A breakthrough therapy, fast track
product, priority review, or other designation by the FDA for our product candidates may not lead to faster development or regulatory
review or approval process, and it does not increase the likelihood that our product candidates will receive marketing approval.
We have received a
breakthrough therapy and fast track product designation for vonapanitase for improving vascular access and decreasing the need
for surgery in patients with CKD who are on hemodialysis or being prepared for hemodialysis. As applicable, we may seek breakthrough
therapy, fast track, priority review, or other designations for other uses of vonapanitase. Breakthrough therapy and fast track
product designations are designed to facilitate the clinical development and expedite the review of drugs and biologics intended
to treat a serious or life-threatening condition which demonstrate the potential to address an unmet medical need. Priority review
designation is intended to speed the FDA marketing application review timeframe for drugs and biologics that treat a serious condition
and, if approved, would provide a significant improvement in safety or effectiveness. For drugs and biologics that have been designated
as breakthrough therapy or fast track products, interaction and communication between the FDA and the sponsor of the trial can
help to identify the most efficient path for clinical development. Sponsors of drugs and biologics designated as breakthrough therapies
or fast track products may also be able to submit marketing applications on a rolling basis, meaning that the FDA may review portions
of a marketing application before the sponsor submits the complete application to the FDA, as long as the sponsor pays the user
fee upon submission of the first portion of the marketing application. For products that receive a priority review designation,
the FDA’s marketing application review goal is shortened to six months, as opposed to ten months under standard review. This
review goal is based on the date the FDA accepts the marketing application for review (i.e., filing), which typically occurs two
months after the date of submission.
Designation as a breakthrough
therapy, fast track product, priority review product, or under another program is within the discretion of the FDA. Accordingly,
even if we believe one of our product candidates meets the criteria for designation as a breakthrough therapy, fast track product,
priority review product, or other designation, the FDA may disagree and instead determine not to make such designation. In any
event, the receipt of any such designation for a product candidate may not result in a faster development process, review or approval
compared to drugs and biologics considered for approval under conventional FDA procedures and does not assure ultimate marketing
approval by the FDA. In addition, the FDA may later decide that the products no longer meet the conditions for qualification as
a breakthrough therapy, fast track product or under another designation program or decide that the time period for FDA review or
approval will not be shortened.
We may expend our limited resources
to pursue a particular product candidate or indication and fail to capitalize on product candidates or indications that may be
more profitable or for which there is a greater likelihood of success.
Because we have limited
financial and managerial resources, we have focused on developing one product candidate, vonapanitase, and have focused on developing
this product candidate for specific indications that we identify as most likely to succeed, in terms of both its regulatory approval
and commercialization. As such, we are currently primarily focused on the development of vonapanitase for vascular access, and
our Phase 3 trials will be limited to the application of vonapanitase in radiocephalic fistulas.
In the future we intend
to pursue additional indications such as the application of vonapanitase in brachiocephalic fistula creation and/or patients undergoing
placement of an arteriovenous graft and/or patients with symptomatic PAD. As a result, we may forego or delay pursuit of opportunities
with other product candidates or for other indications that may prove to have greater commercial potential. Our resource allocation
decisions may cause us to fail to capitalize on viable commercial products or profitable market opportunities. Our spending on
current and future research and development programs and product candidates for specific indications may not yield any commercially
viable products. If we do not accurately evaluate the commercial potential or target market for a particular product candidate,
we may relinquish valuable rights to that product candidate through collaboration, licensing or other royalty arrangements in cases
in which it would have been more advantageous for us to retain sole development and commercialization rights to such product candidate.
Even if we obtain and maintain approval
for vonapanitase or additional product candidates from the FDA, we may never obtain approval for vonapanitase or additional product
candidates outside of the United States, which would limit our market opportunities and adversely affect our business.
Even if we obtain approval
of a product candidate in the United States from the FDA, such approval does not ensure approval of that product candidate by regulatory
authorities in other countries or jurisdictions, and approval by one foreign regulatory authority does not ensure approval by regulatory
authorities in other foreign countries or by the FDA. Sales of vonapanitase or any additional product candidates outside of the
United States will be subject to foreign regulatory requirements governing clinical trials and marketing approval. Even if the
FDA grants marketing approval for a product candidate, comparable regulatory authorities of foreign countries must also approve
the manufacturing and marketing of the product candidate in those countries. Approval procedures vary among jurisdictions and can
involve requirements and administrative review periods different from, and greater than, those in the United States, including
additional preclinical studies or clinical trials. In many countries outside the United States, a product candidate must be approved
for reimbursement before it can be approved for sale in that country. In some cases, the price that we intend to charge for our
products, if approved for sale, is also subject to approval. Moreover, the failure to obtain approval in one jurisdiction may negatively
impact our ability to obtain approval in another jurisdiction.