Application Seeks Full Approval for Duvelisib for the Treatment of
Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia/Small
Lymphocytic Lymphoma and Accelerated Approval in Relapsed/Refractory
Follicular Lymphoma
FDA Target Action Date of October 5, 2018
BOSTON--(BUSINESS WIRE)--Apr. 9, 2018--
Verastem, Inc. (NASDAQ:VSTM), a biopharmaceutical company focused on
developing and commercializing medicines to improve the survival and
quality of life of cancer patients, today announced that the U.S. Food
and Drug Administration (FDA) has accepted for filing with Priority
Review its New Drug Application (NDA) for its lead product candidate
duvelisib. Duvelisib is a first-in-class oral dual inhibitor of
phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, for which
Verastem is seeking full approval 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). The FDA target action date is
October 5, 2018.
“Obtaining Priority Review in the U.S. for duvelisib marks another
important milestone for Verastem and speaks to the unmet need in
relapsed/refractory CLL/SLL and FL and the urgency to identify effective
therapies to treat these patients,” said Robert Forrester, President and
Chief Executive Officer of Verastem. “As an orally administered therapy,
we believe duvelisib will provide an important treatment option for
patients with CLL/SLL and FL, and for the physicians who treat them. We
look forward to working with the FDA during the review process. We are
continuing our commercial preparations for duvelisib to execute the
launch promptly in the U.S. if approved. In parallel, we are exploring
ex-U.S. partnering opportunities for duvelisib and plan to file a
European Marketing Application towards the end of the year.”
Priority Review is granted by the FDA to drugs that, if approved, would
provide significant improvements in the safety or effectiveness of the
treatment, diagnosis, or prevention of a serious condition. Duvelisib
has received Fast Track Designation from the FDA for patients with CLL
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.
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/small lymphocytic
lymphoma (CLL/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 the FDA is reviewing 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), which has Fast Track status, 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
developing and commercializing drugs to improve the survival and quality
of life of cancer patients. 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 indolent non-Hodgkin
lymphoma (iNHL) and a Phase 3 clinical trial in patients with chronic
lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). 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). 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 (NSCLC), and mesothelioma. Verastem’s product candidates seek to
treat cancer by modulating the local tumor microenvironment and
enhancing anti-tumor immunity. 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, Verastem’s potential collaboration opportunities 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 approval of the NDA will not occur on the expected
timeframes or at all, including by the FDA’s target action date; that a
filing of a European Marketing Application may not be achieved before
the end of the year, if 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,
2017 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 D.G., Faia K.L., DiNitto J.P. 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 K 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 M 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.
4www.clinicaltrials.gov,
NCT02004522
5www.clinicaltrials.gov,
NCT01882803
6www.clinicaltrials.gov,
NCT02783625, NCT02158091
View source version on businesswire.com: https://www.businesswire.com/news/home/20180409005377/en/
Source: Verastem, Inc.
Verastem, Inc.
Marianne M. Lambertson, +1-781-292-4273
Vice
President, Corporate Communications
Investor Relations/Public
Relations
mlambertson@verastem.com