Updated Interim Analysis of AIR Trial Shows C21 Promise for IPF


Currently, there are two FDA-approved treatments for people living with idiopathic pulmonary fibrosis (IPF): Ofev (nintedanib) and Esbriet (pirfenidone). While these therapies are effective in improving lung function, they may also come with intense and sometimes debilitating side effects. As a result, many people discontinue treatment. Novel treatment options with less side effects could significantly improve quality-of-life for people with IPF. 

That’s why Vicore Pharma Holding AB (“Vicore”), a Swedish clinical-stage pharmaceutical company, is developing Compound 21 (C21). The Pulmonary Fibrosis Foundation explains that C21, also known as VP01, is:

a first-in-class, orally available low molecular weight angiotensin II receptor type 2 (AT2R) agonist that activates the ‘protective arm’ of the renin angiotensin system (RAS).

Clinical Trial Information

Vicore recently reported that the company performed an updated interim analysis of its Phase 2a AIR study. Within the study, Vicore is exploring the therapy’s safety, efficacy, and pharmacokinetics. Pharmacokinetics means how the drug is absorbed, distributed, and moved within your body. 

So far, 51 participants with IPF have enrolled in the study. Following the interim analysis, Vicore reported that:

  • C21 shows sustained efficacy. At 36 weeks, patients had improved forced vital capacity (FVC), which can show how well their lungs are working. These patients had better FVC than a comparison group of untreated individuals with IPF. 
  • This therapy had no worrying safety signals. It was also relatively well-tolerated by participants. While some individuals had their IPF progress while on this treatment, most people felt that there were more benefits to C21 than risks. 
  • Treatment also helped reduce continued lung fibrosis (scarring), which was shown in some of the biomarkers. 

Ultimately, Vicore believes that this treatment could confer significant benefits over the current available treatments. The company hopes to soon move forward with a Phase 2b study to continue its evaluation of C21. 

An Exploration of Idiopathic Pulmonary Fibrosis (IPF)

What’s in a name? Well, for idiopathic pulmonary fibrosis, its name gives away a lot of insight into the condition:

  • Idiopathic: unknown cause
  • Pulmonary: relating to the lungs
  • Fibrosis: the development of fibrous scar tissue 

IPF refers to the hardening or scarring of lung tissue without a known cause. The current hypothesis is that IPF has a genetic and environmental basis: genetic susceptibility that is later triggered by an environmental factor such as viral illness, metal or wood dust, or cigarette smoking. 

As lung tissue hardens, oxygen has a much more difficult time entering the bloodstream. This chronic condition makes it harder to breathe. Unfortunately, IPF comes with a poor prognosis, especially after symptoms appear. Most cases come with a life expectancy of three to five years. Symptoms may include: 

  • Shortness of breath
  • A dry, hacking cough
  • Clubbed fingers or toes
  • Gastroesophageal reflux (GERD)
  • Appetite loss
  • Unintentional weight loss
  • Pulmonary embolisms 
  • Pneumonia (complication)
  • Lung cancer (complication)
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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