Proteostasis Therapeutics Announces Completion of Enrollment in Global Phase 2 Study in Cystic Fibrosis
The trial is designed to assess the efficacy, safety and tolerability of PTI's proprietary combinations over four weeks and with higher doses of proprietary CFTR corrector and potentiator. Dose selection (600 mg of PTI-801 and 300 mg of PTI-808, with or without 10 mg of PTI-428) was based on the totality of dose range finding data from approximately 250 CF subjects studied thus far. Study endpoints include safety, changes in sweat chloride concentration and changes in percent predicted FEV1 (ppFEV1). The study design targeted up to 30 F508del homozygous and up to 30 F508del heterozygous subjects. Due to rapid enrollment from centers in
"We are working hard to introduce the power of choice to the CF community and the completion of enrollment in our global Phase 2 study is an important step forward to completing that mission. We heard loud and clear at our recently hosted Cystic Fibrosis Summit, as well as at the last week's
The 28-day Phase 2 study follows the positive results of the 14-day Phase 1 clinical studies of PTI's proprietary doublet and triplet combinations in F508del homozygous patients, including those predisposed to rapid pulmonary decline based on their bacterial colonization status. The previous studies demonstrated a favorable safety and tolerability profile for the combinations, as well as a statistically significant improvement in ppFEV1 and sweat chloride concentration that was superior to the available dual CFTR modulator standard of care.
About PTI-428, PTI-801, PTI-808
PTI-428 is an investigational CFTR amplifier in development for the treatment of CF in patients with at least one F508del mutation in the CFTR gene, as part of PTI's proprietary triple combination regimen that includes PTI-808, a novel potentiator, and PTI-801, a third-generation CFTR corrector. PTI-801 received Fast Track Designation from the U.S. Food and Drug Administration (
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 ongoing trials of our product candidates, the expected timing for completion and reporting of topline results of our Phase 2 clinical trial and our expectations regarding expanding available therapeutic options for CF patients. 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 potential of our proprietary combination therapies for the treatment of CF, the potential benefit of our proprietary combination therapies to patients, expected completion of our clinical studies and cohorts for our clinical programs, including our planned Phase 2 program and initiation of a pivotal study, 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 by the Company (noting that these results are based 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 that