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targeted drug to be approved for treating sarcomas, which are malignancies of the bones and soft tissues.The Ariad/Merck drug has the potential to be a new treatment option for patients who have ended treatment with chemotherapy and who would take it rather than waiting to see whether their tumors stop progressing without their taking anti-cancer drugs.
Ariad doesn’t yet have the definitive proof it needs to win FDA approval, but it has been encouraged by what it has seen so far. Clackson recalled fondly his memory of meeting the first patient to be treated with the drug, a Texas woman whose sarcoma had been in remission for eight years, he said. The drug is also in development for breast tumors and other types of cancer.
Taking the long view, Ariad decided this spring to give up some of its ownership in ridaforolimus to Merck (amending its July 2007 partnership deal) in exchange for a $50 million upfront payment from Merck. Merck also paid Ariad $19 million to reimburse the firm for certain R&D costs related to the drug, took over all expenses of its development for sarcoma and other tumor types, and agreed to give Ariad double-digit royalties on potential sales of the drug and as much as $514 million in milestone payments. Importantly, the deal gave Ariad cash and resources to focus on the No. 2 drug in its pipeline, ponatinib, which has the potential to treat both blood cancers and solid tumors.
Last month, Ariad began a Phase II clinical trial to test ponatinib as a treatment for certain patients with chronic myeloid leukemia (CML) as well as those with lymphoblastic leukemia that overexpresses what’s known as the Philadelphia chromosome. The drug is another targeted cancer therapy and one for patients with a mutation makes their cancer immune to imatinib (Gleevec) and other treatments. The company hopes to seek approval of the drug based on the results of the current Phase II. Companies ordinarily need to complete three phases of study to win FDA approval, but in this case the agency has said a well-controlled mid-stage study might be sufficient if it shows compelling results for this rare condition.
There are 320 patients who are expected to enroll in the global single-arm, Phase II study of ponatinib. For patients with chronic disease, the study’s primary endpoint will be to measure the drug’s impact on cytogenetic response rates identified in patients’ tissue samples. And for patients with accelerated or blast forms of disease, the study will measure how much signs of the cancer are reduced in the patients’ blood samples as a primary endpoint, according to Ariad. The trial will also test whether patients have the specific gene mutation that makes their cancer resistant to existing drugs.
“We knew that we had something quite special with ponatinib, in terms of its expedited path to approval, so we wanted to be able to focus on it as much as we could,” Clackson said.
Ponatinib is following a similar development path as the current treatments for CML patients whose cancer is resistant to Gleevec, including Bristol-Myers Squibb’s dasatinib (Sprycel) and Novartis’s nilotinib (Tasigna). Ariad was able to show in the Phase I the safety of its drug and the effective dosage of it. If approved, ponatinib would be used for CML patients who have not responded to dasatinib or nilotinib. There are estimated 4,9000 new cases of CML in the U.S. per year, and most of them respond to Gleevec or the second-line therapies, so this would only be a serious option for a small number of people. Still, Ariad estimates that its initial annual revenue for the drug in this population would be about $450 million per year.
In a September 13 note to investors, the New York-based investment firm Oppenheimer was bullish about the prospects of Ariad’s latest ponatinib trial. “We see a high probability of positive (ponatinib) data in 2012, leading to a late-2012 [or] early-2013 approval,” the firm’s analysts wrote. The firm rated Ariad’s stock at “outperform.”
So it’s not out of the question that Ariad could go from having none to two products on the market in less than three years. If that becomes the case, Ariad can join Millennium in Cambridge’s small and distinguished club of biotech companies with revenue from product sales.