What started as an undergrad project at the University of Washington has now seeded a fully-fledged biotech startup, complete with a high-profile pharma partnership that could see the nascent company acquired in a few short years.
On Thursday, San Diego-based PvP Biologics and Takeda Pharmaceuticals announced a global agreement to further develop KumaMax, an enzyme owned and engineered by PVP Biologics to help treat celiac disease.
In a phone interview, CEO and President Adam Simpson said the collaboration arms the startup with the financing and freedom needed to power its science forward.
“Obviously we are really excited to announce this development agreement with Takeda, which provides financing from preclinical — where we are today — through a Phase 1 proof of principle study, which is around $35 million.”
At that point, Takeda can choose to exercise its option agreement, acquiring the company for an undisclosed upfront sum, milestone payments and royalties, Simpson said.
Unlike most autoimmune conditions, celiac disease is triggered by an external compound — dietary gluten. The protein, found in wheat, barley and rye, sets off an immune response when it reaches the small intestine, causing inflammation and damage to the intestinal lining.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Around one in 100 individuals worldwide has the disorder. However, it remains hard to diagnose and the prevalence varies greatly depending on genetic ancestry.
For all patients, the current standard of care is to observe a gluten free diet. It sounds simple, but it is rarely 100 percent effective. Even small quantities manufactured into foods or introduced through cross-contamination continue to drive inflammation.
With a platform licensed from the University of Washington in December, PvP Biologics is fine-tuning an enzyme that breaks down the gluten protein before it reaches the small intestine.
It’s not an license for patients to eat whole wheat bagels and donuts, cautions Malcom Hill, PVP Biologics’ chief development officer. But when taken with food, it could alleviate some of the discomfort and intestinal damage that occurs with low-level exposure.
“You administer the enzyme prior to each meal so that any gluten that happens to be in the meal is broken down prior to the immunoreactive species being formed — in the stomach,” Hill explained. “So the immunoreactive gluten fragments don’t make it into the intestine.”
The science all started in the summer of 2011, with three undergrad students at the University of Washington; Elizabeth Stanley, Sydney Gordon and Sarah Wolf. They formed a team to compete in the International Genetically Engineered Machine (iGEM) competition, which encourages students to solve real-world challenges through the creation of genetically engineered molecules or biological systems.
Ingrid Swanson Pultz, chief scientific officer and cofounder of PvP Biologics was advising the students at the time. In a phone interview, Swanson Pultz said the students focused their efforts on a therapeutic enzyme for celiacs disease, inspired by friends with the condition.
The gluten protein is notoriously difficult to break down. Compounding the challenge, the therapeutic enzyme would need to survive and thrive in the harsh stomach environment. If it didn’t do its job before the small intestine, the patient’s disease would be triggered.
No enzyme appeared inherently suitable for the job, said Swanson Pultz.
“So we thought, how can we use the tools of protein design to engineer therapies for this disease?”
The team started with an enzyme that is highly active in the acidic stomach environment. They then used a sophisticated protein design platform to optimize the enzyme for gluten digestion. The resulting prototype took out first place in the iGEM competition.
It wasn’t ready for the clinic, but it served as a solid foundation for future development work.
Swanson Pultz picked it up as her Ph.D., project, partnering with David Barker, a renowned protein engineering expert and a fellow cofounder of PVP. Together they advanced the enzyme, incubating it within the University. Approximately one year ago, they decided the time was right to fully spin-out the company.
Board Chairman Tadataka (Tachi) Yamada helped guide the company through its next steps, making some key introductions.
“Tachi is really the one who had the idea to go directly to pharma and avoid the traditional financing,” Simpson said. “In terms of what it means for us, and celiac patients, we think there is an improved probability of technical success because we have access to a world leader in GI.”
The company will now begin its preclinical research with a business headquarters in San Diego, California and a scientific lab in Seattle, Washington.
Photo: minoandriana, Getty Images
