Fellows is CEO of Beacon Therapeutics, which is developing several potential treatments for patients with a range of prevalent and rare retinal diseases such as the treatment for X-linked retinitis pigmentosa (XLRP).
"These patients, mostly young boys and men, will eventually go blind, and generally the symptoms start when they're in their early teens," he says. "By the time they're in their fourth decade of life, they're down to pinhole vision, and ultimately they lose their vision completely."
Fellows, who has worked in the ophthalmology field for more than 40 years, has a passion for finding a solution for this condition that stems from the many patients and families he has met who are affected by XLRP. He remembers one such experience in the UK, when he looked on as a physician met with a family whose young son was slowly going blind from XLRP.
The doctor, Fellows says, was explaining to the parents that one of the first symptoms of retinitis pigmentosa is losing night vision. The boy chimed in that he was already experiencing night vision loss. He said that had been playing tag with his friends in the evening and had run into a bench that he couldn't see. At that point, the boy said, he quit playing the game.
The boy then explained how the disease is affecting him.
“The thought that I might not be able to get a driver's license is devastating,” the boy said, according to Fellows, “but the biggest fear for me is I'm going to come to that point where I meet my date with darkness. That's the day I wake up and everything is black.”
"It's devastating for these kids because they're concerned that they will never be able to get their driver's license," Fellows says. "They're concerned they won't be able to get accepted into college. They're concerned that they won't be able to choose the career they want because of the limitations to their sight."
A Mission to Prevent Blindness
That incident and many more like it have inspired Fellows and his colleagues to be at the forefront of finding ways to manage XLRP. And interacting with patients is the key to pursuing that goal.
"We reach out to our patient support groups and work directly with them, and we also talk to the patients and families," Fellows says. "Our employees meet with patients because I firmly believe that you really have to get to know [them] to understand the journey that they're on. It really helps connect us to what we're trying to do on a day-to-day basis."
There are no current treatments for XLRP, which affects approximately 17,000 people in the U.S. and Europe.
"The most important thing for us is to find a way to get treatments to patients quickly because these people are all going blind," Fellows says. "They are living with the prospect of blindness their whole life. And now we have something that not only will save the vision, but potentially improve it."
David Fellows, CEO of Beacon Therapeutics. Photo courtesy Beacon Therapeutics.
Beacon recently presented robust 12-month data from its Phase 2 SKYLINE trial. According to researchers, males with XLRP demonstrated a response rate of 63% in eyes treated with a high dose of AGTC-501. Response rates in patients’ eyes treated with a low dose of AGTC-501 were similar to the untreated eyes in the high dose cohort. In addition, patients treated with a high dose demonstrated a robust improvement in visual function, including mean retinal sensitivity. Finally, according to the researchers, AGTC-501 was generally well tolerated, with no clinically significant safety events associated with treatment, and any treatment-related adverse events were mostly non-serious and mild to moderate in severity.
Beacon's second program focuses on an intravitreally (IVT) delivered novel AAV-based gene therapy for dry Age-related Macular Degeneration (Dry AMD), a leading cause of irreversible vision loss in people over 60. IVT delivery is less invasive, requires less clinician training and can be delivered in a clinic rather than via surgery.
"We're injecting the drug into the vitreous in the eye,” Fellows says. “And we're using an AAV capsid as the delivery vehicle, [which] will allow the complement factors to be expressed in the retina and presumably be able to affect the course of this disease and prevent the loss of vision.”
Leadership Begins With Listening
Fellows began his career as a sales rep, and discovered early on that a key element to success is listening. He says he listened to what the doctors were saying, identified their needs, and converted that into something that he could act on to solve a problem.
"From a leadership standpoint, listening to what people are telling you about what their needs are, and then trying to find a way that you can address those needs, builds successful relationships with physicians, patients, and employees," he says. "As long as you have your two ears open and you're listening, you're processing and acting, I think that's a good guide to leadership."
From Private to Public, And Vice Versa
Fellows has also had the unusual opportunity of both leading a company through an IPO and taking a public company private.
"Most people don't get the opportunity to do that," he says.
The publicly traded company going private was the Applied Genetic Technologies Corporation (AGTC), a clinical-stage biotechnology company focused on the development and commercialization of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases. AGTCwas one of the original gene therapy companiesand had been in businessfor more than 20 years.
"AGTC was purchased in November of 2022 by Syncona, one of the leading European investors, and that's when they asked me to take over the company and take it private," Fellows says. "I'm used to the other way around: taking a private company public. It's been a really interesting experience from that standpoint."
He says the biggest challenge in the privatization process is finding new funding sources after losing public financing.
"We've been in the process of raising money, and we anticipate we will be very successful,” he says. “But it is challenging, especially in this environment in the biotech world."
Another challenge involved orchestrating change management in an operation that had been in business for more than a decade.
"Asking people to unlearn behaviors and processes and convincing them there are different ways to do that" is difficult, Fellows says. "I spent a lot of time coaching people that it was now acceptable to stand up and give their opinion and to work new solutions."
Outside the Office
Creating a new work environment for more than 100 employees while driving several programs forward that may help prevent blindness keeps Fellows busy. For relaxation, he enjoys his collection of classic cars, especially his black 1969 280SL Mercedes.
But everyday his focus is on the patients.
"There's only one reason to be in the drug development business and that's to get something to the market that's going to help patients,” he says. “And if you're not interested in that, then I think you're in the wrong career.”
Adam Pearson’s life experiences and a recent diagnosis of a rare disease give him a unique perspective to guide the pharma company’s strategy
Adam Pearson, chief strategy officer at Astellas Pharma, has been working in pharma for 20 years, almost all at the Tokyo-based company.
Coming from a consulting background, he says he was eager to work inside the industry for which he had been consulting because he was intrigued by the challenges of a commercial business that is highly scientific and highly regulated.
"It struck me as a fascinating combination of different factors and piqued my interest in learning how to make that business successful," Pearson says.
Pearson began on the operational side of pharma, but recently moved to the global chief strategy position. About four years ago, Pearson had taken the role of head of corporate strategy and in April 2023 took the reins as chief strategy officer.
"I had built my career on the operational side, owning the P&L for different geographies," he says. "But I couldn't turn down this opportunity. I am glad to return [to strategy] to be able to steer the bigger questions and the bigger picture of the whole organization."
Dealing with a rare disease
In the past couple of years Pearson has faced several challenges, including being diagnosed with AL amyloidosis as he took on his new leadership role at Astellas. He says the diagnosis, which he calls one of the most impactful experiences of his life, has transformed both his personal and professional perspective.
Adam Pearson, chief strategy officer at Astellas Pharma. Photo courtesy Astellas Pharma.
Pearson says his illness was discovered early, and with continual treatments it is currently under control.
"I'm still under treatment," he says. "But that process, that experience, has helped me to reflect and understand much better what it means to be a patient."
Pearson has brought his new perspective to his leadership role at Astellas and to his team. He says he is much more aware of how pharmaceutical companies can support patients and incorporate an understanding of their perspectives.
"It's certainly changed my point of view," he says. "My unique experience as a patient has informed my leadership perspective. This has allowed me to relate to patients on a more personal level and reflect on how the actions we take in support of our drugs are perceived by patients.”
“I understand the gratitude patients feel from receiving treatment, as well as the challenges they face while trying to find the best treatment options, coping with side effects, and facing the uncertainties of the disease," he adds.
With a new R&D focus comes new challenges and a reorganization
About the same time he was managing his health issues, Astellas shifted its business model and created a new leadership team to manage the transition. He was promoted to chief strategy officer, at the same time that Naoki Okamura became the CEO and other executives took over new roles.
"There has been lots of change and it's been very interesting," Pearson says. "Working with a new leadership team has been an important time of reflection on how Astellas is doing and what comes next. We made a large acquisition last year with Iveric Bio and that's also been a major part of what I've been involved in."
Astellas' R&D strategy, called the Focus Area Approach, consists of three components: Biologies with high disease relevance, versatile modalities/technologies, and diseases with high unmet medical needs.
"We're trying to evolve our R&D operating model," Pearson says. "We are met with having to make difficult choices around how to allocate our resources and there are tough decisions to make. We are facing new unknown challenges in development that we never faced before with small molecules or antibodies."
One of those challenges has been determining how to maintain a high level of investment in R&D while working on other promising areas.
"One of the challenges that's certainly at the heart of it is: how can we make the best [of] these investments?” he says. “How do we get our R&D engine working better and better?"
The leadership team has reorganized R&D in one way by giving more empowerment to teams and allowing more agile decision-making at the team level. This creates a more fast-paced environment.
Another priority was to ensure that the new leadership team works together to create a clear focus for the organization. As a result, Astellas established three enterprise priorities for the company.
"Broadly speaking, the priorities are maximizing the potential of our products to reach as many patients as possible,” Pearson says. “It's around raising our sights and improving our capabilities in R&D, really driving products faster through the pipeline, and third, it's around responsible management of our margins and costs and transforming the business so that we become more sustainable in the long term.”
"It is very exciting,” he adds. “It gives us all the fuel to take on the challenges for products that show that they can make a difference to patients. For example, we've had some dramatic results from PadCev, a treatment for bladder cancer, which in combination with Keytruda has shown to double the lifespan compared to the standard of care for patients with bladder cancer."
Leadership skills needed for leading a global transition
Leading through a company-wide transition takes an accurate and realistic assessment of the situation and a deep understanding of one’s team members, all while creating a new model for the organization. Pearson believes this requires trust in one’s employees, a deep understanding across cultures, and clear communication.
"I think my role as chief strategy officer requires me to be able to articulate as clearly as possible where we are trying to go as a business," he says. "We need to articulate clearly what the challenges are and the choices that we need to make to get us to our goal."
Pearson says he is all too happy to empower his teams to work autonomously and is not interested in micromanaging. His first step to building a solid team is to hire the right people for the job, and then give them the freedom to do it.
"I put trust in them and provide them with a combination of support and coaching and challenges, but I also allow them to get on with their job," he says. "I love watching this growth happening in my team, when team members feel confident and empowered to own their responsibilities and make their own choices around them. My job is to be a sounding board mostly, and sometimes to challenge them a bit, and certainly to try to make sure that they understand how they operate in the full organizational context.”
“The ideal situation is when they're owning the plan,” he adds. “They all know their job well and do it better than I can and I'm there to empower and support them in that."
While it is difficult coordinating with people across so many countries and cultures, Pearson has had plenty of practice. Astellas' center of gravity is in Japan, but it has a large presence in the US, Europe, and other parts of the world. Pearson says this makes arranging meetings quite a challenge from a time zone perspective.
"It's difficult getting everybody on the same call," he says.
But more important than meeting times is the many cultural differences that must be accounted for and understood. The cultural differences across Astellas might be a bit broader than in a typical global company.
"Leaders need to invest more time to understand what is behind the way someone is acting and behaving because what is going on in their heads may not be what your instinct tells you,” he says. "It might be something quite different in fact."
Pearson has the experience to manage a global company, having lived in nine countries. He was born in Australia and spent much of his childhood in the UK.
"We moved around a lot and sometimes we lived abroad, sometimes we lived in the states," he says. "I lived in Greece when I was at university. I worked a bit in France, and I studied in the US. While at Astellas I have been based in five different countries; at one of my positions, I had responsibility over 10 countries. And I now live in Japan."
Pearson and his wife are settling into Japan and enjoying the many aspects of its culture, including learning the language.
"We are doing a lot of exploring and hiking and just trying to get to know the country and stay active," he says.
While it would be easy to visit other parts of Asia, Pearson says there is more than enough to explore just in Japan.
"It's really a fascinating country, and it's a privilege to live somewhere internationally," he says.
Stephen Ranjan believes teamwork is just as crucial as technology in building successful digital healthcare solutions in the pharma space
Stephen Ranjan learned how to be innovative while growing up in India.
"Living in India, you learn to be resourceful," the global head of digital health at Roche Pharma recalls. "You don't have access to everything, and yet you still find ways to solve the challenges that you encounter. I brought that attitude with me when I came to the U.S."
When Ranjan moved to America to attend college, he found a market for his skills, and a career was born.
"In the U.S., if you can help people solve problems, they will give you opportunities to do so,” he says. “I ended up working at the help desk of my university. I built software that allowed the help desk to track student loaner laptops. This job paid for my college."
At his first job at Epic Systems, Ranjan was introduced to how software can enable efficiency in healthcare. He says he wanted to introduce those technology solutions to the pharma industry, where there were plenty of problems to be solved.
He's had plenty of opportunities. Prior to joining Roche, Ranjan held positions at Merck & Co. and Johnson & Johnson. He says he has seen digital health technology advance, but it still has many more opportunities to improve the patient's health journey.
"About 15 years ago, the industry was just beginning to dabble with technology in the form of websites, social media, and those types of things," he says. "But over the years, pharma has gone through a transformation. The uptake at first started on the sales and marketing side, but in the last 10 years, there has been more investments in the R&D side, especially right now with AI. That has been a huge, huge transformation."
Ranjan says most of the advancements in patient care have been made in cardiovascular, metabolic disease, and mental health, where technologies like continuous glucose monitors are helping patients self-manage their care.
A Lifetime of Experience
Today, Ranjan is using his years of experience to steer the company toward more digital health innovations that improve patient health management. Before he became global head at Roche, he was responsible for the Roche Diabetes Care Digital Health business, where he was instrumental in the growth of engaged users on mySugr and the Roche Diabetes Care Platform.
Ranjan says one of his biggest achievements happened in 2017, while he was working at Merck. He and his team collaborated with the Alexa team at Amazon and Luminary Labs to create an open innovation challenge in diabetes. The task for contestants was to discover a way to use voice technology to help diabetes patients manage their disease.
The challenge generated an increased focus on voice technology worldwide and spurred more companies to develop their technologies to become more healthcare-compatible.
"We're seeing a lot more voice applications being developed," Ranjan says. "The highlight for me from running a challenge like this was to see the impact ripple across the globe and motivate the market to think more about solving some of these problems."
Teamwork Over Technology
Ranjan has focused often on building new teams that can carry out the mission of the company.
"I've learned a lot about how to build teams," he says. "I don't invest in ideas. I invest in teams who are solving complex problems."
He believes his approach to building teams has influenced their success. And although the technology they use is an important part in reaching goals, he says building teams that can work together well to create solutions is a bigger priority.
Building a strong team, Ranjan says, requires hiring people who are equipped to bump up against a brick wall and still persevere, and to get creative and find other ways to make things work.
"Great team members are willing to learn new things and adjust to the new learnings,” he says, “because [in] this area [that] we're in, there's never a straightforward answer.”
"You have to be willing to try and experiment and maybe even fail,” he adds, “but [you] learn from it so that you can get better at making decisions the next time around.”
According to Ranjan, team members need to possess the potential and willingness to learn and grow as they face problems within the business or other challenges in an organization. Tolerance for failure and moving on are also important.
"The last bit for me, personally, has been patience," he says. "This is a long game. If we really want to impact patient lives and use technology to impact them, you can't rush through these things.”
Staying on the Path to Innovation
Ranjan sees a bright future ahead.
“For me, this is such an exciting space,” he says. “We are working to empower patients to manage their disease and live a better life, and that's the vision that I'm excited about--watching our teams push and innovate. We will have some successes, but we will also have a lot of learnings. And that's going to become all part of the innovation."
Ranjan and his family recently moved to Switzerland, where he's taking advantage of the Alpine scenery and climate, as well as the cycling culture.
"I'm not quite as good at cycling as people here [in Switzerland]," he says. "But I'm embracing it and I'm enjoying bike rides with my boys. My other hobby is I love to take photos of birds in flight. There's something about the way birds fly and how they move that's fascinating to me, and I love capturing those moments."
Ranjan's long-term vision is to continue using digital health to overcome obstacles and better the lives of those dealing with chronic diseases.
"We, as an industry, still have a long ways to go in digital health and I'm hoping that I can continue to lead teams to figure out new ways to meet the needs of patients,” he says. “Because at the end of the day, what motivates me is knowing that we are resolving problems for patients, but potentially also for someone we know. We are really in the service of helping people."
As general counsel for the clinical-stage biopharma company, Alex Nemiroff is guided by his experiences as the father of a child with a rare and incurable disease
When he was 36 years old, Alex Nemiroff's life changed forever.
That was the day his first son, Roger, was born with an extremely rare genetic epilepsy (SCN2A mutation) that currently has no cure. Roger started having seizures within hours of his birth, and he was diagnosed with a condition that carries every symptom imaginable and most often leads to death.
"He was our first child, so we really had no idea what to expect and certainly never expected this," Nemiroff says. "It was completely terrifying."
That was nine years ago. Within six months of Roger's birth, Nemiroff, an attorney, and his wife committed themselves to finding a treatment. At first, they focused only on learning enough to try to improve Roger's life.
"Doing the research was a knee-jerk reaction," says Nemiroff, now general counsel at Praxis Precision Medicines. "I thought learning more about the condition might open up some opportunity to potentially change my son's health trajectory. In retrospect, it is clear that we were trying to find a ‘fix’ so that we and our son would not have to deal with the realities of how sick he was."
With no experience in drug development, the lawyer-turned-biopharma-entrepreneur ventured out on a mission that he admits was way over his head, but he wasn’t deterred. Along with learning all he could about Roger’s condition, he began raising money and cofounded RogCon, a biotechnology company, to develop an antisense oligonucleotide to treat it.
Alex Nemiroff, general counsel for Praxis Precision Medicines. Photo courtesy Praxis Precision Medicines.
RogCon forged several partnerships on its way to developing a proof of concept. Then it enlisted the help of Praxis Precision Medicines.
"What was actually really helpful was the fact that we had no idea how challenging this was," Nemiroff says. “Throughout this process, people kept telling us we weren't going to be able to do this. ‘Go home and take care of your kids,’ they said. ‘They're very sick and need you at home. You're not going to cure this disease in their lifetime.’ But we continued to push, and here we are."
Nemiroff also began embracing spirituality, which included a deep, introspective dive into his own fears and insecurities. By creating grace, he says, he began functioning at a more wide-ranging, compassionate level, including maintaining a broader mindset around all children who are born with SCN2A.
"I really started to work on myself and to face a lot of the fears, issues and insecurities that have plagued me my entire life,” he says. “And for me, that meant diving into psychoanalysis and spirituality. What I discovered is that what holds so many of us back, whether it's being a leader, being a part of a team, driving a program, or a company, are our own issues. And so when I look back at the past with my son, I see so many things that held us up, that slowed progress down, and the way I dealt with challenges could have been a lot better. I have become more effective than ever before in confronting challenges through the personal work that I've been doing to transform these issues."
Nemiroff credits his shift in mindset to the successes along the road to development.
"Looking back, I realize how unlikely it was to end up where we ended up," he says.
“What I learned is that for any endeavor to succeed over the long term, you have to have a true, genuine, almost pure desire that is motivating and driving whatever you're doing so that it can really withstand all the attacks, challenges, and anything else that comes up along the way. I think about a time when this drug program started to slow down and get stalled, and it was incredibly frustrating and heartbreaking. It was around that time that I started to realize that trying to help my son is actually a very selfish desire. And I started to refocus my thoughts on the bigger picture, on how this will help all these other kids, and that's when things started to move."
"I was very worried about being seen as the parent of a sick child who was pushing this drug as fast as possible, and who has no appreciation for risk or drug development,” he says.
According to Nemiroff, Praxis President and CEO Marcio Souza disagreed, and he encouraged Nemiroff to be authentic.
"Your perspective is the strongest thing that you bring to the table,” Nemiroff says he was told. “You absolutely should push the urgency that you feel because otherwise, nobody is going to do it.”
“That completely changed my perspective, and transformed the way I looked at my role,” says Nemiroff, who now encourages parents and patients to speak up and add their perspective wherever they can.
“My role at the Praxis table goes beyond just general counsel, but also includes being a parent of a sick child, which informs my perspective,” he says. “And it really allows me to participate in discussions about things like drug development, science, and engaging with patients in a way I otherwise would not have been able to do. And what’s so fascinating to me is the interaction really spurs very interesting conversations and angles to look at these drugs and programs in a very different way.”
Collecting topline results of PRAX-628 in a Phase 2a PPR study in the first quarter of 2024;
The expected initiation of the Phase 2b study in focal epilepsy;
Releasing results of the Phase 2 EMBOLD study of PRAX-562 in SCN2A and SCN8A developmental epilepsies, which should be available in the first half of 2024.
Collecting topline results for both Phase 3 studies evaluating ulixacaltamide in essential tremor later in 2024;
Completing regulatory interactions to advance elsunersen (PRAX-222) towards a pivotal study for the treatment of SCN2A gain-of-function (GOF) developmental epilepsies.
Late last year, Praxis posted a preliminary analysis of PRAX-222 Part 1 that showed 44% median reduction in seizures after three doses for SCN2A-gain-of-function pediatric patients.
Designed as a safety study only, the results were a surprise and a win.
“What we saw was completely shocking and incredible to us,” Nemiroff says. “If we had had even just 10% to 15% efficacy, just to demonstrate there’s a signal, that would have been phenomenal. To see 45% median seizure reduction and a significant increase in the number of seizure free days for these kids was just incredible for us to see.”
While these statistics are beyond expectations, Nemiroff has learned to be cautiously optimistic.
“There’s been so many setbacks and challenges along the way that, sure, a fear of the other shoe dropping builds up," he says. "Until this drug is really over the line and available for all these kids, it's going to be hard to actually let go, breathe and celebrate."
Be brave enough to know what you don't know and to learn things you haven't learned before, she says
As president and CEO of Sumitomo Pharma America (SMPA), Myrtle Potter sets a high level of work performance for herself and her team members. And she doesn't apologize for it.
"I set really high standards as a leader and I demand a lot, but at the same time, I'm also a nurturer," she says. "I think my employees would be disappointed in me if I didn't expect great things from them. I believe they can deliver great things.”
“My job is to make sure the organization can deliver against its objectives, and to ensure it is prepared to do that," she adds.
Setting high standards of performance for your employees doesn't mean you aren't a caring boss, Potter says. There are many layers to running an organization, and an important one is being responsible for the growth and development of your employees.
Leaders must drive the strategy plan, the implementation, the follow-up, and meeting the financial goals. At the same time there's a responsibility to the health and well-being of the employees.
Potter says her ability to build strong, diverse teams is the key to her success.
"The secret to my success is I think strategically about team building, about leadership development, and the selection of leaders who I choose to have on my team," she says. "I make it a point not to hire people who are just like me, because I believe that different points of view are essential for the advancement of innovation and for the best problem solving."
Myrtle Potter, president and CEO of Sumitomo Pharma America. Photo courtesy SMPA.
Potter grew up in a small southwest town in a family of eight. She credits learning how to share a bathroom with five other siblings for her team-building skills. Rather than being a hindrance to her sizable aspirations, she says her small-town upbringing set her on the path to becoming a strong leader.
"I was raised to be very brave," Potter says. "Fearlessness was instilled in me. Even though I grew up in a small town, where everybody knew each other, my aspiration was to go to the University of Chicago. My parents helped me financially to get there. It took a lot of guts to get on that plane by myself and head off to the big city of Chicago. But I grew up in an environment where I was encouraged to stretch, and I believe this is an important trait for anyone looking to do great things in their career. "
When Potter is mentoring others, she sets up the same encouraging environment that her parents did.
"I advise people to leave their handprint on every job they take. In other words, don't just warm a seat, make a difference," she says. "People will remember you because of the contributions you've made and the difference that you've made in the business."
Potter also encourages others to be brave, as her parents taught her.
"Be brave enough to know what you don't know and to learn things you haven't learned before. Seek out the education and the support that you need to round out your understanding and your knowledge base."
Establishing a Career Trajectory
At the University of Chicago, Potter took a couple of jobs at the University Hospital. She worked in a lab, as well as on a patient floor for seriously ill patients with dermatological diseases and neurological diseases.
"I was a ward clerk,” she says. “I really loved it because I got to essentially be a part of the medical teams. That was the defining moment that motivated me to be in the biopharmaceutical industry, that and my dad having been a medic in the Army. I was really close to medicine my whole life. And so that was really the impetus for me joining the pharmaceutical industry."
Over the past 30 years Potter has served as vice president at Merck, president at Bristol-Myers Squibb, and chief operating officer and president of commercial operations at Genentech, as well as the Vant Operating Chair at Roivant Pharma. She also ran her own consulting business, Myrtle Potter & Company, for more than 13 years.
Over this time frame, she says she has honed her leadership skills.
"Today, I understand the power of listening,” she says. “Listening for congruence, listening for alignment, listening for gaps in strategy, listening for times when an organization might feel freer, or they need to be bolstered up, listening for when they feel empowered. Developing others, thinking ten to 12 steps ahead, supporting the organization through the good times, and the tough times. Those are the kinds of things I think about now, versus when I got my very first management job."
Facing the Challenges of a Large Merger
Earlier this year Potter faced a new challenge: The merger of not two companies, but seven. She was instrumental in successfully bringing Sumitovant, Myovant Sciences, Urovant Sciences, Enzyvant Therapeutics, Sumitomo Pharma America Holdings, Sumitomo Pharma Oncology, and Sunovion Pharmaceuticals together to form SMPA.
"As you can imagine, that was just a Herculean task," she says. "Combining a couple of companies is challenging, and I have done that before, but we're talking about seven."
Potter led the companies through the challenge, following a strategy that allowed the transitioning employees of all seven companies to choose whether to join the new company. Mergers can be stressful for employees, and she says she tried to give everyone the opportunity to engage in their future role at the new company.
As opposed to just bringing all the employees together and saying, 'This is your new job, now go off and do it,' Potter says she brought people to the organization with a lot of integrity and honor.
"We literally gave every single transitioned employee a new offer letter, a new description of their role, and gave them the choice to say yes or no," Potter says. "It was very empowering to hear people say, 'Yes, I want to be a part of what you and your leaders are building.'"
While that was a huge task accomplished in just a matter of months, it isn't the proudest moment of Potter's career. That was at Genentech, when the biotech held record sales and earnings growth for 19 of 20 consecutive quarters and launched seven novel therapies in just five years.
"When I joined Genentech, we thought we might have two product launches," she says. "We never anticipated that everything would just keep growing the way that it did. I was co-chair of the Product Portfolio Committee, and we created the most valuable drug pipeline in the world at that time. That's something I'm very, very proud of. We really made a big difference for a lot of patients."
Charting the Future of SMPA
Potter is also proud of SMPA's endeavors to deliver needed therapies to patients who live with difficult to manage conditions.
The company’s portfolio includes treatments in psychiatry, neurology, oncology, urology, women's health, and gene therapy for cystic fibrosis. One of SMPA’s most interesting projects is a treatment for children with congenital athymia, an ultra-rare disease characterized by the absence of a functioning thymus. Most congenital athymia babies die within three years, even with supportive care.
The product, Rethymic, was approved by the FDA in November 2021; it’s the only FDA-approved tissue-based treatment for this condition.
"Rethymic greatly improves [the] survival [rate] for these children," Potter says. "There may be only 20-25 of these children diagnosed in the United States a year, but we care greatly about all of them."
Another SMPA objective is to use technology and AI to advance new technologies to transform operations through the acceleration of digital.
"Our industry is changing so much and one of the things that's really exciting is we truly are leveraging technology in a way that other companies just simply aren't doing," Potter says. "When you're thinking about driving innovation and cultivating leaders, you need to think about who is comfortable talking about AI and natural language processing and using it to solve problems."
Sumitomo has built two in-house technology platforms that use the latest generative AI and machine learning tools to improve the efficiency and effectiveness of research to find new targets, clinical trials, and commercialization of new therapies.
Leonard Mazur looks back on a 50-year career in pharma and forward to the anticipated launch of new treatments for three health concerns.
Leonard Mazur is a 50-year veteran in the pharmaceutical field with a myriad of product launches, patents, and titles under his belt. But it wasn't until he turned 50 that he decided to become an entrepreneur and started establishing his own companies.
Today, in his late 70s, he is steering Citius Pharmaceuticals toward anticipated launches of potential first-and-only prescription treatments for three health concerns and a next-generation, scalable stem cell therapy program.
"I didn't really go entrepreneurial till I was 50 years old, which is the worst possible age that you can try something like this," the co-founder, CEO, and chairman of Citius Pharmaceuticals says. "I remember I used to bring documents home for my wife to sign, signing over our house, to keep everything going."
When Mazur commits to something, he says, he's all in.
"I put money directly into the company, as does my business partner and co-founder, Myron Holubiak," he says. "We both have millions of dollars invested in the company. That's something you normally don't see."
This strategy sets up Citius Pharmaceutical in the enviable position of having little to no debt burden.
A long career path
Mazur spent the first 10 years of his pharma career at Cooper Laboratory, where he got his taste for working in smaller, faster moving environments.
"It was a dynamic company led by a dynamo CEO and I loved the environment," he says. "Smaller companies are much more driven towards getting results quickly."
Leonard Mazur, CEO of Citius Pharma. Photo courtesy Citius Pharmaceuticals.
He also worked in various roles at the Medicis Pharmaceutical Corporation, ICN Pharmaceuticals, Knoll Pharma, and Cooper Laboratories. He then founded Akrimax Pharmaceuticals, Triax Pharmaceuticals, and Genesis Pharmaceuticals.
Prior to its merger with Citius in March 2016, he was the co-founder and chairman of Leonard-Meron Biosciences.
Years later, Mazur refers to his time at Cooper Labs as setting the foundation for his success and setting him on the path to entrepreneurship.
"There was no formal training program, but you did have a chance to witness decision-making firsthand," he says. "Being a part of a company like that, I found that you learned very quickly a lot about the business in general. So that all played an important part for me as far as my own development and my own way of looking at things and also, in all likelihood, my own desires as far as pursuing an entrepreneurial career."
The future at Citius
Citius has three potential first-and-only prescription treatments in their indications and a next-generation, scalable stem cell therapy program in the works.
Lymphir, a purified reformulation of denileukin diftitox, is waiting for FDA approval as a cancer immunotherapy treatment for a rare form of non-Hodgkin lymphoma. Citius filed a BLA in 2022 for the treatment, and earlier this summer the FDA asked the company to incorporate enhanced product testing and additional controls during the market application review. There were no concerns relating to the safety and efficacy clinical data package submitted with the BLA, or the proposed prescribing information. Citius plans to complete the CRL remediation activities by the end of the year and file the resubmission in early 2024.
Two other products are Halo-Lido, which could become the first FDA-approved prescription product to treat hemorrhoids in the United States, and Mino-Lok, an antibiotic lock solution to treat patients with catheter-related blood stream infections that Citius has licensed from The University of Texas MD Anderson Cancer Center.
The company is also working on a stem cell platform for the treatment of respiratory conditions associated with acute inflammation, with an initial indication in the treatment of acute respiratory distress syndrome (ARDS).
Citius' most recent news is its merger with TenX Keane Acquisition, creating a separate oncology company, Citius Oncology. Citius Pharma will continue to focus on completing the Mino-Lok trial and evaluating next steps with the Halo-Lido program. The acquisition is expected to be finalized in early 2024.
"Our objective there is the NASDAQ listing that would come with this backing," Mazur says. "That's the primary reason we're doing it that way. Once it starts trading, we don't have to go through a whole separate process to get NASDAQ-qualified, which will enable us to raise the funds a lot quicker. The objective is to raise the funding to launch, without diluting the Citius shareholders any further. Down the road we would start distributing the shares in a subsidiary to the Citius shareholders."
Growing as a leader
Mazur says he has honed his leadership skills over the years to focus on motivating every person in the company and creating a work environment that values everyone's input. To do that, he believes in "giving everyone all the space that they need" to take responsibility for their work.
"I think if you've got professional people employed, they are the ones that have to make the decisions,” he says. “You are relying upon them to make the right calls to make things happen."
It’s also important, he says, for employees to feel they are a part of the company's success and have the opportunity to participate in that success.
Mazur says he learned from a former boss that stock options are a great way to create that atmosphere.
"Every single person from the lowest to the highest position has stock options in our company," he says. "In reality, I think stock options are an important motivational tool for everybody. And over the years I've had people that received stock options from years ago, come up to me and thank me for it because they were able to pay for their children's college tuition and other things of that nature. So, you know, it usually works out very, very well."
To lead successfully, Mazur says, one has to first believe in oneself, as well as one’s ideas.
"You have to have confidence, you have to believe in what you're working towards, and what you have in your portfolio," he says. "When all those things come together, it gives you a strong feeling that you will succeed. From the beginning, one of my favorite sayings has always been 'Failure is not an option.'"
One of Mazur's many career highlights occurred at ICN Pharmaceuticals. He had been brought in as vice president of sales and marketing to launch Virazole, the brand name for ribavirin, to treat RSV in infants.
"I love our industry," he says. "I think it's a great industry to be part of because you get involved in something and you never know how it's going to work out. For example, ribavirin was the very first drug approved for infants to treat a rare respiratory virus, called RSV. Most of the RSV episodes would occur in a neonatal intensive care unit. And if the infants were immune compromised, they could die.”
“The great thing that happened was [that] once the drug first launched, I can't tell you the number of times we received phone calls at the office from parents, nurses, and doctors thanking us for the availability of that drug because it had saved an infant's life,” he says. “I remember telling everybody in our small marketing group at the time: 'Pay attention to this moment. It's never going to get any better than this. This is what we're here for. What better thing can you experience than being a part of saving infants' lives?'"
Born in Germany, Mazur emigrated to the U.S. when he was young, and later in life became a recipient of the Ellis Island Medal of Honor. The medal is presented to those who immigrated to the United States during the Ellis Island era and have shown an outstanding commitment to serving the United States either professionally, culturally, or civically.
Mazur believes strongly in giving back, and his charitable contributions have been many.
“I was deeply moved to receive the Ellis Island Medal of Honor," he says. "The United States is the only place in the world where immigrants have access to opportunities that far exceed those from where they came. I am very grateful to have been able to tap into those opportunities while celebrating my Ukrainian heritage, which has always been central to my personal growth and professional success. I am living the American Dream and feel honored to work in the healthcare sector where my team and I continue to strive to improve the everyday lives of those in need.”
Mazur has made two significant contributions to his Pennsylvania alma maters: West Catholic Prep High School and Temple University. He says he believes in the value of a liberal arts education and credits his undergraduate degree in psychology from Temple with his entrepreneurial success.
A few years ago, he and his wife donated $5 million to support scholarships and professional development opportunities to students in the College of Liberal Arts. in recognition, Temple is renaming its liberal arts building from Anderson Hall to the Leonard and Helena Mazur Hall.
The Mazurs also donated $5 million to his high school, West Catholic Preparatory, of which he is a member of its Hall of Fame. It’s the largest single donation in the school’s nearly 100-year history.
"I totally believe you have to give back," Mazur says.
Driven at the age of 11 to educate himself on math, science, and computer engineering, he used college textbooks handed down from family members to learn calculus and college-level chemistry. He started his own training in computer engineering while in the 10th grade, learning to run certain software that required adding a different type of processor.
"By then I'd learned enough electrical engineering to make it feasible, and I ran that hybrid system for a couple years," he says. "I've never been happy with what computing has to offer. And so that's why I started designing computers to do things, so we can answer time-sensitive questions today, rather than waiting five or 10 years for the commodity market to give us what we need."
Nicholas Nystrom, chief technology officer of Peptilogics. Photo courtesy Peptilogics.
Nystrom received his PhD in computational chemistry from the University of Pittsburgh. Since then, he has created several innovative platforms, each enabling up to 30,000 users to conduct more than 2,500 projects, particularly in AI for the life sciences.
"I've been doing computational science for my whole career," he says. "I could see the ability of computational science to help us look at things we can't observe experimentally."
After 28 years leading scientific research teams at Pittsburgh Super Computing Center, Nystrom moved clinical-stage biotechnology company Peptilogics. He was excited by the opportunity to speed up lead compounds to patients.
"We're trying to get to those lead compounds as fast as possible," he says. "We are striving to make more of a difference."
He was also a part of the partnership between Carnegie Mellon University and the University of Pittsburgh, sponsored by the National Science Foundation (NSF), that created the supercomputer Bridges.
"Around 2014, I designed the first computer in the world that brought together high-performance computing, artificial intelligence and big data," he says.
This was at a time when high-performance computing had not yet been used by researchers in other fields, so Bridges was designed to enable these researchers to work easily with supercomputing.
Bridges beat the world’s best human poker players, improved predictions of severe weather to lengthen warning times, and offered gene researchers an easy-to-use tool to assemble the largest DNA and RNA sequences, according to Carnegie Mellon. In 2019, Bridges’ AI capabilities were enhanced with the latest GPU technology, fueling more sophisticated AI work on Bridges. In 2021, an advanced version, Bridges-2, was launched, integrating new technologies for converged, scalable HPC, machine learning, data, and more.
Nystrom was Peptilogics’ fifth employee, joining in 2021 as SVP and head of computation and data, with the goal to scale drug design using generative AI, HPC, and physics-based simulation. He was promoted to chief technology officer one year later.
He had met Peptilogics CEO and founder Jonathan Steckbeck in 2020 and discovered they had the same long-term vision: to use computational science to scale therapeutics design. Steckbeck's vision was to take what he had done through biochemistry and wet lab work to make that scalable through a machine learning approach.
"At Peptilogics, we recognize that AI is transforming the life sciences," Nystrom says. "Where we are focused today is in developing therapeutics. We are focused on being very general, being able to treat arbitrary targets and arbitrary therapeutic areas and that makes it scalable. That means we can go after much more in the long run than we were ever able to do historically. And that's what I was enthused to do."
Nystrom says the biotech is researching very diverse biological targets and diverse diseases that range from membrane proteins down through the target in the cell nucleus.
“We're looking at diseases covering rare disease or genetic disease, cancer and immunology, with others in the pipeline and we are focused on algorithms which led us to the capability to treat general targets and hence general therapeutic areas, rather than having a specific focus on the target class or disease,” he says. “In fact, we could not have done what we did just for one disease because there would not be the data algorithms that can work with finite biological data."
This progressive work environment requires a certain culture of open-minded thinkers, he says. As CTO, Nystrom has built these teams from the ground up.
"The team we have built is focused on people who are very inquisitive, who embrace continuous learning, because this field is moving so fast, and people who really want to make that transformative difference," he says. "It's a very interdisciplinary team.”
“As a leader, I bring a culture of thinking broadly, recognizing people have deep expertise in science and in machine learning, but that everyone is always learning something from others, because there's never anyone who's a master at all, including myself,” he adds. “The biggest challenge in this field is not the implementation, because we know how to do that. It's that continuous learning culture and finding the people that actually have this forward-looking mindset of doing things in a new, better way without saying this is the way I've always done it. And so that's what we hire for, people who embrace that constant curiosity."
This type of culture also requires tackling problems from a "monkey first" mentality, he says, referring to the theory of Astro Teller, the CEO of X, Google's innovation hub, who believes prioritizing the most difficult challenges of a project first is key to success. For example, if your objective is to have a monkey stand on a pedestal and recite Shakespeare, you start by teaching Shakespeare.
"If you build the pedestal first, you will feel like you're making progress because it's easy to build a pedestal," Nystrom says. "But in the end, the really hard thing is teaching a monkey to recite Shakespeare."
At Peptilogics, the principle is to start with the hard thing.
"We bring these different complementary pieces together between understanding science, understanding artificial intelligence, [and] understanding how to compute them, and make them run really well," he says. "And then get to work."
And there is plenty to work on. A 2018 paper, illuminating the druggable genome project, determined only 3% of known targets have been commercially drugged. It identified 62% of targets as having chemical or biological support, many of which Nystrom and his team expect to go after.
Development and application of machine learning architectures and models will create safer, more efficacious medicines and help us to understand key aspects of systems biology that drive disease.
"That's where AI-driven design can potentially make a truly meaningful difference," Nystrom says.
The company's US head for the cardiovascular portfolio is leading the effort to find new treatments for women living with heart disease, especially those in underserved populations.
Elena Livshina came to the cardiac space by falling in love. Not with a person, but through a new discovery.
Back in 2016, the US head of the cardiovascular portfolio at Boehringer Ingelheim was deeply involved in the diabetes market as senior brand director at a company developing treatments for diabetes care, obesity care, hemophilia care, and growth hormone therapy.
When study results showed that a diabetes drug that she had been working with for years also reduced the risk of heart disease, she says it was "shocking" to suddenly discover that the drug had more than one benefit.
Elena Livshina, US head for the cardiovascular portfolio, Boehringer Ingelheim. Photo courtesy Boehringer Ingelheim.
"It surprised us all to discover that the diabetes drug was actually good for people with cardiovascular disease," she says. "And that's when I fell in love. I have to be honest, that was an amazing time."
Livshina shifted her focus from diabetes and became immersed in the cardiac therapeutic arena, creating ways to educate physicians and patients alike about the benefits of using anti-diabetes therapeutics as a cardiac treatment.
She began by leading the launch of the diabetes drug as a cardiovascular drug, which was the first in its class to move in this direction. It was a once-in-a-lifetime opportunity to introduce a new way of treating heart disease.
In 2021, Livshina took the reins of Boehringer Ingelheim's cardiovascular portfolio. A drug in that portfolio had been established as a diabetes treatment in 2014, but recently was approved to reduce the risk of cardiovascular death plus hospitalization for heart failure in adults with reduced ejection fraction.
The newer indication prompted Livshina to take on her next mission: To close the gap in care for women living with heart disease.
Through study results, Livshina and her team identified a huge disparity between heart disease care for men and care for women, and a more prominent difference in care between white, Black, and Hispanic women. She and her team decided to tackle this discrepancy head-on by creating unbranded initiatives that would address underserved populations.
"We had to go deeper than just women," Livshina says. "We developed a campaign for Black and Latino women to see what they needed, and what we as a company can do to bring education and practical tools to these women and their care partners. Unless we addressed those patient populations' needs and developed programs specifically for them and by them, we would not be successful."
Working with the Lilly Alliance, Livshina's team created Hear Your Heart, a healthcare initiative that provides resources and education for women living with heart failure, especially Black and Latina women.
"We needed to raise awareness of the issues and explore how [we can] help patients in very different ways, because it's not just about the medicine," she says. "We have an obligation not just as an industry, but as a healthcare community, to do more."
The campaign produced an immediate effect within the women's heart health community.
"What surprised me was the impact that it made from the very beginning," Livshina says. "We produced brochures and the website, and just by starting that conversation we could see already a ripple effect. Our next step is to offer unbranded content to organized health systems and providers, because it's important to them. It's important to everyone, quite honestly."
The campaign has seen 19.9 million social impressions, 1.7 million video views, and 2,400 resource downloads.
"The campaign stands out because it is specific to addressing gaps in health inequality, and really going deep into particular patient population groups and seeing what can be done there," Livshna says.
Establishing leadership values
Livshina uses her 19 years of work experience and her passion to motivate her team members. Being a woman in the industry has its challenges, so she helps others, especially women, to step up and take on challenges that stretch their skill sets.
"I love mentoring women, because you know what? We can do it," she says. "A lot of times … we are our own worst enemy, holding ourselves back."
She says mentoring is one of the more rewarding parts of her job, especially when she witnesses others being courageous and successful in taking their career to the next level.
"That's something that is extremely satisfying for me," she says. "I get goosebumps just thinking about [it]."
Much of her strength as a leader comes from her personality, she says.
"What's critically important about me is that I'm an extreme extrovert, and I like working with people," she says. "I love working in teams. But another very important skill is being open to learning from others."
She creates a learning culture within her teams so that everyone, no matter the level, is encouraged to speak up and express their point of view.
"This is so critically important, because two heads are always better than one," she says, "I firmly believe that you don't have to be the most senior or the most paid or have the highest title to have the best ideas."
A global perspective
Livshina describes herself and her family as "global people." Born in Russia, she has lived in Denmark, Switzerland, the UK, the Czech Republic, Australia, Canada, and the US.
"I am a Russian who married a New Zealander and we have American children," she says.
Growing up, Livshina says she loved math, and decided to start her career in finance. However, once she started working in the banking world, she found it didn't fulfill her need for purpose. "My career switch was very intentional," she says. "I went back to school, and that's how I ended up in pharma."
The move to the US was strategic, because it presented the type of career opportunities she was looking for.
"I really wanted to get to the US because it was the biggest market in the pharmaceutical space and I wanted to get closer to the patient and physician." She says. "I wanted to get closer to the market. So we moved about 10 years ago to the US from Switzerland, and I will never regret it. We absolutely love it here. Professionally, as well as personally, we've had the most amazing experiences."
Livshina and her husband still love to travel, and when they aren't working they are exposing their American-born children to all the different cultures of the world.
"We really enjoy bringing our kids to different worlds because it gives them exposure to different cultures, people, and experiences," she says, noting the family has been to New Zealand and the UK this year.
The chief scientific officer at Ferring Pharmaceuticals USA, born and raised in Nigeria, has seen the dire consequences of inequalities in women's healthcare play out before her eyes. For example, 20 years ago in Nigeria her young cousin, Georgina, died from undiagnosed uterine cancer.
Growing up observing a Nigerian culture where women and men were not treated equally, Garner developed a lifetime mission to swing the pendulum to a more balanced position in women’s health--not only in Nigeria, but around the world.
"I often speak about having close relatives in Nigeria who are literally dying from conditions that women just shouldn't be dying from anymore," she says. "While there has been progress made in some areas of women's health, as long as there are women dying from conditions as common as pregnancy, I feel like we've made virtually no progress."
Elizabeth Garner, chief scientific officer at Ferring Pharmaceuticals USA. Photo courtesy Ferring Pharmaceuticals USA.
In the US, for example, the maternal mortality rate is increasing, not decreasing.
"As far as its rates of maternal mortality, the US is looking very much like a developing nation," Garner says. Worse yet, Black women are approximately three times more likely to die from a pregnancy-related cause compared to white women. There are many causes for this, including lack of access, distrust of the healthcare system, and ongoing racism.
Among the health challenges faced in maternal care is the threat of preeclampsia, which occurs when new blood vessels developing in the placenta don't work properly, leading to high or erratic blood pressure for the mother. The condition is commonly treated with magnesium.
"Preeclampsia is a major cause of death during and after pregnancy in this country," Garner says. "And we still treat that condition with intravenous magnesium. How is that possible in 2023?"
Breaking Down the Challenges in Women's Healthcare
Garner says more research is needed to identify and understand the threats to women's health, including conditions that happen only with women.
"We need desperately to just understand the science of women's health," she says. "Even as women, we don't understand most of what happens to us. Without the science, how do you develop the medicines to address these conditions?"
But more research also creates a need for more investment in developing treatments for women's health conditions.
"In women's health the industry has underinvested for so many years," says Garner. "In 2020, only 1% of the R&D spend of $200 billion went to conditions that solely affect women."
Recently, while speaking before an audience of investors, Garner asked how many had invested in women's health.
"There was nobody in that room who had invested in women's health, and incidentally there were only about four women in the audience," she says.
There is a perception in the industry that there is no profit in women's health, but Garner says that's a wrong assumption.
"There are huge opportunities to make money and we need to get the business case out there so investors will figure that out," she says.
Another factor is that women may be hesitant to talk openly about their health. Women as patients need to shoulder some of the responsibility for their healthcare, Garner says, meaning they need to speak out about uncomfortable topics and clearly outline symptoms for their physicians no matter how embarrassing it may feel.
"It's time women moved beyond the stigma of women's health conditions and speak honestly and descriptively to their practitioners," she says. "This is a global issue. The US has the same issues around stigmatization of women's conditions as in Nigeria. I firmly believe that the fact that historically women have not been comfortable talking about all their issues is a big reason why there hasn't been the attention paid to women's health needs."
Garner says she is motivated to create ways to reduce the stigma so that patients will open up more about their conditions, allowing physicians to learn how to address those issues. She says programs like Fertility Out Loud and Safe Birth are helping to increase this awareness.
Ferring partners with advocacy organizations, such as Resolve, to increase education as well as government organizations to increase access.
"For me, it's all about access and making everything available, no matter where or who you are," she says.
Another factor that will help improve women's healthcare, according to Garner, is putting more women in the top seats.
"The more women in leadership roles, the more we'll see a true investment in women's health, because women understand there is absolutely money in the space, and a need, because we are willing to spend the money to improve our health," she says.
Calculated Risks on a Career That Resonates
With 30 years of experience in reproductive medicine and maternal health, Garner is using her passion and her professional skills to move the needle on improving women's health globally. Her goals include becoming the CEO of a women’s healthcare company.
Prior to joining Ferring in 2022, she held chief medical officer roles for ObsEva and Agile Therapeutics, both women’s healthcare companies. At Agile, she led the Phase 3 clinical development of Twirla, a low-dose contraceptive patch. In 2010, at Merck Research Laboratories, she was instrumental in obtaining FDA approval for Gardasil, the human papillomavirus (HPV) vaccine.
Taking calculated risks has been her MO from the beginning of her career, when she switched to OB GYN practice during her residency. She then jumped from clinical practice to industry, which at that time was an unheard-of career move.
"I definitely got a lot of pushback," she says. "But I wanted to have more impact on patients than I could by caring for one at a time. It was certainly worth that risk."
As a leader Garner gets a boost from showing others how to take calculated risks and stretch themselves in their job.
"What I enjoy the most is working with teams, and growing people, and allowing them to shine," she says. "That aspect of drawing people out and showing them what they can do is one of the things I like best about being a leader. "
The New Jersey-based mom of three takes her downtime seriously and enjoys spending it at home with her family.
"I'm a family person. I have three children, two sons and a daughter, all born in July," she says. Her oldest recently got married, and her youngest lives with autism.
"I love the opportunity on the weekends to just literally be home, just sitting with my son," she says.
Ryan Davies' career has been defined by taking risks. His newest company is taking on one of healthcare's biggest challenges.
The biotech industry isn't for the faint of heart. And that's why Ryan Davies jumped at the chance to lead CancerVax, a high-stakes, pre-clinical biotech partnering with UCLA to develop immunotherapy cancer treatments that use the body’s immune system to fight cancer.
CancerVax's mission is to develop a breakthrough universal cancer vaccine. That, Davies says, is more than just hairy and audacious.
It's "big," "high-risk," and "unique," he says. And right up this serial entrepreneur's alley.
"This opportunity really spoke to me," he says. "I like taking risks. I love diving in and achieving difficult goals. CancerVax is an exciting chance to make a difference."
A risk-taker by nature, Davies' entire career has been entrepreneurial. As a young man, he had anticipated a career in business management, but he began founding companies straight out of college. He established companies in e-commerce, technology, and software, and worked in energy and investments, before he fell in love with the challenges of biotech.
Ryan Davies, CEO of CancerVax. Photo courtesy CancerVax.
"My first company was a software ecommerce company, and I sold it and then started and sold another one. And then started and sold another one, until I ended up in the venture capital space," he says. "While there, we found a really cool technology being developed between the biology and chemistry departments at a major university. We licensed it and built a company around this crazy idea for developing a new class of antibiotics. I was put in as the interim CEO with no science experience. I was a business guy. But I absolutely fell in love with the biotech space."
The company was Curza, which he led for seven years, educating himself with For Dummies books about cell biology and attending organic chemistry classes. The 'crazy idea' developed into the CZ-02 platform of antibiotics for the treatment of drug-resistant bacterial infections. The CZ-02 platform represents a novel class of antibacterial agents with a unique mode of action.
The science learning curve in this experience was "tricky," Davies says, but not knowing everything upfront was also an advantage.
"One of the reasons that I've had success in the biotech space is because I don't know what I don't know," he says. "In the beginning, we were renegades. We didn't know that there was a very specific way of developing a drug, so we went down avenues and did things that were very atypical and very different. And I actually think that worked to our advantage."
Davies' biotech background has grown. He has co-founded several biotech companies, including wound-care company Advanox and medical device company Purgo Scientific.
One of the biggest challenges facing an "outsider" in the industry, he says, was learning to shift gears to align with the slower pace of drug development.
"I remember going straight from the business world into a partnership with a major university on the first biotech that I worked on, and how frustrated I was at the slow pace of academia," he said. Later, during a meeting with the US Food & Drug Administration (FDA), a staffer told him that the government process was going to be frustrating because the FDA wheels grind slow.
"'We're not fast like academia,' he said to me, and I nearly fell off my chair," Davies says.
He says he had to reframe his mindset to a more realistic view of the speed of the development process.
"I looked at other biotech companies and compared how long it took them to get to market," he says, "and as long as I'm moving at a pace that's a little quicker than what some of these companies have done historically, then I'm OK with that."
Davies took the reins at CancerVax in 2022 and is already applying his entrepreneurial skills to its two leading programs. CancerVax is focused on a novel universal cancer vaccine targeting multiple cancers, and a single disease immunotherapy targeting Ewing sarcoma.
According to Bloomberg Business, the cancer immunotherapy market is expected to grow to $196 billion by 2030. Davies believes immunotherapy drugs like Merck's Keytruda and Opdivo from Bristol-Myers Squibb, which started out as niche products and became blockbusters with multiple cancer indications, are proof that not only is immunotherapy a growing and successful market, but also an opportunity for hope for cancer patients.
The newer cancer vaccine program involves creating a methodology that only targets cancer cells and leaves healthy cells alone.
"What is unique about cancer cells is there are markers specific to the cancer cells that healthy cells don't have," he says. "But most immunotherapy treatments today overlap between the cancer cell and the healthy cells, and a lot of healthy cells get killed. We're trying to correct that."
Davies cautions that this is not a preventative vaccine. Rather, it's a proactive approach to treatment.
The second disease-specific immunotherapy program is targeted at treating Ewing sarcoma, a rare and deadly bone and soft tissue cancer that affects children and young adults. There are currently no treatment options available for the disease, which has a high mortality rate and is diagnosed in only 200 to 250 patients a year.
In partnership with CancerVax, the Ewing Sarcoma Program at the UCLA Jonsson Comprehensive Cancer Center has made positive progress toward developing a treatment. This was CancerVax's first partnership with UCLA; the company has raised $2 million of seed funding to date and plans to raise an additional $10 million to further the development of these two programs.
Sluggers and Success
The Utah-based father of eight is a huge baseball fan. And surprisingly, given his location, his favorite Major League team is the Boston Red Sox.
"I love the strategy and the statistics behind baseball and my wife is a fan as well," he says. "We enjoy traveling and hearing live music, but we also like going to Major League ball games. It's something we like to do together."
He says baseball is not all that different from biotech in terms of strategy. Like legendary Red Sox sluggers Ted Williams and Carl Yastrzemski, biotech leaders need to position themselves for the big wins without swinging too fast or too hard out of the gate.
Since the onset of sports analytics, building a baseball team has changed dramatically, as has research that uses healthcare data to fine-tune the development of medicine. Some days you win, and some days you don't.
"Biotech is high-risk," Davies says. "It's an expensive business and it takes a long time to get established. But there's a lot of different areas to pursue, so if we fail in one area, there are 100 different ways that we can pivot."
