BOSTON--(BUSINESS WIRE)--Feb. 7, 2018--
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,
NCT02004522
5 www.clinicaltrials.gov,
NCT01882803
6 www.clinicaltrials.gov,
NCT02783625, NCT02158091
View source version on businesswire.com: http://www.businesswire.com/news/home/20180207005046/en/
Source: Verastem, Inc.
Verastem, Inc.
Marianne M. Lambertson, 781-292-4273
Vice
President, Corporate Communications
Investor Relations/Public
Relations
mlambertson@verastem.com