The very preliminary success of a clinical trial usually isn't big news. But don't tell that to Northfield Laboratories Inc.
The Evanston, Ill.-based company reported the recommendation by an independent monitoring group to move ahead, without modification, with its pivotal Phase III study of PolyHeme - a human hemoglobin-based oxygen carrier.
And that go-ahead "is a big deal," said Steven Gould, Northfield's chairman and CEO. The OK to move forward comes after the group's initial review of blinded data on mortality and serious adverse events in the study.
"It's a big deal, if you've been following this," Gould told BioWorld Today, an oblique reference to the ongoing regulatory vicissitudes that have been suffered by Northfield and other companies in the blood-substitute sector over the past months and years. In particular, the FDA turned down the company's application for PolyHeme in 2001, citing concerns about the design of an earlier trial that used historical models to compare effectiveness. And, like other developmental-stage firms, the company has struggled with financial shortfalls.
The PolyHeme Ambulance Study is designed to evaluate the safety and efficacy of PolyHeme, when administered to patients in hemorrhagic shock following severe injury. Northfield called it "the first study in the United States in which treatment with a blood substitute begins at the scene of injury." Patients are currently being enrolled in the study at 11 Level I trauma centers throughout the U.S, with the primary endpoint being survival at 30 days.
Gould said that other than raising no safety issues, the monitoring committee provided no specifics to Northfield concerning the initial data, and the company will remain shielded until enrollment completion, which includes a 30-day patient follow-up.
He reported that ramp-up of the study sites for the trial has been gradual but is accelerating as more facilities come on line - eventually to reach about 20.
The process has been slowed because the trial, he said, is "way more complex than a standard drug trial. It's very complex in terms of the logistics, regulatory, legal, ethical and clinical challenges, and we've been very diligent in addressing each of those challenges. Our major focus is to provide maximum assurance of patient safety."
Offering no promises concerning the timeline for an FDA application on PolyHeme, Gould said the trial will take several months to complete, with more months for data analysis. The product would be submitted via a biologics license application to the FDA's Center for Biologics and ER.
After financial problems earlier in the decade, Northfield over the past year has completed three registered direct offerings, Gould said, raising "just under" $54 million. And as of May 31, it had $42 million in its coffers.
"A year ago, we had only four months of operating capital left," he said. "Financially, we're in better shape than in a long time. We're good for 12 to 18 months or longer at this point.
"Not that we can sit back and savor," he said. "But we feel good that we've gotten a special protocol assessment from the FDA, which is key in this field of blood substitutes. That really provides some clarity going forward and [gives us] the ability to raise funds. We're getting this trial up and running, which is very important, and added key executives that have pharma expertise. And our investor relations program is much more proactive and successful than in the past."
Northfield's stock (NASDAQ:NFLD) gained 13 cents Wednesday to close at $12.31.