The Primary Outcome of Progression Free Survival (PFS) via
Independent Review Committee (IRC) in the Intent to Treat (ITT)
Population Significantly Favored Duvelisib Monotherapy Over Ofatumumab
(Median PFS of 13.3 versus 9.9 Months, Respectively; Hazard Ratio (HR)
of 0.52, p<0.0001)
Similar Efficacy Benefit for Duvelisib Monotherapy Over Ofatumumab
for Patients with 17p Deletion (Median PFS of 12.7 versus 9.0 Months,
Respectively; HR of 0.41, p=0.0011)
Oral Duvelisib Continues to Demonstrate a Consistent and Manageable
Safety Profile
Conference Call Scheduled for Today at 8:00 AM ET
BOSTON--(BUSINESS WIRE)--Sep. 6, 2017--
Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing
drugs to improve the survival and quality of life of cancer patients,
today reported positive top-line results from the Phase 3 DUO study
evaluating the efficacy and safety of duvelisib, a first in class oral
dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma,
in patients with relapsed or refractory chronic lymphocytic leukemia
(CLL)/small lymphocytic lymphoma (SLL). Regarding the DUO study’s
primary endpoint of progression free survival (PFS) as determined by
Independent Review Committee (IRC), oral duvelisib monotherapy showed
superiority over ofatumumab, an approved standard of care treatment for
patients with CLL/SLL, achieving 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. Median PFS in the subset of patients
with 17p deletion randomized to duvelisib was also significantly higher
(12.7 months compared to 9.0 months for ofatumumab; HR of 0.41,
p=0.0011).
“Although the treatment of CLL/SLL has advanced in recent years, there
remains a substantial unmet need with many patients progressing or
relapsing following the available therapies,” commented Ian Flinn, MD,
PhD, Director of the Blood Cancer Research Program at Sarah Cannon
Research Institute and the Lead Investigator on the DUO study. “These
positive results from the randomized DUO study demonstrate that
duvelisib prolongs progression-free survival (PFS) with a manageable
safety profile in patients with relapsed or refractory CLL/SLL,
including in high risk patients with the 17p deletion. For our patients
with CLL/SLL, and for the physicians who treat them, a convenient, oral
monotherapy that is taken at home would be a valuable addition to the
treatment landscape.”
Verastem plans to share these clinical data with the U.S. Food and Drug
Administration (FDA) with the goal of filing a New Drug Application
(NDA) with the FDA during the first half of 2018. The duvelisib NDA
submission will be supported by favorable results from both the DUO
study in CLL/SLL and the DYNAMO™ study in indolent non-Hodgkin’s
lymphoma (iNHL), which also achieved its primary endpoint with an ORR of
46% (p<0.0001).
In the Phase 3 DUO study, 319 patients were randomized 1:1 to receive
either duvelisib 25mg twice daily until disease progression or
unacceptable toxicity or ofatumumab, an approved standard of care
treatment for use in CLL/SLL, per its label with an initial infusion of
300 mg followed by 7 weekly infusions and 4 monthly infusions of 2,000
mg. In addition to the primary endpoint of PFS in the ITT population a
stratification factor to evaluate the outcome in the patients with 17p
deletion CLL/SLL, a known poor prognostic subgroup, was conducted. PFS
and other efficacy endpoints were analyzed using response determinations
per the IRC using modified iwCLL/IWG criteria.
Duvelisib monotherapy had a manageable safety profile, with results from
this study consistent with the well-characterized safety profile of
duvelisib monotherapy in patients with advanced hematologic
malignancies. Verastem intends to submit detailed results from the Phase
3 DUO study for publication in a peer-reviewed medical journal and for
presentation at an upcoming scientific meeting.
“We are extremely grateful to the patients, caregivers, and
investigators who participated in the DUO study and we are pleased to be
that much closer to delivering on our mission to develop drugs that
improve the lives of patients with cancer,” said Robert Forrester,
President and Chief Executive Officer of Verastem. “Duvelisib was an
important strategic acquisition for Verastem. Both of our late-stage
trials with duvelisib monotherapy (DUO and DYNAMO) have now achieved
their primary endpoints, highlighting the significant potential of
duvelisib in the treatment of advanced hematologic malignancies. We
anticipate sharing these results with the FDA in preparation for a
potential NDA filing during the first half of 2018 and look forward to
exploring subsequent development opportunities for duvelisib in
additional cancers.”
Conference Call Information
The Verastem management team will host a conference call today,
Wednesday, September 6, 2017, at 8:00 AM (ET). The call can be accessed
by dialing 1-877-341-5660 (toll-free) or 1-315-625-3226 (international)
five minutes prior to the start of the call and providing the passcode
81095627.
The live, listen-only webcast of the conference call can be accessed by
visiting the investors section of the Company’s website at www.verastem.com.
A replay of the webcast will be archived on the Company’s website for 90
days following the call.
About Duvelisib
Duvelisib is an 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 clinical trials, including DUO™, a randomized,
Phase 3 monotherapy study in patients with relapsed or refractory
CLL/SLL,4 and DYNAMO™, a single-arm, Phase 2 monotherapy
study in patients with refractory iNHL.5 Both DUO and DYNAMO
achieved their primary endpoints upon topline analysis of efficacy data.
Duvelisib is also being evaluated for the treatment of other hematologic
malignancies, including T-cell lymphoma, 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 the primary
endpoints in both a Phase 2 study in double-refractory iNHL and a Phase
3 clinical trial in patients with relapsed/refractory 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, ovarian, 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. 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 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. will fail to fully perform
under the duvelisib license agreement; that Verastem will not pursue or
submit regulatory filings for its product candidates; 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, NCT02783625, NCT02158091
View source version on businesswire.com: http://www.businesswire.com/news/home/20170906005599/en/
Source: Verastem, Inc.
Verastem, Inc.
Brian Sullivan, 781-292-4214
Director,
Corporate Development
bsullivan@verastem.com