Big Pharma Abandoned Alzheimer’s, This Good Unicorn Stayed And Found A Potential Cure

The biggest companies in the world gave up.

Wall Street wrote it off as a lost cause.

17 years and hundreds of billions of dollars later, a potential cure for Alzheimer’s has finally been created...by a Good Unicorn.

Let’s dive into the deep end with Martin Tolar, Founder and CEO of Alzheon.

Diana Tsai: A pill to stop Alzheimer’s. Is it real?

Martin Tolar: Yes. We’ve developed the first oral pill with the potential to slow or even prevent the disease.?

Tsai: Does the pill slow, stop, or reverse Alzheimer’s??

Tolar: Since our pill is attacking the initial toxic insult that leads to Alzheimer’s disease, we believe it has the potential to stop the disease in its tracks. The favorable safely profile of our treatment allows such early intervention, years and decades before the brain damage results in clinical onset of the disease. Since the main and overwhelming risk for Alzheimer is aging (one third of 80 year-olds have clinical disease), everyone over 40 or 50 could be a candidate for treatment.

?That said, for approval, we must follow a path of evaluating the effect of our pill on the disease in symptomatic patients, because this is where we have clinical tests & tools to evaluate the efficacy. Our current Phase study, which we believe could lead to approval, is conducted in patients with early Alzheimer’s. Preventive Phase 3 study will come next, also aided by effects we have shown on biomarkers of the disease in our ongoing Phase 2 biomarker study.

Tsai: How did you make these strides when so many have failed?

Tolar: Well, my colleagues and I have been at the forefront of Alzheimer’s research for over 20 years, working for many of the companies who have failed over the years. Our team at Alzheon of?about 150 people includes leading scientists and clinicians. So we’ve really had some of the most talented people in the world working on this for the last decade.

What we discovered was the reason why we and so many others in the past had failed: because we focused on the wrong thing. We focused on trying to solve the problem of the amyloid plaque that forms in Alzheimer’s. When in fact, the key to solving Alzheimer’s is inhibition of toxic soluble aggregates of amyloid protein, that forms during earliest stages of the disease and drive progression.

So, we developed a pill that’s able to block the formation of this toxin in the first place.?

Tsai: Whoa. And it’s working?

Tolar: We’ve seen compelling results already in Phase 3 studies that involved more than 2,000 patients, far outperforming anything that’s ever been developed. Now we need to confirm this data in our ongoing APOLLOE 4 Phase 3 study.

The implications are huge. What we’re talking about here is an opportunity to help millions of patients and their families. There are 6 million patients in the US and 35 million worldwide for a disease that currently does not have any safe and effective?oral treatments that can change the course of the disease.

Tsai: I really want to hear the story about how this came to be. How difficult it was to find the cure. Can you share how hard it was to get to this point?

Tolar: You have to realize that trying to solve Alzheimer's has been the worst idea for investors for decades. Hundreds of billions of dollars have been lost trying to figure this out. The biggest companies in the world, Pfizer, Bristol Myers Squibb, gave up and abandoned the space. It was too risky for them, too expensive.?

We had to go completely against the common wisdom, against everyone’s worst fears. Including Wall Street, who totally avoided us. That was one of the hardest things for us, working on a solution in a space where no one wants to invest. I mean statistically, this is literally the worst business investment ever, with more than a 99% fail rate. Who wants to put money into that?

Tsai: Wow. And so, question for you, how did you survive as a company? How did you raise money when no one wanted to fund this?

Tolar: We were able to finance through people who believed in us, in our science, mostly private investors, in the beginning. But now we do now have investments from large institutional funds, especially given the data in the last year, there’s been a full 180 degree turn as investors have realized the potential of this space with the new data from our recent trials.

Tsai: Medicine is so different from the quick feedback loops of building software that traditionally define Unicorns. How did you keep your faith and belief through all these years as you’ve been refining, working on the solution?

Tolar: Well, I was born and raised in Prague, in the Czech Republic. I came of age during the revolution against Communism, I mean, I was part of it, and with my fellow students we started and led the Velvet Revolution that overthrew the communist regime in Czechoslovakia. And then basically my friends and I, a bunch of 20-year-olds, helped write the new laws for the country, form the new society, the new government. So, after doing that, the world was our oyster, it gave us wings to fly, you know??

I came here to the United States with one suitcase, over 20 years ago. And compared to that entire journey of overthrowing the Communists and building the new society of Czechoslovakia, starting a new life in a new country with nothing, solving Alzheimer’s was a problem that didn’t feel so daunting.

This has been an incredibly exciting time right now, for people like me, who work in this space as clinicians and scientists to be at the forefront of a major scientific breakthrough.

Tsai: How can non-science people support critical scientific missions like yours?

Tolar: Fund the unorthodox, the solutions outside of the mainstream. Private funding is what’s given us the ability to stay independent and work at Alzheon on this problem for the last decade. And our independence has allowed us as a company to focus on what’s best for the product, the patients, rather than returning immediate profits to VCs.

Tsai: Are you saying that focusing on what's best for the product doesn't always line up to what's most profitable in the near term?

Tolar: Yes, you said near-term, that’s right. Long-term, product vision might align with profitability. But near-term, often does not. I've been through this with my previous companies, you lose that ability to do what’s best for the company and for the product and for the science, because you lose that independence, the moment you give someone else ownership of the company who wants a quick return.?

Tsai: Powerful perspective. What would your advice be to entrepreneurs who want to build the next Good Unicorn?

Tolar: I’ll just say, we never thought about being a Unicorn. It’s never crossed our minds. We’re physicians and scientists, coming from a situation where we’re trying to solve this seemingly impossible problem. You cannot plan for commercial success in a situation where the success rate for drug development is a fraction of one percent, that’s a near perfect fail rate.?

I guess my advice would be, pick a big hairy problem and just try to figure it out. Try to stay independent. Focus on the truth, the data, the rest will come along. Stick with it and you might just build something no one would ever believe is possible.

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