Prana Biotechnology (ASX:PBT) has delivered a presentation to the World Orphan Drug Congress in the US outlining its unique approach to treating age-related neurodegenerative disorders.
The congress brings together hundreds of the world’s leading researchers and pharmaceutical companies to share experiences and discuss how to expedite access to orphan drugs for patients with rare diseases.
Prana Acting Vice President of Business Development, Dr Birgit Anderegg, presented the company's approach to treating various neurological indications using its platform of Metal-Protein Attenuating Compounds.
“We’re aiming to close therapeutic gaps by addressing unmet medical needs in orphan indications such as Huntington’s disease and various atypical Parkinsonian movement disorders,” said Dr Anderegg.
Prana’s compounds target the metal induced build-up of toxic aggregated forms of proteins linked to neurodegenerative disorders, including alpha-synuclein, A-beta and tau.
Prana’s PBT2 compound has received orphan drug designation from the European Commission and US FDA for Huntington’s disease while its PBT434 compound has potential to treat a range of orphan disorders such as progressive supranuclear palsy, frontotemporal dementia and chronic traumatic encephalopathy.
PBT2 is currently on Partial Clinical Hold by the US FDA and the company continues to work on its substantive submission to reinstate clinical development in the United States. The company is continuing to explore opportunities outside of the US.