Proteostasis Therapeutics (PTI) is a small-cap company developing small molecule therapeutics to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing. Recently, the company announced positive preliminary results from its ongoing Phase 1 study of combination therapies for CF.
Cystic fibrosis (CF) is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe. More than 30,000 people in the US and greater than 70,000 worldwide live with the disease.
Each year approximately 1,000 new cases are diagnosed, according to the Cystic Fibrosis Foundation. Of those, more than 75% of patients diagnosed by age 2.
People with CF have a defective gene that causes a buildup of mucus in the lungs, pancreas, and other organs. Airways can become clogged causing respiratory failure and experience the blocking of digestive enzymes in the pancreas that can impair the body's ability to absorb vital nutrients
The combination treatments Proteostasis Therapeutics is developing showed improvement in the percent predicted forced expiratory volume and sweat chloride concentration across all cohorts. The improvement was significantly greater than the norm using the current standard of care.
The stock spiked significantly on this report and had been trading between $6 to $8. Proteostasis took advantage of its recent increase in share price to raise $74 million by pricing 11 million shares in a Form S-3 shelf registration.
Proteostasis is committed to developing novel therapeutics to treat diseases caused by an imbalance in the "proteostasis" or the protein homeostasis network set of biological pathways that control the creation, folding, transportation, maintenance and degradation of proteins in the body.
An imbalance in this network can lead to poor protein quality control contributing to diseases such as CF and gain-of-toxic function disorders like Alzheimer’s, Huntington’s and Parkinson’s diseases.
Proteostasis went public in 2016 with a market cap of under $200 million. The recent developments may provide astute investors with an opportunity to get on board and benefit from the growth of this exciting company that despite its success may still be a great value and growth opportunity.