Biotechs have long dominated the stage at the American Society of Clinical Oncology (ASCO), unveiling their latest innovations like magicians pulling rabbits out of hats. But last week's annual ASCO bash in Chicago showed that Big Pharma wants in on the $34 billion-a-year market--and already has a few tricks up its sleeve to show for it.

Bayer and Onyx Pharmaceutical's liver-cancer-fighting Nexavar (sorafenib), for instance, looks to be the biggest threat to biotech stalwarts like Genentech. The Nexavar presentation sent Onyx's stock soaring to a two-year high and Bayer's to an all-time high.

Nexavar is already approved for advanced kidney cancer, but promising new data suggest that it could be an option for people with late-stage liver cancer. Patients on Nexavar lived an average of 2.8 months longer, buoying survival time from eight months to 11. About 19,000 Americans a year are diagnosed with liver cancer, and more than half a million worldwide suffer from it, providing an ample global market. ''The results for Nexavar were fantastic,'' said Fleur Pijpers, senior oncology analyst at Datamonitor. ''It's going to be the new standard reference therapy for liver cancer.''

In stark contrast to Nexavar's smashing success, biotech Telik's Phase-III ovarian cancer drug Telcyta (Canfosfamide) crashed and burned, with FDA calling for the company to shutter the trial ASAP. ''Everyone expected their data to be bad, but this was unbelievably bad,'' said Morningstar biotechnology analyst Karen Andersen. ''This was harmful to everyone involved, and brought up a lot of safety issues.''

Pfizer's Sutent (sunitinib) got major ASCO play this year, with the pharma dynamo's signature cancer drug--approved for advanced forms of kidney and gastrointestinal cancers--appearing in 33 abstracts, up from 17 last year.

Pfizer also made headlines with its signal inhibitor axitinib, raising hopes in a slew of tests for advanced pancreatic cancer patients, thyroid cancer, metastatic breast cancer, and advanced renal cell carcinoma. Andersen noted that after anti-angiogenics, the class of inhibitors is the fastest-growing group of oncology drugs. Pfizer also announced that 20 percent of its '07 budget will be dedicated to cancer drug development, up from 12 percent last year. That adds up to a total of $1.9 billion, and Andersen predicted that that percentage will only grow in the coming years.

Sanofi-Aventis jumped on the blood-vessel bandwagon with its anti-angiogenic aflibercept, currently in late-stage breast cancer and lung cancer studies.

With the cancer market set to top $55 billion in 2009, Pijper noted that there's no ceiling for companies to hit--at least not until a safe, side-effect-free cure makes its ASCO debut. But she warned that the rush to oncology is likely to result in some overcrowded niches. ''If you have 12 drugs for liver cancer, one of them is going to be the best, and there'll be no use for the others,'' she said.

But Andersen noted that there is hope for finding new uses for "old" treatments. ''Even drugs that have already been approved can be used in combination studies,'' she said. ''Many of the data at ASCO were from combination studies with drugs like Avastin''--Genetech's all-points blockbuster.

These combo studies will play a major role in the next few years. ''Anytime you have a drug that's that effective, you're going to have a lot of piggy-backing,'' Andersen said.

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News Analysis

The take-home from the American Society of Clinical Oncology.

What's Hot and What's Not in Oncology

Post-marketing studies--at the forefront of congressional debate around the Prescription Drug User Fee Act (PDUFA)--may not yield eye-opening results for most sponsors, a new study has found.

A recent analysis from the Tufts Center for the Study of Drug Development (CSDD) suggests that these much-ballyhooed studies are not only expensive and hard to complete on time but, for all that, not very informative. Yet no news is often good news in the post-marketing stage--and that may offer encouragement for drug companies to start fulfilling their commitment to complete these studies.

Only 32 percent of respondents said post-marketing clinical studies ''significantly or very significantly'' added to their knowledge of a product's safety and quality, while the majority of both clinical and non-clinical study sponsors (68 percent and 79 percent, respectively) said post-marketing studies ''contributed either marginally or not at all'' to their understanding of the product.

CSDD Associate Director Christopher-Paul Milne said the results are neither unexpected nor as bad as they sound. ''You could look at this two ways,'' said Milne, who co-conducted the study. ''One way is that 68 percent of these sponsors aren't finding any surprises in their product--good ones or bad ones.'' The other way, he says, is that 32 percent of the sponsors are finding previously unknown side effects or new uses for their drugs.

And that's an added value of post-marketing surveillance--to explore possible new avenues for development. In non-clinical studies, including animal testing and chemical manufacturing, Milne said that new findings are uncommon because such testing has already been done in early R&D. Clinical studies with human subjects are where the significant data emerge, which explains why there are about twice as many clinical than non-clinical studies conducted.

But the real problem lies in the difficulties post-marketing studies present, rather than any potential lack of relevant results. According to the CSDD study, 45 percent of these studies were delayed, and 56 percent of the delayed studies needed ''at least a year beyond their expected completion date'' to finish. And sometimes, Milne said, the FDA asks too much: Genentech's Avastin (bevacizumab) had to go through 20 post-marketing studies to win approval for use in colorectal cancer patients. At $5.3 million a pop for clinical studies and $610,000 per non-clinical study, that's a steep price for one new application for a single drug.

But Milne noted that the way to minimize these extra costs and time crunches is to start a scientific dialogue with FDA during the approval process. ''This isn't just something that will go away,'' he said. ''Both sides have work to do.''

And for foot-dragging companies that ignore the carrot, PDUFA is set to give FDA a stick to prod them into compliance. Whether or not the agency will choose to use it remains to be seen.

But no news is good news for a drug?s safety profile, a new study says

Most Post-Marketing Studies Deliver ''Duh'' Data

Amgen and Johnson & Johnson continue to defend erythropoiesis stimulating agents (ESAs) as the Centers for Medicare and Medicaid Services (CMS) move to limit reimbursement of these lightening-rod agents.

Scientific debate has centered on whether the use of red-blood-cell boosters Aranesp (darbepoetin alfa) and Procrit (epoetin alfa) increases the risk of death in cancer patients. But CMS' move--and reverberations among commercial payers--may do more bottom-line harm than any black-box warning.

In its comments to CMS, Amgen urged the public payer not to overstep its bounds--implying that it might be acting out of turn in setting reimbursement restrictions before scientific conclusions have been reached.

"CMS should acknowledge the role of the FDA in its judicious evaluation of the safety profile of ESAs," officers wrote, "and avoid using coverage policy to play the role of the FDA by issuing prescribing instructions."

Both Amgen and J&J note that the problems have turned up only when investigators studied the drugs for experimental uses. Amgen also cautioned CMS to avoid "coverage parameters that have never been studied in clinical trials or utilized in clinical practice." It also refuted 10 of 18 CMS coverage recommendations.

The topic of ESAs was heavily anticipated at J&J's analyst meeting last week, too. In their joint presentation, group chair Kris Peterson and group president of R&D Jay Siegel defended the therapies as "safe and effective."

They presented several studies that showed no effect on either survival or tumor progression in patients with cancer who take the medications for chemo-related anemia--though they did acknowledge that the risk of blood clots is "well-known and reflected in the label." But they blamed the mortality risks on the medical conditions linked to these blood clots, like obesity and higher baseline levels of hemoglobin.

Amgen has been hardest hit by the ESA controversy, which has dogged the biotech all season and even overshadowed its R&D news at last week's big American Society of Clinical Oncology confab. The company has also gone on a bit of a shopping spree, snatching up San Franciso-based Ilypsa and Cambridge, MA-based Alantos, both with strength in nephrology.

Biotech takes issue with move to limit reimbursement.

Pharmaceutical Executive-06-13-2007

What's Hot and What's Not in Oncology

Most Post-Marketing Studies Deliver ''Duh'' Data

Amgen Lashes Out at Feds over Anemia Drug Coverage