![]() This support Blueprint by using “data and diagnostics to identify patients based on the specific characteristics of their tumours” and mean pralsetinib can be brought to “patients in need around the world more quickly”. “Roche has unmatched capabilities to use data and diagnostics to identify patients based on the specific characteristics of their tumours,” states Law. The purpose of this design was to provide patients with long-lasting benefits”.Ī Roche spokesperson adds: “Based on clinical data available to date, the depth of response has been very encouraging, with a complete response rate in NSCLC patients that is uncommon in advanced cancer and differentiated from other RET inhibitors.” Pralsetinib was also well tolerated in this cancer type. ![]() Not only does pralsetinib target certain genetic RET alterations that cause the tumours, Law notes “it was also built to inhibit additional mutations that occur over time and make cancer resistant to treatment. The idea is that a precision therapy, such as pralsetinib, will “selectively target oncogenic RET alterations, with the goal of enabling durable clinical benefit and a favourable safety profile,” Law explains. To this end, the company has filed a new drug application to the US Food and Drug Administration for pralsetinib for the treatment of RET-altered NSCLC and thyroid cancers. Instead, Blueprint hopes to improve the treatment paradigm with its “medicines that precisely target the genetic causes of cancer and rare diseases”, with the aim of providing “targeted therapies to patients most likely to benefit from them”, explains Law. “In addition, frequent dose modification or interruption due to off-target toxicities limits the opportunity for sustained response.” “Current treatment of RET-altered cancers is generally limited to drugs called multi-kinase inhibitors, which non-selectively inhibit a range of targets at once, and chemotherapy, which often have limited efficacy and carry significant off-target toxicities,” notes Law. Need for precision medicine to treat RET-altered cancers Blueprint will be eligible for tiered royalty payments for net sales of the drug outside of the US. ![]() While Blueprint and Genentech will co-commercialise the drug in the US, Roche will obtain exclusive rights for pralsetinib outside of the US, except Greater China. Blueprint will also be eligible for up to $927m in contingent payments related to sales and regulatory milestones. “We are very excited to enter into this collaboration with Blueprint Medicines with the goal of bringing a potentially transformative new treatment option to patients with this rare type of RET-altered cancers as quickly as possible.” Terms of the agreement between Blueprint and Rocheīlueprint will receive $775m in upfront payments from Roche $100m of this comes in the form of an equity investment in Blueprint’s common stock. “While RET-activating fusions and mutations are key disease drivers in many cancer types, including NSCLC and MTC, current treatment options that selectively target these alterations are limited,” notes a spokesperson for Roche.
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