Concert Pharmaceuticals Presents CTP-656 Phase 1 Results at European Cystic Fibrosis Conference

LEXINGTON, Mass.–(BUSINESS WIRE)–Concert
Pharmaceuticals, Inc. (NASDAQ: CNCE) today presented results from a
Phase 1 multiple ascending dose trial of CTP-656, a next generation CFTR
potentiator being developed for the treatment of cystic fibrosis. The
Phase 1 healthy volunteer trial included a single dose tablet crossover
comparison with Kalydeco®, the current standard of care for certain
gating mutations of cystic fibrosis, showing that CTP-656 provided
substantially superior key exposure parameters. The Phase 1 results
support the development of CTP-656 as a once-daily potentiator for the
treatment of cystic fibrosis. The results were presented by Concert
during an oral presentation at the European Cystic Fibrosis Conference
being held in Basel, Switzerland.
“We are impressed with the pharmacokinetic profile of CTP-656 that has
emerged from our initial clinical evaluation. Importantly, we believe
this profile supports simplified dosing, with the potential for improved
efficacy,” said James Cassella, Ph.D., Chief Development Officer of
Concert Pharmaceuticals. “We are committed to opening the
Investigational New Drug Application by year-end and advancing CTP-656
into its Phase 2 efficacy study.”
The multiple ascending dose Phase 1 trial was conducted in two parts and
enrolled 38 healthy volunteers to assess safety, tolerability and
pharmacokinetics of CTP-656 in a tablet formulation. The first
part assessed a single dose pharmacokinetic comparison of 150 mg of
CTP-656 versus 150 mg of Kalydeco. The second part assessed three doses
of CTP-656, 75 mg, 150 mg and 225 mg, dosed daily for seven days
compared to placebo.
In part one, the results from the single dose crossover study
demonstrated that 150 mg of CTP-656 provided a superior pharmacokinetic
profile compared to 150 mg of Kalydeco. The key exposure parameters of C₂₄
and AUC were approximately three-fold greater with CTP-656
compared to Kalydeco. In addition, a reduced rate of clearance with
CTP-656 was observed. The half-life of 150 mg of CTP-656 was
approximately 40% longer than 150 mg of Kalydeco. These results confirm
the findings from Concert’s first direct comparison with Kalydeco in the
previously reported Phase 1 single ascending dose trial.
In part two, at steady state, CTP-656 maintained its superior
pharmacokinetic profile with greater exposure to the more potent parent
drug than to less active metabolites. CTP-656 showed a dose-proportional
increase in exposure with repeated dosing for the 150 mg dose relative
to the 75 mg dose. Steady state with CTP-656 was achieved after 3 days
of dosing. Results of the Phase 1 trial also showed that CTP-656 was
well-tolerated and its safety profile was comparable to that of
Kalydeco. No serious adverse events were reported.
A copy of the presentation is available online at: https://www.concertpharma.com/technology-overview/presentations/.
Concert plans to conduct a single Phase 2 clinical trial with CTP-656 in
cystic fibrosis patients with gating mutations. The Company expects to
open an Investigational New Drug Application (IND) to support the Phase
2 trial in the fourth quarter of 2016. Topline results from the Phase 2
trial are expected in the second half of 2017.
About CTP-656 and Cystic Fibrosis
CTP-656 is a novel
potentiator that may enable once-daily dosing that was developed by
applying deuterium chemistry to modify ivacaftor. Concert is initially
developing CTP-656 as a potential monotherapy treatment for cystic
fibrosis due to gating mutations of the gene that encodes for cystic
fibrosis transmembrane conductance regulator (CFTR), a protein, which
regulates components of sweat, mucus clearance and digestion. Cystic
fibrosis is a life-threatening, hereditary genetic disease that has
systemic effects and can cause significantly reduced lung and digestive
system function. According to the Cystic Fibrosis Foundation, an
estimated 70,000 people worldwide have cystic fibrosis.
About Concert
Concert
Pharmaceuticals is a clinical stage biopharmaceutical company
focused on applying its DCE
Platform® (deuterated chemical entity platform) to create novel
small molecule drugs. This approach starts with approved drugs, advanced
clinical candidates or previously studied compounds that have the
potential to be improved with deuterium substitution to enhance clinical
safety, tolerability and efficacy. The Company is developing a broad
pipeline targeting genetic diseases, autoimmune disorders, CNS
disorders and inflammatory diseases, among others. For more information,
please visit www.concertpharma.com.
Cautionary Note on Forward Looking Statements
Any statements in this press release about our future expectations,
plans and prospects, including statements about clinical development of
CTP-656 and other statements containing the words “anticipate,”
“believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,”
“plan,” “potential,” “predict,” “project,” “should,” “target,” “would,”
and similar expressions, constitute forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of 1995.
Actual results may differ materially from those indicated by such
forward-looking statements as a result of various important factors,
including: the uncertainties inherent in the initiation of future
clinical trials, availability and timing of data from ongoing and future
clinical trials and the results of such trials, whether preliminary
results from a clinical trial will be predictive of the final results of
that trial or whether results of early clinical trials will be
indicative of the results of later clinical trials, expectations for
regulatory approvals and other factors discussed in the “Risk Factors”
section of our most recent Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission and in other filings that we make
with the Securities and Exchange Commission. In addition, any
forward-looking statements included in this press release represent our
views only as of the date of this release and should not be relied upon
as representing our views as of any subsequent date. We specifically
disclaim any obligation to update any forward-looking statements
included in this press release.

Concert Pharmaceuticals Inc., the CoNCERT Pharmaceuticals Inc. logo and
DCE Platform are registered trademarks of Concert Pharmaceuticals, Inc.

Kalydeco is a registered trademark of Vertex Pharmaceuticals, Inc.