Every so often, along comes someone who just determines that the game is a tad “tilted” and decides not to belly up to that table. On second thought, decides to construct a new game of their own making and constructed in such a level way that public good ranks right up there with making a fair profit.

The game in question is Big Pharma, of course, which everyone knows has manipulated the research, manufacturing, and sales of life-saving medicines to generate as fat a margin as can be mustered – public be damned, and shareholders be pleased.

As Barbara Handelin, founder and CEO of Audacity Therapeutics explains it, developing medicines is a high-risk investment and high risk demands a high return. It makes sense for Wall Street to encourage the development of medicines for a select few diseases that can satisfy its investors.

Pharma companies have to focus on either very large markets (common diseases) in chronic conditions (e.g. high blood pressure or cholesterol) where billions of pills can be sold at good profit margins, or as above, on bespoke medicines in small patient groups where a single lifesaving medicine is very expensive per patient.

So, what’s her company’s problem with these choices?

For one, she says, “We have developed drugs for only 10 percent of the roughly 6,500 human diseases we know about – and at increasingly stratospheric prices.

I feel the need for greed

(Forgive my dragging Top Gun into this, but the comparison to Top Dollar was irresistible. The comparison strengthens when you realize that our high-flying friends in Pharma want to stay “up there” – cuz that’s where the money is. There is no incentive to be found in drugs priced below the stratosphere.)

Once upon a time, biotech companies set out to radically redefine human disease and treatment from the DNA on up and bring medicinal chemistry (pharmaceuticals) out of the dark ages. So far, so good.

As Dr Handelin describes it, the problem was the evolution of biotech financing which has established a general expectation of spectacular returns in this industry. This expectation is set by the sources of investment capital available to build these companies, from private (e.g., Venture) seed capital, seeking extraordinary ROI, to shareholder pressures in the public markets.

She sighs, “No one seems to have asked a basic social question: should we be generating huge profits in the medical industry?  Or is medicine one of those sectors that is too essential for our basic welfare to allow for financial ‘gaming’ here everyone but the patient is financially rewarded?”

The Audacity alternative

“What if there were biomedical and pharmaceutical companies with a primary purpose and duty to make as many affordable medicines for as many disease conditions as possible?” she asks.

Companies like these might develop therapies for untreated or under-treated human diseases, focus on disease solution opportunities rather than 10X market opportunities, even guarantee that drugs are produced at prices affordable and accessible to all patients…worldwide!

Some of these prospective diseases and drugs were discussed in a recent KPFK fm Los Angeles interview with broadcaster Cary Harrison – Multiple Sclerosis, PTSD and autism.

“Members of the Audacity team have developed evidence that an old anti-HIV drug can be extremely effective in treating multiple sclerosis,” she reveals, “but traditional biotech investors will reject this opportunity because it undermines the high profit $60B multiple sclerosis market by introducing an effective competitor that needs no market exclusivity (patents) and has lower cost to develop.”

A new class of investors

“Funding for drug research and development just described has to come with an expectation of a more modest return,” Barbara says. “Think of it as ‘I want to put a part of my 401K into, umm, bonds and I am going to make a nice return over a long period of time.’”

To facilitate and hasten the day when that sort of investor can make that sort of informed decision, Dr. Handelin and her team are forming a Commission on Financing a Public Benefit Biotech Industry (CFPBBI) to meet over the next several months to produce that blueprint.

Out of this will come a critically needed investment model which rewards the heart as much as the wallet – and not a moment too soon.

In a world where medication costs are skyrocketing and pharmaceutical companies search out ever-higher profit, KPFK’s Cary Harrison interviews Barbara Handelin Ph.D. of Audacity Therapeutics. Barbara talks about how repurposing existing medications can bring down costs while treating a broader range of illnesses with possibly greater therapeutic effect. You can get in touch with Audacity Therapeutics by calling 888-440-2520.

Transcript

Cary Harrison here with you. And I want to welcome you to this special program because today we're going to talk about a solution to a series of issues that plague pretty much everybody, you know, has one of these or the other, whether it's autism, whether it's PTSD, whether it's Alzheimer's disease and people feel hopeless, they feel limited.

They're not sure where to go. And medically speaking, you know, there's cures on the horizon. There's a lot being done, but did you know that existing pharmaceutical medicines, existing ones could be repurposed to actually attack these very conditions and many, many others. But unfortunately, the industry, as we all know, is not poised to allow that yet.

There are some brilliant scientists out there who have found a new pathway. One of them is Barbara Handelin. She's with us today. And Barbara, you are what we would call a superhero because what you're doing is bringing to the fore, the possibility that we can have existing medications, we don't have to invent brand new ones, but we could repurpose them to actually change the course of medicine.

Barbara Handelin: Yes, that's right. But as you said in your introduction, that's not happening. And it will never happen until, and unless we change how we incentivize our industry and we all know that whatever we incentivize is the behavior that we get. And right now our biotech and pharmaceutical industries are heavily incentivized to maximize profits, as opposed to maximizing the number of diseases that we can be treating and at what cost.

Barbara Handelin, about 75,000 children are going to be born in the next four years or so, we're going to find out that their brains are developing into an autistic profile. Uh, this is horrifying for the amount of people that are born in the United States and around the world with so far limited possibilities.

Tell us a little bit about what your plan is to change the nature of medicine.

Barbara Handelin: Well, we have, uh, an interest and an opportunity, uh, to stand up a young company that has been diligently working on trying to understand what autism actually is, you know, very today when we just diagnosed with them by behavioral patterns.

Barbara Handelin: And those are hard to pick up on until a child is, you know, 3, 4, 5, 6, 7, 8 years role as speech and various more normative social behaviors, um, start to fall away. So this company took the attitude of maybe we could understand what the actual underlying biochemistry of autism is. And if we could, then we'd be able to take a blood sample or urine sample.

Barbara Handelin: And instead of guessing, based on behavior, you could make a diagnosis and you could do that really early. While that child's brain is going through its major developmental processes, and you would have an opportunity to intervene using even the behavioral, uh, methods that are being used today, which we know are effective, especially the earlier we start.

Barbara Handelin: So this has been a lot of science and technology to figure all that out. The company actually poised to bring this to paper.

So I'm guessing there is a price tag to this. We hear from the pharmaceutical companies that the whole reason they can't actually really help us is that it's impossibly expensive, the research and development, um, the yacht payments, whatever it might be.

Barbara Handelin: Um, the fact that, you know, the industry spends at least 50 cents of every dollar in sales and marketing, but you know, we're, none of us are standing around at the pharmacy saying, oh, I, I don't know. Do I want this right? This brand or that product? No. You know, in sales and marketing, they teach you, you need to get the prospective customer to the point where they feel they must have your product.

Barbara Handelin: Well, we walk in, must have it.

I have a friend, Barbara, in Switzerland who works for Novartis and they make Claritin. And I said, well, you know, if this were the US, we would simply fly a crop duster and put in all kinds of allergens in the welfare crop dusting and give everyone hideous hay fever for a thousand square miles and then run Claritin ads.

And he said, that's brilliant. I said, no, I'm not, I'm not recommending it, but that's how we do it in the states. He goes, I don't think that's legal here. I said, good.

But to your point 50% goes to sales and marketing. It's like, it's like having an insurance company, you know, a third entity in that doctor, patient insurance company. Like what are they doing here? Well, they're taking their cut. Yes.

Barbara Handelin: Yeah, no it's um, so, uh, the, the numbers, you know, the numbers say yards about why we are aware we are.

But you've found a new way around this through a public benefits company where the public benefits, Hey, wow.

Um, but you have to raise money for it.

Barbara Handelin: Yeah, yeah. That's right. That's right. And the existing investor base for all things, medical, uh, is very expensive. Capital comes from starting with venture capitalists who are in the business of returning very, very exciting returns to their investors. So we need to be free as a company like ours.

Barbara Handelin: We need to be free to say notes and thanks to that kind of capital, because we want to work on medicines and diagnostics that are going to give us good margins but that are not going to produce the kind of outsized profits that are required, um, for investors, including wall street.

The three biggies that you're looking at right now are PTSD that would cover virtually every single military person who has returned from Iraq, from Afghanistan, from any other, uh, battlefield of conflict.

Going back to the Vietnam war, going back to world war II, the Korean war and wars regrettably, yet to come, they come back with severe PTSD. Uh, they're incapable of often carrying on even basic tasks. There they have difficulties having social skill sets, um, romantic capabilities anymore. And it's such a horrific and crippling disease, PTSD invisible as it is for you to be able to offer real solutions to that helps at least 10% of America right off the bat.

Autism helps at least 10 to 20% of America right off the bat. Multiple sclerosis helps again, a huge number of Americans right off the bat. What does it cost for you to put on one of these clinical trials so that we get an understanding of what investors might be looking at?

Barbara Handelin: Well, um, the cost for us to engage in clinical trials for any of those and the myriad of other, um, diseases disorders that we are looking to address with existing medicines are costs for significantly less than what we will hear in the general industry.

Barbara Handelin: And that's because we are extracting and capitalizing on the fact that these medicines have been trialed. Some of them have actually been used and prescribed in general practice. They may even have gone all the way to being a generic drug. So we know their thing, um, or we know what their limitations are.

Barbara Handelin: And now we are simply saying, let's use that same drug compound. We've invested a lot. Somebody invested a lot of money previously. Let's use that same thing and try it in a new indication. For new important indications.

So I'm going to make an analogy one that's popular, at least with the menfolk Viagra. Well, happy Viagra.

Viagra is now generic. You can get it on TV. You can get it in magazines. You can get it from the doctor of your choice. You don't have to explain, you can just get it. And that's because it's generic and you can get it for cheap. And we know it works. We know it's safe. We know how it works. Everything is already all figured out.

So you're taking something, for example, like a Viagra, which everyone understands, which is universally accepted and you're saying, but you know, it also does something else. It also helps lower blood pressure. I think it actually does. Um, and some others. Okay. Okay. And so we're going to actually focus it on lowering blood pressure and we're going to do it at a 10th of the price as it would ordinarily be.

Well, that's a big win for people with high blood pressure, like most Americans. Um, that would just be a made up example, but that's what you're looking to do for your big three right now, multiple sclerosis (MS), Autism and PTSD. It’s finding ways like this, where you can make major changes at affordable prices.

Barbara Handelin: Correct. So for example, multiple sclerosis, uh, members of the audacity team have developed evidence that an old AIDS drug, an old anti-HIV drug can be extremely effective in treating multiple sclerosis. And you know, why do we care about that? Jeez, there's 60 other medications that are on the market right now for multiple sclerosis.

Barbara Handelin: But if you talk to people who have multiple sclerosis, most of them will tell you my disease is inadequately treated. I keep progressing. And some of my symptoms, like my debilitating fatigue ruining my life and I'm getting worse. That's an enormous market for a number of companies. So do you think they have an interest in picking up an old generic AIDS drug and letting that come on the market as a potentially more effective treatment for multiple sclerosis?

Barbara Handelin: Certainly not. And the investor base who would be wanting to invest in some exciting new cure for multiple sclerosis, they say, well, you're not going to be able to make that kind of, that kind of profit on an old drug. So therefore that investment doesn't flow in. Cary, we need a new class of investors.

Interesting question here, new class of investors, they get a major bang on return.

Plus they get to help the human race of which they are members personally. Watch out of that part. I mean, it's got so much upside that it's absolutely crazy. Um, these investors get to participate in that and we get to repurpose drugs that most pharmaceutical companies could literally not give a darn about.

As far as they're concerned. There ain't no more profit. The patents have expired. See ya. You're sitting on a treasure trove of dynamite data and information that can repurpose these things into nothing less, than magical cures. On your staff, on your team, part of your collective, part of your collaborative, uh, organization is a woman who was instrumental in us, understanding TB, tuberculosis, and getting very close to that cure.

Barbara Handelin: Oh, that's a, it's a great example of, um, a medicine that will come and we will be treating tuberculosis around the world, but it has taken 25 years of slaving under always underfunded conditions to eek forward the scientific basis and then the clinical trials, which are happening outside the United States states, because we don't have a lot of TB here.

Barbara Handelin: It will come to, to be, but it could have come to be, at least a decade ago, had the sufficient amount of funding been made available. But that funding, Cary, has to come with an expectation of simply a more modest return. Think of it as I want to put part of my 401k into, um, bonds, and I'm going to make a nice return over a long period of time.

Barbara Handelin: I will let my money sit, it's going to get invested in all the medicines we actually need and I'll get a nice regular return.

Barbara Handelin of Audacity Therapeutics is on an important mission to change the way in which funds can be raised for drug development and/or repurposing to meet the needs of the 90 per cent of us who have a condition with no effective treatment. She and her team’s work may likely change an industry.

What I am talking about here is a change from focusing on headline-grabbing “blockbusters” engineered by Wall Street/Pharma,  to instead developing products to treat any disease based on huge medical impact vs market value.  ( much less profitable…but, nonetheless much needed.)

Given she is a woman with a Ph.D. with significant credentials as a pioneer in her field, is it proper to portray her in a cartoon? To place her important work and mission into a mini graphic-novel? A piece of art that appears as a circular mandala exhorting in an almost spiritual way why change is needed in the business model in her industry?

Yes.

If for one reason alone, the ability of a cartoon to get complex messages across in a visual fashion that is simple, colorful and impactful is undisputed. Otherwise, consider the near-impossibility of rendering 40 years of professional and industry experience and scholarly writings into a medium that doesn’t require its audience to have an advanced degree. But, to still come away educated and appreciative.

As any schoolteacher can attest to, the interest generated by a cartoon in its reader can often lead that person into a deeper dive and more traditional way in which to get educated and then take action on that learning.

So, take a tour of Barbara’s professional life and see if you can identify with the purpose and importance of her mission.  You do? You can reach her directly at This email address is being protected from spambots. You need JavaScript enabled to view it.. Before doing so, you might want to read her article titled “ We can have our medicines and afford them too.”

After earning a Ph.D. in human genetics in 1987, I chose to enter the nascent biotechnology industry rather than pursue conventional academic science. Venture capitalists in Cambridge, Mass, and elsewhere had caught wind of revolutionary genetic discoveries in university labs and academic medical centers. They saw a new gold rush opportunity.

The credo, then and now, is to bet big money on ten startup biotech companies, and if just one develops a high valuation in a buy out or IPO, presto! Instant millionaires! While saving patient lives too!

I joined a small army of scientists, engineers, patent attorneys, and young CEOs who set out to radically redefine human disease and treatment from the DNA on up. We knew that once we delineated the molecular pathway of any disease, we could prevent it with early detection and target treatment right at the root cause; ‘targeted therapeutics’ like the “Silver Bullet” for cancer, remember? All this transformation was enabled by loads of venture capital. Those were heady days. We intended to bring medicinal chemistry (pharmaceuticals) out of the dark ages. I contributed to early game-changing products and technological innovations. Some of the early companies and products are still impacting people’s lives today.

Most public life science companies back then and today have zero revenue. That’s right, companies trading on NASDAQ value their stocks based on intellectual property (patent applications) and potential revenue. These are high risk investments. It seems only right that those early investors in life-saving technologies would or should expect high returns on these investments. Some of the early investors became, and are still becoming, very rich from IPOs and acquisitions.

My colleagues and I asked, so what is wrong with that? In fact, as scientists, we felt that we should be well-rewarded for our role in inventing life-changing medicines, as some of us did.

There’s a but…

This 35 year-old biotechnology funding model should no longer be exalted because it’s now working against our collective interests.

Despite a tsunami of molecular knowledge generated by academicians and industry researchers concerning human diseases, we have developed drugs for only 10 percent of the roughly 6500 human diseases we know about — and at increasingly stratospheric prices.

I attribute this to two significant and important facts:

1. About 90 percent of human diseases are not addressed with medicines today.
2. any medicines that are developed are made available only because they can be priced for profit, not for the larger human needs they address.

We perverted the purpose of biotech companies by how we financed them. As is true in life, so it is in industry. You get the behavior you reward. And the process is addictive. It starts with a new company accepting high-cost early venture capital funds, followed by private equity and bankers doing IPOs and mergers and acquisitions, and finally the intoxicating experience of arriving in public markets. Everyone is financially rewarded. Except the patient.

High-cost capitalization

The behaviors incentivized by what I call “high-cost capitalization” include:

• Maximize prices, especially if the drug is novel or rare. David Axelrod saw something upside down in that thinking as he tweeted, “Your money or your life is a hell of a choice that people shouldn’t have to make.”
• Patient populations are framed as “markets” to entice the discovery of costly blockbuster drugs.
• Ignoring prevention and early detection technologies because they are not blockbusters.
• Trade secrets and price preservation are pillars of the industry
• Development and purchase of potential cures can be shelved to protect companies and shareholders if the profit potential is inadequate.
• Generated profits are used contrary to the purpose of furthering healthcare research. Only 7-15 percent of profits are reinvested in R&D for new products; the rest goes to executive pay, stock buybacks, marketing and sales.
• Lean efficiency is not a standard. As of 2022, it takes 10-15 years and about $1-3 billion to commercialize a new drug, positioning pharma in the top three most profitable industries on the planet.

Wake up!

In 2019, some colleagues and I said, “Wait! Are our labs in the business of forming companies so that we can ring the bell on Wall Street, or in the business of creating medicines that heal people? And if we want to work on any opportunity for improving lives such as repurposing old drugs for new disease targets (frowned upon) or any preventive or early detection test (no money in that), how could we ever get financed?”

Sometimes events collide with ideas.

COVID happened. We saw what our industry could do when it addressed an urgent need, as opposed to targeting a “market.”

We saw companies choosing to make low-profit vaccines, and doing it within a single year. Not for $3B! We saw how testing companies were immediately funded to create PCR and home tests to meet a healthcare need, not a marketing plan. They found ways to capitalize that effort — for the public good. We can now see that placing health above profit is achievable – and desirable.

We saw an effort to share all data and technical know-how globally. Government and private industry worked together to meet this emergency. COVID vaccines and therapeutics have remained private intellectual property, not placed in the public domain as were the polio vaccines in the 1950s. Nevertheless, with taxpayer support we now can have all the medicines America needs. And we can afford them too.

Is this socialized medicine?

If you are talking about the industry sector in which my company is located, then no. It’s “conscious capitalism.” Audacity Therapeutics does not need charity, nor government programs, nor social or public services. We believe that private industry can and should be delivering on the public’s need for medicine.

But we do need some rethinking to bring that about.

We need more biotech and pharma companies, like ours, that are organized to operate differently. Companies that are financially able to say “No Thanks!” to high-cost capital.

Audacity Therapeutics is so-named because we had the temerity to ask what if there were biomedical and pharmaceutical companies with a healthier primary purpose and duty that could:

• Develop effective therapies for untreated or under-treated human diseases, as many as possible.
• Demand capital efficiency and a culture of deliverables, not promises
• Focus on disease solutions opportunities rather than 10X return market opportunities
• Prioritize medically valuable products for patients rather than capital value to financial markets
• Guarantee that drugs are produced at prices affordable/accessible to all patients worldwide.
• Provide fulfilling jobs and contribute to a balanced and just healthcare economy

From this ethical framework, we aim to prove that quality healthcare can be both profitable and affordable.

For more details, visit AudacityTherapeutics.com