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The hottest thing in cancer drug development just won a $9 billion endorsement

Lydia Ramsey   

The hottest thing in cancer drug development just won a $9 billion endorsement

Cancer immunotherapy

Reuters

Cancer cells are seen on a large screen connected to a microscope at the CeBit computer fair in Hanover, Germany, March, 6, 2012.

  • Biotech giant Celgene is acquiring Juno Therapeutics for $9 billion.
  • Juno is known for its experimental, highly personalized cancer treatments called CAR T-cell therapy, which harnesses the body's immune system to go after certain blood cancers.
  • It's part of a new field of cancer therapies that. In 2017, the FDA approved two such treatments, one to treat acute lymphoblastic leukemia in people up to age 25 and another to treat aggressive B-cell non-Hodgkin lymphoma.
  • Celgene said on Monday that Juno's farthest along treatment - which is expected to be approved in 2019 - has the potential to hit $3 billion in global sales.

Celgene is making a $9 billion bet on a new form of personalized cancer treatment that harnesses the body's immune system.

It's the second acquisition this month for the biotech giant, which picked up Impact Biosciences in a $7 billion deal on January 7.

Juno is known for its experimental, highly personalized cancer treatments called CAR T-cell therapy (CAR is short for chimeric antigen receptor). So far, the 2017 Food and Drug Administration has approved two treatments, one to treat pediatric acute lymphoblastic leukemia in people up to age 25 called Kymriah and another to treat aggressive B-cell non-Hodgkin lymphoma called Yescarta.

JCAR017, Juno's furthest-along treatment, is expected to be approved in the US to treat diffuse large B-cell lymphoma (or DLBCL) in 2019.

Celgene said the "potentially best-in-class" treatment had the potential to hit $3 billion in global sales. It's a high target to put on a highly personalized treatment that faces competition and side effects that could keep it from being used as frequently.

Here's how CAR-T cell therapy works

These treatments aren't your run-of-the-mill pill - or even a biologic drug, like insulin - that can be mass-produced. Since the therapy is made from a person's own immune system, the process can take about three weeks.

  • To start, a doctor removes some white blood cells, the part of our body's immune system responsible for combatting infections and foreign substances, from a patient. In a healthy body, the immune system can recognize abnormal, cancerous cells, but for people with cancer, it doesn't recognize that the cells are spreading.
  • The cells are taken to a manufacturing facility where they are re-engineered to recognize cancer cells and wipe them out.
  • Those reprogrammed cells are sent back and administered to the patient.

How Car-T Therapy Works cancer

Ana Pelisson/Business Insider; Novartis

While the treatments don't work in all patients, it can have dramatic results in those who do respond. For example, in a trial of 63 patients treated with Kymriah - the first cell therapy approved - 83% were in remission after three months, and 64% were still in remission after a year.

In part because of the extensive process that's involved, these CAR-T cell therapies don't come cheap: Yescarta has a list price of $373,000, while Kymriah price tag is $475,000.

And the treatments can also have some serious - and deadly - side effects, which can keep them from being used earlier in treating a patient's cancer. It's a challenge researchers are looking to overcome with newer treatments.

If approved, JCAR017 would compete with Yescarta, which is already approved for DLBCL. DLBCL will likely be the second type of cancer that Kymriah's approved for, too. The $3 billion peak-sales figure would be higher than the $2 billion that's been estimated for Yescarta. Previously, investors have estimated that the peak sales from all the programs Juno has in the works would hit $2.5 billion.

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