On November 8, 2021 Day One Biopharmaceuticals (Nasdaq: DAWN), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for patients of all ages with genomically defined cancers, reported financial results for the third quarter of 2021 and highlighted recent corporate achievements (Press release, Day One, NOV 8, 2021, View Source [SID1234594718]).
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"We continue to build momentum in our lead program with the granting of Rare Pediatric Disease Designation for DAY101 for the treatment of pediatric low-grade glioma during the third quarter," said Jeremy Bender, Ph.D., chief executive officer of Day One. "Additionally, with the further strengthening of our leadership team and continued progress with enrollment in our pivotal Phase 2 study, FIREFLY-1, we remain on track to present initial clinical data from this trial in the first half of 2022. We continue to grow our company, advance our development programs, and work to achieve our mission of impacting the lives of all people with cancer, whatever their age, starting from Day One."
Program Highlights
FIREFLY-1, a pivotal Phase 2 clinical trial of DAY101 in pediatric low-grade glioma (pLGG), continues active enrollment; initial data are expected in the first half of 2022. The single arm, open-label, global registrational Phase 2 study is anticipated to enroll 60 patients and has activated approximately 25 sites globally.
Day One will present a poster at the 2021 Connective Tissue Oncology Society (CTOS) Virtual Annual Meeting on November 12, 2021. The poster reviews a compassionate use case of a child with recurrent spindle cell sarcoma harboring a novel SNX8-BRAF gene fusion who had exhausted all treatment options, including a MEK inhibitor, and was treated with DAY101 monotherapy. Following treatment, the patient’s symptoms resolved and there was no evidence of measurable disease at the site of previously visualized tumor, indicating a complete response to treatment with DAY101.
Day One is also conducting a Phase 2 monotherapy trial of DAY101 in adult patients with recurrent, progressive, or refractory solid tumors harboring MAPK pathway aberrations.
Corporate Highlights
Day One strengthened its leadership team with appointment of Jaa Roberson as chief people officer. Ms. Roberson will oversee all aspects of Day One’s human resources and talent acquisition efforts as the Company continues to grow.
The Company recently expanded its board of directors with the appointment of Scott Garland. Mr. Garland is a 30-year veteran of the biopharmaceutical industry with deep commercial and executive leadership experience.
Third Quarter 2021 Financial Highlights
Cash Position: Cash and cash equivalents and short-term investments totaled $297.2 million at September 30, 2021. Based on Day One’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into the second half of 2023.
R&D Expenses: Research and development expenses were $9.8 million for the third quarter 2021 compared to $2.5 million for the third quarter 2020. The increase was primarily due to additional employee compensation costs, clinical trial expenses, and CMC activity.
G&A Expenses: General and administrative expenses were $9.4 million for the third quarter 2021 compared to $1.0 million for the third quarter 2020. The increase was primarily due to additional employee compensation costs, legal, and professional expenses associated with operating as a public company.
Net Loss: Net loss totaled $19.2 million and $3.8 million for the third quarters of 2021 and 2020, respectively, with non-cash stock compensation expense of $5.1 million and $0.1 million for the third quarters of 2021 and 2020, respectively.
Upcoming Events
Connective Tissue Oncology Society (CTOS) Virtual 2021 Annual Meeting (November 10-13, 2021) Day One will present the following poster:
Title: "Activity of Pan-Raf Inhibitor Day101 in a Pediatric Patient with a Recurrent Spindle Cell Sarcoma Harboring a Novel SNX8:BRAF Gene Fusion"
Presenter: Katherine Offer, MD
Date: Friday, November 12, 2021
Time: 2:30 PM – 3:15 PM EST
30th Annual Credit Suisse Virtual Healthcare Conference, November 8-11, 2021
33rd Annual Piper Sandler Healthcare Conference, November 29-December 3, 2021
About DAY101
DAY101 is an investigational, oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway. Studies have shown DAY101 has high brain distribution and exposure in comparison to other MAPK pathway inhibitors, thus potentially benefiting patients with primary brain tumors or brain metastases of solid tumors. DAY101 is a type II RAF inhibitor found to selectively inhibit both monomeric and dimeric RAF kinase, which may broaden its potential clinical application to treat an array of RAF-altered tumors.
DAY101 has been studied in over 250 patients, and as a monotherapy demonstrated good tolerability and encouraging anti-tumor activity in pediatric and adult populations with specific MAPK pathway-alterations. In November 2020, Day One announced preliminary results from PNOC014, an ongoing Phase 1 Pacific Pediatric Neuro-Oncology Consortium (PNOC) network study with DAY101 sponsored by the Dana-Farber Cancer Institute. Preliminary results demonstrated that of the eight relapsed pLGG patients in the study with RAF fusions, two patients achieved a complete response by Response Assessment for Neuro-Oncology (RANO), three had a partial response, two achieved prolonged stable disease, and one experienced progressive disease. DAY101 also demonstrated a tolerable safety profile with the most common side effects being skin rash and hair color changes.
DAY101 has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration who require systemic therapy and who have either progressed following prior treatment or who have no satisfactory alternative treatment options. The FDA has also granted Rare Pediatric Disease Designation to DAY101 for the treatment of low-grade gliomas harboring an activating RAF alteration that disproportionately affects children. In addition, DAY101 has received Orphan Drug designation from the FDA for the treatment of malignant glioma and orphan designation from the European Commission for the treatment of glioma.
Day One is conducting a pivotal Phase 2 trial (FIREFLY-1) of DAY101 in pediatric, adolescent and young adult patients with pLGG. Day One also plans to study DAY101 alone or in combination with other agents that target key signaling nodes in the MAPK pathway, such as the Company’s MEK inhibitor pimasertib, in patient populations where various RAS and RAF alterations are believed to play an important role in driving disease.