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Roche to Pay $775M to Blueprint Medicines for Rights to Pralsetinib for RET-Altered Tumors

This story has been updated to clarify comments made by Blueprint Medicines' Chrissy Ross during a call with investors. 

NEW YORK – Roche said on Tuesday that it will pay Blueprint Medicines $775 million for certain rights to its RET inhibitor pralsetinib under a collaboration agreement between the companies.  

Under the agreement, Blueprint will receive a cash payment of $675 million and Roche will purchase $100 million in Blueprint's common stock at $96.57 per share. Blueprint can also receive up to $927 million for developmental, regulatory, and sales-based milestones for pralsetinib and any licensed product containing a next-generation RET compound. Blueprint will also receive royalties on annual net sales of pralsetinib outside the US.

Blueprint and Roche subsidiary Genentech will co-commercialize pralsetinib in the US. Roche will also have exclusive commercialization rights for pralsetinib outside the US, except in China, Hong Kong, Macau, and Taiwan. CStone Pharmaceuticals will retain development and commercialization rights for pralsetinib in greater China under a previous collaboration with Blueprint Medicines.

Blueprint CEO Jeff Albers said in a statement that the collaboration will help the company commercialize pralsetinib on a global scale and allow it to further invest in other programs in its pipeline. The companies may also codevelop a next-generation RET inhibitor.

Blueprint, Roche, and Genentech plan to develop and commercialize pralsetinib for patients with RET-altered non-small cell lung cancer, medullary thyroid cancer, as well as other solid tumors. "Fortunately, we've seen pretty limited on-target resistance [with pralsetinib] but we understand that with targeted therapies, resistance does emerge over time," Albers said in a call with investors. "It's always been our plan when we initiated efforts with RET to continue development of backup molecules, as well as focus on where and how resistance could emerge."

Blueprint Chief Commercial Officer Chrissy Ross noted during the call that diagnostics and real-world data assets within Roche subsidiaries Foundation Medicine and Flatiron Health are also attractive tools for homing in on likely responders to treatments. 

Blueprint said in June that the new drug application for pralsetinib in RET fusion-positive NSCLC had been accepted by the US Food and Drug Administration and the company was expecting a decision by Nov. 23. The European Medicines Agency also accepted the company's marketing authorization application for the same indication. Additionally, the company recently submitted new drug applications for pralsetinib in RET-mutant and RET fusion-positive thyroid cancers.

During the call, Blueprint COO Kate Haviland said that the company is interested in the continued pursuit of a tumor-agnostic indication for pralsetinib in patients with RET-driven cancers. "We are in conversations to think about how to move pralsetinib earlier in the lines of therapy, as well as combination opportunities with other medicines within the Roche portfolio," she said.  

Roche has been investigating the efficacy of its ALK inhibitor alectinib (Alecensa) in patients with RET-altered cancers. Genentech had previously collaborated with the Dana-Farber Cancer Institute to conduct a Phase I/II trial of the drug in RET-rearranged NSCLC or RET-mutated thyroid cancer, but it was closed last year due to "slow accrual and lack of efficacy." 

However, Roche is still collaborating with the European Thoracic Oncology Platform to conduct a Phase II trial of alectinib in pretreated NSCLC patients with confirmed RET rearrangements. 

If pralsetinib is successfully marketed, it will compete with Eli Lilly's selpercatinib (Retevmo), which the FDA approved in May for three RET-driven cancer indications: line-agnostic RET fusion-positive metastatic NSCLC; RET-mutant advanced or metastatic medullary thyroid cancer that requires systemic therapy; and RET fusion-positive thyroid cancer that requires systemic therapy and has stopped responding to radioactive iodine therapy.