Spectrum Pharmaceuticals Announces Completion of Enrollment, in Pivotal Trial of Captisol-Enabled® (Propylene Glycol-Free) Melphalan
October 30, 2013
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