Spectrum Pharmaceuticals Gains Rights to Pivotal-Stage Captisol-Enabled® Melphalan
Product candidate is being investigated as a conditioning treatment
prior to autologous stem cell transplant for patients with multiple
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 melphalan.
Spectrum anticipates NDA filing in the first half of 2014 with
potential commercial launch the following year, subject to FDA
HENDERSON, Nev.--(BUSINESS WIRE)--
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 potential commercialization.
"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
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
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 hematologic toxicities.
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 thrombocytopenia.
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
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 chemotherapy.
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
Please see full Prescribing Information, including BOXED WARNINGS, for
ZEVALIN and rituximab. Full prescribing information for ZEVALIN can be
found at www.ZEVALIN.com.
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
© 2013 Spectrum Pharmaceuticals, Inc. All Rights Reserved.
Spectrum Pharmaceuticals, Inc.
Shiv Kapoor, 702-835-6300
President, Strategic Planning & Investor Relations
Source: Spectrum Pharmaceuticals, Inc.
News Provided by Acquire Media
Close window | Back to top