Blood substitute firm Northfield Laboratories Inc. reported a new agreement with the FDA's Center for Biologics Evaluation and Research (CBER) that the company said will have at least two important benefits: It could provide a clearer path to product approval with successful completion of a new Phase III trial, and it should provide a positive story to tell financial institutions as the company goes to knock on their doors.
Meanwhile, another firm in the blood substitute space, Hemosol Inc., of Toronto, reported significantly less positive news, saying its initial analysis of adverse results in the trial of its Hemolink product must be followed by additional in-depth study.
Northfield, of Evanson, Ill., said it reached agreement with CBER on a special protocol assessment (SPA) for its pivotal Phase III prehospital trial of PolyHeme, the company's oxygen-carrying product. This follows approval of the trial granted by the agency in March.
Steven Gould, chairman and chief executive officer of Northfield, said the added importance of the SPA is to create strong contractual expectations with CBER concerning the specific protocol and outcomes of the trial, thus producing a much greater sense of exactness in the potential path to commercialization.
An SPA "does provide clarity and predictability on the regulatory issues going forward," Gould said in summarizing the most important benefit of the SPA.
In comparison, he said the traditional regulatory pathways of a series of trials often are unclear. He said that in particular the blood substitute sector has shown "a fair amount of uncertainty" and that it has been "quite confounding to try to bring some predictability and clarity to our own particular process."
By contrast, Gould characterized the SPA as "an agreement, a written document, part of the administrative record, in essence a contract that can't be changed unless both sides agree [to changes]."
The SPA that Northfield has struck with CBER in particular covers "the primary basis for an efficacy claim" that in turn controls specific marketing claims, which are often unclear in traditional regulatory processes, Gould said.
Because of the nature of the patient population, there can be little exactness in predicting how long the trial will take, but Gould said that "if all goes well," it could be completed in about 12 months.
A projected two- to three-year pathway to clearance presents a problem for the company that has found product clearance an uphill battle.
Northfield's current burn rate will take it out to only six or nine more months, according to Gould, and the new trial "is going to be expensive" - requiring at least $15 million, he predicted.
"We're pursuing a number of options with regard to fund-raising," Gould said, but he noted that the announcement of the new SPA agreement should provide a major boost to those efforts.
"We think this will be particularly valuable, very, very helpful in allowing us to solidify our plans and raise funds in the near future. It should have a large positive impact on the business side."
Meanwhile, embattled blood substitute firm Hemosol reported that it completed an internal review of data from its halted cardiac trial (HLK 213/304) for the use of Hemolink (hemoglobin raffimer) in patients undergoing cardiac bypass grafting surgery and that the review confirmed the conclusions of the company's data and safety monitoring board concerning "an imbalance in the incidence of certain adverse events" in the two groups.
The larger number of adverse events occurred in the Hemolink group and so Hemosol terminated the study early in March to conduct a safety analysis. As a precautionary measure, the company also suspended enrollment in its Phase II study involving the use of Hemolink in patients undergoing orthopedic surgery.
"As expected," Hemosol said in a statement, "limiting enrollment has decreased the planned statistical power of the study with respect to efficacy, and the study was unable to meet its primary objective to demonstrate efficacy in the total patient population."
Lee Hartwell, interim chief executive officer of Hemosol, in a statement said that the company confirmed the DSMB's observation "that there was a statistically significant difference in the data, reflective of myocardial infarctions, between the Hemolink group and the control." But he said that no conclusions could be drawn from this because "additional analyses are necessary."
Early last year, the company received a rejection of its Hemolink submission from Health Canada, with that agency requesting additional data. Since then the company has gone through two series of layoffs and in April it instituted more cost reduction measures to reduce its cash burn to about $1 million per month. It said it would be able to fund operations through the end of 2003.
Besides evaluating Hemolink, the company said it is "developing additional oxygen therapeutics and a hemoglobin-based drug delivery platform to treat diseases such as hepatitis C and cancers of the liver, as well as a cell therapy initially directed to the treatment of cancer through its cell expansion and stem cell research activities."
Northfield's stock (NASDAQ:NFLD) gained $1.16 Thursday, or 15.1 percent, to close at $8.85. Hemosol's stock (NASDAQ:HMSL) fell 34 cents, or 36.2 percent, to close at 60 cents.