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.

The Cftr-knockout (CF) mouse model studies were undertaken at Case Western Reserve University, and were supported by a Therapeutics Development Award from Cystic Fibrosis Foundation Therapeutics, Inc.

Cystic Fibrosis Program

Phase 1 Complete

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Paranta is developing an inhaled form of PB01 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.

Positive safety and tolerability with inhaled PB01 has been demonstrated in three Phase 1 clinical studies in healthy volunteers. The studies were undertaken in Australia and in the UK. Paranta is now focused on advancing the clinical development of inhaled PB01 in CF with input from local and global CF experts.

Nephropathy Program


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An injectable form of PB01 is being developed as a treatment for diabetic nephropathy, a major comorbidity of diabetes. Preliminary preclinical studies have demonstrated PB01’s ability to reduce kidney damage and fibrosis in a mouse model of diabetic nephropathy. This work forms part of a collaborative research project with McMaster University, Canada. This research collaboration has recently been expanded to include other forms of chronic kidney disease.

Oncology Program


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Paranta is investigating the effect of intravenous PB01 on the body’s immune system as a potential therapy for treating cancer. This research is being undertaken in collaboration with a leading Australian medical research institute. Encouraging preliminary results in animal studies have been produced.

Pipeline and Clinical Trials


Paranta Biosciences has progressed its recombinant human form of follistatin PB01





Phase 1
Market Opportunity
(Inhaled PB01)
Cystic fibrosis
~75,000 people with CF No anti-inflammatories approved for CF lung disease
(s.c. PB01)
Diabetic kidney disease
Affects >150 M diabetic patients Significant need for disease modifying treatments
(i.v. PB01)
Significant need for novel treatments that target the body’s immune response to cancer
Phase ongoing
Phase complete   


Paranta Biosciences is focused on the development and commercialization of transformative medicines, based on the follistatin-activin pathway, to treat inflammatory, fibrotic and metabolic diseases. We welcome interest from parties looking to establish new alliances or collaboration opportunities that complement our research efforts and align with Paranta's corporate strategy.

We are open to collaborations that enable our partners to develop and commercialize Paranta's technologies worldwide, including out-licensing and R&D partnerships, and collaborations that maximize the potential of our pipeline, and our ability to deliver treatments to patients around the globe.

If you would like to discuss partnering with Paranta, please contact: