– MOTION Study Met Primary Endpoint with
Objective Response Rate (ORR) at Week 25 of 40% Compared to 0% for
Placebo (p<0.0001) –
– MOTION Study Met All Key Secondary Endpoints
with Statistically Significant and Clinically Meaningful
Improvements at Week 25 Compared to Placebo, including ORR by Tumor
Volume Score (TVS) of 67% vs. 0% (p< 0.0001) –
– Updated Results from Phase 1/2 Study of
Vimseltinib Demonstrated Best ORR of 72% (Phase 1) and 64% (Phase 2
Cohort A) with Median Treatment Duration of 25.1 Months (Phase 1)
and 21.0 Months (Phase 2 Cohort A) –
– Treatment with Vimseltinib was Well-tolerated
with No Evidence of Cholestatic Hepatotoxicity in MOTION and Phase
1/2 Studies –
– Company Expects to Submit New Drug
Application (NDA) in Second Quarter 2024 and Marketing
Authorisation Application (MAA) in Third Quarter 2024 –
– Conference Call to be Held Today at 8:00 AM
ET –
Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a
biopharmaceutical company focused on discovering, developing, and
commercializing important new medicines to improve the lives of
people with cancer, today announced positive top-line results from
the MOTION pivotal Phase 3 study of vimseltinib in patients with
TGCT not amenable to surgery. Vimseltinib is the Company’s
investigational, orally administered, potent, and highly selective
switch-control kinase inhibitor of CSF1R.
“Patients suffering from TGCT are in need of a new treatment
option that offers both strong clinical benefit and a
well-tolerated safety profile,” said Hans Gelderblom, M.D., Ph.D.,
Chair of the Department of Medical Oncology at Leiden University
Medical Center. “TGCT has a significant negative impact on the
daily life of patients who face substantial pain, stiffness, and
impaired mobility. Success across both the primary and all key
secondary endpoints in MOTION underscores vimseltinib’s ability to
help TGCT patients feel and function better. The top-line results
from MOTION, together with the impressive data announced today from
the Phase 1/2 study showing that the response rates with
vimseltinib continue to increase over time, and that patients
continue to receive long-term clinical benefit as evidenced by the
median duration of treatment, demonstrates vimseltinib’s potential
to become a best-in-class agent.”
“We are excited about the potential for vimseltinib to become
our next approved medicine, supporting our continued evolution to a
company with multiple marketed products,” said Steve Hoerter,
President and Chief Executive Officer of Deciphera Pharmaceuticals.
“The totality of data shown today demonstrate the potential for
vimseltinib to offer a new and differentiated treatment option for
patients with TGCT. On behalf of the entire Deciphera team, I would
like to thank the patients, their caregivers, and the healthcare
professionals who participated in the MOTION and Phase 1/2 studies.
We look forward to working with regulatory agencies worldwide as we
focus on delivering this important new treatment option to patients
with TGCT.”
Top-line Results from the MOTION Pivotal Phase 3 Study of
Vimseltinib in TGCT
The MOTION pivotal Phase 3 study is a two-part, randomized,
double-blind, placebo-controlled study to assess the efficacy and
safety of vimseltinib in patients with TGCT not amenable to surgery
with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib
or nilotinib allowed). In Part 1, patients (n=123) were randomized
two-to-one to receive either 30 mg twice weekly of vimseltinib
(n=83) or placebo (n=40) for 24 weeks. The primary endpoint of the
study is ORR at Week 25 as measured by Response Evaluation Criteria
in Solid Tumors (RECIST) version 1.1 by blinded independent
radiologic review (IRR). The open-label Part 2 portion of MOTION,
in which patients from both the vimseltinib and placebo arms
receive treatment with vimseltinib, remains ongoing. The results
for Part 1 of the study are based on a data cutoff date of August
22, 2023.
The study met its primary endpoint in the intent-to-treat (ITT)
population demonstrating statistically significant and clinically
meaningful improvement versus placebo in ORR at Week 25 based on
IRR per RECIST v1.1. In the ITT population, the ORR at Week 25 was
40% (95% CI: 29%, 51%) for the vimseltinib arm and 0% (95% CI: 0%,
9%) for the placebo arm resulting in a response difference
(vimseltinib versus placebo) of 40% (95% CI: 29%, 51%)
(p<0.0001).
In addition to meeting the primary endpoint, the study also
achieved statistically significant and clinically meaningful
improvements versus placebo in all key secondary endpoints assessed
at Week 25 including ORR per tumor volume score (TVS), active range
of motion (ROM), physical function, stiffness, quality of life, and
pain.
In the ITT population, the ORR at Week 25 based on IRR per TVS
was 67% (95% CI: 56%, 77%) for the vimseltinib arm and 0% (95% CI:
0%, 9%) for the placebo arm (p<0.0001). Treatment with
vimseltinib also demonstrated an improvement in mean change from
baseline in active ROM at Week 25 of 18.4% versus a 3.8%
improvement for placebo (p=0.0077).
Vimseltinib was well-tolerated and the safety profile in the
MOTION study was consistent with previously disclosed data. There
was no evidence of cholestatic hepatotoxicity in patients treated
with vimseltinib. Patients with treatment-emergent adverse events
(TEAEs) leading to treatment discontinuation was 6% in the
vimseltinib arm. The table below lists TEAEs >15% in either arm during Part 1 of the
study.
Preferred Term n (%)
Vimseltinib
(n=83)
Placebo (n=39)1
All Grades
Grade 3/4
All Grades
Grade 3/4
Periorbital edema^
37 (45%)
3 (4%)
5 (13%)
0
Fatigue ^
27 (33%)
0
6 (15%)
0
Face edema^
26 (31%)
1 (1%)
3 (8%)
0
Pruritus^
24 (29%)
2 (2%)
3 (8%)
0
Headache^
23 (28%)
1 (1%)
10 (26%)
0
Asthenia^
22 (27%)
1 (1%)
9 (23%)
1 (3%)
Nausea ^
21 (25%)
0
8 (21%)
1 (3%)
CPK increased
20 (24%)
8 (10%)
0
0
AST increased
19 (23%)
0
1 (3%)
0
Arthralgia ^
16 (19%)
0
6 (15%)
1 (3%)
Rash ^
16 (19%)
0
2 (5%)
0
Rash maculo-papular^
16 (19%)
1 (1%)
0
0
Edema peripheral ^
15 (18%)
0
3 (8%)
0
Hypertension
14 (17%)
4 (5%)
4 (10%)
1 (3%)
Diarrhea
10 (12%)
0
8 (21%)
1 (3%)
(1) Does not include one patient
randomized to placebo that did not receive study drug.
TEAE incidence is based on maximum grades
per CTCAE v5.0. The only Grade 4 adverse events were CPK Increased
observed in two patients. TEAEs leading to dose interruption were
44 (53%) and dose reduction 35 (42%). ^ Denotes adverse events
without Grade 4 criteria per CTCAE v5.0.
The Company expects to submit a New Drug Application (NDA) to
the U.S. Food and Drug Administration (FDA) for vimseltinib for the
treatment of patients with TGCT in the second quarter of 2024 and a
Marketing Authorisation Application (MAA) to the European Medicines
Agency in the third quarter of 2024.
Additional efficacy and safety results from Part 1 of the MOTION
study are expected to be presented at an upcoming medical
meeting.
Updated Results from the Phase 1/2 Study of Vimseltinib in
TGCT
The Company also announced today updated results from the Phase
1/2 open-label, multicenter study evaluating the safety and
efficacy of vimseltinib in patients with TGCT, which will be
presented at the Connective Tissue Oncology Society 2023 Annual
Meeting in Dublin, Ireland on November 1-4, 2023.
As of the June 27, 2023 cutoff date, 97 patients were enrolled
in the study as follows:
- Phase 1: 32 patients enrolled in three cohorts across multiple
doses.
- Phase 2: 65 patients enrolled in two cohorts at the recommended
Phase 2 dose of 30 mg twice weekly: Cohort A (n=46) in TGCT
patients with no prior anti-CSF1/CSF1R therapy (prior therapy with
imatinib or nilotinib allowed); and Cohort B (n=19) in TGCT
patients with prior anti-CSF1/CSF1R therapy (prior therapy with
imatinib or nilotinib alone not eligible).
Antitumor Activity and Treatment
Duration:
93 patients were evaluable for efficacy by RECIST v1.1 at the
data cutoff date; response data is based on IRR summarized as
follows:
Phase 1
(n=32)
Phase 2 Cohort A
(n=45)
Phase 2 Cohort B
(n=16)
Best ORR per RECIST v1.1 by IRR (%)
72%
64%
(38% at Week 25)
44%
Median Duration of Response
(months) (Range)
NR (3.8+, 45.2+)
NR (0.03+, 25.4+)
NR (4.0+, 21.0+)
Median Treatment Duration
(months) (Range)
25.1 (0.7, 46.9)
21.0 (0.2, 30.3)
7.3 (0.7, 27.4)
Patients Active on Treatment at Cutoff
Date (%)
47%
48%
74%
NR: Not Reached by Kaplan-Meier
analysis.
In addition, updated data from Cohorts A and B of the Phase 2
study demonstrate that patients experienced clinically meaningful
symptomatic benefit at Week 25 across multiple secondary efficacy
measures including best ORR per TVS (Cohort A), active range of
motion, physical function, stiffness, and pain.
Safety and Tolerability
- Treatment with vimseltinib was well-tolerated in patients with
TGCT and consistent with previously disclosed data.
- There was no evidence of cholestatic hepatotoxicity.
- Treatment discontinuation due to TEAEs occurred in 9% of
patients.
Results from the Phase 1 portion of the study are being
presented in an oral presentation, titled “Safety and Efficacy
Updates from a Phase 1 Study of Vimseltinib in Patients with
Tenosynovial Giant Cell Tumor.” Results from Cohorts A and B of the
Phase 2 portion of the study are being presented in two poster
presentations, titled “Safety, Efficacy, and Patient-Reported
Outcomes with Vimseltinib in Patients with Tenosynovial Giant Cell
Tumor Who Received No Prior Anti-Colony-Stimulating Factor 1
Therapy: Ongoing Phase 2 Update” and “Safety, Efficacy, and
Patient-Reported Outcomes with Vimseltinib in Patients with
Tenosynovial Giant Cell Tumor Who Received Prior
Anti-Colony-Stimulating Factor 1 Therapy: Ongoing Phase 2 Update.”
The presentations are available on the Company’s website at
www.deciphera.com/presentations-publications.
Conference Call and Webcast
Deciphera will host a conference call and webcast to discuss
this announcement today, October 30, 2023, at 8:00 AM ET. The
conference call may be accessed via this link:
https://register.vevent.com/register/BIc2885b197da74145bfc30deb5fb11858.
A live webcast of the conference call will be available in the
“Events and Presentations” page in the “Investors & News”
section of the Company’s website at
https://investors.deciphera.com/events-presentations. A replay will
be available on the Company’s website approximately two hours after
the conference call and will be available for 30 days following the
call.
About Deciphera Pharmaceuticals
Deciphera is a biopharmaceutical company focused on discovering,
developing, and commercializing important new medicines to improve
the lives of people with cancer. We are leveraging our proprietary
switch-control kinase inhibitor platform and deep expertise in
kinase biology to develop a broad portfolio of innovative
medicines. In addition to advancing multiple product candidates
from our platform in clinical studies, QINLOCK® is Deciphera’s
switch-control inhibitor for the treatment of fourth-line GIST.
QINLOCK is approved in Australia, Canada, China, the European
Union, Hong Kong, Israel, Macau, New Zealand, Singapore,
Switzerland, Taiwan, the United Kingdom, and the United States. For
more information, visit www.deciphera.com and follow us on LinkedIn
and Twitter (@Deciphera).
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, including, without limitation, our expectations
and timing regarding the potential for our preclinical and/or
clinical stage pipeline assets to be first-in-class and/or
best-in-class treatments; the potential for vimseltinib to become
our second approved medicine, the potential for vimseltinib to
become a new treatment option for patients with TGCT, plans to
submit an NDA for vimseltinib in the second quarter of 2024 and an
MAA in the third quarter of 2024, and plans to present additional
data at upcoming medical congresses. The words “may,” “will,”
“could,” “would,” “should,” “expect,” “plan,” “anticipate,”
“intend,” “believe,” “estimate,” “predict,” “project,” “potential,”
“continue,” “seek,” “target” and similar expressions are intended
to identify forward-looking statements, although not all
forward-looking statements contain these identifying words. Any
forward-looking statements in this press release are based on
management’s current expectations and beliefs and are subject to a
number of risks, uncertainties and important factors that may cause
actual events or results to differ materially from those expressed
or implied by any forward-looking statements contained in this
press release, including, without limitation, our ability to
successfully demonstrate the efficacy and safety of our drug or
drug candidates, the preclinical or clinical results for our
product candidates, which may not support further development of
such product candidates, comments, feedback and actions of
regulatory agencies, including the FDA and the EMA, our ability to
commercialize QINLOCK and execute on our marketing plans for any
drugs or indications that may be approved in the future, the
inherent uncertainty in estimates of patient populations and total
addressable markets, competition from other products, our ability
to obtain and maintain reimbursement for any approved product and
the extent to which patient assistance programs are utilized and
other risks identified in our Securities and Exchange Commission
(SEC) filings, including our Quarterly Report on Form 10-Q for the
quarter ended September 30, 2023, and subsequent filings with the
SEC. We caution you not to place undue reliance on any
forward-looking statements, which speak only as of the date they
are made. We disclaim any obligation to publicly update or revise
any such statements to reflect any change in expectations or in
events, conditions or circumstances on which any such statements
may be based, or that may affect the likelihood that actual results
will differ from those set forth in the forward-looking
statements.
The Deciphera logo, QINLOCK, and the QINLOCK logo are registered
trademarks and Deciphera is a trademark of Deciphera
Pharmaceuticals, LLC.
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version on businesswire.com: https://www.businesswire.com/news/home/20231030355437/en/
Investor Relations: Maghan Meyers Argot Partners
Deciphera@argotpartners.com 212-600-1902
Media: David Rosen Argot Partners
david.rosen@argotpartners.com 212-600-1902
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