Emerging Drug Developer: Archemix

Archemix finds eager partners for aptamer tech

It's been four years since Archemix last raised a round of venture capital. And it's been more than a year since the biotech backed away from an attempt to go public. But there's no shortage of cash in its coffers.

Archemix has racked up $80 million from development pacts--the kind of undilutive cash growing biotechs thrive on--and hundreds of millions more in promised milestones. The developer counts seven candidates in the clinic, but only one remains in Archemix's hands. The others have been partnered out to a string of pharma companies. Just a week ago Archemix inked an R&D deal with Eli Lilly and more collaboration deals are in the works.

Cambridge, MA-based Archemix, which counts a staff in the low 90s, has a simple strategy: Rely on the company's platform aptamer technology--synthetically-derived oligonucleotides, or short nucleic acid sequences, that bind to protein targets--to provide new therapeutics to advance for rare hematological disorders while inking partnerships with companies like Lilly and Merck KGaA, which are better equipped to take on the bigger studies for broader indications.

Inside the company, attention is focused on ARC1779, which is finishing a Phase IIa trial and is expected to begin a Phase IIb before the end of the year. The therapy is being advanced as a first-in-class anti-platelet agent in patients suffering from a blood disorder known as thrombotic thrombocytopenic purpura, or TTP.

"It's a devastating disease where there is no drug out there," says CEO Errol DeSouza, PhD. Roughly one in five victims die from the rare disease. "You're dealing with just a few thousand patients worldwide. Phase IIa was a pilot trial to work out infusion protocols and get indications and mechanisms that were relevant, and we have that.

"One of the patients is being written up," says the CEO about the trial, which recruited about 20 patients for the study. "The patient came in with a coma and nothing helped. Our drug allows platelet count to be normalized, the patient came out of a coma and is back home."

The Phase IIb study--which should recruit about 100 volunteers--will have three dose cohorts. And while DeSouza expects it to last a couple of years, there will be interim data along the way to inspect.

"We will continue to have a dialogue with the regulators," says DeSouza. "Depending on the outcome and considering the high unmet medical need, this trial could serve as a registration trial. We may need a Phase III, but we just don't know right now."

In the meantime, Archemix--a 2007 Fierce 15 company--is also focused on preclinical work for sickle cell disease and has several other preclinical programs underway in hemophilia, "a core area" for the developer.

"This is an area that really plays to the strength of aptamers," explains DeSouza. When the company focuses on rare hematological disorders, researchers can get an early read on the mechanism of action, even in Phase I. And the company is advancing critically needed therapeutics where current standards of treatment either don't exist or are woefully inadequate.

"That's the sweet spot," says DeSouza, "where we can truly look at taking it all the way to the goal line and where we're looking at tens to hundreds of patients, rather than the massive numbers" needed for most large clinical trials.

Archemix was willing to take its case to the public market in 2007, but even then DeSouza and the owners decided the time wasn't right. These days, the idea of taking a biotech public elicits a quick laugh.

Perhaps at some stage the company can take that route, says DeSouza. But at this stage, Archemix isn't ruling anything out, including a buyout at some point.

"We can't speculate," says the CEO. "We will always be opportunistic in terms of how we build shareholder value. I can tell you that the ball is rolling very rapidly in terms of pharma's interest in aptamers as the next generation of therapeutics," he adds, comparing the field to antibodies a few years back.

Emerging Drug Developer: Archemix

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