ASCO Wrap-Up: The Skinny on Cancer News From All Corners of the U.S.

Xconomy National — 

Whoever came up with the phrase “drinking from the fire hose,” could have easily been thinking of the American Society of Clinical Oncology annual meeting.

The information on cancer R&D has been flowing furiously the past few days at ASCO, which draws about 30,000 cancer doctors, drug company executives, investors, and journalists. I didn’t attend the meeting this year in person, but I’ve still had plenty to write about. Here are some quick thumbnail summaries and links that caught my eye from companies in our national network.


Ariad Pharmaceuticals (NASDAQ: ARIA). The Cambridge, MA-based company built up a lot of suspense around its presentation of a pivotal study for its lead drug candidate for sarcoma, a deadly bone cancer. We already knew heading into the conference that this drug, ridaforolimus, hit its primary goal in a study of 711 patients, showing it kept tumors in check for a median of 17.7 weeks, compared with 14.6 weeks for a placebo. Yesterday at ASCO, researchers said an interim look at survival times suggests an advance for the Ariad drug over placebo—a median of 21.4 months vs. 19.2 months—but more patients need to be followed over time before statisticians can say with any confidence that the drug helps people live longer. Ariad’s partner, Merck, repeated that it plans to seek approval in the U.S. and European Union based on the success of the trial.

Infinity Pharmaceuticals (NASDAQ: INFI). Infinity is the Cambridge, MA-based company that has dared to take on pancreatic cancer, a nasty malignancy that has pretty much whipped every drugmaker that has tried to take it on. When taken in combination with chemotherapy, Infinity’s drug, IPI-926, was able to produce meaningful tumor shrinkage in 31 percent of pancreatic cancer patients (5 of 16). There was no control group in this early-phase safety study, so this finding needs to be taken with a grain of salt, but historically less than 10 percent of patients have had that kind of response. Infinity is now in the process of attempting to prove this drug is a winner—through a randomized study of 120 patients that will measure whether IPI-926 in tandem with chemotherapy can help patients live longer than the chemo alone.

Synta Pharmaceuticals. Lexington, MA-based Synta (NASDAQ: SNTA) offered up some preliminary data that showed it has been thinking hard about how to pick patients with the right genetic profiles who are most likely to respond to its experimental drug, ganetespib. This drug, which works against a target called Hsp90, appeared most effective in lung cancer patients whose tumors expressed mutant forms of the genes ALK and KRAS. Patients with those mutations are especially difficult to treat, and the numbers of patients studied in that group is small, so it would be premature to raise any hopes about what this drug can do. Synta said six of the eight patients with ALK mutations (75 percent) had some tumor shrinkage in target lesions.

Alnylam Pharmaceuticals. The Cambridge, MA-based developer of RNA interference drugs (NASDAQ: ALNY) offered an update on an early-stage safety study of its experimental treatment for liver cancer, ALN-VSP. The company said most of the side effects experienced by the first 41 patients were mild in (fatigue, nausea, and fever) although there was one patient who suffered liver failure and died, in a case that Alnylam said “was deemed possibly related to study drug.” About two-thirds (7 of 11 patients) had their tumors stabilize at a dose of 1 milligram per kilogram of body weight—the dose Alnylam has selected as ideal for further development. The company didn’t talk specifically about next steps in clinical trials for ALN-VSP, but CEO John Maraganore did say “Clearly, these data are not only important for the continued advancement of our ALN-VSP program, but they also significantly increase our confidence in our entire pipeline of systemically delivered RNAi therapeutics.”

Constellation Pharmaceuticals. The venture-backed company in Cambridge, MA, developing cancer drugs based on advances in epigenetic science gave everybody a break on crunching hard data on drug response rates and side effects. It made some financing news, by raising $15 million in an extension of its Series B venture round, pulling in cash from Third Rock Ventures, The Column Group, Venrock Associates, SR One and Altitude Life Science Ventures.

San Francisco

Genentech/Plexxikon. Many of the big headlines at this year’s ASCO went to South San Francisco-based Genentech and its partner, Berkeley, CA-based Plexxikon, for their development of a new drug for melanoma patients with a mutation of protein called BRAF. Melanoma is notorious as one of the toughest cancers of all to treat, so enthusiasm has been running high all year long, since Genentech and Plexxikon said a study of 675 patients showed their new drug, vemurafenib, helped prolong lives. At ASCO, researchers said 84 percent of patients taking the new drug were still alive after six months, compared with 64 percent of those getting the standard chemotherapy, dacarbazine.

More data needs to come in to determine how long patients can really expect to live on the new drug, but it was clearly superior, producing meaningful tumor shrinkage for 48.4 percent of patients, compared with 5.5 percent for the standard chemo—about a nine-fold improvement. “This is an unprecedented time of celebration for our patients,” Dr. Lynn Schuchter, a melanoma specialist at the University of Pennsylvania, told reporters Sunday at ASCO, according to a New York Times account.

Exelixis. The South San Francisco-based cancer drug developer (NASDAQ: EXEL) has been generating a lot of buzz among prostate cancer researchers for its lead drug’s ability to reduce the nasty toll that tumor metastases take on the bones. In 108 prostate cancer patients whose malignancy had spread to the bones, the Exelixis drug was able to partially or completely wipe out bone lesions. That translated into reduced pain and less dependence on narcotic pain medications, researchers said. There has been a lot of innovation lately in the prostate cancer field, but none of the new agents has demonstrated had an effect like that on bone pain, one of the devastating complications of prostate cancer as it moves into its late stages.

That story helped Exelixis raise almost $180 million earlier this year for an aggressive development plan, but some new disclosures at ASCO yesterday caused some nerves to rattle in the market. Exelixis said there were two drug-related deaths out of 70 patients with ovarian cancer treated with the company’s lead compound. Out of 490 patients with nine different malignancies who enrolled in studies of the drug, Exelixis reported six deaths. The stock fell 20 percent yesterday to $8.69 largely on the news.

ACT Biotech. San Francisco-based ACT Biotech offered a surprising bit of positive data from a small study of patients with stomach cancer. The tiny 7-person company, led by veterans of Onyx Pharmaceuticals and Proteolix, said that its lead drug produced significant tumor shrinkage in about two-thirds of patients (25 of 39), with mostly mild side effects like fatigue and high blood pressure. Plans are underway to take this into a more rigorous trial of 750 patients to see if it can help patients live longer.

Onyx Pharmaceuticals. South San Francisco-based Onyx (NASDAQ: ONXX) had a bigger data presentation for its lead drug candidate, carfilzomib, last December at the American Society of Hematology meeting. There weren’t any major updates on this drug for multiple myeloma, and investors are waiting for the company to take that one across the finish line for FDA approval. A bigger concern, cited by analyst Chris Raymond of Robert W. Baird, was how Onyx’s existing franchise drug, sorafenib (Nexavar), is holding up competitively after this year’s ASCO. “Post-ASCO, Nexavar’s competitive position—already tenuous in our view—took what we see as a bit of a body blow from Pfizer’s axitinib. Combining this with our skepticism of carfil’s accelerated approval chances, we remain cautious,” Raymond wrote.

Genomic Health. The Redwood City, CA-based company, which makes a genetic test that predicts whether cancer will recur, rolled out results from 10 studies that seek to demonstrate its usefulness in guiding treatment for breast, colorectal, and prostate cancers.


Dendreon. Researchers continued to comb through the data on Dendreon’s sipuleucel-T (Provenge) to better understand what the immune-boosting treatment is doing for prostate cancer patients. In one analysis of a subgroup of patients who entered the Dendreon without pain or the need for addictive painkillers, researchers found that 39 percent of Provenge patients were pain free after 12 months, compared with 19 percent in the control group. The finding wasn’t statistically significant, so there’s a chance it could be a fluke. Still, JP Morgan analyst Cory Kasimov said the chance to offset the pain of prostate cancer could be a selling point. “We see this as added help for Provenge to gain better understanding and traction,” he wrote.

Seattle Genetics. Seattle Genetics (NASDAQ: SGEN) had a good day yesterday as its stock climbed after researchers saw reports on its drug, brentuximab vedotin (Adcetris), for rare lymphomas. “The doctors around the posters yesterday afternoon were uniformly positive to the point of being a little giddy,” David Miller of Biotech Stock Research wrote in a note to clients.

The Seattle Genetics drug, which is being developed in partnership with Cambridge, MA-based Millennium: The Takeda Oncology Company, showed last December at another meeting that it could completely shrink tumors of 34 percent of patients with relapsed forms of Hodgkin’s disease, and when partial responses were included, the number climbed to 75 percent. Now, with more follow up time, the companies reported that the complete responses are long-lasting—a median of 20 months before their disease worsened. One researcher from City of Hope was quoted in a company statement, calling the follow-up reports “clinically meaningful.”

New York

Pfizer. There are two big new drug names from Pfizer (NYSE: PFE) to remember from this year’s ASCO—crizotinib and axitinib.

The crizotinib data has had researchers buzzing for months now, and the data at ASCO kept the hope going. The drug, for about 4 percent of lung cancer patients with a genetic mutation, was found to keep 74 percent of patients alive after a year and 54 percent alive after two years, according to a recap from the Associated Press. Researchers still couldn’t calculate the median survival time, because more than half the 82 patients are still alive. Pfizer has already applied for FDA approval on a faster-than-usual timeline, and the FDA has granted a speedy review, which it sometimes does for potentially lifesaving medicines.

Pfizer’s other drug that got some attention at ASCO was axitinib for patients with kidney cancer. One study found that the Pfizer drug was able to keep tumors from spreading an additional two months, when compared with Onyx Pharmaceuticals and Bayer’s sorafenib (Nexavar). One researcher told HemOnc Today that the trial was important because most studies to date in this group of patients have compared new drugs against placebos—not against existing standards of care like this Pfizer trial.

Merck. The biggest news from the Whitehouse Station, NJ-based drugmaker was from a joint statement in which it detailed the pivotal trial results of its new sarcoma drug, along with partner Ariad Pharmaceuticals. Today, Merck also said it has reached an agreement with Roche under which the Switzerland-based healthcare giant will develop diagnostic tests for Merck’s experimental cancer drugs. Terms of that deal weren’t disclosed.

San Diego

VentiRx Pharmaceuticals. This small company, which has operations in both San Diego and Seattle, presented some data from a small study of 33 patients who got its immune booster, VTX-2237, for a variety of solid tumors. The company found its drug’s side effects were mostly mild to moderate reactions around injection sites, and flu-like symptoms. Based on the findings, the company said it plans to move ahead with four clinical trials of this immune-booster in combination with chemotherapies, antibodies, and radiation treatments.

Ligand Pharmaceuticals. San Diego-based Ligand (NASDAQ: LGND) released some preliminary data from a new chemical formulation of a chemotherapy known as melphalan. Ligand’s reformulation is supposed to allow for longer administration times and slower IV infusion rates, which it says could “enable clinicians to safely achieve a higher dose intensity of pre-transplant chemotherapy.”

Results of a multiple myeloma study found patients were able to get about 10 percent more of the chemo agent into the bloodstream with the reformulated version without adding any new side effects. One researcher quoted in a company release said this could represent a “significant improvement.”

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