AGMB-447, a small-molecule inhibitor of ALK5, is being evaluated in a phase 1 clinical trial.
RT’s Three Key Takeaways
- The FDA has awarded Orphan Drug Designation to an inhaled small molecule inhibitor of ALK5, aimed at treating idiopathic pulmonary fibrosis (IPF).
- The designation highlights the potential of the drug’s mechanism of action to provide meaningful therapeutic benefits to patients suffering from IPF.
- The investigational drug is currently being evaluated in a phase 1 clinical trial, assessing its safety and tolerability in both healthy subjects and IPF patients.
Agomab Therapeutics NV announced it has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for AGMB-447, its inhaled, small-molecule inhibitor of ALK5.
Agomab is evaluating AGMB-447 as a potential treatment for idiopathic pulmonary fibrosis (IPF) in a phase 1 clinical trial.
The FDA’s Orphan Drug Designation program is designed to facilitate development of medicinal treatments for rare diseases that affect fewer than 200,000 people in the United States. The designation provides companies with various development and commercial benefits, including market exclusivity and a range of financial incentives, such as tax relief for clinical research costs.
AGMB-447 for IPF Treatment
ALK5 (or TGFβRI) is a known master regulator of fibrosis in IPF, and preliminary clinical data supports targeting the pathway. AGMB-447 is specifically designed to potently and safely inhibit ALK5 only in the lung due to its rapid metabolism through hydrolysis in plasma, which prevents clinically relevant systemic exposure.
“Receiving Orphan Drug Designation from the FDA provides further support that AGMB-447’s mechanism of action has the potential to achieve meaningful therapeutic benefits to IPF patients,” says Philippe Wiesel, chief medical officer at Agomab, in a release. “As we progress through our ongoing first-in-human phase 1 trial, we look forward to evaluating the data from the single ascending dose and multiple ascending dose evaluation of AGMB-447 in healthy subjects and IPF patients.”
AGMB-447 is an investigational drug and not approved by any regulatory authority. Its efficacy and safety have not been established.
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