Proteostasis’ Proprietary Triple Combination Therapy Study Protocol Receives Endorsement and High Strategic Fit Score from the Largest Cystic Fibrosis Patient Advocacy Groups in the U.S. and Europe
The CFF and ECFS are the most impactful CF patient advocacy organizations and shepherd the execution of clinical trials in the U.S. and
In the U.S., upon the protocol endorsement, the TDN provides access to 89 accredited care centers with demonstrated expertise in clinical research, and supports study participant recruitment and execution of studies. Since its founding in 1998, the TDN has conducted more than 130 clinical studies for CF, including studies of CFTR modulators. Similarly, in
Patrick A. Flume, MD, Professor of Medicine and Pediatrics and Director of the Cystic Fibrosis Center at the
Co-administration of PTI-428, PTI-808 and PTI-801 in CF subjects on track to initiate in 1H 2018 with initial clinical data in 2H 2018
PTI recently completed a healthy volunteer co-administration safety and tolerability study of its three proprietary CFTR modulators. Safety and PK profiles achieved with seven days of once-a-day oral dosing of PTI-428, PTI-801 and PTI-808 indicate these compounds were generally well-tolerated and are potentially amenable for once a day dosing.
PTI-801, a third generation CFTR corrector, and PTI-428, a CFTR amplifier, have shown synergy in vitro with Orkambi® and have been or are being investigated in clinical studies in CF subjects as add-on modulators in patients on background Orkambi® therapy. Preliminary safety and PK data with the first dose of PTI-801 from the ongoing in 14-day dose-range finding study in CF subjects on background Orkambi® supports its further development as part of PTI’s proprietary triple combination therapy. PTI recently announced Phase 2 data for PTI-428 in CF subjects on background Orkambi®, which showed an improvement in lung function in this patient population and helped to identify the initial dose for further combination development.
PTI plans to initiate dosing of CF patients with its proprietary triple combination in the first half of this year in U.S. and European CF centers within the TDN and CTN networks, with initial clinical data expected in the second half of 2018.
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 the completion of, our clinical studies and cohorts for PTI-428, PTI-801, PTI-808 and our dual and triple combination therapy candidates. 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 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
1 Orkambi® is a registered trademark of
Source: Proteostasis Therapeutics, Inc.