Duvelisib is Well Positioned to Meet the Evolving Unmet Need in
CLL/SLL and FL
Company is Laying the Foundation to Optimize the Launch and
Commercial Value of Duvelisib
BOSTON--(BUSINESS WIRE)--May 3, 2018--
Verastem, Inc. (NASDAQ:VSTM) (Verastem Oncology or the Company), focused
on developing and commercializing drugs to improve the survival and
quality of life of cancer patients, yesterday hosted in New York City an
Analyst and Investor Day, titled, “Duvelisib: Harnessing the Power of
Dual PI3K Inhibition.”
The program featured key opinion leaders in the hematologic oncology
field: Jennifer Brown, MD, PhD, Associate Professor of Medicine,
Harvard Medical School Director, and Director, CLL Center of the
Division of Hematologic Malignancies, Dana-Farber Cancer Institute; Ian
Flinn, MD, PhD, Director, Blood Cancer Research Program at Sarah
Cannon Research Institute, and Lead Investigator of the DUO and DYNAMO
Studies; Steven Horwitz, MD, Medical Oncologist, Memorial Sloan
Kettering Cancer Center and NYC Health + Hospitals/Bellevue; Brian
Koffman, MDCM, DCFP, FCFP, DABFP, MSEd, Physician, Medical Director
of the Chronic Lymphocytic Leukemia (CLL) Society and CLL Patient; and Lori
Kunkel, MD, Former Chief Medical Officer, Pharmacyclics.
These leading experts in hematologic malignancies, including chronic
lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), follicular
lymphoma (FL), and peripheral T-cell lymphoma (PTCL), provided an
in-depth discussion regarding the current U.S. treatment landscape,
where phosphoinositide-3-kinase (PI3K)- inhibitors fit into the
treatment paradigm and the need for new anti-cancer agents such as
duvelisib, the Company’s first-in-class oral dual inhibitor of
phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma. Joe Lobacki,
Chief Commercial Officer of Verastem, outlined the Company’s
commercialization strategy and plans, including an overview of ongoing
pre-commercial and launch initiatives, in preparation for the potential
launch of duvelisib in the U.S. following the assigned target action
date of October 5, 2018. Following the presentations, Robert Forrester,
President and Chief Executive Officer of Verastem, moderated an expert
panel discussion which highlighted the unmet need in CLL/SLL, FL and
T-cell lymphomas, the clinical utility of PI3K-inhibitors and in
particular duvelisib, as well as current treatment practices and their
perspectives on the medical need for new treatment options.
“At Verastem Oncology, we care differently. We are driven by the
strength, tenacity and courage of those battling cancer and are
single-minded in our resolve to deliver new therapies to patients in
need,” said Robert Forrester, President and Chief Executive Officer of
Verastem. “I want to extend my thanks to the outstanding panel of
experts for their presentations and thoughtful discussion. Their
comments further reinforce our belief in the unmet need of CLL/SLL and
FL patients, the importance of PI3K inhibitors, as well as the gap that
duvelisib will fill, if approved.”
Presentation Highlights
Brian Koffman, MDCM, DCFP, FCFP, DABFP, MSEd, Physician, Founder &
President of the CLL Society, and CLL patient kicked off the morning by
taking the audience through the CLL patient journey, along with the
learnings and challenges of living with high risk disease. Dr. Koffman,
who was diagnosed twelve years ago with CLL, highlighted his perspective
on what patients want, “I believe that more targeted options are needed
for relapsed patients and therapy should be matched to each individual’s
profile and preference.”
Lori Kunkel, MD, former CMO at Pharmacyclics with global approval of
cancer therapeutic IMBRUVICA®, presented the evolving landscape of
therapies for hematologic malignancies. As Dr. Kunkel noted, there are a
number of factors to consider when treating patients with CLL/SLL or FL,
including resistance or intolerance to first-line therapies, patient
sub-types, and comorbidities. She went on to illustrate that every
patient is different, concluding that there is no single solution for
all patients and more options, like duvelisib, are needed for these
incurable diseases.
Jennifer Brown, MD, PhD, Director of the CLL Center of the Division of
Hematologic Malignancies, Dana-Farber Cancer Institute, and Associate
Professor of Medicine at Harvard Medical School, gave a compelling
presentation on the role of PI3K inhibitors as treatment evolves towards
a chemo-free future for many patients with B-Cell malignancies. “While
there are other, efficacious targeted therapies available, each comes
with its own limitations,” noted Dr. Brown. Her presentation focused on
the role of the PI3K inhibitor, the large and growing population of
patients intolerant of BTK inhibitors, particularly older patients, and
a steadily increasing population progressing on BTK and BCL-2
inhibitors. “While venetoclax is an effective therapy, it can be very
challenging to give in a community setting. Duvelisib has a novel
mechanism that is easily given with no infusions required. This may
provide a benefit to older patients in the community, which represents
the majority of the patients,” she concluded.
Ian Flinn, MD, PhD, Director, Blood Cancer Research Program at Sarah
Cannon Research Institute was the lead Investigator of the DUO™ and
DYNAMO™ studies, the basis for the New Drug Application for duvelisib.
“In the Phase 3 DUO study, oral duvelisib monotherapy achieved a
statistically significant improvement in progression-free survival (PFS)
versus the approved standard of care treatment ofatumumab, along with a
well characterized and manageable safety profile, in patients with
previously treated CLL/SLL,” noted Dr. Flinn. During an overview of the
Phase 2 DYNAMO data, Dr. Flinn commented, “The clinical activity and
durability of responses observed in the DYNAMO study, seen across highly
refractory disease subtypes such as FL, highlight the potential of this
drug in lymphoid malignancies. These results were seen in patients who
were refractory to both rituximab and chemotherapy, a specific
population with unmet medical need.” Dr. Flinn concluded by saying,
“Additional options are needed for a physician’s armamentarium in the
treatment of chronic indolent lymphomas and leukemias and the sequential
use of clinically manageable treatments may extend the period of disease
control. Continued development of oral, targeted therapies such as
duvelisib, is necessary to address the medical unmet need. The DYNAMO
and DUO results support duvelisib oral monotherapy as a potential new
and convenient treatment option for previously treated CLL/SLL or FL
patients.”
Dr. Steve Horwitz, Medical Oncologist at Memorial Sloan Kettering,
presented an overview of the unmet need for new strategies in T-cell
lymphoma. He presented encouraging Phase 1 clinical data where duvelisib
demonstrated a 50% investigator-assessed overall response rate in 16
heavily pre-treated patients with relapsed or refractory PTCL, including
a 19% complete response rate and a 31% partial response rate. Dr.
Horwitz also presented data showing that oral duvelisib, in combination
with either romidepsin or bortezomib, has an acceptable safety profile
in patients with relapsed or refractory TCL with meaningful response
rates, noting, “While still preliminary, the results appear promising
when compared to those seen with currently approved therapies. We were
especially pleased to see that these response rates were even higher in
patients with PTCL, a rare and aggressive type of non-Hodgkin lymphoma.”
Dr. Horwitz is an investigator on PRIMO, an open-label, multicenter,
Phase 2 clinical trial evaluating the efficacy and safety of duvelisib
in patients with relapsed or refractory PTCL that is currently being
initiated in the US, EU and Japan.
Mr. Lobacki, former Chief Commercial Officer at Medivation, concluded
the presentations by highlighting the current limited options for
CLL/SLL and FL patients and where duvelisib meets the unmet needs of
both patients and physicians. He provided an overview of how duvelisib
could potentially fit into the treatment landscape, given its profile as
a first in class dual PI3K inhibitor with demonstrated clinical efficacy
in relapsed lymphomas, a single CLL/SLL and FL therapy option with a
safety profile that is well characterized and manageable, a chemo-free
option that has shown signs of clinical efficacy regardless of tumor
burden or genetic alterations, and a daily oral monotherapy that can be
taken at home with no planned hospitalization or infusions required. Mr.
Lobacki also outlined Verastem’s plan to commercialize duvelisib in the
U.S. including sales force size, geographic coverage, reimbursement and
access as well as plans to prioritize a seamless patient experience.
“With this go-to-market plan and this commercial launch, we anchor
duvelisib in relapsed CLL and FL. However, this is just the start, as we
have a strong foundation for future growth into other important
markets,” he concluded.
At the Analyst and Investor Day event, the Company also announced that
it will change its name to Verastem Oncology. Shares of the Company’s
common stock will continue to trade on the Nasdaq Global Market under
the symbol “VSTM.” The name change reinforces Verastem’s commitment to
advance innovative treatment options to improve the lives of patients
battling cancer.
An archived webcast of the event is available on the “Events and
Presentations” page in the “Investors” section of the Company’s website
at www.verastem.com.
About Duvelisib
Duvelisib is a first-in-class investigational oral, 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. Verastem Oncology’s 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) was
accepted for filing by the U.S. Food and Drug Administration (FDA),
granted Priority Review and assigned a target action date of October 5,
2018. Duvelisib is also being developed by Verastem Oncology 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 Oncology
Verastem, (NASDAQ:VSTM), operating as Verastem Oncology, is a
biopharmaceutical company focused on developing and commercializing
drugs to improve the survival and quality of life of cancer patients.
Verastem Oncology 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 Oncology’s 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) was accepted for filing by the U.S.
Food and Drug Administration (FDA), granted Priority Review and assigned
a target action date of October 5, 2018. In addition, Verastem Oncology
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 Oncology’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.
Forward-looking statements notice:
This press release includes forward-looking statements about Verastem
Oncology’s strategy, future plans and prospects, including statements
regarding the development and activity of Verastem Oncology’s
investigational product candidates, including duvelisib and defactinib,
and Verastem Oncology’s PI3K and FAK programs generally, the structure
of our planned and pending clinical trials, Verastem Oncology’s
financial guidance 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
Verastem Oncology’s New Drug Application for duvelisib will not occur on
the expected timeframe or at all, including by the U.S. Food and Drug
Administration’s target action date; that a filing of a European
Marketing Application may not be achieved in fiscal year 2018 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 Oncology’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 DUOTM
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 Oncology will be unable to successfully
initiate or complete the clinical development and eventual
commercialization of its product candidates; that the development and
commercialization of Verastem Oncology’s product candidates will take
longer or cost more than planned; that Verastem Oncology may not have
sufficient cash to fund its contemplated operations; that Verastem
Oncology or Infinity Pharmaceuticals, Inc. will fail to fully perform
under the duvelisib license agreement; that Verastem Oncology 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 Oncology will not pursue or submit regulatory
filings for its product candidates, including for duvelisib in patients
with CLL/SLL or iNHL; and that Verastem Oncology’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 the Company’s Annual Report on Form 10-K for the year
ended December 31, 2017 as filed with the Securities and Exchange
Commission (SEC) on March 13, 2018 and in any subsequent filings with
the SEC. The forward-looking statements contained in this press release
reflect Verastem Oncology’s views as of the date hereof, and the Company
does not assume and specifically disclaims any obligation to update any
forward-looking statements whether as a result of new information,
future events or otherwise, except as required by law.
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/20180503005616/en/
Source: Verastem, Inc.
Verastem Oncology
Marianne M. Lambertson, +1 781-292-4273
Vice
President, Corporate Communications
Investor Relations/Public
Relations
mlambertson@verastem.com
