Blueprint tears up plans, dropping 2 lung cancer programs after seeing early clinical data

Blueprint Medicines is pulling back from one of its three areas of focus, dropping two non-small cell lung cancer (NSCLC) programs in response to early clinical data on the EGFR tyrosine kinase inhibitors.

One year ago, Blueprint CEO Kate Haviland used a presentation at the J.P. Morgan Healthcare Conference to explain how the company designed a phase 1 EGFR-driven lung cancer study to give “the confidence that we can win in first line.” The decision to spend 2023 gathering data to inform a go/no-go decision reflected Haviland’s recognition that lung cancer was an ambitious and potentially “expensive endeavor.”

Now, Blueprint has early evidence of whether its two drug candidates, BLU-945 and BLU-451, can deliver on its aim to develop best-in-class molecules capable of replacing the standard of care in frontline, EGFR lung cancer. Haviland pulled investment in the programs after getting a look at the data. 

“Combining this emerging data, from both the BLU-945 and the BLU-451 programs, with the evolving EGFR landscape, we have decided to deprioritize continued investment in EGFR-driven non-small cell lung cancer and to focus our R&D investments in the most compelling and highest value programs,” the CEO said at a JPM presentation this week.

Blueprint’s interest in EGFR-driven lung cancer was built on evidence that the first three generations of TKIs had failed to address the need for a molecule that maintains activity against sensitizing mutations, notably L858R. The L858R mutation is associated with worse outcomes on the current standard of care. Blueprint believed a combination with AstraZeneca’s Tagrisso could provide more durable benefits. 

While the biotech has opted against spending its own cash to pursue that belief, it will “explore strategic options, including potential out-licensing” to support further development of BLU-945 and BLU-451.

Blueprint disclosed the news alongside an update on its plans for Gavreto, the approved RET inhibitor that Roche decided to drop 11 months ago. Lacking global infrastructure in lung and thyroid cancer, the approved indications for Gavreto, Blueprint is stopping global development and marketing of the drug outside of the U.S. and China.

CStone Pharmaceuticals owns the rights to Gavreto in China. In the U.S., Blueprint has found “a potential alternate partner” and “is continuing to work with the involved parties to define a scenario that enables continued availability of Gavreto.” Winding down work on Gavreto outside of those markets will reduce operating expenses without affecting the $175 million Blueprint received from Royalty Pharma in 2022.