U.S. Commercial Launch of COPIKTRA™
Underway
$1.7 Million in 2018 Net Product Revenues from
COPIKTRA
Cash, Cash Equivalents and Short-Term
Investments of $249.7 million as December 31, 2018
Verastem, Inc. (Nasdaq:VSTM), operating as Verastem Oncology,
(or “the Company”), focused on developing and commercializing
medicines to improve the survival and quality of life of cancer
patients, today reported financial results for the three and twelve
months ended December 31, 2018, including revenue from its first
commercial product, COPIKTRA™ (duvelisib), which was approved by
the U.S. Food and Drug Administration (FDA) on September 24,
2018.
“Upon the early FDA approval we received, our commercial team
was mobilized the same day and began educating physicians, patients
and payors on the clinical benefits and appropriate use of COPIKTRA
and to secure access to therapy,” said Robert Forrester, President
and Chief Executive Officer of Verastem Oncology. “This year is
poised to be an exciting one as we continue to drive awareness of
COPIKTRA and work to expand upon the potential of PI3K inhibition
through the investigation of duvelisib, initially as a monotherapy,
and through novel combinations, in additional hematologic
malignancies like peripheral T-cell lymphoma (PTCL).”
“Following FDA approval, COPIKTRA was quickly added to the
National Comprehensive Cancer Network® (NCCN) guidelines, and as of
December 31, 2018, we had secured formulary listing and
reimbursement for approximately 75% of targeted health plans. As of
March 11, 2019, that number increased to 90%, underscoring our
efforts to provide access to treatment for appropriate patients,”
said Joseph Lobacki, Executive Vice President and Chief Commercial
Officer of Verastem Oncology. “Looking ahead to 2019, we are
focused on further identifying appropriate patients for treatment
with COPIKTRA, and we intend to continue to work with the leukemia
and lymphoma community to increase awareness and help ensure
physicians and patients are able to get the support they need.”
Key 2018 Accomplishments:
- Launched COPIKTRA in the United
States – Verastem Oncology launched COPIKTRA, an oral inhibitor
of phosphoinositide 3-kinase (PI3K), and the first approved dual
inhibitor of PI3K-delta and PI3K-gamma, in the United States
following FDA approval 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. Accelerated approval
in FL was based on overall response rate and continued approval may
be contingent upon verification and description of clinical benefit
in confirmatory trials, the first of which is expected to start in
2019.
Use 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 has implemented a Risk Evaluation and Mitigation Strategy
to provide appropriate dosing and safety information to better
support physicians in managing their patients on COPIKTRA.
Additionally, use of COPIKTRA is associated
with additional adverse reactions which may also require dose
reduction, treatment delay or discontinuation of COPIKTRA.
Please see
www.COPIKTRAHCP.com/prescribinginformation for full Prescribing
Information including BOXED WARNING and Medication Guide in
addition to the Important Safety Information provided below.
- COPIKTRA Added to NCCN Guidelines
for CLL/SLL, FL and Marginal Zone Lymphoma (MZL) – 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.
COPIKTRA is not approved for use in MZL.
- Presented COPIKTRA Data at the
23rd Annual International Congress on Hematologic
Malignancies (ICHM) – The Company presented four COPIKTRA
abstracts at ICHM 2019, including an abstract highlighting Phase 3
DUO data in patients with relapsed or refractory CLL/SLL who have
progressed following two prior lines of the therapy. This is the
same indication for which COPIKTRA received approval from the FDA
in September 2018. In this analysis, COPIKTRA demonstrated
progression-free survival (PFS) of 16.4 months and an ORR of 78%,
with a manageable safety profile. The remaining three abstracts
featured data from a long-term (>2 years) efficacy and safety
analysis, the Phase 3 DUO crossover extension study, and prognostic
and immune-related factors associated with response to duvelisib
from the Phase 2 DYNAMO™ study in indolent non-Hodgkin’s lymphoma
(iNHL). Collectively, the data presented at ICHM 2019 continue to
support the use of COPIKTRA in its approved indications of relapsed
or refractory CLL/SLL after at least two prior therapies and FL
after at least two prior systemic therapies. PDF copies of all of
the ICHM 2019 poster presentations are available here.
- Presented Updated Duvelisib
Combination Data in PTCL at the American Society of Hematology 2018
Annual Meeting (ASH 2018) – The oral presentation highlighted
updated data from an investigator-sponsored Phase 1 study
evaluating duvelisib in combination with romidepsin in relapsed or
refractory T-cell lymphomas, including PTCL and cutaneous T-cell
lymphoma (CTCL). Of the 27 patients with PTCL evaluable for
efficacy, 16 responded (9 complete responses (CRs) and 7 partial
responses (PRs)) for an overall response rate (ORR) of 59%.
Importantly, of the 27 patients with PTCL treated with the
combination of duvelisib and romidepsin, 6 (22%) responded deeply
enough to allow them to bridge to potentially curative stem cell
transplant (SCT). Median progression-free survival (PFS) for
patients with PTCL was 6.72 months, which was confounded by 6
subjects that proceeded to SCT. Among the 31 patients at the
maximum tolerated dose who were evaluable for safety, the most
common Grade ≥3 adverse events occurring in ≥10% of patients were
neutropenia (32%), diarrhea (19%), increased transaminase (23%;
alanine aminotransferase 16% and aspartate aminotransferase 6%),
hyponatremia (13%) and platelet count decrease (10%).
- Presented Frontline Duvelisib
Combination Data in Younger CLL Patients at European Hematology
Association 2018 Annual Meeting (EHA 2018) – Dr. Matthew
Davids, M.D., MMSc, Assistant Professor of Medicine, Harvard
Medical School, and Associate Director, Center for Chronic
Lymphocytic Leukemia at the Dana-Farber Cancer Institute, presented
Phase 1b/2 clinical data from 31 patients who received duvelisib in
combination with fludarabine (F), cyclophosphamide (C), and
rituximab (R) (dFCR) as frontline therapy. The ORR was 94%, with
26% (n=8) of patients achieving a CR or CRi, and 68% achieving a
PR. The best rate of minimum residual disease (MRD) negativity in
the bone marrow (BM) in patients with at least one evaluation was
76% (22 of 29 patients). The two-year progression-free survival and
overall survival rates for patients in the study were both 97%. The
recommended Phase 2 dose for duvelisib in combination with FCR was
established as 25mg twice daily. The most common all grade
non-hematologic adverse events (AEs) were nausea (72%, all Grade
1/2), fatigue (69%, 3% Grade 3), fever (53%, all Grade 1/2),
diarrhea (47%, 3% Grade 3), transaminitis (34%, 28% Grade 3/4),
anorexia (34%, all Grade 1/2), vomiting (28%, all Grade 1/2),
pruritus (16%, 3% Grade 3), arthritis (9%, all Grade 2) and
Cytomegalovirus (CMV) reactivation (6%, both Grade 2). The most
common all grade hematologic adverse events were thrombocytopenia
(65%; 34% Grade 3/4), neutropenia (59%; 50% Grade 3/4), and anemia
(38%, 16% Grade 3/4). Serious AEs included febrile neutropenia
(n=6, all Grade 3) and pneumonia (n=6, including 3 cases of PJP
despite planned prophylaxis).
- Investigator-Sponsored Study
Initiated Evaluating COPIKTRA in Combination with Venetoclax –
In early September 2018, the first patient was dosed in a
multicenter Phase 1/2 clinical trial investigating COPIKTRA in
combination with venetoclax, an oral selective inhibitor of BCL-2,
in patients with relapsed or refractory CLL/SLL. Preclinical data
support this combination, as COPIKTRA has been shown to upregulate
BCL-2 transcript and protein expression levels and potentially
enhance the ability of venetoclax to induce apoptosis in ex vivo
human CLL cells. The primary objectives of the Phase 1 portion of
the trial are to determine the maximum tolerated dose and the
recommended Phase 2 dose of venetoclax for this combination
regimen. The trial is being led by Dr. Matthew Davids.
- 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 (collectively, the CSPC Territory), and Yakult Honsha Co.,
Ltd. (Yakult) to develop and commercialize COPIKTRA in Japan. Both
agreements are for the treatment, prevention or diagnosis of all
oncology indications.
- Under the terms of the agreement with
CSPC, Verastem Oncology received an upfront payment of $15.0
million and is entitled to receive aggregate payments of up to
$160.0 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 $100.0 million, includes a one-time
upfront payment of $10.0 million and up to an additional $90.0
million if certain 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 products containing
duvelisib in Japan. Yakult has a strong presence in development and
commercialization of therapeutic products in the field of oncology
and markets several branded anti-cancer therapies, including
Elplat® and Campto®.
- Collaboration with The Leukemia
& Lymphoma Society for Development of Duvelisib in PTCL –
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 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
University.
- 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 et al). The publication was accompanied by a review
article by Jennifer R. Brown, M.D., Ph.D., Director of the Center
for Chronic Lymphocytic Leukemia at the Dana-Farber Cancer
Institute, 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.
- Entering 2019 with Cash, Cash
Equivalents and Short-Term Investments of $249.7 Million –
During 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 (Convertible Senior Notes). The Company also raised
funds through the sale of shares of common stock under its
at-the-market equity offering program. These fundraising
transactions helped to provide the Company with a strong cash, cash
equivalents and short-term investments balance of $249.7 million as
of December 31, 2018.
- Key Commercial, Clinical and
Investor Relations Team Additions – In February 2019, the
Company expanded its commercial and clinical teams through the
appointment of several new employees, including Amy Cavers as
Senior Vice President, Strategic Engagement and Alignment, Robert
Morgan as Senior Vice President, Development Operations, and Erin
Cox, Senior Director, Investor Relations and Corporate
Communications.
Selected posters and presentations are available within the
“Media” section of the Company’s website at www.verastem.com.
Fourth Quarter 2018 Financial Results
Net loss for the three months ended December 31, 2018 (2018
Quarter) was $11.3 million, or $0.15 per share (basic), as compared
to $18.2 million, or $0.43 per share (basic), for the three months
ended December 31, 2017 (2017 Quarter). Net loss for the 2018
Quarter includes a non-cash gain of $25.6 million, or $0.35 per
share (basic), relating to the accounting impact of a financial
derivative related to our Convertible Senior Notes. In addition,
net loss includes non-cash stock-based compensation expense of $1.8
million and $1.0 million for the 2018 and 2017 Quarters,
respectively.
Net product revenue for the 2018 Quarter was $1.2 million which
reflects the first full quarter of recorded sales for COPIKTRA. The
Company did not have any product revenue for the 2017 Quarter as
the FDA approved COPIKTRA on September 24, 2018.
Research and development expense for the 2018 Quarter was $8.8
million compared to $11.3 million for the 2017 Quarter, a decrease
of $2.5 million or 22%, primarily related to lower R&D costs
associated with the development of COPIKTRA.
Selling, general and administrative expense for the 2018 Quarter
was $26.2 million compared to $6.8 million for the 2017 Quarter.
The increase of $19.4 million, or 285%, from the 2017 Quarter to
the 2018 Quarter was due to higher personnel and related costs, as
well as promotional and consulting costs in support of the
commercial launch of COPIKTRA.
Other income of $25.6 million for the 2018 Quarter relates
entirely to a non-cash gain for the accounting impact of a
financial derivative related to our Convertible Senior Notes.
Full-Year 2018 Financial Results
Net loss for the year ended December 31, 2018 (2018 Period) was
$72.4 million, or $1.12 per share (basic), as compared to $67.8
million, or $1.76 per share (basic), for the year ended December
31, 2017 (2017 Period). Net loss for the 2018 Period includes a
non-cash gain of $25.6 million, or $0.39 per share (basic),
relating to the accounting impact of a financial derivative related
to our Convertible Senior Notes. In addition, net loss includes
non-cash stock-based compensation expense of $6.7 million and $5.0
million for the 2018 and 2017 Periods, respectively.
Total revenue for the 2018 Period was $26.7 million which
reflects net product revenue of $1.7 million for sales of COPIKTRA
and license revenue of $25.0 million relating to our license
agreements with Yakult and CSPC. The Company did not have any
product revenue for the 2017 Period as the FDA approved COPIKTRA on
September 24, 2018. The Company did not have any license revenue
for the 2017 Period.
Research and development expense for the 2018 Period was $43.6
million compared to $46.4 million for the 2017 Period. The decrease
of $2.8 million, or 6%, from the 2017 Period to the 2018 Period was
primarily related to a decrease of $6.0 million in license
fees related to a one-time milestone earned pursuant to our
Infinity license agreement which was recognized in the 2017 Period,
offset, in part, by a net increase of $3.2 million in personnel
related costs, including non-cash stock-based compensation,
clinical trial costs and consulting fees for COPIKTRA.
Selling, general and administrative expense for the 2018 Period
was $77.3 million compared to $21.4 million for the 2017
Period. The increase of $55.9 million, or 261%, from the 2017
Period to the 2018 Period primarily resulted from higher personnel
and related costs, promotional and consulting costs in support of
the commercial launch of COPIKTRA.
Other income of $25.6 million for the 2018 Period relates
entirely to a non-cash gain for the accounting impact of a
financial derivative related to our Convertible Senior Notes.
In October 2018, the Company completed an offering of 5.00%
Convertible Senior Notes due 2048 through a registered direct
offering. The Company received net proceeds of $145.3 million,
after transaction fees and expenses. Verastem Oncology ended 2018
with cash, cash equivalents and short-term investments of $249.7
million.
For more information about Verastem Oncology, including its
leadership, product and pipeline, please visit verastem.com
Important Safety Information
WARNING: FATAL AND SERIOUS TOXICITIES: INFECTIONS, DIARRHEA OR
COLITIS, CUTANEOUS REACTIONS, and PNEUMONITIS
- 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. Withhold COPIKTRA.
WARNINGS AND PRECAUTIONS
Infections: Serious, including fatal (18/442; 4%),
infections occurred in 31% of patients receiving COPIKTRA 25 mg BID
(N=442). The most common serious infections were pneumonia, sepsis,
and lower respiratory infections. Median time to onset of any grade
infection was 3 months (range: 1 day to 32 months), with 75% of
cases occurring within 6 months. Treat infections prior to
initiation of COPIKTRA. Advise patients to report new or worsening
signs and symptoms of infection. For grade 3 or higher infection,
withhold COPIKTRA until infection has resolved. Resume COPIKTRA at
the same or reduced dose.
Serious, including fatal, Pneumocystis jirovecii pneumonia (PJP)
occurred in 1% of patients taking COPIKTRA. Provide prophylaxis for
PJP during treatment with COPIKTRA and following completion of
treatment with COPIKTRA until the absolute CD4+ T cell count is
greater than 200 cells/μL. Withhold COPIKTRA in patients with
suspected PJP of any grade, and permanently discontinue if PJP is
confirmed.
Cytomegalovirus (CMV) reactivation/infection occurred in 1% of
patients taking COPIKTRA. Consider prophylactic antivirals during
COPIKTRA treatment to prevent CMV infection including CMV
reactivation. For clinical CMV infection or viremia, withhold
COPIKTRA until infection or viremia resolves. If COPIKTRA is
resumed, administer the same or reduced dose and monitor patients
for CMV reactivation by PCR or antigen test at least monthly.
Diarrhea or Colitis: Serious, including fatal (1/442;
<1%), diarrhea or colitis occurred in 18% of patients receiving
COPIKTRA 25 mg BID (N=442). Median time to onset of any grade
diarrhea or colitis was 4 months (range: 1 day to 33 months), with
75% of cases occurring by 8 months. The median event duration was
0.5 months (range: 1 day to 29 months; 75th percentile: 1
month).
Advise patients to report any new or worsening diarrhea. For
patients presenting with mild or moderate diarrhea (Grade 1-2)
(i.e., up to 6 stools per day over baseline) or asymptomatic (Grade
1) colitis, initiate supportive care with antidiarrheal agents,
continue COPIKTRA at the current dose, and monitor the patient at
least weekly until the event resolves. If the diarrhea is
unresponsive to antidiarrheal therapy, withhold COPIKTRA and
initiate supportive therapy with enteric acting steroids (e.g.,
budesonide). Monitor the patient at least weekly. Upon resolution
of the diarrhea, consider restarting COPIKTRA at a reduced
dose.
For patients presenting with abdominal pain, stool with mucus or
blood, change in bowel habits, peritoneal signs, or with severe
diarrhea (Grade 3) (i.e., > 6 stools per day over baseline),
withhold COPIKTRA and initiate supportive therapy with enteric
acting steroids (e.g., budesonide) or systemic steroids. A
diagnostic work-up to determine etiology, including colonoscopy,
should be performed. Monitor at least weekly. Upon resolution of
the diarrhea or colitis, restart COPIKTRA at a reduced dose. For
recurrent Grade 3 diarrhea or recurrent colitis of any grade,
discontinue COPIKTRA. Discontinue COPIKTRA for life-threatening
diarrhea or colitis.
Cutaneous Reactions: Serious, including fatal (2/442;
<1%), cutaneous reactions occurred in 5% of patients receiving
COPIKTRA 25 mg BID (N=442). Fatal cases included drug reaction with
eosinophilia and systemic symptoms (DRESS) and toxic epidermal
necrolysis (TEN). Median time to onset of any grade cutaneous
reaction was 3 months (range: 1 day to 29 months, 75th percentile:
6 months) with a median event duration of 1 month (range: 1 day to
37 months, 75th percentile: 2 months).
Presenting features for the serious events were primarily
described as pruritic, erythematous, or maculo-papular. Less common
presenting features include exanthem, desquamation, erythroderma,
skin exfoliation, keratinocyte necrosis, and papular rash. Advise
patients to report new or worsening cutaneous reactions. Review all
concomitant medications and discontinue any medications potentially
contributing to the event. For patients presenting with mild or
moderate (Grade 1-2) cutaneous reactions, continue COPIKTRA at the
current dose, initiate supportive care with emollients,
antihistamines (for pruritus), or topical steroids, and monitor the
patient closely. Withhold COPIKTRA for severe (Grade 3) cutaneous
reaction until resolution. Initiate supportive care with steroids
(topical or systemic) or antihistamines (for pruritus). Monitor at
least weekly until resolved. Upon resolution of the event, restart
COPIKTRA at a reduced dose. Discontinue COPIKTRA if severe
cutaneous reaction does not improve, worsens, or recurs. For
life-threatening cutaneous reactions, discontinue COPIKTRA. In
patients with SJS, TEN, or DRESS of any grade, discontinue
COPIKTRA.
Pneumonitis: Serious, including fatal (1/442; <1%),
pneumonitis without an apparent infectious cause occurred in 5% of
patients receiving COPIKTRA 25 mg BID (N=442). Median time to onset
of any grade pneumonitis was 4 months (range: 9 days to 27 months),
with 75% of cases occurring within 9 months. The median event
duration was 1 month, with 75% of cases resolving by 2 months.
Withhold COPIKTRA in patients with new or progressive pulmonary
signs and symptoms such as cough, dyspnea, hypoxia, interstitial
infiltrates on a radiologic exam, or a decline by more than 5% in
oxygen saturation, and evaluate for etiology. If the pneumonitis is
infectious, patients may be restarted on COPIKTRA at the previous
dose once the infection, pulmonary signs and symptoms resolve. For
moderate non-infectious pneumonitis (Grade 2), treat with systemic
corticosteroids and resume COPIKTRA at a reduced dose upon
resolution. If non-infectious pneumonitis recurs or does not
respond to steroid therapy, discontinue COPIKTRA. For severe or
life-threatening non-infectious pneumonitis, discontinue COPIKTRA
and treat with systemic steroids.
Hepatotoxicity: Grade 3 and 4 ALT and/or AST elevation
developed in 8% and 2%, respectively, of patients receiving
COPIKTRA 25 mg BID (N=442). Two percent of patients had both an ALT
or AST > 3 X ULN and total bilirubin > 2 X ULN. Median time
to onset of any grade transaminase elevation was 2 months (range: 3
days to 26 months), with a median event duration of 1 month (range:
1 day to 16 months).
Monitor hepatic function during treatment with COPIKTRA. For
Grade 2 ALT/AST elevation (> 3 to 5 X ULN), maintain COPIKTRA
dose and monitor at least weekly until return to < 3 X ULN. For
Grade 3 ALT/AST elevation (> 5 to 20 X ULN), withhold COPIKTRA
and monitor at least weekly until return to < 3 X ULN. Resume
COPIKTRA at the same dose (first occurrence) or at a reduced dose
for subsequent occurrences. For grade 4 ALT/AST elevation (> 20
X ULN), discontinue COPIKTRA.
Neutropenia: Grade 3 or 4 neutropenia occurred in 42% of
patients receiving COPIKTRA 25 mg BID (N=442), with Grade 4
neutropenia occurring in 24% of all patients. Median time to onset
of grade ≥3 neutropenia was 2 months (range: 3 days to 31 months),
with 75% of cases occurring within 4 months.
Monitor neutrophil counts at least every 2 weeks for the first 2
months of COPIKTRA therapy, and at least weekly in patients with
neutrophil counts < 1.0 Gi/L (Grade 3-4). Withhold COPIKTRA in
patients presenting with neutrophil counts < 0.5 Gi/L (Grade 4).
Monitor until ANC is > 0.5 Gi/L, then resume COPIKTRA at same
dose for the first occurrence or at a reduced dose for subsequent
occurrences.
Embryo-Fetal Toxicity: Based on findings in animals and
its mechanism of action, COPIKTRA can cause fetal harm when
administered to a pregnant woman. Advise pregnant women of the
potential risk to a fetus. Conduct pregnancy testing before
initiating COPIKTRA treatment. Advise females of reproductive
potential and males with female partners of reproductive potential
to use effective contraception during treatment and for at least 1
month after the last dose.
ADVERSE REACTIONS
B-cell Malignancies Summary
Fatal adverse reactions within 30 days of the last dose occurred
in 8% (36/442) of patients treated with COPIKTRA 25 mg BID. Serious
adverse reactions were reported in 289 patients (65%). The most
frequent serious adverse reactions that occurred were infection
(31%), diarrhea or colitis (18%), pneumonia (17%), rash (5%), and
pneumonitis (5%).
Adverse reactions resulted in treatment discontinuation in 156
patients (35%) most often due to diarrhea or colitis, infection,
and rash. COPIKTRA was dose reduced in 104 patients (24%) due to
adverse reactions, most often due to diarrhea or colitis and
transaminase elevation. The most common adverse reactions (reported
in ≥ 20% of patients) were diarrhea or colitis, neutropenia, rash,
fatigue, pyrexia, cough, nausea, upper respiratory infection,
pneumonia, musculoskeletal pain and anemia.
CLL/SLL: Fatal adverse reactions within 30 days of the
last dose occurred in 12% (19/158) of patients treated with
COPIKTRA and in 4% (7/155) of patients treated with ofatumumab.
Serious adverse reactions were reported in 73% (115/158) of
patients treated with COPIKTRA and most often involved infection
(38%; 60/158) and diarrhea or colitis (23%; 36/158). COPIKTRA was
discontinued in 57 patients (36%), most often due to diarrhea or
colitis, infection, and rash. COPIKTRA was dose reduced in 46
patients (29%) due to adverse reactions, most often due to diarrhea
or colitis and rash. The most common adverse reactions with
COPIKTRA (reported in ≥20% of patients) were diarrhea or colitis,
neutropenia, pyrexia, upper respiratory tract infection, pneumonia,
rash, fatigue, nausea, anemia and cough.
FL: Serious adverse reactions were reported in 58% of
patients and most often involved diarrhea or colitis, pneumonia,
renal insufficiency, rash, and sepsis. The most common adverse
reactions (≥20% of patients) were diarrhea or colitis, nausea,
fatigue, musculoskeletal pain, rash, neutropenia, cough, anemia,
pyrexia, headache, mucositis, abdominal pain, vomiting,
transaminase elevation, and thrombocytopenia. Adverse reactions
resulted in COPIKTRA discontinuation in 29% of patients, most often
due to diarrhea or colitis and rash. COPIKTRA was dose reduced in
23% due to adverse reactions, most often due to transaminase
elevation, diarrhea or colitis, lipase increased and infection.
DRUG INTERACTIONS
- CYP3A Inducers: Coadministration with a
strong CYP3A inducer may reduce COPIKTRA efficacy. Avoid
coadministration with strong CYP3A4 inducers.
- CYP3A Inhibitors: Coadministration with
a strong CYP3A inhibitor may increase the risk of COPIKTRA
toxicities. Reduce COPIKTRA dose to 15 mg BID when coadministered
with a strong CYP3A4 inhibitor.
- CYP3A Substrates: Coadministration of
COPIKTRA with sensitive CYP3A4 substrates may increase the risk of
toxicities of these drugs. Consider reducing the dose of the
sensitive CYP3A4 substrate and monitor for signs of toxicities of
the coadministered sensitive CYP3A substrate.
See full Prescribing Information, including Boxed Warning,
at www.COPIKTRA.com
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. In 2018, there were
approximately 200,000 patients in the United States affected by
CLL/SLL with nearly 20,000 new diagnoses. 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 with CLL/SLL.
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. In 2018, this lymphoma subtype
accounted for 20 to 30 percent of all NHL cases, with more than
140,000 people in the United States with FL and more than 13,000
newly diagnosed patients. 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”1 which
circulate with the lymphatic system.2 PTCL accounts for between
10-15% of all non-Hodgkin lymphomas (NHLs) and generally affects
people aged 60 years and older.1 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.2 There is currently no established standard
of care for patients with relapsed or refractory disease.1
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.3,4,5 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.6 For more information on
COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib
clinical trials 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 COPIKTRA, and Verastem Oncology’s PI3K
program generally, its commercialization of COPIKTRA, the potential
commercial success of COPIKTRA, the anticipated adoption of
COPIKTRA 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 the risks and
uncertainties, among other things, regarding: the commercial
success of COPIKTRA in the United States; physician and patient
adoption of COPIKTRA, including those related to the safety and
efficacy of COPIKTRA; the uncertainties inherent in research and
development of COPIKTRA, such as negative or unexpected results of
clinical trials; whether and when any applications for COPIKTRA 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
COPIKTRA, 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 COPIKTRA will be commercially successful in such
jurisdictions; our ability to obtain, maintain and enforce patent
and other intellectual property protection for COPIKTRA and our
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 COPIKTRA; the fact that regulatory
authorities in the U.S. or other jurisdictions, if approved, could
withdraw approval; whether preclinical testing of our 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 our product candidates is uncertain; that third-party payors
(including government agencies) may not reimburse for COPIKTRA;
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
COPIKTRA or our 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 COPIKTRA will be ineffective at
treating patients with lymphoid malignancies; that we will be
unable to successfully initiate or complete the clinical
development and eventual commercialization of our product
candidates; that the development and commercialization of our
product candidates will take longer or cost more than planned; that
we may not have sufficient cash to fund our contemplated
operations; that we, CSPC Pharmaceutical Group, Yakult Honsha Co.,
Ltd. or Infinity Pharmaceuticals, Inc. will fail to fully perform
under the duvelisib license agreements; that we may be unable to
make additional draws under our debt facility or obtain adequate
financing in the future through product licensing, co-promotional
arrangements, public or private equity, debt financing or
otherwise; that we will not pursue or submit regulatory filings for
our product candidates, including for duvelisib in patients with
chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL)
or indolent non-Hodgkin lymphoma (iNHL) in other jurisdictions; and
that our 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 its Annual Report on Form
10-K for the year ended December 31, 2018 as filed with the SEC on
March 12, 2019 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 The Leukemia & Lymphoma Society. Peripheral T-Cell
Lymphoma Facts. July 2014.
2 Leukemia Foundation. http://www.leukaemia.org.au/blood-cancers/lymphomas/non-hodgkin-lymphoma-nhl/peripheral-t-cell-lymphoma
3 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.
4 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.
5 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.
6 www.clinicaltrials.gov, NCT03372057
Verastem, Inc.
Consolidated Balance Sheets
(in thousands)
December 31, December 31, 2018
2017 Cash, cash equivalents and investments $ 249,653
$ 86,672 Accounts receivable, net 306 — Inventory 327 — Prepaid
expenses and other current assets 2,973 1,115 Property and
equipment, net 1,369 861 Intangible assets, net 21,577 — Other
assets 1,031 1,143
Total assets $
277,236 $ 89,791 Accounts payable,
accrued expenses and other current liabilities $ 37,077 $ 17,128
Long-term debt 19,506 14,828 Convertible senior notes 95,231 —
Other liabilities 1,123 151 Stockholders’ equity 124,299
57,684
Total liabilities and stockholders’ equity
$ 277,236 $ 89,791
Verastem, Inc.
Consolidated Statements of
Operations
(in thousands, except per share
amounts)
Three months ended
December 31,
Year ended
December 31,
2018 2017 2018 2017 Revenue: Product
revenue, net $ 1,210 $ — $ 1,718 $ — License revenue —
— 25,000 — Total revenue 1,210 —
26,718 — Operating expenses: Costs of revenues,
excluding amortization of acquired intangible assets 116 — 165 —
Research and development 8,762 11,253 43,648 46,423 Selling,
general and administrative 26,199 6,799 77,265 21,381 Amortization
of acquired intangible assets 392 — 423
— Total operating expenses 35,469 18,052
121,501 67,804 Loss from operations (34,259)
(18,052) (94,783) (67,804) Other income 25,556 —
25,556 — Interest income 1,306 145 2,603 561 Interest expense
(3,952) (328) (5,810) (559) Net loss
$ (11,349) $ (18,235) $
(72,434) $ (67,802) Net loss per share—basic
$ (0.15) $ (0.43) $
(1.12) $ (1.76) Net loss per share—diluted
$ (0.37) $ (0.43) $
(1.37) $ (1.76) Weighted average common shares
outstanding used in computing: Net loss per share—basic
73,766 42,027 64,962 38,422 Net loss
per share—diluted
91,061 42,027 69,321
38,422
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190312005881/en/
Verastem Oncology:Erin S. CoxSenior Director, Investor Relations
& Corporate Communications+1 781-469-1553ecox@verastem.com
Investors:Joseph RayneArgot Partners+1
617-340-6075joseph@argotpartners.com
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