Proteostasis Therapeutics Inc (NASDAQ: PTI) shares rose by leaps and bounds Tuesday, after the company disclosed that the U.S. Food and Drug Administration has granted breakthrough therapy designation for PTI-428 in Cystic Fibrosis (CF)
The firm, based in Cambridge, Mass., is a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat CF and other diseases caused by dysfunctional protein processing.
FDA Breakthrough Therapy Designation is intended to expedite the development and review of a drug aimed at treating a serious or life-threatening disease where there is a significant unmet need and preliminary clinical evidence indicates that the drug may offer substantial improvement over existing therapies.
Less than half of the drugs submitted for Breakthrough Therapy under the Food and Drug Administration Safety and Innovation Act have been granted the designation since the inception of the program. In the CFTR modulator category, Breakthrough Therapy Designation was granted to Kalydeco® and Orkambi®.
Proteostasis CEO Meenu Chhabra said "We believe the Breakthrough Therapy Designation for PTI-428 reflects the strength of the recent Phase 2 study results for our amplifier, a novel and proprietary class of CFTR modulators.
"PTI-428 can potentially be added to current and future standards of care, offering the potential for improvement in pulmonary function for patients with cystic fibrosis."
Proteostatis shares ballooned early Tuesday afternoon by $2.29, or 55.3%, to $6.43, within a 52-week high of $1.41 to $6.43.
