The Pharma Exec behind the First Approved Gene Therapy Is Hunting for His Next Big Break - DiscoveryCampus
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The Pharma Exec behind the First Approved Gene Therapy Is Hunting for His Next Big Break

The Pharma Exec behind the First Approved Gene Therapy Is Hunting for His Next Big Break

13:27 25 January in Uncategorized
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Joseph Jimenez retires at the end of the month after eight years in the top job at Novartis, one of the world’s largest pharmaceutical companies. Under his leadership the company sold off its vaccine division and shifted its focus to developing generic drugs as well as products for treating retinal diseases, glaucoma and dry eye, among other conditions. But his tenure will be remembered largely for putting gene therapies on the map.

Last year Novartis became the first company to get approval from the U.S. Food and Drug Administration for this type of treatment. The company’s CAR-T (short for chimeric antigen receptor T cells) therapy is a type of personalized medicine in which a patient’s own immune cells are removed, reengineered to target and kill cancer cells and infused back into the body. Marketed under the name Kymriah, this therapy is currently only approved to treat children and young adults with acute lymphoblastic leukemia, but the company expects it will eventually be approved for other cancers.

The catch, of course, is the price tag. Kymriah, developed in collaboration with researchers at the University of Pennsylvania under the name CTL019, is a one-time infusion that costs $475,000 per person. In an unusual move designed to counter complaints about unaffordability, Jimenez has promised Novartis will only charge for Kymriah if a patient sees a positive response within the first 30 days. (Studies show the treatment triggers a complete remission in more than 80 percent of patients, who typically start to see results within a few days.) Some patients relapse in the long term, or develop serious side effectsincluding an immune system overreaction that leads to intense flulike symptoms. Yet the extraordinarily high response rate puts Kymriah in the very small category of truly transformative treatments.

Scientific American spoke with Jimenez about CAR-T therapy, his hopes for new game-changing treatments that could target disease-causing proteins and problems with the U.S. health care system.

[An edited transcript of the interview follows.]

Do you think CAR-T immune therapies like Kymriah may ever be useful beyond blood cancers?

We have mixed views at the company about that. Some [of us] believe that we will find a way to impact solid tumors, particularly in combination with other drugs. Right now we’re very confident in the technology for different blood cancers but we’re still in the proof-of-concept stages for solid tumors.

What made you pursue CAR-T, even as other large pharmaceutical companies decided not to bet on the technology?

Novartis was known for breakthrough innovations in oncology back in the early 2000s. We developed Gleevec [which treats certain types of blood and intestinal cancers with less collateral damage to healthy cells than a typical chemotherapy]. When we saw the data on CTL019 CAR-T coming out of Penn—the really early data about five years ago—we decided that this was a place where we were going to place a big bet. The data reminded us of the early days of Gleevec, and so we went out on a limb.

Why is Novartis able to invest in such a long-term gamble while other companies aren’t?

We’re a Swiss company, which makes us a little bit more insulated from the quarter-to-quarter pressures that some of the other pharmaceutical companies feel. We have a longer-term view and we have an investor base that is perhaps more patient than some others, so that gives us the ability to invest for the long term.

Is there another gamble like that in the works now?

There’s a new technology that Jay Bradner, the head of our research organization, is championing: protein degradation [essentially tricking the body into disposing of harmful proteins], which is a new approach to addressing intractable [disease] targets. We’re also investing heavily in gene editing because we do believe there will be therapeutic value coming out of gene editing.

How confident are you that CRISPR will be usable to edit genes in sick people?

So far it’s promising, but I think everybody is in a wait-and-see mode. There will be twists and turns along the path, as there is with any new technology.

After more than a decade working for a European drug company, have you come up with any ideas for fixing America’s health care system?

Obviously, the current system is not sustainable. I really believe that the only way it’s going to shift is if we shift away from this transactional approach of selling pills or fee-for-service towards an outcomes-based one, where we get paid for the results that we deliver. If you were to say, “We’re only going to get paid based on the outcome we deliver,” you could eliminate a lot of the waste in the system.

Do you have a solution for the high cost of drugs?

A pharmaceutical company gets 60 percent of the dollar that is paid for the drug. Forty percent goes to the supply chain: the pharmacy benefit manager, the wholesaler, the retailer. That seems wrong. We’re the ones taking all the risk. We’re the ones developing the drug. That 40 percent—how do we find a way to get [some of] that money into the hands of the patient at the pharmacy benefit counter? If we can do that, we can put a serious dent in pharma costs in the U.S.

So, you’re saying drug costs could come down by cutting the money made by insurance companies and retailers? How do you make that happen?

Most Medicare patients are on fixed incomes, and when they go to the pharmacy counter they’re not able to get any kind of co-pay assistance. One way we could help these seniors that are seeing big increases in either deductibles or co-pays is to help them with that out-of-pocket expense. There is a legislative and regulatory approach that we’re working on to try to effect that.

What’s next for you? Will you be starting a company, or joining one?

I haven’t decided if I want to do a portfolio of companies and be on the investing side—helping these companies get formed and started—or whether I want to enter an early-stage company and actually operate it.


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