THE WOODLANDS, Texas,
Oct. 9, 2013 /PRNewswire/ -- Lexicon
Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced top-line
results from a pilot study exploring the use of telotristat
etiprate in ulcerative colitis. Telotristat etiprate, a serotonin
synthesis inhibitor, is currently in Phase 3 development for
carcinoid syndrome, a condition caused by metastatic, functioning
neuroendocrine tumors. Elevated serotonin is a hallmark of
carcinoid syndrome, and two previous Phase 2 clinical trials have
shown that clinical benefits of telotristat etiprate treatment were
associated with significant reductions in 5-HIAA, a biomarker of
serotonin production. Genetic and pharmacological studies in
preclinical models had also suggested that reducing serotonin
levels might benefit patients with other forms of gastrointestinal
disease such as ulcerative colitis.
In this pilot study of mild to moderate ulcerative colitis,
telotristat etiprate achieved the primary objective of
demonstrating safety and tolerability. Adverse events and
discontinuations were evenly distributed between treatment and
placebo and there were no serious adverse events attributed to the
investigational drug. In addition, telotristat etiprate showed
dose-dependent, statistically significant reductions in 5-HIAA from
baseline compared to placebo (p < 0.001), with a relationship
between the biomarker and certain measures of clinical benefit
being observed in the high dose group. Although these results
provide a clear signal of activity of the mechanism of action of
telotristat etiprate in this patient population, they were not
accompanied by other findings that would indicate a large impact on
disease modification.
"The top-line results clearly support a favorable safety profile
of telotristat etiprate and, importantly, will contribute to our
overall safety database for this investigational, orphan drug,"
said Pablo Lapuerta, M.D., Lexicon's
chief medical officer. "The reductions in serotonin biosynthesis in
this study are consistent with beneficial gastrointestinal effects
we have observed in other settings; however, the results from this
limited trial do not provide compelling evidence of an effect on
the underlying pathogenesis of ulcerative colitis, a standard we
require to move forward in this indication. We plan,
therefore, to maintain the focus of Lexicon resources on the
ongoing Phase 3 program in carcinoid syndrome as the primary
indication for telotristat etiprate."
In this multicenter study, 59 patients with mild-moderate
ulcerative colitis were randomized to placebo, a 500 mg dose of
investigational drug telotristat etiprate taken orally once daily,
or a 500 mg dose of telotristat etiprate three times daily.
Treatment was given for 8 weeks. In the context of a pilot study,
additional analyses will be conducted to best understand the
clinical significance of the results, which are planned for
presentation at a scientific congress in 2014.
About Lexicon
Lexicon is a biopharmaceutical company focused on discovering
breakthrough treatments for human disease. Lexicon currently
has multiple programs in clinical development for diabetes,
irritable bowel syndrome, carcinoid syndrome and other indications,
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 extensive 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 telotristat etiprate, characterizations of the
results of and projected timing of clinical trials of telotristat
etiprate, and the potential therapeutic and commercial potential of
telotristat etiprate. The 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 telotristat
etiprate 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.
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, 2012, 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.