Pralsetinib, an investigational precision therapy in late-stage development for individuals with alterations in the RET gene, would be jointly sold in the United States by the 2 companies if it is approved.
Roche said Tuesday it will invest heavily in Blueprint Medicines on a global codevelopment and commercialization agreement for pralsetinib, an investigational precision therapy in late-stage development for individuals with alterations in the RET gene.
Roche will pay $675 million in cash in addition to a $100 million equity investment; Blueprint Medicines is eligible to receive up to $927 million in potential milestones, plus royalties on net product sales outside the United States.
The 2 companies will jointly market pralsetinib in the United States, if it is approved, while Roche will be responsible for commercial activities outside the United States, excluding Greater China. Pralsetinib is an investigational inhibitor of RET fusions and mutations.
Earlier this month, Blueprint Medicines submitted a new drug application (NDA) for pralsetinib to the FDA under priority review for treatment of patients with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) and RET fusion—positive thyroid cancers. In May, it submitted an NDA for pralsetinib in locally advanced or metastatic RET fusion—positive non–small cell lung cancer (NSCLC), with an expected decision by November 23, 2020.
It has also submitted a marketing authorization application to the European Medicines Agency.
The once-daily oral precision therapy has also shown promise as a tumor-agnostic treatment.
“Beyond lung cancer, pralsetinib’s tumour-agnostic potential further expands Roche's ongoing commitment to finding new approaches to treat cancer in a more personalised way based on the genetic mutation of the disease, irrespective of the tumour site of origin,” Roche said in a statement.
In May, the FDA approved another oral RET therapy, Eli Lilly’s selpercatinib (Retevmo), also for NSCLC and MTC.
In a cohort of 13 patients with RET fusion—positive MTC in the phase 1/2 ARROW study of pralsetinib in patients with select RET fusion—positive solid tumors, the MTC cohort achieved an objective response rate (ORR) of 74% in the treatment-naïve patients and an ORR of 60% in patients who had previous treatment.
The disease control rate seen in patients with RET fusion—positive thyroid cancers was 100% (95% CI, 72%-100%). The best response was a partial response, which was achieved in 91% of patients. Additionally, stable disease was observed in 9 patients and no patients had progressive disease.
“We are very excited to enter into this collaboration with Blueprint Medicines, a partner we have already been working with for four years, with the goal of bringing a potentially transformative treatment option to patients with rare RET-altered cancers as quickly as possible,” said James Sabry, head of Roche Pharma Partnering, in a statement. “In bringing pralsetinib to patients, we will leverage our global reach and expertise in oncology, as well as our capabilities in diagnostics and the use of real-world data toward our aim of providing personalised treatments for patients."
“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, Blueprint’s CEO.