Proteostasis Therapeutics, Inc. (NASDAQ: PTI) leaped Thursday following positive preliminary results from its proprietary doublet in cystic fibrosis patients. PTI-801 and PTI-808 achieved statistical significance on all endpoints.
These studies were designed to assess the safety, tolerability, and pharmacokinetics (PK) of PTI-801, a third-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector, PTI-808, a novel CFTR potentiator, and PTI-428, a novel CFTR amplifier, compared to placebo over a 14-day dosing period in CF subjects across dose levels of each modulator.
Changes in sweat chloride (SC) concentration and in percent predicted FEV1 (ppFEV1) were assessed and evaluated as endpoints.
At the time of data cut, 21 subjects were randomized and treated in the first three dose cohorts of the doublet combination of PTI-801 (100 mg, 200 mg, 300 mg) and PTI-808 (50 mg, 100 mg, 300 mg) or received placebo. PK results were as expected and confirmed a once-a-day dosing profile for both PTI-801 and PTI-808. In terms of safety, the compounds were generally well tolerated. No pulmonary exacerbations were reported in the dosing or follow up period of the study.
In a note, RBC's Brian Abrahams says any dip in Vertex Pharmaceuticals (NASDAQ:VRTX) following Proteostasis’ positive early-stage data on its cystic fibrosis (CF) candidates will be a buying opportunity. He believes that PTI could be an "incremental negative" for VRTX but could emerge as a more serious threat to its CF franchise if the early-stage data are confirmed or if a larger company steps in to partner or acquire PTI.
Abrahams upgraded PTI to outperform with a $9.00 (376% upside) price target.
Shares in PTI exploded $5.78, or 306%, to $7.67.
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