AGMB-447 is an investigational, lung-restricted, inhaled small molecule inhibitor of ALK5 (TGFβR1) in clinical development for Idiopathic Pulmonary Fibrosis (IPF).  

Core description  

Idiopathic Pulmonary Fibrosis (IPF) is a rare, progressive fibrotic lung disease with poor prognosis and limited treatment options. While there are available therapies that slow the decline of disease progression, there is still significant unmet need. AGMB-447 is being developed to address this gap.  

We seek to address a root cause of lung fibrosis while reducing the risks associated with systemic ALK5 inhibition. AGMB-447 is designed to inhibit ALK5 signaling directly in the lung, targeting the TGFβ-driven activation of fibroblasts and excessive deposition of extracellular matrix that underlie progressive lung scarring in IPF. Through inhalation we aim to achieve high lung exposure, where efficient hydrolysis minimizes systemic exposure. AGMB-447 may have potential either as monotherapy or in combination with other anti-fibrotic therapies.   

Phase 1 program
AGMB-447 has been evaluated in a Phase 1 study assessing safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy participants and participants with IPF. We reported positive topline interim results in 108 healthy participants showing low systemic exposure with high lung exposure, together with dose-dependent target engagement measured by reduction of pSMAD3 in bronchoalveolar lavage cells. (NCT06181370)

AGMB-447 continues to be evaluated in 10 participants with IPF to further characterize its safety, pharmacokinetic profile, and pharmacodynamic activity in the target patient population. We expect to report data from the IPF patient cohort in the second half of 2026.  

Planned Phase 2 study
Based on the Phase 1 observations reported to date, we plan to initiate a Phase 2 study of AGMB-447 in patients with IPF before year end 2026.

 

AGMB-447 has received U.S. FDA Orphan Drug Designation, a status granted by the FDA to promising treatments in development for rare diseases.
AGMB-447 is an investigational product candidate and is not approved by any regulatory authority. Its safety and efficacy have not been established.