Roche to Co-Develop Investigational Therapy for RET-Altered Cancers

Roche and Blueprint Medicines Corporation recently signed an agreement to provide exclusive rights to Roche for global co-development and commercialization of an investigational therapy for patients with RET-altered cancers.

Genentech, a member of Roche Group, will obtain co-commercialization rights to the therapy, pralsetinib, which is intended for RET-altered cancers, including non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC), and other types of thyroid cancer.

Pralsetinib demonstrated tumor-agnostic potential as well.

Roche will pay upfront of $675 million in cash in addition to a $100 million equity investment in Blueprint Medicines, which is eligible to receive up to $927 million in potential milestones, plus royalties on net product sales outside the US.

Through the collaboration, the companies plan to expand the development of pralsetinib in numerous treatment settings and explore development of a next-generation RET inhibitor.

“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,” James Sabry, head of Roche pharma partnering, said 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 personalized treatments for patients,” he added.

RET-activating fusions and mutations are key disease drivers in many cancer types, including NSCLC and MTC, Roche officials said. But treatment options for these mutations are limited.

In lung cancer, pralsetinib will complement Roche’s already approved medicines, such as Alecensa, Rozlytrek, Tecentriq, Avastin and Tarceva.

At the end of May, FDA approved Roche’s first-line treatment, Tecentriq (atezolizumab), for adult patients with metastatic NSCLC.

Atezolizumab was the fourth indication in metastatic non-small cell lung cancer and fifth indication in lung cancer overall in the US.

The approval was based on results from Roche’s clinical trial, IMpower110: a multicenter, international, open-label trial for patients with stage IV non-small cell lung cancer whose tumors express PD-L1 and who had received no prior chemotherapy for metastatic disease.

Researchers found that atezolizumab improved overall patient survival by 7.1 months compared with chemotherapy.

Median survival was 20.2 months for patients for atezolizumab compared with 13.1 months for chemotherapy. There was no statistically significant difference in survival for the other two PD-L1 subgroups at the interim or final analyses.

Additionally, median progression-free survival per investigator was slightly more than eight months for atezolizumab and five months for the platinum-based chemotherapy.

“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,” Jeff Albers, Blueprint Medicines CEO, said in the recent announcement.

Recently, Blueprint Medicines submitted a new drug application (NDA) to FDA and a marketing authorization application to the European Medicines Agency (EMA), for pralsetinib.

FDA granted priority review with an expected decision date of November 23, 2020, Roche officials said.

Additionally, FDA recently accepted Blueprint Medicines MTC application for its Real-Time Oncology Review (RTOR) pilot program, which aims to enhance the review process to ensure safe and effective treatments are available to patients as early as possible.