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Proteostasis Stock Surges On Positive Data From Cystic Fibrosis Studies


Proteostasis Therapeutics, Inc.'s PTI shares surged more than 160% on Dec 12 after the announcement of positive results from studies evaluating its three cystic fibrosis ("CF") pipeline candidates. The three candidates include PTI-428 (a cystic fibrosis transmembrane conductance regulator [CFTR] amplifier), PTI-801 (a CFTR corrector) and PTI-808 (a CFTR potentiator).

PTI-428 and PTI-801 were evaluated in patients who were on background therapy with Vertex Pharmaceuticals Incorporated's VRTX Orkambi.

Shares of the company were up 60.7% in the past six months, outperforming the industry's decline of 0.6% in that period.

The most advanced candidate, PTI-428, was evaluated in a phase II study in CF patients on background Orkambi therapy. Data showed that the addition of PTI-428 to Orkambi had mean absolute improvements in ppFEV1 (a measure of the lung function used for the disease) of 5.2% from baseline compared to placebo. It also showed a mean relative improvement of 9.2%. Data demonstrates that PTI-428 amplifies the effect of Orkambi in responding patients (ppFEV1 <70% at baseline).

The dose ranging phase I study evaluating PTI-801 showed a mean improvement in ppFEV1 of approximately 4% from baseline. Moreover, a phase I study evaluating PTI-808 showed that the candidate is well tolerated in patient population.

Proteostasis is also evaluating the combination of PTI-428, PTI-801 and PTI-808 in CF patients in an early stage study. A preliminary analysis showed that the combination is well tolerated in patients and has the potential to be developed for once-daily dosing.

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The preceding article is from one of our external contributors. It does not represent the opinion of Benzinga and has not been edited.

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