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- the data collected from clinical trials of PRT-201 or any additional product candidate may not be sufficient to the
satisfaction of the FDA or comparable foreign regulatory authorities to support the submission of a BLA, or other comparable submission in foreign jurisdictions or to obtain regulatory approval in the
United States or elsewhere;
- our manufacturing processes or facilities may not be adequate to support approval of our product candidates; or
- regulatory agencies may change their approval policies or adopt new regulations in a manner rendering our clinical data
insufficient for approval.
is possible that neither PRT-201 nor any product candidates we may seek to develop in the future will ever obtain the appropriate regulatory approvals necessary for us or any future
collaborators to commence product sales. Any delay in obtaining, or failure to obtain, required approvals would materially adversely affect our ability to generate revenue from the particular product
candidate, which likely would result in significant harm to our financial position and adversely impact our stock price.
We may face difficulty in enrolling patients for clinical trials.
We may find it difficult to enroll patients in our clinical trials, which could delay or prevent clinical trials of PRT-201 or any
additional product candidates. We have never previously limited a trial to patients undergoing a surgical procedure to create a radiocephalic AVF, as we will do for our Phase 3 trials.
Identifying and qualifying patients to participate in clinical trials of
PRT-201 or any additional product candidates are critical to our success. The timing of our clinical trials depends on the speed at which we can recruit patients to participate in testing product
candidates. The enrollment timeline for radiocephalic AVF patients is lengthy and there are limited numbers of sites from which we can enroll pre-hemodialysis or hemodialysis patients. If patients are
unwilling to participate in our trials because of negative publicity from adverse events or for other reasons, including competitive clinical trials for similar patient populations, the timeline for
recruiting patients, conducting trials and obtaining regulatory approval of potential products may be delayed or prevented. These delays could result in increased costs, delays in advancing our
product development, delays in testing the effectiveness of our technology or termination of the clinical trials altogether. We may not be able to identify, recruit and enroll a sufficient number of
patients, or those with required or desired characteristics to achieve diversity in a trial, to complete our clinical trials in a timely manner. Patient enrollment is affected by factors
- severity of the disease under investigation;
- design of the trial protocol;
- size of the patient population;
- eligibility criteria for the trial in question;
- perceived risks and benefits of the product candidate under study;
- proximity and availability of clinical trial sites for
- availability of competing therapies and clinical trials;
- efforts to facilitate timely enrollment in
- patient referral practices of physicians; and
- ability to monitor patients adequately during and after
may not be able to initiate or continue clinical trials if we cannot enroll a sufficient number of eligible patients to participate in the clinical trials required by regulatory
agencies. If we have difficulty enrolling a sufficient number of patients to conduct our clinical trials as planned, we may need to delay, limit or terminate ongoing or planned clinical trials, any of
which would have an adverse effect on our business.