Proteostasis Therapeutics Announces Progression of PTI-428 and PTI-801 to Longer Duration Studies in CF Subjects
PTI-801 Moving to 14 Day Proof of Concept Study in CF Subjects on Orkambi®
PTI-428 Continues to Enroll 28 Day Proof of Concept Study in CF Subjects on Orkambi®
PTI-808 Investigational New Drug Application Now Active; Phase 1 Study in Healthy Volunteers Initiated
Proteostasis announced that it has completed dosing of 19 patients as part of the ongoing Phase 1/2 study designed to evaluate the safety and pharmacokinetics of PTI-428, the Company’s CFTR amplifier. PTI-428 was administered together with background Orkambi® (lumacaftor/ivacaftor) or as the only CFTR modulator therapy in CF subjects over a 14-day period (7-day dosing followed by 7-day follow-up period). The trial met its primary safety and pharmacokinetic endpoints, confirming PTI-428’s safety, tolerability and lack of clinically meaningful drug-drug interaction with ivacaftor and lumacaftor.
“Preliminary data suggests that PTI-428 continues to demonstrate a favorable safety and pharmacokinetic profile, which has enabled the initiation of Phase 2, enrolling CF subjects on background Orkambi® taking PTI-428 or placebo for 28 days,” said
In the Phase 1 portion of the PTI-428 study, 11 subjects in the Orkambi® cohort and eight in the PTI-428 monotherapy cohort were enrolled, with each group enrolling 2 placebo subjects. All adverse events (AEs) were mild or moderate and none occurred in more than one subject. There were no hematology-related adverse events and no serious adverse events (SAEs) reported. Safety endpoints evaluated included lung function as measured by forced expiratory volume in one second (FEV1), although the study was not designed to show a statistically significant difference. In the subjects who received PTI-428 in addition to their background Orkambi, there was no significant improvement of FEV1 compared to placebo, although there was a numerical increase in FEV1 at day 7. Measurements of sweat chloride and mRNA in nasal mucosa were used as exploratory biomarkers but the changes were not significant nor correlated with lung function changes.
Ms. Chhabra added, “While confirming the safety of PTI-428, the phase 1 portion of this study was not expected to demonstrate efficacy over a 7-day dosing period, as PTI-428, as a CFTR amplifier, is designed to deliver substrate to correctors and was investigated in combination with Orkambi®, whose signal of efficacy required a 28-day study. As a result, we look forward to generating data in our 28-day proof-of-concept study to begin understanding the activity profile of PTI-428.” Proteostasis is enrolling patients in the Phase 2 safety and efficacy portion of the study, which explores PTI-428 dosed over a 28-day period, and preliminary data is expected in Q4 2017.
Proteostasis also announced today that the protocol for the CF portion of its Phase 1/2 study of PTI-801, a new generation CFTR corrector with Fast Track designation from the
The Company also announced today that its Investigational New Drug application with the
To the extent that statements in this release are not historical facts, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as “aim,” “may,” “will,” “expect,” “anticipate,” “estimate,” “intend,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Examples of forward-looking statements made in this release include, without limitation, statements regarding the expected timing of the initiation of, patient enrollment in, data from, and our completion of, our clinical studies and cohorts for PTI-428, PTI-801, PTI-808 and our triple combination therapy candidate, PTI-NC-733. Forward-looking statements made in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved. Such risks and uncertainties include, without limitation, the possibility final or future results from our drug candidate trials (including, without limitation, longer duration studies) do not achieve positive results or are materially and negatively different from or not indicative of the preliminary results reported in this release, (noting that these results are on a small number of patients and small data set), uncertainties inherent in the execution and completion of clinical trials (including, without limitation, the possibility
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