2018 Highlighted by Approval and Commercial Launch of Lead Asset
Entering 2019 with a Strong Balance Sheet to Achieve Corporate
Company’s Focus in 2019 Will Be on Continued Investigation of
Duvelisib in Additional Tumor Types, Including Peripheral T-Cell
Lymphoma, Both as a Monotherapy and in Combination with Other
BOSTON--(BUSINESS WIRE)--Jan. 3, 2019--
Verastem, Inc. (Nasdaq: VSTM) (Verastem Oncology or the Company), a
biopharmaceutical company focused on developing and commercializing
medicines to improve the survival and quality of life of cancer
patients, today highlighted the company’s recent progress and outlined
strategic priorities for 2019.
“2018 was a pivotal year for Verastem Oncology, as the U.S. Food and
Drug Administration’s (FDA) approval of COPIKTRA™ and other key
accomplishments strongly positioned us to execute on our 2019 corporate
priorities that are focused on increasing revenues, initiating
additional clinical studies of COPIKTRA and advancing our pipeline,”
said Robert Forrester, President and Chief Executive Officer of Verastem
Oncology. “We are pleased with the strong vote of confidence we have
received in duvelisib, including validating licensing agreements in key
Asian markets, recognition of our pivotal Phase 3 data in the medical
journal Blood, and more. We are also entering 2019 with a
strong balance sheet derived from the successful completion of multiple
financing transactions, which we believe provides us with important
financial strength to achieve our planned corporate objectives. We look
forward to keeping the momentum going, and to sharing ongoing updates on
“Since the launch of COPIKTRA, we’ve been encouraged by the positive
feedback we are hearing from physicians and other healthcare providers
about this important new oral monotherapy within the treatment
landscape,” said Joseph Lobacki, Executive Vice President and Chief
Commercial Officer of Verastem Oncology. “Following the approval,
COPIKTRA was quickly added to the National Comprehensive Cancer Network®
(NCCN) guidelines, which has led to its inclusion on formularies and
extensive reimbursement coverage, including on the top national health
plans, reaching approximately 75% of U.S. Pharmacy lives and providing
critical access to treatment for appropriate patients. In 2019, the
commercial team will be diligently working to engage with physicians and
other health care professionals to focus on ensuring COPIKTRA reaches
the patients who need it.”
COPIKTRA (duvelisib) Capsules Approved by the U.S. FDA – On
September 24, 2018, the FDA approved COPIKTRA, an oral inhibitor of
phosphoinositide 3-kinase (PI3K), and the first approved dual
inhibitor of PI3K-delta and PI3K-gamma. COPIKTRA was approved for the
treatment of adult patients with relapsed or refractory chronic
lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at
least two prior therapies. COPIKTRA also received accelerated approval
for the treatment of adult patients with relapsed or refractory
follicular lymphoma (FL) after at least two prior systemic therapies.
The indication in FL is approved under accelerated approval based on
overall response rate. Continued approval for this indication is
contingent upon verification and description of clinical benefit in a
confirmatory trial. The commercial launch of COPIKTRA is ongoing.
of COPIKTRA is associated with a BOXED WARNING for four fatal and/or
serious toxicities: infections, diarrhea or colitis, cutaneous
reactions, and pneumonitis. Verastem Oncology is implementing an
informational Risk Evaluation and Mitigation Strategy to provide
appropriate dosing and safety information to better support physicians
in managing their patients on COPIKTRA.
of COPIKTRA is also associated with adverse reactions which may
require dose reduction, treatment delay or discontinuation of COPIKTRA.
for full Prescribing Information including BOXED WARNING and
Medication Guide in addition to the Important Safety Information
Established a Commercial Franchise in the U.S. – In 2018, the
Company established a full commercial infrastructure in the U.S. The
sales, market access and medical affairs teams are fully deployed and
calling on medical institutions, oncology healthcare professionals and
payors in support of the COPIKTRA launch. COPIKTRA product was
available at specialty distributors and specialty pharmaceutical
providers immediately following approval. Top national health plans
are now offering reimbursement coverage and the majority of COPIKTRA
sales territories have patients being treated. The Company also
successfully launched Verastem Cares™, a comprehensive, personalized
program designed to provide information and assistance to patients who
have been prescribed COPIKTRA, which is now fully operational
COPIKTRA Added to NCCN Guidelines for CLL/SLL and FL – The NCCN
added COPIKTRA to the Clinical Practice Guidelines in Oncology (NCCN
Guidelines), the standard physician resource for determining the
appropriate course of treatment for patients. The Company believes
these updated guidelines will increase awareness for COPIKTRA and help
health care providers make informed decisions for patients battling
these difficult to treat advanced cancers.
Phase 3 DUO Study Results Published in the Journal Blood – The
results of the randomized, multicenter, open-label Phase 3 DUO™ study
(NCT02004522), which evaluated COPIKTRA versus ofatumumab in patients
with relapsed or refractory CLL/SLL, were published in the
peer-reviewed journal Blood (Flinn at al). The publication was
accompanied by a review article by Jennifer R. Brown, M.D., Ph.D.,
Director of the Chronic Lymphocytic Leukemia Center at Dana-Farber
Cancer Center, discussing the role of PI3K inhibitors and duvelisib in
current CLL therapy. The full manuscript titled “The phase 3 DUO
trial: duvelisib versus ofatumumab in relapsed and refractory
CLL/SLL,” is available at www.bloodjournal.org.
Eight Abstracts Presented at the American Society of Hematology
2018 Annual Meeting (ASH 2018) – The Company presented eight
abstracts, including one oral presentation, at ASH 2018 in San Diego.
The oral presentation highlighted data from the Phase 1 study
evaluating duvelisib in combination with romidepsin in relapsed or
refractory peripheral T-cell lymphoma. Additional poster presentations
showcased preclinical and clinical data reinforcing the potential of
Signed Exclusive License Agreements in China and Japan –
Verastem Oncology entered into exclusive license agreements with CSPC
Pharmaceutical Group Limited (CSPC) to develop and commercialize
COPIKTRA in China, Hong Kong, Macau and Taiwan, and Yakult Honsha Co.,
Ltd. (Yakult) to develop and commercialize COPIKTRA in Japan. Both
agreements are for the treatment, prevention or diagnosis of all
Under the terms of the agreement with CSPC, Verastem Oncology
received an upfront payment of $15 million and is entitled to
receive aggregate payments of up to $160 million if certain
development, regulatory and commercial milestones are successfully
achieved, plus double-digit royalties on net sales of products
containing duvelisib in the CSPC Territory. CSPC is a leading
pharmaceutical group in China.
The transaction with Yakult carries a total deal value of up to
$100 million, includes a one-time upfront payment of $10 million
and up to an additional $90 million if certain future
pre-specified development, regulatory and commercial milestones
are successfully achieved by Yakult. In addition, Verastem
Oncology is also eligible to receive double-digit royalties based
on future net sales of duvelisib in Japan.
Collaboration with The Leukemia & Lymphoma Society for Development
of Duvelisib in Peripheral T-Cell Lymphoma – Duvelisib was
selected for The Leukemia & Lymphoma Society’s (LLS) Therapy
Acceleration Program® (TAP) which provides additional resources to
support the development of therapies for patients with blood cancers.
The Company plans to use the TAP funds to conduct certain
translational and clinical activities relating to the development of
duvelisib for the treatment of Peripheral T-Cell Lymphoma (PTCL). LLS
and Verastem Oncology will share the cost of the PTCL development
program, portions of which will be conducted in collaboration with
Memorial Sloan Kettering Cancer Center, The Dana-Farber Cancer
Institute, The Washington University in St. Louis and Stanford
Entering 2019 with a Strong Balance Sheet– In May 2018,
Verastem Oncology successfully completed multiple fundraising
transactions, including an underwritten registered offering in May
2018, a registered offering in June 2018, and a registered direct
offering of 5.00% convertible senior notes in October 2018. The
Company also raised funds through the sale of shares of common stock
under its at-the-market equity offering program. The Company has
approximately $279 million in cash and cash equivalents pro-forma to
the close of the third quarter of 20181.
Verastem Oncology’s 2019 focus is to execute on business priorities
aimed at increasing the company’s sales and revenues:
Continuing to expand on the commercial traction of COPIKTRA in CLL/SLL
and FL for appropriate patients;
Expansion of the open-label, multicenter, Phase 2 clinical trial (the
PRIMO study) evaluating the efficacy and safety of duvelisib
monotherapy in adult patients with histologically confirmed relapsed
or refractory PTCL. This study is expected to enroll approximately 120
Initiating a confirmatory Phase 3 study evaluating duvelisib for the
treatment of patients with relapsed or refractory FL after at least
two prior systemic therapies. The confirmatory study is expected to
start in the second half of 2019;
Initiating additional investigational studies of duvelisib as a
monotherapy and in combination with other anti-cancer agents, such as
checkpoint inhibitors, in both hematological and solid tumor
Working with the LLS to advance the PTCL program including the
expansion of the Phase 2 combination study of duvelisib and romidepsin
for patients with relapsed or refractory PTCL;
Additional ex-U.S. partnerships for duvelisib;
Presenting and publishing additional duvelisib data; and
Advancing the Company’s focal adhesion kinase (FAK) inhibitor
defactinib, which is designed to treat cancer through modulation of
the tumor microenvironment and enhancement of anti-tumor immunity.
Defactinib 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, non-small cell lung cancer (NSCLC), and mesothelioma.
For more information about Verastem Oncology, including its leadership,
product and pipeline, please visit verastem.com
COPIKTRA™ (duvelisib)- Select Important Safety Information
WARNING: FATAL AND SERIOUS TOXICITIES: INFECTIONS, DIARRHEA OR COLITIS,
CUTANEOUS REACTIONS, and PNEUMONITIS
See full prescribing information for complete boxed warning
Fatal and/or serious infections occurred in 31% of COPIKTRA-treated
patients. Monitor for signs and symptoms of infection. Withhold
COPIKTRA if infection is suspected.
Fatal and/or serious diarrhea or colitis occurred in 18% of
COPIKTRA-treated patients. Monitor for the development of severe
diarrhea or colitis. Withhold COPIKTRA.
Fatal and/or serious cutaneous reactions occurred in 5% of
COPIKTRA-treated patients. Withhold COPIKTRA.
Fatal and/or serious pneumonitis occurred in 5% of COPIKTRA-treated
patients. Monitor for pulmonary symptoms and interstitial infiltrates.
WARNINGS AND PRECAUTIONS
Hepatotoxicity: Monitor hepatic function.
Neutropenia: Monitor blood counts.
Embryo-Fetal toxicity: COPIKTRA can cause fetal harm. Advise patients
of potential risk to a fetus and to use effective contraception.
The most common adverse reactions (> 20%) are diarrhea or colitis,
neutropenia, rash, fatigue, pyrexia, cough, nausea, upper respiratory
infection, pneumonia, musculoskeletal pain, and anemia. (6)
To report SUSPECTED ADVERSE REACTIONS, contact Verastem, Inc.
(Verastem) at 877-7RXVSTM or 1-877-779-8786, or U.S. Food and Drug
Administration (FDA) at 1-800-FDA-1088 or www.fda.gov/medwatch.
CYP3A inducers: Avoid co-administration with strong CYP3A inducers.
CYP3A inhibitors: Monitor for COPIKTRA toxicities when co-administered
with strong or moderate CYP3A inhibitors. Reduce COPIKTRA dose to 15
mg twice daily when co-administered with strong CYP3A4 inhibitors.
CYP3A substrates: Monitor for signs of toxicities when co-administering
COPIKTRA with sensitive CYP3A substrates.
About Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL)
are cancers that affect lymphocytes and are essentially the same
disease, with the only difference being the location where the cancer
primarily occurs. When most of the cancer cells are located in the
bloodstream and the bone marrow, the disease is referred to as CLL,
although the lymph nodes and spleen are often involved. When the cancer
cells are located mostly in the lymph nodes, the disease is called SLL.
The symptoms of CLL/SLL include a tender, swollen abdomen and feeling
full even after eating only a small amount. Other symptoms can include
fatigue, shortness of breath, anemia, bruising easily, night sweats,
weight loss, and frequent infections. However, many patients with
CLL/SLL will live for years without symptoms. There are approximately
200,000 patients in the US affected by CLL/SLL with nearly 20,000 new
diagnoses this year alone. While there are therapies currently
available, real-world data reveals that a significant number of patients
either relapse following treatment, become refractory to current agents,
or are unable to tolerate treatment, representing a significant medical
need. The potential of additional oral agents, particularly as a
monotherapy that can be used in the general community physician’s
armamentarium, may hold significant value in the treatment of patients
About Follicular Lymphoma
Follicular lymphoma (FL) is typically a slow-growing or indolent form of
non-Hodgkin lymphoma (NHL) that arises from B-lymphocytes, making it a
B-cell lymphoma. This lymphoma subtype accounts for 20 to 30 percent of
all NHL cases, with more than 140,000 people in the US with FL and more
than 13,000 newly diagnosed patients this year. Common symptoms of FL
include enlargement of the lymph nodes in the neck, underarms, abdomen,
or groin, as well as fatigue, shortness of breath, night sweats, and
weight loss. Often, patients with FL have no obvious symptoms of the
disease at diagnosis. Follicular lymphoma is usually not considered to
be curable, but more of a chronic disease, with patients living for many
years with this form of lymphoma. The potential of additional oral
agents, particularly as a monotherapy that can be used in the general
community physician’s armamentarium, may hold significant value in the
treatment of patients with FL.
About Peripheral T-Cell Lymphoma
Peripheral T-cell lymphoma (PTCL) is a rare, aggressive type of
non-Hodgkin lymphoma (NHL) that develops in mature white blood cells
called “T cells” and “natural killer (NK) cells”2 which
circulate with the lymphatic system.3 PTCL accounts for
between 10-15% of all non-Hodgkin lymphomas (NHLs) and generally affects
people aged 60 years and older.2 Although there are many
different subtypes of peripheral T-cell lymphoma, they often present in
a similar way, with widespread, enlarged, painless lymph nodes in the
neck, armpit or groin.3 There is currently no established
standard of care for patients with relapsed or refractory disease.2
About COPIKTRA™ (duvelisib)
COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and
the first approved dual inhibitor of PI3K-delta and PI3K-gamma, two
enzymes known to help support the growth and survival of malignant
B-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.4,5,6 COPIKTRA is
indicated for the treatment of adult patients with relapsed or
refractory chronic lymphocytic leukemia/small lymphocytic lymphoma
(CLL/SLL) after at least two prior therapies and relapsed or refractory
follicular lymphoma (FL) after at least two prior systemic therapies.
COPIKTRA is also being developed by Verastem Oncology for the treatment
of peripheral T-cell lymphoma (PTCL), for which it has received Fast
Track status, and is being investigated in combination with other agents
through investigator-sponsored studies.7 For more information
on COPIKTRA, please visit www.COPIKTRA.com.
Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.
Defactinib is an investigational inhibitor of focal adhesion kinase
(FAK), a non-receptor tyrosine kinase that mediates oncogenic signaling
in response to cellular adhesion and growth factors.8 Based
on the multi-faceted roles of FAK, defactinib is used to treat cancer
through modulation of the tumor microenvironment and enhancement of
anti-tumor immunity.9,10 Defactinib 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. These studies are combination clinical
trials with pembrolizumab and avelumab from Merck & Co. and Pfizer/Merck
KGaA, respectively.11,12,13 Information about these and
additional clinical trials evaluating the safety and efficacy of
defactinib can be found on www.clinicaltrials.gov.
About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a commercial biopharmaceutical
company committed to the development and commercialization of medicines
to improve the lives of patients diagnosed with cancer. We are driven by
the strength, tenacity and courage of those battling cancer –
single-minded in our resolve to deliver new therapies that not only keep
cancer at bay, but improve the lives of patients diagnosed with cancer.
Because for us, it’s personal.
Our first FDA approved product is now available for the treatment of
patients with certain types of indolent non-Hodgkin’s lymphoma (iNHL).
Our pipeline comprises product candidates that seek to treat cancer by
modulating the local tumor microenvironment. 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 lead product duvelisib, and Verastem Oncology’s PI3K and FAK
programs generally, its intent to commercialize duvelisib, the potential
commercial success of duvelisib, the anticipated adoption of duvelisib
by patients and physicians, the structure of its planned and pending
clinical trials and the timeline and indications for clinical
development, regulatory submissions and commercialization activities.
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,
among other things, uncertainties regarding the commercial success of
duvelisib in the United States; uncertainties regarding physician and
patient adoption of duvelisib, including those related to the safety and
efficacy of duvelisib; the uncertainties inherent in research and
development of duvelisib, such as negative or unexpected results of
clinical trials; whether and when any applications for duvelisib may be
filed with regulatory authorities in any other jurisdictions; whether
and when regulatory authorities in any other jurisdictions may approve
any such other applications that may be filed for duvelisib, which will
depend on the assessment by such regulatory authorities of the
benefit-risk profile suggested by the totality of the efficacy and
safety information submitted and, if approved, whether duvelisib will be
commercially successful in such jurisdictions; Verastem Oncology’s
ability to obtain, maintain and enforce patent and other intellectual
property protection for duvelisib and its other product candidates; the
scope, timing, and outcome of any legal proceedings; decisions by
regulatory authorities regarding labeling and other matters that could
affect the availability or commercial potential of duvelisib; that
regulatory authorities in the U.S. or other jurisdictions, if approved,
could withdraw approval; whether preclinical testing
of Verastem Oncology’s product candidates and preliminary or interim
data from clinical trials will be predictive of the results or success
of ongoing or later clinical trials; that the timing, scope and rate of
reimbursement for Verastem Oncology’s product candidates is uncertain;
the risk that third party payors (including government agencies) will
not reimburse for duvelisib; that there may be competitive developments
affecting its product candidates; that data may not be available when
expected; that enrollment of clinical trials may take longer than
expected; that duvelisib or Verastem Oncology’s other product candidates
will cause unexpected safety events, experience manufacturing or supply
interruptions or failures, or result in unmanageable safety profiles as
compared to their levels 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 FL in other jurisdictions; 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 Quarterly Report on Form 10-Q for the
quarterly period ended September 30, 2018 as filed with the Securities
and Exchange Commission (SEC) on November 7, 2018, its Annual Report on
Form 10-K for the year ended December 31, 2017 as filed with
the 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.
1 Pro-forma cash and cash
equivalents represents cash and cash equivalents at September 30, 2018
of $145.6 million, plus estimated net proceeds of $145.1 million from
the issuance of our 5.00% Convertible Senior Notes in October 2018, and
the remaining $10.0 million of the $15.0M non-refundable upfront payment
due from CSPC pursuant to the exclusive license agreement executed in
September 2018, less the $22.0 million payment the Company owes to
Infinity Pharmaceuticals, Inc. pursuant to the terms of the amended and
restated license agreement.
2 The Leukemia & Lymphoma
Society. Peripheral T-Cell Lymphoma Facts. July 2014.
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.
5 Reif K et al. Cutting Edge:
Differential Roles for Phosphoinositide 3 kinases, p110-gamma and
p110-delta, in lymphocyte chemotaxis and homing. J Immunol
6 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
8 Schaller M.D. and Parsons J.T. Focal
adhesion kinase: an integrin-linked protein tyrosine kinase. Trends Cell
Biol. 1993 3: 258-62.
9 Jiang H et al. Targeting focal
adhesion kinase renders pancreatic cancers responsive to checkpoint
immunotherapy. Nat Med 2016: Aug 22(8) 851-60.
Sulzmaier F.J. et al. FAK in cancer: mechanistic findings and clinical
applications. Nature Rev Cancer. 2014 14: 598-610.
View source version on businesswire.com: https://www.businesswire.com/news/home/20190103005706/en/
Source: Verastem, Inc.