Proteostasis surges as FDA grants Breakthrough Therapy Designation its cystic fibrosis treatment

Breakthrough status in cystic fibrosis sends Proteostasis up by half

Breakthrough status in cystic fibrosis sends Proteostasis up by half

In a compelling tale for investors, shares of Proteostasis Therapeutics Inc (NASDAQ:PTI) are surging almost 50% higher as of this writing.

Proteostasis Therapeutics (NASDAQ:PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for PTI-428, the Company's cystic fibrosis transmembrane conductance regulator (CFTR) amplifier.

Ultimately, the FDA Breakthrough Therapy Designation is meant to expedite the development and review of a drug aimed at treating a serious or life-threatening disease when there is a significant unmet need and preliminary clinical evidence indicates that the drug may offer substantial improvement over existing therapies.

A recent phase II study showed that PTI-428 did indeed help to boost the levels of CFTR proteins in cystic fibrosis patients.

"We believe the breakthrough therapy designation for PTI-428 reflects the strength of the recent Phase II study results for our amplifier, a novel and proprietary class of CFTR modulators", said Meenu Chhabra, president and CEO of Proteostasis, in a statement. "We look forward to working closely with the FDA as we advance our clinical programs for PTI-428, including as part of our proprietary triple combination with PTI-801 and PTI-808, our third generation corrector and potentiator, respectively".

Proteostasis hopes the drug can be added to current and future treatments to improve lung function in those patients.

Proteostasis' stock is up 52% in pre-market trading, going for $6.29 per share at time of publishing.

Abrahams wrote, "As one of the only companies developing multiple clinicalstage CFTR modulators for the treatment of CF, we believe that PTI's potential for commercial success is now underappreciated. Given the large potential opportunity for a proprietary triple-combo in CF and the many shots-on-goal for individual modulators to be approved as adjunctive therapies for now marketed combinations, we believe the shares are undervalued".