BPMC Blueprint Medicines Corporation

80.36
+1.21  (+2%)
Previous Close 79.15
Open 80.02
52 Week Low 43.29
52 Week High 102.98
Market Cap $4,354,705,507
Shares 54,189,964
Float 46,361,822
Enterprise Value $3,717,207,650
Volume 122,576
Av. Daily Volume 626,153
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Upcoming Catalysts

Drug Stage Catalyst Date
Avapritinib BLU-285 - PATHFINDER
Advanced Systemic mastocytosis (SM)
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
Pralsetinib (BLU-667)
RET-mutant medullary thyroid cancer
NDA Filing
NDA Filing
NDA filing announced July 1, 2020.
Avapritinib - EXPLORER
Advanced Systemic mastocytosis (SM)
Phase 1
Phase 1
Phase 1 updated presented at EHA June 12, 2020.
BLU-263
Healthy volunteers
Phase 1
Phase 1
Phase 1 initiation of dosing announced June 6, 2020.
Avapritinib BLU-285 - PIONEER
Indolent and smoldering systemic mastocytosis (SM)
Phase 2
Phase 2
Phase 2 enrolment in Part 2 of the trial to commence June 2020.
Pralsetinib BLU-667 - ARROW
RET-altered solid tumors
Phase 1/2
Phase 1/2
Phase 1/2 updated data at ASCO May 29, 2020. ORR 61% in patients who previously received platinum-based chemotherapy.
Avapritinib
Fourth-line GIST
CRL
CRL
CRL issued May 15, 2020.
Avapritinib - Voyager
Gastrointestinal stromal tumors (GIST)
Phase 3
Phase 3
Phase 3 data did not meet primary endpoint - April 28, 2020.
Pralsetinib (BLU-667)
First-line RET-fusion non-small cell lung cancer (NSCLC)
NDA Filing
NDA Filing
NDA filing announced April 1, 2020.
Pralsetinib (BLU-667) - AcceleRET
First-line RET-fusion non-small cell lung cancer (NSCLC)
Phase 3
Phase 3
Phase 3 trial initiation announced January 13, 2020.
Avapritinib BLU-285
PDGFRA exon 18 mutant Gastrointestinal stromal tumors (GIST)
Approved
Approved
FDA Approval announced January 9, 2020.
Fisogatinib and CS1001
Hepatocellular Carcinoma
Phase 1/2
Phase 1/2
Phase 1b/2 trial initiation announced January 6, 2020.
BLU-782
Fibrodysplasia ossificans progressiva (FOP)
Phase 2
Phase 2
Phase 2 trial to be initiated 4Q 2019.

Latest News

  1. CAMBRIDGE, Mass., July 2, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ:BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced that, effective on July 1, 2020, the Compensation Committee of Blueprint Medicines' Board of Directors granted non-qualified stock options to purchase an aggregate of 30,778 shares of its common stock and an aggregate of 15,388 restricted stock units (RSUs) to 13 new employees under Blueprint Medicines' 2020 Inducement Plan.

    The 2020 Inducement Plan is used exclusively for the grant of equity awards to individuals who were not previously an employee or non-employee director of Blueprint Medicines, as an inducement material to such individual's entering into employment with Blueprint Medicines, pursuant to Rule 5635(c)(4) of the NASDAQ Listing Rules.

    The options have an exercise price of $79.02 per share, which is equal to the closing price of Blueprint Medicines' common stock on July 1, 2020. Each option will vest as to 25% of the shares underlying such option on the first anniversary of the grant date and as to an additional 1/48th of the shares underlying the option monthly thereafter, in each case, subject to each such employee's continued employment on each vesting date. Each RSU will vest as to 25% of the shares underlying the RSU award on the first anniversary of the grant date and as to an additional 25% of the shares underlying the RSU award annually thereafter, subject to each such employee's continued employment on each vesting date. The options and RSUs are subject to the terms and conditions of Blueprint Medicines' 2020 Inducement Plan, and the terms and conditions of the stock option and RSU agreement covering the grant.

    About Blueprint Medicines

    Blueprint Medicines is a precision therapy company striving to improve human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational medicines rooted in our leading expertise in protein kinases, which are proven drivers of disease. Our uniquely targeted, scalable approach empowers the rapid design and development of new treatments and increases the likelihood of clinical success. We have one FDA-approved precision therapy and are currently advancing multiple investigational medicines in clinical development, along with a number of research programs. For more information, visit www.BlueprintMedicines.com and follow us on Twitter (@BlueprintMeds) and LinkedIn.

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    SOURCE Blueprint Medicines Corporation

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  2. CAMBRIDGE, Mass., July 1, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ:BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for pralsetinib for the treatment of patients with advanced or metastatic RET mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancers. Pralsetinib is an investigational, once-daily precision therapy designed to potently and selectively inhibit RET fusions and mutations, including predicted resistance mutations.

    Blueprint Medicines submitted the NDA under the Real-Time Oncology Review pilot program (RTOR program), an initiative of the FDA's Oncology Center of Excellence. The RTOR program aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible, while maintaining and improving review quality by the FDA.

    "Pralsetinib has broad potential to address the medical needs of patients with RET-altered cancers, who have not traditionally benefited from targeted therapy even though their tumors have a known disease driver," said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. "There are now pending marketing applications for pralsetinib in RET-altered non-small cell lung cancer and thyroid cancers, supporting our goal to advance treatment standards for these patients. We are working closely with the FDA and aim to bring this promising treatment to patients as expeditiously as possible."

    In May 2020, Blueprint Medicines announced that the U.S. and EU marketing applications for pralsetinib for the treatment of locally advanced or metastatic RET fusion-positive non-small cell lung cancer (NSCLC) were accepted by the FDA and validated by the European Medicines Agency, respectively.

    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, 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.

    About Pralsetinib

    Pralsetinib is an investigational, once-daily oral precision therapy specifically designed for highly potent and selective targeting of oncogenic RET alterations. Blueprint Medicines is developing pralsetinib for the treatment of patients with RET-altered NSCLC, thyroid cancer and other solid tumors. The FDA has granted Breakthrough Therapy Designation to pralsetinib for the treatment of RET fusion-positive NSCLC that has progressed following platinum-based chemotherapy, and RET mutation-positive MTC that requires systemic treatment and for which there are no acceptable alternative treatments.

    Pralsetinib was designed by Blueprint Medicines' research team, leveraging the company's proprietary compound library. In preclinical studies, pralsetinib consistently demonstrated sub-nanomolar potency against the most common RET fusions, activating mutations and predicted resistance mutations. In addition, pralsetinib demonstrated markedly improved selectivity for RET compared to pharmacologically relevant kinases, including approximately 80-fold improved potency for RET versus VEGFR2. By suppressing primary and secondary mutants, pralsetinib has the potential to overcome and prevent the emergence of clinical resistance. Blueprint Medicines believes this approach will enable durable clinical responses across a diverse range of RET alterations, with a favorable safety profile.

    Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of pralsetinib and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for pralsetinib in the rest of the world.

    About Blueprint Medicines

    Blueprint Medicines is a precision therapy company striving to improve human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational medicines rooted in our leading expertise in protein kinases, which are proven drivers of disease. Our uniquely targeted, scalable approach empowers the rapid design and development of new treatments and increases the likelihood of clinical success. We have one FDA-approved precision therapy and are currently advancing multiple investigational medicines in clinical development, along with a number of research programs. For more information, visit www.BlueprintMedicines.com 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, statements regarding plans and timelines for the development of pralsetinib; plans and timelines for commercializing pralsetinib, if approved; the potential benefits of pralsetinib in treating patients; 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 plan in establishing a commercial infrastructure, and successfully launching, marketing and selling its approved product; the delay of any current or planned clinical trials or the development of Blueprint Medicines' drug candidates or licensed drug candidate; 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 develop and commercialize companion diagnostic tests for its current and future drug candidates; and the success of Blueprint Medicines' current and future collaborations 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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    SOURCE Blueprint Medicines Corporation

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  3. CAMBRIDGE, Mass., June 29, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ:BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced that The Lancet Oncology published data from the NAVIGATOR clinical trial showing an unprecedented overall survival (OS) rate and a well-tolerated safety profile for AYVAKIT™ (avapritinib) in patients with advanced PDGFRA D842V mutant gastrointestinal stromal tumor (GIST). The paper, titled "Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial," was published online in The Lancet Oncology on June 29, 2020.

    CAMBRIDGE, Mass., June 29, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ:BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced that The Lancet Oncology published data from the NAVIGATOR clinical trial showing an unprecedented overall survival (OS) rate and a well-tolerated safety profile for AYVAKIT™ (avapritinib) in patients with advanced PDGFRA D842V mutant gastrointestinal stromal tumor (GIST). The paper, titled "Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial," was published online in The Lancet Oncology on June 29, 2020.

    "The data published in The Lancet Oncology show that patients with PDGFRA D842V mutant GIST treated with AYVAKIT had deep and durable clinical responses as well as a high overall survival rate. These results represent a transformative advancement for patients whose tumor type is resistant to other approved therapies," said Michael Heinrich, M.D., Professor of Medicine at Oregon Health & Science University and primary author of the paper. "As a scientist who has dedicated my career to understanding the molecular basis of GIST and as a clinician treating patients, it's tremendously rewarding to be able to offer – for the first time – a highly effective treatment option to my patients with PDGFRA D842V mutant GIST."

    "Based on the compelling clinical data from the NAVIGATOR trial, AYVAKIT was granted a full approval by the FDA earlier this year and has become the new standard of care for patients with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation," said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. "We are now focused on bringing AYVAKIT to additional patients with this genomically defined form of GIST in other geographies, including Europe."

    Highlights from The Lancet Oncology Publication Data

    The Lancet Oncology paper reported efficacy and safety results from the NAVIGATOR trial, including all patients enrolled in the dose escalation part of the trial and the subset of patients with PDGFRA D842V mutant GIST enrolled in the expansion part of the trial. The efficacy population comprised 56 patients with PDGFRA D842V mutant GIST. The safety population comprised 82 patients, including 26 patients with non-PDGFRA D842V mutant GIST enrolled in the dose escalation part of the trial. All results were as of a data cutoff date of November 16, 2018.

    In patients with PDGFRA D842V mutant GIST, the overall response rate (ORR) was 88 percent (95% CI: 76-95%) with 9 percent of patients achieving a complete response. AYVAKIT demonstrated durable clinical benefit in this patient population with a 12-month duration of response rate of 70 percent (95% CI: 54-87%), a 12-month progression-free survival (PFS) rate of 81 percent (95% CI: 69-93%) and a 24-month OS rate of 81 percent (95% CI: 67-94%).

    AYVAKIT was generally well-tolerated with most treatment-related adverse events (AEs) reported as Grade 1 or 2. The most common treatment-related AEs were nausea, fatigue, diarrhea, periorbital edema, anemia, decreased appetite, vomiting and memory impairment. Cognitive effects occurred in 40 percent of patients, with the majority of events reported as Grade 1.

    About AYVAKIT (avapritinib)

    AYVAKIT (avapritinib) is a kinase inhibitor approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. AYVAKIT is the first precision therapy approved to treat a genomically defined population of patients with GIST and the only highly active treatment for PDGFRA exon 18 mutant GIST. The FDA granted Breakthrough Therapy Designation to avapritinib for the treatment of unresectable or metastatic GIST harboring the PDGFRA D842V mutation. For more information, visit AYVAKIT.com.

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

    Blueprint Medicines is developing avapritinib globally for the treatment of advanced, smoldering and indolent systemic mastocytosis (SM). The FDA granted Breakthrough Therapy Designation to avapritinib for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.

    Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of avapritinib and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.

    About GIST

    GIST is a sarcoma, or tumor of bone or connective tissue, of the GI tract. Tumors arise from cells in the wall of the GI tract and occur most often in the stomach or small intestine. Most patients are diagnosed between the ages of 50 to 80, and diagnosis is typically triggered by GI bleeding, incidental findings during surgery or imaging and, in rare cases, tumor rupture or GI obstruction.

    About 5 to 6 percent of primary GIST cases are caused by a PDGFRA D842V mutation, the most common PDGFRA exon 18 mutation. Prior to the approval of AYVAKIT, there were no highly effective treatments for PDGFRA D842V mutant GIST. Published data have shown poor outcomes in patients with PDGFRA D842V mutant GIST treated with imatinib and other approved therapies, including a median OS of 15 months, a median PFS of 3 months and an ORR of 0 percent.1

    Important Safety Information

    Intracranial hemorrhage (e.g., subdural hematoma, intracranial hemorrhage, and cerebral hemorrhage) occurred in 1% of 267 patients (0.7% Grade 3 or 4) with GIST and overall in 3% of 335 patients (1.2% Grade 3 or 4) who received AYVAKIT. Overall, 0.9% of patients receiving AYVAKIT required permanent discontinuation for an intracranial hemorrhage. Withhold AYVAKIT and then resume at a reduced dose upon resolution, or permanently discontinue AYVAKIT based on severity.

    In 335 patients receiving AYVAKIT, CNS adverse reactions occurred overall in 58% of patients including cognitive impairment (41%; 3.6% Grade 3 or 4), dizziness (20%; 0.6% Grade 3 or 4), sleep disorders (15%; 0.3% Grade 3 or 4), mood disorders (13%; 1.5% Grade 3 or 4), speech disorders (6%; none Grade 3 or 4), and hallucinations (2.1%; none Grade 3 or 4). Overall, 3.9% of patients required permanent discontinuation of AYVAKIT for a CNS adverse reaction. Depending on severity, withhold AYVAKIT and then resume at the same dose or at a reduced dose upon improvement, or permanently discontinue AYVAKIT.

    AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential and pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective method of contraception during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for two weeks after the final dose. Advise females and males of reproductive potential that AYVAKIT may impair fertility.

    In 204 patients with unresectable or metastatic GIST, the most common adverse reactions (≥ 20%) were edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, hair color changes, increased lacrimation, abdominal pain, constipation, rash and dizziness.

    Avoid coadministration of AYVAKIT with strong and moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong and moderate CYP3A inducers.

    Please click here to see the full Prescribing Information for AYVAKIT.

    About Blueprint Medicines

    Blueprint Medicines is a precision therapy company striving to improve human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational medicines rooted in our leading expertise in protein kinases, which are proven drivers of disease. Our uniquely targeted, scalable approach empowers the rapid design and development of new treatments and increases the likelihood of clinical success. We have one FDA-approved precision therapy and are currently advancing multiple investigational medicines in clinical development, along with a number of research programs. For more information, visit www.BlueprintMedicines.com and follow us on Twitter (@BlueprintMeds) and LinkedIn.

    Reference

    1 Cassier PA, Fumagalli E, Rutkowski P., et al. Outcome of patients with platelet-derived growth factor receptor alpha-mutated gastrointestinal stromal tumors in the tyrosine kinase inhibitor era. Clin Cancer Res. 2012;18(16):4458-4464.

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    SOURCE Blueprint Medicines Corporation

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  4. CAMBRIDGE, Mass., June 6, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ:BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced updated clinical data from part 1 of the PIONEER trial showing robust and consistent clinical activity for avapritinib across multiple qualitative and quantitative measures of cutaneous disease in patients with indolent systemic mastocytosis (SM). Additional data showed avapritinib treatment resulted in deepening improvements in overall disease symptoms, as measured by the Indolent SM Symptom Assessment Form (ISM-SAF) total symptom score (TSS), and was well-tolerated through 24 weeks of follow-up. These data were presented today…

    CAMBRIDGE, Mass., June 6, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ:BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced updated clinical data from part 1 of the PIONEER trial showing robust and consistent clinical activity for avapritinib across multiple qualitative and quantitative measures of cutaneous disease in patients with indolent systemic mastocytosis (SM). Additional data showed avapritinib treatment resulted in deepening improvements in overall disease symptoms, as measured by the Indolent SM Symptom Assessment Form (ISM-SAF) total symptom score (TSS), and was well-tolerated through 24 weeks of follow-up. These data were presented today during the European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress 2020.

    SM is a rare disease driven by the KIT D816V mutation in nearly all patients and characterized by uncontrolled mast cell proliferation and activation. The disorder can lead to debilitating systemic, gastrointestinal and neurocognitive symptoms, including life-threatening anaphylaxis. Additional skin manifestations such as itching, flushing and pigmented skin lesions are common and can significantly impact quality of life. Avapritinib is a potent and highly selective inhibitor of D816V mutant KIT.

    "Patients with indolent systemic mastocytosis often suffer from extensive skin lesions and severe mediator symptoms that can be life-threatening," said Karin Hartmann, M.D., Professor of Medicine and Head of the Division of Allergy at the University of Basel and an investigator on the PIONEER trial. "The updated PIONEER trial data showed that patients treated with avapritinib had clinically meaningful reductions in mediator symptoms, patient-reported quality of life and mast cell burden across multiple measures. With further evidence that these reductions deepen over time, avapritinib has the potential to provide important clinical benefits to patients with this debilitating disease."

    "These promising data reinforce our commitment to rapidly advance development of avapritinib to meet persistent medical needs in indolent systemic mastocytosis, which are poorly addressed by a patchwork of available symptomatic therapies," said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. "Avapritinib was specifically designed to potently target the underlying disease driver of systemic mastocytosis and has the potential to fundamentally change the treatment of this disease."

    The presentation is available on-demand via the EAACI Digital Congress 2020 website at www.eaaci.org/eaaci-congresses/eaaci-2020.

    Highlights from the EAACI Presentation of PIONEER Trial Data

    Previously reported data from part 1 of the PIONEER trial showed that treatment with avapritinib was well-tolerated and resulted in robust and clinically meaningful improvements on measures of mast cell burden, disease symptoms and patient-reported quality of life through 16 weeks. Based on these data, avapritinib 25 mg once daily (QD) was selected as the recommended part 2 dose. Updated data on disease symptoms through 24 weeks and new skin assessment results were reported in the EAACI presentation.

    Updated clinical activity and safety data

    As of a data cutoff date of March 31, 2020, updated data from part 1 of the PIONEER trial showed a deepening of symptom reductions in patients treated with avapritinib through 24 weeks of follow-up. The mean percent change from baseline in ISM-SAF TSS was -35 percent in patients treated with avapritinib 25 mg QD (n=10) compared to -4 percent in patients treated with placebo. In addition, the mean percent change from baseline in ISM-SAF skin domain score was -38 percent for avapritinib 25 mg QD versus +11 percent for placebo.

    The updated data also showed a 60 percent response rate in patients treated with avapritinib 25 mg QD compared to a 0 percent response rate in patients treated with placebo at 24 weeks, with response defined as a 30 percent or greater reduction in ISM-SAF TSS. Based on these data and feedback from the U.S. Food and Drug Administration (FDA), Blueprint Medicines has selected response rate at 24 weeks as the primary endpoint for the registration-enabling part 2 of the PIONEER trial and plans to enroll approximately 200 patients. Blueprint Medicines continues to plan to initiate patient screening in part 2 of the PIONEER trial in June 2020.

    As of a data cutoff date of March 31, 2020, avapritinib 25 mg QD was well-tolerated and safety results were consistent with previously reported data, with no Grade ≥3 adverse events or discontinuations due to adverse events.

    Additional clinical activity data on measures of skin disease

    High resolution skin photographs were taken at baseline and every 12 weeks during treatment for patients with significant cutaneous involvement who consented to photography. To assess changes in skin disease, photographs were assessed by a blinded independent review committee and a computational image analysis algorithm. Images and data as of a cutoff of March 31, 2020 were evaluated by the independent committee.

    Based on skin photography at 24 weeks or the last available assessment, results showed that skin lesions lightened in 71 percent of patients treated with avapritinib (n=17; all doses) compared to 25 percent of patients treated with placebo (n=8), per blinded review by the independent committee. In addition, the median percent change from baseline in most affected surface area was -35 percent for avapritinib (n=18; all doses) compared to -8 percent for placebo (n=8), based on a computational image analysis algorithm.

    Mast cell infiltration in skin lesions was also assessed by lesional skin biopsies obtained at baseline and 12 weeks. The median percent change from baseline in mast cell infiltration was -46 percent for avapritinib (n=18; all doses) compared to +51 percent for placebo (n=7).

    Phase 1 Trial of BLU-263 in Healthy Volunteers

    Blueprint Medicines today announced it has initiated dosing in a Phase 1 trial of BLU-263, an investigational next-generation KIT inhibitor, in healthy volunteers. Following completion of the Phase 1 trial and the analysis of data, the company plans to initiate clinical development of BLU-263 in patients with indolent SM, with the goal of complementing the ongoing avapritinib development program.

    About SM

    SM is a rare disease driven by the KIT D816V mutation. Uncontrolled proliferation and activation of mast cells result in chronic, severe and often unpredictable symptoms for patients across the spectrum of SM. The vast majority of those affected have non-advanced (indolent or smoldering) SM, with debilitating symptoms that lead to a profound, negative impact on quality of life. A minority of patients have advanced SM, which encompasses a group of high-risk SM subtypes including aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia. In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor overall survival.

    Debilitating symptoms associated with SM, including anaphylaxis, maculopapular rash, pruritis, brain fog, fatigue and bone pain, often persist despite treatment with a number of symptomatic therapies. Patients often live in fear of attacks, have limited ability to work or perform daily activities, or isolate themselves to protect against unpredictable triggers. Currently, there are no approved therapies that selectively inhibit D816V mutant KIT.

    About AYVAKIT™ (avapritinib)

    AYVAKIT™ (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. For more information, visit AYVAKIT.com.

    Avapritinib is not approved for the treatment of any other indication, including SM, in the U.S. by the FDA or for any indication in any other jurisdiction by any other health authority.

    Blueprint Medicines is developing avapritinib globally for the treatment of advanced, smoldering and indolent SM. The FDA granted Breakthrough Therapy Designation to avapritinib for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.

    Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of avapritinib and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.

    About the Phase 2 PIONEER Trial

    PIONEER is a randomized, double-blind, placebo-controlled, registration-enabling trial evaluating avapritinib in patients with indolent SM. The trial includes three parts: dose-finding part 1, registration-enabling part 2 and long-term treatment part 3. All patients who complete parts 1 or 2 will have an opportunity to continue to receive treatment with avapritinib in part 3. Key trial endpoints include the change in patient-reported disease symptoms as measured by the ISM-SAF TSS, patient-reported quality of life, quantitative measures of mast cell burden and safety. Part 1 has completed patient enrollment. Blueprint Medicines plans to initiate patient screening for part 2 in June 2020 at sites in the United States, Canada and European Union.

    Patients and healthcare providers with questions about opportunities to participate in the PIONEER trial can contact the Blueprint Medicines study director at or 1-617-714-6707. Additional details are also available at www.blueprintclinicaltrials.com or www.clinicaltrials.gov.

    About Blueprint Medicines

    Blueprint Medicines is a precision therapy company striving to improve human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational medicines rooted in our leading expertise in protein kinases, which are proven drivers of disease. Our uniquely targeted, scalable approach empowers the rapid design and development of new treatments and increases the likelihood of clinical success. We have one FDA-approved precision therapy and are currently advancing multiple investigational medicines in clinical development, along with a number of research programs. For more information, visit www.BlueprintMedicines.com 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, statements regarding plans and timelines for the development of its drug candidates, including the timing, design, implementation, enrollment, plans and announcement of results regarding Blueprint Medicines' ongoing and planned clinical trials for avapritinib and BLU-263; plans, timelines and expectations for initiating patient screening in part 2 of the PIONEER trial; expectations regarding the potential benefits of avapritinib and BLU-263 in treating patients with SM and mast cell disorders; 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 plan in establishing a commercial infrastructure, and successfully launching, marketing and selling current or future approved products; the delay of any current or planned clinical trials or the development of Blueprint Medicines' drug candidates or licensed product candidate; 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 develop and commercialize companion diagnostic tests for its current and future drug candidates; and the success of Blueprint Medicines' current and future collaborations 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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    SOURCE Blueprint Medicines Corporation

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  5. CAMBRIDGE, Mass., June 3, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ:BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced that, effective on June 1, 2020, the Compensation Committee of Blueprint Medicines' Board of Directors granted non-qualified stock options to purchase an aggregate of 49,493 shares of its common stock and an aggregate of 24,745 restricted stock units (RSUs) to four new employees under Blueprint Medicines' 2020 Inducement Plan.

    The 2020 Inducement Plan is used exclusively for the grant of equity awards to individuals who were not previously an employee or non-employee director of Blueprint Medicines, as an inducement material to such individual's entering into employment with Blueprint Medicines, pursuant to Rule 5635(c)(4) of the NASDAQ Listing Rules.

    The options have an exercise price of $66.24 per share, which is equal to the closing price of Blueprint Medicines' common stock on June 1, 2020. Each option will vest as to 25% of the shares underlying such option on the first anniversary of the grant date and as to an additional 1/48th of the shares underlying the option monthly thereafter, in each case, subject to each such employee's continued employment on each vesting date. Each RSU will vest as to 25% of the shares underlying the RSU award on the first anniversary of the grant date and as to an additional 25% of the shares underlying the RSU award annually thereafter, subject to each such employee's continued employment on each vesting date. The options and RSUs are subject to the terms and conditions of Blueprint Medicines' 2020 Inducement Plan, and the terms and conditions of the stock option and RSU agreement covering the grant.

    About Blueprint Medicines

    Blueprint Medicines is a precision therapy company striving to improve human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational medicines rooted in our leading expertise in protein kinases, which are proven drivers of disease. Our uniquely targeted, scalable approach empowers the rapid design and development of new treatments and increases the likelihood of clinical success. We have one FDA-approved precision therapy and are currently advancing multiple investigational medicines in clinical development, along with a number of research programs. For more information, visit www.BlueprintMedicines.com and follow us on Twitter (@BlueprintMeds) and LinkedIn.

     

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    SOURCE Blueprint Medicines Corporation

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