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.

Delayed Graft Function


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Paranta has developed PB01 to be administered intravenously during kidney transplantation for the prevention of delayed graft function. Prior to committing to a full clinical development program in patients undergoing kidney transplantation, Paranta will confirm preclinical proof of concept in a model of ischemia reperfusion injury. Results are anticipated by end Q1 2020.

Acute Kidney Injury


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PB01 efficacy in renal DGF underpins the rationale to expand its use to other causes of AKI, such as: cardiovascular surgery, traumatic injury, clots and other causes of reduced blood flow to the kidneys; drug and toxin induced injury including chemotherapy; and sepsis.

Program Status


Paranta Biosciences has progressed its recombinant human form of follistatin PB01





Phase 1
Market Opportunity
(i.v. PB01)
Kidney DGF
Significant need for therapy to increase organ donor pool, reduce transplant waiting times, reduce morbidity and mortality; estimated $600M market opportunity in US
Acute Kidney Injury
(i.v. PB01)
Drug-induced AKI
Significant need for effective therapy to reduce end-stage renal failure
Phase ongoing
Phase complete   


Paranta Biosciences is focused on the development and commercialization of PB01 to modulate the activin pathway for the treatment of inflammatory and fibrotic 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: