Spectrum Pharmaceuticals Gains Rights to Pivotal-Stage Captisol-Enabled®
*Product candidate is being investigated as a conditioning treatment prior
to autologous stem cell transplant for patients with multiple myeloma.
*Melphalan has been granted Orphan designation by the FDA for this
*In a previous Phase 2 study, Captisol-enabled melphalan had acceptable
safety findings, and it met the requirements for establishment of
bioequivalence to the current commercial intravenous formulation of
*Spectrum anticipates NDA filing in the first half of 2014 with potential
commercial launch the following year, subject to FDA approval.
HENDERSON, Nev. -- March 14, 2013
Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biotechnology company with fully
integrated commercial and drug development operations with a primary focus in
hematology and oncology, today announced the Company has gained global
development and commercialization rights to Ligand Pharmaceuticals’ (NASDAQ:
LGND) Captisol-enabled^®, propylene glycol-free (PG-free) melphalan.
Captisol-enabled melphalan is currently in a pivotal trial for use as a
conditioning treatment prior to autologous stem cell transplant for patients
with multiple myeloma.
Spectrum is assuming the responsibility for the ongoing pivotal clinical trial
and will be responsible for filing an NDA, which is anticipated in the first
half of 2014. Under the license agreement, Ligand will receive a license fee
and is eligible to receive milestone payments, as well as royalties following
“We are pleased to add this late-stage program to our portfolio, which
includes belinostat, for which we anticipate an NDA filing mid-year, and
apaziquone, for which we expect to file an NDA in 2014,” stated Rajesh C.
Shrotriya, M.D., Chairman, President and Chief Executive Officer of Spectrum
Pharmaceuticals, Inc. “Captisol-enabled melphalan is designed to meet the need
for a formulation of melphalan that is free of propylene glycol, which has
been associated with renal and cardiac side effects. The Captisol technology
may allow longer duration of administration and slower infusion rates,
potentially enabling a higher dose intensity of pre-transplant chemotherapy to
optimize efficacy. We look forward to rapid progress of the program.”
“We are pleased to have forged this agreement,” commented John Higgins,
President and Chief Executive Officer of Ligand Pharmaceuticals. “Spectrum has
an established oncology and hematology business, and this melphalan product is
an ideal complement to their two commercial hematology products, ZEVALIN^® and
FOLOTYN^®, including an expected high degree of commercial call overlap.
Spectrum’s highly experienced, oncology-focused R&D team is committed to the
efficient development of Captisol-enabled melphalan, and has established
relationships with key investigators.”
About Captisol-Enabled Melphalan
Captisol-enabled, PG-free melphalan is a novel intravenous formulation of
melphalan being investigated for the multiple myeloma transplant setting,
which has been granted Orphan designation by the FDA. This formulation avoids
the use of propylene glycol, which has been reported to cause renal and
cardiac side effects that limit the ability to deliver higher doses of
therapeutic compounds. The use of the Captisol^® technology to reformulate
melphalan is anticipated to allow for longer administration durations and
slower infusion rates, potentially enabling clinicians to safely achieve a
higher dose intensity of pre-transplant chemotherapy.
In December 2012 Ligand announced the initiation of a pivotal trial of
Captisol-enabled melphalan. This multi-center trial is evaluating safety and
efficacy in 60 patients, and is intended to confirm the results from an
earlier Phase 2 study demonstrating that the Captisol-enabled melphalan
formulation showed acceptable safety findings, and met the requirements for
establishment of bioequivalence to the current commercial intravenous
formulation of melphalan (sold by GlaxoSmithKline as Alkeran^® for Injection).
About Multiple Myeloma and Melphalan
Multiple myeloma is a cancer of plasma cells, a type of white blood cell
present in the bone marrow. In multiple myeloma a group of plasma cells
(myeloma cells) becomes cancerous and multiplies, raising the number of plasma
cells to a higher-than-normal level. There are an estimated 20,000 new cases
of multiple myeloma in the United States each year, with an incidence of new
cases increasing by approximately 1.7% per year.
The current intravenous melphalan market is approximately $130 million
annually, with predominant use in stem cell transplants. The rate of
autologous stem cell transplants for patients with multiple myeloma is growing
by approximately 3.3% annually.
Captisol is a patent-protected, chemically modified cyclodextrin with a
structure designed to optimize the solubility and stability of drugs. Captisol
was invented and initially developed by scientists in the laboratories of Dr.
Valentino Stella at the University of Kansas’ Higuchi Biosciences Center for
specific use in drug development and formulation. This unique technology has
enabled six FDA-approved products, including Onyx Pharmaceuticals’ Kyprolis^®,
Baxter International’s Nexterone^® and Pfizer’s Vfend^® IV. There are also
more than 30 Captisol-enabled products in clinical development.
FOLOTYN, (pralatrexate injection), a folate analogue metabolic inhibitor, was
discovered by Memorial Sloan-Kettering Cancer Center, SRI International and
Southern Research Institute and developed by Allos Therapeutics. In September
2009, the U.S. Food and Drug Administration (FDA) granted accelerated approval
for FOLOTYN for use as a single agent for the treatment of patients with
relapsed or refractory PTCL. This indication is based on overall response
rate. Clinical benefit such as improvement in progression-free survival or
overall survival has not been demonstrated. FOLOTYN has been available to
patients in the U.S. since October 2009. An updated analysis of data from
PROPEL, the pivotal study of FOLOTYN in patients with relapsed or refractory
PTCL, was published in the March 20, 2011 issue of the Journal of Clinical
Oncology. FOLOTYN has patent protection through July 2022, based on a
five-year patent term extension through the Hatch-Waxman Act. Please see full
Prescribing Information for FOLOTYN at www.FOLOTYN.com.
Important FOLOTYN^® Safety Information
Warnings and Precautions
FOLOTYN may suppress bone marrow function, manifested by thrombocytopenia,
neutropenia, and anemia. Monitor blood counts and omit or modify dose for
Mucositis may occur. If greater-than or equal to Grade 2 mucositis is
observed, omit or modify dose. Patients should be instructed to take folic
acid and receive vitamin B12 to potentially reduce treatment-related
hematological toxicity and mucositis.
Fatal dermatologic reactions may occur. Dermatologic reactions may be
progressive and increase in severity with further treatment. Patients with
dermatologic reactions should be monitored closely, and if severe, FOLOTYN
should be withheld or discontinued. Tumor lysis syndrome may occur. Monitor
patients and treat if needed.
FOLOTYN can cause fetal harm. Women should avoid becoming pregnant while being
treated with FOLOTYN and pregnant women should be informed of the potential
harm to the fetus.
Use caution and monitor patients when administering FOLOTYN to patients with
moderate to severe renal function impairment.
Elevated liver function test abnormalities may occur and require monitoring.
If liver function test abnormalities are greater-than or equal to Grade 3,
omit or modify dose.
The most common adverse reactions were mucositis (70%), thrombocytopenia
(41%), nausea (40%), and fatigue (36%). The most common serious adverse events
are pyrexia, mucositis, sepsis, febrile neutropenia, dehydration, dyspnea, and
Use in Specific Patient Population
Nursing mothers should be advised to discontinue nursing or the drug, taking
into consideration the importance of the drug to the mother.
Co-administration of drugs subject to renal clearance (e.g., probenecid,
NSAIDs, and trimethoprim/sulfamethoxazole) may result in delayed renal
Please see FOLOTYN^® Full Prescribing Information at www.FOLOTYN.com.
About ZEVALIN^® and the ZEVALIN Therapeutic Regimen
ZEVALIN (ibritumomab tiuxetan) injection for intravenous use, is indicated for
the treatment of patients with relapsed or refractory, low-grade or follicular
B-cell non-Hodgkin's lymphoma (NHL). ZEVALIN is also indicated for the
treatment of patients with previously untreated follicular non-Hodgkin's
Lymphoma who achieve a partial or complete response to first-line
ZEVALIN is a CD20-directed radiotherapeutic antibody. The ZEVALIN therapeutic
regimen consists of two components: rituximab, and Yttrium-90 (Y-90)
radiolabeled ZEVALIN for therapy. ZEVALIN builds on the combined effect of a
targeted biologic monoclonal antibody augmented with the therapeutic effects
of a beta-emitting radioisotope.
Important ZEVALIN^® Safety Information
Deaths have occurred within 24 hours of rituximab infusion, an essential
component of the ZEVALIN therapeutic regimen. These fatalities were associated
with hypoxia, pulmonary infiltrates, acute respiratory distress syndrome,
myocardial infarction, ventricular fibrillation, or cardiogenic shock. Most
(80%) fatalities occurred with the first rituximab infusion. ZEVALIN
administration can result in severe and prolonged cytopenias in most patients.
Severe cutaneous and mucocutaneous reactions, some fatal, can occur with the
ZEVALIN therapeutic regimen.
Please see full Prescribing Information, including BOXED WARNINGS, for ZEVALIN
and rituximab. Full prescribing information for ZEVALIN can be found at
About Spectrum Pharmaceuticals, Inc.
Spectrum Pharmaceuticals is a leading biotechnology company focused on
acquiring, developing, and commercializing drug products, with a primary focus
in oncology and hematology. Spectrum and its affiliates market three oncology
drugs ─ FUSILEV^® (levoleucovorin) for Injection in the U.S.; FOLOTYN^®
(pralatrexate injection), also marketed in the U.S.; and ZEVALIN^®
(ibritumomab tiuxetan) Injection for intravenous use, for which the Company
has worldwide marketing rights. Spectrum's strong track record in in-licensing
and acquiring differentiated drugs, and expertise in clinical development have
generated a robust, diversified, and growing pipeline of product candidates in
advanced-stage Phase 2 and Phase 3 studies. More information on Spectrum is
available at www.sppirx.com.
Forward-looking statement — This press release may contain forward-looking
statements regarding future events and the future performance of Spectrum
Pharmaceuticals that involve risks and uncertainties that could cause actual
results to differ materially. These statements are based on management's
current beliefs and expectations. These statements include, but are not
limited to, statements that relate to our business and its future, including
certain company milestones, Spectrum's ability to identify, acquire, develop
and commercialize a broad and diverse pipeline of late-stage clinical and
commercial products, leveraging the expertise of partners and employees around
the world to assist us in the execution of our strategy, and any statements
that relate to the intent, belief, plans or expectations of Spectrum or its
management, or that are not a statement of historical fact. Risks that could
cause actual results to differ include the possibility that our existing and
new drug candidates may not prove safe or effective, the possibility that our
existing and new applications to the FDA and other regulatory agencies may not
receive approval in a timely manner or at all, the possibility that our
existing and new drug candidates, if approved, may not be more effective,
safer or more cost efficient than competing drugs, the possibility that our
efforts to acquire or in-license and develop additional drug candidates may
fail, our lack of sustained revenue history, our limited marketing experience,
our dependence on third parties for clinical trials, manufacturing,
distribution and quality control and other risks that are described in further
detail in the Company's reports filed with the Securities and Exchange
Commission. We do not plan to update any such forward-looking statements and
expressly disclaim any duty to update the information contained in this press
release except as required by law.
SPECTRUM PHARMACEUTICALS, INC.^®, FUSILEV^®, FOLOTYN^®, and ZEVALIN^® are
registered trademarks of Spectrum Pharmaceuticals, Inc and its affiliates.
REDEFINING CANCER CARE^™ and the Spectrum Pharmaceuticals logos are trademarks
owned by Spectrum Pharmaceuticals, Inc.
© 2013 Spectrum Pharmaceuticals, Inc. All Rights Reserved.
Spectrum Pharmaceuticals, Inc.
Shiv Kapoor, 702-835-6300
Vice President, Strategic Planning & Investor Relations
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