Verastem, Inc. (Nasdaq:VSTM) (Verastem Oncology or the Company),
a biopharmaceutical company focused on developing and
commercializing medicines seeking to improve the survival and
quality of life of cancer patients, today announced duvelisib
(COPIKTRATM) has received orphan drug designation from the U.S.
Food and Drug Administration for use in the treatment of T-Cell
lymphoma. The designation was created to encourage the development
of drugs that may provide significant benefit to patients suffering
from rare diseases.
“Receiving orphan drug designation for T-Cell Lymphoma, in
addition to the previously-granted Fast Track status, for
Peripheral T-Cell lymphoma, marks another important regulatory
milestone to bring COPIKTRA to patients who are faced with this
aggressive type of disease with limited therapeutic options,” said
Brian Stuglik, Chief Executive Officer of Verastem Oncology. “We
look forward to sharing the results of our Phase 2 PRIMO study and
efficiently advancing our development program in this
indication.”
COPIKTRA is approved in the United States for the treatment of
chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL)
after at least 2 prior therapies and accelerated approval in
follicular lymphoma (FL) after at least 2 prior systemic therapies.
COPIKTRA is not currently approved for the treatment of T-cell
lymphoma. The Company’s ongoing Phase 2 PRIMO study will provide
guidance on a duvelisib monotherapy dosing regimen in patients with
relapsed or refractory peripheral T-cell lymphoma (PTCL) and
further characterize its efficacy and tolerability in this
population.
In the U.S., under the Orphan Drug Act, the FDA’s Office of
Orphan Products Development (OOPD) grants orphan drug status to a
drug or biologic intended for the safe and effective treatment,
diagnosis or prevention of rare diseases/disorders, which is
generally a disease that affects fewer than 200,000 individuals in
the U.S. and that are expected to provide a significant therapeutic
advantage over existing treatments. Orphan designation qualifies a
company for benefits that apply across all stages of drug
development, including an accelerated approval process, seven years
of market exclusivity following marketing approval, tax credits on
U.S. clinical trials, eligibility for orphan drug grants, and a
waiver of certain administrative fees.
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
SELECT IMPORTANT SAFETY INFORMATION
This does not include all information needed to use COPIKTRA™
(duvelisib) safety and effectively. See full Prescribing
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% (4% fatal) 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%
(<1% fatal) of COPIKTRA-treated patients. Monitor for the
development of severe diarrhea or colitis. Withhold COPIKTRA.
- Fatal and/or serious cutaneous reactions occurred in 5% (<1%
fatal) of COPIKTRA-treated patients. Withhold COPIKTRA.
- Fatal and/or serious pneumonitis occurred in 5% (<1% fatal)
of COPIKTRA-treated patients. Monitor for pulmonary symptoms and
interstitial infiltrates. Withhold COPIKTRA.
INDICATIONS AND USAGE
COPIKTRA is a kinase inhibitor indicated for the treatment of
adult patients with:
- Relapsed or refractory chronic lymphocytic leukemia (CLL) or
small lymphocytic lymphoma (SLL) after at least two prior
therapies.
- Relapsed or refractory follicular lymphoma (FL) after at least
two prior systemic therapies. Accelerated approval based on overall
response rate and continued approval may be contingent upon
confirmatory trials
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.
ADVERSE REACTIONS
The most common adverse reactions (≥20%) are diarrhea or
colitis, neutropenia, rash, fatigue, pyrexia, cough, nausea, upper
respiratory infection, pneumonia, musculoskeletal pain, and
anemia.
To report Adverse Reactions, contact FDA at 1-800-FDA-1088
(1-800-332-1088) or www.fda.gov/medwatch and Verastem Oncology at
1-877-7RXVSTM (1-877-779-8786).
DRUG INTERACTIONS
- 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.
USE IN SPECIFIC POPULATIONS
Lactation: Advise women not to breastfeed.
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 within
the meaning of the Private Securities Litigation Reform Act of 1995
that are subject to risks, uncertainties and other factors,
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; that
enrollment of clinical trials may take longer than expected;
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;; and 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. These risks, uncertainties and other
factors could cause actual results to differ materially from those
referred to in the forward-looking statements. The reader is
cautioned not to rely on these forward-looking statements.
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 June 30, 2019, as filed with
the Securities and Exchange Commission (SEC) on August 1, 2019, 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20191003005764/en/
Investors: John Doyle Vice President, Investor Relations &
Finance +1 781-469-1546 jdoyle@verastem.com
Media: Lisa Buffington Corporate Communications +1 781-292-4205
lbuffington@verastem.com
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