Roche has expanded its existing collaboration with Blueprint Medicines, signing a new deal which will see it gain rights to the biotech’s investigational RET inhibitor pralsetinib.
In 2016, Roche signed an initial deal with Blueprint worth a potential $1bn, which included $45m upfront along with a further $965m in possible option fees and milestone payments, plus royalties, dependent on the clinical development and regulatory approval of five small-molecule candidates.
At that point, Blueprint was still in the pre-clinical stages of development – now, pralsetinib is poised for approval, with new drug applications already submitted to the US Food and Drug Administration (FDA) for the treatment of RET fusion-positive non-small cell lung cancer (NSCLC), RET mutation-positive medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer.
Although RET activating fusions and mutations are only observed in a small number of cancer patients – around 1-2% in NSCLC and 10-20% in papillary thyroid cancer – they are thought to be key disease drivers. By inhibiting RET, researchers have found that significant benefits can be achieved in patients with these specific mutations.
Under the terms of the deal, Blueprint and Roche’s Genentech division will co-commercialise pralsetinib in the US, while Roche will have exclusive commercialisation rights for the drug outside the US, excluding Greater China.
Blueprint will gain a substantial $775m in upfront payments, including a cash payment of $675m and an equity investment by Roche of $100m. The biotech will be eligible to receive an additional $927m in contingent payments, including certain development, regulatory and sales-based milestones for pralsetinib. Roche will also have the right to opt in to a next-generation RET compound, co-developed under the collaboration.
“With Roche’s global reach and unparalleled expertise in personalised healthcare, this collaboration will accelerate our ability to bring pralsetinib to patients with significant medical needs around the world and expand development of pralsetinib across multiple treatment settings where there is potential to benefit even broader patient populations,” said Jeff Albers, chief executive officer of Blueprint Medicines.
Blueprint’s main rival, Loxo Oncology, also has an oral RET inhibitor – Retevmo (selpercatinib) – in its arsenal that is FDA-approved in RET fusion-positive NSCLC, advanced or metastatic RET-mutant MTC and advanced or metastatic RET fusion-positive thyroid cancer.
Eli Lilly scooped up Loxo in January 2019 for $8bn to gain access to the drug, as well as an oral BTK inhibitor, LOXO-305, which is designed to address resistance to currently available BTK inhibitors, such as Johnson & Johnson’s Imbruvica (ibrutinib).