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Deciphera Pharmaceuticals Announces Positive, Preliminary, Top-Line Clinical Data for the Ongoing Phase 1 Clinical Study with DCC-3014 and an Update on Future Development Plans

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- DCC-3014, A Highly-Selective and Potent Small Molecule Inhibitor of
CSF1R, Demonstrates Tolerability, Pharmacokinetics and Biomarker
Mechanistic Proof-of-Concept (mPoC) in Patients with Advanced
Malignancies -

- Deciphera Pharmaceuticals Plans Expansion of Ongoing Phase 1 Study
to Include Patients with Tenosynovial Giant Cell Tumors -

Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage
biopharmaceutical company focused on addressing key mechanisms of tumor
drug resistance, today announced positive, preliminary, top-line data
from the ongoing dose escalation part of the Phase 1 clinical study with
DCC-3014, the Company's investigational small molecule switch control
inhibitor of CSF1R, in patients with advanced malignancies. In addition,
the Company announced a plan to expand the Phase 1 study to evaluate
DCC-3014 in patients diagnosed with Tenosynovial Giant Cell Tumors
(TGCT). A review of further data from this Phase 1 study will be
presented at a medical meeting in 2019.

The Phase 1 study was designed to evaluate the safety, pharmacokinetics
and pharmacodynamics of multiple doses of DCC-3014 in up to 55 patients.

  • As of the data cut-off date of November 9, 2018, increasing doses of
    DCC-3014 were assessed in five dose cohorts across 24 patients with
    advanced solid tumor malignancies. This included one dose cohort that
    received 10 mg once daily (QD) and four dose cohorts that followed a
    schedule of once or twice-weekly maintenance dosing preceded by a
    five-day loading dose regimen at doses of up to 30 mg per dose. In
    addition, four patients are currently enrolled in a dose cohort that
    will receive 40 mg twice weekly preceded by a five-day loading regimen.
  • Preliminary pharmacokinetic (PK) analysis showed dose-proportional
    exposure for DCC-3014 that we believe supports twice-weekly
    maintenance dosing preceded by a five-day loading dose regimen.
  • Biomarker data demonstrated strong target engagement of CSF1R,
    including material reductions in CSF1R positive macrophages in the
    blood that we believe constitutes mechanistic proof-of-concept (mPoC)
    for DCC-3014.
  • DCC-3014 was generally well tolerated in patients enrolled in the dose
    cohorts that received twice-weekly maintenance doses of DCC-3014
    preceded by a five-day loading regimen at doses of up to 30 mg, which
    is summarized below:
    • Treatment emergent adverse events (TEAEs) were mostly Grade 1 or 2;
    • No Grade 3 or 4 DCC-3014 related TEAEs in ≥10% of patients;
    • No dose-limiting toxicities; and
    • A maximum tolerated dose has yet to be established.
  • In the dose-cohort that received 10 mg QD, clinically asymptomatic
    laboratory values were recorded as dose-limiting toxicities in two of
    seven patients.

"We are very pleased with the progress made to date in the Phase 1
clinical study with DCC-3014, our selective, and potent small molecule
inhibitor of CSF1R," said Michael D. Taylor, Ph.D., President and Chief
Executive Officer of Deciphera. "Based on the preliminary tolerability,
PK, and mechanistic proof-of-concept data observed to date, we believe
DCC-3014 is well suited for further development. While we will continue
to enroll further patients in this Phase 1 study, we are planning to
expand this study to enroll patients with tenosynovial giant cell tumors
and to explore combinations with other therapies in the first half of
2019."

About DCC-3014

DCC-3014 is an investigational, orally administered, potent and highly
selective inhibitor of CSF1R. DCC-3014 was designed using the Company's
proprietary switch control kinase inhibitor platform to selectively bind
to the CSF1R switch pocket. DCC-3014 has greater than 100-fold
selectivity for CSF1R over the closely related kinases FLT3, KIT,
PDGFRα, PDGFRß and VEGFR2 and has an even greater selectivity for CSF1R
over approximately 300 other human kinases. CSF1R controls the
differentiation and function of macrophages including Tumor Associated
Macrophages (TAMs) whose density within certain tumors including cancers
of the breast, cervix, pancreas, bladder and brain correlates with poor
prognosis. Tumors induce TAMs to suppress a natural immune response
mediated by cytotoxic T-cells, a type of lymphocyte that would otherwise
eradicate the tumor; a process known as macrophage checkpoints. Through
inhibition of CSF1R DCC-3014 has in preclinical studies demonstrated
potent macrophage checkpoint inhibition as both a single agent and in
combination with PD1 inhibitors and other T-cell checkpoint inhibitors.
DCC-3014 is currently being evaluated in a Phase 1 clinical study. For
more information about the clinical trial design please visit www.clinicaltrials.gov
(NCT03069469).

About Tenosynovial Giant Cell Tumors (TGCTs)

Tenosynovial giant cell tumors (TGCTs) are a group of benign tumors that
involve the synovium, bursae and/or tendon sheath. Although benign,
these tumors can grow and cause damage to surrounding tissues and
structures inducing pain, swelling, and limitation of movement of the
joint. Surgery is the main treatment option; however, these tumors tend
to recur, particularly in pigmented villonodular synovitis, a
diffuse-type of TGCT. If untreated or if the tumor continually recurs
damage and degeneration may occur in the affected joint and surrounding
tissues, which may cause significant disability. A genetic mutation in
certain cells within the tumor causes overproduction of CSF-1, the
ligand for the CSF1R receptor, which attracts macrophages and certain
other cells that become the bulk of these tumors and cause the
associated inflammatory changes.

About Deciphera Pharmaceuticals

Deciphera Pharmaceuticals is a clinical-stage biopharmaceutical company
focused on improving the lives of cancer patients by tackling key
mechanisms of drug resistance that limit the rate and/or durability of
response to existing cancer therapies. Our small molecule drug
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, such as macrophages. We have used our
platform to develop a diverse pipeline of tumor-targeted and
immuno-targeted drug candidates designed to improve outcomes for
patients with cancer by improving the quality, rate and/or durability of
their responses to treatment.

Availability of Other Information About Deciphera Pharmaceuticals

Investors and others should note that Deciphera Pharmaceuticals
communicates with its investors and the public using its company website
(www.deciphera.com),
including but not limited to investor presentations and scientific
presentations, Securities and Exchange Commission filings, press
releases, public conference calls and webcasts. The information that
Deciphera Pharmaceuticals posts on these channels and websites could be
deemed to be material information. As a result, Deciphera
Pharmaceuticals encourages investors, the media and others interested in
Deciphera Pharmaceuticals to review the information that it posts on
these channels, including Deciphera Pharmaceuticals' investor relations
website, on a regular basis. This list of channels may be updated from
time to time on Deciphera Pharmaceuticals' investor relations website
and may include other social media channels than the ones described
above. The contents of Deciphera Pharmaceuticals' website or these
channels, or any other website that may be accessed from its website or
these channels, shall not be deemed incorporated by reference in any
filing under the Securities Act of 1933, as amended.

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 clinical trials with our investigational
agent DCC-3014, including, without limitation, the potential for
DCC-3014 as an effective and well tolerated therapy, the plan to present
data at a medical meeting in 2019, the timing of and plan for expansion
of the Phase 1 clinical study with DCC-3014 to include patients with
TGCT and the timing of and plan to explore combinations with other
therapies. 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 drug candidates, including DCC-2618, rebastinib, and
DCC-3014, our advancement of multiple early-stage and later-stage
efforts, our ability to successfully demonstrate the efficacy and safety
of our drug candidates including in later-stage studies, the preclinical
and clinical results for our drug candidates, which may not support
further development of such drug candidates, our efforts to scale up
drug product manufacturing, our ability to implement commercial
readiness, actions of regulatory agencies, any or all of which may
affect the initiation, timing and progress of clinical studies and other
risks identified in our SEC filings, including our Quarterly Report on
Form 10-Q for the quarter ended September 30, 2018, 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.

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