Note: This post will be updated regularly as more data from the conference is released. Make sure to also catch our writeup of Sunday's developments here.
CHICAGO, IL—BioPharma Dive is in the Windy City attending the 2015 American Society of Clinical Oncology (ASCO) conference, where the biggest players in the cancer game gather to present data on their most promising and closely-watched oncology candidates.
The conference took off with a bang on Friday, with major news—some good, and some not-so-great—from major pharma companies such as Bristol-Myers Squibb, AbbVie, Pfizer, Johnson & Johnson, and others. As expected, new classes of checkpoint inhibitors and immuno-oncology candidates have been dominating the conversation.
We'll have daily posts that will be regularly updated throughout the course of the conference to cut through the clutter and keep you up-to-date on what you need to know.
Here are the major takeaways from Friday and Saturday:
1) Bristol-Myers Squibb's "good news/bad news" day for Opdivo
Investors and industry observers got some telling news from data presented by Bristol-Myers Squibb on its blockbuster cancer immunotherapy Opdivo (nivolumab). As you might recall, BMS won a record-fast FDA approval for Opdivo in advanced squamous non-small cell lung cancer (NSCLC) back in March—the first therapy in the new PD-1 checkpoint inhibitor class to win a lung cancer indication (several others, including Merck's competing treatment Keytruda, have already been granted melanoma approvals).
The NSCLC indication was particularly significant for BMS since lung cancer is the most common cancer in the world, affecting about 17% of all male cancer patients and 13% of all overall cancer patients.
But the big test for BMS was always going to be the therapy's effectiveness in non-squamous NSCLC, the most common form of lung cancer. And while Opdivo did indeed show efficacy in treating that condition, the data the company presented at ASCO came with some major caveats that disappointed some observers and led to a huge drop in the pharma giant's stock price.
For some lung cancer patients, a more limited benefit
In a phase III trial of non-squamous NSCLC, treatment with Opdivo improved patients' overall survival by 27% compared with chemotherapy with docetaxel. But one of the most striking elements of the data was that patients who were PD-L1-positive had a far stronger response to the therapy, and those with the highest levels of the protein showed the strongest advances in overall survival and diminished death risk.
What that means is that there is a distinct possibility that, once Opdivo does gain FDA approval for a non-squamous NSCLC indication, it might be recommended for patients who are PD-L1 positive, thereby significantly diminishing the drug's market penetration potential for this cancer. As some have pointed out, that could also mean that patients taking the drug may be recommended to have a biopsy to determine their PD-L1 status—an invasive procedure that, while common, still does come with the risks of surgery.
BMS' stock dropped about 7% on Friday on the news.
...but early promise in liver cancer
Now, for the good news: An early trial of Opdivo showed promise in one of the hardest-to-treat cancers that also faces a dearth of available treatments: advanced liver cancer.
As of now, there is only one approved targeted therapy for the disease: Amgen's Nexavar (marketed by Bayer outside the U.S.). But in a (small) phase I/II trial, Opdivo elicited an objective response in 19% of advanced liver cancer patients and led to tumor shrinkage of more than 30%—a truly impressive result. Many of these patients had tried other therapies that had failed, making the Opdivo liver cancer trials important ones to follow.
2) Merck: King Keytruda?
A competing cancer immunotherapy from Merck, Keytruda (pembrolizumab, the first-ever approved PD-1 inhibitor in the U.S.), had significant and promising data across several cancers. And the U.S. pharma giant also announced a significant partnership for its immuno-oncology superstar.
An Amgen alliance
In a potentially major move, Merck announced on Friday that it would be teaming up with Amgen to test Keytruda in combination with the biotech's investigational compound talimogene laherparepvec in a phase I, open-label study of patients with head and neck cancers.
Overall, these types of cancers make up about 3% of all cancers in the U.S. and cause more than 13,000 deaths per year. The Amgen investigational compound is intented to initiate an immune response against cancer cells.
A potential winner in colon, and other, cancers
But perhaps the more significant news came from new colon cancer data released by Merck that shows promise for use of Keytruda in patients with a biomarker other than PD-1 and its ligands.
The biomarker that was identified in this trial is called a "mismatch repair deficiency," or MMR deficiency, and may be present in a host of cancers. Merck announced that patients with non-colon gastrointestinal cancers and colon cancer who had this genetic marker showed a 60% and 62% response rate, respectively, to treatment with Keytruda. That means that the presence of this genetic marker could be a major sign that a patient will respond to immunotherapies such as Keytruda, although it will take more trials before anyone can be certain of that.
3) Johnson & Johnson could have a multiple myeloma game-changer on its hands
Some of the most significant announcements of the Saturday sessions came from Johnson & Johnson, which presented early data suggesting that its investigational daratumumab therapy could be a real game-changer for patients with the blood cancer multiple myeloma. Even more impressive was the fact that the patients in the trial had already attempted as many as five prior treatments, including with drugs like Celgene's blood cancer blockbuster Revlimid, Amgen's Kyprolis, and J&J's own Velcade, to no avail.
Enter daratumumab, which elicited responses in 29% of patients in a phase II trial and increased patients' progression free survival, on average, by four months. A small number of study participants even underwent complete remission, and adverse events were only slight-to-severe. In fact, no patients discontinued therapy.
J&J is expected to file the therapy for FDA approval before the end of the year.
4) A paradigm-shifting cancer treatment payment proposal from ASCO
In some non-clinical trial news, ASCO itself made a major proposal on Saturday. The group dubbed its proposal the "Patient-Centered Oncology Payment" (PCOP) system, and ASCO president Dr. Peter Yu promoted it to a packed house during his presidential address to the conference.
You can read the highlights of the proposal here, but in short, it's a bundled payment system that would move away from the traditional fee-for-service systems that an increasing number of critics across healthcare have been pushing back against in recent years.
For instance, the PCOP system would propose new bundled billing codes for services such as cancer patient prep, case management, and clinical trial participation in exchange for achieving metrics such as avoiding the need for emergency admittance and—significantly—avoiding the use of higher-cost drugs if cheaper alternatives have proven to be just as effective.
We'll have more on this proposal later.
5) Other notable developments from CTI, Pfizer, AbbVie, and J&J
Keeping up with all of the studies and data from ASCO can be overwhelming. Here are some other ones to note:
- CTI Biopharma presented data finding that pacritinib is particularly effective for myelofibrosis patients with especially low platelet counts.
- Pfizer's Ibrance (palbocicilib) significantly extended progression free survival in women with one of the most common forms of breast cancer (HR+, HER2- metastatic breast cancer) whose disease progressed despite endocrine therapy.
- J&J and AbbVie showed even more data finding that the rock star therapy Imbruvica extended PFS in chronic lymphocytic leukemia (CLL) patients who had already undergone therapy. The interim analysis of the phase III trial found that patients treated with a combo of Imbruvica, Teva's Treanda, and Roche's Rituxan had an 80% reduction in progression or death risk compared to those treated with placebo and the two latter drugs. The trial was so successful that it was halted early and placebo patients were allowed to switch over to the Imbruvica-included combo therapy.