Verastem Submits New Drug Application to U.S. FDA for Duvelisib for the Treatment of Patients with Relapsed or Refractory Chr...
07 February 2018 - 11:00PM
Business Wire
Verastem, Inc. (NASDAQ: VSTM), focused on discovering and
developing drugs to improve the survival and quality of life of
cancer patients, today announced it has submitted a New Drug
Application (NDA) to the U.S. Food and Drug Administration (FDA)
seeking full approval for its lead product candidate duvelisib, a
first-in-class oral dual inhibitor of phosphoinositide-3-kinase
(PI3K)-delta and PI3K-gamma, for the treatment of relapsed or
refractory chronic lymphocytic leukemia/small lymphocytic lymphoma
(CLL/SLL) and accelerated approval for the treatment of relapsed or
refractory follicular lymphoma (FL). Duvelisib has received Fast
Track Designation from the FDA for patients with CLL or peripheral
T-cell lymphoma (PTCL) who have received at least one prior therapy
and for patients with FL who have received at least two prior
therapies. In addition, duvelisib received orphan drug designation
in the United States and the European Union for patients with CLL,
SLL and FL.
“The submission of our first NDA for duvelisib is a major
milestone for Verastem and is the culmination of substantial effort
by our employees and the investigators who have dedicated
themselves toward developing a potential treatment option for
patients who are in need of additional therapies. We are immensely
grateful to all of the patients that participated in the duvelisib
clinical trial program over the last few years,” said Robert
Forrester, President and Chief Executive Officer of Verastem. “Oral
duvelisib is the first PI3K inhibitor to show efficacy as a
monotherapy in a randomized Phase 3 study in patients with relapsed
or refractory CLL/SLL. Duvelisib monotherapy has also demonstrated
significant clinical activity in patients with double-refractory
FL. We believe duvelisib will offer a convenient oral treatment
alternative. We look forward to working with the FDA during the
review process and to a potential U.S. approval decision for
duvelisib in early 2019.”
The NDA is supported by clinical data from the randomized Phase
3 DUO™ study demonstrating significant efficacy, along with a
consistent and manageable safety profile, of duvelisib monotherapy
in patients with relapsed or refractory CLL/SLL. The DUO study met
its primary endpoint with oral duvelisib monotherapy achieving a
statistically significant improvement in progression-free survival
(PFS) compared to ofatumumab in patients with relapsed or
refractory CLL/ SLL (median PFS of 13.3 months versus 9.9 months,
respectively; HR=0.52; p<0.0001), representing a 48% reduction
in the risk of disease progression or death. The NDA is also
supported by results from the Phase 2 DYNAMO™ study in patients
with indolent non-Hodgkin’s lymphoma that are double-refractory to
both rituximab and chemotherapy or radioimmunotherapy, which also
achieved its primary endpoint with an objective response rate (ORR)
of 46% (p<0.0001). In the subset of patients enrolled in DYNAMO
with double-refractory FL (n=83), duvelisib demonstrated an ORR of
41%.
About Duvelisib
Duvelisib is a first-in-class investigational, dual inhibitor of
phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes
known to help support the growth and survival of malignant B-cells
and T-cells. PI3K signaling may lead to the proliferation of
malignant B- and T-cells and is thought to play a role in the
formation and maintenance of the supportive tumor
microenvironment.1,2,3 Duvelisib was evaluated in late- and
mid-stage extension trials, including DUO™, a randomized, Phase 3
monotherapy study in patients with relapsed or refractory chronic
lymphocytic leukemia (CLL)/small lymphocytic lymphoma
(SLL),4 and DYNAMO™, a single-arm, Phase 2 monotherapy study
in patients with refractory indolent non-Hodgkin lymphoma
(iNHL).5 Both DUO and DYNAMO achieved their primary endpoints
and Verastem has submitted a New Drug Application (NDA)
requesting the full approval of duvelisib for the treatment of
patients with relapsed or refractory CLL/SLL, and accelerated
approval for the treatment of patients with relapsed or refractory
follicular lymphoma (FL). Duvelisib is also being developed
by Verastem for the treatment of peripheral T-cell
lymphoma (PTCL), and is being investigated in combination with
other agents through investigator-sponsored studies.6 Information
about duvelisib clinical trials can be found
on www.clinicaltrials.gov.
About Verastem, Inc.
Verastem, Inc. (NASDAQ:VSTM) is a biopharmaceutical company
focused on discovering and developing drugs to improve outcomes for
patients with cancer. Verastem is currently developing duvelisib, a
dual inhibitor of PI3K-delta and PI3K-gamma, which has successfully
met its primary endpoint in a Phase 2 study in iNHL and a Phase 3
clinical trial in patients with CLL/SLL. In addition, Verastem is
developing the FAK inhibitor defactinib, which is currently being
evaluated in three separate clinical collaborations in combination
with immunotherapeutic agents for the treatment of several
different cancer types, including pancreatic cancer, ovarian
cancer, non-small cell lung cancer, and mesothelioma. Verastem’s
product candidates seek to treat cancer by modulating the local
tumor microenvironment, enhancing anti-tumor immunity, and reducing
cancer stem cells. For more information, please visit
www.verastem.com.
Verastem, Inc. forward-looking statements notice:
This press release includes forward-looking statements about
Verastem's strategy, future plans and prospects, including
statements regarding the development and activity of Verastem's
investigational product candidates, including duvelisib and
defactinib, and Verastem's PI3K and FAK programs generally, the
structure of our planned and pending clinical trials and the
timeline and indications for clinical development and regulatory
submissions. The words "anticipate," "believe," "estimate,"
"expect," "intend," "may," "plan," "predict," "project," "target,"
"potential," "will," "would," "could," "should," "continue," and
similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words. Each forward-looking statement is subject
to risks and uncertainties that could cause actual results to
differ materially from those expressed or implied in such
statement. Applicable risks and uncertainties include the risks
that acceptance or approval of the NDA will not occur on the
expected timeframes or at all; that even if data from clinical
trials is positive, regulatory authorities may require additional
studies for approval and the product may not prove to be safe and
effective; that the preclinical testing of Verastem's product
candidates and preliminary or interim data from clinical trials may
not be predictive of the results or success of ongoing or later
clinical trials; that the full data from the DUO study will not be
consistent with the previously presented results of the study; that
data may not be available when expected, including for the Phase 3
DUO™ study; that the degree of market acceptance of product
candidates, if approved, may be lower than expected; that the
timing, scope and rate of reimbursement for our product candidates
is uncertain; that there may be competitive developments affecting
our product candidates; that data may not be available when
expected; that enrollment of clinical trials may take longer than
expected; that our product candidates will cause unexpected safety
events or result in an unmanageable safety profile as compared to
their level of efficacy; that duvelisib will be ineffective at
treating patients with lymphoid malignancies; that Verastem will be
unable to successfully initiate or complete the clinical
development of its product candidates; that the development of
Verastem's product candidates will take longer or cost more than
planned; that Verastem may not have sufficient cash to fund its
contemplated operations; that Verastem or Infinity Pharmaceuticals,
Inc. (Infinity) will fail to fully perform under the duvelisib
license agreement; that Verastem may be unable to make additional
draws under its debt facility or obtain adequate financing in the
future through product licensing, co-promotional arrangements,
public or private equity, debt financing or otherwise; that
Verastem will not pursue or submit regulatory filings for its
product candidates, including for duvelisib in patients with
CLL/SLL or iNHL; and that Verastem's product candidates will not
receive regulatory approval, become commercially successful
products, or result in new treatment options being offered to
patients. Other risks and uncertainties include those identified
under the heading "Risk Factors" in Verastem's Annual Report on
Form 10-K for the year ended December 31, 2016 and in any
subsequent filings with the U.S. Securities and Exchange
Commission. The forward-looking statements contained in this press
release reflect Verastem's views as of the date of this release,
and Verastem does not undertake and specifically disclaims any
obligation to update any forward-looking statements.
References
1 Winkler et al. PI3K-delta and PI3K-gamma inhibition by IPI-145
abrogates immune responses and suppresses activity in autoimmune
and inflammatory disease models. Chem Biol 2013; 20:1-11.2 Reif et
al. Cutting Edge: Differential roles for phosphoinositide 3
kinases, p110-gamma and p110-delta, in lymphocyte chemotaxis and
homing. J Immunol 2004:173:2236-2240.3 Schmid et al. Receptor
tyrosine kinases and TLR/IL1Rs unexpectedly activate myeloid cell
PI3K, a single convergent point promoting tumor inflammation and
progression. Cancer Cell 2011;19:715-727.4 www.clinicaltrials.gov,
NCT020045225 www.clinicaltrials.gov, NCT018828036
www.clinicaltrials.gov, NCT02783625, NCT02158091
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version on businesswire.com: http://www.businesswire.com/news/home/20180207005046/en/
Verastem, Inc.Marianne M. Lambertson, 781-292-4273Vice
President, Corporate CommunicationsInvestor Relations/Public
Relationsmlambertson@verastem.com
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