Prothena (PRTA) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Pipeline and scientific focus

• Emphasis on differentiated therapeutics targeting protein misfolding in CNS and peripheral amyloid diseases, including Alzheimer's, Parkinson's, AL and ATTR amyloidosis.

• Programs include phase III for birtamimab (AL amyloidosis), phase I for PRX012 (Alzheimer's), and partnered programs with Roche, Bristol Myers Squibb, and Novo Nordisk.

• Each therapeutic approach is tailored to the disease biology, targeting abnormal protein conformations while sparing normal function.

• PRX012 is designed to target both deposited and soluble toxic amyloid in Alzheimer's, aiming for a best-in-class, once-monthly, subcutaneous administration.

• Prasinezumab (Parkinson's) and PRX005 (tau, Alzheimer's) are engineered for high selectivity and functional benefit, with PRX005 now in phase II with Bristol Myers Squibb.

Clinical progress and upcoming milestones

• Birtamimab's phase III AFFIRM trial in advanced AL amyloidosis is nearing readout, with prior data showing robust survival benefit in high-risk patients.

• PRX012 phase I multiple dose trial is progressing well, with a data or timing update expected by year-end.

• PADOVA readout for prasinezumab in Parkinson's is anticipated later this year.

• PRX005 phase II study for tau in Alzheimer's is enrolling, with design details recently presented at scientific meetings.

• Ongoing innovation includes enrolling APOE4 homozygotes in PRX012 trials, a population previously understudied.

Strategic partnerships and commercialization

• About half the pipeline is partnered, balancing internal expertise and capital with external bandwidth and value-add.

• Strong collaborations with Roche (prasinezumab), Bristol Myers Squibb (tau, PRX019), and Novo Nordisk (ATTR) support clinical advancement.

• Birtamimab is a wholly owned program, with plans to commercialize independently if phase III is successful.

• Commercial planning for birtamimab is underway, leveraging deep experience in the amyloidosis space.

• The company is prepared to file an NDA and build commercial infrastructure following a positive AFFIRM readout.