BioPharma

Blueprint throws down targeted therapy gauntlet with new lung cancer data, FDA submission

The biotech company announced data from its Phase I/II study of pralsetinib in RET fusion-positive non-small cell lung cancer and kicked off a rolling submission for FDA approval. Eli Lilly Oncology announced data for its own RET inhibitor in September.

A company developing targeted therapies for cancers expects to complete submission of data for one of its drugs in lung cancer by the end of the first quarter, based on top-line data from a trial of patients whose disease carries a particular genetic anomaly.

Cambridge, Massachusetts-based Blueprint Medicines said Wednesday that it had started a rolling submission to the Food and Drug Administration for the drug BLU-667 (pralsetinib), based on data from its Phase I/II study in patients with RET fusion-positive non-small cell lung cancer.

Shares of the company were up about 1.8% on the Nasdaq following the news. The company also plans to submit for regulatory approval of the drug in medullary thyroid cancer in the second quarter of 2020.

Blueprint is one of multiple companies developing RET inhibitors. Its most advanced competitor is Indianapolis-based Eli Lilly’s oncology division, whose RET inhibitor LOXO-292 (selpercatinib) has also shown positive data in RET-positive NSCLC, per an announcement by the company in September, while also being tested in other cancers that carry the mutation. Lilly acquired selpercatinib’s original developer, Loxo Oncology, last year for $8 billion.

In its Wednesday announcement, Blueprint said that pralsetinib showed a 61% overall response rate in 80 patients who had received prior platinum-based chemotherapy, and the median duration of response had not yet been reached. Overall, 95% of patients had tumor shrinkage, with 14% showing complete regression of tumors. Among 26 patients who had not received prior treatment, the overall response rate was 73%, including a 12% complete response rate. The drug was deemed well-tolerated, with most side effects being mild to moderate.

In a note to investors in September, following Lilly’s announcement, Cowen analysts expressed a positive view toward selpercatinib, but wrote that pralsetinib remains competitive, with Lilly’s drug not appearing clearly superior to Blueprint’s, as proposed points of differentiation appeared to weaken with each data update. The most recent data for selpercatinib, in 105 NSCLC patients, showed an overall response rate of 68%, with a 20.3-month median duration of response and 18.4-month median progression-free survival. Meanwhile, an analysis of 34 patients who had not received prior therapy showed an 85% overall response rate.

In a note Wednesday, Cowen’s Marc Frahm wrote that Blueprint’s latest data confirm pralsetinib’s competitive profile in NSCLC. On the one hand, he wrote, Lilly has a competitive edge in being a much larger company with data for selpercatinib that is ahead of Blueprint’s in terms of timing and number of patients treated. As such, he forecast that Blueprint would likely command a minority share of the market, around 30%, while needing a partner outside the U.S., with peak sales reaching about $300 million per year. Still, pralsetinib’s overall response rate is only slightly below that of selpercatinib’s 68%, while the complete response rate of 12-14% appears directionally higher than selpercatinib’s 2-3%, he wrote.

Photo: Donald Miralle, Getty Images

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