FDA Grants Orphan Drug Designation to AGMB-447 for Idiopathic Pulmonary Fibrosis


Currently, the drug is being evaluated in a phase 1 clinical trial to determine the safety and efficacy in patients.

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About the Trial

Trial Name: Phase I Study to Assess Safety, Tolerability, PK and PD of AGMB-447 in Healthy Participants and Participants With IPF

ClinicalTrials.gov ID: NCT06181370

Sponsor: Agomab Spain S.L.

Completion Date (Estimated): March 2025

The FDA has granted an orphan drug designation (ODD) to AGMB-447 (Agomab Therapeutics) for idiopathic pulmonary fibrosis (IPF). Currently, the investigational drug is being evaluated in a phase 1 clinical trial (NCT06181370) to determine its safety and efficacy.1

AGMB-447 is an inhaled small molecule lung-restricted inhibitor of ALK5, or TGFβRI, intended for the treatment of IPF and other fibrotic respiratory conditions. The drug is specifically designed to potently—and safely—inhibit ALK5 only in the lungs to prevent clinically relevant systemic exposure. Although there are some medicinal treatments without a lung transplant are available to patients with IPF, average survival is typically 3 to 5 years for those with the disease.1

IPF—which is often characterized by unregulated production of fibrotic, scar-like tissue that builds up in the lungs—is a disease that affects up to 100,000 patients in the US. It can cause the fibrotic lung to become stiff and hinder breathing while reducing the absorption of inhaled oxygen in the blood. TGFβ is master regulator of fibrosis in IPF, with preliminary clinical data supporting targeting the pathway.1

The phase 1 clinical trial (NCT06181370) is a randomized, single center, double-blind, placebo-controlled, single and multiple ascending dose, 3-part study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of AGMB-447 in patients with IPF. Patients were randomly assigned to receive either AGMB-447 or placebo, and receive a single dose in part A, multiple doses over 7 days in part B, and multiple doses again over 14 days in part C. Both AGMB-447 and placebo will be identical doses and administrated via inhalation.2

The primary end points of the trial are the number of participants who experience adverse events, as well as participants who have abnormal clinical laboratory values, electrocardiogram parameters, vital signs, physical exams, and spirometry parameters, all of which are being monitored from screening through study completion and up to 8 weeks. Additionally, secondary end points include plasma levels of AGMB-447 and the major metabolite to characterize pharmacokinetics of the drug by measuring the amount of plasma. Like the primary end points, secondary end points will also be monitored from screening through study completion and up to 8 weeks following the trial.2

Further, in addition to AGMB-447, investigators with Agomab Therapeutics are also looking at 3 other drugs, including AGMB-129, which is being evaluated in a phase 2a trial for the treatment of fibrostenosing Crohn disease; AGMB-101, which is undergoing IND-enabling studies for fibrotic indications; and AGMB-102, which is currently in discovery and is also intended for fibrostic indications. Philippe Wiesel, chief medical officer at Agomab Therapeutics notes that the pipeline will continue to grow with these antibody and molecule drug candidates with the goal of treating patients with difficult disease states.3

“Receiving ODD from the FDA provides further support that AGMB-447’s mechanism of action has the potential to achieve meaningful therapeutic benefits to patients [with IPF],” said Wiesel in a news 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.”1


1. Businesswire. Agomab Receives FDA Orphan Drug Designation for AGMB-447 in Idiopathic Pulmonary Fibrosis. News release. June 6, 2024. Accessed June 7, 2024. https://www.businesswire.com/news/home/20240606970963/en
2. Phase I Study to Assess Safety, Tolerability, PK and PD of AGMB-447 in Healthy Participants and Participants With IPF. ClinicalTrials.gov identifier: NCT06181370. Updated March 6, 2024. Accessed June 7, 2024. https://clinicaltrials.gov/study/NCT06181370
3. Agomab Therapeutics. Our Pipeline. Accessed June 7, 2024. https://agomab.com/pipeline/
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