ALK5 inhibitor (Alchemedicine)

Agomab is now eligible for market exclusivity and a range of financial incentives. Image credit: Shutterstock / HammadKhn. Six months after kicking off a Phase I trial for idiopathic pulmonary fibrosis (IPF) treatment AGMB-447, Agomab Therapeutics has now secured a US Food and Drug Administration (FDA) orphan drug designation for the asset. Recommended Buyer's Guides Buyer's Guide Leading Guide to Packaging Materials, Containers and Containment Services for the Pharmaceutical Industry Buyer's Guide Leading Guide to Compliance Software for the Pharmaceutical Industry The Belgium-based biotech will now be in line for tax credits for US-based clinical trials and, if the therapy is approved, the potential for seven years of market exclusivity in the designated indication. IPF is a rare, chronic lung disease that affects the air sacs – called alveoli – in the lungs. The condition is characterised by thickening of lung tissue that becomes stiff, causing breathing difficulties. It is not known what causes the disease, though the risk is increased with smoking and a family history of IPF. There are currently two FDA-approved drugs for IPF, Boehringer Ingelheim’s Ofev (nintedanib) and Genentech’s Esbriet (pirfenidone). Ofev is a tyrosine kinase inhibitor whilst Esbriet targets TGF-beta, a chemical mediator that plays a role in development of fibrosis. Agomab has gone down a similar route to Genentech, with AGMB-447 blocking the action of the TGFβ type 1 receptor. Agomab’s USP is that AGMB-447, which is inhaled, inhibits the TGFβ type 1 receptor, also known as ALK5, only in the lung. The biotech has achieved this by designing the drug to be rapidly metabolised through hydrolysis in the plasma, preventing exposure throughout the whole body. See Also: The tiny pills that may send shockwaves across immunology Roche’s PI3K inhibitor secures breakthrough status in breast cancer Agomab is evaluating AGMB-447 in a placebo-controlled Phase I trial that aims to enrol 76 patients with IPF. Participants will either receive a single dose or multiple doses of AGMB-447, the latter treatment will occur in two cohorts over seven days or 14 days. The randomised trial has primary endpoints comprising safety assessments up to eight weeks after therapy. Secondary endpoints include pharmacokinetic measurements. The biotech dosed the first patient in December 2023. Agomab’s chief medical officer Philippe Wiesel said: “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 secured $100m in Series C financing in October 2023. In a statement at the time, the biotech said it would use part of the funds to progress Crohn’s disease candidate AGMB-129 – also an ALK5 inhibitor – through Phase II trials. Agomab’s designation comes only a few weeks after Alentis Therapeutics won orphan drug status for its candidate lixudebart in IPF. In the same announcement as its orphan drug designation, Alentis reported positive safety results from a Phase I trial evaluating lixudebart in IPF patients.

AGMB-447 is an inhaled lung-restricted ALK5-inhibitor currently in a Phase 1 clinical trial ANTWERP, Belgium--(BUSINESS WIRE)-- Agomab Therapeutics NV (‘Agomab’) today announced that it has received Orphan Drug Designation from the U.S. 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 (NCT06181370). 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 U.S. 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. “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,” said Philippe Wiesel, Chief Medical Officer at Agomab Therapeutics. “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. About AGMB-447 AGMB-447 is a small molecule lung-restricted inhibitor of ALK5 (or TGFβRI) for the treatment of Idiopathic Pulmonary Fibrosis (IPF) and other fibrotic respiratory indications. IPF is a devastating disease affecting 100,000 patients in the U.S. IPF is characterized by unregulated production of fibrotic, scar-like tissue that builds up in the lungs. As a result, the fibrotic lung becomes stiff which hampers breathing and reduces the absorption of inhaled oxygen in the blood. Even though some medicinal treatments are available, without a lung transplant, the average survival following diagnosis is only three to five years. TGFβ 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. About Agomab Agomab is focused on achieving disease modification by modulating fibrosis and regeneration in chronic indications such as Fibrostenosing Crohn’s Disease and Idiopathic Pulmonary Fibrosis. We do this by targeting biologically validated pathways – including Transforming Growth Factor β and Hepatocyte Growth Factor - and by applying specialized capabilities in organ-restricted small molecules and high affinity antibodies. With a differentiated clinical pipeline across several fibrotic disorders, end-to-end research and development capabilities, a proven BD track-record and a strong investor base, Agomab is building a leading European biopharma company. View source version on businesswire.com: Contacts For Agomab Therapeutics Tim Knotnerus, CEO E-Mail: tim.knotnerus@agomab.com Media Requests for Agomab Dr. Stephanie May or Gretchen Schweitzer Trophic Communications Phone: + 49 (0) 172 861 8540 E-Mail: agomab@trophic.eu Source: Agomab Therapeutics NV View this news release online at: