Product Development Pipeline

Pipeline Code Therapeutic Area Design Chemistry Pre-Clinical Clinical Collabration/Licensing Regulatory
ORPHAN DISEASES:
CB-ND-002 Nephropathic Cystinosis IND Filling 2020

Cellomatics BioSciences Ltd announced its collaboration with Krisani Biosciences to advance a novel molecular conjugate prodrug of cysteamine and eicosapentaenoic acid (EPA) for the therapeutic management of nephropathic cystinosis. This novel molecular conjugate prodrug of cysteamine and eicosapentaenoic acid (EPA) is patented in the United States.

Cellomatics BioSciences is leading the development of CB-ND-002 portfolio of cysteamine and eicosapentaenoic acid (EPA) conjugates/salts from non-clinical studies to Investigational New Drug Application filing with FDA. Cellomatics BioSciences has exclusive rights to develop the CB-ND-002 portfolio for global market. The collaboration also will advance the IND approved molecules through clinical stage studies, regulatory approval and commercialization of therapy for Nephropathic cystinosis.

Nephropathic Cystinosis:

Description:

  • Nephropathic cystinosis (NC) is a genetic error of metabolism disabling the mechanism for clearing the amino acid cystine, a breakdown product of cellular proteins. This results in an accumulation of cystine crystals in all organs and tissues.
  • Condition is usually diagnosed in early childhood when patients exhibit poor growth, vision problems (photophobia) and specific kidney problems (called Fanconi syndrome) that result in increased urination, thirst and dehydration. If untreated, NC destroys major organ systems including the kidneys, eyes, liver, muscles, pancreas and the brain. Additional complications include muscle wasting, poor growth, difficulty swallowing, diabetes and hypothyroidism.

Incidence:

  • NC is a rare disease with an incidence estimated to be 1 case / 100,000-200,000 living births. It is estimated to have around 500 patients in US and 2,000 worldwide with therapy cost of ~$750,000 per annum

Therapy and Reactions:

  • Cysteamine therapy may delay and/or prevent kidney transplant and other clinical manifestations of disease. Goal of cysteamine treatment of NC is to reduce cystine levels in cells. However, patient compliance is challenging due to frequent dosing and gastrointestinal side effects.

Our Unique Proposition:

Cellomatics BioSciences and KrisaniBio collaboration seek to significantly enhance the value proposition of new drug discovery in different unmet need therapeutic areas:

  • Proprietary and Innovative Platform based drug discovery and development
  • Designed & Developed I.P. backed Portfolio of novel potential therapeutic molecules
  • Drug candidates resulting have lower regulatory hurdles and mitigate the risk of product registration and launch
  • Cost and time to develop products is significantly reduced due to the use of existing and established molecules in some of our portfolios

Vision for CB-ND-002:

  • To develop once-a-day formulation with sustained response and efficacy
  • To further improve the acute (tolerability – nausea, vomiting, diarrhoea) and chronic (gastric and intestinal ulceration, bleeding) Adverse Effect profile of currently available PROCYSBI ®
  • Improve the compliance in paediatric population and reduce the healthcare burden