This weekend a slate of biopharma companies will be presenting new data at ASCO, underscoring the big new role that immunotherapy drugs will be playing in the fight against cancer. In part, that's because of the success of Yervoy, which sells for $40,000 a month. Now Bristol-Myers Squibb ($BMY), Merck ($MRK) and others believe they have a key to further advances in a variety of cancers. And that's raising some big questions about the impact these drugs will have in a market that is feeling the impact of six-figure oncology treatments.
Bristol-Myers Squibb is one of the leaders in this movement. Two years after it gained an approval for Yervoy, the drug developer is back with a closely watched combo that adds nivolumab to the mix. The drug targets the PD-1 cloaking device, opening up a fresh T-cell attack on a variety of solid tumors. The FDA has put it on the agency's fast track for melanoma, lung and kidney cancer.
These new therapies will likely add considerably to the cost of care. As they rescue a growing number of patients from an early death, they'll also become chronic disease therapies that could be needed for years to come. A Nature article picks up on the cost trend, noting that cancer drug costs are rising at 15% a year, twice the rate of overall healthcare inflation. That's likely to make these drugs a lightning rod for private and public payers looking to contain the cost of healthcare in the U.S.
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"Cancer is a very complicated and expensive disease," Scott Ramsey, a healthcare economist at the Fred Hutchinson Cancer Research Center in Seattle, Washington, tells Nature. "But now it's turning into a chronic disease, and we're talking about years of maintenance therapy with drugs that cost $10,000 a month."
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Oncology is the most crowded area of drug research. So the shorter development cycle helps limit companies' risk of chasing the same or very similar targets as a bevy of other companies, wagering on their program to come out on top. Novartis might have the frontrunner with LDK378, but it is one of no fewer than four experimental compounds against ALK in lung cancer. These drugs home in on misfit proteins in an ALK-positive subgroup that represents as little as 3% of the NSCLC population.
The FDA is opening the inside track to more genetic therapies in response to overwhelming patient need, as the efficacy of the first wave of gene-targeting drugs fades after clever cancers adapt and find new pathways to grow out of control. Roche ($RHHBY) and other companies also face patent expirees to existing drugs, and the Swiss drugmaker has mounted an effort to make one of its best drugs, Rituxan, even greater with a glycoengineered successor called GA101.
Targeted or not, none of these drugs is a silver bullet, and patients must still endure side effects. Yet there's hope for some cancers to become manageable chronic illnesses as opposed to death sentences.
Ibrutinib has sparked a frenzy of activity in the clinical and investment worlds with promising evidence of efficacy in a lineup of B-cell cancers. The blockbuster prospects of the Bruton's tyrosine kinase (BTK) inhibitor have driven the market value of its developer, Pharmacyclics ($PCYC), to more than $5 billion, the highest among clinical-stage companies in the oncology arena, and stamped company CEO and Chairman Bob Duggan's ticket into the billionaire's club.
As one physician described ibrutinib to a Forbes scribe recently, the compound could become the Gleevec of B-cell cancers such as chronic lymphocytic leukemia (CLL) and mantle cell lymphoma. Now in Phase III studies, ibrutinib has shown impressive response rates and progression-stealing results in midstage tests. In a Phase Ib/II study in CLL patients, for instance, cancer progression halted in an estimated 96% of patients without prior treatment and 75% of those with high-risk cases at up to 26 months after they started to take ibrutinib.
Johnson & Johnson ($JNJ) wants to add the compound to its growing roster of cancer therapies, and the pharma giant and Pharmacyclics have raced ahead with 5 late-stage clinical trials for the experimental drug through their collaboration that began in December 2011. The partners have already found a supportive audience with the FDA, which has granted three "Breakthrough Therapy" designations for the compound for use in separate pools of cancer patients.
With the first submission for FDA approval expected in the third quarter of 2013, Pharmacyclics and J&J could win a quick stamp from the agency and have ibrutinib cleared for the U.S. market before the end of the calendar year. The pace of the agency's action on the application will speak volumes about the value of "Breakthrough" status. If approved for CLL and other uses, analysts estimate that ibrutinib could become one of the biggest moneymakers in oncology with up to $5 billion in annual sales.
Idelalisib targets a subset of PI3K proteins that is known to accelerate the development of cancer, the kind of clear biologic pathway the FDA enjoys. As a result, it's in direct competition with IPI-145, a PI3K delta and gamma inhibitor in development at Infinity Pharmaceuticals ($INFI), which has been winning some enthusiastic supporters with evidence of possibly greater activity and lower liver toxicity. But while Infinity has been focusing on restructuring the company around IPI-145, which is now delivering Phase I data, Gilead has been ramping up its late-stage clinical campaign, putting its drug well in the lead. And now Infinity may be forced to settle for next-gen drug status as it watches Gilead hustle ahead.
It certainly doesn't lack for attention. ASCO President Sandra Swain called the recent results "pretty incredible," while Dana-Farber's Jennifer Brown enthused: "Drugs like idelalisib are probably going to change the landscape of the disease in the next few years."
PSMA-targeted nanoparticle containing docetaxel
While easily the least proven candidate in this report, BIND-014 has shown early promise in combatting a range of solid tumors in Phase I trials, and Bind Therapeutics plans to prove the mettle of its lead candidate in three Phase II trials slated for this year in lung, prostate and bladder cancers.
Cambridge, MA-based Bind highlighted the Phase I data on 014, which encapsulates the chemo drug docetaxel in a nanoparticle, in an oral presentation at the American Association for Cancer Research. Getting the spotlight at AACR is no small deal. In 28 cancer patients who had already received a battery of treatments, Bind's drug shrank or stabilized tumors in 9 patients, including a complete response in a patient with cervical cancer as well as partial responses in patients with non-small cell lung, prostate and ampullary tumors.
Bind has a long way to go with this program. Yet based on early work with its Accurin nanotechnology platform, the company has landed a troika of major corporate partnerships this year on other assets with Amgen ($AMGN), Pfizer ($PFE) and AstraZeneca ($AZN). Those deals improve the odds of a success from Bind's technology, which came from labs of MIT inventor Bob Langer and his colleagues.
A DNA cancer vaccine developed by Blue Bell, PA-based Inovio Pharmaceuticals caused tumor cell death and increased the rate of survival in animal studies. Using the company's adaptive electroporation delivery technology, the hTERT DNA vaccine broke the immune system's tolerance to its self-antigens and induced T cells with a tumor-killing function. High levels of human telomerase reverse transcriptase, also known as hTERT, are detected in over 85% of human cancers.