Company Plans to Submit a Single NDA Requesting Full Approval of
Duvelisib for the Treatment of Patients with Relapsed or Refractory
Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma and Accelerated
Approval for the Treatment of Patients with Relapsed or Refractory
Follicular Lymphoma
Company Plans to Submit NDA for Duvelisib During First Quarter of 2018
BOSTON--(BUSINESS WIRE)--Oct. 31, 2017--
Verastem, Inc. (NASDAQ: VSTM), focused on discovering and developing
drugs to improve the survival and quality of life of patients with
cancer, today announced that a meeting was held with the U.S. Food and
Drug Administration (FDA) regarding the regulatory path for duvelisib,
the Company’s first-in-class, oral, monotherapy, dual inhibitor of
phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, which is being
developed for the treatment of patients with lymphoid malignancies.
Based on the meeting with, and written feedback from the FDA, Verastem
intends to submit a New Drug Application (NDA) requesting the full
approval of duvelisib for the treatment of patients with relapsed or
refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma
(SLL), and accelerated approval for the treatment of patients with
relapsed or refractory follicular lymphoma (FL). The Company expects to
submit the duvelisib NDA during the first quarter of 2018.
“With FDA guidance now in hand, we have a defined regulatory path
forward for the duvelisib NDA,” said Robert Forrester, President and
Chief Executive Officer of Verastem. “Obtaining the FDA’s guidance for
this submission represents an important milestone for Verastem and for
duvelisib as a potential new treatment for patients with relapsed or
refractory CLL/SLL and patients with relapsed or refractory FL. Our
near-term focus will be on preparing the NDA, which we expect to submit
during the first quarter of next year.”
In September 2017, Verastem reported that the Phase 3 DUOTM
study met its primary endpoint with oral duvelisib monotherapy
demonstrating superiority over ofatumumab for progression free survival
(PFS) in patients with relapsed or refractory CLL/SLL. In this study,
duvelisib achieved a statistically significant improvement in median PFS
of 13.3 months, compared to 9.9 months for ofatumumab with a hazard
ratio (HR) of 0.52 (p<0.0001), representing a 48% reduction in the risk
of progression or death. Along with the clinical data from the DUO
study, the duvelisib NDA submission will also contain the favorable
results from the Phase 2 DYNAMO™ study in double-refractory indolent
non-Hodgkin’s lymphoma (iNHL), which also achieved its primary endpoint
with an ORR of 46% (p<0.0001). In the subset of patients enrolled in the
DYNAMOstudy 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-cells and is thought to play a role in the formation and maintenance
of the supportive tumor microenvironment.1,2,3 Duvelisib is
currently being 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 intends to submit 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 indolent non-Hodgkin lymphoma (iNHL) and
a Phase 3 clinical trial in patients with chronic lymphocytic leukemia
(CLL)/small lymphocytic lymphoma (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 the full data
from the DUO study will not be consistent with the top-line results of
the study; 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 data may not be available when expected, including for the
Phase 3 DUO™ study; 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 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
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/20171031005343/en/
Source: Verastem, Inc.
Verastem, Inc.
Brian Sullivan, 781-292-4214
Director,
Corporate Development
bsullivan@verastem.com