European Commission approves Orphan Designation for Alterity’s lead drug candidate
Alterity Therapeutics Limited (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”) announces that the European Commission (EC) has granted Orphan Drug designation for its lead molecule, PBT434, for the treatment of Multiple System Atrophy (MSA).
Orphan Drug designation by the EC entitles Alterity to ten years of market exclusivity in the European Union for the use of PBT434 in the treatment of MSA and other benefits including assistance in developing clinical protocols, reduced fees and access to EU-funded research grants.
The approval was based on the recommendation of a positive opinion from the European Medicines Agency’s Committee for Orphan Medicinal Products, which was announced on the 18th of November 2019.
“This is an important milestone for Alterity as we advance PBT434 toward patient studies and it follows Orphan Drug designation from the US FDA for treating MSA. It reinforces the dire need for treatment options for this particularly debilitating disease and supports our efforts in preparing for our Phase 2 clinical trial,” said Dr David Stamler, Chief Medical Officer and Senior Vice President, Clinical Development.
Last year the company announced Phase 1 clinical trial results. PBT434 was found to be safe and well-tolerated in adult and older adult (≥ 65 years) subjects with an adverse event profile comparable to placebo. The clinically tested doses achieved concentrations in brain that exceeded those associated with efficacy in animal models of MSA and Parkinson’s disease (PD).
PBT434 looks to treat Parkinsonian disorders such as MSA and PD. These neurogenerative diseases result from accumulation of aggregated alpha-synuclein protein. PBT434 was found to inhibit the alpha-synuclein aggregation, preserve neurons and improve motor function in pre-clinical models of PD and MSA.
Authorisation & Additional information
This announcement was authorised by Geoffrey Kempler, CEO and Chairman of Alterity Therapeutics Limited.