Novel Compounds Have Therapeutic Potential
in Broad Range of Diseases, Including Certain Rare Genetic
Disorders
La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the Company or
La Jolla), a leader in the development of innovative therapies
intended to significantly improve outcomes in patients suffering
from life-threatening diseases, and Vanderbilt University
(Vanderbilt) today announced an exclusive, worldwide research and
license agreement covering Vanderbilt’s research program and
intellectual property rights relating to small-molecule kinase
inhibitors designed to selectively block specific members of the
bone morphogenetic protein (BMP) type-I receptor family.
The seven members of the BMP type-I receptor family, activin
receptor-like kinase (ALK) 1-7, play critical roles in human
development and physiology. In turn, the improper activation of
these receptor pathways is responsible for a wide range of disease
conditions. For example, fibrodysplasia ossificans progressiva
(FOP), a rare genetic disorder where the body turns muscle into
bone, is caused by a genetic mutation in ALK2 that results in
excessive signaling of this pathway.
Members of the BMP type-I receptor family also are involved in
other diseases such as acquired heterotopic ossification (formation
of bone in soft tissue caused by injury or trauma), muscular
dystrophies including Duchenne muscular dystrophy, anemia of
chronic disease (decrease of red blood cells or hemoglobin from
chronic infection, chronic immune activation and malignancy),
cancer, cardiovascular diseases and inflammatory bowel disease.
After the first publication of his discovery in 2008, Charles C.
Hong, M.D., Ph.D., in conjunction with Vanderbilt, has led a
dedicated research team focused on discovering highly selective,
small-molecule kinase inhibitors designed to selectively block
specific members of the BMP type-I receptor family. Dr. Hong's team
is complemented by the robust medicinal chemistry expertise of
Vanderbilt's Craig W. Lindsley, Ph.D., and Corey R. Hopkins, Ph.D.
Under the research and license agreement with La Jolla, La Jolla
will fund further research under this program at Vanderbilt in
return for rights to acquire compounds emerging from this
program.
Dr. Hong is an Associate Professor of Cardiovascular Medicine,
Pharmacology, and Cell and Developmental Biology at Vanderbilt
University School of Medicine and a member of the Veterans Affairs
Tennessee Valley Healthcare System. He is also a member of the
Vanderbilt Institute of Chemical Biology and the Vanderbilt Center
for Stem Cell Biology. Dr. Hong discovered the first pharmacologic
inhibitor of the BMP pathway in 2008, and he was instrumental in
the discovery of the critical role of the BMP pathway in stem cell
maturation, cholesterol homeostasis and cancer.
Dr. Lindsley is a Professor of Pharmacology and Chemistry at
Vanderbilt University School of Medicine. He is also the Director
of Medicinal Chemistry at the Vanderbilt Center for Neuroscience
Drug Discovery and the Director of the Vanderbilt Specialized
Chemistry Center for Accelerated Probe Development.
Dr. Hopkins is an Assistant Professor of Pharmacology and
Chemistry at Vanderbilt University School of Medicine. He is also
the Associate Director of Chemistry at the Vanderbilt Center for
Neuroscience Drug Discovery and a co-Director of the Vanderbilt
Specialized Chemistry Center for Accelerated Probe Development.
“We are excited to see our work move forward from a basic
research program to a therapeutic program in partnership with La
Jolla. Together, we see this as an opportunity to position our BMP
inhibitors as potential treatments for rare and neglected
diseases,” said Drs. Hopkins and Lindsley on behalf of the
Vanderbilt team.
“Tremendous credit goes to Dr. Hong for his discovery of BMP
receptor inhibitors and Dr. Lindsley and Dr. Hopkins for their
advancements in medicinal chemistry. Their breakthrough discoveries
offer hope to improve the lives of patients suffering from a broad
range of debilitating diseases,” said George F. Tidmarsh, M.D.,
Ph.D., President and Chief Executive Office of La Jolla. “In
collaboration with our innovative partners at Vanderbilt University
Medical Center, we will work hard to translate their pioneering
discoveries into novel treatments for patients in need.”
About La Jolla Pharmaceutical Company
La Jolla Pharmaceutical Company is a biopharmaceutical company
focused on the discovery, development and commercialization of
innovative therapies intended to significantly improve outcomes in
patients suffering from life-threatening diseases. The Company has
several product candidates in development. LJPC-501 is La Jolla’s
proprietary formulation of angiotensin II for the potential
treatment of catecholamine-resistant hypotension. LJPC-401 is La
Jolla’s novel formulation of hepcidin for the potential treatment
of iron overload, which occurs as a result of diseases such as
hereditary hemochromatosis and beta thalassemia. LJPC-30Sa and
LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for
the potential treatment of serious bacterial infections and rare
genetic disorders, such as cystic fibrosis and Duchenne muscular
dystrophy. For more information on La Jolla, please visit
www.ljpc.com.
About Vanderbilt University Medical Center
Vanderbilt University Medical Center is home to Vanderbilt
University Hospital, the Monroe Carell Jr. Children’s Hospital at
Vanderbilt, the Vanderbilt Psychiatric Hospital and the Vanderbilt
Stallworth Rehabilitation Hospital. These hospitals experienced
more than 61,000 inpatient admissions during fiscal year 2015.
Vanderbilt’s adult and pediatric clinics treated nearly 2 million
patients during this same period.
Both the Vanderbilt University School of Medicine and School of
Nursing are recognized by U.S. News & World Report’s annual
Best Graduate Schools as among the nation’s best, with the School
of Medicine ranked 14th and the School of Nursing 11th. The School
of Medicine’s biomedical research program has earned its place
among the nation’s top 10 academic medical centers in terms of
public and private research funding, receiving more than $500
million in total funding during fiscal year 2015.
Vanderbilt University Hospital and the Monroe Carell Jr.
Children’s Hospital at Vanderbilt are recognized again this year by
U.S. News & World Report’s Best Hospitals as among the nation’s
best, with 18 nationally ranked specialties.
Forward-Looking Statement Safe Harbor
This document contains forward-looking statements as that term
is defined in the Private Securities Litigation Reform Act of 1995.
These statements relate to future events or the Company’s future
results of operations. These statements are only predictions and
involve known and unknown risks, uncertainties and other factors,
which may cause actual results to be materially different from
these forward-looking statements. The Company cautions readers not
to place undue reliance on any such forward-looking statements,
which speak only as of the date they were made. Certain of these
risks, uncertainties, and other factors are described in greater
detail in the Company’s filings with the U.S. Securities and
Exchange Commission (SEC), all of which are available free of
charge on the SEC’s web site www.sec.gov. These risks include, but are not
limited to, risks relating to: the timing for the filing of an
Investigational New Drug Application, commencement of clinical
studies and the anticipated timing for completion of such studies;
the success of future development activities for the Company’s drug
candidates; and potential indications for which the Company’s drug
candidates may be developed. Subsequent written and oral
forward-looking statements attributable to the Company or to
persons acting on its behalf are expressly qualified in their
entirety by the cautionary statements set forth in the Company’s
reports filed with the SEC. The Company expressly disclaims any
intent to update any forward-looking statements.
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version on businesswire.com: http://www.businesswire.com/news/home/20150818005463/en/
La Jolla Pharmaceutical CompanyGeorge F. Tidmarsh, M.D.,
Ph.D.President & Chief Executive OfficerPhone: (858)
207-4264Email: gtidmarsh@ljpc.comorDennis M. MulroyChief Financial
OfficerPhone: (858) 433-6839Email: dmulroy@ljpc.com
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