IPF treatment AGMB-447 granted FDA orphan drug status

Phase 1 trial expected to wrap up in March 2025

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by Andrea Lobo |

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AGMB-447, an inhaled treatment for idiopathic pulmonary fibrosis (IPF) now being tested in a Phase 1 clinical trial, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA).

The FDA gives this designation to therapies that may improve care for people with rare diseases, defined as those affecting fewer than 200,000 people in the U.S. The designation provides certain incentives to therapy developers,  including a guarantee of seven years of market exclusivity upon approval and tax credits for clinical trials.

“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,” Philippe Wiesel, MD, chief medical officer of Agomab Therapeutics, the company developing AGMB-447, said in a company press release.

Pulmonary fibrosis (PF) is characterized by fibrosis, or scarring, of the lungs. Fibrotic tissue causes lungs to become stiffer, making breathing more difficult and causing patients to experience symptoms like shortness of breath and cough. In IPF, the most common PF type, the exact cause of lung scarring is unknown.

AGMB-447 is a small molecule designed to specifically block ALK5 in the respiratory tract. ALK5 is the receptor of transforming growth factor-beta (TGF-beta), a well-established driver of fibrosis.

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Three-part trial underway

The therapy’s safety, tolerability, and pharmacological properties are being evaluated in a three-part Phase 1 trial (NCT06181370), which is expected to enroll 76 participants, including healthy volunteers (parts A and B) and people with IPF (part C). Participants will be randomly assigned to receive either AGMB-447 or a placebo, via inhalation through a nebulizer.

In part A, participants will receive single ascending doses of the therapy or a placebo, while in part B they will receive multiple ascending doses of AGMB-447 or a placebo for seven days. In part C, IPF patients ages 40 and older will receive multiple doses of the therapy or a placebo for 14 days.

The trial’s main goals are to assess AGMB-447’s safety and tolerability, as indicated by lab tests, vital signs, and side effects, which will all be assessed for up to eight weeks.

Secondary goals include characterizing AGMB-447’s pharmacokinetics, or its movement into, through, and out of the body, over the same period.

Launched last year, the Phase 1 study is expected to be completed in March 2025.

“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,” Wiesel said.