Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) announced
today that its partner Retrophin, Inc. (NASDAQ:RTRX) has received orphan
drug designation from the U.S. Food and Drug Administration (FDA) for
Sparsentan for the treatment of focal segmental glomerulosclerosis
(FSGS).
“Sparsentan has the potential to be a significant medicine, and could
transform the treatment of FSGS,” said John Higgins, President and Chief
Executive Officer of Ligand Pharmaceuticals. “The team at Retrophin
continues to make great progress advancing the program, and obtaining
orphan designation provides an additional layer of potential market
exclusivity. This is a valuable late-stage asset in our broad portfolio
of over 100 shots-on-goal.”
Higgins added, "Late last year, Ligand highlighted a selection of major
pipeline programs that we called the Big Six for investors to follow in
2015. In recent weeks, three of the six programs have had positive
developments with the NDA submission for Captisol-enabled®
Melphalan, positive Phase 3 data for Captisol-enabled Delafloxacin
IV, and now orphan designation for an important drug targeting a rare
disease with major unmet medical need. We are pleased with the momentum
we have for these key programs early in the year."
Sparsentan is an investigational therapeutic agent which acts as both a
selective endothelin receptor antagonist and an angiotensin receptor
blocker. Retrophin is conducting the Phase 2 DUET trial of Sparsentan
for the treatment of FSGS, a leading cause of end-stage renal disease.
There are currently no therapies approved for the treatment of FSGS in
the United States.
Ligand licensed worldwide rights of Sparsentan (RE-021) (formerly known
as DARA) to Retrophin in 2012, at the time of Retrophin’s formation.
About Orphan Drug Designation
The Orphan Drug Designation program is intended to encourage companies
to develop therapeutics for diseases that affect fewer than 200,000
individuals in the U.S. Orphan designation will provide Sparsentan with
seven years of marketing exclusivity for FSGS if it is approved by the
FDA for this indication. Prior to FDA approval, orphan designation
provides incentives for sponsors including tax credits for clinical
research expenses, the opportunity to obtain government grant funding to
support clinical research, and an exemption from FDA user fees.
About Retrophin, Inc.
Retrophin is a pharmaceutical company focused on the development,
acquisition and commercialization of drugs for the treatment of serious,
catastrophic or rare diseases for which there are currently no viable
options for patients. The Company's approved products include Chenodal®,
Thiola® and Vecamyl®, and its pipeline includes compounds for several
catastrophic diseases, including focal segmental glomerulosclerosis
(FSGS), pantothenate kinase-associated neurodegeneration (PKAN),
schizophrenia, infantile spasms, nephrotic syndrome and others. For
additional information, please visit www.retrophin.com.
About Ligand Pharmaceuticals
Ligand is a biopharmaceutical company with a business model that is
based upon the concept of developing or acquiring royalty revenue
generating assets and coupling them to a lean corporate cost structure.
Ligand’s goal is to produce a bottom line that supports a sustainably
profitable business. By diversifying the portfolio of assets across
numerous technology types, therapeutic areas, drug targets and industry
partners, we offer investors an opportunity to invest in the
increasingly complicated and unpredictable pharmaceutical industry. In
comparison to its peers, we believe Ligand has assembled one of the
largest and most diversified asset portfolios in the industry with the
potential to generate revenue in the future. These therapies seek to
address the unmet medical needs of patients for a broad spectrum of
diseases including diabetes, hepatitis, muscle wasting, Alzheimer's
disease, dyslipidemia, anemia, asthma 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, Onyx
Pharmaceuticals (a subsidiary of Amgen Inc.), Merck, Pfizer, Baxter
International, Lundbeck Inc., Eli Lilly & Co. and Spectrum
Pharmaceuticals. Please visit www.captisol.com
for more information on Captisol and www.ligand.com
for more information on Ligand.
Forward-Looking Statements
This news release contains forward looking statements by Ligand and
Retrophin that involve risks and uncertainties and reflect Ligand’s and
Retrophin’s judgment as of the date of this release. Actual events or
results may differ from Ligand’s or Retrophin’s expectations. For
example, there can be no assurance that Sparsentan or any product in the
Ligand or Retrophin pipelines will be successfully developed, that any
of the milestone triggers will be achieved, that regulatory approvals
will be granted, that patient and physician acceptance of these products
will be achieved, that final results of human clinical trials will be
consistent with any interim results, that final results will be
supportive of regulatory approvals required to market products or that
any revenue will be achieved from this partnered program. In addition,
if Retrophin chooses to sub-license the program the actual financial
amounts Ligand receives may be lower than licensing terms specified in
this press release. 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 and Retrophin disclaim 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.
Copyright Business Wire 2015