In the past, medical research has shown that the STAT3 protein plays a role in regulating inflammatory responses throughout the body; it also plays a role in cell proliferation, migration, and apoptosis. As a result, STAT3 has been implicated in the development of several cancers causing solid tumors, such as breast, liver, and lung cancer. According to Pulmonary Fibrosis News, TTI-101, a STAT3 inhibitor designed to treat idiopathic pulmonary fibrosis (IPF), recently earned Orphan Drug designation from the FDA. 

TTI-101: An Overview

Developed by Tvardi Therapeutics, TTI-101 is a small molecule STAT3 inhibitor. Because STAT3 can prompt inflammatory responses, such as those which cause fibrosis (scarring) in IPF, Tvardi Therapeutics believes that TTI-101 can mediate those responses and improve patient outcomes. Preclinical studies have shown that TTI-101 treatment has helped improve lung function, as well as stopped or even reversed fibrosis. 

Currently, a Phase 1/2 clinical trial is evaluating TTI-101 in conjunction with other therapies for breast cancer. Another Phase 1 study is evaluating the safety, pharmacokinetics, and tolerability of TTI-101. This study may help find the ideal dose for future clinical trials, of which two are already planned. 

TTI-101 received Orphan Drug designation for IPF. Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases (affecting fewer than 200,000 Americans). As a benefit, Tvardi Therapeutics earns fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity upon drug approval. 

What is Idiopathic Pulmonary Fibrosis (IPF)? 

Idiopathic pulmonary fibrosis (IPF) can be understood by unpacking its name: idiopathic (of unknown cause), pulmonary (relating to the lungs), fibrosis (scarring). So IPF is basically the hardening or scarring of lung tissue without a known cause. Although the cause is unknown, some researchers hypothesize that a mix of genetics and environmental triggers may prompt the disease. As the lungs become increasingly scarred, adequate oxygen is unable to enter the bloodstream. Symptoms associated with IPF can include:

• A dry, hacking cough
• Shortness of breath and/or difficulty breathing
• Clubbed fingers and toes
• Gastroesophageal reflux (GERD)
• Appetite loss
• Unintended weight loss
• Blood clots in the lungs
• Pneumonia
• Increased risk of lung cancer