The head of the FDA is calling for an investigation into her agency’s controversial decision to approve a new Alzheimer’s drug

Janet Woodcock
Interim FDA Commission Janet Woodcock.

  • Interim FDA Commissioner Janet Woodcock has asked for an outside investigation into an Alzheimer’s drug approval.
  • The agency approved the drug, Aduhelm, last month, despite a lack of evidence showing the drug slowed the disease.
  • Officials from Biogen and the FDA worked closely together to revive the drug, raising concerns.
  • See more stories on Insider’s business page.

The top leader at the US Food and Drug Administration is asking for an investigation into the agency’s controversial decision to approve an expensive new Alzheimer’s disease treatment.

Janet Woodcock, acting commissioner of the FDA, published a letter Friday asking the federal government’s top inspector to investigate if FDA staff worked inappropriately with biotech company Biogen to approve the new drug.

“To the extent these concerns could undermine the public’s confidence in FDA’s decision, I believe that it is critical that the events at issue be reviewed by an independent body such as the Office of the Inspector General, in order to determine whether any interactions that occurred between Biogen and FDA review staff were inconsistent with FDA policies and procedures,” she wrote in the open letter.

The drug in question, Aduhelm, was approved by the agency last month and has caused an uproar in the scientific community. Three of the agency’s scientific advisors resigned following the decision.

Documents released by the FDA last month show that people within the agency thought that the drug should not have been approved because human testing data didn’t show it worked, but their concerns were overridden by top staff.

Biogen officials used back channels to get the drug approved after a late-stage clinical trial failed in March 2019, according to a report from STAT News.

The interactions pre-dated Woodcock, who became interim commissioner in January when the Biden Administration took office. She has been with the FDA for more than two decades and is one of the top contenders to permanently take the role, according to biotech analysts.

After the FDA approved Aduhelm, Biogen set its price at about $56,000 a year. Analysts estimate the US Medicare program could pay anywhere from $6 billion to $29 billion per year for Aduhelm, according to a recent New York Times analysis.

Biogen’s stock, which surged when the FDA approved Aduhelm, declined 3.2% to $357.09 a share on Friday afternoon.

Read the original article on Business Insider

What a controversial drug approval could mean for future Alzheimer’s treatments

Hello,

Welcome to Insider Healthcare. I’m Lydia Ramsey Pflanzer, and this week in healthcare news:

If you’re new to this newsletter, sign up here. Tips, comments? Email me at lramsey@insider.com or tweet @lydiaramsey125. Let’s get to it…


We’re still looking for nominees for our annual 30 under 40 list!

Know someone under 40 transforming the healthcare industry? Submit their name here. Nominations are due by June 30.


alzheimers research 4x3

A massive – and controversial – Alzheimer’s drug approval

Wow, I can’t believe it was just this Monday that the Food and Drug Administration approved the first new Alzheimer’s treatment in nearly two decades.

There were a few surprises that came with the approval of the drug, Aduhelm. For one, there were no real limitations placed by FDA on who will be eligible for the new drug. And the drug costs roughly $56,000 a year.

…. And let’s not forget this is a drug that an expert panel voted against approving last November.

Allison DeAngelis and Shelby Livingston this week dug into a little-known barrier could stand in the way of millions of patients clamoring to get the treatment.

Since the approval, doctors have started resigning from the FDA’s expert panel – the one that voted against approving the drug.

Allison and Andrew Dunn analyzed the problems facing Aduhelm – and future Alzheimer’s drugs.

Find out more>>

The first new Alzheimer’s drug in decades costs $56,000, probably doesn’t work, and could stand in the way of better treatments


Noom
Noom is an app-based weight management startup.

Noom’s big bet on preventive care

This week, Megan Herbroth and Gabby Landsverk went digging into Noom.

The weight-loss startup stunned the healthcare industry in May when it raised $540 million from investors including Silver Lake and Oak HC/FT. And it’s betting big that it can do for sleep and anxiety what it did for weight loss.

It’s taken 13 years to get to this point, and not every idea worked.

Megan and Gabby spoke to Noom’s CEO, investors, and experts to get a sense of the journey that now convinces investors Noom’s worth $3.7 billion.

Get the full history>>

Inside the rise of $3.7 billion Noom: How a 13-year-old weight-loss startup is taking on one of the fastest-growing markets in digital health


Amazon Pharmacy

Amazon and Walmart face off over prescriptions

Is it just me, or has a lot of news about the health ambitions of Amazon and Walmart come out in near tandem?

This week, it was the news that Amazon and Walmart are competing to drive down the cost of medications.

And prescriptions isn’t the only place where competition between the two retail giants is heating up. Both are beefing up their virtual care presence. Last Friday, Shelby had the scoop that Walmart quietly filed to be a provider in 16 new states.

In a rare public appearance, a top Amazon VP shared that Amazon’s medical service will announce multiple companies it’s working with in the next few months.

When it comes to prescriptions, the two have the potential to shake up the pharmacy industry, Morgan Stanley analysts wrote in a note this week.

Here’s what’s at stake>>

Amazon and Walmart are competing to offer you the cheapest prescriptions, and it could shake up the entire pharmacy industry


More stories we covered this week:


– Lydia

Read the original article on Business Insider

Scientists are reaching a new understanding about Alzheimer’s disease

Hello,

Welcome to Insider Healthcare. I’m Lydia Ramsey Pflanzer, back after a week away to ski and feeling refreshed. Today in healthcare news:

If you’re new to this newsletter, sign up here. Comments, tips, tricks for successfully scheduling vaccine appointments in the state of Colorado? Email me at lramsey@insider.com or tweet @lydiaramsey125. Now, let’s get to it…


alzheimers research 4x3

Scientists are coming around to a surprising new understanding of Alzheimer’s disease, and it could supercharge drug development for the $1.1 trillion problem

Find out more here>>


Vaccine distribution

CDC: Real-world data shows Pfizer’s and Moderna’s COVID-19 vaccines are 90% effective at preventing infection

Get the full story here>>


One of Crossover Health's exam rooms at its Midtown, Manhattan location.
One of Crossover Health’s exam rooms at its Midtown, Manhattan location.

An investor presentation lays out how a key healthcare partner for Amazon is thinking about the future of its business

See the presentation here>>


More stories we’re reading today:


Lydia

Read the original article on Business Insider

New data on Eli Lilly’s experimental Alzheimer’s drug backs a hotly debated scientific theory, but shows mixed benefits for patients

FILE PHOTO:  Patients with Alzheimer's and dementia are sit inside the Alzheimer foundation in Mexico City April 19, 2012. REUTERS/Edgard Garrido
Patients sit inside the Alzheimer’s Foundation in Mexico City

  • Eli Lilly’s experimental treatment slowed down cognitive decline by 32% in a midstage trial.
  • But the drug missed most of the secondary trial goals, including one watched by experts.
  • An Eli Lilly executive told Insider the data was mixed, but very exciting.
  • See more stories on Insider’s business page.

The latest test of Eli Lilly’s Alzheimer’s disease drug ended with a mixed bag of results, but experts say there are signs the experimental treatment may curb the disease’s decimation of cognition and memory.

Lilly gave an in-depth look at the performance of its drug, called donanemab, on Saturday at the International Conference on Alzheimer’s and Parkinson’s Diseases. The drug met the trial’s primary goals, reducing a harmful substance that builds up in the brains of patients with Alzheimer’s disease. It also slowed down the rate of cognitive decline by 32%, according to data scheduled to be published Saturday in a medical journal. But it missed the mark on several secondary tests.

The Alzheimer’s drug-development field has been marked by failure after failure, so the news that Lilly may have a working drug sent a surge of energy through the field when the drugmaker first disclosed some information about the results in January.

Those earlier failures – even within Lilly’s own drug-development pipeline – may have tempered expectations for donanemab. Dr. Mark Mintun, the vice president of the Alzheimer’s disease unit at Lilly, said he couldn’t think of a similar Alzheimer’s disease trial that hit its primary goal.

At this point, any news is good news, Dr. Howard Fillit, executive director of the Alzheimer’s Drug Discovery Foundation, told Insider.

“We’ve basically had a 100% failure rate on drug approvals since 2003. The thing that we’re going for is incremental benefits,” he said.

The donanemab data may give hope to the scientific community and other drug companies, but Fillit said the jury is still out on how effective the drug will be for patients themselves.

Donanemab missed the mark on a memory assessment experts were watching

Donanemab was designed to target a plaque that builds up in the brains of Alzheimer’s patients called amyloid beta. The hope is that getting rid of the plaque will reduce the rate of decline of a person’s memory and cognition.

Scans of trial participants’ brains showed that people taking donanemab did see their plaques fade away. That’s not only a boon to Eli Lilly, but other drugmakers that are working on treatments targeting amyloid beta, such as Biogen and Roche.

FILE - In this May 19, 2015, file photo, a doctor points to PET scan results that are part of a study on Alzheimer's disease at a hospital in Washington. Scientists know that long before the memory problems of Alzheimer's become obvious, people experience more subtle changes in their thinking and judgment. (AP Photo/Evan Vucci, File)
FILE – In this May 19, 2015, file photo, a doctor points to PET scan results that are part of a study on Alzheimer’s disease at a hospital in Washington. Scientists know that long before the memory problems of Alzheimer’s become obvious, people experience more subtle changes in their thinking and judgment. (AP Photo/Evan Vucci, File)

It wasn’t as clear how much benefit donanemab had on subjects’ ability to recall words, follow instructions and other abilities lost to dementia.

The drug passed the main test of memory and cognition.

But Alzheimer’s experts had their eyes on one datapoint in particular: the scores from a widely accepted dementia assessment known as CDR-SB. The tool is used to measure things like memory and judgement, as well as a person’s ability to do household chores and maintain their personal hygiene. The higher the score, the more skills have been lost to dementia like Alzheimer’s.

Eli Lilly’s drug didn’t meet expectations there.

The company reported that trial participants taking donanemab had a 0.36 point score difference compared to the participants that received a placebo treatment.

For comparison, Dr. Lon Schneider, director of the California Alzheimer’s Disease Center at University of Southern California, had hoped to see at least a one-point difference, according to the Wall Street research firm Mizuho.

Mintun, the Eli Lilly executive, said he hadn’t expected that donanemab would pass all five dementia tests, calling it statistically impossible. But giving patients more time to hold onto memories is meaningful for them, he said.

The clinical trial took a ‘Goldilocks approach,’ and more testing is needed

The donanemab trial assessed a small and very specific group of patients. The trial was limited to participants with amyloid plaques, as well as another protein linked to Alzheimer’s called tau. Fillit called it a Goldilocks approach – in the end, Lilly ended up with participants whose Alzheimer’s disease wasn’t too early or too advanced.

Fillit and Alzheimer’s disease researcher Rudy Tanzi said that Lilly will need to run another clinical trial, preferably a much larger late-stage test with participants from multiple countries, if it wants to bring the treatment to the US Food and Drug Administration for approval. In particular, the company would need to show the drug works, as measured by the CDR-SB score, Tanzi said.

Eli Lilly’s current plan is to run at least one more mid-stage trial. The format of that trial had already been set, but Mintun said the drug giant is discussing possible changes with the FDA, including potentially removing a significant CDR-SB change as the main goal.

Changing that CDR-SB requirement, particularly given that the original Phase 2 trial failed on that regard, would make investors skeptical of the drug, according to a note from Morgan Stanley analysts before the full results were announced.

Read the original article on Business Insider

Cassava Sciences jumps 16% on meeting with FDA paving the way for phase 3 study of Alzheimer’s treatment

brain scan alzheimer's dementia
  • Shares of Cassava Sciences jumped as much as 16% on Monday on news that it successfully completed an end-of-phase two meeting with the FDA for its Alzheimer’s treatment Simufilam.
  • The biotech company will be moving forward with a pivotal phase-three study in the second half of this year. 
  • Simufilam is a novel drug that targets “both neuroinflammation and neurodegeneration.
  • Sign up here for our daily newsletter, 10 Things Before the Opening Bell

Shares of Cassava Sciences jumped as much as 16% on Monday on news that the biotech company successfully completed an end-of-phase 2 meeting with the US Food and Drug Administration for Simufilam, its leading drug candidate for the treatment of Alzheimer’s disease. The meeting paved the way for a pivotal phase-three study in the second half of this year. 

The Austin, Texas-based company surged toward an almost 10-year high, nearing a share price last seen in June 2011. 

“For over 10 years we’ve been doing basic research and early drug development with simufilam,” Remi Barbier, President and CEO of the company said in a statement. “We believe the underlying science is solid, the drug appears safe and the clinical roadmap makes sense. We’ve crossed the Rubicon.

Simufilam is a novel drug that targets “both neuroinflammation and neurodegeneration,” the statement said.

The discussion during the meeting with the FDA, which took place in January, was supported by years of scientific and clinical data. This included positive results from a previously completed phase two clinical program.

More than five million Americans are living with the disease, according to the Alzheimer’s Association.

Shares of Cassava Sciences traded at $59.07 at 12:20PM E.T. on Monday.

Read the original article on Business Insider