Ipsen obtains ex-US rights to Day One's pediatric cancer drug with $111M upfront investment

Cancer specialist Ipsen has partnered with Day One Biopharmaceuticals to expand the global reach of tovorafenib, also known as Ojemda in the U.S., just three months after its accelerated FDA approval. The licensing agreement, signed on Thursday, grants Ipsen the rights to develop and commercialize tovorafenib outside the U.S. for pediatric low-grade glioma (pLGG) and any future indications identified by Day One.

Ipsen is making a significant upfront investment of $111 million, which includes a $40 million equity stake in Day One. The agreement also opens the door for Day One to receive up to $350 million in milestone payments and tiered double-digit royalties based on future performance.

Ipsen emphasized that its extensive experience and expertise in oncology would enable it to expedite regulatory activities and the delivery of tovorafenib outside the U.S. The company highlighted the severe impact of pLGG on children, noting symptoms such as vision and speech problems, neurological issues, fatigue, and nausea, all of which can significantly affect a child's development and quality of life.

Tovorafenib, marketed as Ojemda in the U.S., received accelerated FDA approval as a treatment for pLGG in patients six months and older with tumors exhibiting a BRAF fusion or rearrangement, or a BRAF V600 mutation. The approval was granted just three months ago, and pLGG is described as the most common childhood brain tumor. The disease sees tovorafenib competing against Novartis' combination of Tafinlar and Mekinist, which also received FDA approval in 2023 for treating pLGG cases with BRAF V600 mutations, albeit in a more limited scope.

Ojemda represents Day One's first commercial product, and analysts at JPMorgan estimate its peak sales could reach $750 million. This potential has likely attracted Ipsen to the partnership.

In other updates from Ipsen, the company released its first-half financial results on the same day as the partnership announcement. Ipsen's sales for the first half of the year increased by 8% year-over-year, totaling 1.659 billion euros ($1.8 billion). This growth was driven by sales of cancer drug Cabometyx, in collaboration with Exelixis, along with Dysport for spasticity and cervical dystonia, Bylvay for Alagille syndrome, and Onvyde for certain types of cancer.

However, Ipsen's profit margin saw a slight decline in the first half of the year, which the company attributed to increased spending on research and development. Despite this, Ipsen adjusted its full-year sales growth expectations to 7%, up from its previous forecast of 6%.

This partnership between Ipsen and Day One underscores the ongoing efforts to enhance treatment options for pediatric low-grade glioma, aiming to improve the quality of life for affected children globally. As Ipsen takes on the challenge of expanding tovorafenib's reach, both companies are poised to make significant strides in the oncology field.