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.--(BUSINESS WIRE)--
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 research infrastructure."
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
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
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 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 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
Source: Spectrum Pharmaceuticals, Inc.
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