To close the pediatric innovation gap, this biotech has to think differently
Welcome to today’s Biotech Spotlight, a series featuring companies creating breakthrough technologies, strategies and products. Today, we’re looking at Day One Biopharmaceuticals, a clinical-stage biotech developing targeted cancer therapies for patients of all ages.
In focus with: Jeremy Bender, CEO and president, Day One Biopharmaceuticals
The company’s focus: Day One Biopharmaceuticals was founded “to address the innovation gap between children and adults living with cancer,” Bender said. But the company doesn’t focus exclusively on developing medicine for children. Instead, what makes Day One distinctive is that it “pushes for early clinical trials in children and adolescent patients,” rather than waiting for a drug to first be approved for adults before pursuing a pediatric indication.
“We do that in parallel with the work that we do seeking trials in adults. That effort requires coordination with regulators, coordination with trial sites, sometimes specific investments in formulation and early toxicology studies, for example,” he said. “But fundamentally, our mission is to develop all-new medicines in patients of all ages. And so we do that early. It sounds simple, but it is quite distinct from what other companies pursue.”
Day One submitted a rolling new drug application to the FDA for its lead candidate, tovorafenib, as a monotherapy in relapsed or progressive pediatric low-grade glioma in September. The company anticipates the FDA’s filing decision by mid-November.
“The mantra that we have [is]: right program, right patient population, right biology.”
Jeremy Bender
CEO, Day One Biopharmaceuticals
The pipeline also includes pimasertib, a small molecule inhibitor of mitogen‐activated protein kinases 1 and 2. And Day One has entered into an exclusive, worldwide license agreement and research collaboration with Sprint Bioscience for its VRK1 program, which aims to develop treatments for pediatric and adult cancers with high unmet need.
Why it matters: There’s a well-known disparity in the availability of innovative drugs for adult and pediatric patients. Often, that’s because drugs are first approved for adults and much later for children. Day One wants to change that.
“Over the past several decades, you’ve seen a lot of breakthrough medicines for adults with cancer, but very few for children,” said “On average, it takes six to seven years longer for something to be approved in children than adults, and that’s when it’s even approved at all.”
Although research has shown the success of combined adult and pediatric trials submitted to the FDA, there are several barriers to this approach.
“The reason it’s more difficult has to do with, to some extent, safety considerations and the regulatory process for getting clinical trials approved,” Bender said.
For instance, “regulatory authorities do require some exposure to adults before you expose children to a new agent in order to address any potential safety concerns associated with the new agent.”
He also points to a lack of infrastructure and experience among academic sites for pediatric oncology trials, as well as the fact that there are far fewer children with cancer than adults.
Here, Bender explains more about Day One and its approach to drug development.
This interview has been edited for brevity and style.
PHARMAVOICE: Why would you describe Day One’s process and business strategy as innovative?
JEREMY BENDER: In Day One’s case, our innovative approach really comes from two components. It comes from pushing where we can to do that pediatric work early. [That] is unique and differentiated.
And it also is a process we employ to look at indications and biology that may not be addressed or thought through by competitors. That leads to the mantra that we have: right program, right patient population, right biology. And if you put those things together and have insights about each element, you can develop new medicines more quickly and with a higher probability of success. Employing that, which is what we all in the industry strive to do, is why we’ve been successful so far with tovorafenib and what we hope to apply to other programs as well.
You say you want to be part of what changes pediatric cancer patients’ lives. Do you see the company doing that and how?
That’s absolutely our aspiration, and it animates everybody in the company. Our employees have joined Day One in order to have that impact on patients, and on pediatric patients specifically. I hope we’re able to achieve that aspiration, and I think we’re on the right path. The data from our lead program, tovorafenib in relapsed or progressive pediatric low-grade glioma indicate that we’re on the right path. The clinical trial for that patient population is called Firefly-1, and we’ve released some of the results of that trial over the past 18 months in particular and are really encouraged by what we’re seeing.
I would highlight that of the approximately 76 patients in that trial, we’re seeing a stabilization and decrease in tumor size for 80% to 90% of the patients on the trial, depending on exactly how you measure the tumors in those children. And that’s a meaningful early signal for making a difference for these patients.
What does that mean in terms of how these patients’ lives progress? By stabilizing and in many cases, decreasing the size of their tumors in their brains, you’re arresting any functional deficits that may have been resulting from tumor growth. In some cases, you’re improving that function and just as importantly, preventing the use of subsequent therapies that may be more challenging or more toxic or have longer-term safety effects than that we hypothesize tovorafenib to have.
In the end, that can translate, and we hope that it will, into a truly better quality of life for these children in the form of visual function, cognitive function [or] motor function. Of course, the families and caregivers of these children as well can be impacted by that kind of improvement.
What should other biotech and pharma executives know about how you’re approaching this?
For our model to work and frankly, for the biotech and pharma industries (or biotech, specifically) to thrive and make progress depends on a couple of components that I emphasize. The first is ongoing and significant collaboration with regulatory authorities. The extent to which that dialogue is productive and clear and supportive of innovative approaches in pediatric patients in particular is critical to our model.
The second is the degree to which our industry relies on capital formation and the ability to raise money from investors, whether that’s through venture capital or through the public markets. Both components — the availability of capital and the regulatory process, of course, those two are connected — are crucial to having the opportunity to continue trying to build a sustainable business that aims to do what we aim to do.
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