Building Promise from Precision

Amy Simon, MD, Chief Medical Officer, Beam Therapeutics

For Amy Simon, MD, the key to being a good leader is authenticity. She believes a leader who is able to admit what they know and don’t know is going to have more success than someone who doesn’t. In her role as chief medical officer at Beam Therapeutics, Simon sees herself as someone that enables every member of her team to be contributors who feel empowered to share ideas—even in areas where they may not necessarily be experts.

At Beam, Simon works across multiple departments to ensure that new, innovative treatments are being discovered and developed. Her role is special to her because it’s something that she was able to help build.

“When I came to Beam, there were only two people in clinical development, so I helped to build out the clinical development organization, which then helped us to get our incredible science safely and efficiently to patients,” she tells Pharm Exec. “Some of the projects I’m working on, which are really our clinical priorities right now, are our sickle cell disease program and our Beam 302 program.”

The latter, she explains, is attempting to provide a one-time treatment for patients with alpha-1 antitrypsin deficiency and target the root cause of the disease: DNA mutation. The hope is to apply the approach to lung and liver manifestations. Beam has a similar program for glycogen storage disease in the clinic as well.

Simon’s goal is to solve problems that are preventing these new types of therapies from reaching more patients.

“We’re trying to think about how we might be able to get cell and gene therapy to more patients, because we’re seeing limitations with some people having genotoxic conditioning,” she says, referring to the impetus behind Beam's lead program in sickle cell disease. “If we could get rid of that genotoxic conditioning, we could treat the more severe patients who couldn’t tolerate that. We could also treat more patients who don’t want to have genotoxic conditioning because it could affect their fertility or even lead to long-term malignancies in the future.”

Rise of precision therapy

Simon notes that there’s been an increased interest in precision therapy in recent years, which she credits to a number of elements. First, the scientific tools needed to build these therapies are finally available. Second, she’s noticed a trend in the industry toward trying to prevent diseases, as opposed to just treating them once they’ve already manifested symptoms.

“This sort of stuff seemed like science fiction in the past,” she says. “Now we’re showing that we actually can do this. Being a doctor myself who’s taken care of patients for a long time, we have often focused on treating the disease once it’s already happened and once things are broken. There’s now this idea about moving away from waiting until things are not working anymore and trying to go closer to the root cause of the disease. The precision allows you to potentially be more on mechanism. And No. 2, by being so proximal, you can potentially prevent a lot of the different manifestations that occur in the disease.”

By treating people earlier, it’s becoming possible to prevent things from breaking. As Simon describes it, it’s the difference between tuning up a car versus waiting for the tires to go bald.

“As a clinician, I’ve always thought that we never had enough time to spend talking to patients about prevention or focusing on disease prevention. It feels like the system was not set up for that,” she tells Pharm Exec.

A new era of medicine

Simon believes that biotech is entering a phase where the industry can really start to focus on treating diseases that have a high unmet need. New technologies and discoveries are allowing researchers to take fresh looks at diseases that may have been neglected for a long time, such as sickle cell disease. She also believes that many of these new drugs will avoid some of the issues that previous treatments placed upon patients.

“If there’s a known mechanism of action of a drug so you specifically know how it works, and you know that everybody has that mechanism naturally occurring in their body, that medicine just works better,” she says. “It doesn’t have all these unintended effects or effects beyond what you’re trying to do. It cuts down on the variability in response. We all have similar genetic processes in our bodies. Precision therapy isn’t like a small molecule where some people may respond and some may not respond. That helps in the response rate and in mitigating unwanted side effects. The efficacy has been so outstanding, and we are just starting to see what can be done with precision medicine.”