7 Biopharma CEOs On Who And What To Pay Attention To In 2021 And Beyond

By Rob Wright, Chief Editor, Life Science Leader
Follow Me On Twitter @RfwrightLSL

When you make the decision to involve 15 biopharmaceutical industry CEOs in an article for the annual outlook issue, you risk having way more content than can possibly be wedged into the printed article. Thank goodness Life Science Leader developed the online-exclusive Beyond The Printed Page section of the magazine. For this installment, we have six biopharmaceutical company CEOs providing their perspectives on companies, people, trends, and technologies they are paying attention to for 2021 and beyond. And if you like what you read, be sure to subscribe to Life Science Leader today. It’s quick, easy, and free.  

WHAT INNOVATIVE BIOPHARMA COMPANY OR PERSON HAVE YOU BEEN PAYING CLOSE ATTENTION TO AND WHY?

DIETRICH STEPHAN CEO, NEUBASE THERAPEUTICS

Only a handful of companies are working to fundamentally disrupt the traditional pharmaceuticals development process, something I believe is critical if we hope to enter an era of high-impact scalable medicine. One company I always have admired is Vertex. They started as a small biotech focusing on cystic fibrosis (CF) and have grown tremendously. As one of the earliest adopters of rational drug design, their process has demonstrated the advantages of moving away from classical drug screening strategies. As they have grown, they have been quick to adopt disruptive technologies, which can offer drugs that address the underlying cause of a disease, instead of its manifestations. Their latest work, developing personalized therapies, is a powerful undertaking, showing us where medicine is heading, along with the potential to harness the genetic revolution that started with the human genome project.

KAREN ZADEREJ, CEO, AXOGEN

In general, I like to watch companies that are pioneering in their area of science, and those with a mission-driven, patient-centric focus. It’s interesting to watch how that passion for innovation and patients drive them, and it can be very inspiring.

One of those companies I’ve been watching closely is Biogen, which is leading the development of therapies for neurological diseases. While Biogen is much larger than Axogen, I see similarities in their development of science and technology as a patient-centric organization. They are also balancing their mission and their innovation with their need to deliver long-term value to shareholders, and it’s always valuable to see how other companies are creating that necessary balance.

WHAT ARE THE MOST IMPORTANT TRENDS/ISSUES BIOPHARMA SHOULD PAY ATTENTION TO IN 2021 AND WHY?

DOUG FAMBROUGH, III, PH.D., CEO, DICERNA PHARMACEUTICALS

The two I’d highlight are clinical trials going remote and the resurgence of science.

COVID-19 has forced clinical trials to adapt, some of which may become best practices that should continue. Some trial sites, including academic medical centers, have become more restrictive on the types of research they will conduct, such as allowing research only in areas of greatest unmet need. Trial sponsors have had to allow participants to take part in remote monitoring sessions, replacing hospital visits with nurse visits to participant’ homes. These and other changes not only enabled trials to continue but broke down barriers to those patients limited by geographic or logistical constraints. Continuing these practices post-pandemic has the potential to improve efficiency and productivity without compromising scientific integrity. Speaking of science, in the current environment there has been an erosion of respect for its importance. However, I expect science to come back to the fore in 2021, led by a successful COVID-19 vaccine. The challenge will be in navigating the political environment and sustaining public trust given the inherent difficulty of being guided by science in an era rife with misinformation and strong opinions. Our industry has been thrust into the spotlight like never before, and we have an opportunity — and a duty — to clearly explain the science and show how clinical research can combat the coronavirus and so many other diseases that cause suffering.

RICK MODI, CEO, AFFINIA THERAPEUTICS

This year has been a wake-up call in many respects. One positive outcome is it has brought the importance of diversity and inclusivity (D&I) to the forefront, not just with words, but action. D&I is not a “trend,” but a necessity. In my mind, it encompasses the more traditional ways we think about equality in terms of race, gender, age, and other protected characteristics, but also more broadly with regard to ideas. We are seeing a trend where talent is coming into biopharma from many different paths, creating a melting pot of professionals with high tenures across multiple companies. This requires a non-hierarchical culture in the workplace, along with the all-important soft skills (i.e., openness to different ways of thinking, working, and communicating with each other). It is important that we role model and champion D&I and proactively shape work environments that allow all people to be their authentic selves and feel “at home.”

Another important trend is the increasing portfolio of technologies that can be applied to biopharma, especially digital technologies. These technologies can be applicable to patients, clinicians, payers, or employees. The technologies I believe are still underestimated are those that can assist with every day biopharma needs. This is even more important now and going forward, as social distancing and remote operations become further entrenched as part of the new norm. Imagine the increasing roles of wearable devices for patient monitoring at home, more telemedicine for clinical trial follow ups, and more automated solutions for taking minutes and action items from meetings. A home wireless network can provide 24-hour monitoring of a patient’s health status, from activity level, to range of motion, to quality of gait. Handwriting recognition and voice recognition software can summarize an hour-long meeting in seconds. Emerging technologies require surveillance, assessment, and adoption by employees and the consumers they support. For employees, it’s as much a mindset as it is training and development. The impact of these technologies can be transformational.

The third trend I would point out is the use of data. Data is generated in everything we do, but seldom leveraged to the extent it can be. Sometimes it’s justifiably (i.e., privacy reasons), but many times it’s just not a foresight. Here, too, the applications can be for every function within a biopharma company, to every segment of its external customers. Proper use of data, such as with machine learning (ML), can decrease the diagnosis odyssey for patients with a rare disease and help biopharma companies increase awareness of clinical trials to these families. Employees who can foresee when and how to generate and leverage datasets to make better informed decisions, as well as render proper judgment on when it will lead to unnecessary analysis or when it is simply inappropriate to even collect information, will have an outsized impact in their companies and be sought after.

WHAT NONBIOPHARMA COMPANY OR PERSON DO YOU THINK WILL HAVE THE BIGGEST IMPACT ON THE BIOPHARMA INDUSTRY OVER THE NEXT THREE TO FIVE YEARS AND WHY?

MICHAEL WEISS, CEO, TG THERAPEUTICS

I don’t think one singular person or company will have a material impact on the biopharma industry. And while companies like Amazon and people like Bill Gates may impact small segments, the nonbiopharma people who will have the greatest impact on biopharma are those in congress, and the person who sits in the White House for the next four years. I believe how the government handles healthcare over the next administration may have the biggest impact on how new medicines are developed for the next 20+ years. How the next government decides to address innovation incentives (either enhancing or reducing them) through policy decisions (e.g. government “negotiated” prices) will have long lasting effects on the development of new medicines and the biopharma industry.

WHAT TECHNOLOGY ARE YOU PAYING CLOSEST ATTENTION AND WHY?

DOUG FAMBROUGH, III, PH.D., CEO, DICERNA PHARMACEUTICALS

AI holds the potential to make drug discovery less expensive, more precise, and speedier. Our industry is challenged by the vast amount of biomedical research and data being generated, and the inherent complexity of diseases and life itself. AI may permit a deeper, more effective analysis of this complexity, leading to better approaches to disease, and ultimately, better treatments. While some of the boldest predictions about AI’s potential are likely overblown, at least in terms of what will happen in the next 5-10 years, its continued development is intriguing, and our industry should embrace this exciting new tool.

PETER HEIFETZ, PH.D., CEO, ORPRO THERAPEUTICS

It is hard for me to think of a technology with greater potential for long-term impact on biopharma than gene therapy, including CRISPR. This will be the means to ultimately treat genetic disorders and enable true cures for so many devastating diseases. However, today that vision is still far off, and there are three key areas that must be addressed before gene therapy is a reality. First and foremost is safety. Gene therapy in the past has been fraught with unintended consequences. Targeting has to be highly precise, and the target itself needs to be unambiguous in order to avoid side effects that might be life threatening. CRISPR has potential to change the game, but we still need to have better tools to ensure that precision is high, and off-target effects are negligible. Second is efficacy. We have to hit the correct target, at the correct time, and at the correct gene dosage. This is easier for straight deficiency diseases, but more challenging when defects are subtle or involve dysregulation. Our knowledge of the disease mechanism and control may not be sufficient to affect a therapy even if the technology is adequate. Last is establishing a robust ethical framework. The reports from China of germ-line editing of human embryos leading to live birth was a wakeup call. Once the technology is robust and straightforward, then we need clear guidelines for how it will be applied. “Just because you can doesn’t mean you should,” must be a central question to ask at all stages, with strong justifications needed for moving forward with any gene therapy. In addition to IACUC (Institutional Animal Care and Use Committee) and IRB (institutional review board), there will need to be a third formal level of institutional oversight for gene therapy that is built into the regulatory and approval process.

ADRIAN RAWCLIFFE, CEO, ADAPTIMMUNE

Cell therapy in the clinic and on the market has been focused on autologous approaches that require harvesting a patient’s own cells, modifying them, and providing them back to the patient to fight disease. This is a time consuming and complicated process for patients and providers. There are potentially alternative ways to provide cell therapy, and one of these is “off-the-shelf” products using allogeneic technology that eliminate the need to harvest cells from patients. These therapies can be ready when a patient needs them. This would be game changing for the cell therapy field, taking it from a niche, high priced product, to one available to the majority of the world’s population.