Horizon Therapeutics Now Amgen

Jeffrey W. Sherman, MD, FACP, is a seasoned physician and pharmaceutical executive with over 35 years of industry experience. He has held clinical and medical roles at Horizon Therapeutics, IDM Pharma, Takeda, NeoPharm, Searle/Pharmacia, and Squibb/ Bristol-Myers Squibb. A former DIA board chair, he serves on multiple boards and advisory committees, focusing on rare diseases, clinical research innovation, and patient advocacy and access to breakthrough therapies.

My Path Into The Pharmaceutical Industry

I started out as a physician, completing my internal medicine internship, residency, and chief medical residency at Northwestern University in Chicago. From there, I pursued an Infectious Diseases fellowship at the University of California, San Francisco, and conducted research in allergy immunology at the Howard Hughes Medical Institute. My focus was purely on medicine and research at the time—I wasn’t thinking about the corporate world.

My transition into the pharmaceutical industry began in translational medicine and clinical pharmacology at Squibb, which later merged with Bristol Myers. I went on to work at Searle/Pharmacia (now part of Pfizer) and then at several small biotech companies, along with Takeda, before joining Horizon Therapeutics in 2009. At that point, Horizon was a virtual organization, operating out of a rental house in Palo Alto, California, with no marketed products and only a handful of employees. Under the leadership of Chairman, President, and CEO Tim Walbert, the company was built from the ground up, eventually going public, acquiring and developing marketed products, and establishing our U.S. headquarters in Deerfield, Illinois.

In late 2023, Horizon was acquired by Amgen, and my role shifted to Chief Medical Officer Emeritus. Now, I advise Amgen while also serving on the boards of several biotech companies, both public and private. Beyond my corporate work, I’ve been involved in non-profit organizations dedicatedto healthcare innovation and increasing clinical research participation. I also represented Horizon on the BIO board, a major biotechnology trade organization, which broadened my perspective on industry-wide challenges.

The High-Stakes Role Of A CMO In Small Biotech

Throughout my career, I have seen firsthand how the role of a CMO has evolved. In small biotech companies, one of the biggest hurdles is securing funding and advancing technology. It’s the job of the CMO to take highly complex scientific and clinical data and make it understandable to investors who might have a limited medical or scientific background. Investors need to see the potential of a company’s technology, and if they don’t, the company may struggle to survive.

Small biotechs operate with tight budgets, and the reality is that only a small percentage of drugs make it through clinical trials. Unlike large pharmaceutical companies that can absorb financial setbacks, small biotechs often rely on a single lead asset. If that fails, the company may not have another shot. You have to balance scientific optimism with financial realism—ensuring that we de-risk programs as much as possible while using capital efficiently.

“No one can predict every challenge, but surrounding yourself with the right people can make all the difference.”

Hiring the right people is another challenge. Many earlystage biotechs have groundbreaking technology, but they need top talent to bring those innovations to life. Attracting experts to a company that’s operating from one funding round to the next isn’t easy, especially when larger firms offer morestability and resources. Partnerships also play a huge role— smaller companies don’t have the same purchasing power as industry giants, so they must be strategic in how they build relationships.

Regulatory hurdles add another layer of complexity. From early-stage clinical trials to drug approval, you have to navigate evolving government regulations, compliance requirements, and reimbursement challenges. Even after approval, the work doesn’t stop—payers and insurers have to be convinced of a drug’s value, and long-term safety monitoring is essential. External factors, like geopolitical instability or global health crises, can further complicate things. COVID-19, for example, disrupted clinical trials and supply chains across the industry, forcing companies to quickly adapt.

Navigating Uncertainty: Strategy, Risk, And Purpose In Biotech

To improve our chances of success, you have to focus on derisking our programs while making the most of our resources. This means making smart choices about study designs, selecting the right patient populations, and anticipating potential regulatory roadblocks. Unlike large pharmaceutical companies with in-house manufacturing, smaller biotechs often rely on contract manufacturers. Managing those relationships carefully is critical to maintaining quality and controlling costs.

Proactive risk management has also been key. Unexpected events—a global pandemic, an economic downturn, or a supply chain disruption—can throw even the best-laid plans off course. While you can’t predict every challenge, you have to learn the importance of having contingency plans and staying flexible. Investors also want as much certainty as possible in an unpredictable industry, so clear, transparent communication is vital.

Rare diseases have been a major focus of my career. With over 7,000 rare diseases identified but treatments availablefor only a small fraction, the need is enormous. However, working in this space comes with challenges. Because patient populations are small, clinical trials are difficult to design and expensive to run. Reimbursement can also be tricky, as many insurers hesitate to cover treatments for ultra-rare conditions. Still, the impact we can have on patients’ lives makes it all worth it. Many people living with rare diseases would trade for what most consider a bad day just to regain simple functions—brushing their teeth, making breakfast, or walking without pain. Knowing that our work gives them hope is what keeps me going.

What’s Next For The Industry

Despite the challenges, I remain optimistic about the future of biopharma. Diseases continue to evolve, and we need new therapies to keep up. Infectious diseases, for example, present ongoing threats due to microbial resistance and genetic mutations, much like what we see in oncology. Meanwhile, new therapeutic areas, such as obesity treatments, are attracting significant investment, showing how quickly the industry can adapt to emerging health concerns.

Rare diseases will remain a focus, but their market potential is often underestimated. The reality is that for patients and their families, access to treatment is lifechanging. External factors, such as government funding policies, clinical research site capabilities, and regulatory changes, will also shape the industry’s direction. Making sure that trial sites have the resources and infrastructure they need is critical to accelerating drug development.

Advice For Future Leaders

For those stepping into leadership roles in biopharma, my biggest piece of advice is to stay open-minded and build strong networks. No one can predict every challenge, but surrounding yourself with the right people can make all the difference. I have learned so much from my peers, mentors, and team members over the years, and I continue to learn every day.

Above all, never lose sight of why we do this work. At the end of the day, we are all patients at some point in our lives. Whether we’re developing treatments for genetic diseases, lifestyle-driven conditions, or complex multi-factorial disorders, the goal should always be to improve health outcomes and quality of life.

The biopharma industry is not for the faint of heart, but it is one of the most rewarding fields to be in. The work we do have the potential to change—and even save—lives. That’s what makes all the challenges worth it.