The Icahn School of Medicine at Mount Sinai has entered into two exclusive licenses to develop the drug pentosan polysulfate sodium for the treatment of mucopolysaccharidosis (MPS), with Australia-based company Paradigm Biopharmaceuticals Ltd. (ASX: PAR) and ReqMed Company, Ltd. of Japan.
MPS is a group of 11 lysosomal storage diseases, metabolic disorders caused by genetic defects that affect the skeletal system, skin, heart, brain, and other organ systems and can lead to disability or death. These conditions occur in about 3.5 out of 100,000 births. Calogera Simonaro, PhD, Research Professor in the Department of Genetics and Genomic Sciences at the Icahn School of Medicine, demonstrated the potential use of the drug through several proof-of-concept studies carried out in animal models of MPS. Pentosan polysulfate sodium, normally used as an anti-inflammatory for bladder pain, was repurposed in Dr. Simonaro’s lab to improve connective tissue affected by MPS. Current therapies that exist for these diseases, such as enzyme replacement therapy and bone marrow transplantation, are costly, require highly invasive procedures, and can have limited efficacy. These therapies can slow, but often do not halt, progression of the disease.
“By using a drug that is already approved, we should be able to fast-track [pentosan polysulfate sodium] to patients and address an important unmet medical need,” said Edward Schuchman, PhD, the Genetic Disease Foundation – Francis Crick Professor of Genetics and Genomic Sciences at the Icahn School of Medicine and the Department’s Vice Chair for Research. “Using one drug for several different lysosomal storage diseases changes the approach currently used for treating these diseases.”
Paradigm will develop the drug in the United States, the European Union, and other territories, and ReqMed will develop the drug in Japan and other select Asian territories. The financial backing and infrastructure provided by these partnerships will facilitate blinded studies for regulatory approval. Drs. Schuchman and Simonaro believe pentosan polysulfate sodium can also be used to treat other lysosomal storage diseases, including Fabry and Gaucher diseases.
“Mount Sinai innovators recognized the value of repurposing an existing therapeutic for a new indication,” said Erik Lium, PhD, Executive Vice President of Mount Sinai Innovation Partners. “We look forward to working with Paradigm and ReqMed to advance pentosan polysulfate sodium for the treatment of MPS.”
About ReqMed Company, Ltd.
ReqMed Company, Ltd. is a privately held, Japanese company established in 1998 to serve as a bridge between biotechnology ventures and pharmaceutical firms in Japan. In 2008 Reqmed entered an exclusive license agreement with Asahi Kasei Pharma Corporation, in partnership with Bene pharmaChem, to develop and sell PPS in Japan for patients with osteoarthritis. Reqmed also has obtained small business grant funding from the Japanese government to conduct PPS proof-of-concept studies in patients with MPS. A phase 1/2 clinical trial in adult patients with MPS II has been completed, and studies are currently underway in adult and pediatric patients with MPS I and VI, and in patients with a related disorder known as a mucolipidoses type II. For more information please visit www.reqmed.co.jp.
About Mount Sinai Innovation Partners (MSIP)
Mount Sinai Innovation Partners (MSIP) is responsible for driving the real-world application and commercialization of Mount Sinai discoveries and inventions, and the development of research partnerships with industry. Our aim is to translate discoveries and inventions into health care products and services that benefit patients and society. MSIP is accountable for the full spectrum of commercialization activities required to bring Mount Sinai inventions to life. These activities include evaluating, patenting, marketing and licensing new technologies building research, collaborations and partnerships with commercial and nonprofit entities, material transfer and confidentiality, coaching innovators to advance commercially-relevant translational discoveries, and actively fostering an ecosystem of entrepreneurship within the Mount Sinai research and health system communities. For more information please visit www.ip.mountsinai.org.