A biotech founded by a 32-year-old has a shot to launch 'the first new Alzheimer's drug in 15 years'

• Alzheimer's affects 5 million Americans, a number that's expected to balloon to 13.8 million by 2050.
• On average about 99% of all Alzheimer's drugs in clinical trials never actually make it to approval.
• Axovant is hoping its Alzheimer's drug will be the first one in 15 years to get approved, with key data from its late-stage trial coming in September.
• The company's also looking at how the drug works in people who have Lewy Body Dementia, and has three other drugs which are earlier in development.

The search for treatments for Alzheimer's disease hasn't been going well.

The past decade has been filled with failures, with 99% of all Alzheimer's drugs failing in clinical trials. There are only four approved drugs that treat the symptoms of the disease, and the most recent drug approval happened in 2003.

That could change if Axovant, a company with a 32-year-old founder, gets positive results in a late-stage trial for its Alzheimer's drug in September. If the results are good and the FDA agrees, the drug could be approved by 2018.

"It's remarkable that we have a shot to become the first new Alzheimer's drug in 15 years," Axovant CEO David Hung told Business Insider.

The drug has an unusual backstory: GlaxoSmithKline sold intepirdine to Axovant in 2014 for $5 million. At GSK, the drug had done poorly in clinical trials, with one expection: when the drug was combined with donepezil, an Alzheimer's drug currently in use. Taken together, the two seemed to slow the loss of cognition and ability to perform daily tasks more effectively than when donepezil was taken alone.

Axovant, which is one of a number of companies in the Roivant family founded by 32-year-old Vivek Ramaswamy, latched onto those results, and decided to keep pursuing the drug. After getting positive results from a phase 2 trial of intepirdine, Axovant launched a phase 3 trial in 2015, which sets it up to go before the FDA. The results of that trial are expected in late September.

How intepirdine works

There's been a lot of debate in Alzheimer's research about two possible ways to tackle the disease and possibly reverse its effects: by targeting and trying to remove either the amyloid-beta plaques or tau protein tangles that build up in the brains of people who have Alzheimer's.

Axovant's drug doesn't deal with those hypotheses at all, however. Instead, intepirdine works on something called a 5HT6 receptor as a 5HT6 antagonist.

Hung compares it to a glass of apple juice, with the juice signifying acetylcholine, a chemical in the brain that transmits signals, and the neuron serving as the glass. In healthy humans, that cup is full. But in people with Alzheimer's, the cup is maybe a quarter of the way full, and it's leaking.

Donepezil, a cholinesterase inhibitor and the drug that Axovant is pairing with intepirdine in its trials, works by trying to keep that quarter cup of apple juice from leaking out quite as fast. intepirdine, on the other hand, adds more juice, Hung said. "If Mindset [the trial set to read out in September] hits and the drug gets approved, it'll be the first drug ever approved that adds more juice to your cup."

Why it might not be the 'breakthrough' people are looking for

The goal of the drug is to treat the symptoms associated with the disease, such as cognition and behavior changes. intepirdine, as far as researchers know, can't reverse the effects of Alzheimer's or cure the disease. More realistically, Hung said he's hopeful they might find a disease-modifying effect, which could push back the timeline on the disease.

Axovant isn't the only drug that's taken this approach: In February, Danish drugmaker Lundbeck said two of its trials on the company's 5HT6 antagonist had failed. In 2016, Pfizer shut down its trials on a drug that acted on the 5HT6 receptor as well.

Should intepirdine fail, Hung still has big plans for Axovant.

The company's also looking at how intepirdine works in people who have Lewy Body Dementia, a neurodegenerative disease with symptoms that look like Alzheimer's or Parkinson's. In that trial, people received a higher dose of intepirdine. It's expected to have results by the end of the year. Axovant also has three other drugs, which are earlier in development.

"Some people think that we're a one-trick pony. And I think we have seven tricks," Hung said.