PROT-001: Our Lead Program
Targeting AL Amyloidosis
In immunoglobulin light chain (AL) amyloidosis, light chains can misfold and form species that contribute to organ injury. Our lead program focuses on stabilizing lambda light chains.
Intended mechanism of action
PROT-001 is designed to bind lambda light chains and stabilize their native conformation, with the aim of reducing misfolding and the formation of toxic species.
By targeting the light chain protein itself, PROT-001 is designed to complement plasma cell-directed therapies, which reduce light chain production. We are investigating whether this approach can address light chain toxicity and contribute to the goal of preserving organ function.
Clinical development
PROT-001 is in clinical development for AL amyloidosis. It completed a Phase 1 study (PRO-1A-101) in healthy volunteers. In October 2026, the U.S. Food and Drug Administration cleared the Investigational New Drug (IND) application for PROT-001, enabling initiation of SHIELD-AL, a global pivotal Phase 2/3 study in newly diagnosed lambda AL amyloidosis patients. The study is planned to begin in the first quarter of 2027.
PROT-001 is investigational. Its safety and efficacy in patients with AL amyloidosis have not been established.
