Biotechnology company Paradigm Biopharmaceuticals (ASX: PAR) is continuing to expand the gamut of applications for its proprietary Pentosan Polysulfate Sodium (iPPS) drug by signing an agreement with US-based not-for-profit corporation, the Icahn School of Medicine at Mount Sinai.
Development of iPPS is currently making strong progress as part of clinical trials in Australia with its pain reduction rate remaining well above initial expectations and continuing to gain plaudits amongst existing patients.
Paradigm announced today that it had executed an exclusive “In-License Agreement” with the Icahn School of Medicine at Mount Sinai, New York, for the use of iPPS in the treatment of mucopolysaccharidoses (MPS), a group of inherited lysosomal storage disorders.
Icahn is a US-based entity that is considered as a leader in medical and scientific training and education, biomedical research and patient care.
The corporation operates within the Mount Sinai Health System – New York’s largest integrated health delivery system encompassing seven hospital campuses, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region.
Tackling mucopolysaccharidoses (MPS)
The mucopolysaccharidoses (MPS) are a family of disorders caused by genetic defects in the catabolism of sulfated components of connective tissue known as glycosaminoglycans (GAGs).
According to scientific research conducted last year, MPS affects around 3.5 in 100,000 live births and presents in three different ways.
First, it can appear as what’s known as “dysmorphic syndrome” which tends to induce early onset of middle ear disease, deafness, or upper airways obstruction
Secondly, patients tend to experience learning difficulties, behavioural disturbance, dementia and mild somatic abnormalities. Lastly, MPS can also manifest severe bone dysplasia, colloquially known as “dwarfism”.
Paradigm looks to capitalise
Under the terms of the agreement, Paradigm will gain access to attractive commercial terms including a single-digit royalty with “no payment of equity or dilution to existing shareholders,” the company said.
The deal effectively means Paradigm will obtain highly valuable intellectual property (IP) including granted patents in all key regions such as the US, Japan, Europe, Australia and New Zealand.
Furthermore, Paradigm has declared that its successfully completed Phase 2a clinical trial demonstrated excellent safety and strong efficacy data, which also demonstrated that the data set includes long-term safety of iPPS in very sick and debilitated patients with chronic joint pain.
The company also said that iPPS would be included in future FDA submissions for Paradigm’s primary indications, namely osteoarthritis and treating bone marrow lesions.
Paradigm has stated that the licensing agreement carries with it accompanying Phase 2a human data which is “very complementary and synergistic” to the company’s OA/BMEL program. Therefore, the licensing agreement constitutes a logical and valuable addition to Paradigm’s product pipeline and IP portfolio.
If successful, Paradigm’s iPPS could potentially be developed into a billion-dollar “blockbuster” product, although the pharma company must still obtain successful registration in several territories around the world.
Paradigm has confirmed this newly in-licensed indication is fully funded for the next 12 months “and beyond”.
The next milestone for Paradigm is the conclusion of its Phase 2b OA/BMEL clinical trial which is expected to deliver head-line results in sometime in December 2018 – a future event which remains Paradigm’s primary focus.