Roche and its Genentech subsidiary will partner to develop and commercialize the once-daily oral cancer drug pralsetinib, through an expansion of a four-year-old immuno-oncology collaboration between the companies that could generate more than $1.7 billion for the Cambridge, MA, precision therapy developer. [Rita Elena Serda, Duncan Comprehensive Cancer Center at Baylor College of Medicine, National Cancer Institute, NIH]

Roche and its Genentech subsidiary will partner to develop and commercialize the once-daily oral cancer drug pralsetinib, through an expansion of a four-year-old immuno-oncology collaboration between Roche and Blueprint Medicines that could generate more than $1.7 billion for the Cambridge, MA, precision therapy developer.

The expanded collaboration is intended to combine Roche’s global commercial reach, integrated personalized healthcare capabilities, and cancer treatment portfolio with Blueprint Medicines’ expertise in targeted therapies—as reflected in pralsetinib (formerly BLU-667), a selective inhibitor of oncogenic RET alterations that has generated positive clinical results over the past year.

Blueprint Medicines is developing pralsetinib for the treatment of patients with RET-altered non-small cell lung cancer (NSCLC), various types of thyroid cancer and other solid tumors.

Most recently in May at the American Society for Clinical Oncology (ASCO) 2020 Virtual Scientific Program, researchers from Blueprint and numerous research institutions presented data from the ongoing Phase I/II ARROW clinical trial (NCT03037385). The ARROW trial consisted of a Phase I dose escalation (30–600 mg once or twice daily) followed by a Phase II expansion (400 mg once daily) in patients with advanced RET-altered solid tumors. Primary objectives were overall response rate (ORR)  and safety.

In 80 patients with RET fusion-positive NSCLC who were previously treated with platinum-based chemotherapy, pralsetinib showed an ORR of 61% (49 patients), as well as two partial responses confirmed after the data cutoff on November 18, 2019. Five percent of patients showed a confirmed response (CR) while 14% of patients had complete regression of target tumors. In 26 patients with no prior systemic therapy, the confirmed ORR was 73% and the CR rate was 12%.

“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 personalized treatments for patients,” James Sabry, Head of Roche Pharma Partnering, said in a statement.

Added Blueprint Medicines CEO Jeff Albers: “The collaboration is transformative for Blueprint Medicines and our efforts to build the leading precision medicine company, as it enables us to continue to build best-in-class commercial capabilities, further invest in our rapidly growing pipeline including our systemic mastocytosis programs and fortify our strong financial position to bridge the company to a self-sustaining future.”

Blueprint Medicines and Genentech, a member of the Roche Group, have agreed to co-commercialize pralsetinib in the U.S., with the companies equally sharing responsibilities, profits and losses. Roche will obtain exclusive commercialization rights for pralsetinib outside of the U.S., excluding Greater China, where CStone Pharmaceuticals retains all rights to developing and commercializing pralsetinib through an existing collaboration with Blueprint Medicines.

The companies also agreed to expand development of pralsetinib in multiple treatment settings and explore development of a next-generation RET inhibitor.

Blueprint Medicines and Roche agreed to co-develop pralsetinib globally in RET-altered solid tumors, including NSCLC, MTC and other thyroid cancers, as well as other solid tumors. The companies also agreed to share global development expenses based on pre-specified cost-sharing.

Roche will pay Blueprint Medicines $775 million upfront, including $675 million cash and a $100 million equity investment by Roche in Blueprint’s common stock at a purchase price of $96.57 per share, a 22% premium over yesterday’s closing stock price of $78.97. Roche also agreed to pay Blueprint up to an additional $927 million in payments tied to achieving development, regulatory, and sales-based milestones for pralsetinib and any licensed product containing a next-generation RET compound.

In addition, Blueprint Medicines is eligible to receive tiered royalties ranging from high-teens to mid-twenties on annual net sales of pralsetinib outside the U.S.

The closing of a minority portion of the equity investment is subject to expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and other customary closing conditions.

Roche and Blueprint Medicines began collaborating in 2016, when the companies agreed to develop up to five small-molecule therapeutics targeting kinases deemed important in cancer immunotherapy. At the time, Roche agreed to pay Blueprint $45 million upfront and up to an additional approximately $965 million in option fees and milestone payments.

Blueprint Medicines said it anticipated its existing cash and investments, together with the upfront payments and anticipated product revenues, to provide sufficient capital to enable the company to achieve financial self-sustainability as the company continues to commercialize and expand development of its approved medicine AYVAKIT™ (avapritinib) and advance new research programs.

“With Roche’s global reach and unparalleled expertise in personalized 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,” Albers added.

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