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."
However, influenced by his physician-father, graduating from Princeton, becoming a lawyer, being a cancer survivor, rising to COO at Seattle Genetics, serving as CEO of Silverback Therapeutics, Dobmeier then joined Chinook Therapeutics in April 2019. His career path may have been haphazard, but his goal at Chinook is crystal clear: Make dialysis and kidney transplant unnecessary for patients living with rare, severe chronic kidney diseases including Berger's disease or Immunoglobulin A nephropathy (IgAN), a condition that damages the glomeruli inside kidneys and can lead to kidney disease.
"I didn't have a grand master plan for my career, but I tried to work jobs that interested me, and that I thought I could be good at and challenged by," Dobmeier says. "My focus has been on doing meaningful work, challenging myself and putting myself in situations where I felt I could make a difference. It worked out."
Dobmeier might not have planned to head a biopharma as part of his career, but his timing couldn't have been better to have started. In the past few years, there has been an increased focus on glomerular diseases. For example, in December 2022,Tarpeyo (developed by Calliditas) became the first FDA-approved product specifically for IgAN, while in mid-February 2023, Travere gained approval for Filspari, according to a report by Spherix Global Insights.
Dobmeier thinks Chinook could be next in line to receive FDA approval for its two lead drugs for IgAN. Chinook's lead program is atrasentan, a phase 3 endothelin receptor antagonist for the treatment of IgAN and proteinuric glomerular diseases. BION-1301, an anti-APRIL monoclonal antibody, is being evaluated in a phase 1/2 trial for IgAN, with a phase 3 trial initiation planned for mid-2023.
"We have two potentially best-in-class drugs for this disease," Dobmeier says. "Our goal is to make dialysis and kidney transplant unnecessary for these patients. We hope that by having different therapies with different mechanisms of action, we can turn IgAN into a truly chronic disease and allow patients the possibility to outlive it. We've got a good chance to make a big impact on these patients."
It is that impact on people's lives that drove Dobmeier out of law and into life sciences.
"I'm a recovering lawyer," he says. "I practiced law for almost 10 years in different capacities and I realized I didn't want to do litigation. I really wanted to be involved with helping companies build their businesses and doing deals."
As an attorney, Dobmeier worked with high-tech companies, but he felt his work was not improving the lives of others as much as he hoped. With his physician-father, he had always been interested in medicine. After working with Seattle Genetics as outside counsel, Dobmeier was offered a job as in-house counsel. He accepted.
"One of the things that really attracted me to biotech is the possibility of helping people live better, healthier lives," he says.
He stayed 16 years at Seattle Genetics, which was "a wildly successful company," he says, although it had its share of failures over that time period as well. After serving 16 years at Seattle Genetics and rising to COO, Dobmeier moved to Silverback Therapeutics as CEO, a position that lasted six months.
His next step was to lead Chinook into its next chapter of growth.
"I've always been someone who's willing to say, 'sure, I'll try it. I'll figure it out.' Not being afraid to try new things has really helped me in my career," he says.
One of Dobmeier's major challenges when he took the reins at Chinook was keeping pace with the rapid growth of the company during the virtual environment of the pandemic.
"When I started [at] the company, which was about a year before COVID, we had 15 employees in Vancouver, British Columbia, and I was the only employee in the United States. We in-licensed atrasentan from AbbVie and started building a development team, mostly in the U.S. At that time, we were a team of about five people in the U.S., and 18 people in Canada. Since 2020, we've grown very quickly to more than 240 people, all working virtually in both countries."
Dobmeier says the reverse merger with Aduro in October 2020 and going public as a result was a major catalyst for propelling the company forward so quickly.
"In the early days, we were all doing six jobs and it was crazy," he says. "We were a research-only company. Then we did the reverse merger and became a public company all during a pandemic. But you know, sometimes doing the crazy thing and taking some chances pays off and it has so far."
The new Chinook team functioned well working virtually during the pandemic, but Dobmeier would prefer to have more in-person interactions with and between employees. To that end, he is establishing a hybrid operating model at Chinook. He'd like to have employees in the office whenever it is a reasonable request; for example, twice a week, or once a month.
Dobmeier says he recognizes the benefits of a virtual environment, such as the ability to hire the best people regardless of their location, but by not having people together face-to-face he feels employees miss opportunities for greater creativity and teamwork. But the days of working five days a week on site is a thing of the past.
"I think a lot of CEOs want to see more people in the office and a lot of employees don't really want that," he says. "We'll have to figure out the right balance, because we're not going back to five days a week in the office. "I think everyone in the industry is struggling right now with what does the 'new normal' look like."
In the years before joining Chinook, both Dobmeier and his wife were diagnosed with cancer, making them living examples of the benefits of life saving treatments developed by the industry. Both are doing well today, but Dobmeier was diagnosed with lymphoma while at Seattle Genetics and his wife was diagnosed with breast cancer in 2018. This experience solidifies his commitment to developing treatments that improve and save patient lives.
"It really brings the mission home and personalizes it and drives home the importance of what we're doing," he says. "If not for the work that biotech and pharma companies have done, my wife and I might not be alive today. This makes my work with Chinook very meaningful."
Editor's note: This story was updated on June 2, 2023.
As an advisor to hundreds of therapeutic and tech startups over the years, this innovator observed both good and bad business processes.
Nevan Elam, JD, CEO and founder of Rezolute, practiced law for more than two decades, working as an advisor to companies in the life sciences and tech industries, when his real passion emerged from the experience. His calling was not driven by high-tech solutions and corporate law, but rather children, in particular, children with rare diseases in need of new treatments.
As an advisor to hundreds of therapeutic and tech startups over the years, Elam observed both good and bad business processes, which prepared him well to launch his own biopharma.
"I spent a good number of years as a partner in a law firm in Palo Alto, and I saw what works and a lot of what doesn't. And it was there that I also discovered what my passion really was, and it wasn't about all things bits and bytes. Working with therapeutic company clients was the most personally rewarding to me because they were making a difference."
The father of three says there's nothing more impactful for him than working to help children.
"If you can actually make a difference in a child's life, there's nothing like it," he says. "I witnessed that in my prior roles and that spurred my interest in creating Rezolute."
Elam established the clinical stage biopharmaceutical company in 2010 with a focus on developing transformative therapies for rare and metabolic diseases, specifically therapies targeting congenital hyperinsulinism and diabetic macular edema therapeutics (DME).
Saving children from hypoglycemic brain damage
Congenital hyperinsulinism is a genetic disorder in which the insulin cells of the pancreas secrete too much insulin. Rezolute's lead candidate, RZ358, is in Phase 2b development. In 2022, the RIZE study exceeded expectations for correction of hypoglycemia, including a highly significant reduction of approximately 75% in hypoglycemia events by blood glucometer. "The treatment substantially corrects the hypoglycemia that these children have experienced," Elam says. "We're poised to start Phase 3 of RIZE this year, which is very exciting for families, physicians, key opinion leaders and all of us at Rezolute."
According to NORD, congenital hyperinsulinism (HI) is the most frequent cause of severe, persistent hypoglycemia in newborns, infants, and children. In most countries it occurs in approximately one out of 25,000 to one out of 50,000 births. About 60% of babies with HI are diagnosed during the first month of life. An additional 30% will be diagnosed later in the first year and the remainder after that. With early treatment and aggressive prevention of hypoglycemia, brain damage in these children can be prevented.
"RZ358 will enter its Phase 3 program mid-year this year and is expected to be complete and announced in the first half of 2025," Elam says.
No more needles in the eye for Diabetic Macular Edema
Rezolute's DME candidate, RZ402, is a plasma kallikrein inhibitor in a pill form instead of a shot that would radically change the treatment of patients with DME. The disease is treated historically with a monthly anti-VEGF (vascular endothelial growth factor) injection into the eye. This uncomfortable method of delivery reduces patient adherence and can affect adequate dosing.
"Our exciting oral therapy for DME will be a potential game changer," Elam says. "We just started a Phase 2 study in the US, and we'll be doing it at 25 sites this year, and with 100 patients for proof of concept to demonstrate that a daily tablet can actually reduce the swelling in the macula and also prevent a vascular leakage. If we're right, it'll have a tremendous impact on how we actually treat these patients."
The study should be completed this year and Rezolute plans to announce the results in Q1 of 2024.
"With our success in our clinical studies to date, we've been fortunate to raise enough capital in the last year to be able to fund both those programs," Elam says. "So now it's all about execution."
Leadership skills and team building
Before Elam launched Rezolute, he knew what kind of leader he wanted to be as a CEO, and he knew what kind of team he wanted to build. Throughout his career, he had encountered egocentric leaders and that was a model did not work for him. He decided he would get to know his employees as individuals, work to understand their motivations, and allow them to contribute in the best way possible for both parties. At Rezolute, he has intentionally created a collaborative work environment where individuals have the freedom to voice their opinion, excel in their expertise, and be included in discussions.
"I pride myself on building a company that's really focused on the common mission and goal but is also an environment where individuals have the freedom to contribute and are encouraged to be forthright and bring their expertise to the forefront," he says. "This is how you create a good team and retain, as well as excel, with individuals who love where they work. We've been very successful at building that type of team."
Reflecting on his own leadership style, Elam says the most effective leaders are those that work in the spirit of collaboration, "where egos are left at the door."
"Part of the job as a CEO, especially with respect to the senior leaders, is doing your best to understand the individuals," he says. "That's how I operated as a senior executive, even before becoming a CEO and a founder. And definitely the way I operate as a CEO today."
Elam manages the stress and responsibilities of running the biopharma by taking three- to four-hour bike rides through the redwoods and mountains near his California home. "Staying active is my superpower. It's my therapy," he says. Each ride averages about 40 miles at about 5,000 feet of elevation, over the hilly range that runs along the Pacific Ocean.
"It's a pretty intense ride but so beneficial in terms of getting outside, seeing the redwoods, seeing the ocean and getting amazing exercise," he says. "My favorite thing is to get under the 2000- year-old redwood trees; those giants put everything into perspective."
Elam also spends time contemplating the works of Nietzsche, Plato, Aristotle, Kant and others. "Exploring philosophy is a very different hobby than probably most folks have who are in drug development, but I am fascinated by what makes us human and why it is we do what we do."
And it is that type of deep thinking and seeking understanding that brought Elam and his team at Rezolute to continue advancing toward two major milestones in the coming years: the approval of RZ358 and RZ402. These novel treatments could fill an unmet need that would have a long-term impact on the lives of patients with diabetes and children with congenital hyperinsulinism.
And that puts Elam and Rezolute on track to make a meaningful difference in the lives of others.
After years in the industry, Isaacsohn identified gaps in drug development and established a company to solve them.
Editor's note: This article appears in the June 2023 edition of HealthLeaders magazine.
CinRx co-founder and CEO Jonathan Isaacsohn, MD, FACC, wants to speed up the drug development process and provide medicines to patients faster than historically possible. To achieve this, however, his company will never bring an actual product to market. That's not his MO.
After working in the industry for several years, Isaacsohn decided he had seen enough failures in the drug development process. With more than 90% of drugs from biotech companies failing, he took his 30-plus years of experience in the industry and created a solution that would improve the process. The objective was to use abandoned or repurposed drugs that had not been optimized by other companies and create an entire functional company around each product to fully research and develop it to its full potential.
"Many drugs are discontinued because of problems that could not have been anticipated," Isaacsohn says. "Safety issues, lack of efficacy, lack of benefit over and above other approved drugs, are all reasons to be unsuccessful, but so many drugs fail because of ill-conceived strategies and poor execution. That is the driving force behind why we conceived CinRx, and what we aim to solve in the model that we put together."
This model includes several biotechnology companies all supported under one dedicated funding system which Isaacsohn says helps accelerate the process. CinRx is not just a financial investor in these companies. Instead, it establishes a fully functioning company around each candidate and helps drive its success. Most VC-funded companies receive outside funding and operate on their own.
"At CinRx, we do a fair amount of capital raising at the holding company level, but there's an integration between that money and the execution," Isaacsohn says. "That is quite differentiated from the general model of how biotechs get funded."
"We bring funding into the holding company, and we use that funding to help us establish companies or partnerships," he says. "Ultimately each company follows a trajectory appropriate for that company to a point of exit, wherever that may be, early in the process or later in the process. We never intend to be a pharma company."
As an example, the recent sale of CinCor to AstraZeneca was one of CinRx's first steps toward realizing the company vision, which was set in motion nearly eight years ago when CinRx was founded in 2015.
In 2019, CinCor entered in an agreement with Roche to acquire the rights to a novel aldosterone synthase inhibitor (ASI) compound, CIN-107 also known as baxdrostat.
CinCor conducted phase one studies, safety studies, and a proof-of-concept study. The positive results showcased the value of the drug, and that caught AstraZeneca's attention, who bought the portfolio company for $1.9 billion this February.
"What happened with CinCor actually validates the model that we work in," Isaacsohn says. "Each one of our companies ultimately will find a new home."
Other companies under the CinRx umbrella include CinDome, CinSano, Cinphloro, and CinFina.
As Isaacsohn mentioned, CinRx also operates a second model by partnering with existing companies that, for whatever reason, don't have adequate capacity to develop the drug on their own. CinRx partners with the company by investing in it, so it has "skin in the game," and it then drives the development on the other company's behalf. Two of these partnerships currently exist: vTv Therapeutics and Retromer Therapeutics, bringing the total number of portfolio companies CinRx is working with to seven.
It is this multiple shots on goal approach in the drug portfolio that expedites the process. "By sharing resources across companies combined with an efficient process, we're being good shepherds of the funding," Isaacsohn says.
CinRx's pipeline of transformational medicines include therapies for diabetes, gastroparesis, IBS, metabolic and neurodegenerative diseases, and oncology.
Being the CEO of CinRx puts all of Isaacsohn's previous work experience to good use. Throughout his career, he has worked in drug development from early phases through global submissions in various therapeutic areas. He is also a cardiologist, trained in internal medicine and cardiology at Harvard Medical School, and has taught on the cardiology faculty at Yale Medical School. Isaacsohn was one of the founders at the ground floor of global CRO Medpace and worked there for 14 years, until it was sold.
CinRx works closely with Medpace as its development arm, testing and developing the compounds it acquires. This keeps CinRx on the cutting edge of drug development, Isaacsohn says, as Medpace has many of the resources needed to develop a drug and as drug development has evolved, so have they.
After the sale of Medpace, Isaacsohn served as CMO for Teva Pharmaceuticals where he drove development of Teva's specialty drugs and the new therapeutic entities initiative across all therapeutic areas.
This collective experience prepared him well for the challenges at CinRx.
"I have been in this industry for a long time from all different vantage points, and different roles. I'm a trained cardiologist and was in academic medicine. I did the first human study with Mevacor. I was a principal investigator on hundreds of trials. I was among the founding group that established the CRO Medpace. I ran the medical and regulatory side and that's where I got exposed to biotech companies. I did literally hundreds of programs on behalf of other biotech companies, and that's where I learned the trade."
Isaacsohn believes himself to be a democratic leader and he aims to keep the organizational structure flat, giving everybody a role, and a chance to participate in decision making. "We don't have the kind of hierarchy that makes people feel they need to keep quiet, or they can't express themselves. We value everybody's opinion. It's very much a family feel in our group."
Isaacsohn credits his leadership success to his varied experience. "I've been around the block," he says. "A lot of it just has to do with experience."
Editor's note: This story was updated to correct the spelling of Cinphloro. Updated 3/28/23.
The company prioritizes unmet needs for women in contraception, fertility, and vaginal and sexual health.
Sabrina Martucci Johnson never planned on being a founder and CEO of a women’s healthcare company, but after identifying gaps in women's healthcare, she asked herself, “If not me, then who?"
"I'm not one of those people who always wanted to be a CEO," she says. "But I had a light bulb moment: if I don't do this, it's not going to happen."
Over the years, Johnson had always championed for women in her volunteer and charity work, supporting philanthropic activities surrounding STEM education for women, domestic violence awareness, and women's healthcare issues.
On the professional side, Johnson spent her entire career in the drug development industry, in a variety of roles. She began as a research scientist with Baxter Healthcare and went on to hold marketing and sales positions there.
Later, she held C-suite positions, including CEO, CFO and COO at WomanCare Global Trading, a specialty pharmaceutical company in female reproductive healthcare, and before that she served as CFO of Cypress Bioscience, a publicly traded pharmaceutical company.
"I felt there should be a way to match my extracurricular passion with what I do professionally," Johnson says. "There are all these needs of women that are not being met. So, I put my money where my mouth is, and started Daré Bioscience in 2015.
"In addition to aligning her life goals and working toward filling unmet needs for women, Johnson saw the business sense of investing in women's health. Market research shows that only approximately 1% of healthcare research is invested in female-specific conditions beyond oncology, and women’s health conditions outside of oncology comprise less than 2% of the current healthcare pipeline.
Yet, women’s health products outside of oncology make up 27% of the total number of blockbuster products (products that each have over $500 million in annual sales) and contribute 35% of the total sales dollars generated by blockbuster products. "This is why investment in women’s health is efficient and impactful in terms of translating dollars invested in R&D to ultimate revenue generated, and therefore, not only good for 50% of the population, but it is also a good business model," she says.
The company prioritizes unmet needs for women in contraception, fertility, and vaginal and sexual health. Daré’s first FDA-approved product, Xaciato, is a vaginal gel for the treatment of bacterial vaginosis in female patients 12 years and older, which is under a global license agreement with Organon.
Daré’s portfolio also includes potential first-in-category candidates in clinical development: Ovaprene, a novel, hormone-free monthly intravaginal contraceptive whose U.S. commercial rights are under a license agreement with Bayer; Sildenafil cream, a novel cream formulation of sildenafil to treat female sexual arousal disorder utilizing the active ingredient in Viagra; and DARE-HRT1, a combination bio-identical estradiol and progesterone intravaginal ring for hormone therapy following menopause. There are currently no FDA-approved products that continuously deliver this type of hormone therapy with both estradiol and progesterone together.
"It was a little bit shocking that there was no FDA approved product that met the guidelines that the medical experts in the space recommend for hormone therapy, which is the need that the two hormones be administered together," Johnson says. "When we started the company, this is why hormone therapy for menopause was high on our list."
DARE-HRT1 could become the first FDA-approved monthly intravaginal ring delivering such therapy. Due to its positive early phase results, the company has been cleared by the FDA to advance directly to Phase 3.
Johnson said that even though it was out of the ordinary, the company decided to ask the FDA if it would consider allowing them to go straight to Phase 3 trials.
"After the trial results we started discussions with the FDA to propose that we just go right to a single Phase 3 so that we can get this product moving forward and into the market to women," she says.
The trail blazer didn't think it was much of risk to take the request to the FDA, as long as they could pass the 'red face' test, meaning they had justification for asking and the ability to prove it. This was a case of 'you never know until you ask', she says.
"We made an ask that wasn't the normal path, but we scientifically could justify it," she says.
"If you can scientifically make a case for something, there is room to color outside the lines, and DARE-HRT1 is a great example of that."
If the FDA denied the request, Johnson reasoned, Daré would be in the same place as if it didn't ask. But surprisingly, the FDA said yes.
"We were really pleasantly surprised that the FDA came back and said yeah, that's reasonable," Johnson says. "Now we have a lot of work to do to get ready, beginning with formally filing the IND."
Currently, Daré's portfolio only contains products that aim to deliver drugs in a way that's never been delivered before and pushing the envelope on indications that haven't been studied before. Thinking outside the box this way is the company's credo and striving to be first in these areas is not without challenges.
"We are all about the firsts, and firsts are hard," she says, "It's challenging but it's such an incredible opportunity. And I would also say, in the same breath, it's the most fun program to work on in our portfolio because while it's hard to be first, it is also fun to be first and to get to set the stage and blaze the trail."
For example, the company is developing a cream formulation of sildenafil --the same active ingredient in Viagra--for women with female sexual arousal disorder. There's nothing approved for this indication. "We are really blazing a trail getting all the work that goes into a brand-new indication: getting the alignment on what to study, what are the endpoints. Like how to design a clinical trial for this, for starters."
Johnson keeps the company focusing on firsts by creating opportunities for employees to be free and open with brainstorming new ideas and solutions. It is cliché, Johnson says, but to her, there really is no bad idea or dumb question that employees can pose.
"I try really hard to be a very authentic leader," Johnson says. "I have a very authentic style and create very much a culture of dare to be different and be bold. Let's push the envelope. It's okay to suggest something that hasn't been done before. It's okay to put those ideas out there. I very much try to create a culture of 'let's think outside the box and be aggressive.'"
Personal tragedies involving family and friends motivated this CEO to launch Peptilogics.
When thinking of life-threatening surgical risks, hip or knee replacement does not immediately spring to mind. Maybe it should.
No one knows that better than Jonathan Steckbeck, CEO and co-founder of Peptilogics. The replacement procedures aren't necessarily risky, but the infections that can occur after surgery are. Antibiotic resistant bacteria --often staphylococcus aureus-- can accumulate on the hard surfaces of the prosthetic and cause infections that are very difficult, if not close to impossible, to eliminate. This is what can cause knee replacement and other joint replacement surgeries to turn deadly.
Most prosthetic joint infections (PJI) are the result of bacteria already present in the body or introduced during the surgery or other procedures. The usual treatment is additional surgery and antimicrobial therapy, often taking place over a span of many weeks or months. These infections put the patient at a high risk of morbidity and the five-year mortality rate after catching one of these infections is 25%, according to Steckbeck.
"That's not common," he says. "Ever since we've had antibiotics, we've been able to control most types of infections. That this can’t be controlled in this day and age is alarming."
Steckbeck has seen loved ones struggle with PJI, including his father-in-law, who died from antibiotic resistant infection, and a family friend—a fit Navy fighter pilot who required multiple surgeries over 18 months before he was cured of the infection. Steckbeck says the pilot fears that the infection might return.
These tragedies motivated Steckbeck's first entrepreneurial venture. In 2013 he left his work in HIV research and launched Peptilogics.
"There's no effective drug to treat this infection," he says. "When my father-in-law passed, I thought there must be something more we can do to solve this problem."
With a PhD in biochemistry and molecular genetics and an MBA from the University of Pittsburgh, Steckbeck turned his research mind to business. He saw an opportunity to not only improve patient lives, but to also fill an unmet healthcare market need.
While the patient population for PJI is small, controlling devastating infection is life altering for the patient. It can also reduce costs by negating the need for 12 to 18 months of expenstive post-operative treatment. "Per patient direct costs are in the $100,000 range, and the total cost per patient for certain joints can be in the $500,000 range," Steckbeck says. "PJI ends up being a really large economic burden. We have an opportunity to bring a good therapeutic to patients and create a market around that."
More than 1 million total joint replacements are performed in the U.S. each year, a number projected to grow to nearly 4 million by 2030 due to the aging, active population.
PLG0206 is Peptoligics' tool to address persistent bacterial pathogens. It attacks bacterial pathogens within the biofilm that evade standard-of-care antibiotics by targeting and disrupting bacterial membranes to trigger bacterial cell death. If approved by the FDA, the anti-biofilm, anti-infective peptide therapy will be the first specifically indicated for the treatment of periprosthetic joint infection.
Still in investigational stages, PLG0206 has been granted FDA Orphan Drug, Fast Track and Qualified Infectious Disease Product Designations for the treatment of PJI. Peptilogics began dosing patients in October 2022 through its LOGIC-1 clinical trial.
The importance of computational technology
As Peptilogics designs better therapy for PJI patients, it is also pioneering machine learning methods and artificial intelligence that create previously unattainable efficiencies and accuracies. This works not just in biotech, Steckbeck says, adding that any business can benefit from using computational technology, even hospitals. The challenge is finding the right application to solve the problem.
"Using computational technology is going to be increasingly important no matter what business you're in," he says. "Companies need to have a base-level understanding of how some of these technologies work, so that they can apply them more effectively. It's how do you build and understand which tools can be applied to your problem, so that you can answer the question most effectively."
At Peptilogics, its computational design accelerates the discovery and development of peptide therapeutics by combining peptide research, AI, and machine learning to quickly extract insights from biomedical data. Traditionally, the industry uses more of a screening method for discovery, which takes much longer.
"Through advances in peptide synthesis technology and computational design, we can move beyond broad, random screening to chart the universe of functional peptides and design novel therapeutics," Steckbeck says. "We are trying from the very beginning to design for the characteristic that we want, better, faster, and cheaper."
These are the technologies that make scalable peptide drug design a reality, Steckbeck says.
"Using modern techniques around machine learning is how we believe we're going to be able to bring more solutions to patients."
Lessons learned in first biotech venture
Steckbeck credits his MBA for honing his entrepreneurial skills and giving him the balance needed to start a biotech from scratch. When speaking with others about to enter the field, he encourages them to broaden their education to include business.
"I tell grad students that learning business on a broad level is important if you think you might go the entrepreneurial route. It's a hard transition from the super detailed work of science to pulling back 30,000 feet and telling the business story, and a background in business helps," he says.
The biggest lesson he has learned, Steckbeck says, is how important it is to build a strong team. He suggests finding the best people possible for each role. "The biggest lesson I've learned is how important having a good team is," he says. "My job became much easier with a good team supporting me."
An additional benefit is if that team consists of "people who are actually really fun to work with, because a lot of what we do is really, really hard. It is hard on top of hard. If you don't have a good group of people that you want to work with every day, it's going to be that much harder," he says.