RARE Daily

Blueprint Enters Collaboration with Roche to Develop Treatment for RET-Altered Cancers

July 14, 2020

Blueprint Medicines entered into a $1.7 billion global collaboration with Roche and its subsidiary Genentech to develop and commercialize pralsetinib, an experimental therapy for the treatment of patients with cancers driven by oncogenic RET alterations.

Photo: Jeff Albers, CEO of Blueprint Medicines

RET activating fusions and mutations are key disease drivers in many cancer types, including non-small cell lung cancer (NSCLC) and multiple types of thyroid cancer. RET fusions are implicated in approximately 1 to 2 percent of patients with NSCLC and approximately 10 to 20 percent of patients with papillary thyroid cancer, while RET mutations are also implicated in approximately 90 percent of patients with advanced medullary thyroid cancer (MTC), both rare cancers. In addition, oncogenic RET alterations are observed at low frequencies in colorectal, breast, pancreatic and other cancers, and RET fusions have been observed in patients with treatment-resistant EGFR-mutant NSCLC.

BluePrint, a precision therapy company focused on genomically defined cancers, rare diseases, and cancer immunotherapy, developed pralsetinib as a once-daily oral precision therapy that selectively targets oncogenic RET alterations. The company is developing it to treat NSCLC, MTC, and other solid tumors, and has submitted pralsetinib to regulatory agencies in the United States and Europe for marketing approval for the treatment of patients with RET-altered NSCLC.

Blueprint has been collaborating with Roche for the past four years. This new deal will allow the biotech, which markets a drug for rare gastrointestinal stromal tumors, to achieve a self-sustainable financial business.

“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,” said Jeff Albers, CEO of Blueprint Medicines.

As part of the collaboration with Roche, Blueprint Medicines and Genentech will co-commercialize pralsetinib in the United States and Roche will obtain exclusive commercialization rights for pralsetinib outside of the United States, excluding Greater China, where it is under license to CStone Pharmaceuticals. The companies also plan to expand development of pralsetinib in multiple treatment settings and explore development of a next-generation RET inhibitor as part of the collaboration, for which Roche will have a right to opt in.

Roche will pay Blueprint Medicines $775 million in upfront payments, including a cash payment of $675 million and an equity investment by Roche of $100 million of Blueprint Medicines stock at a purchase price of $96.57 per share, a 26 percent premium to its share price at the time of the deal announcement. Blueprint Medicines will be eligible to receive up to an additional $927 million in additional payments contingent to specified development, regulatory and sales-based milestones for pralsetinib and any licensed product containing a next-generation RET compound. Blueprint is also eligible to receive tiered royalties ranging from high-teens to mid-twenties on annual net sales of pralsetinib outside the United States.

Blueprint Medicines and Roche have also 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 will share global development expenses based on pre-specified cost sharing.

The deal leverages Roche’s diagnostic capabilities. “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,” said James Sabry, head of Roche Pharma Partnering.

The U.S. Food and Drug Administration has granted priority review for pralsetinib for RET fusion-positive NSCLC, with a PDUFA action date of November 23, 2020.

Author: Rare Daily Staff

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