THE WOODLANDS, Texas,
Aug. 3, 2015 /PRNewswire/
-- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today
announced that the pivotal TELESTAR Phase 3 clinical trial met its
primary endpoint, showing the benefit of oral telotristat etiprate
in treating cancer patients with carcinoid syndrome that is not
adequately controlled by the current standard of care. Telotristat
etiprate was discovered using Lexicon's gene science, based on
Nobel Prize-winning technology, and is the company's first
discovery to complete a pivotal Phase 3 clinical trial. If
approved, telotristat etiprate would be the first oral treatment
successfully developed for carcinoid syndrome and the first
addition to the standard of care in more than 16 years[1].
Top-line results from the Phase 3 study show that patients who
added telotristat etiprate to the standard of care at both the 250
mg and 500 mg doses experienced a statistically significant
reduction from baseline compared to placebo in the average number
of daily bowel movements over the 12-week study period
(p<0.001), meeting the study's primary endpoint.
"We are extremely pleased with these top-line results," said
Lexicon President and Chief Executive Officer Lonnel Coats. "Carcinoid syndrome is severely
debilitating, preventing many patients from leading active and
predictable lives, and unfortunately, a majority of patients will
not be adequately controlled over time with the current standard of
care. We are committed to working closely with the FDA to file our
first new drug application (NDA) and to bring this innovative new
treatment to patients whose lives are already impacted by the
challenges of cancer."
"The TELESTAR results are promising, and the community of
patients and caregivers who live and deal with carcinoid syndrome
are excited about the prospect of a new treatment becoming
available," said principal investigator Matthew H. Kulke, M.D., Director, Program in
Neuroendocrine and Carcinoid Tumors and Senior Physician, Dana
Farber Cancer Institute, and Associate Professor of Medicine,
Harvard Medical School.
Lexicon received Fast Track designation and Orphan Drug status
for telotristat etiprate from the U.S. Food and Drug Administration
(FDA) in 2008 and 2012, respectively. The company plans to announce
complete results from the Phase 3 TELESTAR study at an upcoming
scientific conference.
About Carcinoid Syndrome
Carcinoid syndrome is a rare disease affecting thousands of
patients with neuroendocrine tumors that originate in the
gastrointestinal tract and metastasize or spread to the liver or
other organs. Overproduction of serotonin within these metastatic
neuroendocrine tumor (mNET) cells is a driver of carcinoid
syndrome, which is characterized by debilitating diarrhea, facial
flushing, abdominal pain, heart valve damage and other serious
consequences. The severe and unpredictable diarrhea
associated with carcinoid syndrome has a profound impact on cancer
patients' lives, often preventing them from participating in daily
activities.
The current standard of care for carcinoid syndrome is
somatostatin analog depot injection (SSA), first approved in 1998.
SSA therapy fails to maintain adequate control of carcinoid
syndrome for most patients, with many becoming not adequately
controlled within the first two years after the therapy is
initiated. Patients with carcinoid syndrome can live for many
years with metastatic cancer, requiring the need for long-term
treatment options to effectively manage their disease[2].
About TELESTAR
The double-blind Phase 3 study known as TELESTAR (Telotristat
Etiprate for Somatostatin Analogue Not Adequately Controlled
Carcinoid Syndrome) enrolled 135 patients with carcinoid syndrome
which was not adequately controlled on SSA therapy, the current
standard of care. The three-arm study evaluated two doses of oral
telotristat etiprate – 250 mg and 500 mg, each taken three times
daily – against placebo over a 12-week period and measured the
reduction from baseline in the average number of daily bowel
movements. Patients in both the treatment and placebo arms
continued their SSA therapy throughout the study.
Top-line results from TELESTAR show that patients who added
telotristat etiprate to the standard of care at both the 250 mg and
500 mg doses experienced a statistically significant reduction from
baseline compared to placebo in the average number of daily bowel
movements over the 12-week study period (p<0.001), meeting the
study's primary endpoint.
In a key secondary measure, a substantially greater proportion
of patients on telotristat etiprate achieved a durable response (44
percent and 42 percent in the 250 mg and 500 mg arms,
respectively), defined as at least a 30 percent reduction in daily
bowel movements over at least half the days of the study period, as
compared to 20 percent response on placebo (p<0.040).
Patients who received 250 mg of telotristat etiprate experienced
a 29 percent reduction in the average number of daily bowel
movements during the final week (week 12) of the study period
compared to baseline, and those in the 500 mg arm experienced a 35
percent reduction, while the placebo group showed a 17 percent
reduction. These results are consistent with those seen in the
12-week Phase 2 study of telotristat etiprate.
The proportion of patients with treatment-emergent adverse
events (AEs), serious AEs and discontinuation due to AEs were
generally similar in all three treatment arms. The tolerability
profile of the telotristat etiprate 250 mg dose appeared similar to
placebo and somewhat better than the 500 mg dose with respect to
gastrointestinal discomfort and mood. The overall incidence and
nature of AEs in TELESTAR was consistent with those reported in
previous studies. Further in depth analysis of safety and
tolerability data will be conducted.
The 12-week study period is being followed by a 36-week
open-label extension where all patients receive telotristat
etiprate 500 mg three times daily.
About Telotristat Etiprate
Discovered using Lexicon's unique approach to gene science,
telotristat etiprate is the first investigational drug in clinical
studies to target tryptophan hydroxylase (TPH), an enzyme that
triggers the excess serotonin production within mNET cells that
leads to carcinoid syndrome. While existing treatments for
carcinoid syndrome work to reduce the release of serotonin outside
tumor cells, telotristat etiprate works at the source to reduce
serotonin production within the tumor cells. By specifically
inhibiting serotonin production, telotristat etiprate seeks to
control this important driver of carcinoid syndrome and, in turn,
provide patients with more control over their disease.
Lexicon retains rights to market telotristat etiprate in the
U.S. and Japan, and is building
the in-house commercial infrastructure to serve the U.S. market.
The company has a license and collaboration agreement with Ipsen to
commercialize telotristat etiprate in Europe and other countries outside the U.S.
and Japan.
Lexicon Conference Call
Lexicon management will hold a conference call and webcast to
discuss the TELESTAR Phase 3 top-line results at 8:00 a.m. Eastern Time on August 3, 2015. The dial-in number for the
conference call is 888-645-5785 (within the US/Canada) or 970-300-1531 (international).
The conference ID for all callers is 37172080.
Investors can access a live webcast of the call at
www.lexpharma.com. An archived version of the webcast will be
available on the website through September
2, 2015.
About Lexicon
Lexicon is a fully integrated biopharmaceutical company that is
applying a unique approach to gene science based on Nobel
Prize-winning technology to discover and develop precise medicines
for patients with serious, chronic conditions. Through its
Genome5000™ program, Lexicon scientists have studied the role and
function of nearly 5,000 genes over the last 20 years and have
identified more than 100 protein targets with significant
therapeutic potential in a range of diseases. Through the precise
targeting of these proteins, Lexicon is pioneering the discovery
and development of innovative medicines to safely and effectively
treat disease. Lexicon has a pipeline of promising drug candidates
in clinical and pre-clinical development in oncology, diabetes and
metabolism. For additional information 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 (LX1032), including characterizations of the
results of and projected timing of clinical trials and the
potential therapeutic and commercial potential of telotristat
etiprate. In addition, 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 meet its capital requirements, successfully conduct clinical
development of telotristat etiprate and preclinical and clinical
development of its other potential drug candidates, 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, 2014, 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.
[1] Octreotide acetate for injectable suspension was approved by
FDA for the treatment of carcinoid syndrome in 1998.
[2] Shebani KO, Souba WW, Finkelstein DM, et al. (1999).
Prognosis and Survival in Patients With Gastrointestinal Tract
Carcinoid Tumors. Annals of Surgery. 1999;229(6):815.
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SOURCE Lexicon Pharmaceuticals, Inc.