Cancer drug developers angling for center stage at ASCO

Posted under Ariad Pharmaceuticals, ArQule, Array BioPharma, ASCO, axitinib, Blog, breast cancer, cabozantinib, Cancer, Cancer Drugs, Companies, Diagnostics, Eli Lilly, Exelixis, Funding, Medical Devices, Medical Supply, Pfizer, Pharmaceuticals, Pipeline, renal cell carcinoma, Roche, Startups, Universities, Videos by john

Later this week the cancer drug research crowd will examine a tsunami of abstracts filed ahead of the ASCO meeting in early June in the hopes of finding a few new gems in the mountain of data being readied for the confab. ASCO is the place where anyone who's doing anything in cancer tries to make a splash in the spotlight. And with close to a thousand cancer drugs in the pipeline, that's no easy task.

Pfizer ($PFE) and Eli Lilly ($LLY) were both out bright and early this morning touting their work in the cancer arena. Pfizer's best hope at drawing attention lies in updating the data on axitinib, its recently approved treatment for renal cell carcinoma. And Lilly clearly hopes to impress analysts with ramucirumab, which is in 6 late-stage cancer studies.

Digging deeper, Executive Editor Ryan McBride's recent take on the top 10 cancer drugs in late-stage studies includes some of the closest watched therapies likely to elbow their way to center stage. Aveo ($AVEO) will be working hard to distinguish tivozanib--its kidney cancer drug with so-so head-to-head data with Nexavar--as it takes a monumentally important run at an approval later this year. Anything new on Roche's ($RHHBY) T-DM1 for breast cancer, which has racked up positive late-stage data on breast cancer on its way to an odds-on approval at the FDA, will be studied closely. Onyx ($ONXX) has the promising carfilzomib to tout, which is already at the FDA for review, while regorafenib (majority-owned by Bayer) is also in the media mix.

Today in Xconomy, the savvy biotech scribe Luke Timmerman assesses 8 drugs likely to make it into the center ring at ASCO. In addition to the drugs already mentioned, Timmerman includes abiraterone (Zytiga) from J&J ($JNJ) and Exelixis's ($EXEL) cabozantinib, a promising cancer drug that has earned Exelixis CEO Mike Morrissey both acclaim and exceptionally harsh criticism. The development program for cabo has been raked over the coals by a number of analysts. Medivation's enzalutamide (MDV3100), meanwhile, could wind up stealing the thunder for prostate cancer. And there's follow-up data from Ariad ($ARIA) on ponatinib.

Back in mid-March Adam Feuerstein at TheStreet got the first crack at an ASCO preview, listing prospects from Celldex, ArQule, Array BioPharma and Oncothyreon among the more frequently mentioned drug developers. But Celldex's ($CLDX) CDX-011 presentation was scrapped due to a clerical error. Now Celldex is planning a preemptive strike, with plans to hold a press conference of its own on May 23.

If you can't join them at ASCO, you can always try to beat them to the punch.

- here's the story from Xconomy
- read Adam Feuerstein's piece in TheStreet

Special Report: Top 10 Late-Stage Cancer Drugs - 2012

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FDA panel shoots down Merck/Ariad cancer drug as GSK marches on

Posted under Ariad Pharmaceuticals, Blog, Companies, Diagnostics, FDA, Funding, GlaxoSmithKline, Medical Devices, Medical Supply, Merck, Pharmaceuticals, Startups, Universities, Videos, Votrient by Ryan McBride

Merck ($MRK) and Ariad Pharmaceuticals ($ARIA) saw their hopes for approval of their targeted cancer drug ridaforolimus dim after an FDA panel voted not to recommend approval of the therapy for sarcomas. U.S. regulators have the final say on the companies' bid for approval and will likely decide its fate before a June 5 deadline.

Thirteen experts on the panel voted "no" to the question of approval, with only one "yes" vote. Many analysts expected the panel to deny Merck and Ariad, especially after an FDA staff briefing questioned whether the experimental treatment's marginal benefits justified the health hazards that could hit patients while taking it. FDA staff noted that the drug kept patients' cancer from advancing for 3.1 weeks longer than a placebo, Bloomberg reported.

If Whitehouse Station, NJ-based Merck fails to sail to an FDA approval with ridaforolimus, the blow won't sink the pharma giant. Cambridge, MA-based Ariad, which has no products on the market, and its investors have banked heavily on the success of the second-most advanced drug in its pipeline, ponatinib, which has yielded promising data as a targeted treatment for chronic myeloid leukemia. And Ariad has already reaped nearly $200 million in payments from Merck related to ridaforolimus, Xconomy reported. Those funds have helped back development of ponatinib and other cancer programs in its pipeline.

Merck announced last night that it would keep pursuing approval for ridaforolimus, a small molecule mTOR inhibitor, for soft-tissue or bone sarcomas after the tough FDA panel vote.

"Merck remains confident in the potential of the investigational agent ridaforolimus for an indication where patients have limited options," Dr. Eric Rubin, vice president of Merck's clinical oncology unit, said. "We remain committed to bringing forward this promising therapy for patients with metastatic sarcoma and look forward to further discussions with the FDA regarding this application."

In related the news, the same FDA panel that shot down ridaforolimus backed approval of GlaxoSmithKline's ($GSK) Votrient (previously approved for kidney cancer) for treating sarcomas in an 11-2 vote, Bloomberg reported. The FDA will take the panel's vote into account in making its decision about approval of the drug for combating sarcomas, with a decision expected by a deadline of May 6.

- here's Merck's release
- see Bloomberg's article
- and Xconomy's report

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FDA questions benefit of Merck and Ariad’s bone cancer drug

Posted under Ariad Pharmaceuticals, Blog, Companies, Diagnostics, FDA, Funding, GSK, Medical Devices, Medical Supply, Merck, Pharmaceuticals, ridaforolimus, Startups, Universities, Videos by Ryan McBride

Merck ($MRK) and Ariad Pharmaceuticals ($ARIA) face a big test for their experimental cancer drug ridaforolimus next week. In briefing docs released today, FDA staffers questioned whether the drug should be approved given the slim survival benefits that it provides. That will be a central question for FDA advisers to consider at an advisory committee meeting slated for March 20.

Merck and Ariad are seeking FDA approval of their drug as a maintenance therapy for patients with a rare form of cancer called soft tissue and bone sarcoma. In a late-stage trial, the drug helped patients live without their bone cancer getting worse for a median of 16.1 weeks compared with 14 weeks in the placebo arm, the FDA said, and patients on the drug had median overall survival of 20.8 weeks, slightly more than the 19.6 weeks in the placebo group. FDA staffers said those gains might fall short of providing enough of a benefit, Bloomberg reported. 

GlaxoSmithKline ($GSK) will also face next week's FDA advisory panel with its application to gain approval for Votrient, which is already marketed for treating kidney cancer, to treat bone cancer. Bloomberg noted that the FDA staff review of the drug in bone cancer indicated that it doesn't increase overall survival.

No new red flags about the safety of Merck's or GSK's drugs were raised in the FDA staff documents, Reuters said.

Ariad has no drugs on the market and could garner a $25 million payment from Merck if the FDA approves ridaforolimus--an mTOR targeting compound--for bone cancer. If the drug doesn't fair well with regulators, Ariad also has in late-stage development a targeted drug known as ponatinib for treatment of chronic myeloid leukemia.

The FDA often follows the advice of its advisory committees in its approval decisions.

- see Bloomberg's report
- check out the Reuters article
- get the FDA briefing docs here

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Ariad’s ponatinib takes center stage with promising pivotal leukemia data

Posted under Ariad Pharmaceuticals, Blog, Companies, Diagnostics, Funding, leukemia, Medical Devices, Medical Supply, Pharmaceuticals, ponatinib, Startups, Universities, Videos by John Carroll

Ariad Pharmaceuticals ($ARIA) took center stage at the big American Society of Hematology (ASH) meeting over the weekend with interim pivotal data demonstrating that close to half of all chronic myeloid leukemia patients who had failed standard treatment had a "major" response to its experimental drug ponatinib. In what was widely heralded as a promising step toward a potential approval, 39% of the patients who responded to the drug experienced a complete remission. A "major" response was recorded when at least two thirds of their bone marrow returned to normal. The data build on Ariad's first look at the promising interim data back in November.

Ariad has big plans for ponatinib. The biotech plans to hold on to U.S. rights, planning an FDA filing by the middle of next year. And Reuters notes that the company plans to decide in the next few months whether it will line up a European partner. Ariad also plans to begin a study next year in a group of patients at an earlier stage of the disease.

There were some serious side effects noted in the study. Six percent of the patients developed pancreatitis and four of the subjects died. Investigators concluded that the deaths of the four patients with advanced leukemia and co-morbidities could be related to the drug. But the deaths weren't unexpected. "The side effect profile is every bit as good as other drugs in this class," Ariad Chief Executive Officer Harvey Berger told Reuters.

Analysts were clearly impressed with the positive data. Leerink Swann's Howard Liang called it a possible "highlight of the conference."

"This is a very hard disease to treat as patients become resistant to earlier lines of therapy," Berger told Bloomberg. "Even in these very advanced patients, we're seeing what most physicians would say is a very important and clinically beneficial response."

- read the Reuters story
- here's the Bloomberg piece

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