New Analyses Show Statistically Significant
Differences from Placebo for the 8 mg and 12 mg Twice-Daily Cohorts
Achieving a SALT Score of ≤ 20
CTP-543 Expected to Advance into Phase 3
Evaluation in the Fourth Quarter of 2020
Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today released new
data analyses from its Phase 2 dose-ranging clinical trial of its
investigational agent CTP‑543 for the treatment of
moderate-to-severe alopecia areata. The results were selected as an
oral presentation at the Late-Breaking Research Program at the
American Academy of Dermatology (AAD) Annual Meeting; however, the
in-person meeting, originally scheduled for March 21, 2020, in
Denver, was converted to a virtual meeting. The corresponding
abstract was published by AAD in connection with the virtual
meeting. The data analyses released by Concert build on the
previously-reported Phase 2 primary efficacy analysis which showed
that administration of 8 mg twice-daily and 12 mg twice-daily doses
of CTP-543 for 24 weeks produced a statistically significantly
greater number of responders compared to placebo. A responder was
defined as a ≥ 50% relative reduction in Severity of Alopecia Tool
(SALT) score at Week 24 compared to baseline. In the new analyses,
statistically significant results were reported for the 8 mg and 12
mg twice-daily doses at more stringent response thresholds, which
may be more clinically meaningful to patients, and positive
findings were reported for clinician and patient reported outcome
measures of scalp hair loss.
“We believe that these data from our Phase 2 dose-ranging study
set a new benchmark for clinical efficacy in treating alopecia
areata and provide important information to inform our planned
Phase 3 trials with CTP-543. Since both the 8 mg and 12 mg
twice-daily doses resulted in statistically significant improvement
in SALT scores compared to placebo and exhibited a generally
well-tolerated safety profile, we plan to assess both doses in our
Phase 3 program,” said James V. Cassella, Ph.D., Chief Development
Officer of Concert Pharmaceuticals. “Importantly, we would like to
thank the alopecia areata patients for their participation in this
study, as well as the clinical investigators and research staff for
their significant efforts to support the development of
CTP-543.”
Key new findings further highlight the positive efficacy results
in the Phase 2 study, including:
- Meaningful hair regrowth: At Week 24, 26% and 42% of
patients who received CTP-543 in the 8 and 12 mg twice-daily
cohorts, respectively, achieved an absolute SALT score ≤ 20 (p
<0.05 vs. placebo), indicating a clinically-meaningful 80
percent or greater scalp hair present. In addition, 36% of patients
in the 12 mg twice-daily cohort achieved an absolute SALT score ≤
10 (p <0.05 vs. placebo) at Week 24.
- Clinical Global Impression of Improvement: Data from the
Clinician Global Impression of Improvement scale showed 75% of
clinicians rated the response in the 12 mg twice-daily cohort and
61% of clinicians rated the response in the 8 mg twice-daily cohort
as “much improved” or “very much improved” at Week 24. For both
doses there was a statistically significant difference from placebo
(p <0.001).
- Similarly, previously-reported data from the Phase 2 trial for
the Patient Global Impression of Improvement scale showed 78% of
patients receiving 12 mg twice-daily and 58% of patients receiving
8 mg twice-daily rated their alopecia areata as “much improved” or
“very much improved” at Week 24. For both doses there was a
statistically significant difference from placebo (p
<0.001).
In the Phase 2 dose-ranging trial, the most common (≥10%) side
effects in the 8 mg or 12 mg CTP-543 dose groups were headache,
nasopharyngitis, upper respiratory tract infection, acne, nausea
and low-density lipoprotein increase. One serious adverse event of
facial cellulitis was reported in the 12 mg dose group as possibly
related to treatment. After a brief dosing interruption, treatment
was continued, and this patient completed the trial. No
thromboembolic events were reported during the trial.
Slides summarizing the new analyses are available in the
Scientific Presentations section of Concert’s website.
About the Phase 2 Trial Design of CTP-543 in Alopecia
Areata
The Phase 2 trial was a double-blind, randomized,
placebo-controlled, sequential dose trial to evaluate the safety
and efficacy of CTP-543 in adult patients with moderate-to-severe
alopecia areata. A total of 149 patients were randomized to receive
one of three doses of CTP-543 (4 mg, 8 mg or 12 mg) or placebo,
administered twice-daily. The primary outcome measure utilized the
SALT score after 24 weeks of dosing. The average baseline SALT
score across all patients was approximately 88%, where 0% is no
scalp hair loss and 100% represents total scalp hair loss. All
patients who completed 24 weeks of treatment in the 12 mg dosing
cohort had the opportunity to continue in a separate extension
study to evaluate long-term safety and efficacy of CTP-543.
Previously-reported data from the Phase 2 trial demonstrated a
relative reduction in overall SALT scores from baseline at Week 24,
the primary efficacy endpoint of the study. In the 12 mg
twice-daily cohort, 58% of patients achieving a ≥ 50% relative
reduction in their overall SALT score from baseline compared to 9%
for placebo (p <0.001). In the 8 mg twice-daily cohort, 47% of
patients achieved the primary endpoint compared to placebo (p
<0.001).
About CTP-543 and Alopecia Areata
CTP-543 is an oral selective inhibitor of Janus kinases JAK1 and
JAK2. The FDA has granted Fast Track designation for CTP-543 for
the treatment of alopecia areata. The Company intends to advance
CTP-543 into Phase 3 evaluation in the fourth quarter of 2020.
Alopecia areata is an autoimmune disease in which the immune
system attacks hair follicles, resulting in partial or complete
loss of hair on the scalp and body. Alopecia areata may affect
approximately 700,000 Americans at any given time1. The scalp is
the most commonly affected area, but any hair-bearing site can be
affected alone or together with the scalp. Onset of the disease can
occur throughout life and affects both women and men. Alopecia
areata can be associated with serious psychological consequences,
including anxiety and depression. There are currently no drugs
approved by the FDA for the treatment of alopecia areata.
The FDA selected alopecia areata as one of eight new disease
areas that it focused on under its Patient-Focused Drug Development
Initiative (PFDDI) in 2016-2017. The goal of the PFDDI is to bring
patient perspectives into an earlier stage of product development.
Following the FDA’s Patient-Focused Drug Development meeting held
in September 2017 on alopecia areata, the FDA summarized the input
shared by patients and patient representatives in a Voice of the
Patient report. Additional information on the PFDDI is available
online.
About Concert
Concert Pharmaceuticals is a clinical stage biopharmaceutical
company focused on applying its DCE Platform® (deuterated chemical
entity platform) to create novel medicines designed to treat
serious diseases and address unmet patient needs. The Company’s
approach starts with previously studied compounds, including
approved drugs, in which deuterium substitution has the potential
to enhance clinical safety, tolerability or efficacy. Concert’s
pipeline of innovative medicines targets autoimmune diseases and
central nervous systems (CNS) disorders. For more information
please visit www.concertpharma.com or follow us on Twitter at
@ConcertPharma or on LinkedIn.
Cautionary Note on Forward Looking Statements
Any statements in this press release about our future
expectations, plans and prospects, including, among others,
statements about our expectations on the progress of clinical
development of CTP-543, and any other statements containing the
words “anticipate,” “believe,” “continue,” “could,” “estimate,”
“expect,” “intend,” “may,” “plan,” “potential,” “predict,”
“project,” “should,” “target,” “will,” “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
and timing of future clinical 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.
1 Benigno M. Clinical, Cosmetic and Investigational Dermatology.
2020.
###
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version on businesswire.com: https://www.businesswire.com/news/home/20200612005427/en/
For additional information contact: Justine Koenigsberg
(investors) Concert Pharmaceuticals, Inc. (781) 674-5284
ir@concertpharma.com
Kathryn Morris (media) The Yates Network (914) 204-6412
kathryn@theyatesnetwork.com
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