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Blueprint Medicines is a fully integrated global biopharmaceutical company that invents life-changing medicines across two strategic focus areas of allergy/inflammation and oncology/hematology. Our approach combines deep scientific expertise, modality-agnostic drug discovery capabilities, and a proven track record of R&D success. Currently, we are commercializing our approved medicine AYVAKIT/AYVAKYT® (avapritinib) in the U.S. and Europe, as well as in other regions of the world through partnerships.

News release details

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

May 19, 2021 at 5:00 PM EDT
-- 88% ORR and median duration of response not reached in treatment-naïve patients with non-small cell lung cancer enrolled after expansion of trial inclusion criteria to include patients eligible for standard-of-care front-line therapy --
-- 53% ORR in heavily pre-treated patients with RET fusion-positive tumors beyond non-small cell lung cancer and thyroid cancer --

CAMBRIDGE, Mass., May 19, 2021 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC) today announced the presentation of updated Phase 1/2 ARROW trial data demonstrating durable clinical benefits of GAVRETO® (pralsetinib) in metastatic RET fusion-positive non-small cell lung cancer (NSCLC) and other advanced solid tumors. GAVRETO showed high response rates in treatment-naïve patients with RET fusion-positive NSCLC, clinical activity across a number of RET fusion-positive tumor types and a safety profile consistent with previously reported results. These data will be presented during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting, June 4-8.

"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 Giuseppe Curigliano, M.D., Ph.D., Associate Professor of Medical Oncology at the University of Milano and the Head of the Division of Early Drug Development at the European Institute of Oncology, IRCCS, Italy. "In treatment-naïve metastatic NSCLC, these results are particularly encouraging, with many patients remaining in response. The data highlight the critical importance of identifying RET alterations before initiating treatment, so that more patients have the opportunity to benefit from targeted therapy."

"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 Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "The results in treatment-naïve patients with RET fusion-positive NSCLC reflect the compelling clinical profile of GAVRETO, with high response rates and durability of response that have strengthened over time. In other RET-altered cancers, target lesions were reduced across a diverse range of tumor types, with the majority of patients responding to treatment. In collaboration with Genentech, we look forward to engaging with the FDA based on the strength of these data in metastatic RET fusion-positive solid tumors."

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 November 6, 2020. Tumor response was assessed by blinded independent central review using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1, and all responses were confirmed.

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 July 2019, with the goal of including a population more reflective of real-world practice. In an exploratory analysis of treatment-naïve patients enrolled after this expansion of inclusion criteria (n=25), the ORR was 88 percent (95% CI: 69%, 98%), and all responses were PRs.

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.

Safety Data

As of a data cutoff date of November 6, 2020, a total of 471 patients were enrolled with a GAVRETO dose starting at 400 mg once daily. Across tumor types, GAVRETO was well-tolerated with no new safety signals observed. The most common treatment-related adverse events (AEs) reported by investigators (≥20 percent) were neutropenia, increased aspartate aminotransferase (AST), anemia, decreased white blood cell count, increased alanine aminotransferase (ALT), hypertension, constipation and asthenia. Overall, 6 percent of patients discontinued GAVRETO due to treatment-related AEs.

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 June 4, 2021 in the "Science―Publications and Presentations" section of Blueprint Medicines' website.

About GAVRETO (pralsetinib)

GAVRETO (pralsetinib) is a once-daily oral targeted therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of three indications: adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA approved test, adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy, and adults and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). These indications are approved under accelerated approval based on ORR and DOR. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials. In addition, GAVRETO is approved by the National Medical Products Administration (NMPA) of China for the treatment of adult patients with locally advanced or metastatic RET fusion-positive NSCLC after platinum-based chemotherapy.

GAVRETO is not approved for the treatment of any other indication in the U.S. by the FDA or in China by the NMPA, or for any indication in any other jurisdiction by any other health authority.

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

Blueprint Medicines and Roche are co-developing GAVRETO globally (excluding Greater China) for the treatment of patients with RET-altered NSCLC, various types of thyroid cancer and other solid tumors. The European Medicines Agency validated a marketing authorization application for GAVRETO for the treatment of RET fusion-positive NSCLC. The FDA granted breakthrough therapy designation to GAVRETO for the treatment of RET fusion-positive NSCLC that has progressed following platinum-based chemotherapy and for RET mutation-positive MTC that requires systemic treatment and for which there are no acceptable alternative treatments.

Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of GAVRETO in Greater China, which encompasses Mainland China, Hong Kong, Macau and Taiwan.

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 or

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.

About Blueprint Medicines

Blueprint Medicines is a global precision therapy company that invents life-changing medicines for people with cancer and hematologic disorders. Applying an approach that is both precise and agile, we create therapies that selectively target genetic drivers, with the goal of staying one step ahead across stages of disease. Since 2011, we have leveraged our research platform, including expertise in molecular targeting and world-class drug design capabilities, to rapidly and reproducibly translate science into a broad pipeline of precision therapies. Today, we are delivering our approved medicines to patients in the United States and Europe, and we are globally advancing multiple programs for genomically defined cancers, systemic mastocytosis, and cancer immunotherapy. For more information, visit and follow us on Twitter (@BlueprintMeds) and LinkedIn.

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 Blueprint Medicines' current or future approved drugs and drug candidates, including timelines for regulatory interactions for GAVRETO for the treatment of RET-altered cancers; the potential benefits of Blueprint Medicines' current and future approved drugs or drug candidates in treating patients; and Blueprint Medicines' strategy, goals and anticipated milestones, business plans and focus. The words "aim," "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks and uncertainties related to the impact of the COVID-19 pandemic to Blueprint Medicines' business, operations, strategy, goals and anticipated milestones, including Blueprint Medicines' ongoing and planned research and discovery activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Blueprint Medicines' ability and plans in continuing to establish and expand a commercial infrastructure, and successfully launching, marketing and selling current or future approved products; Blueprint Medicines' ability to successfully expand the approved indications for AYVAKIT™/AYVAKYT® (avapritinib) and GAVRETO or obtain marketing approval for AYVAKIT/AYVAKYT in additional geographies in the future; the delay of any current or planned clinical trials or the development of Blueprint Medicines' current or future drug candidates; Blueprint Medicines' advancement of multiple early-stage efforts; Blueprint Medicines' ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; the preclinical and clinical results for Blueprint Medicines' drug candidates, which may not support further development of such drug candidates; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; Blueprint Medicines' ability to obtain, maintain and enforce patent and other intellectual property protection for AYVAKIT/AYVAKYT, GAVRETO or any drug candidates it is developing; Blueprint Medicines' ability to develop and commercialize companion diagnostic tests for AYVAKIT/AYVAKYT, GAVRETO or any of its current and future drug candidates; and the success of Blueprint Medicines' current and future collaborations, partnerships or licensing arrangements. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in Blueprint Medicines' filings with the Securities and Exchange Commission (SEC), including Blueprint Medicines' most recent Annual Report on Form 10-K, as supplemented by its most recent Quarterly Report on Form 10-Q and any other filings that Blueprint Medicines has made or may make with the SEC in the future. Any forward-looking statements contained in this press release represent Blueprint Medicines' views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, Blueprint Medicines explicitly disclaims any obligation to update any forward-looking statements.


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