Cystic Fibrosis Program – Phase 1 Complete

Paranta is developing an inhaled form of PB01 (recombinant human follistatin-288) for the treatment of cystic fibrosis lung disease.

Inhaled PB01 offers a potential step change in the treatment landscape for CF patients, by targeting the underlying lung inflammation and fibrosis which occurs in CF. To date there are no safe and effective anti-inflammatories approved for the treatment of CF.

Activin A, a key regulator of lung inflammation, has been shown to be upregulated in CF. Adult CF patients have elevated levels of Activin A in their serum, and this increase in Activin A is correlated with decreased lung function and body weight.

PB01, a potent Activin A antagonist, is being developed as an anti-inflammatory treatment for CF patients to prevent on the chronic inflammation and fibrosis which develops in the lungs of CF patients.

Preclinical Results

Paranta’s preclinical studies have demonstrated the benefit of inhaled PB01 in two different, and clinically relevant, mouse models of CF lung disease. Both models demonstrate the capacity of inhaled PB01 to reduce lung neutrophil levels to similar levels found in healthy lungs (neutrophils are a type of white blood cell implicated in the progression of CF lung disease).

Preclinical studies in a transgenic mouse model of CF lung disease, treatment with inhaled PB01 significantly inhibited airway inflammation and reduced mediators of lung fibrosis.

In a second mouse model of CF, Cftr-knockout (CF) mouse model, inhaled PB01 also modulated key markers of lung inflammation. Importantly, inhaled PB01 did not worsen or impair the clearance of Pseudomonas aeruginosa lung infection. This is a clinically important outcome as it indicates that inhaled PB01 will not exacerbate lung infections in CF patients. Pseudomonas aeruginosa lung infection is a clinically important and common bacterial infection, affecting 50% of people with CF.