MediQuest, Crawling Out of the Bunker, Eagerly Awaits Results From Pivotal Raynaud’s Study

MediQuest Pharmaceuticals has barely made a peep since the FDA declined to approve its drug for Raynaud’s disease back in October 2008. But now the Bothell, WA-based company is starting to poke its head up again as it takes another shot at getting its first drug cleared for sale in the U.S.

This week, MediQuest said it has finished screening 284 patients into a clinical trial for Raynaud’s, a condition of limited blood circulation that causes pain and numbness in the fingers and toes in cold temperatures. That means MediQuest should have some fresh clinical trial results on the effectiveness of its gel treatment by August, and, if they are positive, be able to file an updated application to the FDA before the end of the year.

MediQuest was hopeful once before that it would win FDA approval. The drug, MQX-503 (Vascana), is a squeeze-on gel designed to improve blood circulation in the hands and feet. There’s nothing currently approved by the FDA for treating Raynaud’s, and while most of the time it’s considered a minor annoyance that doesn’t require medical treatment, an estimated 2.1 million people in the U.S. are considered candidates for therapy, MediQuest says. Since nobody had ever tried to win regulatory clearance to treat Raynaud’s before, there are no precedents to follow, and the path forward after the rejection took several months to figure out in talks with the FDA. Now, MediQuest says it has the clarity it needs from regulators, and success will hinge on whether its clinical trial is positive.

“We had some logistical hurdles we had to overcome first,” Dechow says.

MediQuest’s original application to the FDA didn’t pass muster because of what Dechow called “intrasubject variation.” Even while the trial reached its main goal of reducing pain and numbness, each patient saw their benefits fluctuate based on variables like how cold the weather was on a certain day. To really confirm that the MediQuest drug was offering a benefit, the FDA proposed a different kind of study. This time, patients would go into a consistent, controlled environment—a cold room, sort of like a meat locker, kept at 40 degrees Fahrenheit.

That might sound great as an experimental design, to see whether the drug relieves pain in a consistent setting, but it wasn’t easy for MediQuest to execute. Each of the 12 clinical trial sites that MediQuest enlisted for this study needed to get approval from the local Institutional Review Board (IRB), which is charged with protecting patient safety. Some objections were raised that such a study would knowingly subject patients to pain, given that some patients would get the drug while others were randomly assigned to a placebo. Plus, since such a study had never been done before, MediQuest needed to make sure a proper “cold room” existed at each of the clinical trial sites, without any noxious chemicals or biological tissues that might introduce new variables into the experiment, Dechow says.

It was late spring of 2009 before the company and the FDA were on the same page about what needed to be done. After a quick pilot study, it was November when MediQuest started screening patients into this new pivotal study.

Screening is important, because researchers need to weed out all the patients with mild symptoms of Raynaud’s, who aren’t likely to be good subjects to show the real difference between drug and placebo. MediQuest needs about 60 good candidates for study, who will then be randomly assigned to the drug or the control group.

The way this works is actually pretty simple. Patient walks into the cold room, applies drug or placebo, and stays in there for 15 minutes. Patient is then asked to rank their pain on a standard scale of 0 to 10. When it’s over, they’re compensated with a trip to a warming room.

The FDA has agreed that if MediQuest can show an average of a 1.8-point improvement in pain scores, that would be medically meaningful, Dechow says. That means if placebo patients report their average pain score is a 6.0, then the drug group would have to average a 4.2 score or better.

It’s not a stretch to say the fate of the company is riding on this trial. MediQuest cut half its staff, trimming its workforce to 15 people, after the FDA rejection in October 2008. MediQuest raised $23 million in this same quarter a year ago to finish its development plan, which represented some new money and a conversion of old debt into equity, Dechow says. But that won’t last forever. The company is continuing to raise money from angels and venture capitalists to make sure it can complete the study, Dechow says.

Most importantly, MediQuest is trying to work the business development front hard, by having talks with five potential partners who are weighing possibilities of buying the drug, or the company, Dechow says. Some talks are designed to strike such a deal before the data is available, after the data comes out, or after FDA grants approval, Dechow says. Obviously, the further along the drug progresses on that timeline, the higher the price would go.

I can’t really imagine anyone would pay much to buy the company before at least seeing the results from this cold room study. No study like this has been done before, so there’s not much of a basis to handicap it. And even if the data is promising, there will be doubts about just how big a market exists to treat a disease that is considered mild 80 percent of the time. But that’s all part of the risk inherent to drug development. Dechow sure seems to relish it, and is looking forward to his precious results.

“We are pioneers,” he says.

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