Proteostasis Therapeutics Announces FDA Grants Orphan Drug Designation for PTI-428 in Cystic Fibrosis
“This is the second important regulatory designation PTI-428 has been granted from the
PTI-428 is an investigational CFTR amplifier in development for the treatment of CF in patients who are homozygous for the F508del mutation in the CFTR gene as an add-on therapy to approved CFTR modulators or as part of PTI’s proprietary triple combination regimen that includes PTI-808, a potentiator, and PTI-801, a corrector. PTI-428 has been shown to work early during CFTR biogenesis to increase levels of newly synthesized CFTR protein, suggesting potential therapeutic benefits in combination with CFTR correctors and potentiators. In addition to Orphan Drug Designation, PTI-428 has been granted Breakthrough Therapy Designation as well as Fast Track Designation from the
In December, Proteostasis announced the results from a Phase 2, randomized, placebo controlled study of PTI-428, in 24 CF subjects on background Orkambi therapy which showed that treatment with PTI-428 led to mean absolute improvement in percent predicted forced expiratory volume in 1 second (ppFEV1) of 5.2 percentage points from baseline through Day 28 compared to placebo (p<0.05).
Proteostasis plans to initiate a triple combination CF Study of PTI-428 with PTI-801, its third generation corrector, and PTI-808, its potentiator, in the second quarter of 2018, with preliminary results anticipated in the second half of 2018. The Company is also planning an additional study of PTI-428 in CF Subjects on Symdeko™, with initial data anticipated in early 2019.
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 benefits of Orphan Drug designation and 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 double 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 FDA requires us to run cohorts sequentially or conduct additional cohorts or pre-clinical or clinical studies), in the enrollment of CF patients in our clinical trials, in the timing of availability of trial data, in the results of the clinical trials, in possible adverse events from our trials, in the actions of regulatory agencies, in endorsement, if any, by therapeutic development arms of CF patient advocacy groups, and in the possibility that we do not maintain Orphan Drug status and therefore lose the benefits thereof, and those set forth in our Quarterly Report on Form 10-Q for fiscal quarter ended
Orkambi® and Symdeko™ are trademarks of
Source: Proteostasis Therapeutics, Inc.