AGMB-447 Nabs Orphan Drug Designation as Potential IPF Treatment

People with idiopathic pulmonary fibrosis (IPF) already have two FDA-approved treatment options: Ofev (nintedanib) and Esbriet (pirfenidone). But biotech company Agomab Therapeutics (“Agomab”) is working to introduce another potential therapeutic candidate to the scene. The company’s inhaled small molecule lung-restricted inhibitor of ALK5 (of TGFβR1), which they call AGMB-447, is currently being explored in the clinical setting. AGMB-447 is designed to reduce or reverse lung fibrosis (scarring).

The ongoing Phase 1 clinical study, which launched six months ago, will enroll up to 76 individuals living with idiopathic pulmonary fibrosis. Some participants will receive a single AGMB-447 dose, while others will receive multiple doses. The trial is assessing tolerability, safety, efficacy, pharmacokinetics, and pharmacodynamics.

Potential benefits of AGMB-447 are already being recognized. In fact, reports Robert Barrie in Pharmaceutical Technology, the FDA granted Orphan Drug designation to the treatment in early June 2024. Orphan Drug designation stems from a need to improve and incentivize drug development in rare and orphan diseases. This designation, granted to therapies intended for indications affecting fewer than 200,000 people nationwide, comes with incentives: fee waivers, increased regulatory communication, tax credits, and seven years of market exclusivity if/when the drug is approved. Learn more about Orphan Drug designation here.

Get Educated on Idiopathic Pulmonary Fibrosis (IPF)

Idiopathic pulmonary fibrosis is a rare, serious, and chronic lung disease. The exact cause of IPF isn’t yet understood, hence the name (idiopathic translates to “of an unknown cause”). Physicians and scientists theorize that IPF results from a genetic susceptibility triggered by environmental triggers such as cigarette smoke or viral infections. Regardless of the cause, idiopathic pulmonary fibrosis causes lung tissue to become thickened, scarred, and hardened. This makes it much harder to breathe. Since this condition worsens over time, symptoms like shortness of breath only worsen. Right now, the prognosis associated with IPF is not always promising. However, treatment advances are working to slow disease progression and help people live longer.

Individuals with idiopathic pulmonary fibrosis may show signs or symptoms such as:

  • A dry, hacking cough that doesn’t really go away over time
  • Joint and muscle aches
  • Appetite loss
  • Fatigue and general weakness
  • Clubbed fingers and toes
  • Losing weight without trying or meaning to

IPF may also cause complications. These can include pulmonary embolisms (blood clots in the lungs), an increased risk of lung cancer, pulmonary hypertension, respiratory failure, chest pain, and pneumonia. Please speak with your physician about IPF management and what options are best for you to reduce complications and preserve your overall health. For example, your doctor may recommend pulmonary rehabilitation, oxygen therapy, or, in severe cases, a lung transplant.

Looking for additional support and resources? Head to the Pulmonary Fibrosis Foundation.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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