Cancer therapies are notoriously elusive.
The disease is complex, clinical development is expensive, and failure rates are high.
But biotech and pharmaceutical companies around New Jersey are building a pipeline of potential breakthroughs, largely centered on drugs that stimulate the body's own immune system to fight cancer, known as immunotherapies.
The newfound optimism stems from clinical results showing improved survival rates among patients receiving immunotherapy, fueling hope that cancer can become more manageable and less of a death sentence.
That someday there could be a cure.
“It is the hottest area of cancer research today,” said Daniel O'Connor, the CEO of Advaxis, a company we profile on page 13. “I think it will be the future of where cancer research will be.”
Tibor Keler, the chief scientific officer for Celldex Therapeutics Inc., a Hunterdon County biotech with several immunotherapies in the pipeline, agrees.
“The biggest recognition is how powerful your immune system can be in terms of fighting cancer. And that's been proven unequivocally at this point,” Keler said.
The progress is welcome news for an industry hungry for new revenue in an increasingly post-blockbuster age. Many immunotherapies are being developed by emerging biotechs, but bigger pharmaceutical companies reeling from expired patents are also investigating.
Merck & Co., whose revenue suffered when the blockbuster asthma and allergy drug Singulair came off patent in 2012, received a boost in April when federal regulators granted breakthrough status for its experimental immunotherapy drug MK-3475 for treatment of melanoma. The designation expedites Food and Drug Administration reviews for drugs on the grounds such medicines serve an unmet need.
Immunotherapy is appealing because it lessens the dependence on traditional approaches, such as chemotherapy and radiation and their negative side effects.
As recently as the mid-1990s, immunotherapy offered little proof of success. But clinical results have dramatically improved in the last decade thanks to technological advances.
“Nobody looks at it as a fluke or a one-off opportunity anymore,” Keler said.
Celldex's lead drug candidate, Rindopepimut, attacks glioblastoma, a form of brain cancer that can quickly lead to death. About 14 percent of patients taking Rindopepimut in Phase II clinical trials continued to live for five years and reported median survival of 24 months. That's for a cancer with a median survival rate of about 15 months using standard treatment alone.
Keler said the results demonstrate why people are excited about immunotherapy. Unlike chemotherapy, which tends to make big initial impact in shrinking tumors but then declines in effectiveness, Keler said immunotherapy makes less of an initial splash but works effectively for longer durations.
“Years ago, nobody would have thought you have had a five-year survival for brain cancer,” said Celldex CEO Anthony Marucci. “It was unheard of.”
Rindopepimut is currently in late-stage trials that are expected to finish in 2014. If plans move accordingly, Celldex expects FDA approval by 2016. The company envisions a $500 million market for the vaccine.
Merck is boasting results for immunotherapy, too. The Readington Township company's MK-3475 drug demonstrated an 81 percent survival rate among advanced melanoma patients after 12 months of therapy. Analyst projections for MK-3475 have ranged from $500 million for melanoma to $2 to $3 billion — blockbuster range — if treatment is approved for additional cancers. The company said an initial launch should happen by 2015.
To date, only two immunotherapies have been approved by the FDA, both in 2011: Yervoy, a melanoma drug made by Bristol-Myers Squibb and the prostate cancer drug Provenge, made by Dendreon.Yervoy generated $706 million in sales in 2012 while Provenge recorded $325 million in sales.
Experts say cancer drugs have blockbuster potential, though less so than conventional blockbuster drugs aimed at larger populations suffering more common ailments.
Celldex said it hasn't had problems raising money. The publicly traded company raised about $300 million in two stock offerings in 2013 and reported $136.6 million in cash at the end of the third quarter.
The pre-revenue company will need cash and capital as it completes clinical trials of Rindopepimut and CDX-011 and advances other drugs through the pipeline.
Plus, Marucci expects to bolster the company's sales team pending FDA approvals. Confident in its growth, Celldex moved last year from Phillipsburg to larger headquarters in a Union Township corporate park off Interstate 78.
Chalk up the optimism to wider acceptance of immunotherapy supported by stronger clinical data.
“I think immunotherapy is just scratching the surface,” Marucci said.
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