Synergy Pharmaceuticals Presents Long-Term Data for Plecanatide in Chronic Idiopathic Constipation at the American College of Gastroenterology (ACG) Annual Scientific Meeting

NEW YORK–(BUSINESS WIRE)– Synergy Pharmaceuticals Inc. (NASDAQ:SGYP) today presented new long-term
safety data of plecanatide, its investigational, orally-administered
compound currently being evaluated by the U.S. Food and Drug
Administration (FDA) for the treatment of chronic idiopathic
constipation (CIC) and irritable bowel syndrome with constipation
(IBS-C). These data, which were presented at the American College of
Gastroenterology (ACG) annual scientific meeting, showed that
plecanatide was associated with low adverse events and low
discontinuation rates in patients with CIC who received plecanatide (3
mg or 6 mg) once-daily for up to 72 weeks.

Plecanatide is the first investigational therapy designed to replicate
the activity of uroguanylin, a naturally occurring human
gastrointestinal (GI) peptide, by working locally in the proximal small
intestine to stimulate digestive fluid movement and support regular
bowel function.

In this long-term study, which evaluated 2,370 patients, the most common
adverse events in both dose groups were diarrhea (7.1%) and urinary
tract infection (2.2%). The remainder of adverse events occurred in less
than 2% of patients treated with plecanatide. Adverse events leading to
discontinuation occurred in 5.3% of patients treated with plecanatide,
with discontinuation due to diarrhea occurring in 3.1% of patients. In
addition, this study asked patients about level of treatment
satisfaction and desire to continue treatment. The median score for
treatment satisfaction was 4.0 (4=quite satisfied) and for continuation
of treatment was 4.0 (4=quite likely).

“Chronic idiopathic constipation is a complex and often debilitating
condition, and there remains an unmet need for new, effective therapies
for the millions of Americans who may not have found a treatment option
with which they are satisfied,” said Satish S.C. Rao, M.D., PhD,
Professor of Medicine, Division Chief Fellowship Program Director and
Director, Digestive Health Center at Augusta University. “The data
presented today at ACG provide additional information regarding the
Phase 3 efficacy and safety clinical trials and support the long-term
safety of plecanatide, reinforcing the potential of plecanatide as a new
treatment option for this condition.”

Synergy also presented plecanatide data from an integrated efficacy and
safety analysis which are consistent with findings of two previously
presented double-blind, placebo-controlled Phase 3 trials that evaluated
more than 2,600 patients with CIC over a 12-week treatment period. These
additional analyses confirmed a significantly greater response rate of
durable overall complete spontaneous bowel movements (CSBM) — a primary
endpoint defined by the FDA for regulatory approval in CIC—in each of
the two plecanatide dose groups (3 mg, 20.5%; 6 mg, 19.8%) when compared
to the placebo group (11.5%, p<0.001 for both doses). These significant
increases in CSBMs with both plecanatide dose groups were seen as early
as the first week and maintained through the treatment period compared
with placebo. Results at 12 weeks also found:

• Secondary endpoints (stool consistency, straining and bloating) were
  significantly improved compared to placebo.
• Adverse event rates were similar across plecanatide-treatment groups
  and placebo (30.6% in 3 mg and 31.1% in 6 mg dose groups compared to
  28.7% in placebo), Diarrhea was the most common adverse event (4.6% in
  3 mg and 5.1% in 6 mg compared to 1.3% in placebo).
• Discontinuation rates were low across all treatment groups (3 mg,
  4.1%; 6 mg, 4.5%; and placebo, 2.2%).

“Synergy has pioneered a different way to think about constipation,
which is reflected in our approach to plecanatide,” said Patrick H.
Griffin, MD, FACP, Executive Vice President and Chief Medical Officer of
Synergy Pharmaceuticals. “We are excited to bring these new data to
gastroenterologists at ACG. We are committed to developing and bringing
to market novel therapies that offer the potential to significantly
improve the lives of patients living with CIC and other GI conditions.”

The Prescription Drug User Fee Act (PDUFA) target action date for
plecanatide in CIC is January 29, 2017.

In addition, Synergy has already completed patient recruitment for the
two double-blind, placebo-controlled Phase 3 clinical trials with
plecanatide in IBS-C and remains on-track to report top-line data from
both trials in the fourth quarter of this year. Pending approval in the
CIC indication, Synergy plans to file a New Drug Application Supplement
with Clinical Data (sNDA) for plecanatide in IBS-C in the first quarter
of 2017 and expects a 10-month review period from submission.

About Chronic Idiopathic Constipation (CIC)

CIC affects 14 percent of the population in North America,
disproportionately affecting women and older adults. People with CIC
have persistent symptoms of difficult and infrequent bowel movements.
CIC can severely impact people’s daily lives, increasing stress levels
and anxiety.

About Plecanatide

Plecanatide is a peptide made up of 16 amino acids and, with the
exception of a single amino acid substitution, it is identical to
uroguanylin. Plecanatide is the first investigational drug designed to
replicate the function of uroguanylin, a naturally occurring and
endogenous human GI peptide which acts in a pH-dependent manner
targeting GC-C receptors primarily in the proximal small intestine.
Plecanatide stimulates fluid secretion and promotes stool consistency
necessary to support normal bowel function.

About Synergy Pharmaceuticals

Synergy is a biopharmaceutical company focused on the development and
commercialization of novel GI therapies. The company has pioneered
discovery, research and development efforts around uroguanylin analogs
for the treatment of functional GI disorders and inflammatory bowel
disease. Synergy’s proprietary uroguanylin analog technology platform
includes two lead product candidates – plecanatide and dolcanatide. For
more information, please visit www.synergypharma.com.

Forward-Looking Statement

Certain statements in this press release are forward-looking within the
meaning of the Private Securities Litigation Reform Act of 1995. These
statements may be identified by the use of forward- looking words such
as « anticipate, » « planned, » « believe, » « forecast, » « estimated, »
« expected, » and « intend, » among others. These forward-looking statements
are based on Synergy’s current expectations and actual results could
differ materially. There are a number of factors that could cause actual
events to differ materially from those indicated by such forward-looking
statements. These factors include, but are not limited to, substantial
competition; our ability to continue as a going concern; our need for
additional financing; uncertainties of patent protection and litigation;
uncertainties of government or third party payer reimbursement; limited
sales and marketing efforts and dependence upon third parties; and risks
related to failure to obtain FDA clearances or approvals and
noncompliance with FDA regulations. As with any pharmaceutical under
development, there are significant risks in the development, regulatory
approval and commercialization of new products. There are no guarantees
that future clinical trials discussed in this press release will be
completed or successful or that any product will receive regulatory
approval for any indication or prove to be commercially successful.
Investors should read the risk factors set forth in Synergy’s Form 10-K
for the year ended December 31, 2015 and other periodic reports filed
with the Securities and Exchange Commission. While the list of factors
presented here is considered representative, no such list should be
considered to be a complete statement of all potential risks and
uncertainties. Unlisted factors may present significant additional
obstacles to the realization of forward-looking statements.
Forward-looking statements included herein are made as of the date
hereof, and Synergy does not undertake any obligation to update publicly
such statements to reflect subsequent events or circumstances.

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