Emerging Drug Developer: Fate Therapeutics

Fate takes a hand in cancer therapy

Fate Therapeutics has set out to take the oldest stem cell therapy in use today and make it much better.

Hematopoietic stem cell support is a common theme in cancer therapy. Physicians use it to help reconstitute the blood and immune systems of patients who have undergone harsh chemotherapy treatments. Fate believes it has a small molecule--FT-1050--that can enhance the efficacy of umbilical cord blood transplants in adult patients with leukemia and lymphoma. And researchers recently began a Phase Ib trial to start determining just how safe and effective its small molecule can be in improving hematopoietic stem cell proliferation and homing to the bone marrow, spurring the activity of these blood-forming stem cells.

"People don't think of it the way we do. It's the oldest stem cell therapy out there used in every single patient that gets a bone marrow transplant," says CEO Paul Grayson. "Patients don't start chemotherapy until they are sure they have two adequately matched cord blood units. We have to match to HLA (human leukocyte antigen) type; of 8 types they have to have six matching between the patient and cord blood units.

"The patient comes in, goes through chemotherapy," he adds. Two cords are matched to the patient and for the first six patients in the study the untreated cord is then rinsed and perfused in patients and the second cord gets treated with 1050 for a short period of time. It goes through an additional rinse, and then is transfused into the patient. It fits beautifully into the standard of care. The cord doesn't go off to be treated someplace. There's no process, just a short treatment with a small molecule."

The trial--which is being undertaken at the Dana-Farber Cancer Institute in Boston--is broken into three cohorts, adds Grayson. "We should have data on the first before the end of the year; the next two cohorts come in 2010. Depending on how one and two look, we may go straight for a pure efficacy study. With a remarkable improvement in patient outcome, which is time to engraftment, decrease in hospital stay and underlying disease, there's a potential for us to see efficacy with a smallish number of patients, say 60 to 100."

Fate has a number of ties to top scientists in the stem cell and cancer fields. And Grayson credits three prominent investigators for creating the company's scientific foundation: Harvard Medical School's Leonard Zon, a pioneer in stem cell biology; Randy Moon at the University of Washington, and David Scadden, the co-director of the Harvard Stem Cell Institute.

At this stage, Fate is still very much focused on demonstrating the value of the work. Once that's done, though, Grayson is laying the groundwork for new partnerships to help fund the company.

"At this point we are pushing forward with development at least through Ib on our own," says the CEO. "We're talking to a lot of really smart companies very interested in this application as well as other applications of stem cell modulation."

Fate has brought in $25 million in equity and debt. A Series A captured $15 million, a convertible debt deal brought in another $7.5 million and the developer inked a growth capital deal with Silicon Valley Bank. Even without new revenue coming in from partnering deals or grants, Fate has enough money to operate for a year. And there are a variety of options on the table for raising additional cash.

Fate has 34 staffers on the payroll, including several contract workers. They're now preparing to move into a 19,000-square-foot facility in San Diego that's close to Scripps Research Institute in La Jolla.

Emerging Drug Developer: Fate Therapeutics
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