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ASCO Wrapup: Immunotherapy Shines, Hope For Brain Tumors, & The Great Cancer Drug Price Debate

This article is more than 8 years old.

Hundreds of stories about new developments against cancer came out the past few days at the American Society of Clinical Oncology (ASCO) meeting in Chicago. I didn’t cover the meeting on site this year, but here are a few of the highlights that caught my eye.

Bristol-Myers Squibb Pushes Hard To Maintain Cancer Immunotherapy Edge

Bristol-Myers Squibb , as it has been the past couple years, was at the center of attention with its antibody that unleashes the immune system to fight tumors by binding with a molecular target called PD-1. In one study, patients with the non-squamous form of lung cancer who took its drug, nivolumab (Opdivo), lived a median time of 12.2 months, compared with 9.4 months for chemotherapy, and with fewer severe side effects. The survival time advantage was even wider for patients if their tumors expressed more of the PD-L1 target protein. Andrew Pollack of the New York Times put the news in context, as this is another development in what is becoming a historic movement for immunotherapy against cancer. Opdivo has already won FDA clearance for melanoma, and the squamous cell form of lung cancer, so the new data should help extend the drug’s reach to a broader group of lung cancer patients. Reuters’ Deena Beasley quoted an exuberant Bristol-Myers executive who said, "this marks the end of the chemotherapy era in second-line treatment of lung cancer.”

Investors were less enthused, as some entered the meeting with sky-high expectations of $6 billion in sales or more. Some were spooked by data on the PD-L1 biomarker, which can difficult to measure consistently, and because it raises questions about whether it will narrow the drug’s addressable market. Wall Street, seeking to absorb all the data from a busy meeting, continued to debate what it all meant. The company lost about $3 a share at the meeting, and traded at $65.86 as of 1:10 pm ET. Some analysts thought the reaction was overdone. "We think BMY has strengthened its leadership position in I-O," wrote Credit Suisse analyst Vamil Divan, in a note to clients near the end of the meeting. Divan continued to rate the company an "outperform" with a price target of $75.

Merck’s Immunotherapy Horse Stays in the First Wave

Merck came from behind to win the first FDA approval of a PD-1 inhibitor antibody last year, when it was initially cleared for sale as a melanoma treatment. The company is now seeking the green light to market its pembrolizumab (Keytruda) as as a treatment for lung cancer, and it isn’t stopping there. One Merck study presented at ASCO showed that about one-fourth of patients with head and neck cancer had their tumors shrink after getting the new drug. The data were noteworthy partly because it showed the drug is working against yet another tough-to-treat tumor type, and partly because the patients who responded appeared to enjoy a long-term benefit (see MedPage Today story). All told, Merck reported on studies at ASCO in which showed the drug delivering anti-tumor effects against a remarkable 13 different tumor types, showing the breadth of its program, and reminding everyone why immunotherapy has such exciting potential. Many researchers are working on combo drug regimens that mix and match immunotherapies like Merck’s and Bristol’s, although these studies raise a wide range of business challenges, which I wrote about at Timmerman Report in March (Subscribers Only).

Genentech Follows Fast in the PD-1/PD-L1 Immunotherapy Field

While Bristol-Myers and Merck are at the forefront with their PD-1/PD-L1 inhibiting drugs already on the market for certain uses, Genentech , a unit of Roche that has a storied history in cancer drug development, sees plenty of opportunity for its fast-follower. The company unveiled a new name for its drug candidate, atezolizumab, and showed off an aggressive development plan with 10 Phase III clinical trials to find niches of patients who will respond the best. See the story from FierceBiotech's John Carroll for more. Given the unbelievable heterogeneity of cancer, there are bound to be plenty of niches to explore. Daniel Petrylak of Yale Cancer Center presented data from the Genentech immunotherapy that showed about half of bladder cancer patients with high levels of the PD-L1 biomarker responded to treatment with “atezo” while those with low levels responded, but at a lower rate, just 17% of patients. See the Cancer Research Institute blog for more.

Celldex Reports Progress Against Brain Cancer

Researchers haven’t had much to brag about in recent years for treating a type of primary brain tumor, known as glioblastoma multiforme. Hampton, NJ-based Celldex Therapeutics released further data from its immunotherapy rindopepimut (Rintega), from a clinical trial that randomly assigned 73 patients to get the new drug in addition to the standard treatment-- Genentech’s bevacizumab (Avastin)--or the standard treatment alone. About 28% were alive and with tumors that hadn’t worsened after six months of treatment compared with 16% who did that well on standard therapy. After 18 months of follow-up, 30% of patients on the Celldex drug were alive compared with 15% of those in the control group. The company is discussing whether the data are compelling enough to seek accelerated approval from the FDA, according to this report by TheStreet.com’s Adam Feuerstein.

While effective new treatments are welcome, cancer doctors need to be aware of the perils of overtreatment as well. That’s sometimes true when cancer spreads to the brain. Researchers reported on a study that raised doubts about the use of radiation treatment against brain metastases. See a story by the Wall Street Journal’s Ron Winslow for more.

Cancer Drug Prices Continue to Stoke Controversy

Leonard Saltz, an oncologist at Memorial Sloan-Kettering Cancer Center in New York, stirred the pot at ASCO this year by reminding everyone that “these drugs cost too much.” See Joe Walker’s coverage in the Wall Street Journal, as well as Matthew Herper’s analysis in FORBES on the strange economic forces that have worked over the past decade to drive up cancer drug prices. One insurer, Anthem , said it was trying to curb spending by paying oncologists $350 a month for each patient it keeps on Anthem's recommended treatment regimens. Anna Matthews had the story at the WSJ.

Progress Isn’t All About Drugs. Information Matters, Too

Stories of progress in cancer wouldn’t be complete without mentioning the quest for better information about the molecular nature of tumors, to help form better personalized treatment strategies. Many different companies are working on this concept, including the drugmakers who are seeking to select patients for trials based on whether their tumors carry certain biomarkers like PD-L1. Cambridge, Mass.-based Foundation Medicine, built on DNA sequencing technology that helps doctors find common genetic abnormalities that drive tumors, reported on one study done with MD Anderson Cancer Center that showed hunting for these abnormalities led to promising new treatment ideas against biliary tract cancers. One of Foundation’s challenges as a business has been payers who are unwilling to pay thousands of dollars for its test. If Foundation can continue to gather mounting evidence that such personalized treatment strategies do indeed deliver better results for patients, then it will be in position to be reimbursed well enough to someday become profitable. Roche already bet more than $1 billion to acquire a majority stake in Foundation Medicine earlier this year, so it obviously sees an encouraging trend. See a feature on the company from Bloomberg’s Caroline Chen.

Another company seeking to slash through thorny reimbursement challenges with molecular tests, InVitae, unveiled a new low-price strategy. For just $475, consumers can pay for out of their own pocket to get genetic information that could help guide their treatment for cancer or other diseases. Matthew Herper of Forbes spoke to InVitae founder Randy Scott about the strategy.

Rare Encouragement Against Ovarian Cancer

Ovarian cancer is often detected late in the game, making it especially tough to treat. But Waltham, Mass.-based Immunogen, a company that makes targeted antibodies loaded with toxins to make them more potent tumor-killers, presented some encouraging results. Data were from a small study of just 17 patients with ovarian cancer, but these were severely ill women, and more than half (53%) saw their tumors shrink significantly after getting the ImmunoGen drug. Blurred vision was observed in about half of the patients, however, and one patient dropped out of the study because of it, researchers said. See Adam Feuerstein’s coverage from TheStreet.com.

A Mixed Bag for Treatment of Breast Cancer

Breast cancer surgeons had to take note of a study presented at ASCO which showed that if they sliced out just a little more tissue when removing a tumor, the patients performed better, without noticing any cosmetic difference. AP’s Marilynn Marchione reported on that interesting study.

On the drug front, Seattle-based Oncothyreon offered some encouraging, albeit preliminary, results from a pair of small trials that enrolled heavily pre-treated patients with tumors that overexpress the HER2 protein. The company’s drug appeared to help some patients whose tumors had spread to the brain, which gives them a particularly grim prognosis. That contrasted with Los Angeles-based Puma Biotechnology, which had a rough ASCO after it presented data from a Phase III study that offered what one researcher called an "awfully small" marginal benefit for HER2-positive patients, along with significant diarrhea as a side effect. Once again, TheStreet.com’s Adam Feuerstein had the story.

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