Index Ventures taps Big Pharma-backed fund to hatch anticoagulant upstart

With an experimental therapy that could save "millions of lives," biotech startup XO1 Ltd. said it has raised $11 million in a Series A round of financing from Index Ventures. Index Ventures has formed the startup and invested in the young company from a $200 million fund closed in 2012 with capital from GlaxoSmithKline ($GSK) and Johnson & Johnson ($JNJ) to fuel new asset-focused drug companies.    

XO1 fits the bill. The company was created for the sole purpose of advancing an anticoagulant antibody from the University of Cambridge, and one full-time chief executive is expected to oversee the program with research contractors and consultants, said David Grainger, a venture partner at Index serving as interim CEO of XO1, in an interview with FierceBiotech.

Cambridge, U.K.-based XO1 has an extremely promising drug candidate unlike any other anticoagulant on the market, the company's founders say. The synthetic antibody, called ichorcumab, could combat blood clots without causing life-threatening bleeding, which has plagued warfarin and newer anticoagulants. Based on a natural antibody discovered in a woman treated for a head injury at Addenbrooke's Hospital in 2008, ichorcumab is designed to bind to the exosite-1 region of the enzyme thrombin to provent clots.

"It is absolutely unheard of that any anticoagulant that has been tested on a preclinical basis or has eventually gotten to market didn't cause bleeding--they all do," said Professor Jim Huntington, a coagulation expert from the Cambridge Institute for Medical Research, in a telephone interview.

Huntington collaborated with Dr. Trevor Baglin, who treated the head-injury patient at Addenbrooke's, to discover ichorcumab, and he's working with Index partners on the startup to develop the candidate. Index has made one of its largest first-round investments ever in XO1, according to the VC firm.

Ichorcumab has never been tested in humans, even though researchers modeled the antibody after the one found in Baglin's patient, who made a full recovery from her head injury within days of arriving at the hospital. The antibody could save millions of lives, but it could also turn out to be a dud. That is the risk of all promising drug candidates, most of which never make it to market.

"If the profile that we currently have is capable of being delivered safely in people on scale it could render it virtually impossible to have a heart attack or a stroke," XO1 interim CEO Grainger says. "And in such circumstances, you can begin to imagine the kind of dollar-market sizes that it could achieve."

Imagine billions of dollars, potentially, especially if the drug could be used as a long-term treatment for heart disease, which is the top killer of Americans. In patients with acute coronary syndrome, which includes heart attack, anticoagulents such as warfarin are used under close clinical supervision because of the bleeding risks. If an anticoagulant can be given to patients without causing deadly bleeding, it could be used on a regular basis to prevent the blockages that trigger heart attacks and stroke.

"One of the interesting things about all the new oral anticoagulants that are in development or already on the market," Huntington says, "is that they are not being approved for [acute coronary syndrome] and part of the reason is they cause bleeding."

Johnson & Johnson, which markets the oral anticoagulant Xarelto in the U.S., and GlaxoSmithKline backed Index's life sciences fund to spark novel drug research, especially in Europe. GSK and J&J get to elect one representative each to serve with Index's partners on a committee that meets quarterly with the portfolio companies. Yet XO1 and other companies financed with the fund keep full rights to their drug candidates.

Heart disease drugs are notoriously expensive to bring to market because advanced trials typically need to include thousands of patients and span years. If ichorcumab prevails in early human studies, Grainger says, the program would require more resources than any biotech startup alone can muster.

Yet if the drug proves to be as good as billed, it's easy to imagine a bidding war among pharma companies to acquire the antibody. It's got a long way to go, however.

- here's the press release
- read Grainger's post about the deal