PTGP-150

PTGP-150 is a small molecule kinetic stabilizer designed to treat λ light chain amyloidosis (AL). Developed by Protego Biopharma, it works by binding to a conserved induced-fit pocket at the interface of the λ free light chain (FLC) variable domains. This binding stabilizes the FLC homodimers and monomers, preventing the misfolding, fragmentation, and aggregation that lead to toxic amyloid fibril formation. PTGP-150 has demonstrated the ability to thermodynamically and kinetically stabilize a wide range of amyloidogenic λFLC sequences across multiple subfamilies (v1, v2, v3, and v6) and protect them from proteolytic degradation. In ex vivo studies using patient plasma, PTGP-150 reduced the levels of unstable FLC and decreased protein toxicity.