Blueprint Medicines Presents ARROW Trial Data for GAVRETO® (pralsetinib) Highlighting Durable Clinical Activity in Patients with Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer and Other Advanced Solid Tumors
"Precision therapies have significantly improved outcomes in non-small cell lung cancer driven by actionable biomarkers, and these pralsetinib data show the transformative impact of targeting RET alterations in the front-line treatment setting," said
"Updated data in metastatic RET fusion-positive non-small cell lung cancer underscore how GAVRETO may transform the standard of care, including in the first-line treatment setting," said
Clinical Activity Data
The ASCO data included response-evaluable populations comprising 216 patients with RET fusion-positive NSCLC who had measurable disease at baseline and received a starting GAVRETO dose of 400 mg once daily, and 19 patients with other RET fusion-positive solid tumors, as of a date cutoff date of
RET Fusion-Positive NSCLC
With a median follow-up of 17.1 months, GAVRETO showed durable clinical benefits in patients with RET fusion-positive NSCLC with or without prior therapy. In 68 treatment-naïve patients, the overall response rate (ORR) was 79 percent (95% CI: 68%, 88%). The complete response (CR) rate was 6 percent, 10 percent of patients had complete regression of target tumors, and 74 percent of patients had a partial response (PR). The median duration of response (DOR) was not reached (95% CI: 9.0 months, not reached).
For treatment-naïve patients, the initial study protocol limited enrollment to those determined by the investigator to be ineligible for standard platinum-based chemotherapy, which may be due to age, comorbidities or other poor prognostic factors. This eligibility restriction was removed in
In 126 patients who previously received platinum-based chemotherapy, the ORR was 62 percent (95% CI: 53%, 70%). The CR rate was 4 percent, 12 percent of patients had complete regression of target tumors, and 58 percent of patients had a PR. The median DOR was 22.3 months (95% CI: 15.1 months, not reached).
Other RET Fusion-Positive Solid Tumors
In a heavily pre-treated patient population who had a median follow-up of 12.1 months, GAVRETO showed clinical activity across multiple additional RET-driven tumor types. In 19 patients with a variety of RET fusion-positive solid tumors beyond NSCLC and thyroid cancer, the ORR was 53 percent (95% CI: 29%, 76%) and the median DOR was 19.0 months (95% CI: 5.5 months, not estimable). Tumor reductions were shown in patients with the following cancers – pancreatic, cholangiocarcinoma, colon, lung (except NSCLC), mesenchymal, salivary duct, sweat gland and thymus – as well as patients diagnosed with cancers of unknown primary origin. In the three patients with pancreatic cancer, a particularly difficult-to-treat tumor type, there was one CR and two PRs.
As of a data cutoff date of
These updated data will be reported in two ASCO poster presentations: one on RET fusion-positive NSCLC (Abstract #9089) and one on RET fusion-positive solid tumors (Abstract #3079). Copies of the data presentations will be available starting
About GAVRETO (pralsetinib)
GAVRETO (pralsetinib) is a once-daily oral targeted therapy approved by the
GAVRETO is not approved for the treatment of any other indication in the
GAVRETO is designed to selectively and potently target oncogenic RET alterations, including secondary RET mutations predicted to drive resistance to treatment. In preclinical studies, GAVRETO inhibited RET at lower concentrations than other pharmacologically relevant kinases, including VEGFR2, FGFR2 and JAK2. For more information, visit GAVRETO.com.
Enrollment is ongoing in the Phase 1/2 ARROW trial, including for patients with various RET fusion-positive solid tumors, and in the Phase 3 AcceleRET Lung trial for treatment-naïve patients with RET fusion-positive NSCLC. For more information about GAVRETO clinical trials, visit www.clinicaltrials.gov or www.blueprintclinicaltrials.com.
About RET-Altered Solid Tumors
RET activating fusions and mutations are key disease drivers in many cancer types, including NSCLC and multiple types of thyroid cancer. RET fusions are implicated in approximately 1 to 2 percent of patients with NSCLC and approximately 10 to 20 percent of patients with papillary thyroid cancer, while RET mutations are implicated in approximately 90 percent of patients with advanced MTC. In addition, oncogenic RET fusions are observed at low frequencies in colorectal, breast, pancreatic and other cancers, as well as in patients with treatment-resistant EGFR-mutant NSCLC.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, plans, strategies, timelines and expectations for
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