PROTHENA CORP PUBLIC LTD CO (PRTA)

Prothena is a late-stage clinical biotechnology company focused on protein dysregulation. It discovers and develops investigational therapeutics for neurodegenerative diseases and rare peripheral amyloid diseases, which are caused by misfolded, aggregated proteins. The company was formed in Ireland in 2012 and is an Irish public limited company. Its ordinary shares trade on the Nasdaq Global Select Market under the symbol "PRTA". It aims to become a fully integrated research, development and commercial biotechnology company.

Its wholly-owned programs include birtamimab for AL amyloidosis and a portfolio of Alzheimer's disease programs. These include PRX012, a subcutaneously dosed anti-amyloid beta antibody, and PRX123, a dual amyloid beta-tau vaccine. Birtamimab is in the Phase 3 AFFIRM-AL study in Mayo Stage IV patients, run under a Special Protocol Assessment agreement with the FDA. It has Fast Track Designation and Orphan Drug Designation from the FDA and EMA. Partnered programs include prasinezumab, an anti-alpha-synuclein antibody for Parkinson's disease, which Roche is developing in the Phase 2b PADOVA study. Programs targeting tau (PRX005), TDP-43 for ALS, and an undisclosed target (PRX019) are partnered with Bristol Myers Squibb. Prothena is also entitled to potential milestone payments from Novo Nordisk, which acquired its ATTR amyloidosis business and is running a Phase 2 study of NNC6019 (formerly PRX004) in ATTR cardiomyopathy. The clinical pipeline has five antibody programs. Discovery and preclinical work covers Aβ, tau and TDP-43. The company describes itself as modality agnostic, with antibody, small molecule and vaccine approaches.

The company targets serious diseases that lack effective therapies. These include Alzheimer's disease, Parkinson's disease (an estimated 10 million people worldwide), AL amyloidosis (an estimated 200,000 to 400,000 patients globally, about 30% of them Mayo Stage IV) and ATTR amyloidosis. For commercialization it is weighing several options: building its own sales force, co-selling partnerships, regional licensing, or out-licensing for royalties. The Roche collaboration and the Novo Nordisk transaction are examples of its partnering approach.

Its biology-directed discovery engine relies on pathophysiology-directed targeting, epitope mapping and disease-driven antibody engineering. These are meant to target pathogenic protein forms while leaving normal proteins unaffected. The company combines internal talent with outside consultants, advisors and academic and industry collaborators. Roche and Novo Nordisk run the late-stage trials of prasinezumab and NNC6019. The text gives no information on manufacturing, suppliers, competitors or headquarters.