- Results support the safety and efficacy
of high-dose Captisol-enabled (CE)-Melphalan as a high-dose
conditioning treatment prior to autologous hematopoietic stem cell
transplantation (AHCT) in patients with multiple myeloma (MM).
- Overall Response Rate improved from 79%
at study entry to 95% after CE-Melphalan and AHCT; Complete
Response Rate increased from 10 to 31%.
- Spectrum’s CE-Melphalan formulation is
free of propylene glycol and does not use a custom, propylene
glycol-containing solvent for its reconstitution.
- CE-Melphalan is more stable with a
longer use time, simplifying clinical administration
logistics.
- CE-Melphalan was shown to be
bioequivalent to the currently approved commercial intravenous
formulation of melphalan.
- FDA decision is expected in late 2015.
If approved, the Company plans to launch CE-Melphalan with its
existing hematology/oncology sales force.
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 results of a clinical study of Propylene
Glycol (PG)-Free, Captisol-enabled Melphalan (CE Melphalan)
Conditioning for Autologous Hematopoietic Stem Cell Transplantation
(AHCT) in Patients with Multiple Myeloma (MM). Spectrum filed a New
Drug Application (NDA) for approval of CE-Melphalan in December
2014 and expects a decision from the Food and Drug Administration
(FDA) by late 2015.
“CE-Melphalan is a key late-stage drug for the company, and we
are excited to share these clinical data,” said Rajesh C.
Shrotriya, MD, Chairman and Chief Executive Officer of Spectrum
Pharmaceuticals. “CE-Melphalan is a new injectable formulation of
melphalan that incorporates the Captisol brand of β-cyclodextrin
improving the solubility and stability of melphalan. It also
facilitates the use of an aqueous diluent (normal saline) instead
of propylene glycol, which is associated with toxicities including
renal dysfunction and arrhythmias. CE-Melphalan will fit seamlessly
into our existing commercial infrastructure if it is approved by
the FDA. Spectrum is committed to deliver improved treatment
options to patients suffering with cancer.”
“Intravenous melphalan has become the standard of care
conditioning agent used for high-dose treatment of MM patients
undergoing AHCT, and the substitution of Captisol in CE-Melphalan
addresses some of the limitations of the currently approved
formulations,” said Dr. Parameswaran Hari, Armand J. Quick/William
F. Stapp Professor of Hematology at the Medical College of
Wisconsin, Director of the Adult Blood and Marrow Transplant
Program at Froedtert Hospital and the Section Head of Hematologic
Malignancies and Transplantation, in the Division of Hematology and
Oncology in the Department of Medicine. “The improved stability of
the CE-Melphalan HCl formulation may potentially ensure that cancer
patients receive the full, intended therapeutic dose of IV
melphalan by increasing the use time and infusion time of IV
Melphalan, and simplifying clinical administration logistics.”
Following is the summary of the presentation:
Abstract #155 Results of a Phase II Study
of Propylene Glycol (PG)-Free, Captisol-Enabled Melphalan
Conditioning for Autologous Hematopoietic Stem Cell Transplantation
(AHCT) in Patients with Multiple Myeloma (MM)
Sixty-one patients with MM received 200 mg/m2
of CE-Melphalan (100 mg/m2/day x 2) followed by AHCT. The number of
lines of prior therapy ranged from 1 to 4, and 9 (15%) pts had high
risk cytogenetics. Disease status Pre-treatment included Complete
Response (CR) in 10% of subjects, Very Good Partial Response (VGPR)
in 36% and Partial Response (PR) in 33% subjects. All subjects
(100%) achieved myeloablation followed by successful bone marrow
engraftment. Median time to neutrophil engraftment was 12 days and
to platelet engraftment was 13 days post-AHCT. There were no deaths
by Day 100, and the most common Grade 3 and 4 toxicities were the
expected hematologic events (neutropenia, leukopenia, lymphopenia,
thrombocytopenia and anemia). The most frequent non-hematologic
adverse events included diarrhea, nausea, and fatigue. Importantly,
the incidence of severe mucositis was low (Grade 3/4; 10%). At Day
100 post-AHCT, the ORR increased to 95% with 74% of subjects
achieving a >= VGPR response including stringent CR in 16%, CR
in 15% and VGPR in 43%.
Conclusions: CE-Melphalan led to
successful myeloablation and subsequent engraftment in MM patients
with no mortality or unexpected transplant-related toxicity; the
incidence of Grade 3-4 mucositis was low. Overall, 95% of subjects
(n=61) responded to high dose CE-Melphalan, and in the subgroup of
high risk patients (15%), 67% VGPR or better responses were
achieved.
In December 2014, Spectrum submitted a NDA to the FDA for the
approval of CE-Melphalan (propylene glycol-free) for use as a
high-dose conditioning treatment prior to AHCT in patients with MM.
Spectrum is also seeking approval for the palliative treatment of
patients with MM for whom oral therapy is not appropriate.
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 was responsible for filing the NDA. Under the license
agreement, Ligand received a license fee and is eligible to receive
milestone payments, as well as royalties following potential
commercialization.
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 five 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; MARQIBO® (vinCRIStine sulfate LIPOSOME
injection) for intravenous infusion, for which the Company has
worldwide marketing rights and BELEODAQ® (belinostat) for Injection
in the U.S. 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.
About Captisol-Enabled Melphalan
Captisol-Enabled, Propylene Glycol -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 need to use propylene glycol containing custom diluent, which
has been reported to cause renal and cardiac side effects, which in
turn 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.
About Captisol®
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 development.
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®,
MARQIBO®, and BELEODAQ® 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.
© 2015 Spectrum Pharmaceuticals, Inc. All Rights Reserved
Spectrum PharmaceuticalsShiv KapoorVice President, Strategic
Planning & Investor
Relations702-835-6300InvestorRelations@sppirx.com
Spectrum Pharmaceuticals (NASDAQ:SPPI)
Historical Stock Chart
From Mar 2024 to Apr 2024
Spectrum Pharmaceuticals (NASDAQ:SPPI)
Historical Stock Chart
From Apr 2023 to Apr 2024