Ipsen acquires ex-US rights to Day One's brain cancer therapy in $460m deal

Ipsen has announced a significant global partnership with Day One, valued over $460 million, to commercialize a monotherapy for treating common pediatric brain tumors. As part of this collaboration, Ipsen will gain all regulatory and commercial rights outside the United States for tovorafenib, used in treating pediatric low-grade gliomas (pLGG) and any future indications. Meanwhile, Day One retains exclusive global development and U.S. commercial rights.

In terms of the financial arrangement, Day One will receive an upfront payment totaling approximately $111 million. This includes $71 million in cash and a $40 million equity investment at a premium. Additionally, there is the potential for up to $350 million in launch and sales milestone payments, along with tiered royalties.

Tovorafenib is an oral, once-weekly type 2 RAF kinase inhibitor that targets mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. It is marketed in the U.S. under the brand name Ojemda and is indicated for patients aged six months and older with relapsed or refractory pLGG. Pediatric low-grade gliomas are the most common type of brain tumor diagnosed in children and can develop in various parts of the brain and spinal cord.

David Loew, the CEO of Ipsen, expressed his enthusiasm about the partnership with Day One, highlighting the goal of making tovorafenib accessible to all eligible patients worldwide. Jeremy Bender, the CEO of Day One, also emphasized the shared commitment of both companies to bring new therapeutic options to patients with limited treatment choices.

Ojemda has previously been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA). Recently, it received FDA approval for use in patients six months and older with relapsed or refractory pLGG that have a BRAF fusion or rearrangement, or a BRAF V600 mutation. These genetic markers are present in over 50% of pLGG cases globally.

Currently, tovorafenib is being evaluated in different clinical studies. The phase 3 FIREFLY-2/LOGGIC study is investigating its use as a front-line treatment for patients aged six months to 25 years with pLGG harboring BRAF fusion or rearrangement or BRAF V600 mutation. Additionally, the FIRELIGHT-1 study is examining the combination of tovorafenib with the MEK inhibitor pimasertib for adolescent and adult patients with recurrent or progressive solid tumors that have MAPK pathway alterations.