DAWN - Day One stock surges 20% as brain tumor drug shows promise in mid-stage study

Day One Biopharmaceuticals ( NASDAQ: DAWN ) reported data from an ongoing phase 2 trial of tovorafenib to treat recurrent or progressive pediatric low-grade glioma (pLGG).

The main goal of the study, dubbed FIREFLY-1, is overall response rate (ORR) and the company is evaluating tovorafenib as once-weekly monotherapy in patients aged 6 months to 25 years with recurrent or progressive pLGG — a type of tumor which occurs in the brain in children.

The company said that in 69 heavily-pretreated patients, ORR of 64% was seen and 91% clinical benefit rate (CBR - complete response + partial response/unconfirmed partial response + stable disease).

Three patients with confirmed complete responses, 59% (n=41) partial responses (31 confirmed and 10 unconfirmed), and 28% (n=19) patients with stable disease.

Median duration of 8.4 months on therapy as of Sept. 28, 2022, with 77% (n=59) of patients remaining on treatment, according to the company.

DAWN noted that tovorafenib was generally well-tolerated. The most common side effects related to the drug were change in hair color (75%), increased creatine phosphokinase (64%), anemia (46%), fatigue (42%) and maculopapular rash (42%).

Increased creatine phosphokinase levels may suggest injury or stress to muscle tissue, heart or the brain.

"Based on the efficacy and safety profile of tovorafenib observed to date from the FIREFLY-1 trial population, we plan to submit a New Drug Application in the first half of this year that will include additional follow up from the full study population," said Day One CEO Jeremy Bender.

The company noted that additional data will be presented at a medical meeting in Q2 2023.

DAWN +23.20% to $26.29 premarket

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