Day One Biopharmaceuticals

Day One Biopharmaceuticals reported OJEMDA net product revenue of $33.6 million in Q2 2025, representing a 310% year-over-year increase driven by expanding adoption among pediatric oncologists.

Day One Biopharmaceuticals has appointed Michael Vasconcelles, M.D., as Head of Research and Development, bringing over 25 years of oncology research and development expertise to the company.

• XOMA Royalty doubled its portfolio to over 120 royalty assets through five strategic transactions in 2024, strengthening its position as a biotech royalty aggregator. • The company completed two whole company acquisitions and celebrated FDA approvals for Day One's OJEMDA™ (tovorafenib) and Zevra's MIPLYFFA™ (arimoclomol), generating significant milestone payments. • Despite reporting a net loss of $13.8 million for 2024, XOMA Royalty received $46.3 million in cash receipts and maintains over $100 million in cash, positioning it for sustainable cashflow from royalties.

• The FDA has approved FoundationOne CDx as a companion diagnostic for tovorafenib (Ojemda) in pediatric low-grade glioma (pLGG) patients with specific BRAF alterations. • FoundationOne CDx detects BRAF fusions, rearrangements, and V600 mutations, enabling precise identification of pLGG patients who may benefit from tovorafenib treatment. • Tovorafenib received accelerated approval in April 2024 for relapsed/refractory BRAF-altered pLGG, supported by a 67% overall response rate in the FIREFLY-1 trial. • This approval marks Foundation Medicine’s first companion diagnostic indication exclusively supporting pediatric patients, enhancing precision medicine in pediatric oncology.

The FDA approved Madrigal's Rezdiffra, the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), addressing a significant unmet need in liver disease.

Over 80% of U.S. clinical trials fail to meet patient enrollment timelines, highlighting the significant challenges in rare disease research and patient recruitment.