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Ariad wins key patent for leukemia drug

Treatment could reach $1b in sales

Details of a drug called ponatinib, which treats a form of leukemia, was diplayed on a computer screen at Ariad Pharmaceuticals.

Erik Jacobs for the Boston Globe/File 2011

Details of a drug called ponatinib, which treats a form of leukemia, was diplayed on a computer screen at Ariad Pharmaceuticals.

Ariad Pharmaceuticals Inc. is set to disclose today that it has won a key patent on its drug to treat a form of leukemia, giving the Cambridge biotechnology company patent protection through 2026 on what could become a major cancer-fighting therapy.

Shares of Ariad have more than doubled in price over the past year, climbing sharply since the start of 2012, amid projections that ponatinib, its treatment for chronic myeloid leukemia, or CML, eventually could generate annual sales of more than $1 billion.

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Ariad expects to file a new drug application next fall for ponatinib, which was discovered by scientists at the company’s labs over the past decade.

The Food and Drug Administration would then decide early next year whether to approve it for CML patients who have been failed by multiple other treatments and have no other treatment options.

After that, Ariad would seek approval to use the drug for newly diagnosed CML patients.

If successful, ponatinib would transform Ariad from a discovery and research company into a full commercial company and catapult it into the top rank of Boston area biotechs.

Vertex Pharmaceuticals Inc., another Cambridge company, followed a similar path, securing FDA approval for two drugs over the past nine months.

Ariad’s new “composition of matter’’ patent issued by the US Patent and Trademark Office covers the compound behind ponatinib and related compounds.

Ariad has applied for separate patents covering the drug’s method of treatment and formulation.

“This has been at the heart of our plans for many years,’’ chief executive Harvey J. Berger said yesterday.

“We were the first to discover ponatinib and the class of compounds ponatinib is within. Winning this patent protection fits perfectly with our plans to commercialize ponatinib in the United States, in Europe, and ultimately in Japan, and make it the foundation to develop a global commercial company.’’

Unlike many biotechnology companies, which license compounds, Ariad has its own scientists working on drug discovery.

“Our goal is to have a fully integrated cancer company - to discover drugs, develop them, and sell them worldwide,’’ Berger said.

The company takes a structure-based approach to drug design, working to block the mutations through which particular cancers can overcome treatments.

Its first drug candidate, ridaforolimus, treats what is projected to be a modest market: patients suffering from metastatic soft-tissue and bone sarcomas who had a favorable response to chemotherapy.

While it discovered the compound, Ariad struck a deal allowing pharmaceutical giant Merck & Co. to take the lead in marketing it.

Last fall, the FDA accepted the Merck and Ariad new drug application for ridaforolimus.

An advisory panel is set to convene next month to review the results of the drug’s clinical trials, and the agency is scheduled to decide on June 5 whether to approve it.

Merck would handle about 80 percent of the marketing for the sarcoma drug.

In return, Merck gave Ariad upfront payments of $125 million and milestone payments totaling $78.5 million, investment capital that enabled it to develop ponatinib and other drugs in its pipeline. Ariad plans to market those medicines through its own sales force.

In anticipation, Berger said, Ariad has begun expanding its staff. It has hired 40 employees since Jan. 1, boosting its Cambridge workforce to 180, and plans to add another 100 in the next 12 to 18 months.

Ariad is also planning to open a European headquarters in Switzerland later this year.

Robert Weisman can be reached at weisman@globe.com.
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