IPF Drug TTI-101 Earns Orphan Drug Designation


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
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.

Follow us