Emerging Drug Developer: Argos Therapeutics

Argos Therapeutics journeys into Phase II trials

Argos Therapeutics’ approach to developing cutting-edge therapies for cancer and infectious diseases is really quite simple to understand: Load a patient’s dendritic cells with tumor DNA and then unleash them back into the patient, killing the disease by triggering a lethal immune system response directed right at the specific antigens causing the disease.It was figuring out a way to make the process commercially viable that proved a tough obstacle. But company CEO John Bonfiglio says he’s confident that the company has accomplished the task.

A tissue sample can be sent directly to Argos’ facility in North Carolina, which can extract and amplify enough RNA from that sample to provide enough of the therapeutic to last for three years. Argos has a patented method to mature monocytes--part of the body’s immune system that bears down on infected tissue--into dendritic cells. The RNA is placed in the dendritic cells, which are allowed to fully mature, and the therapeutic is placed in a vial, frozen and shipped overnight back to the hospital where the patient is being treated.

“It’s an automated process, which reduces our costs quote a bit,” says Bonfiglio, who’s been able to rely on an ambitious partnership with Kirin Pharma to advance their work on the process. Bonfiglio has already had a chance to review the process with the FDA, and “they’re fine with it.”

Argos, though, still has quite a ways to go before Bonfiglio finds out if the FDA is also fine with these new therapies. Bonfiglio doesn’t expect an FDA approval before 2013.

Argos’ lead therapy--AGS-003--is in Phase IIa for metastatic renal cell carcinoma. “We’re looking for partial and complete remissions in these trials as a stand-alone agent in stage 4 renal cell cancer.” Another mid-stage trial is being prepared to test its therapy in combination with a new standard of care in the field. A mid-stage trial for an HIV therapy--an immune response was tracked in the early-stage trial--is scheduled to get underway shortly. And Bonfiglio is clearly excited about a preclinical program for CD83, a prime candidate for a partnership deal, which has demonstrated potential for preventing transplant rejection.

“We’ll have data in primates by the end of the year or early next year,” he says. “This is one of the hidden gems of the company.”

Bonfiglio can already point to proof-of-concept data from an earlier Phase II trial in renal cell carcinoma based on an earlier version of the process. Improvements in the process, he says, have allowed researchers to increase the potency of the dendritic cells.

The biotech company has been financing much of its recent work with new deals. Following a Series A round at close to $40 million in 2001, when the company was called Merix Bioscience, Argos netted $33 million in a deal with Geron to give it access to its IP on dendritic cells and RNA. Last year it inked a $21 million contract with the NIH to develop new HIV vaccines. A deal with Novo Nordisk provided an up front fee with a schedule of milestones. A smaller deal is being finalized now. And Kirin is matching Argos’ research spending dollar for dollar on the dendritic cells program, a pact worth about $30 million so far. “The company’s been very successful in leveraging non-dilutive cash to keep going,” says Bonfiglio. “We’re raising a Series C now,” he adds, targeting a range of $30 million to $40 million.

Bonfiglio says he has enough money to get the company through ’09, taking it to the point where it will have the Phase II data on hand to determine the company’s valuation at that stage. At that point, he says, just about anything is possible. An IPO? New partnerships? A buyout? More venture funds?

Says Bonfiglio: “Anything’s possible for the right price.”

And at the right time.

“This is truly a platform technology,” says the CEO, who got his PhD in organic chemistry from UC San Diego and had earlier been CEO of Immune Response Corp. And with Dendreon looking for an approval in the field of immune stimulation--which has been no easy task in the face of regulators’ skepticism--dendritic cell technology could soon be in investors’ spotlight.

“This whole area could explode,” he says enthusiastically. And not in a bad way.