Naseem Amin: CEO of Orphalan

Naseem Amin,  CEO of Orphalan, shares his thoughts with BioBoss host John Simboli about leadership in biopharma and how Orphalan is working to transform rare disease treatment.

Transcript

John Simboli: 0:00

Today I'm speaking with Naseem Amin, CEO of Orphalan, headquartered in Paris. Welcome to BioBoss, Naseem.

Naseem Amin: 0:07

Thank you for having me.

John Simboli: 0:09

What led to your role as CEO at Orphalan?

Naseem Amin: 0:12

I've been in large corporations for almost 25 years of my career. And I thought I was semi-retiring back in 2015. And I got a call from a venture capital firm in London saying, they wanted to meet me and see whether I'd be interested in coming on as an advisor. And when I joined the company as an advisor, Orphalan, at that time, was called GMP Orphan. And it just started its development of its product. And it was going to be a several year development and commercialization. And I went on the board as an advisor, representing this venture capital firm. And then two years later, the product received a non approvable letter from the European Medicine Agency. And the board were informed that the product couldn't be manufactured. And we had a first-time CEO. And this came as a bit of a surprise for the board. And I remember after the board meeting, two of the partners said, we have full time jobs, we can't step in, would you step in to try and see how we can turn this around? So that's how I became an accidental CEO. And the original commitment was six months. And then, come five years later, I'm still here.

John Simboli: 2:02

So when you got into the job, and you find yourself the CEO, how did it turn out to be, being a CEO? Your experience of it versus your perception of what it would

Naseem Amin: 2:13

So the first couple of years, it was very similar to be like? what my perception was, that we had a series of technical challenges that we had to overcome. And I relied on my experience in the industry in a variety of different roles. And I worked with a lot of great experts in different areas. It was a virtual company. There were only two and a half employees, myself, my assistant and a part time CFO. And then the rest of it was a virtual network of experts. Because we had so many challenges, I just went and found the best and brightest people to help us. And we were able to turn it around. And several years later, we got the product approved. And that was back in 2018. Then suddenly, we found ourselves with a rare drug that we had to think, okay, what are we going to do with it now because it's approved in Europe. And I had spent many years in rare orphan diseases at Genzyme. And then at Biogen, and I knew that you could do this with very small organizations. So the board said, how would you commercialize it? Do you want to find a partner? And I foolishly said, well, you can do this with a small organization. And they said, well go ahead and do it. And we ended up building in three months, actually a commercial organization across Europe, gradually across Europe. And again, I was very lucky. A former colleague who had been head of Genzyme, Southern Europe, I reached out to him and said, Look, I face this problem and challenge, can you help? And he was actually working for another company. But this was the Genzyme family way of doing things. He said, but let me help. And he introduced me to half a dozen people who I ended up hiring, many of them still in the company. And we directly commercialized a product all across Europe. And we now have gone from two and a half people to over 70 people in the company. And we are one of those very fortunate rare biotech rare disease companies that has been successful in launching— and even more rare, profitable biotech company. And, you know, from those early years, we've been very lucky on the journey we've embarked upon. And so I've actually grown and learned so much that I never had to do before. My background was in R&D in large organizations, and then in business development, some marketing, but never running a company with a commercial product with a P&L. And I had to learn on the job and made more than my fair share of mistakes along the way, but always found there were people who, if you ask them, they help you. That's one of the things I've learned, that if you ask, and you ask yourself and people tough questions, there's usually a solution.

John Simboli: 5:58

That sounds like a willingness to help, not just knowledge on the part of the people you were reaching out to, but a genuine interest and in solving.

Naseem Amin: 6:08

Yes. And that's because, I think, in rare orphan diseases I think it just recruits people who want to be in that field, because of the nature of the patients, the nature of the disease, then often the lack of, you know, they really have been orphans, no one has cared about these patients. And so the people who come want to help. So those who joined the industry, whether it's in R&D, whether it's on the commercial side, they have an empathy and an affinity for the patients, the people providing the care. And I think that goes also into their professional engagement and interaction, as well. And so that's what I was able to draw upon—a lot of empathy, a lot of wanting to really make a difference

John Simboli: 7:09

In looking at your LinkedIn and so forth, I noticed that you were both a scientist, someone who's skilled in management, and also a physician. So how does being a physician prepare you or not prepare you to be a CEO of a biopharma company?

Naseem Amin: 7:27

That's a very good question, because I think it's a strength and a weakness. The strength is that you very easily can understand the patient, and how a drug may be able to help the patient, because all your academic and professional training has been. As a physician, you're used to making decisions, and often being the individual who makes the decision. I always say, you know, when things get really crazy, I become calmer and calmer, because when you're a physician, for instance, if you're in an emergency room, as everything around you is becoming more crazy, and more and more people are trying to provide care to the patient, you have to step back and say, Okay, what do we really need to do here? What are the priorities? And I will say, you know, in medicine you're taught ABC, in an emergency situation, you secure the airway, you make sure the patient is breathing, then you make sure that you've got adequate circulation. And so as a physician, you're very good at being decisive and making decisions. The weakness is that you say, think that you're the one who should always be making the decisions. And that transition, I'm not sure I fully perfected it even today, because I'm not sure we fully change. But I've tried to be more nuanced in decision making. I also have learned over time, that you have to rely on a lot of people and rely on their expertise. One thing I have got is, you know, medicine is very Socratic. There's a lot of back and forth of debate. You also always have to be listening, because you know, you listen to the patient, but you're immediately formulating a hypothesis of what's wrong. And I think that combination of the Socratic decision making, just being decisive, being a listener but also making sure that you're not the only one doing that, that's where the dynamic tension is always, I think, been in my career,

John Simboli: 10:12

I do know that being a biopharma CEO means being the head of what can be a fragile thing, because it's hard to get a company off the ground. So how does a CEO properly lead that and stay calm and, and positive?

Naseem Amin: 10:28

I think as long as you're tough on the issue, and not on the person, an empathy for the person, I think is incredibly important. If there is a mistake, you immediately put the person at ease, that this is not their mistake, mistakes happen, its nature. I always say I can handle anything, don't surprise me, when you've known it for a long time, and you've hidden it from me, then I have a bigger issue. Because there's a breakdown in trust. And ultimately, what we do requires trust of each other. And if someone is not open and telling you what the issues are, trying to solve it themselves and comes to you too late, then it's very difficult to fix. But if something happens, even if they didn't tell you, you don't immediately become the tough guy. Sometimes you do have to make tough calls. And that is a game, you have to try and do it with empathy. The most difficult things I've ever had to do is when I know someone is not performing, and you give them a lot of leeway, you try your best. But if you just think there is a inherent conflict of the mission of the company, and where it's going, and the skill set of the person, and you've got to make a decision, those are the toughest decisions. The rest of it, you know, environmental situational things, if you have smart, bright people, you can overcome almost anything.

John Simboli: 12:17

How does a capable CEO resist the temptation to be the "smartest guy in the room?"

Naseem Amin: 12:24

I remember when I first started, you know, I had just come out of a hospital environment, you know, dealing with incredibly sick people. I joined a company and there are all these people, marketing people, others. You know, I was used to evidence, data, decisions. And it was a lot softer. And the people were conjecturing. And, you know, in my very blunt doctor way, I said, you know, you've got no evidence for that. How can you make that statement? And I remember a very smart, my first boss, she was Director of Marketing at Baxter Healthcare. She took me aside and she said, Naseem, look, you probably are the smartest person in the room. But you don't need to point out to people that they don't know what they're talking about. You can phrase it in a different way. You could say, Look, that's a very interesting thought, you know, is that your hunch? Have we got any market research to support that? You can soften how you ask the question. Without making people feel like they're an idiot, and you're the smartest person in the room. And always when I start going down that drilling mode of trying to get to the fact, I always remember what she tells me, it's not always successful. But I hope I'm doing it more often than I did it the first time. And I also found that I'm not the smartest person in the room. There is always someone who knows something more about every topic. And the trick is to find out, get it out of them. And I've got a colleague, an old friend who works for that company. And he said to me, just ask the person who's the one who's quietest in the room. They're usually the ones who have the information and the best information. And again, I don't think I fully learned that yet, but it's a work in process.

John Simboli: 14:36

When you built Orphalan and you've done the recruiting process, not directly, of course you have people that worked on your team who helped you to do that. But surely you're looking to hire people who are in their own way, the smartest people in the room.

Naseem Amin: 14:55

Actually I think most people are smart at our level with our level of education they're smart. I'm looking for people with passion, a desire to make a difference, empathy, and not political. And the reason I say that we're a smart, small organization. In a large organization, politics can thrive because you know, you've got multiple stakeholders, you're trading off, you know, you do this for me, I'll do that for you. And a lot smaller organization like that, there's no place to hide. Someone told me in our organization, it's, you come here, you know who you are, you know what you can contribute. And you just want to get on and contribute. Because, you know, a lot of your time in your career has been spent on not directly making a difference, but tried to make a difference. And here, you can make a difference. And I think that's what I look for when we're hiring people.

John Simboli: 16:07

Naseem, can you remember when you were maybe eight or nine or 10, or some early age when you had a self-image of what you might be when you got to be an adult? And I'm guessing for many of us, it's what the what did you think are your parents pictured you doing? Who knows? But can you remember that time? Does it have anything to do with your work at Orphalan now?

Naseem Amin: 16:28

I'm about seven or eight, and a group of friends and I decided we were going to make a spaceship. And we were going to go into space. And I remember walking along the street and picking up aluminium foil from like a KitKat. And saying this would need to be a heat shield. And I also know my mother told me that whatever she was doing, I said, I can make that. Those are the two things I remember from my childhood, that may give a clue about who I was. You know, people who know me now think I'm quite loud and talk a lot. But I was actually very quiet and observed a lot. Then I come from first-generation immigrants who, you know, there are only three careers that I could choose. And it was in the order of pecking of how capable I was academically. First was a doctor. Second was a lawyer. And third was an engineer or maybe an accountant. And I remember being four or five and my father introducing me to people. So this is my only son, four daughters. He's going to be a doctor. When I was 16, or 18, I rebelled against that. And I said, I don't want to be a doctor. I want to actually go and study Russian literature. And for a first-generation immigrant family, what are you going to do with that? How you're going to earn a salary? And for them, it was all about economic success. And then I think around 18, I suddenly realized, okay, now I don't need to rebel against my father's desires. And I said I really love science, maybe medicine is not a bad choice. And that was when I applied for medicine.

John Simboli: 18:29

Do you remember a time maybe in college, graduate school, at some point where you said after after you received your medical degree where you said, I love this, but there's something else that's intriguing me. Do you remember that cycle?

Naseem Amin: 18:44

I was working in the National Health Service in England, and I was working in nephrology. It was tragic, because if you were 50 years old and diabetic and your kidneys failed, we basically told you you had to go home and die, because we didn't have enough hemodialysis machines in the country. I think we had a couple of thousand machines for a whole country. I found that soul destroying. And I remember we had a very socialist physician who wrote, he drafted a letter and said give it to every patient and tell them to send this letter to their member of parliament. Why they should get hemodialysis because transplantation is not available. It's available for very few patients. And if you're diabetic, you didn't even get on the transplantation list. I thought that the delivery of health care and how we managed the management side of health care was broken if we had to make those decisions. And my other head of the department became the medical director of the hospital. At a time when Margaret Thatcher was experimenting with a different delivery model. It was called NHS Trust. He was grappling with the questions I thought, were important if we want to improve delivery of care to patients. And I told him, frankly, I said, Look, I'm really struggling with a career in academic medicine. And he said, you know, I'm loving what I'm doing. But I know I'm not equipped to do my job. I'm a physician. If I was your age, I would go and get an MBA and come back into hospital management. So that's how I made that switch. And I will never forget, I had no idea what an MBA was. I went to the local university bookstore. And there was a book by Business Week, saying, top MBA schools in the world or top business schools in the world. And I applied to the top three, and ended up going to Kellogg in Chicago. That's how I ended up doing an MBA. And actually that's how I ended up in the industry because I was originally thinking I'd go back to the UK and go into hospital management. But Kellogg, as you may know, is in the greater Chicago outskirts, and it just happened, again by chance, the company that developed hemodialysis, commercial hemodialysis, was Baxter Healthcare, and they happened to be in Chicago. And in those days, and they still do, your CV is sent to all the companies and they contacted me saying, you have a nephrology background. One of our divisions is called a renal division. And why don't you come and interview? That's how I ended up in industry. And I was one of the very first generation of MD-MBAs joining industry. This was back in 1990. And so that's the story transition from medicine into industry,

John Simboli: 22:19

In the MBA program, did the discipline, interest, fascinate, bore? Because that's very different. It's a very different discipline than being a physician.

Naseem Amin: 22:30

I loved it. I loved it, because I suddenly was exposed to people of all different disciplines. One friend was a shrimp farmer from Indonesia. Another was a marketing guy from Pepsi, who'd been an Olympic swimmer. I had friends from all over the world. Friends, actually I'm working with now who have joined or are helping the company in some way or the other. I was just exposed to looking at a problem much more holistically. You know, as a physician, you become more and more narrow, as you specialize and sub-specialize. So you become an expert at a very small, narrow range. And here, I could, it's almost like, you start a painting with dots, you're painting on a canvas and with broad brushes, but at the end of it, there's a whole painting. And that is what is so fascinating about combining different disciplines. You can bring aspects to a problem that people who don't have those multi-disciplines may find it a little harder. It's not that they can't get there, there are very successful people. And it reminds me, one of the people I think of as a mentor is Henry Termeer, who you may know was pretty much a founder of Genzyme. He was an economist by training. And he approached things very differently. Because he said, you know, he thought of things around price elasticity. Before him, no one thought you could make these very rare orphan diseases economically viable, and he approached it from the economist perspective and said, well, that why should that be the case? Because if the disease has a huge burden on the patient, the family, society, and if it's a very rare disease, then society should be prepared and can reward it. If they can't, then the obligation on us is to be able to make sure that we as a manufacturer, or developer of the drug, never put the patient or the physician between us and the payer. And so if the patient needs a drug we'll provide it

John Simboli: 25:12

How do you like to answer the question, who is Orphalan?

Naseem Amin: 25:15

Well Orphalan changed its name to Orphalan, which phonetically, Orphalan means orphan, in French. And I think that is the core mission of the company, to focus on patients' needs that are being neglected. And either the disease is neglected or the treatment neglects the needs of the patient. And that is our core mission is trying to address that need. And we are focused in rare diseases. Not to say that there aren't needs in broader diseases. But that's where we, I think, feel we can make a difference, mainly because of our backgrounds.

John Simboli: 26:06

Can you tell me how it was that you made the decision to focus initially within rare disease on the Wilson's Disease?

Naseem Amin: 26:14

So when the investment came to the venture capitalists, the theory was that there was an existing product which had been on the market for 40 years, and it had challenges. It's a cold storage product, it's unstable, it needs to be taken up to four times a day. And you cannot eat for an hour before or two hours after. So if you're taking up to four times a day, that's 12 hours a day, you may be fasting, and it's a refrigerated product. That was the thesis, and they thought they had a more stable form of that, that had been originally tested in a hospital in Paris, which was a reference center for Wilson's Disease, but had been abandoned because of issues with manufacturing. When we took it on, those were the things we had to fix. And so we developed a true room temperature stable product, which in our most recent trial, we've done twice a day. And we believe we may be able to improve that even further, with further research, but a lot has yet to be done. So here is a product that has been around a long time, but we've improved it for a patient population. And it reminds me of a drug when I was at Biogen, was a very similar sort of story. That was a 30 year old product used in MS and other auto-immune diseases in Europe. And had been off-label used in MS, had some evidence it worked, but it had formulation issues; it cause rapid flushing. With reformulation, we reduced that side effect, and Biogen licensed it in. And that became a drug called Tecfidera which became the largest selling MS product, lead oral MS product. And so when I saw this story being presented, I said this rings a bell. You can take an existing active ingredient, which has galenic formulation issues, reformulate it and fix it. And that's how the origin of the company began. Then we found out it was not as easy as it was presented. When we went to the EMA, all the issues I raised with you surfaced. And then the hard work began. We rolled up our sleeves for two years and fixed all the problems we were told had already been solved. From that, we've now done very well in Europe, where the lead product in Wilson Disease in that sub population. Our ambitions are to provide this drug all around the world. We've got it approved in Australia, New Zealand, the Middle East, in Saudi. We've submitted in China with priority review. We've got it approved in Latin America, and we just got it approved by the FDA. From this very small hospital in Paris, we were able to fix the problems we first embarked on a journey in Europe and now we're embarking on the next phase of the journey which is to make this improved product available all around the world.

John Simboli: 29:57

How does the way you've set up the pipeline express your vision for what you want the company to be?

Naseem Amin: 30:03

I think if you look truly at the track record of biotech and pharma companies, internal R&D is not very productive. And so if you look at the biotech companies, most of them, licensed in their product, and then developed it. So what I've set the company up to be is a development and commercialization company. So we have very good expertise in regulatory, clinical development, and commercialization. What I don't have is a history of research that I can draw upon to say, Okay, this is the technology platform, we're going to develop a pipeline from. I also think, and this was my experience at Biogen, I joined Biogen when Tysabri had just been pulled off the market. If you remember, this was a drug that was going to be a big MS drug, it had three or four deaths, or was associated with a rare infection, neurological infection, and the product was pulled. And I remember the CEO, when I met him, he said, I've looked at the productivity of our R&D. And it was started by a couple of Nobel Prize winners. And we developed, I think, he said, 10, blockbuster drugs, nine of them were licensed out. Would you say that the process of finding the right asset to And we kept one. But after the first five years, we've never developed a product of our own. So I'm going to change our whole R&D model. And we're going to license in products, and we're going to become a development and commercialization organization. And that's actually what Genzyme was, it was not a basic research organization. So that's the model I've grown up with. And I've seen very successful. And if you think about it, you know, the world is full of hundreds of thousands of scientists working on diseases, working on understanding the pathways. Then you got tens of thousands of companies trying to develop solutions, and they're the true heroes, because the vast majority of them will never succeed. Where I think we can add value is being the development and commercial partner. Now there are other people who are much bigger and better at doing that, than, I would argue, we are today in the orphan disease space. What we offer a partner is, it's important to us, we only have one product. So if we take your baby, it's one of only two babies. It's not 10, or 20, or 30. And it will be nurtured and developed. And that's our model. So we are constantly looking. Right now, we're looking at a gene therapy product, we've been looking at the most complex thing you could think of, you know, a gene therapy in a stem cell into a tissue that becomes a product. And each one of those could be an option, but it has to meet certain criteria. And then we are, as I said, we're continuing with further development work on our own bring internally, would you say that's more science than art? product, to make it even easier for the patient. But would you say there's an art to it as well? We say, you know, in medicine after a few years, you develop a nose. You know, when a patient is sick As soon as they walk in, you can tell that something's not right if you're an observant and good clinician. And I think that's true in our industry as well. When I was at Biogen, I think we used to look at around 500-600 opportunities a year. And in the five years I was there, we did about four or five transactions. One was this product that became Tecfidera. Another was a company we acquired called Syntonix, which became the hemophilia products that Sanofi bought for 12 billion. The third was our Alzheimer programs at Biogen, that was it's first foray into Alzheimer's. And then there's one or two that we licensed in which either failed or we decided not to move on. Out of those 2000-3000 opportunities we looked at, we ended up doing four or five, two became major products. And the third may, you know, not the original product, but the next generation of that may become a real Alzheimer breakthrough, as everyone is saying. So that tells you it's an art, it's a science, but it requires lots of different people's input. And I remember, I mean, it's a good question, because I'm remembering some of the conversations I was having with people. How the outsider program began was, I went to Al Sandrock and said there's this scientist in Switzerland, he's got this idea. What do you think? And Al said, it sounds interesting. And it was his nose that kind of said, we should think about this. And we ended up handshaking that day. And that was the beginning of our journey at Biogen in Alzheimer's disease. So why we made that decision on that day, can't tell you,. Someone told me intuition, all it is is lightning-fast logic. I'm not sure if that is true. But there may be an element of that. You're bringing lots of different things into something that is a coherent, logical thought. I've never believed in strategic planning. You know, you cannot plan success in our area. But you can be a strategic thinker. You can see information and you can make those links. And I've seen that with people who've been successful many times in our industry have that ability

John Simboli: 37:05

Naseem, what are the challenges you see ahead for Orphalan and for our biopharma community?

Naseem Amin: 37:11

I think the challenge for Orphalan is as we grow, and we're growing rapidly, how you maintain that culture, and how you retain that can-do and no problem is not solvable culture. So I think that's going to be the biggest challenge for us. Because as you grow, you put more and more processes in place, you have to be more regulated, more oversight. But balancing that out over time, and different kinds of people coming to companies at different phases in its development. I'm lucky, I've operated in the very large companies. And this is the smallest company I've ever been in. So hopefully I can help it on that journey. For the industry. I think we are a victim of our success. I believe we're just on the cusp of the true century of biotechnology. The last century was digital. And this is, I'm quoting someone who is a very seasoned major investor in the digital age, telling me this, he said, I wish I was in your field, because the next century is of biotechnology. And we now have so many of the tools to really decipher, intervene and correct at a very fundamental level, life at a DNA level. But as we all know, the more you learn about nature, the more complex it is. So we're going to have to decipher that complexity and develop tangible solutions. Then we got the bigger issue of how society copes with that, how it pays for it, how it prioritizes that with all the other challenges we have, whether it's the environment, whether it's polarization in the world, whether it's conflict, and here we are in our little cosm, microcosm, saying we want to improve humanity's lot by improving the health of our people. And this is what I'm always conflicted with is, you know, where do you put your resources? Do you deal with famines, hunger, or do you fix a disease that affects a few thousand people? And at a societal level, you might come to one conclusion As someone who believes as a humanitarian and your humanity begins with each individual person. That's where I'm not sure where we're going. That's why we're here. We love our jobs. I mean, I could never think of doing anything else. Could you?

John Simboli: 40:20

Naseem, thanks for speaking with me today.

Naseem Amin: 40:22

My pleasure. I very much enjoyed the questions and reliving part of my journey. So thank you very much.