Development Pipeline

Our pipeline applies protein stabilization to AL amyloidosis and other protein misfolding diseases. Our lead program, PROT-001, is in clinical development for AL amyloidosis and is designed to stabilize lambda light chains.

Our Development Pipeline

We are also pursuing earlier-stage research programs. Each program's target and development stage are shown below.

Programs and development stages

PROT-001

Disease
AL amyloidosis
Target
Lambda (λ) light chains
Stage
Phase 1 completed; IND cleared for SHIELD-AL, a Phase 2/3 study planned to start in Q1 2027

PROT-002

Disease
AL amyloidosis
Target
Lambda (λ) and kappa (κ) light chains
Stage
Lead optimization

Undisclosed program

Disease
Undisclosed
Target
Undisclosed
Stage
Discovery

Our lead program

PROT-001 is an investigational, first-in-class oral small molecule designed to stabilize lambda light chains in their native conformation, with the aim of reducing misfolding and the formation of toxic species.

PROT-001 completed a Phase 1 study (PRO-1A-101) in healthy volunteers. In October 2026, the U.S. Food and Drug Administration cleared the 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.

Explore PROT-001