Will the FDA Approve a Ketone Drug for Alzheimer's?

Years back, Accera completed clinical trials to show how diet can increase ketone levels to improve Alzheimer's. Now, this subsidiary of Nestle Foods is creating a prescription pill, performing full-blown clinical trials to prove it works.

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The Accera team believes Alzheimer’s stems from a problem with the brain’s energy supply. Brain scans of Alzheimer’s patients show their cerebral metabolism is greatly diminished as their ability to metabolize glucose in their brains decreases.

In other words, the Alzheimer's brain is being starved for energy, and Accera thinks there are other ways to nourish it. Accera is testing whether AC-1204 can help the patient produce ketone bodies, molecules that normally come from the fat reserves the body breaks down when it doesn’t get enough food. The brain will use uses ketone bodies as an energy source when it doesn’t get enough glucose. AC-1204 induces the liver to produce ketone bodies that are released into the blood stream. Some will make their way to the brain, where they are converted into energy.

Until now, doctors and dieticians have sometimes recommended coconut oil and MCT oil in dementia diets, as these foods are known to boost ketone levels in the body and brain. Some doctors also prescribe Axona, a medical food that offers a milkshake powder offering a more concentrated effect.

Winning Strategy

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Accera, an early stage drug company based in Broomfield, CO, thinks it might have a winning strategy with this new drug, AC-1204. Accera is moving beyond its origins as a “medical food” company and is remaking itself into a pharmaceutical company. It wants to use the expertise it developed creating a special supplement for Alzheimer’s patients to create what it says will be this first-of-its-kind drug that attacks Alzheimer’s in a new way.

It’s a big step for the company, which since its founding in 2001 has raised about $155 million, according to its SEC filings. The money comes from investors such as Nestlé, the Swiss food company best known in the U.S. for its candy brands.

The president and CEO of Accera is Charles Stacey, a founder of a medical device company, a surgeon in the United Kingdom, and a senior investment advisor. Stacey said there are other potential adantages of this new drug. For example, there is also some evidence suggesting ketone bodies could protect nerves from damage.

In an interview, Stacey said AC-1204 has the potential to be a much-needed new option for doctors because it takes a different approach to treating the disease and slowing the progression of symptoms.

“There have been no new treatments in 12 years,” Stacey said. The failure rate in clinical trials of potential drugs “has been astronomically high,” with a study in Alzheimer’s Research and Therapy finding that 99.6 percent of the 413 drug trials conducted between 2002 and 2012 failed. That’s led to a rethinking of what causes Alzheimer’s and how new drugs should work.

For the past couple of decades, Alzheimer’s researchers and drug companies have focused on the beta-amyloid proteins that stick together to create the plaques that characteristically form in the brains of Alzheimer’s patients. Many researchers have believed the plaques are the key culprits in damaging the brain, but drugs aimed at clearing the plaques once they’ve developed have not helped patients. Those failures have prompted efforts to intervene much earlier in the course of disease, which is no easy task.

It’s also not proven whether beta-amyloid itself is a culprit or just a byproduct of Alzheimer’s damage, so researchers continue to go after other biological targets.

What Should We Be Targeting?

“In the past few years, people have begun saying, ‘Maybe we have this wrong, maybe we shouldn’t be targeting amyloid, what else should we be targeting?’” Stacey said. “There has to be a new target out there, and that’s where we come in.”

Too many ketone bodies in the blood can be a severe medical problem, but Stacey said the amount generated by fasting or a low-carb, high-fat diet does not create complications. Accera has conducted studies that found a correlation between circulating ketone bodies and improved cognitive performance, which Stacey said supports the hypothesis that the brain can shift to ketone bodies as a consistent fuel source.

Phase III Clinical Trials

Accera has completed enrollment and is conducting  a Phase III trial of AC-1204, with 480 patients from across the U.S. Its endpoints will examine patients’ performance on memory, cognition, and global function as measured by commonly used tests.

AC-1204 isn’t Accera’s first attempt to treat Alzheimer’s by improving the brain’s metabolism. The company originally developed and sold Axona, a so-called medical food. Accera said it worked by increasing the supply of energy to the brain, much like AC-1204. Axona was a dry powder that users could mix with milk or other liquids and drink once a day.

Axona required a prescription and had to comply with truth-in-labeling regulations. Accera did not need FDA approval to sell it, but the company conducted clinical trials anyway to establish its credibility with doctors.

Axona gained a following among doctors, and Accera said it was used by more than 35,000 Alzheimer’s patients in the U.S. Investors bet big on the company, and Axona. Although Stacey declined to say how much the company has raised, SEC filings put the total around $155 million. That includes $84 million the company raised between 2012 and 2014.

Nestlé itself invested directly in the company, with its Health Science division reportedly acquiring a 60 percent stake in the company in 2012 for an undisclosed amount.

Most of the investment came when Accera was developing and then building up its sales force to market Axona. Accera will continue to produce Axona for existing users, but its focus now is becoming a true drug development company and commercializing AC-1204, Stacey said. That’s keeping true to the company’s long-term practices.

“We have always put a very strong emphasis on clinical development,” he said. “This is a new iteration of that.” Switching to a more traditional approach to drug development, Accera “will have access to more patients and allow us to have more impact on this disease,” Stacey said.

SOURCE:

• Accera, Inc.

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