Spectrum Pharmaceuticals Announces Completion of Enrollment, in Pivotal
Trial of Captisol-Enabled® (Propylene Glycol-Free) Melphalan
*Expected NDA filing mid-2014.
*Granted Orphan designation by the FDA as a conditioning treatment for use
in autologous transplant for patients with multiple myeloma. If approved,
will be the only drug approved for this indication.
*In a previous clinical study, Captisol-enabled melphalan met the
requirements for establishment of bioequivalence to the current commercial
intravenous formulation of melphalan.
HENDERSON, Nev. -- October 30, 2013
Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biotechnology company with fully
integrated commercial and drug development operations and a primary focus in
hematology and oncology, announced today the completion of enrollment for the
pivotal trial of Captisol-enabled®, propylene glycol-free (PG-free) high-dose
melphalan as a conditioning treatment prior to autologous transplant for
patients with multiple myeloma.
“We are very pleased to have the patient enrollment completed ahead of
schedule,” said Rajesh C. Shrotriya, MD, Chairman, Chief Executive Officer,
and President of Spectrum Pharmaceuticals. “Melphalan remains a high priority
for Spectrum; if approved, Captisol-enabled melphalan would be the only drug
approved as a conditioning agent for stem cell transplant in multiple myeloma.
This product will also fit seamlessly into our existing commercial and
The phase 2 pivotal trial is a multi-center trial evaluating safety and
efficacy in 60 patients. The primary objective of the study is to determine
the overall safety and toxicity profile in multiple myeloma patients receiving
200 mg/m2 of Melphalan HCl for Injection (Propylene Glycol-Free) as
myeloablative therapy prior to autologous stem cell transplantation (ASCT).
The secondary objective is to evaluate the efficacy of Melphalan HCl
(Propylene Glycol-Free) in this patient population as measured by multiple
myeloma response rate (according to International Myeloma Working Group [IMWG]
criteria), myeloablation, and engraftment. Several endpoints require 90-100
day follow-up of patients on the trial.
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.^1 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.^2
About Captisol-Enabled Melphalan
Captisol-enabled, PG-free melphalan is a novel intravenous formulation of
melphalan being investigated for the multiple myeloma transplant setting, for
which it 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 slower infusion rates and longer
administration durations, potentially enabling clinicians to safely achieve a
higher dose intensity of pre-transplant chemotherapy.
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 currently in clinical development.
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 four oncology
drugs ─ FUSILEV® (levoleucovorin) for Injection in the U.S.; FOLOTYN®
(pralatrexate injection), also marketed in the U.S.; ZEVALIN® (ibritumomab
tiuxetan) Injection for intravenous use, for which the Company has worldwide
marketing rights and MARQIBO® (vinCRIStine sulfate LIPOSOME injection) for
intravenous infusion, 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
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
sales of Spectrum’s drug products, 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 customer
concentration, the possibility for fluctuations in customer orders, evolving
market dynamics, our dependence on third parties for clinical trials,
manufacturing, distribution, information 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®, ZEVALIN® and MARQIBO® ^
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. Any other trademarks are the property
of their respective owners.
© 2013 Spectrum Pharmaceuticals, Inc. All Rights Reserved.
^1American Cancer Society, Cancer Facts & Figures 2012
^2 Leerink Swann Market Research
Vice President, Strategic Planning & Investor Relations
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