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Cancer cells dividing. Source: National Institutes of Health.

The global pharmaceutical market is officially a trillion-dollar business, and no indication appears to have a faster-growing research effort than cancer. Based on estimates from the World Health Organization in 2012, cancer incidence rates are expected to rise by 57% in the coming two decades to 22 million cases diagnosed per year. Thus, focusing research and development dollars on cancer drug development could wind up netting substantial profits for the companies that succeed and making a difference in the lives of millions.

One avenue of cancer research that's garnering most of Wall Street's attention is cancer immunotherapy. Cancer immunotherapies are medicines designed to enhance the ability of a patient's immune system to more effectively fight cancer. The typical mechanism involves either reducing or eliminating the immunosuppressant quality that allows cancer cells to go undetected, supercharging the immune system to destroy cancer cells, or some combination of both.

This past weekend, the kingpin of cancer care research, Roche (NASDAQOTH:RHHBY), announced positive results from three phase 2 studies over two indications for its experimental cancer immunotherapy product atezolizumab, results that I believe are going to seriously turn some heads in the coming days and weeks.

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Source: National Cancer Institute. 

Roche is about to turn some heads
Despite being arguably the dominant force in cancer care with a vast product portfolio and more than six dozen ongoing clinical-stage trials just for oncology, Roche is having to play catch-up to immunotherapies known as checkpoint inhibitors from Bristol-Myers Squibb (with Opdivo) and Merck (with Keytruda). Both Opdivo and Keytruda are approved as treatments for BRAF V600 mutation-positive advanced melanoma, and both are looking to expand to a number of additional indications.

On Sunday, Roche may have closed the gap between it and its competitors in a big way.

In its first midstage study, known as IMvigor 210, which was presented at the 2015 European Cancer Congress, checkpoint inhibitor atezolizumab, an anti-PDL1 therapy, was tested as a treatment for advanced bladder cancer. Advanced bladder cancer (specifically metastatic urothelial carcinoma, or mUC) is particularly difficult to treat, but atezolizumab demonstrated a 27% objective response rate in patients with mUC who had moderate to high levels of PD-L1 expression. Furthermore, a median duration of response had not been reached when Roche released its results, but the company did note that 92% of those with an objective response were still responding to the therapy at the time of its press release.

Although mUC isn't an indication that gets a lot of press, these initial results are impressive because advanced bladder cancer hasn't seen a new therapy hit the market in more than two decades. As a breakthrough-designated therapy drug, this data, along with mature median duration of response data in the coming months, could prove to be enough to get atezolizumab approved as a treatment for mUC.

Roche Chemistry
Source: Roche.

Roche's two other midstage studies, POPLAR and BIRCH, focused on atezolizumab as a treatment for advanced non-small cell lung cancer, or NSCLC. The BIRCH trial was a single-arm study that showed atezolizumab effectively shrank tumors in 27% of patients who had progressed on prior therapies but had high levels of PD-L1 expression.

The POPLAR trial is what really turned my head, even more so than the advanced bladder cancer results. In the randomized phase 2 study, atezolizumab met its primary endpoint by demonstrating a statistically significant overall survival benefit in patients with high PD-L1 expression that was 7.7 months longer than that of the control group, which received docetaxel. Statistically significant median overall survival improvements in advanced cases of NSCLC are often measured in months, often just two or three. To see a therapy improve overall survival by 7.7 months for patients who've failed on prior therapies in a disease with a low five-year survival rate is astounding!

As with mUC, atezolizumab has the breakthrough therapy designation for advanced NSCLC patients with high PD-L1 expression who have progressed on prior therapies, and this phase 2 data will be the impetus for a new drug application filing in the U.S. and EU.

Roche Scientist

Source: Roche.

This could be just the beginning
For Roche's cancer immunotherapy, mUC and NSCLC could be just the beginning. Atezolizumab is being studied in various other indications, including both solid tumor and blood cancer trials.

For instance, a phase 1 trial is under way examining the combination of atezolizumab and Celgene's Revlimid as a treatment for multiple myeloma. Another phase 1 study looking at the combination of atezolizumab and Gazyva as a treatment for relapsed/refractory follicular lymphoma and diffuse large B cell lymphoma is ongoing. Other combo studies include the addition of cobimetinib, an experimental cancer agent from Exelixis, which is being reviewed in solid tumors and metastatic melanoma; a solid tumor combination with Avastin; and an NSCLC study with Tarceva that's focused on EGFR-activating mutations. All aforementioned combinations are currently in early clinical stages.

If Roche's approach with atezolizumab demonstrates anything, it's that cancer immunotherapies likely have their best chance of success as combination therapies with other cancer immunotherapies or existing cancer drugs.

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Source: National Cancer Institute.

Additionally, if Opdivo and Keytruda are any example, atezolizumab, if approved, will probably command a six-digit annual price tag. Being a specialized therapy that targets only patients with high levels of PD-L1 expression, Roche will more than likely be justified in placing a premium price tag on its cancer vaccine. It also doesn't hurt that atezolizumab demonstrated incredible efficacy in the two indications it reported on this weekend.

What could a full gamut of cancer indications hold for atezolizumab? It's hard to say because it's still such a young experimental therapy. We have data from two midstage studies, but have more than a handful of phase 1 studies yet to report. However, tossing out a guess based on its early success in NSCLC and mUC with high PD-L1 expression, it's easily believable that atezolizumab could be a blockbuster drug, and that with additional indications it could become one of a handful of immunotherapies to carve out $3 billion to $5 billion in annual sales within a decade. Again, this is a hypothetical estimate based on atezolizumab seeing numerous label expansions (and gaining FDA approvals for mUC and advanced NSCLC to start with).

Long story short, with immunotherapies continuing to shape near-term cancer care research, I believe you'd be a smart investor if you had Roche firmly planted on your list of potential game-changers worth keeping a close eye on.

Sean Williams owns shares of Exelixis, but has no material interest in any other companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.

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