Biotech Values

[DewDiligence]

FDA puts Ariad drug for cancer on fast track

[As might be expected from a general-purpose newspaper, this article misses the distinction between Fast Track and Accelerated Approval. Does anyone here follow ARIA ?]

http://www.boston.com

By Jeffrey Krasner
April 20, 2005

Ariad Pharmaceuticals Inc. of Cambridge said the Food and Drug Administration gave fast-track designation to an experimental drug for sarcomas, a group of aggressive cancers for which there are few treatment options.

Sarcomas are cancers that attack bones or soft tissues and affect about 100,000 patients in the United States, with 12,000 cases diagnosed yearly.

Dr. Harvey Berger, Ariad's chief executive, said that with the accelerated timetable made possible by fast-track designation, the firm might seek final approval for the drug as early as next year, even though another round of clinical trials remains to be completed. The designation means the firm will work more closely with regulators, and may be able to design a final trial with an end point that is easier to reach quickly.

''The real benefit comes not in starting our pivotal trial three months earlier, but in decreasing the length of follow-up needed to get approval," Berger said. ''That could save a year or so."

Sarcoma tumors often don't generate any symptoms until they are extremely advanced or until the cancer has spread to other parts of the body. That makes such cancers particularly deadly. Ariad said fewer than 15 percent of patients who are diagnosed when the cancers have spread survive for five years.

''There's a huge need for new treatment approaches," said Dr. Jeffrey Clark, an oncologist who works in drug development at Massachusetts General Hospital and the Dana-Farber/Harvard Cancer Center. ''Especially for adult patients, there haven't been any new treatments in the past decade that have been markedly effective."

Ariad's approach involves the proteins that instruct tumor cells to absorb nutrients, grow, and cultivate a robust blood supply. By blocking the protein, Ariad's experimental treatment interferes with the malignant cells' development. One impact is that cells absorb less nourishing blood sugar and amino acids.

''The cell starves itself, when normally it would gobble up nutrients at high rates," Berger said. Blocking the protein also interferes with the tumor's signals that cause blood vessels to grow, ultimately starving the tumor. ''You take away the food, the blood supply, and the stimulants."

The drug candidate is known only as AP23573. Berger said that compared to traditional chemotherapy drugs, Ariad's experimental treatment has an extraordinarily low incidence of side effects. The primary side effect demonstrated in two sets of human trials is an inflammation of the mouth.

But how effective is AP23573? The company won't say just yet. Interim results from a trial will be released at the annual meeting of the American Society of Clinical Oncology next month.

Ariad, founded in 1991, went public in 1994. In the past two years, it has raised about $80 million through additional sales of its shares. The company has no products yet but is collaborating with Medinol Ltd. of Israel in using AP23573 in drug-coated stents. The firm ended last year with $75 million in cash, and said it plans to spend about $40 million on drug development this year.

Though approval could be years away, Ariad is already engaged in ''pre-market launch" activities, Berger said. It plans to develop a US sales force and retain revenue from domestic sales.
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