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A drug company will stop selling lucrative medicine to keep a promise to ALS patients

JUANA SUMMERS, HOST:

A small drug company made a big announcement last month. It said it would voluntarily stop selling a medicine that was bringing in hundreds of millions of dollars. The decision kept a promise the business had made years earlier to people with the fatal condition ALS or Lou Gehrig's disease. NPR's Jon Hamilton has more.

JON HAMILTON, BYLINE: In March, the pharmaceutical company Amylyx got some very bad news. A large study had found that its flagship drug, Relyvrio, was no better than a placebo for people with ALS, amyotrophic lateral sclerosis. Josh Cohen is co-founder and co-CEO of Amylyx. He says the company's first task was to inform several thousand ALS patients who'd been taking Relyvrio.

JOSH COHEN: As hard as it is for us, it's much harder for them. And we've made a commitment at every point to act with integrity, to do the right thing, to follow the science. And that's what we tried to do here as well.

HAMILTON: Justin Klee is the other co-founder and co-CEO of Amylyx. He says the company's next job was to decide the future of Relyvrio, which was sold under a different name in Canada.

JUSTIN KLEE: The answer is pretty simple. This should not remain on the market, but for people who believe they're benefiting, we will continue to provide drug.

HAMILTON: For free. Relyvrio's failure came less than two years after it was approved by the Food and Drug Administration, and it was a huge disappointment for many people with ALS, a nerve disease that usually causes paralysis and death within a few years. But the ALS Association and many individuals with the disease have praised Amylyx for its conduct. Brooke Eby, who is 35 and living with ALS, says she's just glad the drug got a chance.

BROOKE EBY: I think Amylyx did right. I think the FDA was thinking of patients in the right way. I think everyone acted the way they should have, and I hope it sets a good example for the future.

HAMILTON: Unusual praise for a drugmaker. But Amylyx, based in Cambridge, Mass., has an unusual history. It was started by Klee and Cohen in 2013 when they were still undergraduates at Brown University. During an interview in 2020, Cohen said their dream was to find a drug that could help nerve cells withstand the deadly onslaught of diseases like Alzheimer's and ALS.

COHEN: The idea is that you can at least boost up what the neuron has so that it lives longer or in the best case doesn't die at all.

HAMILTON: Klee and Cohen thought they could do that by combining two existing drugs. In animal models of ALS, each of these drugs appeared to help keep nerve cells alive. In 2020, a clinical trial of 137 patients suggested the drug combination could slow down ALS in people. Klee was jubilant.

KLEE: The odds are stacked against you. So to have something that really worked, it was a really special feeling.

HAMILTON: To confirm the result, Amylyx was doing a larger Phase 3 trial. But the company was anxious to get their drug on the market, so they asked the Food and Drug Administration to approve it based solely on the results of the smaller study. FDA advisers initially opposed the move, then changed their mind. Brooke Eby, the ALS patient, remembers when she first heard about the treatment.

EBY: I got diagnosed in March of 2022, and they mentioned there was this drug that was, like, about to be approved.

HAMILTON: The FDA cleared Relyvrio in September. In November, Eby posted a video to her Instagram account.

(SOUNDBITE OF ARCHIVED RECORDING)

EBY: Day 1 of taking Relyvrio, the medicine that was just approved by the FDA. The pharmacist spent 10 minutes telling me how bad this tastes.

HAMILTON: She opens a packet, stirs the powder into a glass of water and lines up a Gatorade chaser.

(SOUNDBITE OF ARCHIVED RECORDING)

EBY: Ready? This is going to be a big one. (Groaning) Oh, my God.

It was the worst-tasting thing I'd ever had (laughter).

HAMILTON: Eby kept drinking it anyway, and she has no regrets.

EBY: If they had that first trial with the positive results and the FDA didn't approve it, we'd all be wondering, like, what if this could've really helped us?

HAMILTON: One reason the FDA decided to approve Relyvrio may have been a public promise that Cohen and Klee made. It involved the results of an ongoing Phase 3 trial called PHOENIX.

(SOUNDBITE OF ARCHIVED RECORDING)

KLEE: If the PHOENIX trial is not successful, we will do what's right for patients, which includes taking the drug voluntarily off the market.

HAMILTON: Klee and Cohen were able to make that promise because of the way they'd built their company. Here's Klee in 2022 explaining their strategy.

(SOUNDBITE OF ARCHIVED RECORDING)

KLEE: We have chosen not to partner and to stay independent, and it's because we want to make sure that this is delivered responsibly, in the right way for the community. It's something we really care about.

HAMILTON: A nice sentiment, but did they really mean it? One skeptic was Holly Fernandez Lynch, an assistant professor of medical ethics and health policy at the University of Pennsylvania.

HOLLY FERNANDEZ LYNCH: At the time I was like, oh, come on, right? You know, what does this mean, we'll do what's right for patients? That could be anything.

HAMILTON: Lynch says the promise wasn't legally binding, and the FDA rarely takes an approved drug off the market. So Lynch was a bit surprised when Amylyx decided to stop selling its drug.

LYNCH: Amylyx did very well here, but they didn't have to by law do very well here. And that's concerning, right?

HAMILTON: Lynch says the FDA needs a better system for reconsidering drugs that have already been approved. And she does have one criticism of Amylyx, the cost of Relyvrio, which came to about $158,000 a year. Lynch says that's a lot for a drug that hasn't been through a Phase 3 clinical trial.

LYNCH: It would have been nice to see the company say, we're not going to charge full price for this drug until the Phase 3 is done, but the market incentives obviously are not set up for that.

HAMILTON: When Amylyx announced the results of the PHOENIX trial, its stock fell by more than 80% in a matter of hours. The market value of Amylyx declined by more than $1 billion. And now some shareholders are suing Amylyx, saying the company withheld information about Relyvrio that would have signaled trouble. Cohen says he's not surprised by the suit.

COHEN: We've always tried to be clear and transparent in our communications, so we feel very good about how we've conducted ourselves. But, you know, of course, there will always be those who argue otherwise.

HAMILTON: It's been more than a decade since Josh Cohen and Justin Klee began hanging out in a college dorm, talking about brain diseases. Klee says their lives are a bit different now.

KLEE: I have a very loving and patient wife. We have a 1-year-old puppy who keeps us grounded.

COHEN: I also have a wife, no dog.

KLEE: Josh and his wife are the godparents to our dog, so you know.

HAMILTON: But Cohen says their goals are the same.

COHEN: We were nerdy before starting Amylyx, we're still nerdy now, still get to do a ton of science, which I think is really exciting.

HAMILTON: Amylyx is now testing Relyvrio on people with another fatal condition called Wolfram syndrome, and once again, early results are promising. The company is also developing a new drug for ALS. Cohen says this one is designed to prevent cells from making a protein that's central to the disease.

COHEN: ALS really needs better therapies. And I think it's critical that just because there are challenges or setbacks that we don't give up.

HAMILTON: Cohen and Klee say they continue to be guided by a plea they once got from a person living with ALS. Research like your lives depend on it because mine does. Jon Hamilton, NPR News.

(SOUNDBITE OF SZA SONG, "GOOD DAYS") Transcript provided by NPR, Copyright NPR.

NPR transcripts are created on a rush deadline by an NPR contractor. This text may not be in its final form and may be updated or revised in the future. Accuracy and availability may vary. The authoritative record of NPR’s programming is the audio record.

Jon Hamilton is a correspondent for NPR's Science Desk. Currently he focuses on neuroscience and health risks.