- Application is Being Reviewed Under FDA's
Real-Time Oncology Review (RTOR) Pilot Program -
- Positive Results from Company’s INVICTUS
Pivotal Phase 3 Clinical Study of Ripretinib in Patients with
Advanced Gastrointestinal Stromal Tumors (GIST) Form Basis of
Submission -
Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) today announced
the submission of a New Drug Application (NDA) to the U.S. Food and
Drug Administration (FDA) for ripretinib, the Company’s
investigational broad-spectrum KIT and PDGFRα inhibitor, for the
treatment of patients with advanced GIST who have received prior
treatment with imatinib, sunitinib and regorafenib.
“The NDA submission for ripretinib marks an exciting milestone
as we work towards delivering a potential new treatment option for
people with advanced GIST,” said Steve Hoerter, President and Chief
Executive Officer of Deciphera. “We look forward to working with
the FDA through their review of our application, and we remain
focused on preparing for the potential launch of ripretinib in the
United States, if approved.”
The NDA is being reviewed by the FDA under the Oncology Center
of Excellence Real-Time Oncology Review, or RTOR, pilot program.
The pilot program aims to explore a more efficient review process
to ensure that safe and effective treatments are available to
patients as early as possible, while maintaining and improving
review quality. Additional information about RTOR can be found at:
https://www.fda.gov/about-fda/oncology-center-excellence/real-time-oncology-review-pilot-program.
In October 2019, FDA granted Breakthrough Therapy Designation
(BTD) for ripretinib for the treatment of patients with advanced
GIST who have received prior treatment with imatinib, sunitinib and
regorafenib. BTD is designed to expedite the development and review
of drugs that are intended to treat a serious condition and
preliminary clinical evidence indicates that the drug may
demonstrate substantial improvement over available therapy.
The NDA submission is supported by positive data from the
Company’s INVICTUS pivotal Phase 3 study of ripretinib in advanced
GIST. INVICTUS is a randomized (2:1), double-blind,
placebo-controlled, international, multicenter study designed to
evaluate the efficacy and safety of ripretinib compared to placebo
in 129 patients with advanced GIST whose previous therapies have
included at least imatinib, sunitinib, and regorafenib. As
previously reported, the study achieved the primary endpoint of
improved progression free survival compared to placebo in patients
with fourth-line and fourth-line plus GIST, as determined by
blinded independent central radiologic review using modified
Response Evaluation Criteria in Solid Tumors (RECIST) version
1.1.
About Gastrointestinal Stromal Tumor
Gastrointestinal stromal tumor (GIST) is a cancer affecting the
digestive tract or nearby structures within the abdomen, most often
presenting in the stomach or small intestine. GIST is the most
common sarcoma of the gastrointestinal tract, with approximately
4,000 to 6,000 new GIST cases each year in the United States and a
similar incidence rate in European and other countries. Most cases
of GIST are driven by a spectrum of mutations. The most common
primary mutations are in KIT kinase, representing approximately 75%
to 80% of cases, and PDGFRα kinase, representing approximately 5%
to 10% of cases. Current therapies are unable to inhibit the full
spectrum of primary and secondary mutations, which drives
resistance and disease progression. Estimates for 5-year survival
range from 48% to 90%, depending on the stage of the disease at
diagnosis.
About Ripretinib
Ripretinib is an investigational tyrosine kinase switch control
inhibitor that was engineered to broadly inhibit KIT and PDGFRα
mutated kinases by using a unique dual mechanism of action that
regulates the kinase switch pocket and activation loop. Ripretinib
is currently in clinical development for the treatment of KIT
and/or PDGFRα-driven cancers, including gastrointestinal stromal
tumors, or GIST, systemic mastocytosis, or SM, and other cancers.
Ripretinib inhibits initiating and secondary KIT mutations in exons
9, 11, 13, 14, 17, and 18, involved in GIST, as well as the primary
exon 17 D816V mutation involved in SM. Ripretinib also inhibits
primary PDGFRα mutations in exons 12, 14 and 18, including the exon
18 D842V mutation, involved in a subset of GIST. Ripretinib has
been granted Fast Track Designation and Breakthrough Therapy
Designation by the U.S. FDA for the treatment of patients with
advanced GIST who have received prior treatment with imatinib,
sunitinib and regorafenib. Ripretinib has also been granted Orphan
Drug Designation for the treatment of GIST by the U.S. FDA and
European Medicines Agency (EMA). For more information about the
Company’s clinical trials with ripretinib, please visit
www.clinicaltrials.gov.
Deciphera Pharmaceuticals has an exclusive license agreement
with Zai Lab (Shanghai) Co., Ltd. for the development and
commercialization of ripretinib in Greater China (Mainland China,
Hong Kong, Macau and Taiwan). Deciphera Pharmaceuticals retains
development and commercial rights for ripretinib in the rest of the
world.
About Deciphera Pharmaceuticals
Deciphera Pharmaceuticals is a clinical-stage biopharmaceutical
company focused on improving the lives of cancer patients by
addressing key mechanisms of drug resistance that limit the rate
and/or durability of response to existing cancer therapies. Our
small molecule product candidates are directed against an important
family of enzymes called kinases, known to be directly involved in
the growth and spread of many cancers. We use our deep
understanding of kinase biology together with a proprietary
chemistry library to purposefully design compounds that maintain
kinases in a “switched off” or inactivated conformation. These
investigational therapies comprise tumor-targeted agents designed
to address therapeutic resistance causing mutations and
immuno-targeted agents designed to control the activation of
immunokinases that suppress critical immune system regulators, and
agents designed to inhibit reprogramming of cancer cell metabolism.
We have used our platform to develop a diverse pipeline of
tumor-targeted, immuno-targeted, and metabolism-targeted product
candidates designed to improve outcomes for patients with cancer by
improving the quality, rate and/or durability of their responses to
treatment.
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, statements
regarding our expectations regarding our goal of bringing
ripretinib to patients with advanced GIST, the potential for
ripretinib to serve as an important new treatment option for people
with advanced GIST, working with the FDA through its review of our
NDA application via the FDA’s Real-Time Oncology Review pilot
program and the possible benefits of that pilot program and
breakthrough therapy designation, and preparing for the potential
launch of ripretinib in the United States, if approved. The words
“may,” “will,” “could,” “would,” “should,” “expect,” “plan,”
“anticipate,” “intend,” “believe,” “estimate,” “predict,”
“project,” “potential,” “continue,” “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, risks and uncertainties related to the delay of any
current or planned clinical studies or the development of our
product candidates, including ripretinib, our ability to
successfully demonstrate the efficacy and safety of our product
candidates including in later-stage studies, the preclinical and
clinical results for our product candidates, which may not support
further development of such product candidates, the possibility
that results experienced in early, preliminary, top-line or initial
data may not be indicative of the results experienced in final
data, our ability to work with the FDA under its RTOR pilot program
and timely respond to information requests or requirements in
connection with our recently-filed NDA for ripretinib in advanced
GIST, that acceptance into the RTOR pilot program does not
guarantee or influence approvability of our NDA for ripretinib in
advanced GIST, which is subject to the standard benefit-risk
evaluation by FDA, and that we may not derive any benefit from
inclusion in the RTOR pilot program, including, but not limited to,
a more efficient review process compared to investigational drugs
evaluated without this pilot program or under standard FDA
procedures, the fact that the RTOR program is a pilot program being
tested by FDA, is not a formal regulatory pathway with regulatory
process, regulations or procedures, and may be suspended or halted
at any time, including, without limitation, because FDA decides not
to continue the pilot, or because FDA determines that our
application no longer meets its criteria for inclusion in the RTOR
pilot program, the fact that receipt of a breakthrough therapy
designation for a product candidate, such as ripretinib, may not
result in us receiving any of the benefits of such designation such
as a faster development process, review or approval compared to
drugs considered for approval under conventional FDA procedures,
the fact such designation does not assure ultimate approval by FDA
and is subject to the risk FDA may later decide that the products
no longer meet the conditions for qualification or decide that the
time period for FDA review or approval will not be shortened, our
ability to manage and our reliance on third parties such as our
third party drug substance and drug product contract manufacturers,
actions of regulatory agencies, any or all of which may affect the
initiation, timing and progress of clinical studies and the timing
of and our ability to obtain regulatory approval, if at all, and
make our investigational drugs available to patients, and other
risks identified in our SEC filings, including our Quarterly Report
on Form 10-Q for the quarter ended September 30, 2019, 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. Any forward-looking statements
contained in this press release represent our views only as of the
date hereof and should not be relied upon as representing its views
as of any subsequent date. We explicitly disclaim any obligation to
update any forward-looking statements.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20191216005211/en/
Investor Relations: Jen Robinson Deciphera Pharmaceuticals, Inc
jrobinson@deciphera.com 781-906-1112 Media: David Rosen Argot
Partners David.Rosen@argotpartners.com 212-600-1902
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