Biotech Insiders Share Five Lessons From Pioneering New Medicines
Last week, I had the good fortune of attending the BIO International Convention in San Diego, Calif., where more than 18,500 biotech professionals from around the globe gathered to exchange ideas, build connections, and form new partnerships with the shared mission of advancing drugs for patients.
It was my first time attending the conference, and I was delighted to meet with my colleagues based near and far, as well as current and potential partners. One of the highlights of my four days there was moderating a panel on first-in-class medicines, featuring several of the savviest CEOs in the business.
A first-in-class medicine means that it uses a new and unique mechanism of action, as opposed to incrementally better versions of previously developed medicines. Why does this matter for patients? Because in many disease indications, patients lack adequate options. They need a novel approach, something no one has developed before. Enter the cutting-edge biotech and pharma companies who embark on the grueling, risky, and time-consuming path to developing a first-in-class drug.
These drugs are the embodiment of high risk, high reward propositions for both the companies and the investors who finance them. Many fail, but those that succeed open up a new therapeutic modality or area of medicine. A good example is Keytruda, which was approved in 2014 as the first in a new class of checkpoint inhibitors. In trials, Keytruda shrunk tumors in nearly a quarter of patients who were suffering from life-threatening forms of skin cancer. Another example is Casgevy, the first CRISPR medicine, approved last December, to treat sickle cell disease and beta thalassemia.
Our standout panelists shared five lessons learned from their combined decades of experience in developing first-in-class medicines:
1) The Advantage of First to Market
John Wu, managing director and partner of the Boston Consulting Group, shared the results of research his team has conducted on the commercial value of first-in-class medicines versus best-in-class medicines that may be second or third to launch, yet have an advantage of efficacy or safety. The results have become even more pronounced in the last decade, Wu said. It’s clear that first-in-class medicines capture more value — even if better drugs follow. It may be that providers and patients become familiar with the first drug, for example, and therefore maintain an advantage.
2) Time Is Your Enemy
Seth Ettenberg, CEO of BlueRock Therapeutics, which is a wholly owned subsidiary of Bayer and the cornerstone of our cell and gene therapy program, offered this tidbit about the necessity of prioritization for platform biotech companies. Such companies usually are spun out of academic labs whose innovative discoveries hold great potential for treatments across a range of disease indications. These companies seek to validate and develop a novel approach to either screening or drug development, rather than a specific asset only. That said, the multiplicity of options, combined with finite capital, means that effective prioritization is essential, Ettenberg said.
Hanadie Yousef, CEO and co-founder of Juvena Therapeutics, corroborated Ettenberg’s advice. Her company’s unique approach to translating secreted proteins into engineered biologics has identified over 50 hits in six organ systems, but it is a practical impossibility to pursue all of those potential drugs at once. Her team decided to prioritize muscle wasting associated with aging because of the founders’ expertise and doctoral work.
3) Know the Conditions That Yield a Breakthrough Drug
Wu at BCG offered five steps for launching a successful first-in-class medicine: determine the fundamental biology, find a way of modulating the biology in a potentially beneficial way, have a good translational animal model of the disease, figure out clear clinical endpoints to see if the drug is having the intended effect, and make sure there is a clear market with enough patients for the drug to be commercially viable.
Sometimes, the choice to first de-risk a new technology platform by developing it for a disease that is extremely rare may end up dooming a company’s valuation in the long run. It can be more strategic to focus on a larger patient population right off the bat. Proof of concept of a drug is not the same as the proof of a drug for patients — and that’s what matters most.
4) AI’s Transformative Power Goes Beyond Drug Discovery
It seems like a foregone conclusion these days that AI will accelerate drug discovery, although we are still in the early days. Enthusiasm is heady, but some skeptics are still waiting for lived experience to bear proof of AI’s transformative capabilities. As tech and biology converge, I am optimistic that we will see shortened discovery timelines and screening analyses of new compounds. But there are other, less discussed opportunities for AI to step in and speed up the process, such as streamlining clinical trials by completing mountains of necessary paperwork. Even a 30 percent increase in speed would be a gamechanger in getting new drugs to patients faster.
5) Talk to Regulators Early and Often
Ettenberg of BlueRock described how his team started discussions with the Food and Drug Administration from an early academic phase of the company, since their approach of using pluripotent stem cells to replace the lost dopamine-producing neurons in Parkinson’s patients represents a completely new way of treating the disease — and even potentially offering patients a functional cure. (The team recently shared positive 18-month data on the first twelve patients in the phase 1 trial.) It’s important to “not be afraid of conversations with regulators,” Ettenberg said, and stressed the importance of an ongoing dialogue to educate as well as receive and address concerns throughout the process. BlueRock has now just received the coveted RMAT designation from the FDA, which means that regulators have designated the therapy as eligible for accelerated approval.
“We all have the same mission,” said Aleksandra Rizzo, the CEO of Vividion, another wholly owned and independently operated subsidiary of Bayer. “We all want to bring medicines to patients, so the more we discuss with regulators, the faster we can progress.”
My time at BIO was a chance to reflect on the fascinating era we are now living through, which I predict we will look back on as the dawn of a golden age in medicine. In a recent interview with John Crowley, the new president and CEO of BIO, he offered a similar takeaway.
“In many respects, this is the third bio-revolution,” he said. “The first was cell-based engineering, the second was decoding the human genome and now we’re coming into this area of precision medicine. We just need to make sure we do everything possible to accelerate this research” for patients.
They are counting on us, and I will keep doing everything in my power — through new deals, collaborations, and partnerships — to deliver them the best medicines our industry has to offer.
Special thanks to Kira Peikoff and David Cox for their additional research and reporting for this article.
The article was initially published on Forbes.com on June 10th, 2024