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

The BTD should help expedite the development and review of PTI-428, which Proteostasis believes can improve lung function in cystic fibrosis patients
Data from a recent phase II trial showed PTI-428 helped to boost levels of the CFTR protein in patients

Proteostasis Therapeutics Inc (NASDAQ:PTI) was the top riser on Wall Street after the small cap biopharma’s cystic fibrosis treatment was granted Breakthrough Therapy Designation (BTD) by the US Food and Drug Administration.

A BTD helps to expedite the development and review of a drug aimed at treating a serious or life-threatening diseases where there is significant unmet need and where clinical evidence has shown the drug may offer substantial improvement over existing therapies.

Strong phase II data

“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,” said President and chief executive Meenu Chhabra.

PTI-428 is Proteostasis’ most advanced candidate and is part of a class of treatments called cystic fibrosis transmembrane conductance regulator (CFTR) modulators.

When working normally, the CFTR protein regulates the proper flow of water and chloride in and out of the cells lining the lungs and other organs.

In people with cystic fibrosis though, mutations in the CTFR gene mean a defective protein, or no protein at all, is produced. That leads to a build-up of sticky mucus which can in turn cause infections in the lungs and damage to the pancreas.

Part of a potential triple combo therapy

CFTR modulators work by correcting the malfunctioning protein made by these CFTR-mutated genes, preventing the mucus from building up in the first place.

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

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

That said, the Massachusetts-based company is also developing its own triple combination regime.

Shares jumped 58.6% to US$6.57.

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