There’s so much going on in the world of cancer drug R&D that it’s hard to keep up with all the news about drugs that are changing the standards of care. And quite a few of those standards are changing, based on a quick rundown of what was new at this year’s American Society of Clinical Oncology meeting.
The annual conference, winding down now in Chicago, is the biggest gathering for cancer doctors, drugmakers, investors, and journalists. Here are some storylines that I’ve been watching for some time, and that caught my eye from companies around our national network.
Genentech’s breast cancer “smart bomb” takes center stage
Genentech impressed doctors and analysts at this year’s conference with data from its souped-up breast cancer drug called trastuzumab emtansine (T-DM1). This drug beat a couple of active treatments in a head-to-head study of 991 patients with relapsed breast cancer that carries a mutated form of the HER2 protein. The new therapy kept tumors in check for a longer period of time, caused fewer toxic side effects, and showed it is on pace to help extend the lives of these very sick patients. The finding is not only big news for Genentech and for HER2-positive breast cancer patients, but also for scientists, because this performance is helping crack open the field of “smart bomb” antibodies, also known as “antibody-drug conjugates.” These kinds of drugs, which have eluded scientists for decades, are designed to combine the targeting capability of an antibody with a toxin to make the drug more potent.
Jason Kantor, an analyst with RBC Capital Markets in San Francisco, called the T-DM1 clinical results, “a real ‘wow’ moment at ASCO. There was no aspect of the T-DM1 efficacy data that was not positive.”
For more, see the T-DM1 story from Andy Pollack in Sunday’s New York Times and from AP’s veteran medical writer Marilynn Marchione. I also had a story on Xconomy with comments from Genentech VP Dietmar Berger.
The prostate cancer battle royal has begun
Prostate cancer was once a backwater for life science innovation, but not anymore. Johnson & Johnson’s abiraterone (Zytiga) made big news at this year’s ASCO with positive (and somewhat controversial) results from a study of more than 1,000 men known as the ‘302 trial. Essentially, the J&J drug is helping delay the spread of tumors in men whose disease no longer responds to chemical castration, but who haven’t yet turned to chemotherapy. It also helps delay the onset of pain. That’s clearly good news, but because of the positive results, an independent monitoring board recommended the study be halted earlier than planned. While the trends in the study suggest the J&J drug is helping men live longer, the study was halted too early for J&J to state unequivocally that it can reach the gold standard measurement of effectiveness. Bloomberg News’ Michelle Cortez had a solid summary of the news, consultant Pieter Droppert criticized the monitoring board’s decision in a guest post for Xconomy, and Matthew Herper wrote a sharp analysis of the situation yesterday on Forbes. Andy Pollack at the NYT weighed in with a report about how all this prostate cancer progress is coming with some very hefty price tags.
While J&J got more of the headlines, San Francisco-based Medivation (NASDAQ: MDVN) got its share of buzz. This company, along with partner Astellas Pharma, showed off data from a clinical trial that showed their drug enzalutamide (MDV3100) was clearly able to help men live longer, delay the onset of pain, and improve quality of life in men with the last stage of prostate cancer—disease that had worsened after a previous round of chemotherapy. That terrific performance in the sickest of the sick has gotten doctors buzzing about whether Medivation will ultimately become the dominant player in this market once it gathers results in 2013 from another study in the larger pre-chemo patient population.
All this progress is bad news for Seattle-based Dendreon (NASDAQ: DNDN), the company that won FDA approval in 2010 for a pioneering treatment that spurs the immune system to fight prostate cancer. David Miller, president of Biotech Stock Research and a longtime Dendreon bull, said in a note to clients that he’s not yet ready to write Dendreon’s obituary, but he warned subscribers that the competition leaves Dendreon little margin for error (after it has made quite a few errors in the past two years already). “Dendreon is not dead yet,” Miller wrote. “It could be soon if management doesn’t get it precisely right.”
Better understanding of cancer biology is paving the way for better drugs
Reuters’ Deena Beasley had an encouraging macro story about how the number of cancer drug applications to the FDA is rising, with 20 expected this year. Last year, 10 of the 30 new drugs the FDA approved were for cancer, including a couple that were approved in tandem with diagnostics that select patients with genetic profiles that make them most likely to benefit. “There is greater understanding of some of the disease processes,” said Richard Pazdur, the head of the FDA’s cancer drug office, in an interview with the financial wire service.
The rise of immunotherapy continues
Treatments that find a way unleash the body’s immune system to fight tumors are hot. There are lots of different ways of going about this task, and one of the newer ones is through aiming at a molecular target known as PD-1. This target, as Bloomberg’s Robert Langreth put it, acts like an “off-switch” for the immune system, which tumors cleverly keep in place to allow them to thrive. By hitting the PD-1 target, researchers at Bristol-Myers Squibb hope to unleash the power of the immune system to fight cancer cells as if they were any other foreign invader, like a virus. Merck is also among the pharma companies in pursuit of drugs to fight cancer via the PD-1 route, according to this ASCO preview story by my Xconomy colleague Arlene Weintraub.
If you’re really tired of looking at statistical p-values and Hazard Ratios and in need of a little more entertaining summary of ASCO news, check this piece on immunotherapy from Liz Szabo at USA Today. This might be the first-ever reference to Mr. T in a health story.
Mid-cap biotech companies are driving much of the innovation in cancer drug R&D
While the average citizen might only be able to recognize a few major drug companies by name—Pfizer, Merck, GlaxoSmithKline et. al—much of the innovation on display at ASCO was coming from mid-sized biotech companies. Most cancer drugs have an effect that diminishes with time, but Cambridge, MA-based Ariad Pharmaceuticals (NASDAQ: ARIA) showed that its ponatinib for chronic myeloid leukemia gets more effective as patients are followed up longer. Seattle Genetics (NASDAQ: SGEN) won FDA approval last year for the first truly effective “empowered antibody” for a couple rare lymphomas, and it showed more data at ASCO this year that said the drug can shrink tumors for a majority of patients, even in a tough “re-treatment” setting among relapsed patients. South San Francisco-based Onyx Pharmaceuticals (NASDAQ: ONXX) had “kind of so-so” data to report on its new regorafenib for colorectal cancer, in the words of JP Morgan analyst Cory Kasimov. But the data was much more impressive for carfilzomib (Kyprolis) in newly diagnosed multiple myeloma patients, where it was generating significant tumor shrinkage rates for 89-96 percent of patients, Kasimov wrote.
And that’s really just scratching the surface. Sunnyvale, CA-based Pharmacyclics (NASDAQ: PCYC) showed its drug, designed to hit a target called Btk, was able to achieve meaningful tumor shrinkage for 81 percent of patients with chronic lymphocytic leukemia in a small study, while only 12 percent reported moderate to severe side effects. South San Francisco-based Exelixis (NASDAQ: EXEL) showed a raft of data presentations for its drug that’s designed to work against multiple types of cancer, by blocking a cancer master switch and cutting off blood flow to tumors.
Cambridge, MA-based Aveo Oncology (NASDAQ: AVEO) presented results from a pivotal trial of its kidney cancer drug, and it announced another bold bet at ASCO, saying it plans to compare its treatment head-to-head against a rival from Pfizer in another study in which patients will get to decide which treatment they prefer. Aveo is clearly betting that since its drug has shown a milder side effect profile—prompting fewer dose reductions or interruptions—that patients will choose its drug. RBC’s Kantor called it “A Welcome and Aggressive Marketing Move for AVEO.”
For a quick rundown of the winners and losers at ASCO, many of them in the mid-cap biotech world, see the coverage from TheStreet.com columnist Adam Feuerstein.
Cancer Diagnostics On the Move
While most of the headlines go to makers of cancer drugs, drugs, drugs—where most of the money is—there are some quite interesting things happening in the world of cancer diagnostics. Cambridge, MA-based Foundation Medicine, a startup backed by Third Rock Ventures and Kleiner Perkins Caufield & Byers, rolled out its product that looks at an array of 200 genes implicated in cancer, which it combines with published scientific literature into a report which is supposed to help doctors select the right drug for an individual patient’s type of cancer. Redwood City, CA-based Genomic Health (NASDAQ: GHDX) had a number of studies to discuss about its Oncotype DX test, which it uses to predict whether certain forms of cancer are likely to recur, and whether patients are likely to respond to a round of chemotherapy.
These diagnostic companies, like drug companies, are under pressure to show that their products are worth their escalating price tags. That makes meetings like ASCO extra-important to them, as they seek to amass bulletproof data sets to show the value of their products. Those debates still feel like they are in the early days, and they will rise in prominence at future ASCOs.