Ipsen allocates $461m for ex-US rights to Day One’s glioma treatment

Day One Biopharmaceuticals has earned accelerated approval from the US FDA for its paediatric glioma medication, Ojemda, intended for children aged six months and older with low-grade glioma. This approval marks a significant milestone for paediatric cancer treatment. Ojemda, which targets type II rapidly accelerated fibrosarcoma (RAF) kinases, addresses gliomas characterized by BRAF rearrangements, a specific genetic mutation.

Low-grade gliomas, a category of slowly progressing tumors, typically manifest in the brain and spinal cord, representing the most prevalent type of central nervous system tumors in children and teenagers. According to the American Brain Tumor Association, these gliomas constitute roughly one-third of brain and spinal cord tumors in this demographic.

Ipsen, a French pharmaceutical company, is poised to enhance its paediatric drug offerings by entering into a licensing agreement with Day One Biopharmaceuticals. This deal, potentially amounting to $461 million, focuses on the commercialization of Ojemda. Ipsen will provide an upfront payment of $111 million, consisting of $71 million in cash and the remainder as an equity investment. Furthermore, Day One could receive up to $350 million in milestone payments and 'double-digit' royalties on sales.

Under the terms of the agreement, Ipsen will secure global commercial rights for Ojemda, excluding the US, while Day One will maintain global development and US commercial rights. This collaboration is expected to boost the global availability of Ojemda, providing a much-needed treatment option for paediatric patients worldwide.

Ojemda recently gained accelerated approval from the US FDA in April for treating children six months and older who have relapsed or refractory low-grade glioma with a BRAF fusion or rearrangement, or BRAF V600 mutation. Market analysts at GlobalData project that Ojemda could generate sales of approximately $797 million by 2030.

Currently, Ojemda is undergoing further evaluation in a Phase III clinical trial named FIREFLY-2. This trial (NCT05566795) aims to compare Ojemda's efficacy against standard chemotherapy in about 400 patients. The primary endpoint is the objective response rate, which will be assessed using response assessment in neuro-oncology (RANO) criteria by an independent review.

In addition to their work on Ojemda, both Ipsen and Day One Biopharmaceuticals have shown interest in developing antibody-drug conjugates (ADCs). Recently, Day One entered a licensing agreement with MabCare Therapeutics for its ADC, potentially worth up to $1.2 billion. Ipsen has also been active in this area, having signed two ADC licensing agreements this year—one with Sutro Biopharma in April and another with Foreseen Biotechnology. Phase I trials for both ADCs are slated to commence in the near future.

This strategic partnership between Ipsen and Day One Biopharmaceuticals represents a significant advance in paediatric cancer treatment. By combining their resources and expertise, these companies aim to bring innovative therapies to market, offering new hope to young patients and their families battling low-grade glioma.