate to demonstrate
proof of concept in patients in Phase 2," said Dr. Arthur T. Sands, Lexicon's president and CEO.
"Based on the positive results of this U.S. study, as well as
encouraging observations from our clinical trial in Europe, we intend to discuss a Phase 3
development plan for carcinoid syndrome with the FDA."
The randomized, double-blind, placebo-controlled study was
conducted in the United States in
23 patients with carcinoid syndrome who were refractory to
currently available therapy. Patients in the study had metastatic
carcinoid disease and were experiencing an average of about six
bowel movements per day at baseline. Patients received either
placebo (n=5) or one of four doses of telotristat etiprate (n=18)
daily for 28 days. The primary endpoint of the study was safety and
tolerability. Efficacy measures included change in bowel movement
frequency, relief of symptoms, and reduction in serotonin
synthesis.
Telotristat etiprate was well tolerated, and adverse events in
the study were usually mild to moderate with similar frequencies
overall between treatment groups and placebo. Five telotristat
etiprate patients achieved clinical responses characterized by
reductions of at least 30% in the number of bowel movements per day
for two weeks or more during the study. Six telotristat etiprate
patients reported adequate relief of carcinoid symptoms at the end
of the study. There were nine telotristat etiprate patients with a
complete biochemical response defined as a reduction of at least
50% in urinary 5-HIAA, a biomarker of serotonin synthesis. No
patients on placebo experienced a clinical response, adequate
relief of symptoms, or biochemical response during the study. The
difference between telotristat etiprate and placebo in bowel
movement frequency ranged between 1.6 and 2.9 bowel movements/day
across telotristat etiprate doses, all favoring telotristat
etiprate treatment. Also of note, all eligible patients elected to
continue treatment with telotristat etiprate under an extension
protocol.
Preliminary data were also reported from a separate, ongoing,
open-label, single-arm study of telotristat etiprate in
Europe. To date, 5 out of 6
patients with refractory carcinoid syndrome have experienced
sustained reductions of at least 30% in bowel movement frequency
when treated with telotristat etiprate. Two of these responses were
within the first 4 weeks of therapy, while three more occurred
between 4 and 8 weeks of treatment under the 12-week protocol.
"The Phase 2 data clearly show a reduction in bowel movements
and symptom relief in a population that has exhausted all
standard-of-care treatment options," said Dr. Pablo Lapuerta,
senior vice president and chief medical officer at Lexicon. "The
Phase 2 data are also consistent with preliminary results from the
European study, where there appear to be some early responses and
additional benefit with continued treatment. We look forward to
presenting detailed results at a scientific meeting later this
year."
Lexicon Conference Call and Webcast:
Lexicon management will be discussing the results of this study
in conjunction with the second quarter earnings conference call
today at 1:00 p.m. Eastern Time.
The dial-in number for the conference call is 888-220-1244
(within the US/Canada) or
706-679-5615 (international). The conference ID for all
callers is 89449241. A live webcast of the call can be
accessed at www.lexpharma.com. An archived version of the
webcast will be available on Lexicon's corporate website through
September 6, 2011.
About Telotristat Etiprate (LX1032)
Telotristat etiprate was discovered and developed at Lexicon to
reduce serotonin production by inhibiting tryptophan hydroxylase
(TPH), a key enzyme in the synthesis of serotonin. Excessive
levels of serotonin have been implicated in symptoms associated
with carcinoid syndrome, especially diarrhea and carcinoid heart
disease. Serotonin's breakdown product, 5-HIAA, is a
biomarker used in the diagnosis of the condition. In
preclinical studies, telotristat etiprate reduced peripheral
serotonin in several different species without affecting serotonin
levels in the brain. In Phase 1 clinical studies, telotristat
etiprate reduced serotonin levels and urinary 5-HIAA in healthy
volunteers consistent with preclinical results. Telotristat
etiprate is being developed under Fast Track designation from the
U.S. Food and Drug Administration and orphan drug designation from
the European Medicines Agency. Telotristat etiprate is a member of
a new class of oral drugs invented by Lexicon, the serotonin
synthesis inhibitors, which are being developed in a spectrum of
gastrointestinal indications. Lexicon is also currently
planning a Phase 2 trial of telotristat etiprate in mild to
moderate ulcerative colitis.
About Carcinoid Syndrome
Carcinoid syndrome is a chronic condition caused by metastatic
neuroendocrine tumors that usually originate from the
gastrointestinal tract. These tumors secrete large amounts of
serotonin, which can cause a variety of symptoms including severe
diarrhea and abdominal discomfort. Patients with carcinoid
syndrome currently have limited therapeutic options, and the
standard of care includes chronic therapy with somatostatin
analogues, which are delivered by injection. With current
therapy, the gastrointestinal symptoms return over time in most
patients, hence the need for new agents.
About Lexicon
Lexicon is a biopharmaceutical company focused on discovering
breakthrough treatments for human disease. Lexicon currently
has four drug candidates in mid-stage development for diabetes,
irritable bowel syndrome, carcinoid syndrome and rheumatoid
arthritis, all of which were discovered by Lexicon's research team.
Lexicon has used its proprietary gene knockout technology to
identify more than 100 promising drug targets. Lexicon has
focused drug discovery efforts on these biologically-validated
targets to create its broad pipeline of clinical and preclinical
programs. For additional information about Lexicon and its
programs, please visit www.lexpharma.com.
Safe Harbor Statement
This press release contains "forward-looking statements,"
including statements relating to Lexicon's clinical development
of LX1032 (telotristat etiprate), including
characterizations of the results of and projected timing of
clinical trials, and the potential therapeutic and commercial
potential of LX1032. This press release also contains
forward-looking statements relating to Lexicon's growth and future
operating results, discovery and development of products, strategic
alliances and intellectual property, as well as other matters that
are not historical facts or information. All forward-looking
statements are based on management's current assumptions and
expectations and involve risks, uncertainties and other important
factors, specifically including those relating to Lexicon's
ability to successfully conduct clinical development of LX1032 and
preclinical and clinical development of its other potential drug
candidates, advance additional candidates into preclinical and
clinical development, obtain necessary regulatory approvals,
achieve its operational objectives, obtain patent protection for
its discoveries and establish strategic alliances, as well as
additional factors relating to manufacturing, intellectual property
rights, and the therapeutic or commercial value of its drug
candidates, that may cause Lexicon's actual results to be
materially different from any future results expressed or implied
by such forward-looking statements. Unless specifically
indicated otherwise, results reported as trends were not
statistically significant. Information identifying such important
factors is contained under "Risk Factors" in Lexicon's annual
report on Form 10-K for the year ended December 31, 2010, as filed with the Securities
and Exchange Commission. Lexicon undertakes no obligation to
update or revise any such forward-looking statements, whether as a
result of new information, future events or otherwise.
SOURCE Lexicon Pharmaceuticals, Inc.