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Archemix Signs Ribomic to Aptamer Deal; $6M Up Front

Assistant Managing Editor

Archemix Corp.'s aptamer technology landed an expanded collaboration deal with Japanese firm Ribomic Inc., the latest in a string of partnerships for the firm over the past few years and in line with the company's overall business strategy.

"We've had eight publicly announced deals in just over two years," said Duncan Higgons, executive vice president of business operations at the Cambridge, Mass.-based firm, which develops drugs based on aptamers, synthetically derived oligonucleotides.

On its own, Archemix pushes an internal pipeline targeting hematologic disorders, such as thrombotic thrombocytopenic purpura (TTP), and has signed deals with large pharma partners such as Darmstadt, Germany-based Merck Serono and Osaka, Japan-based Takeda Pharmaceutical Co. Ltd. But to capture greater potential with its aptamer platform technology while conserving its own resources, the company has a third strategic approach aimed at out-licensing rights to firms that have the capability to take an aptamer-based product through the development process.

The aim is to "get it into other people's hands so they can get out and run with it," Higgons told BioWorld Today, though he added that Archemix has been choosy about potential licensees. "We're not just licensing indiscriminately. We're doing it very carefully."

It helped that the company had worked with Ribomic before. In late 2006, for example, Archemix granted the Tokyo-based firm a worldwide, nonexclusive license to use an IgG aptamer for affinity purification.

Under the terms of the collaboration expansion, Ribomic agreed to pay $6 million up front in exchange for a nonexclusive license to the aptamer technology for use against multiple undisclosed targets. It also has the option of converting that to an exclusive deal on a target-by-target basis. Upon the exercise of that option, Archemix would be entitled to a further undisclosed fee and could get milestones of more than $200 million if products are developed successfully.

Higgons was not able to disclose the number of targets or the specific breakdown of milestone payments but said the terms were "not out of line with some of our previous deals."

Archemix has struck similar "arms-length deals," as Higgons called them, with other companies. It licensed rights to aptamer technology to San Carlos, Calif.-based Nuvelo Inc., which has progressed a candidate, NI172, in Phase I development as a direct thrombin inhibitor in patients undergoing coronary artery bypass graft surgery. Other clinical programs using the company's aptamer technology include London-based Antisoma plc's AS1411 (nucleolin), which is in Phase II in acute myeloid leukemia and set to start Phase II in renal-cell carcinoma, and REG1 (Factor IXa) from Durham, N.C.-based Regado Biosciences Inc., which is in Phase II in CABG surgery patients and percutaneous coronary intervention.

Each of those agreements also is very focused in terms of targets so they don't overlap with Archemix's internal hematological pipeline or targets being pursued by existing partners. Importantly, those collaborations also help accelerate the development of aptamers across multiple indications. As partners move forward, "the class, as a whole, is gaining validation," Higgons said.

In the meantime, revenue from those deals is "funding a lot of our internal development," he added.

The company's lead program, ARC1779, is finishing up a Phase IIa trial in TTP, an indication caused when the von Willebrand Factor (vWF) protein is not properly regulated, leading to the formation of blood clots. ARC1779 is an aptamer drug designed to work by binding and inhibiting vWF to increase or restore platelet counts to normal levels.

Results from a Phase I trial reported last year showed that the drug demonstrated dose- and concentration-dependent inhibition of plasma vWF activity and platelet function when given intravenously to healthy volunteers.

ARC1779 has been designated an orphan drug for TTP in both the U.S. and Europe.

Archemix also is investigating ARC1779's use in other diseases, with exploratory studies in "a couple of other hematological indications," Higgons said.

Earlier-stage programs include a candidate aimed at treating acute manifestation of sickle cell anemia, which should be ready for an investigational new drug application "sometime in 2009," and a preclinical program in hemophilia that is "progressing well," he said.

Archemix completed its last major financing round in early 2004, raising $50 million in a Series B financing. The company filed to go public last year but withdrew its filing in February citing unfavorable market conditions.

Published: August 12, 2008