The hottest area in cancer drugs is going mainstream this year.
If 2014 proved that the most promising new group of oncology drugs in generations could work, 2015 brings a crowded field that sees winners and losers in a market eventually worth $30 billion a year or more in the next decade.
The treatments, known broadly as immunotherapies or immune-oncology, fall into two major categories -- drugs that help take the brakes off the immune response, going after solid tumors like melanoma and lung cancer, and customized treatments that modify immune cells to combat blood malignancies.
“I think 2015 is the end of the beginning in the story of immunotherapy,” said Michael Giordano, head of development of oncology and immunology at Bristol-Myers Squibb Co. “2015 will be a pivot point where I-O will be mainstreamed beyond melanoma and we’ll start seeing it approved and used in large tumors.”
There are 374 experimental cancer drugs in mid-stage trials, according to the IMS Institute for Healthcare Informatics’ global outlook report published last year. That’s more than twice as many drugs as for nervous system disorders, for example. Of the experimental cancer drugs, about 25 percent to 30 percent are immunotherapies, according to IMS.
In trials, Merck & Co. and Bristol-Myers’ drugs showed long-lasting effects in some patients that oncologists have called dramatic.
There a huge unmet medical need from patients, for whom many new treatments extend lives by years, rather than months. And the financial reward for drugmakers is huge. The drugs have annual costs of $150,000, and a slate of personalized treatments are expected to carry even higher price tags.
Kite Pharma Inc., Juno Therapeutics Inc. and Bluebird Bio Inc. have gathered interest from investors for their therapies to modify the immune system cells and retrain them to attack blood cancers, a technique known as CAR. Kite and Bluebird rose more than fourfold in the last year, while Juno’s shares have more than doubled since their initial public offering last month. All three companies are scheduled to present at the JPMorgan Health Care Conference in San Francisco next week.
Kite plans to file for regulatory approval for its treatment by end of 2016, according to spokesman Justin Jackson. Juno plans to file an application by late 2016 or early 2017, according to company filings.
Bristol-Myers and Merck, meanwhile, are racing to expand their drugs beyond melanoma. They expect approvals in lung cancer this year, and are testing on a wide range of solid tumors.
They’re also pushing related immune-oncology drugs through their pipelines that will used in combination with one another. While their drugs have only shrunk tumors in about 30 percent of the population, combinations may help broaden their use.
They’ll have competition. In 2015, AstraZeneca Plc, Roche AG and Merck may all release data on new immune system mechanisms, according to Seamus Fernandez, an analyst with Leerink Partners. In combination treatments, AstraZeneca “has now advanced from ‘dark horse’ to ‘major contender,’’ Fernandez said. ‘‘Real leadership remains wide open.’’
Roche has more than 20 molecules in its immunotherapy pipeline, and looks to leverage the broad portfolio to craft combination therapies, Daniel O’Day, chief operating officer of the drugmaker’s pharmaceuticals division, said in an October conference call.
Large drugmakers are also joining forces with biotechnology companies for their boutique blood cancer treatments. While questions linger about the cost and scalability of the CAR therapies, bigger biotechs and pharma companies are jumping into the fray nonetheless.
There have already been four licensing deals adding up to more than $1 billion, including an agreement between Amgen Inc. and Kite valued at as much $525 per therapy, plus royalties.
Celgene Corp. has a pact for as much as $225 million per product with Bluebird Bio, and Pfizer Inc. has promised Paris-based Cellectis $80 million upfront and milestone payments of as much as $185 million to share in their CAR program. Novartis AG is also working on the therapy.
‘‘A new pillar of cancer care is now firmly established,” Fernandez wrote in a Dec. 29 note to clients. As treatments begin to hit the market and new drug combinations and cancer types are explored, “things are just getting interesting,” he said.