Solving for the medical and business case in pediatric cancer — Day One

CEO Jeremy Bender on building a strategy for pediatric cancer therapies that serves both patients and investors 

Pediatric cancer drug development is difficult: it requires high levels of safety, involves parents as well as the patients, and has small populations that can make it difficult to achieve the revenues that reward the investment. Squaring that circle, according to Day One CEO Jeremy Bender, is not impossible, but takes a dedicated strategy that considers both pediatric and adult development paths from the get-go, and pursues them with equal intensity. 

On The BioCentury Show, Bender discussed the rapid rise of  Day One Biopharmaceuticals Inc. (NASDAQ:DAWN), and how it is building a pipeline and evolving a strategy that allows it to keep pediatric drug development central to its mission.

Day One was founded in 2018 and raised a $60 million series A round in 2020, a few months before Bender was recruited in as president and CEO from Gilead Sciences Inc. (NASDAQ:GILD), where he  served as VP of corporate development. The biotech raised a $130 million B round in 2021, and went public in May 2021, raising $184 million. It’s since raised a further $344 million in two follow-on financing rounds.

In April, Day One received accelerated approval of its first product, a pan-RAF inhibitor Ojemda tovorafenib, to treat patients six months and older with BRAF-altered relapsed pediatric low-grade glioma. The drug is in a confirmatory Phase III trial in frontline pLGG in patients aged six months to under 25 years.

The company is becoming a pioneer in pediatric cancer drug development, an area few are dedicated to but Bender said is foundational to Day One’s model and goals. At its core, the company’s strategy involves finding opportunities that could extend to adult populations, rather than focusing on adult cancers and adding pediatric patients later.

“Any program that we bring in, we need to develop with equal intensity in both pediatrics and adults,” he said, adding that was necessary “in order to make a model work in which we’re able to put children living with cancer first.”

Day One’s first programs have begun in children and will extend to adults. An ADC against PTK7 that the company licensed this month from China-based MabCare Therapeutics, a unit of Multitude Therapeutics USA LLC, will reverse that order, but plans for testing in children are built-in. “That program will lead off in adults, partly because we need to establish safety before we can go into children, and thereafter we’ll develop in both populations,” said Bender.

Bender said that a key element to the strategy was building in commercialization plans from the beginning — something he advises small companies to do “at least a couple of years ahead of a launch.”

Though small companies can face challenges in commercializing their own products, he believes it fits Day One’s model, and plans to continue with the rest of the pipeline. “The pediatric oncology market is small enough and concentrated enough, and the patients are identified early on in their diagnosis and treatment, such that a commercialization process is not as challenging as in other arenas, including adult oncology, where all of those parameters are quite different,” he said. 

Having sat on both sides of the pharma deal table (prior to Gilead he was in several biotechs), he said building plans to commercialize alone is important, rather than relying on a deal to come into place when needed.

“It’s really important for small companies to continue investing and building value, whatever stage they are, in anticipation of what’s next, because those deals are never certain. You have to be in a position where you can build value regardless of whether you’re bought or not,” said Bender.