Despite significant advancements in hemophilia treatments over the past several decades, a fundamental challenge remains for many patients: balancing management of the disorder while maintaining regular day-to-day activities.

Historically, treatments could be cumbersome and time-consuming to administer. They typically required intravenous administration up to several times a week to prevent bleeding episodes.1 Even treatments that could be administered at home required infusions multiple times a week, as well as strict requirements around the medications’ scheduling, preparation, and storage. As of a few decades ago and before the introduction of recombinant protein technologies, treatments could carry a risk of contracting a blood-borne infection due to the fact that medicines were derived from blood.2

While treatment options and the science behind hemophilia treatments have continued to improve, one team of Pfizer researchers wondered if a different and easier to administer approach might be possible. With patients’ experiences, preferences, and needs at the forefront of their minds, they sought to discover a molecule with the potential to be easier to manufacture and deliver, all while maintaining or exceeding the safety and efficacy of the existing standard of care.3

A Collaborator Brings First-Hand Experience

One of the program’s first and most influential collaborators was Will Somers, Head of Biomedicine Design at Pfizer. Somers brings a special expertise to his work: he is a person living with hemophilia.

Although hemophilia didn’t slow Somers down when he was a kid — he was always riding a bike, hiking a trail, or sailing on a boat — there were scary moments. Growing up, Somers often wondered: Would a bruise heal on its own, or would it get worse? Would the next one land him in the hospital for what could be weeks of treatment?

“Staying active was an important thing to do, but that does also occasionally result in injuries,” says Somers. “Hemophilia treatment for me when I was younger was pretty tough.”

Somers dreamt of discovering a treatment that would allow people to manage their disorder with fewer interruptions. His dream became a plan. Somers’ experience with hemophilia led to a career in medicine discovery and development.

“When I went to university and studied biophysics — and in particular learned about protein structure and function — I always thought it would be great if I could use those skills to come up with a better hemophilia treatment,” Somers says.

As the Pfizer team worked to develop a new molecule, Somers often advocated for raising the bar, reminding his teammates what improvements could mean for patients’ everyday lives. He encouraged the team to develop a treatment that would be easy for patients to administer themselves, and a medication that would be easy to manufacture and to deliver.

And the team didn’t stop until they developed a prospective treatment that met these criteria.

“All of us had been trained to think one way about how drugs should work,” says Laird Bloom, Senior Director of Molecular Discovery at Pfizer and a key contributor to the project. “But with the input of all our collaborators – the modelers, discovery scientists, and many more – we learned to think a different way.”

Watch the video below to learn more “Behind the Science” of this team’s work: