Ischemix Faces Moment of Truth with Mid-Stage Heart Drug Trial

3/16/10

Ischemix is facing the biggest hurdle in its 11-year history. The Maynard, MA-based biotech firm is launching the first human study to test the effectiveness of its drug that’s supposed to protect heart tissue from injuries that often occur when blood flow is restored in cardiac surgeries.

The drug, called CMX-2043, is the only contender the company has that’s ready for clinical trials, says CEO Duffy DuFresne. Without backup clinical candidates, the success of the firm’s Phase IIa trial in treating ischemic-reperfusion injury is hugely important to the future of the company.

Success would be a major leap forward for both Ischemix and the treatment of ischemic-reperfusion injuries, for which there are no FDA-approved therapies despite many previous attempts. The closely held company—funded primarily by its chairman, and its medical director—has enough money to pay for the mid-stage clinical and not much else beyond that, according to DuFresne.

“The good news is that we’ve raised enough money to carry us to the end of this trial,” DuFresne says, “the bad news is that we’re not funded beyond that and additional funding is going to have to be determined by how encouraging our results are.”

The clinical trial is expected enroll 200 to 220 patients, many of whom are undergoing surgeries to unblock their arteries and implant stents to restore blood flow, according to DuFresne. The firm’s drug, which was previously tested for safety in healthy people, will need to prove that it’s both safe and effective for patients to succeed in the trial. (To date, the effectiveness of the drug has only been shown in rodents.) The CEO says the results should be in by the end of 2010.

Heart surgeries can actually result in more tissue damage. This is because of the body’s natural response when blood flow is restored to oxygen-starved cells after a blockage is removed. Restoring blood flow all at once unleashes reactive oxygen molecules that can damage heart tissue. Such ischemic-reperfusion injuries are a major cause of heart damage and deaths after surgeries, DuFresne says. (Yet exact statistics on such injuries are hard to come by.)

The biggest need for Ischemix’s drug is in coronary artery bypass surgeries, of which there are about half a million done in the U.S. every year. However, the health status of patients who receive these bypass procedures can vary considerably, DuFresne says, so the company’s latest clinical trial will include patients undergoing catheter-based cardiac surgeries such as angioplasties, whose conditions are more uniform. If the clinical trial shows that the drug is effective, the plan is to later test the treatment in patients undergoing the bypass surgeries.

While there are no approved treatments for ischemic-reperfusion injuries, DuFresne says, heart surgeons typically use blood-thinners during procedures to prevent clotting in arteries. But those treatments don’t address reperfusion injuries. On the other hand, Ischemix’s lead drug is intended to protect against the hazards of the excess oxygen and the buildups of calcium in cells that damage tissues. (David Stipp took a deep dive into the science at Ischemix in an Xconomy story about two years ago.)

Ischemix (pronounced iss-KEY-mix), founded in 1999, is competing with industry giants that are working on the same problem. New York-based drug-maker Bristol-Myers Squibb (NYSE:BMY) is funding KAI Pharmaceuticals’s mid-stage clinical trial of a drug for ischemic-reperfusion injuries in patients undergoing certain heart surgeries. Also, Merck & Co. (NYSE:MRK) has brought on a late-stage program in this field through its blockbuster merger with Schering-Plough last year.

It’s no mistake to find these industry giants pursing this problem: the multi-billion dollar market for heart drugs is one of the largest in the pharmaceutical industry. The two people who stand to gain or lose the most money on Ischemix are company chairman and president Reinier Beeuwkes and Geoffrey Clark, the firm’s medical director. Both men have provided most of the some $20 million the firm has raised, says DuFresne, who took over the role of CEO from Beeuwkes in early 2009. He declined to name the firm’s other investors, who he says are wealthy individuals.

Beeuwkes and Clark have reaped the benefits of the successes of a previous company they founded, Braintree Laboratories, a Braintree, MA-based firm that specializes in bowel-cleansing treatments taken prior to colonoscopies, according to DuFresne. By late this year, we may know whether these entrepreneurs will have an encore for the heart drug market.

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