On November 3, 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 an upcoming poster presentation at the 2021 Connective Tissue Oncology Society (CTOS) Virtual Annual Meeting, being held from November 10-13, 2021 (Press release, Day One, NOV 3, 2021, View Source [SID1234594260]).
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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 had resolved and there was no evidence of measurable disease at the site of previously visualized tumor, indicating a complete response to treatment with DAY101.
"This compassionate use case provides an important experiential data point about the therapeutic activity of DAY101 in pediatric patients with soft tissue sarcomas harboring BRAF gene fusions," said Samuel Blackman, M.D., Ph.D., co-founder and chief medical officer of Day One. "We remain committed to making a difference in the lives of all people with cancer and plan to study DAY101 further in pediatric patients with extracranial RAF-altered tumors."
Details of the poster presentation are as follows:
Title: Activity of Pan-RAF Inhibitor DAY101 in a Pediatric Patient with a Recurrent Spindle Cell Sarcoma Harboring a Novel SNX8-BRAF Gene Fusion
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 an investigational type II RAF inhibitor designed 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.