THRIVE-AA2 Study Met Primary Endpoint for Scalp
Hair Regrowth
Key Secondary Endpoint Met for Patient Reported
Outcome on Hair Satisfaction
Key Secondary Endpoint Met for Hair Regrowth as
Early as 12 Weeks
CTP-543 Has Potential to be Best-in-Class for
the Treatment of Alopecia Areata
Company Completes Pivotal Program with Two
Positive Phase 3 Trials and Expects to File New Drug Application in
First Half of 2023
Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced
positive topline results from its second Phase 3 clinical trial,
THRIVE-AA2, evaluating its oral investigational medicine CTP-543 in
adult patients with moderate to severe alopecia areata, an
autoimmune disorder that results in patchy or complete scalp hair
loss. The primary efficacy endpoint for THRIVE-AA2 was the
percentage of patients achieving an absolute Severity of Alopecia
Tool (SALT) score of 20 or less at Week 24 of treatment, which was
met with statistical significance in both the 8 mg twice-daily and
12 mg twice-daily dose groups relative to placebo. Treatment with
CTP-543 was generally well tolerated.
“It is a new era of innovation for developing treatment options
for patients with alopecia areata, many of whom often suffer
physically and mentally with this challenging autoimmune disease,”
said Maryanne Senna, MD, Director of the Lahey Hair Loss Clinic of
Excellence and clinical investigator of THRIVE-AA2. “These results
are very promising and suggest that CTP-543 has the potential to
address important needs for people living with alopecia
areata.”
“With the successful completion of our two THRIVE-AA Phase 3
trials, Concert is now moving rapidly to prepare our NDA for
submission to the U.S. FDA in the first half of 2023. We are also
continuing to advance our commercial planning as we position
CTP-543 to potentially enter the marketplace for the large
underserved population of patients with alopecia areata,” said
James V. Cassella, Ph.D., Chief Development Officer of Concert
Pharmaceuticals. “Based on the results from our Phase 3 program, we
believe CTP-543, if approved, could be a best-in-class treatment
option for alopecia areata and have a distinct therapeutic profile
with features that are important to patients, including the
percentage of patients with clinically-meaningful hair regrowth,
patient satisfaction levels, and hair regrowth rates.”
Dr. Cassella added, “The Concert team continues to be
enthusiastic and committed to working to bring CTP-543 to market as
soon as possible as a potential new treatment option for patients
living with alopecia areata.”
Patients enrolled in THRIVE-AA2 were required to have at least
50 percent scalp hair loss due to alopecia areata, as measured by
SALT. A SALT score of 100 represents total scalp hair loss, whereas
a score of 0 represents no scalp hair loss. The average baseline
SALT score across all patients was approximately 87.9
(corresponding to approximately 12% average scalp hair
coverage).
A statistically significant proportion of patients treated with
either 8 mg twice-daily or 12 mg twice-daily of CTP-543 experienced
greater scalp regrowth compared to placebo. The proportion of
patients achieving a SALT score of 20 or less (meaning 20 percent
or less scalp hair loss) was 38.3 percent in the 12 mg twice-daily
dose group and 33.0 percent in the 8 mg twice-daily dose group,
compared to 0.8 percent of patients in the placebo group, at the
24-week endpoint. The treatment difference for both dose groups of
CTP-543 relative to placebo was statistically significant
(p<0.0001).
The key secondary endpoints were the percentage of responders on
a Satisfaction of Hair Patient Reported Outcome (SPRO) scale at
Week 24 and the percentage of patients achieving absolute SALT
scores of 20 or less at each of Weeks 20, 16, 12 and 8. 47% of
patients in the 8 mg twice-daily group and 52% of patients in the
12 mg twice-daily group reported being “satisfied” or “very
satisfied,” as compared to 2% of patients in the placebo group. The
treatment difference for both groups relative to placebo was
statistically significant. SALT scores of 20 or less at Weeks 20,
16 and 12 were statistically significant in both dose groups.
The safety profile seen with CTP-543 in THRIVE-AA2 was
consistent with previous studies. The most common (≥5%) side
effects in any dose group were COVID-19 infection, nasopharyngitis,
increased creatine kinase levels, acne and headache. No pulmonary
embolisms or deep vein thromboses were observed in the trial. Two
patients treated with the 8 mg twice-daily dose and two patients
treated with the 12 mg twice-daily dose developed herpes zoster
(shingles). Five serious adverse events were reported in five
patients, with only one in the 8 mg twice-daily dose group that was
assessed as possibly related to treatment.
Concert expects to submit the full results from this study for
future scientific publication and presentation. These data, along
with data from the first Phase 3 clinical trial, THRIVE-AA1, are
intended to form the basis of a New Drug Application (NDA) planned
to be submitted to the U.S. Food and Drug Administration (FDA) in
the first half of 2023.
About THRIVE-AA2
THRIVE-AA2 (NCT04797650) is a randomized, double-blind,
placebo-controlled clinical trial in 517 adult patients age 18-65
with moderate to severe alopecia areata at sites in the U.S.,
Canada and Europe evaluating the regrowth of scalp hair after 24
weeks of dosing using the SALT score. Patients were randomized to
receive either 8 mg twice-daily or 12 mg twice-daily of CTP-543 or
placebo for 24 weeks. The primary endpoint is the percentage of
patients achieving a SALT score ≤ 20 at 24 weeks. All patients who
completed 24 weeks of treatment in THRIVE-AA2 had the opportunity
to continue in a separate extension study to evaluate long-term
safety and efficacy of CTP-543. The THRIVE-AA pivotal program,
consisting of two Phase 3 clinical trials that enrolled over 1,200
patients, is one of the most comprehensive development programs in
alopecia areata.
About CTP-543 and Alopecia Areata
CTP-543 is an investigational oral selective inhibitor of Janus
kinases JAK1 and JAK2. The FDA has granted CTP-543 Breakthrough
Therapy designation for the treatment of adult patients with
moderate to severe alopecia areata and Fast Track designation for
the treatment of alopecia areata.
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 up
to approximately 1.5 million Americans at any given time.1 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
limited treatment options available for 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 late-stage clinical
biopharmaceutical company that is developing small molecule drugs
that it discovered through the application of its DCE Platform®
(deuterated chemical entity platform). Selective incorporation of
deuterium into known molecules has the potential, on a case-by-case
basis, to provide better pharmacokinetic or metabolic properties,
thereby enhancing their clinical safety, tolerability or efficacy.
Concert’s lead product candidate CTP-543 is being developed for the
treatment of alopecia areata, a serious autoimmune dermatological
condition. Concert is also assessing a number of earlier-stage
pipeline candidates. 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 regarding the development of
CTP-543, the potential for CTP-543 to be a best-in-class treatment
for the treatment of alopecia areata and the planned timing for
filing an NDA for 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,
timing and design of future clinical trials, the availability and
timing of data from ongoing and future clinical trials and the
results of such trials, whether preliminary results, including
safety profiles, 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 the timing of the submission of an NDA, the
availability of 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
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220801005238/en/
Justine E. Koenigsberg (Investors) Concert Pharmaceuticals, Inc.
(781) 674-5284 ir@concertpharma.com
Kathryn Morris (media) The Yates Network (914) 204-6412
kathryn@theyatesnetwork.com
Concert Pharmaceuticals (NASDAQ:CNCE)
Gráfico Histórico do Ativo
De Mar 2024 até Abr 2024
Concert Pharmaceuticals (NASDAQ:CNCE)
Gráfico Histórico do Ativo
De Abr 2023 até Abr 2024