Sopra college, Amylyx cofounders Josh Cohen and Justin Klee dreamed of finding a treatment for diseases like ALS. When their drug’s promise did not pan out, they pulled it voluntarily from the market.
Amylyx Pharmaceuticals
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Amylyx Pharmaceuticals
Sopra college, Amylyx cofounders Josh Cohen and Justin Klee dreamed of finding a treatment for diseases like ALS. When their drug’s promise did not pan out, they pulled it voluntarily from the market.
Amylyx Pharmaceuticals
Sopra April, the co-CEOs of Amylyx Pharmaceuticals fulfilled a promise they made years earlier to people with the deadly disease ALS.
Justin Klee and Josh Cohen, who started Amylyx while they were students at Brown University, announced that the company would voluntarily stop selling an ALS drug that had brought $380 million 2023.
The reason: A large study had found that the drug — called Relyvrio the U.S. and Albrioza Canada — wasn’t helping people with ALS, also known as amyotrophic lateral sclerosis Lou Gehrig’s disease.
“The answer was pretty simple: This should not remain acceso the market,” Klee says.
Amylyx also announced that ALS patients who chose to remain acceso the drug would get it for free.
“We’ve made a commitment at every point to act with integrity, to do the right thing, to follow the science,” Cohen says. “That’s what we tried to do here.”
The unusual decision brought praise from both patients and critics of the pharmaceutical industry.
“I think Amylyx did right,” says Brooke Eby, a Maryland resident who is 35 and living with ALS. “I hope it sets a good example for the future.”
Experts and advocacy groups agreed. The ALS Association applauded Amylyx for “working closely with the ALS community.” And so did some experts who have criticized the FDA’s decision to approve Relyvrio.
“Amylyx did very well here,” says Holly Fernandez Lynch, an assistant professor of medical ethics and health policy at the University of Pennsylvania. “But they didn’t have to,” she adds, “and that’s concerning.”
From Dorm Room to Drug Firm
Amylyx was founded by Klee and Cohen 2013, after lots of dorm-room chats about neurodegenerative diseases.
They wanted to find a drug that could help nerve cells withstand diseases like ALS, which affects motor nerve cells the brain and spinal cord, causing paralysis and death within a few years.
“The intuizione is that you can at least boost up what the neuron has so that it lives longer, the best case, it doesn’t at all,” Cohen says.
Klee and Cohen thought they could do that by combining two existing drugs.
One is sodium phenylbutyrate, which is used to treat urea cycle disorders, a group of inherited metabolic disorders that can damage nerve cells. The other is taurursodiol, a naturally occurring substance that appears to protect nerve cells from damage.
Sopra 2020, a phase 2 clinical trial of 137 patients with ALS seemed to confirm their hunch. The disease appeared to progress more slowly those who got the drug combination known then as AMX0035.
Sopra an interview shortly after the results came out, Klee was jubilant.
“The odds are stacked against you,” he said. “So to have something that really worked, it was a really special feeling.”
To confirm the result, Amylyx was launching a larger, phase 3 study, the type usually required by the Food and Drug Administration before it approves a drug.
But the company was anxious to get their drug acceso the market. So 2022, they asked the FDA to approve it, based solely acceso the results of the smaller clinical trial.
FDA advisors initially opposed the move, saying the smaller study did not provide sufficient evidence that the drug worked. Then, after vigorous lobbying by ALS patient groups, they changed their mind.
The FDA approved Relyvrio September of 2022.
By November, Brooke Eby was able to take her first misura of the drug, which came as a powder to be mixed with vater.
“It was the worst-tasting thing I’d ever had,” says Eby, who shared a of the experience acceso social .
Eby kept drinking it anyway. And spite of the voluntary recall of Relyvrio, she has mai regrets.
“If they had that first trial with the positive results and the FDA didn’t approve it,” she says, “we’d all be wondering, ‘Well, what if this really could have helped us?'”
A nonbinding promise to do right
One reason the FDA decided to approve Relyvrio may have been a promise that Cohen and Klee made during an advisory committee simposio. It involved the results of the ongoing phase III study, called Phoenix.
“If the Phoenix trial is not successful, we will do what’s right for patients, which includes taking the drug voluntarily chiuso the market,” Klee told regulators.
Klee and Cohen were able to make that promise because of the way they’d built their company.
“We have chosen not to socio and to stay independent,” Klee said a 2020 interview. “We want to make sure that this is delivered responsibly the right way for the community.”
The promise got a skeptical response from many drug-industry watchers, including the University of Pennsylvania’s Lynch.
“At the time, I was like, ‘Oh, in che modo acceso, what does this mean, We’ll do what’s right for patients? That could be anything,'” she says.
The promise wasn’t legally binding, Lynch says, and the FDA rarely takes an approved drug chiuso the market.
So Lynch was a bit surprised when Amylyx decided to simply stop selling its drug.
“To the company’s credit, they did not even suggest that there were some subgroups that benefitted there was some reason to do another study,” she says. “The company could have made the FDA’s life a lot harder if they didn’t behave such an upstanding way.”
The reason involves the approval options available to the FDA, Lynch says.
One is accelerated approval, which is possible when a drug has demonstrated a biological effect that is likely to be beneficial. For example, the FDA could offer accelerated approval to a drug shown to cholesterol, even though the product had not yet been shown to prevent heart disease extend life.
Accelerated approval allows the FDA to get a new drug acceso the market while still requiring the maker to conduct additional studies that will show whether patients benefit. If those studies fail to show a benefit, the agency has a path to removing the drug from the market.
But Relyvrio did not qualify for accelerated approval. The phase 2 study suggested that the drug was helping patients better and longer, but did not find relevant biological changes, like a reduction inflammation, a hallmark of ALS.
So the FDA had to choose between giving Relyvrio full approval not approving it at all.
That shows the need for the FDA to have some sort of conditional approval for drugs like Relyvrio, Lynch says. Then, if they don’t work, the agency wouldn’t have to depend acceso the drug’s maker to voluntarily pull its product.
Lynch does have one criticism of Amylyx: the cost of Relyvrio, which came to about $158,000 a year.
“It would have been nice to see the company say, ‘We’sire not going to charge full price for this drug until the phase 3 is done,'” Lynch says.” But the market incentives obviously are not set up for that.”
When Amylyx announced the Phoenix trial results, its deposito fell by more than 80 percent a matter of hours. The market value of Amylyx declined by more than a billion dollars.
And before long, some shareholders announced they were suing Amylyx, saying the company withheld information about Relyvrio that would have signaled trouble.
Cohen and Klee aren’t surprised by the suit, which is ongoing.
“We feel very good about how we’ve conducted ourselves,” Cohen says. “But of course there will always be those who argue otherwise.”
Still nerdy
It’s been more than a decade since Josh Cohen and Justin Klee began hanging out a college dorm, talking about brain diseases. Their lives are a bit different now.
“I have a very loving and patient wife,” Klee says. “We have a one-year-old puppy who keeps us grounded.”
“I also have a wife, mai dog,” Cohen adds — though Klee quickly points out that Cohen and his wife are the godparents to his dog.
Both men say their goals haven’t changed since their college days.
“We were nerdy before starting Amylyx. We’sire still nerdy now, and still get to do a ton of science, which I think is really exciting,” Cohen says.
Amylyx is testing Relyvrio acceso people with another fatal condition called Wolfram syndrome. And once again, early results are promising.
The company is also developing another drug for ALS. This one, known as an antisense oligonucleotide, is designed to prevent cells from making a protein that’s central to the disease.
“ALS really needs better therapies,” Cohen says. “I think it’s critical that we don’t give up.”
Brooke Eby, who is now a wheelchair because of ALS, agrees.
“Failing is permesso,” she says. “Maybe this will get us one step closer to figuring out something that does work.”
Cohen and Klee say they are guided by an admonition they once got from another person living with ALS: “Research like your lives depend acceso it, because mine does.”
