Prothena Corporation plc &  Bristol Myers Squibb communicated that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to BMS-986446, a potential best-in-class anti-microtubule binding region-tau (anti-MTBR-tau) antibody currently in Phase 2 development for the treatment of early Alzheimer’s disease. Fast Track Designation is intended to facilitate the development and expedite the review of investigational drugs that treat serious conditions and fill an unmet medical need.

Alzheimer’s disease — the most common type of dementia in adults — is a progressive, multifaceted and devastating neurodegenerative disease in which significant changes occur in the brain that cause neurons to die over time. These changes include the accumulation and spread of pathological tau, an abnormal form of the tau protein. Pathological tau protein fragments containing the microtubule binding region (MTBR) appear to have a key role in the underlying pathology of Alzheimer’s disease. By neutralizing the spread and promoting the clearance of pathological tau, BMS-986446 aims to modify the underlying course of the disease with the ultimate goal of slowing or delaying disease progression.

In preclinical models, BMS-986446 demonstrated significant reductions in tau uptake and spread, protection against behavioral deficits and was localized with tau pathology in Alzheimer’s brain tissue. BMS-986446 was also shown to be safe and well tolerated across three dose cohorts in a Phase 1 study of healthy participants. Bristol Myers Squibb also announced that the ongoing Phase 2 study is fully enrolled and includes several biomarkers of tau and amyloid-beta biology, as well as clinical outcome measures, to evaluate the impact of BMS-986446 on disease progression.

As part of the BMS-986446 global license with Bristol Myers Squibb, Prothena is eligible to receive additional regulatory and sales milestone payments of up to $562.5 million. Prothena also is eligible to receive tiered royalties on net sales. Bristol Myers Squibb is responsible for all communication, development, manufacturing, and commercialization for BMS-986446.