Business Of Biotech
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Business Of Biotech
Risk Mitigation With Meira GTx's Zandy Forbes, Ph.D.
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Zandy Forbes, Ph.D. learned about business risk mitigation in the riskiest of business environments. She was a public markets healthcare investor on Wall Street for well more than a decade. Her hedge fund experience followed the Human Frontiers/Howard Hughes postdoctoral fellowship at the Skirball Institute of Biomolecular Medicine at NYU Langone Medical Center that was preceded by an Oxford Ph.D. in Molecular Genetics. Since 2015, she's been drawing from her immersion in both the business and science disciplines as President & CEO at Meira GTx. On this episode of the Business of Biotech, Dr. Forbes and I go into deep analysis mode as we examine the risks she's taken in her current role, and how she applies her experiences – both good and bad – with handling other people's money to building a biotech company.
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Welcome back to the Business of Biotech. I'm Matt Pillar and since talking with today's guest a couple of weeks ago, I've been so looking forward to getting back together with her to record this episode. She's a brilliant biotech risk taker and I don't use that combination of words lightly. I describe her that way because her scientific, academic and postdoc work, married with well over a decade of investment banking work, serve as the foundation of that brilliance which she's now leaning into when weighing risk as a biotech builder. Her name is Dr Zandy Forbes. She's the president and CEO at Meira GTx and on today's episode we'll get to know her and we'll learn about some of the bold fundamental decisions she's led Mira through on the company's way to building a very deep pipeline of gene therapy candidates across the clinical spectrum and doing the development and manufacturing of those candidates, I might add, in-house. Dr Forbes, welcome to the show.
Zandy Forbes, Ph.D.:Thank you so much. And what a lovely introduction. Thank you.
Matt Pillar:Oh well, I appreciate it and I mean every word of it. You know, it strikes me when I was thinking about that conversation that we had a couple of weeks ago. You know, I kind of went long on the setup, like you know, this is what the business of biotech is all about. This is what we're after. I think I asked you one question and your passion, enthusiasm and intelligence just took over. I mean, I was super excited After doing those pre-briefs. I know like this is going to be a great episode. Not to set the bar too high for you, but I've been excited about this one. By the way, do you go by Myra GTX or is it Myra Gene Therapeutics?
Zandy Forbes, Ph.D.:GTX is fine. Whatever people are willing to say is fine.
Matt Pillar:Very cool. So, as I mentioned, you've got you know from the outset, right. If you look at your CV, your academic record, if you will, you have the makings of what looks like an academic career. You have an Oxford PhD in molecular genetics. You did postdoc fellowships at the Skirball Institute of Biomolecular Medicine at NYU Langone. You did fellowships at Duke University and the Carnegie Institute at Johns Hopkins. After your PhD, it looks like this woman is setting herself up for a career in research and academia. And then in 2000, not long after that, you became a healthcare investor at the Hedge Fund Civic global healthcare yes, investor at the hedge fund civic global healthcare yes. So two questions wrapped up here. One, when you were doing your PhD and doing that fellowship work, your postdoc stuff at that time, in that moment were you thinking academia, research? So that's part one of the question. And then part two of the question is at what point were you exposed to and did you embrace investment banking?
Zandy Forbes, Ph.D.:so my academic career really began as an undergraduate at cambridge and I um, and at the time I was courted as a person who had very good grades in my part ones. I was courted by the newly deregulated City of London city of London and I used to receive letters because we didn't have emails. Then, dear Mr Forbes, please will you consider coming for an interview as a salesperson. I went for one interview where I imagined myself it was for a commodities trader and I knew nothing about Wall Street trading anything. So in my mind's eye I saw myself, my future, as the woman from Del Monte who travels the world and sources and provides the world's commodities. I got to the interview and they asked me about sales and trading. I didn't know what any of these things were and I failed. I had another interview in response to the Dear Mr Forbes letters, and so when I finished at Cambridge and so when I finished at Cambridge, I got a very high grade degree I didn't really know what to do because that seemed to be where people went or where people were courted to right Big city for Goldman and those sort of places year off and ended up finding a topic that was super fascinating and super unknown, which is how does one cell develop into an embryo where every gene is only used in the right cells?
Zandy Forbes, Ph.D.:What is positional information? How does life go from one cell to organisms? And I found someone who was at Oxford to do a PhD in this subject in fruit flies where a screen had just been done that identified every gene that was required from an egg to make a fruit fly, and so that, to me, was a huge. It was a universe of interesting undiscovered things. And I went and I did my PhD and when I finished the PhD I happened to have worked on a gene that was one of these positional information molecules and I was invited. I didn't know what exactly to do. I was invited to do visiting professorships at Hopkins and Duke. So I didn't have a plan. I never had a plan and that is a failing in my life. I feel I would have been much more successful as a particular thing had I had a plan and pursued single-mindedly that plan, and I admire people who do that and encourage people to do that. But given I didn't have a plan, I was falling into position at NYU because I gave a talk at MIT and the woman who I met with offered me a job in her lab. This woman is one of the brightest, most inspiring scientists I know. She's called Ruth Lehman and she was actually the individual that did that screen, that found those genes, and she was moving to NYU where she was able to really broaden her lab, broaden what she did. And I was there because of her and because of her work.
Zandy Forbes, Ph.D.:And towards the end of that tenure, when I was setting up a lab in the UK, I by chance met the person, christian Sood, who at that time the year 2000, was the best performing hedge fund in the world. I didn't intend to meet him. Someone I'd been at Cambridge with was an investor and his bank had money with Christian. So I met him and at the first interview he gave me the reds for some recent IPOs Abgenix, medirex, exelixis and I was a fruit fly geneticist and I looked at them and suddenly explosion. I suddenly saw that outside academia, in biotech, people were doing everything. They were trying to discover everything, not just one gene in one fly in one lab, and that and a number of things.
Zandy Forbes, Ph.D.:I literally went back and met Christian, put out my hand and said, yeah, I accept your job, no contract, no negotiation, nothing. I went back to Ruth and I said put out my hand and said, yeah, I accept your job. No contract, no negotiation, nothing. I went back to Ruth and I said I'm going to do this and they all said you can't do it. You're so good at this. But it wasn't that. I was so good at doing research. They told me that of the postdocs and visiting professors they'd had, what I was good at was teaching. So I took a year off and spent it in these hedge funds and I never went back. But I did if I may spend one more minute, of course when I took the job at this fund, which was at the time a fund called Galleon it was very secretive, as long short funds are and particularly were at that time, still are and I used to go back to my lab to sort virgin fruit flies because you have to get them before they've mated, so you know what progeny they're going to have.
Zandy Forbes, Ph.D.:And I felt this sort of claustrophobic feeling at work. It was the first actual job I'd had and I would go back and I sought my fruit flies and I'd feel I could breathe and I could feel that my mind was flying because I could think, I could daydream about anything, and I did, at that time, realize what an incredible luxury and what an incredible opportunity to think anything you want being an academic is. Would I have kept going as an academic? Yes. Was it my plan? No, I rather abruptly took an opportunity that I knew nothing about, which was incredibly hard for me.
Zandy Forbes, Ph.D.:When I then ended up doing it both emotionally like this being confined for the first time um, both emotionally like this being confined for the first time you're paid for how you're going to use your mind um, but in that job I learned more than I'd learn in 10 years of academia, in every day of school and university, about medicine, science, people, businesses, incentives, science, people, businesses, incentives and failure. Because if you're an investor, you really really feel the failure of everything you invest in because you are responsible for making that money. And it's not money that it's about. It's about being wrong. So I'm wrong on an FDA panel.
Zandy Forbes, Ph.D.:I've watched so many FDA panels. You're wrong on an FDA panel. You're wrong on an FDA decision. You're wrong on a drug development trial. You're wrong on a product. You're wrong on a launch. You feel it and you remember it. I remember Genentech's panel for Avastin in breast cancer like a video right, because I was watching the faces of each person and the patient advocates and how the FDA had put together that panel and I was wrong, you know. So it was an incredible learning experience and what you also learn is when you're in a position where the money is like a really big hedge fund, everyone wants to talk to you. When you are not, no one wants to talk to you. So that contrast of how you deal with people and you deal with your own position as your position of being desired versus being not desired, it's also a real learning experience in life in general. But that's applied to how you run businesses and manage people and do deals.
Matt Pillar:Yeah, yeah, you mentioned I mean you talked about the emotional aspects of that learning and making that transition. Let me put it this way what else was in the very early days, what else was difficult for you to learn and grasp? And I ask that question because you know you're obviously an accomplished academic with a very expandable and malleable learning capacity. You know you think you could do anything. What was challenging for you to embrace and absorb and what had to change? What did you have to adapt about yourself?
Zandy Forbes, Ph.D.:So I think that. So the first year, right, the first year, I didn't know what the word short meant. I didn't know what. I'd never seen a P&M. I didn't.
Zandy Forbes, Ph.D.:I went to one week after my first year at Columbia Business School, right, so it was language. I had the language of science, which was very rapidly translated into the language of medicine, so I found that very intuitive. The language of business and of Wall Street, right, I'd never heard the words I'd like to give you a heads up, just for example. So how salespeople talk to you, I mean as trivial as that. And I kept thinking when they came, oh yeah, I just want to give you a heads up, sandy, and I'd go. I kept thinking of meerkats. You know, I'd never heard these words because I lived in a different world. It was as trivial as that, it was jargon, it was all of those things I will say.
Zandy Forbes, Ph.D.:The claustrophobia aspect related to having sold my brain to focus on things that I didn't understand.
Zandy Forbes, Ph.D.:Now, the first way I started understanding companies I wasn't taught was listening to earnings calls, and that taught me and in fact, reading 10Qs and 10Ks and I couldn't believe it.
Zandy Forbes, Ph.D.:I mean, they're just almost incomprehensible and totally repetitive when you actually read them back to back and just kind of getting a feel of a structure of how things are, as well as learning slightly what these lawyers think about when they are putting together and running companies. But Christian said to me and he was right, he said look, Sandy, you know nothing and in about a year you'll start feeling you're going to be able to give opinions on stocks. In about three years I will listen to everything you say and what actually happened is in that timeframe I took over actually being a portfolio manager of the small biotech fund. And when you ask me about transitions in what you learn and how you do things, that was the most transformative transition. Because when you're an analyst or a stock picker, the general thesis is, as a scientist, you are going to be able to have an edge by understanding the science by looking at data and assessing the risk and there is some vague truth to that.
Zandy Forbes, Ph.D.:However, when I took over that small biotech fund, I looked at the performance of the fund the year before when the market had been up a little bit and they were down. But when you look at why they were down, only because two or three of those positions had gone to zero, because one thing had failed. Everything else was okay so it was. At that moment I realized that in this industry, if you avoid disaster, if you avoid losing money, you will definitely make money, and that really informed how I was in constructing a portfolio. I also recognize that however good you are at science or however great a physician you are, or however brilliant you think you are, you will never really understand the risk of a clinical trial or an FDA outcome. You can feel, guess, it's more positive than negative. So in constructing a portfolio, we tended to take positions where we would look at not what was definitely going to happen. It was going to be positive or negative. But if it was positive for this stock, not this company, for this stock, where would the stock price?
Matt Pillar:go.
Zandy Forbes, Ph.D.:And every company has its own hindrances or supports for valuation. Where would the stock price go If it's negative? Where would the stock price go? So are you willing to take a bet and lose that percent? And that decision depends on your performance.
Zandy Forbes, Ph.D.:It depends on a lot of things, but you can do things, particularly in biotech, which are very constantly done in other markets of selling puts, selling calls, not risk, because that's all it really is for human beings.
Zandy Forbes, Ph.D.:We're really really bad at assessing risk right and formalizing it by deciding how much I was willing to lose, and that is very like my nature as a portfolio manager. I'm sure every one of these incredibly successful fund managers their personality is expressed in their portfolios like a sculptor expresses themselves in the outline of their sculpture, and that way of managing a portfolio did mean that the performance that we had was really good because we didn't lose big amounts of money. We didn't go for binary events to make 10 times the money. We incrementally made money and also, if you are intuitively curious and a bit of a Sherlock Holmes in how you think about things, interestingly understanding who's going to buy whom and strategically, what's best in an industry. A lot of the big bets that we made were on takeouts and a lot of them were right because things are obvious. Not everything that is obviously good always happens, but having a slightly more strategic view of how people and companies fit together, also gained over time, was very helpful and helpful now as well.
Matt Pillar:Yeah, yeah, and I feel like I could spend the next hour asking you questions about that period of your life, but I want to get to what's helpful now, so we're going to move forward a little bit. You spent 12 years, I think, in the industry.
Zandy Forbes, Ph.D.:About a decade.
Matt Pillar:Yeah.
Zandy Forbes, Ph.D.:More or less. I consider academia a decade, hedge fund a decade, this a decade, but there's wiggle room on both sides.
Matt Pillar:Yeah, yeah. Well, so the current decade, when you transitioned to Cadman Holdings, which has since been acquired by Sanofi, you went there as SVP of commercial operations from investment banking. What was the story there? It sounds like you got to a point where you embraced the role, were comfortable with wrong. See, this is one of the things that, when I said we could talk for much longer about your time in investment banking, one of the things I wanted to ask you, which you can reflect on before you get to the Cadman Holdings story, is the difference between wrong as a research scientist, that academician in science, the difference between being wrong there and being wrong as people frequently are in investment banking. I want to stick with that question. Do you want me to answer that question? I do, I do. Yeah, I promise that we move on to cabin holding, but I need to know.
Zandy Forbes, Ph.D.:I will answer it super simply. You're never wrong in science ever because you're finding out something. That's true or not. So if what you thought is wrong, science and I do think that sometimes in science you know where things do go wrong is when people think they're right, and if what they show is not supportive of what they think, they consider it wrong.
Matt Pillar:I do.
Zandy Forbes, Ph.D.:But as a matter of science, right, science is fact-based and if you're proved wrong, that's part of the scientific process, whatever science it is, whether it's cell biology or genetics. However, the complete antithesis is true in public markets trading, there is only one measure of being wrong or right because you're using that market to make your clients money. So, however right you are from a scientific perspective, if the position you've chosen to take ends up being completely wrong, you are wrong. You made a mistake. Now, was it your fault? None of it matters, and that's one of the reasons, from my perspective, that I found it a place of learning, because you are obviously wrong. The stock's up or down, you're wrong or right and it's not, you're wrong like a VC in five years. You're wrong that minute and you deal with it. I remember when Tysabri was pulled right Because I'd worked for a whole year on the MS market.
Zandy Forbes, Ph.D.:It had been approved. I was Long Bargin, I was short, sirono right. All of the whole thing set up really pleased. I'd spoken to everyone in the world about the studies and their patients and it was euphoric. This drug, everyone was going to use it. I think three people dead their brains and also melting brains. I mean total disaster and I felt stock-halted sick. I will never forget that there were lots of things I was right about as a scientist. I could not have been more wrong in that moment. So very different, I suppose, what is wrong, what is right, but very different measures of wrongness and rightness when the same skills are somehow involved.
Matt Pillar:Yeah, yeah. How do you reconcile that? How did you rationalize being wrong when it was being wrong in investment banking was so different from being wrong in science? And then I promise we'll move on.
Zandy Forbes, Ph.D.:It's the recognition that how I rationalized it was this recognition that I could know about science, I could know about a clinical trial, I could know about a mechanism and, in fact, I could be the world expert.
Zandy Forbes, Ph.D.:And I could also have spoken to world experts and 20 of them because no point in speaking one world expert, because you're definitely going to get something wrong from them. But it doesn't matter, because biology is. We know so little about it, really, that it's unpredictable, and I learned that, however right you might think you are, that when you're making decisions about things like clinical outcomes or you know an FDA decision, there are so many things you don't know about that could make you wrong. That, rather than betting on your own rightness which is actually what you're told to do and I think it drives some of the bad behavior in public market biotech investing sometimes you really don't have an edge and you don't know things before other people. So all you can do is have a very, very good understanding of what would happen if you're right, what would happen if you're wrong, and choose to mitigate both of those. You will accept less on the upside, or accepting less on the downside, or accepting less on the downside.
Zandy Forbes, Ph.D.:So not going for the massive bets, but making sure that whatever you decide, you will always make money, and I think it's really when you're wrong over and over again, as one is, you learn that you may think you're a genius, but you've got to think that you could easily be wrong, and that leads to education for everything you later do.
Matt Pillar:Did you carry that mentality into the move in the industry when you joined Cabin Holdings or did you kind of put those pieces together once you were in the industry?
Zandy Forbes, Ph.D.:I think that the way, first of all, with respect to different people having different personalities. I'm a person who looks out into the world. I've got quite a lot of adventure in me, but also quite a lot of fear, so how do those two things marry? I would say that one of the things I do to make myself calm and be adventurous is I go. What's worst case scenario? I remember one time in my life and you can delete this from your podcast One time in my life I found it on this from your podcast.
Zandy Forbes, Ph.D.:One time in my life I found it on my phone when I thought everything was going wrong. I went okay, worst case scenario. I moved to Burma and I go and work as a banker for a friend of mine's father who was a banker in Burma. That's worst case scenario. How bad is that? Not that bad. Okay, I'll proceed.
Zandy Forbes, Ph.D.:So it's checking out worst case scenario and mitigating that, and then going up allows me to build things that are risky, because there are so many layers of alternative, which is really really embedded in the company that we've built at Mira, and it's not necessarily the easiest to convey to investors or to convey to what might be an ultimate buyer, which is pharma, because they're more used to almost like a binary stock pick, like clinical trial. That's going to be a phase three. There's going to be a single product. Are you going to commercialize it? Are you going to sell to pharma? What event do you have in the next 12 months? I find that, from an emotional point of view, very difficult to handle that binary risk. So I am very it's not even controllable. It is wherever I see a potential risk, I put a layer to mitigate it in place where I can, which includes a broad pipeline of things that are de-risked. Then manufacturing that de-risks everything. Optimization, not just of capsids, not just of promoters, but of everything. So it's a very broad view of optimization, which is the opposite side of the coin of risk mitigation, right.
Zandy Forbes, Ph.D.:And that allowed us to have a company that's highly resilient to a failure or the markets, or changes in what's the favorite product, whether it's Alzheimer's or GLP-1. In what's the favorite product, whether it's Alzheimer's or GLP-1. And, at the same time, be able to build a company that actually invents technology like controlling biologics with pills, right. So it's this sort of multi-layered cushions that you can see value in all these layers and they're each supporting one another. So it very much fed through to how we built the company. And the company wasn't obviously just me, but who joined the company when we formed from the fund that I worked with again with again is Rich Giroux, who's our COO and CFO. Who's an unusual person to be senior management in a biotech company, but he was head trader for a decade with me and so he executed my ideas in the world and I trust him more than anyone. Really I've worked with him's. A sort of multi-strat risk approach was not just me, it's the founders of the company and how we built the company.
Matt Pillar:Yeah, well, okay. So let's jump right into that. Give us more color on the origin story of Mira. Give us more color on the origin story of Mira.
Zandy Forbes, Ph.D.:So I was actually on the board of Cadmon and they needed someone at that particular time to go in and do a number of things in the company. So I agreed to do that for a year or so and, without really knowing what I was doing, I went there and ran commercial and learned a little bit. But I wasn't there for two years maybe, so I got some familiarity with how you sell drugs.
Zandy Forbes, Ph.D.:It was very, very complicated and I have a lot of respect for people who do that in the US in. But they had an idea to control genes with small molecules right, and they are not a gene therapy company. So at that particular time we decided to take that idea, which hadn't yet been made out of Catmon, and build a gene therapy company, and so I became one of the founders and CEO, rich joined and with a number of other founders we bought together, we acquired other companies, a few other programs and we set up a company, initially in the UK, because our first deal was with the Moorfields and UCL to buy iProducts and we immediately looked at the world of manufacturing because no one could manufacture it. How can we have a pipeline? And we started building manufacturing. We immediately, having acquired a company from UCL which was extremely strong in vector development, we set up organoids. We focused on promoters and capsids and sequence and from there built this notion of really looking at every aspect of these drugs that allow you to use lower, safer doses and deliver them locally in a way that you avoid a lot of the very obvious risks of doing somewhat difficult things with genetic medicines, like big gene replacement by systemic delivery.
Zandy Forbes, Ph.D.:Fantastic work. Got Zolgensma approved, right, I mean incredible life-saving drug, but definitely a very difficult thing to do, a very expensive thing to do, as we've seen recently, as we've seen over the years big, massive, systemic doses. So we looked at the. We said, okay, so what we're going to do is make genetic medicine, dna, a drug that can be used like small molecules, like biologics, like RNA, as this new modality where those other modalities are not effective enough or not broadly usable enough. Right, so not, as it's intuitively thought of, to just replace broken genes.
Zandy Forbes, Ph.D.:That's what gene therapy has somewhat come to me. But we had to build a company that investors would invest in, that was able to have a pipeline of products that we really thought worked, that we were willing to spend money on working. And so we focused on local delivery of gene replacement in the eye, of changing neural circuits in the brain by tiny doses, locally delivered, of changing self-function in the salivary gland for xerostomia. And so far, nine years later, we've brought those products forward and we have four late stage products one pivotal, one phase three, one about to read out. Phase three in the eye with Johnson children ages one to two.
Zandy Forbes, Ph.D.:Every child born blind treated with our drug can now see which is, you know, miraculous right. So across the board, we took a strategy that slightly avoided some of the obvious risks that other people took on board. But we didn't, because our aim was to broaden the use of gene therapy. And while we were doing all that and we built the best manufacturing world, we're a commercial manufacturer. We have lead optimization. Well before that we quietly like you would if you're MIT quietly invented a completely new way of controlling genes with small molecules of your choice, that is, so many logs, higher dynamic range and usable as a drug and as a platform than we ever imagined.
Zandy Forbes, Ph.D.:We're currently in a position where we can deliver anything encoded by DNA that makes RNA, whether it's GLP-1, leptin, amlin, pyy, any combination of those. Every farmer's antibody, you know, nucleases CARs on CAR-T. We can precisely control how much of whatever that drug is is produced at any time by giving the animal a pill and we're taking that into the clinic. So we were able to do that because we put all these other things in place manufacturing to support our pipeline, optimization to support the pipeline, but all of it which you need if you're going to use DNA to treat large diseases like obesity, you need to be able to use mini doses like three, four logs less than you use for local delivery at another time. So there was an unknown end point which we have achieved more than we anticipated. But we put in place many, many layers of things which are individually valuable but together each support each other's value.
Matt Pillar:Yeah, in retrospect, I mean you know I can see where that de-risks the entire initiative, but that's in retrospect and I mean it's well stated right. But that's in retrospect and I mean it's well stated right, Like if you've got a broad, you know the overused, many shots on goal, you know cliche or metaphor if you will, but leading into this, like from the outset, realizing the risk is subjective, you know, the assessment of risk is subjective. Leading into this, it occurs to me that perhaps some people would go oh, a startup company, a new company, wants to embrace development manufacturing in its entirety, on its own and it wants to build a pipeline of, I mean, what's Mira now 14, like stated program-ish.
Zandy Forbes, Ph.D.:Yeah, we've got four late stage programs we've got yeah multiple, multiple multiple pre-ind. Yeah, which we're not we we need to finance, but yes, so we focus on our late stage.
Matt Pillar:Yeah right, yep, but my point remains. Like to to some people, they'd look at that and they go well, boy, I don't know. As an investor, I'm not sure that I want to jump on board with that, because that sounds kind of risky. They want to build manufacturing capacity, they want to own the entire thing. Some people tell me oh, a giant step in our biotech or a giant decision in our biotech's development was to de-risk by outsourcing or de-risk by being uber-focused on a specific molecule or a specific indication. So tell me about that. What's your headspace around that? Actually, what looks like to some, a risk being risk mitigation in the mind of Dr Zandi Forbes?
Zandy Forbes, Ph.D.:So the original risk mitigation which I just mentioned is looking at the technology. Today, you're going to build a company that, in 10 years time, has transformed DNA into a modality that can be used to treat obesity Right, fantasy, okay. A modality that can be used to treat obesity right, fantasy okay. But you need today to have a pipeline that will be successful. So you start with okay, let's look at everything out there that we have to buy in, license or build. Look at everything out there that we have to buy in, license or build. How, in your original clinical programs, are you going to avoid risk?
Zandy Forbes, Ph.D.:The number one decision that we took was not to focus on an indication right that required large doses that eventually came to be seen as potentially unsafe right, and rather to only look at where we thought there was going to be a less risky clinical path forward. That involved local delivery of small doses in somewhat immune, protected areas where there was human proof, by avoiding the risks associated with large systemic deliveries and not going after super high-priced drugs for really rare pediatric conditions. By not focusing on that, we actually put together a diverse pipeline based not on indication but based on chance of success. And we also looked at the market right, because when you're doing gene replacement it tends to be small markets that you charge a lot for. We looked at our xerostomia market. It's a massive market, it doesn't go away. It's people with head and neck cancer, parkinson's a massive market. We also looked at cost of goods, because small, locally delivered doses are not only safer and may work well in the clinic. Guess what they cost? So much less. So our Parkinson's drug, which is in phase three, just had really good data. It's 1E12 titer, 35 microliters delivered. It's the lowest dose, smallest dose, safe, true. How much does it cost? In the hundreds to thousands of dollars per patient, not hundreds of thousands.
Zandy Forbes, Ph.D.:So there was a lot that went into choosing these indications where, at the very outset, we avoided some of the risks that other people are, you know, heroically taking on and addressing. And the manufacturing thing one of the people who founded the company with us, Stuart Naylor, had been at Oxford Biomedica and had learned how essential it was at that time to have manufacturing in-house. And that decision is one of the reasons we have a pivotal program and a phase three program today, because we built manufacturing, we got our facility licensed. We then did a deal with Johnson Johnson. We had the Johnson Johnson the J&J VC group come and do diligence and they actually told us at that meeting, had they set the company up, they would not have allowed us to spend the money on manufacturing. However, j&j wouldn't do the deal with us unless we had.
Zandy Forbes, Ph.D.:And then we built a plasmid facility under our GMP quality systems, not because, oh, we want to manufacture plasmids which we clearly don't but because it saves about a year in time and a ton of money. Then we had to build our own QC facility in order to release material, because the big vendors were taking up to 18 months to release something that should be six weeks. So you can't have a commercial supply chain with that kind of bottleneck. So Little Mirror went out, built a QC facility, designed and validated every single assay for commercial QC, for stability and release. We have a commercial license, right, we didn't want to do that, but we couldn't be a company with the quality of vendors in the world today and we save now end-to-end manufacturing.
Zandy Forbes, Ph.D.:We're very well known by the FDA. We cross-reference everything. Fda knows us super well from a manufacturer perspective and it probably saves three years in the development timeline of any drug. It is hugely valuable as a potential business. We have partners and other companies wanting us to manufacture. So there's value to our own business. But at this moment in time, because it's unique in how we built it, with a pipeline, with a platform based on more than 20 viral vectors right, it becomes valuable as a supplier ourselves. But that wasn't the intent, we didn't know any of that. We would have loved to use CDMOs, but it turns out to be multiple years faster, at least half the price, probably way more than half the price, and, aside from that, a valuable asset. We bought the land as well, we didn't just lease it. So we own stuff, because I'm a bit of a freak, I like solid things, not just like spending money not just like spending money?
Matt Pillar:yeah, did. Did those that, did those aspects make it um easier or more difficult to fund the company, like as you grew and needed more money? I mean it's? It sounds like a more of a nuanced uh, and sounds like there's some complexity, I guess, to the pitch, but you're very convincing, dr Forbes. So did that desire to own the entire system, to own the very ground that the processes live on, did it make it more difficult or did it sort of enhance your fundraising periods?
Zandy Forbes, Ph.D.:I think that a broad view of a business as a real business, looking to the future, is difficult for certain types of public market investors to think about.
Zandy Forbes, Ph.D.:We have some really really smart long investors with long views who are really supportive and we never had a high valuation. So, while gene therapy companies would be going to multiple billions based on a program, we didn't do that. We've actually raised significantly less money than many, many, many of our peers A because our market cap never went to those levels probably three, four, five times our market cap right and we didn't therefore have that market cap opportunity to raise massive amounts and not money less dilutably. But we've certainly had really supportive investors and investors are of all different types, right. I mean there are investors who just say, well, what's your next catalyst and I only want to know about one program, but they're probably not the people who would be interested in a fundamental investment in the sort of company that we run. There are others which have very different views, which like certain programs over others, which support whole businesses. So in general, has it been more difficult than it might've been had I been in a different company?
Matt Pillar:Yeah, created a virtual company with super low overhead and people you know, people distributed across the world outsourcing everything that you do.
Zandy Forbes, Ph.D.:Maybe, but it would have been different investors and we have had investors that were very happy to support the company we've built. That all said right. The last three years for genetic medicines and small biotech in general has been the most difficult ever in the history of the industry. So in the context of that environment, I don't know if a single product company would have done it. I mean, I think we've done an incredible job navigating the last three years and we do have supportive investors and we have got non-diluted financing and, frankly, in a world where capital is difficult to get, we have been in a position, this year in particular, to look at our business as multiple businesses and start talking to companies and investors about taking positions in different programs, taking positions in different entities, each of which is arguably as or more valuable than our current public market cap. So there's a big change over the last three years as to what investors are thinking, the types of investors that are interested in companies like ours, the source of capital, not necessarily from traditional short-term, long-short, not even from big mutual funds, but these other sources of capital that are looking to look for innovation, looking for diamonds in the rough of this sort of wasteland post-World War, I wasteland.
Zandy Forbes, Ph.D.:That's NASDAQ right. Yeah, that's NASDAQ right. There's an evolution of investors that we think with and alongside as well as pharma companies in any environment, and this is undoubtedly, as, I'm sure, everyone that you speak to. This is a weird and difficult market, right? Even the last two weeks, volatility is enormous, the liquidity is minimal, the selling is maximal, the buying is through private offerings or ATMs. It's a very unusual. Well, it's become usual, but from my experience a decade ago, it's not how things were and one has to adapt and speak to a lot of people, including pharma companies right, about the aspects of value and what they want and how to monetize things that you wouldn't have if you were just a single trial company.
Matt Pillar:Yeah, how often do you? You have time for a couple more.
Zandy Forbes, Ph.D.:Absolutely.
Matt Pillar:Yep, just a couple more. I don't want to abuse our audience either, so just a couple more. How often do you find yourself leaning into your experience, especially in this weird space that we're in or this weird time that we're in right now? How often do you find yourself, day to day, week to week, in the role that you're in now, leaning back into the experiences that you had as an investment banker?
Zandy Forbes, Ph.D.:I think there are two aspects of that experience Daily. I remember drug development failures that I witnessed as an investor. I don't think a day goes by where I don't think, oh my God, that could go wrong. Why do I know it could go wrong? Because I've seen it go wrong and that is all the time. And I didn't realize until I joined biotech and worked with pharma how valuable the breadth of failure that I've witnessed and not just witnessed that I've been stabbed in the heart by, but has seared into my psyche how valuable that is. So that's every day With respect to speaking to investors.
Zandy Forbes, Ph.D.:Speaking to investors, I think probably there's very few days, and certainly almost no weeks, where we don't meet with investors and I don't know what it would be like not to have been a public market investor.
Zandy Forbes, Ph.D.:But there is a certain decorum, language and way of speaking to public markets investors that I respect and one of the reasons we went public so quickly is I didn't have that experience with private. I don't have the same understanding and respect for VC right. It's a different way of being and I just remember the feeling of when we were public and suddenly I was speaking to people with. I call it decorum, because it's a language that you both know, which, whether it's really respect or not, it's a courtesy where you both understand each other without explicitly saying stuff, and I use that all the time in my communications with investors, and it's certainly a relief for me to speak to public investors. I don't know how much of a relief it is for them to speak to me, but I find it a much easier dialogue, which I attribute to having been one of them and having respect for them. Actually, I understand that they know a lot.
Matt Pillar:Do you miss those days at all?
Zandy Forbes, Ph.D.:I don't miss it now, I wouldn't necessarily. I think the last couple of years is equally tough for investors and for people managing companies. I sometimes think, oh my God, it was so easy. It was so easy being an investor because you've got so much opportunity. If you have one company, you have to make it work. You have to fight every day. You have to make it work. You can't get rid of the position and move on and make a success elsewhere. So I, you know, yeah, when things are looking bleak and you have to really, you know, force yourself to do your best, sometimes I go, oh, that was easy, but it's not easy. It's not easy to be an investor.
Matt Pillar:Right. So that's what we're going to end on on the fight and you're going to tell me what's the next fight Like, what's next on your agenda, what's the good fight? You alluded to the clinical progress some of the late stage candidates, so maybe give us an update on next milestones for those and what the next big fight Zandy Forbes is going to sign up for looks like.
Zandy Forbes, Ph.D.:I will say this Over the last three years my fight is to stop. It's a fight to succeed. I have really learned not to fight right. Really work with new investors, new sources of money, pharma companies really, really think out of the box in how you finance and move forward your company and how you do what your duty is right To extract value from all different parts of your business. And that requires a real need to think a little bit differently in many different scenarios. Think a little bit differently in many different scenarios, and that is what we're really doing now Financing, value extraction, new ways of doing something in a public market that's somewhat broken and in a world where there's huge amounts of capital that are interested in innovation. That's on the big thing. So I actually feel right now, as we come to the end of this year, really excited about the things we're doing with respect to building the company and extracting value.
Zandy Forbes, Ph.D.:With respect to the clinical programs, what I'm most excited about which is actually the smallest population, is getting our drug for the blind kids approved. It was something we did for charity right. Never expected to make money. We did for charity right. Never expected to make money, we gave it away and working with the UK regulators and the FDA and working with them to get this really transformative drug approved outside the normal pathway and seeing that in agencies that have been quite rigid in the past right, and being able to really really provide something which really changes kids' lives blind, not blind, autistic-ish behavior, normal behavior and working with those agencies in a really collaborative, somewhat less formal way, that's given me a lot of hope. In speaking to both the FDA and MHRA recently, that recognition we can do things differently here to actually get access for these kids that really need this and I find that exciting.
Matt Pillar:Yeah, that's super exciting. That's awesome work. It's a great story. I uh, you know, I feel like I could talk to you for the rest of the afternoon, uh, but I know you've got to get onto things and I need to get onto things. So we're going to have you, we're going to be back. If you'll come back, there's so much more to unpack and discuss with you and, as I said from the outset, your enthusiasm is contagious. I love the passion and enthusiasm. So, thank you, thank you for coming on. I appreciate it.
Zandy Forbes, Ph.D.:Well, thank you so much. It's super fun for me to talk to you. It was really interesting to be forced to think about things that you don't talk about with investors. It's really interesting, so I've really enjoyed it. So thank you.
Matt Pillar:Yeah, I've enjoyed it too and, like I said, we'll do it again. I'll get with your people, we'll get another date on the podcast.
Zandy Forbes, Ph.D.:Okay, all right, okay, thanks so much.
Matt Pillar:Thank you. So that's Meira GTx president and CEO Zandy Forbes. I'm Matt Pillar and you just listened to the Business of Biotech. If you're digging these conversations, please subscribe to the Business of Biotech anywhere you listen, and, if you'd like, go watch them at bioprocessonlinecom backslash VOB. Come on back for a fresh episode on Monday morning. We drop every Monday. We'll see you then. Thanks for listening.
