What happened
Shares of Day One Pharmaceuticals (DAWN 0.46%) were up by more than 13% on Monday morning a day after the clinical-stage biotech released updated trial information regarding its lead therapy. The stock is still down more than 29% so far this year.
So what
Day One, which specializes in targeted therapies to treat genetically defined life-threatening diseases, released early data from its phase 2 study of DAY101 (tovorafenib) as a treatment for pediatric low-grade glioma at the American Society of Clinical Oncology annual meeting. This type of cancer is the most common cause of brain and spinal tumors in children.
The company said the drug showed an overall response rate of 67%, and among heavily pretreated patients showed a clinical benefit rate of 93% based on the Response Assessment for Neuro-Oncology High-Grade Glioma criteria. DAY101 is an investigational oral therapy that is a pan-RAF kinase inhibitor with the ability to cross the blood-brain barrier.
Now what
Day One has already begun a rolling New Drug Application (NDA) to the Food and Drug Administration (FDA) for DAY101 as a monotherapy for relapsed or progressive pediatric low-grade glioma (pLGG). It said it anticipates the rolling NDA will be completed by October.
As a clinical-stage company, Day One has no revenue, and it reported a $42.4 million loss in the first quarter. As of March 31, it said it had $318.2 million in cash and equivalents on the books, enough to fund operations into 2025. All clinical-stage biotechs present a certain level of risk, but in addition to DAY101, Day One's pipeline includes pimasertib, which the company said has the potential to treat various solid tumors, particularly as a combination therapy.
