Spectrum Pharmaceuticals' Pivotal Trial of Captisol-Enabled™ (Propylene Glycol-Free) Melphalan Meets Primary Endpoint
CE Melphalan is being developed as a novel version of the
well-established conditioning treatment in autologous transplant for
patients with multiple myeloma. CE Melphalan does not contain
propylene glycol, an ingredient in currently available products which
has been associated with renal and cardiac side effects.
In its pivotal trial, CE Melphalan met its primary endpoint. The
improved stability profile of CE Melphalan could lead to rapid
adoption in the transplant conditioning market.
The company expects to file an NDA in the 3rd quarter and plans to
launch this drug with its existing hematology/oncology sales force
next year pending approval.
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 that its
pivotal trial of Captisol-enabled™ (propylene glycol-free) Melphalan met
its primary end points.
"I am pleased with the focus and alacrity in which our clinical team has
executed this trial," said Rajesh C. Shrotriya, MD, Chairman and Chief
Executive Officer of Spectrum Pharmaceuticals. "With the positive
results from this trial, we are one step closer to providing patients
and healthcare providers with a much needed new formulation of
Melphalan. Melphalan is currently a well-established conditioning
treatment in autologous transplant for patients with multiple myeloma.
Our novel Captisol-enabled Melphalan is free of propylene glycol, which
itself is associated with renal and cardiac side effects. Additionally,
the improved stability profile of CE Melphalan could be valuable in its
rapid adoption in the transplant conditioning market. This product also
fits seamlessly into our existing commercial and research
infrastructure. With key upcoming catalysts related to CE Melphalan,
Beleodaq and SPI-2012, we continue to be committed to building a
world-class Hematology/Oncology company."
The phase 2 pivotal trial evaluating CE Melphalan was a multi-center
trial evaluating safety and efficacy. The primary objective of the study
was to determine the overall safety and toxicity profile in multiple
myeloma patients receiving 200 mg/m2 of CE Melphalan as
myeloablative therapy prior to autologous stem cell transplantation
(ASCT). The secondary objectives evaluated the efficacy of CE Melphalan
in this patient population as measured by multiple myeloma response rate
(according to International Myeloma Working Group [IMWG] criteria), and
the rates of myeloablation, and engraftment. The primary endpoint of
this Phase 2 trial was met, and additional analyses are currently
underway. The company plans to file an NDA in the third quarter.
In a previous clinical study, Captisol-enabled Melphalan met the
requirements for establishment of bioequivalence to the current
commercial intravenous formulation of melphalan.
Spectrum Pharmaceuticals gained global development and commercialization
rights to CE Melphalan from Ligand Pharmaceuticals Incorporated (NASDAQ:
LGND) in March 2013. Spectrum assumed the responsibility for the pivotal
clinical trial and is responsible for filing an NDA. Under the license
agreement, Ligand received a license fee and is eligible to receive
milestone payments, as well as royalties following potential
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 an Orphan Drug Designation by the
FDA. This formulation eliminates 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 also improves
its stability and is anticipated to allow for slower infusion rates and
longer administration durations, potentially enabling clinicians to
safely achieve a higher dose intensity for 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 Merck's NOXAFIL IV. There are
also more than 30 Captisol-enabled products currently in clinical
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 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®, ZEVALIN® and
MARQIBO® are registered trademarks of Spectrum Pharmaceuticals, Inc and
its affiliates. BELEODAQ™, 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.
© 2014 Spectrum Pharmaceuticals, Inc. All Rights Reserved.
Vice President, Strategic
Planning & Investor Relations
Source: Spectrum Pharmaceuticals
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