Roche is paying Blueprint Medicines $775 million upfront for rights to RET inhibitor pralsetinib. The deal sets Roche up to compete with Eli Lilly for patients with RET fusion-positive tumors of organs including the lung and thyroid.
Blueprint has filed for approval of pralsetinib in the U.S. and Europe this year on the strength of data linking it to a 91% response rate in RET fusion thyroid cancer. Pralsetinib achieved a 65% response rate in RET fusion-positive non-small cell lung cancer (NSCLC) and a 50% response rate across all RET fusion-positive solid tumors.
The data have persuaded Roche to write a check. Roche is set to pay $675 million in cash and make a $100 million equity investment in Blueprint—at a share price $20 above the biotech’s last close—to seal the deal. Blueprint is also in line to receive up to $927 million in milestones.
In return, Roche will receive an exclusive license to sell pralsetinib outside of the U.S. and China. Roche, through its Genentech unit, will co-promote pralsetinib with Blueprint in the U.S. CStone Pharmaceuticals has the rights to pralsetinib in greater China.
The deal sets the stage for a showdown between Roche and Lilly, which acquired a RET inhibitor now sold as Retevmo in its $8 billion takeover of Loxo Oncology. Lilly won FDA approval for Retevmo in the treatment of NSCLC and two types of thyroid cancer in May.
Blueprint is targeting the same indications and is set to learn whether the FDA will approve its RET inhibitor in RET fusion-positive NSCLC in November. Raymond James analyst Dane Leone has previously speculated that Blueprint could win approval before the PDUFA date. Blueprint is also working to get pralsetinib approved in indications other than NSCLC and in markets outside the U.S., including through the Project Orbis initiative that involves regulators in Australia and Canada.
If pralsetinib wins approval, Roche and Blueprint will face the challenge of competing with a drug that has a head start and comparable response data for patients with RET fusions and mutations. RET fusions are common in medullary thyroid cancer but are only found in 1% to 2% of NSCLC patients. The poor outcomes associated with RET fusion-positive lung cancer mean pralsetinib and Retevmo could corner that subpopulation, but there is still a limited market for the drugs to fight over.
Roche and Blueprint hope to grow the number of patients eligible for treatment with pralsetinib. The deal comes tied to plans to “expand development of pralsetinib in multiple treatment settings.” RET fusions are found at low levels in cancers of the breast, pancreas and other organs. The partners also plan to work on a next-generation RET inhibitor. Roche has the right to opt in to the program.
For Blueprint, the deal represents a chance to achieve financial sustainability. With Ayvakit on the U.S. market—albeit not in all the hoped-for indications—and the Roche deal providing a significant cash boost, Blueprint thinks it has the means to evolve into a self-sustaining commercial company.