A drug from Eli Lilly that targets rare mutations in lung and thyroid cancers has had Food and Drug Administration approval for less than a week, and a competitor is already on the way.
Cambridge, Massachusetts-based Blueprint Medicines announced Thursday the release of an abstract of registration-directed data from its Phase I/II ARROW study of pralsetinib, a RET inhibitor, in 116 patients with advanced non-small cell lung cancer (NSCLC), ahead of the American Society of Clinical Oncology’s 2020 virtual annual meeting.
The ARROW abstract’s release closely follows the May 8 announcement that the Food and Drug Administration had given accelerated approval to Eli Lilly’s RET inhibitor, Retevmo (selpercatinib), in RET fusion-positive non-small cell lung cancer and thyroid cancer and RET-mutated medullary thyroid cancer (MTC). Retevmo was the first RET inhibitor to receive FDA approval.
Blueprint’s shares were up slightly on the Nasdaq when markets closed Thursday.
According to the data, the overall response rate (ORR) was 65%, with a 93% disease-control rate. The ORR included a 61% rate among patients who had received prior platinum chemotherapy treatment, as well as 73% among those who had not received prior treatment. Overall, there was a 6% rate of complete responses – with seven patients achieving a CR – including four among patients with prior platinum chemotherapy and three among treatment-naive patients.
According to safety data, which included 354 patients, most of the treatment-related adverse events were mild to moderate, and 4% of patients discontinued treatment due to them.
Blueprint said in its first quarter earnings last week that it had submitted its approval application for pralsetinib in RET fusion-positive NSCLC to the Food and Drug Administration and European Medicines Agency. It also plans to file under the FDA’s Real-Time Oncology Review pilot program for the drug’s approval in RET-mutant MTC in the second quarter. In a note to investors, Cowen analysts noted that the company had requested a priority review for RET fusion-positive NSCLC, which could result in a six-month review timeline.
The Cowen analysts further wrote that they continue to view Retevmo and pralsetinib as largely interchangeable drugs. “[Blueprint] is optimistic that its directionally superior CR rates will ultimately translate into improved long-term durability, though we think this is far more likely a 2021 story rather than a discussion that will be settled at ASCO,” the report read, adding that Cowen expects pralsetinib will captures about one-third of the market based on its second-place entry.
Lilly is also presenting a poster of updated data from LIBRETTO-1, the trial that led to Retevmo’s accelerated approval, in NSCLC. The poster includes data on 105 patients, which showed an ORR of 70%, including 90% among treatment-naive patients.
Photo: nopparit, Getty Images