Proteostasis Therapeutics, Inc. (PTI), a biotechnology company working on breakthrough therapeutics to treat diseases caused by defects in protein processing, recently announced that it has secured new financing worth $37 million, which will be dedicated to further developing its primary product candidate for cystic fibrosis for testing in human clinical studies.
Cystic fibrosis (CF) is the most common fatal, inherited disease in the United States. Improvement in disease management protocols and approval of new drugs to treat the symptoms have extended the life expectancy for CF patients, which is now approaching 40 years of age on average. However, CF remains incurable and those diagnosed with it typically succumb to the disease. Recently, however, CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) mutation-specific disease modulators such as correctors and potentiators have shown the ability to restore CFTR function in selected genotypes. These therapies do more than simply treat symptoms of CF — they address the underlying cause of the disease.
Proteostasis Therapeutics’ experimental therapy for cystic fibrosis is a completely new class of therapeutic agents known as cystic fibrosis transmembrane conductance regulator (CFTR) amplifiers. These CFTR amplifiers enhance the effect of other already established CFTR modulators, such as correctors and potentiators. Proteostasis’ preclinical studies showed that their CFTR amplifier was effective in treating the disease across a wide range of CFTR mutation classes, and PTI believes that early results point to the possibility of developing their therapy as a broad-acting combination therapy that can treat CF patients with most mutations.
News of the $37 million investment is significant for the CF community, since it will be used to accurate development of an experimental therapy that has the potential to benefit a large proportion of the patient population in the future.
“We are very pleased that we were able to attract such a strong investor group to our company at this important time in our development and we welcome the new investors to our existing group of top-tier investors,” said Meenu Chhabra, PTI’s President and Chief Executive Officer. “As we advance our programs into clinical development, PTI is in a strong position to improve upon present treatments for patients with cystic fibrosis. We are confident that we will continue to build on our promising preclinical results to advance our lead product in to the clinic.”