Assistant Managing Editor

Don't mistake Prolong Pharmaceuticals LLC's lead program Sanguinate for a blood substitute.

That term is a misnomer anyway, said Glenn Kazo, president of the South Plainfield, N.J.-based firm, since creating a true blood requires the ability to deliver oxygen, plus achieving the correct pH and salinity and "whole host of other functions."

Sanguinate is "really a different class of molecule," designed specifically to actively transport oxygen to treat conditions such as anemia and peripheral ischemia due to diabetes, he said. Though it "starts from a similar basis, it's a different focus."

Still, trying to distance the firm's approach from the failure-riddled blood substitute space hasn't been easy, especially when it came to raising money. A lot of venture capitalists carry a bias toward those products, so "we did not go to the traditional VC community," Kazo told BioWorld Today.

Instead, Prolong shared its promising preclinical data, including recently published results in Artificial Cells, Blood Substitutes and Biotechnology showing Sanguinate's efficacy in an ischemic stroke model, with a private investor group. The firm is expected to announce Wednesday that it has secured $30 million in funding from the undisclosed investor, and that money should fund the firm over the next two or three years as it moves into clinical trials with Sanguinate.

Prolong – originally formed around pegylation technology developed by scientific founder Abraham Abuchowski (who previously founded pegylation leader Enzon Pharmaceuticals Inc.) – has been around since 2002. It operated as a virtual research company with a half-dozen employees up until a couple of months ago, when it "came together with all the elements to build a company," said Kazo, who had consulted with the firm until his appointment as president last month.

Abuchowski starting working on oxygen therapeutics in the early 1990s and had created the firm's lead molecule, Sanguinate, technically described as bovine pegylated carboxy hemoglobin.

His previous pegylation work was an obvious fit. Hemoglobin is toxic outside of cells, so a pegylation process was needed to make it nontoxic. But pegylation itself still doesn't work to promote active oxygen transport, and that's where Prolong's other modifications came into play.

It was "tweaked from just a pegylation hemoglobin model," Kazo said, to actually facilitate the transfer of oxygen into surrounding tissues.

In sickle cell disease, an inherited blood disorder characterized by misshapen cells that fail to deliver adequate oxygen to the body, Prolong will be going after the acute setting of sickle cell crisis. Patients in crisis experience full body pain that Kazo likened to "having a muscle cramp or charley horse in every muscle because cells are starved for oxygen."

That pain often sends patients to the hospital, where they are treated with opioid-based painkillers, the standard of care.

Prolong's planned clinical studies, anticipated to start in early 2011, will be designed to test Sanguinate against standard of care to see whether patients experience a reduction in pain and, hopefully, also a reduction in the need for pain meds, Kazo said.

If all goes as planned, the company expects to take Sanguinate to market on its own in the orphan sickle cell space. Prolong already has built up its own manufacturing capability and, with its recent cash infusion, plans to grow its 16-member staff.

While it outsources some activities, "we're not interested in building a company with a proof-of-concept-and-then-sell-it [plan]," Kazo said.

"We're building it as if we're going to be a standalone, integrated company," he noted.

Prolong likely will consider partnerships if Sanguinate moves into broader indications, including peripheral ischemia caused by diabetes.

It also has in its pipeline a long-acting third-generation biologic treatment for anemia, though "that's not getting a lot of focus right now," Kazo said.

Between now and the end of this year, Prolong aims to build out its management team, expand its facilities and finish up remaining clinical work to line Sanguinate up to start clinical testing next year.