Blueprint Medicines Announces Top-line Data for Pralsetinib and Initiates Rolling NDA Submission to FDA for the Treatment of Patients with RET Fusion-Positive Non-Small Cell Lung Cancer
"As the clinical data for pralsetinib have matured, with deep and durable responses along with robust evidence of activity against brain metastases, our confidence has continued to grow in the potential of pralsetinib to provide lasting benefit to a broad population of patients with RET fusion-positive NSCLC, including those with newly diagnosed unresectable or metastatic disease," said
Top-line Data from Phase 1/2 ARROW Trial in RET Fusion-Positive NSCLC
Results from the Phase 1/2 ARROW clinical trial of pralsetinib will be used to support the NDA submission for pralsetinib for the treatment of patients with RET fusion-positive NSCLC. The registration endpoints are ORR and DOR based on independent central radiology and Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST 1.1) criteria.
Top-line efficacy data were reported for patients treated with pralsetinib who were evaluable for response assessment per RECIST 1.1, as determined by blinded independent central review. All patients received the proposed indicated dose of 400 mg once daily (QD).
In 80 patients with RET fusion-positive NSCLC previously treated with platinum-based chemotherapy, the ORR was 61 percent (95% CI: 50-72%) per independent central review (two responses pending confirmation) as of a data cutoff date of
In 26 patients with treatment-naïve RET fusion-positive NSCLC, the ORR was 73 percent (95% CI: 52-88%) per independent central review (all responses confirmed), with 12 percent of patients achieving a complete response. All patients had tumor shrinkage.
Top-line safety data were consistent with those previously reported. Pralsetinib was well-tolerated, and most adverse events (AEs) were Grade 1 or 2. Across all patients enrolled in the ARROW trial treated with the proposed indicated dose of 400 mg QD (N=354), only four percent of patients discontinued treatment with pralsetinib due to treatment-related AEs.
Planned Phase 3 AcceleRET Lung Trial in Treatment-Naïve RET Fusion NSCLC
The global, randomized AcceleRET trial will enroll approximately 250 patients with advanced or metastatic RET fusion-positive NSCLC who have received no prior systemic therapy for metastatic disease. Participants will be randomized to receive either pralsetinib or the investigator's choice of platinum-based chemotherapy regimen with or without pembrolizumab. The trial's primary endpoint is PFS and secondary endpoints include overall survival, ORR and DOR. Patients may receive local testing to identify a RET fusion. In addition, patients randomized to the control arm may crossover upon progression to receive pralsetinib. Multiple trial sites are planned in
About RET-Altered Solid Tumors
RET activating fusions and mutations are key disease drivers in many cancer types, including NSCLC and MTC. 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 (PTC), while RET mutations are implicated in approximately 90 percent of patients with advanced MTC. In addition, oncogenic RET alterations are observed at low frequencies in colorectal, breast, pancreatic and other cancers, and RET fusions have been observed in patients with treatment-resistant, EGFR-mutant NSCLC.
Currently, there are no approved therapies that selectively target RET-driven cancers, although there are several approved multi-kinase inhibitors (MKIs) with RET activity being evaluated in clinical trials. To date, clinical activity attributable to RET inhibition has been uncertain for these approved MKIs, likely due to insufficient inhibition of RET and off-target toxicities. There is a need for precision therapies that provide durable clinical benefit by selectively targeting RET alterations and anticipated resistance mutations.
Pralsetinib is an investigational, once-daily oral precision therapy specifically designed for highly potent and selective targeting of oncogenic RET alterations.
Pralsetinib was designed by
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, statements regarding plans and timelines for the development of its drug candidates, including the timing, design, implementation, enrollment, plans and announcement of results regarding
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