Agomab secures FDA orphan drug status for idiopathic pulmonary fibrosis treatment

Agomab Therapeutics has reached a significant milestone by obtaining the US Food and Drug Administration (FDA) orphan drug designation for its idiopathic pulmonary fibrosis (IPF) treatment, AGMB-447. This progress comes six months after the initiation of a Phase I clinical trial for the drug.

Based in Belgium, Agomab is now poised to benefit from various incentives, including tax credits for clinical trials conducted in the US and the possibility of seven years of market exclusivity if AGMB-447 is approved. This designation is a crucial step for Agomab in its efforts to address IPF, a rare and chronic lung disease.

IPF primarily affects the alveoli, the air sacs within the lungs, causing the lung tissue to thicken and stiffen, which significantly hampers breathing. While the exact cause of IPF remains unclear, factors such as smoking and a family history of the condition are known to increase the risk.

Currently, there are two FDA-approved treatments for IPF: Boehringer Ingelheim’s Ofev (nintedanib) and Genentech’s Esbriet (pirfenidone). Ofev operates as a tyrosine kinase inhibitor, whereas Esbriet targets TGF-beta, a mediator involved in fibrotic processes. Agomab’s AGMB-447 also takes aim at TGF-beta but does so by inhibiting the TGFβ type 1 receptor, known as ALK5, specifically in the lungs. This selective inhibition is achieved by designing AGMB-447 to be quickly metabolized through hydrolysis in the plasma, thereby minimizing systemic exposure.

The ongoing Phase I trial of AGMB-447 is a placebo-controlled study designed to enroll 76 IPF patients. Participants in the trial will receive either a single dose or multiple doses of AGMB-447. The multiple dose regimen is divided into two cohorts, each treated over a period of seven or 14 days. The trial's primary endpoints include safety assessments conducted up to eight weeks post-therapy, while secondary endpoints focus on pharmacokinetic data. The first patient was dosed in December 2023.

Philippe Wiesel, Agomab’s chief medical officer, expressed optimism about the trial, stating, “As we progress through our ongoing first-in-human Phase I 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.”

Agomab's recent advancements also include securing $100 million in Series C financing in October 2023. The company has allocated part of these funds to advance another candidate, AGMB-129, intended for Crohn’s disease, through Phase II trials. AGMB-129, like AGMB-447, is also an ALK5 inhibitor.

The orphan drug designation for AGMB-447 follows closely on the heels of a similar announcement from Alentis Therapeutics, which received orphan drug status for its IPF treatment candidate, lixudebart. Alentis also shared positive safety results from a Phase I trial evaluating lixudebart in IPF patients.

Agomab’s achievements highlight the ongoing efforts and advancements in the development of treatments for rare diseases like IPF, where innovative approaches and regulatory incentives play a crucial role in bringing new therapies to patients in need.