Table of Contents
dose of PRT-201, the population and dose in which, in a non-prespecified analysis, we observed an improvement in primary unassisted patency with PRT-201 in our Phase 2 trial.
- Phase 3 endpoints same as our Phase 2
trial. The primary endpoint in our Phase 3 trials, primary unassisted patency, will be the same as that used in our
Phase 2 trial. In April 2013, we held an end of Phase 2 meeting with the FDA during which we confirmed elements of our Phase 3 development plan including the primary endpoint.
- Safety profile supports approval. Based on results from
our clinical trials and preclinical studies, we believe PRT-201, which is administered once and only acts locally, has demonstrated a safety profile that will support approval if our planned
Phase 3 clinical program is successful. At our end of Phase 2 meeting with the FDA, we confirmed that we do not need to conduct any additional preclinical studies to support a BLA
- Unmet medical need. A 2014 publication estimated the total
cost of managing hemodialysis vascular access dysfunction in the United States to be approximately $2.9 billion annually. We are not aware of any approved preventative treatments to reduce the
AVF failure rate. PRT-201 has received fast track designation from the FDA, which is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an
unmet medical need.
- Substantial and readily-addressable market opportunity. We
estimate a sales force of approximately 75-100 representatives will enable us to call on the approximately 1,300 hospitals that account for more than 90% of the AVF surgical placements performed in
the United States annually. We believe PRT-201 will be reimbursed adequately as costs related to AVF surgical placement, which is typically performed in the hospital outpatient setting, are not
included in the ESRD bundle.
- Experienced team. Both our Chief Executive Officer and
Chief Medical Officer were senior executives at GelTex, a biopharmaceutical company, where they played leading roles in the development and commercialization of Renagel, a treatment for hemodialysis
patients that led to Genzyme's acquisition of GelTex for more than $1 billion.
Our strategy is to develop and commercialize PRT-201 for patients suffering from renal and vascular diseases, beginning with patients
undergoing surgical creation of a radiocephalic AVF. Key elements of our strategy include our plans to:
- complete clinical development of PRT-201 and seek regulatory approval in its lead indication in the United States;
PRT-201 directly in the United States;
- undertake clinical development of PRT-201 in Europe and establish partnerships for commercialization of PRT-201 in all or
parts of Europe;
- pursue additional indications for PRT-201;
- establish partnerships for development and commercialization
of PRT-201 in Japan and other Asian countries; and
- in-license or acquire additional product opportunities.
Risks Associated with Our Business
Our business is subject to numerous risks and uncertainties, including those highlighted in the section entitled "Risk Factors"
immediately following this prospectus summary. These risks include, among others, the following:
- we have a limited operating history and have incurred significant losses since our inception and we anticipate that we
will continue to incur losses for the foreseeable future;
- we will require substantial additional financing to achieve our goals, and failure to obtain this necessary capital when
needed on acceptable terms, or at all, could force us to delay, limit, reduce or terminate our product development, any commercialization efforts or other operations;