Proteostasis Therapeutics Receives Orphan Drug Designation in the EU for PTI-428 for the Treatment of Cystic Fibrosis
ODD in the
"This designation by the EC is further validation of both PTI-428's potential and PTI's mission to offer additional disease modifying treatment options for CF," said
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 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 March, PTI announced results from the Company's 14-day clinical studies of its proprietary combination CFTR modulators in CF subjects. Later this year, the Company is planning to initiate Phase 2, placebo-controlled, 28-day studies in CF subjects with at least one F508del mutation.
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 potential of our proprietary combination therapies for the treatment of CF, the potential benefit to patients of our proprietary combination therapies, expected presentations and expected timing of the initiation of, patient enrollment in, data from, the completion of, our clinical studies and cohorts for our clinical programs, including our planned Phase 2 program, and the potential benefits associated with orphan drug designation. 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 that
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