Roche Collaborates with Blueprint Medicines to Develop Personalized Medicine for Lung and Thyroid Cancer

By Daniel Ojeda, Ph.D.

On July 14th, Roche and Blueprint Medicines Corporation announced a licensing and collaboration agreement to co-develop and co-commercialize Pralsetinib, a once-daily oral therapy for the treatment of RET-altered NSCLC, medullary thyroid and other types of thyroid cancers, as well as other solid tumors.

Under the agreement, Roche will pay $675 million upfront and a $100 million equity investment in Blueprint Medicines. Together the companies plan to explore the development of the next-generation of RET inhibitors, as well as explore the use of pralsetinib in other cancers. The deal also includes up to an additional $927 million in potential milestones, plus royalties on sales outside the US for Blueprint Medicines.

RET Fusion Cancers and Personalized Medicine

The RET (Rearranged during Transfection) protein, a proto-oncogene, is a member of the tyrosine kinase family of proteins [1]. In cancer, the RET gene can recombine with other genes resulting in the protein being constantly activated [2]. This fusion protein can then result in cancer. Non-small cell lung cancer (NSCLC) is the most common type of lung cancer [3], and approximately 2% of them harbor RET fusions [4]. Furthermore, RET fusions can be found in 90% of advanced medullary thyroid cancer (MTC) tumors [5].

The idea of personalized medicine is to use genomic data to determine specific targets for each patient and possibly at different stages of cancer. Pralsetinib now joins Roche’s broad portfolio of cancer treatments and moves the company towards its goal to develop more personalized treatment approaches using genetic mutation markers instead of tumor sites of origin.

James Sabry, Head of Roche Pharma Partnering said: “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.”

After positive results in the Phase 1/2 ARROW clinical trial in early 2020 [6], a new drug application for pralsetinib has been submitted by Blueprint Medicines to the FDA and it is expecting a decision before the end of the year. If approved, pralsetinib will become the second drug approved targeting RET fusions after the approval of retevmo developed by Eli Lilly.

Related Article: Sarepta and Codiak Biosciences Join Hands to Tackle Rare Diseases Using Exosome-Based Therapeutics

References

1. https://www.genecards.org/cgi-bin/carddisp.pl?gene=RET
2. https://pubmed.ncbi.nlm.nih.gov/32326537/
3. https://www.cancer.net/cancer-types/lung-cancer-non-small-cell/statistics#:~:text=NSCLC%20is%20the%20most%20common,be%20diagnosed%20with%20lung%20cancer.
4. https://www.sciencedirect.com/science/article/pii/S2666364320300552
5. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5839408/
6. http://ir.blueprintmedicines.com/news-releases/news-release-details/blueprint-medicines-announces-top-line-data-pralsetinib-and

Author

Daniel Ojeda