Indications

Indications

Paranta Biosciences is developing potentially transformative first-in-class biotherapeutics based on PB01, a unique form of recombinant human follistatin, targeting inflammatory, fibrotic and metabolic diseases with significant unmet need.

The Company's lead program involves the development of an inhaled form of PB01 for treating cystic fibrosis lung disease. This program has completed Phase I clinical development. The Company has additional programs in preclinical development. These programs involve the development of injectable PB01 for treating diabetic kidney disease and other diseases.

Cystic Fibrosis

Lung disease is the leading cause of morbidity and mortality in patients with cystic fibrosis

Cystic fibrosis is a chronic, life threatening genetic disease caused by mutations in the CFTR gene. The disease is characterized by excessive mucus, chronic inflammation and infection in the lungs which leads to loss of lung function and irreversible lung damage (bronchiectasis and fibrosis). In the lungs, the mucus clogs the airways and traps bacteria leading to infections, extensive lung damage, and eventually, respiratory failure.

The median forecast survival age for a person with CF is approximately 40 years. According to the Cystic Fibrosis Foundation, more than 30,000 Americans and over 70,000 people worldwide suffer from the disease, with approximately 1,000 new cases diagnosed each year.

Inflammation plays a critical role in the progression of CF lung disease. Despite the recent expansion in treatment options for CF with the introduction of medicines targeting specific CF mutations, currently there are no safe and effective anti-inflammatories approved for treating CF lung disease.

Paranta is developing inhaled PB01 for the treatment of CF lung disease. PB01 targets lung inflammation, fibrosis and mucus production and therefore has the potential to slow the progression of lung disease and preserve lung function in people living with CF.

For more information, please refer to the Cystic Fibrosis Foundation: www.cff.org

  • ~75,000

    People living with Cystic Fibrosis

  • 40 Years

    Median forecast life expectancy

  • >85%

    Mortality from lung disease

  • 50%

    Cystic fibrosis population >18 years

Oncology

NSCLC accounts for >85% of all lung cancers. Globally, 1.5M people are diagnosed with NSCLC annually. Long term outcomes are very poor for patients with advanced disease, with only approximately 5% surviving 5 years.

PB01 in vivo studies have shown that parenterally administered PB01 enhances the sensitivity of resistant NSCLC cells to chemotherapy agents, and also reduces platinum-based nephrotoxicity. PB01's activity in other cancer cell lines is currently underway.

Diabetic Nephropathy

Diabetic nephropathy, a form of chronic kidney disease, is a major comorbidity of diabetes affecting over 150 million diabetic patients worldwide.

Diabetic nephropathy, a form of chronic kidney disease, is a major comorbidity of diabetes affecting over 150 million diabetic patients worldwide. It is the leading cause of chronic kidney disease with 40% of kidney failures in the US attributed to diabetes. Current treatment options for diabetic nephropathy are only partially effective and offer limited ability to delay disease progression.

Currently approved treatment options include blood pressure medications that delay, but do not stop, the progression of the disease. Treatment options for patients with severely limited kidney function include dialysis or kidney transplantation. There is an increasing, and significant, need for novel therapeutic approaches.

PB01's anti-inflammatory and antifibrotics actions have the potential to offer diabetic kidney disease patients a highly effective and novel treatment to reduce long term kidney damage and fibrosis.

For more information, please refer to the American Diabetes Association: www.diabetes.org

  • >420 Million

    People with diabetes

  • ~40%

    People with diabetes who will develop diabetic kidney disease

  • ~40%

    Kidney failures due to diabetes