Ligand Initiates Pivotal Trial of Captisol-Enabled®, Propylene Glycol-Free Melphalan in Patients with Multiple Myeloma
SAN DIEGO--
Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) announced
today that it has initiated a 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. This multi-center trial will evaluate safety and efficacy in 60
patients, and is intended to confirm the results from an earlier Phase 2
study demonstrating that the PG-free melphalan intravenous formulation
was safe and well-tolerated, and met the requirements for establishment
of bioequivalence to the current commercial intravenous formulation of
melphalan (sold by GlaxoSmithKline as Alkeran® for Injection).
Given the robust dataset compiled to date along with the pivotal study
design, Ligand believes that should this pivotal trial produce positive
results, the Company will be in a position to submit a 505(b)(2) New
Drug Application to the U.S. Food and Drug Administration.
“The initiation of the pivotal trial for our Captisol-enabled, PG-free
melphalan is an important development for this program, and for Ligand,”
commented Matthew W. Foehr, Chief Operating Officer of Ligand
Pharmaceuticals. “This proprietary product has Orphan Drug designation
and a compelling therapeutic profile. While this pivotal trial is
underway, we will be evaluating options to license this valuable asset
to a strategic partner or commercialize it on our own.”
About Ligand's Captisol-enabled, PG-Free Melphalan Program
Ligand's Captisol-enabled, PG-free melphalan program, which has been
granted Orphan designation by the FDA, is a new intravenous formulation
of melphalan for the multiple myeloma transplant setting.
Ligand's 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 quantities of therapeutic compounds. The use of the
Captisol® technology to reformulate melphalan is anticipated
to allow for longer administration durations and slower infusion rates,
potentially enabling clinicians to safely achieve a higher dose
intensity of pre-transplant chemotherapy. The positive Phase 2 results
for this program were announced in 2011. Ligand worked in partnership
with The University of Kansas Cancer Center on the Phase 2 trial, which
was partially funded by grants from the Kansas Bioscience Authority.
About Multiple Myeloma
Multiple myeloma is a cancer of plasma cells, a type of white blood cell
present in the bone marrow. In multiple myeloma, a group of plasma cells
(myeloma cells) becomes cancerous and multiplies, raising the number of
plasma cells to a higher than normal level. 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®
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 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 currently more than 30 Captisol-enabled products in
development, including Lundbeck’s carbamazepine IV, The Medicines
Company's MDCO-157 and Rib-X's delafloxacin IV program.
About Ligand Pharmaceuticals
Ligand is a biopharmaceutical company that develops and acquires assets
it believes will generate royalty revenues and, under its lean corporate
cost structure, produce sustainable profitability. Ligand has a diverse
asset portfolio addressing the unmet medical needs of patients for a
broad spectrum of diseases including thrombocytopenia, multiple myeloma,
diabetes, hepatitis, muscle wasting, dyslipidemia, anemia and
osteoporosis. Ligand’s Captisol platform technology is a
patent-protected, chemically modified cyclodextrin with a structure
designed to optimize the solubility and stability of drugs. Ligand has
established multiple alliances with the world's leading pharmaceutical
companies including GlaxoSmithKline, Merck, Pfizer, Eli Lilly & Company,
Baxter International, Bristol-Myers Squibb, Celgene, Onyx
Pharmaceuticals, Lundbeck Inc. and The Medicines Company, among others.
Please visit www.captisol.com
for more information on Captisol or www.ligand.com
for more information on Ligand.
Follow Ligand on Twitter @Ligand_LGND.
Forward-Looking Statements
This news release contains certain forward-looking statements by Ligand
that involve risks and uncertainties and reflect Ligand's judgment as of
the date of this release. These statements include those related to
clinical trials of Captisol-enabled melphalan's profile, market size and
possibility of commercial success, efficacy, potency, competitiveness
and the strength of Ligand's product portfolio. Actual events or results
may differ from our expectations. For example, there can be no assurance
that Captisol-enabled melphalan or other potential Captisol-enabled
drugs will progress through clinical development or receive required
regulatory approvals within the expected timelines or at all, that
further clinical trials will confirm any safety or other characteristics
or profile described in this press release, that there will be a market
of any size for Captisol-enabled melphalan or that Captisol-enabled
melphalan will be beneficial to patients or successfully marketed. The
failure to meet expectations with respect to any of the foregoing
matters may have a negative effect on Ligand's stock price. Additional
information concerning these and other risk factors affecting Ligand's
business can be found in prior press releases available via www.ligand.com
as well as in Ligand's public periodic filings with the Securities and
Exchange Commission at www.sec.gov.
Ligand disclaims any intent or obligation to update these
forward-looking statements beyond the date of this release. This caution
is made under the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995.
1 American Cancer Society, Cancer Facts & Figures 2012
2 Leerink Swann Market Research
Ligand Pharmaceuticals Incorporated
John L. Higgins, President and
CEO
Jennifer Capuzelo, Investor Relations
(858) 550-7584
jcapuzelo@ligand.com
or
LHA
Don
Markley
(310) 691-7100
dmarkley@lhai.com
Source: Ligand Pharmaceuticals Incorporated
Released December 10, 2012