Northfield Reaches Agreement With FDA on Special Protocol Assessment For Pivotal Phase III Trial; Establishes Basis for PolyHeme(R) Efficacy Claim.
"This is an exceptional achievement for Northfield. The SPA confirms that the data from our landmark trial will form the primary basis for an efficacy claim in a Biologics License Application for PolyHeme," said Steven A. Gould, M.D., Chairman and Chief Executive Officer. "SPA is not a routine event and this is not a routine trial. SPA is particularly useful for studies which assess novel, potentially life-saving therapies, especially when no approved drug is available to address a critical, unmet clinical need. Since agreement on SPA has only occurred in the Center for Biologics Evaluation and Research (CBER) on a limited number of occasions, this is a true milestone."
SPA represents acknowledgement and confirmation of a mutual agreement between a clinical trial sponsor and FDA that successful completion of the proposed trial will form the primary basis for an efficacy claim as part of product approval. SPA documents agreement on the design, primary efficacy endpoints, study conduct, data analysis, and the kind of labeling statements that could be made if the data are supportive and the product is approved. The agreement becomes part of the administrative record and may only be changed by mutual agreement of the parties, or if FDA identifies a substantial scientific issue relevant to safety or efficacy after the trial has begun.
"During the past year, we have been pursuing a regulatory strategy designed to reduce uncertainty and improve predictability by taking advantage of FDA regulations specifically intended to expedite the approval of novel therapies such as PolyHeme. SPA is the cornerstone of that strategy, and as we move towards licensure, this agreement provides the regulatory clarity that will facilitate our efforts to seek fast track designation for PolyHeme as a product intended to treat life-threatening blood loss when blood is unavailable," added Dr. Gould.
The PolyHeme trial is designed to assess the safety and efficacy of PolyHeme in improving survival when used to treat severely injured and bleeding trauma patients beginning at the scene of injury and continuing during ambulance transport to the hospital. Because the patients eligible for the study are unlikely to be able to provide consent due to the extent and nature of their injuries, the trial will be conducted under federal regulations that allow clinical research in emergency settings using an exception from the requirement for informed consent.
Northfield anticipates that approximately 20 Level I trauma centers throughout the United States will eventually participate in the PolyHeme trial, which has an expected enrollment of 720 patients. The process of public disclosure and community consultation required under the regulations is underway at a number of potential trial sites across the country. Patient enrollment will begin as soon as local IRBs complete their review process.
Northfield Laboratories is a leading developer of an oxygen-carrying blood substitute. Its product, PolyHeme(R), is the only blood substitute that has been rapidly and safely infused in sufficiently massive quantities in clinical trials to be considered useful in the treatment of large volume blood loss in trauma and surgical settings. PolyHeme(R) requires no cross matching, making it immediately available and compatible with all blood types, and has an extended shelf life of over 12 months.
Statements in this release that are not strictly historical are "forward-looking" statements that are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements involve known and unknown risks, which may cause the company's actual results in the future to differ materially from expected results. These risks include, among others: competition from other blood substitute products; the possible refusal of the FDA to approve its request for fast track designation; the company's ability to obtain regulatory approval to market PolyHeme commercially; the availability of capital to finance the company's planned clinical trials and ongoing business operations; the company's and/or its representative's ability to successfully market and sell PolyHeme; the company's ability to manufacture PolyHeme in sufficient quantities; the company's ability to obtain an adequate supply of raw materials; the company's ability to maintain intellectual property protection for its proprietary product and to defend its existing intellectual property rights from challenges by third parties; and the extent to which the hospitals and physicians using PolyHeme are able to obtain third-party reimbursement, as described in the company's filings with the Securities and Exchange Commission.
For further information contact: Steven A. Gould, M.D. Sharon L. Weinstein Chairman and Chief Executive Officer Noonan Russo Presence (847) 864-3500 (212) 845-4271 email@example.com Sophia H. Twaddell Vice President, Corporate Communications (847) 864-3500 firstname.lastname@example.org
CONTACT: Steven A. Gould, M.D., Chairman and Chief Executive Officer, +1-847-864-3500, or Sophia H. Twaddell, Vice President, Corporate Communications, +1-847-864-3500, email@example.com, both of Northfield Laboratories Inc.; or Sharon L. Weinstein of Noonan Russo Presence, +1-212-845-4271, firstname.lastname@example.org, for Northfield Laboratories Inc.