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Item 7.01 Regulation FD.
On June 9, 2022, Blueprint Medicines Corporation (the “Company”) is presenting updates on the AYVAKIT/AYVAKYT (avapritinib) development program in systemic mastocytosis and other business updates at the 2022 Jefferies Healthcare Conference. A copy of the presentation is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The information in Item 7.01 of this Current Report on Form 8-K, including Exhibit 99.1 attached hereto, is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 8.01 Other Events.
On June 9, 2022, the Company issued a press release announcing data from the AYVAKIT/AYVAKYT (avapritinib) development program from various presentations at the European Hematology Association 2022 Congress. A copy of the press release is filed herewith as Exhibit 99.2 to this Current Report on Form 8-K and incorporated herein by reference.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
| Exhibit No. | Description | |
| 99.1 | Corporate slide presentation of Blueprint Medicines Corporation dated June 9, 2022 | |
| 99.2 | Press release issued by Blueprint Medicines Corporation on June 9, 2022 | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document and incorporated as Exhibit 101) |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| BLUEPRINT MEDICINES CORPORATION | ||
| Date: June 9, 2022 | By: |
/s/ Kathryn Haviland |
| Kathryn Haviland | ||
| Chief Executive Officer | ||
Exhibit 99.1
| JEFFERIES HEALTHCARE CONFERENCE JUNE 9, 2022 Kristine G. Systemic mastocytosis patient Pioneering the Science of Time Not for promotional use. |
| Forward-looking statements 2 Not for promotional use. This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding plans, timelines and expectations for interactions with the FDA and other regulatory authorities; statements regarding plans and expectations for Blueprint Medicines' current or future approved drugs and drug candidates; the potential benefits of any of Blueprint Medicines' current or 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 report 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 report, 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 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 either as monotherapies or in combination with other agents or may impact the anticipated timing of data or regulatory submissions; the timing of the initiation of clinical trials and trial cohorts at clinical trial sites and patient enrollment rates; 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; Blueprint Medicines' ability to successfully expand its operations, research platform and portfolio of therapeutic candidates, and the timing and costs thereof; Blueprint Medicines' ability to realize the anticipated benefits of its executive leadership transition plan; 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 report 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. Blueprint Medicines, AYVAKIT, AYVAKYT, GAVRETO and associated logos are trademarks of Blueprint Medicines Corporation. |
| Blueprint Medicines is a global leader in precision therapy AYVAKIT is approved for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and adult patients with advanced SM, including aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia. GAVRETO is approved for the treatment of adult patients with RET-fusion positive NSCLC, adult and pediatric patients with advanced or metastatic RET-mutant medullary thyroid cancer who require systemic therapy and adult, and pediatric patients with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory. FDA, U.S. Food and Drug Administration; GIST, gastrointestinal stromal tumor; NSCLC, non-small cell lung cancer; SM, systemic mastocytosis. 3 internally discovered medicines FDA & EMA approved across indications within years, and with breakthrough therapy designations OUR FIRST DECADE OF ACHIEVEMENT Not for promotional use. Ongoing global collaboration with Roche and Genentech for the development and commercialization of GAVRETO |
| Blueprint is uniquely positioned with a diversity of significant growth drivers Ongoing global collaboration with Roche and Genentech for the development and commercialization of GAVRETO. FDA, U.S. Food and Drug Administration; EC, European Commission; PDGFRA, platelet-derived growth factor receptor alpha; GIST, gastrointestinal stromal tumor; SM, systemic mastocytosis; sNDA, supplemental new drug application; R&D, research and development; POC, proof-of-concept; EGFR, epidermal growth factor receptor; NSCLC, non-small cell lung cancer; CDK2, cyclin-dependent kinase 2 4 • Topline results expected late summer '22 • sNDA and launch in non-advanced SM, if approved Not for promotional use. • Global commercial expansion • $115 - $130M in AYVAKIT product revenue in '22 • BLU-222 in breast cancer and other CDK2-vulnerable tumors • First clinical data expected 1H '23 • R&D day 2H 2022 • Two new development candidates by end of 2022 GLOBAL COMMERCIAL EXECUTION CLINICAL STAGE GROWTH LEADING PRECISION MEDICINE DISCOVERY PLATFORM • BLU-945+osi early clinical data 2H '22 • BLU-701 first clinical data expected 2H '22 • BLU-451 first clinical data expected 1H '23 |
| Global Commercial Execution Cyndi N. Systemic mastocytosis patient Not for promotional use. |
| We are establishing the standard of care for advanced SM 6 Not for promotional use. 1. Reported data represent estimations. Analysis based on US claims data AYVAKIT COMMERCIAL GROWTH NEW PATIENT STARTS DURATION OF THERAPY ~65 new accounts activated in Q1 2022 ~70% share of new AdvSM patient starts ~18 month trending average duration of therapy, showing increasing trend ~40% growth in AdvSM market treated with TKIs / cytoreductive agents since launch |
| Retrospective analysis showed a longer OS among AYVAKIT patients A multi-center, global, observational, retrospective chart review study was conducted at 6 study sites (4 European, 2 US) to identify and collect data from AdvSM patients who received BAT. SM-AHN patients were identified using inclusion/exclusion criteria similar to the EXPLORER and PATHFINDER trials. The follow-up times for the midostaurin, cladribine, and BAT cohorts were truncated to match the maximum follow-up time of the avaprtinib cohort. 1. Reiter et al. Overall Survival in Patients with Systemic Mastocytosis with Associated Hematologic Neoplasm Treated with Avapritinib Versus Best Available Therapy. Presented at EHA 2022. Abstract #P1013. 2. Reiter et al. Overall Survival in Patients with Advanced Systemic Receiving Avapritinib Versus Midostaurin or Cladribine. Presented at EHA 2022. Abstract #P1014 BAT, best available therapy; KM, Kaplan-Meier; OS, overall survival; AdvSM, advanced systemic mastocytosis; AVA, AYVAKIT; MIDO, midostaurin; CLAD, cladribine; SM-AHN, systemic mastocytosis with associated hematologic neoplasm 7 OS AMONG PATIENTS WITH SM-AHN TREATED WITH AYVAKIT VS. BAT1 Not for promotional use. OS OF PATIENTS WITH ADVSM TREATED WITH AYVAKIT VS. MIDOSTAURIN OR CLADRIBINE2 46.9 mos (95%CI: 44.9,NE) 18.0 mos (95%CI: 13.0, 26.8) NR (95%CI: 46.9,NE) 23.4 mos (95%CI: 14.8, 40.6) 28.6 mos (95%CI: 18.2, 44.6) |
| Clinical Stage Growth Rob T. Advanced cancer patient Not for promotional use. |
| Non-advanced SM patients have high medical need despite available therapies1 95% of SM cases driven by the KIT D816V mutation SYSTEMIC MASTOCYTOSIS SYMPTOMS2 Brain fog Pruritis, flushing and pigmented skin lesions Life-threatening organ infiltration and damage Unpredictable, life-threatening anaphylaxis GI upset with vomiting, diarrhea and nausea Debilitating fatigue 1. Jennings SV et al. Immunol Allergy Clin North Am. 2018;38(3):505-525. 2. Sperr WR, et al. Lancet Haematol, 2019. 3. Data on file. Adelphi Observational Study. July 2018. Data cutoff March 2018. 4. van Anrooij B et al. Allergy. 2016 Nov;71(11):1585-1593. 9 Not for promotional use. 75% of patients have taken 4+ classes of therapies to address significant symptom burden3 >80% of patients report limitations in their work and/or daily activities3 83% of patients are frustrated at lack of treatment options that do not address the underlying driver of disease4 |
| PIONEER Part 2 primary endpoint to be updated to mean change in TSS 1. PIONEER Part 1. Based upon a data cutoff of March 12, 2020. TSS, total symptom score; FDA, Food and Drug Administration; ISM-SAF, Indolent Systemic Mastocytosis Symptom Assessment Form; SAP, statistical analysis plan; EMA, European Medicines Agency 10 What is the same? • The PIONEER study is powered for key primary and secondary analyses of clinical benefit based on the ISM-SAF TSS o Mean change in TSS o Proportion of patients with a ≥30% reduction in TSS What is changing? • In recent discussions with FDA to finalize the SAP in advance of database lock we have aligned on: o Mean change in TSS to be the primary endpoint - previously a key secondary endpoint o Proportion of patients with a ≥30% reduction in TSS will be a key secondary endpoint – previously the primary endpoint Why? • Characterizes benefit of avapritinib across a wider range of patients • Harmonizes with the EMA Not for promotional use. ALIGNMENT WITH FDA ON PRIMARY EFFICACY ANALYSIS FOR PIONEER PART 2 p=0.0371 PIONEER PART 1 MEAN CHANGE IN TSS AT 24 WEEKS1 AVAPRITINIB 25 MG VS. PLACEBO |
| PIONEER Part 1 showed statistically significant difference in mean change in TSS between avapritinib and placebo 1. Hartmann K. et al. Avapritinib reduces cutaneous symptoms and mast cell burden in patients with indolent systemic mastocytosis in the PIONEER study. Presented at the European Academy of Allergy Clinical Immunology Annual Meeting. June 2020. Based upon a data cutoff of March 31, 2020. 2. PIONEER Part 1. Based upon a data cutoff of March 31, 2020. TSS, total symptom score; SE, standard error 11 MEAN CHANGE IN TSS AT 24 WEEKS2 AVAPRITINIB 25 MG VS. PLACEBO (+/- SE) MEAN CHANGE IN TSS BY SYMPTOM AT 24 WEEKS1 AVAPRITINIB 25 MG VS. PLACEBO PRESENTED AT EAACI 2020 Not for promotional use. Difference is statistically significant (p=0.0371) at 24 weeks |
| Key secondary endpoints are important to fully characterize the impact of avapritinib on patients with non-advanced SM 12 Not for promotional use. 1. Hartmann K. et al. Avapritinib reduces cutaneous symptoms and mast cell burden in patients with indolent systemic mastocytosis in the PIONEER study. Presented at the European Academy of Allergy Clinical Immunology Annual Meeting. June 2020. Based upon a data cutoff of March 31, 2020. *24 weeks or last assessment before, if 24 weeks not available. TSS, total symptom score PIONEER PART 1 ≥ 30% REDUCTION IN TSS AT 24 WEEKS1 AVAPRITINIB 25 MG VS. PLACEBO PIONEER PART 1 ≥ 50% REDUCTION IN TRYPTASE AT 24 WEEKS 1* AVAPRITINIB 25 MG VS. PLACEBO |
| PIONEER Part 2 topline on track for late summer 2022 TSS, total symptom score; QD, once daily; BSC, best supportive care; ISM, indolent systemic mastocytosis; QoL, quality of life; MC-QoL, Mastocytosis Quality of Life Questionnaire; PGIS, Patient's Global Impression of Symptom Severity; PGIS, Patient's Global Impression of Change; SF-12, Short Form Health Survey 13 Not for promotional use. Primary endpoint Avapritinib 25 mg QD + BSC Placebo QD + BSC Mean change in TSS at 24 weeks Avapritinib 25 mg QD + BSC Rollover Randomize 2:1 Part 2 • Age ≥18 years • ISM confirmed by central pathology review • No restriction on prior therapy • Moderate-to-severe symptoms Eligibility • Proportion of patients with reduction in TSS • Reduction in measures of mast cell burden • Change in measures of QoL Key secondary endpoints PLANS TO SUBMIT sNDA BY END OF 2022 |
| Our portfolio of EGFR therapies is purpose-built to address medical needs CS, C797S resistance mutation; Ex20in, activating exon 20 insertion mutations; LR, L858R activating mutation; TM, T790M resistance mutation. 14 Not for promotional use. Effectively block the EGFR pathway Establish 2L+ SOC with combinations that treat on- and off-target resistance TREATMENT GOALS • Potent EGFR mutation coverage: o LR and LR/CS o TM and TM/CS regardless of activating mutation o Potential for broader coverage at higher exposures • Highly selective over wild-type EGFR • Potent EGFR mutation coverage: o Ex19del and LR o CS regardless of activating mutation • Highly CNS penetrant BLU-451 • Potent inhibitor of all common Ex20ins and other uncommon activation mutations • Highly selective over wild-type EGFR • CNS penetrant BLU-701 BLU-945 BLUEPRINT MEDICINES EGFR PORTFOLIO |
| BLU-945 potency and selectivity enable wide therapeutic index and broad EGFR coverage, with promising early clinical monotherapy data 15 aOne patient had two different DNA mutations in C797S. Note: reductions in individual variant allele fractions as shown; therefore, patients with multiple mutations may be represented on both plots. All T790M and C797S allele fractions with available baseline and C1D15 data are shown. Increases of greater than 100% were truncated at 100 bPatients with measurable target lesions at baseline with post-baseline scans (investigator assessed). An unconfirmed PR is a PR in which tumor reduction ≥30% has occurred but has not yet been confirmed via a subsequent scan. ┼ Most common AEs by preferred term in ≥10% of patients included nausea, headache, fatigue, cough, dyspnea, vomiting, hyponatremia, dry mouth, and anemia.ctDNA, circulating tumor DNA; C, cycle; D, day; F1LCDx, FoundationOne Liquid CDx assay; QD, once daily CR, complete remission; PD, progressive disease; PR, partial remission; SD, stable disease; EOT, end of treatment. As of the data cut-off (March 9, 2022), 33 patients have been treated with BLU-945 at 25–400 mg once daily (QD) in the first 5 cohorts. Not for promotional use. GENERALLY WELL-TOLERATED ┼, WITH NO SIGNIFICANT ADVERSE EVENTS ASSOCIATED W ITH W ILD-TYPE EGFRINHIBITION In the 400 mg cohort, all detectable T790M and C797S alleles showed reduction, including three that fell below the limit of detection (clearance) Unconfirmed PR reported in patient with ex19del/T790M/C797S treated at 400 mg QD DOSE-DEPENDENT REDUCTIONS IN ctDNA… …AND ANTI-TUMOR ACTIVITY, WITH TUMOR SHRINKAGE REPORTED AT DOSES ≥200 MG QD |
| Phase 1/2 trials to rapidly generate data in broad populations, informing development and registration strategies PLANNED INITIATION OF PHASE 1/2 SYMPHONY / HARMONY TRIAL COHORTS 1L 2L+ 2023 701 + ADC All comer 945 + osi LR 701 + chemo All comer 701 + osi All comer Q2 2022 945 + osi LR + CS +/- TM 945 LR + CS +/- TM 2H 2022 945 + ADC LR 701 Ex19del/LR + CS 945 + 701 Ex19del/LR + CS 945 + chemo LR 701 + osi Ex19del/LR + CS Biomarker- selected Broad populations 16 945 + 701 All comer Not for promotional use. |
| Opportunity to influence the treatment paradigm for more than 100K patients across multiple CDK2-vulnerable cancers1 17 CDK-CYCLIN COMPLEXES REGULATE THE CELL CYCLE ABERRANT CYCLIN E (CCNE1) DRIVES CELL PROLIFERATION CDK4/6 Cyclin D CDK2 Cyclin E CDK2 Cyclin A CDK1 Cyclin A/B M G1 S G2 CELL DIVISION DNA SYNTHESIS Not for promotional use. 1. Approximate patient numbers covering major markets – US, EU4, UK, and Japan. 2. Data from company reports. 3. CCNE1 amplification frequency represented as percentage of total patient samples. Data from the National Cancer Institute's The Cancer Genome Atlas Program (www.cancer.gov/tcga).. CDK, cyclin dependent kinas; ER+/HER2-, estrogen receptor-positive, HER2-negativ $0.7 $2.1 $3.2 $4.6 $6.0 $7.0 $7.7 $0.0 $1.0 $2.0 $3.0 $4.0 $5.0 $6.0 $7.0 $8.0 2015 2016 2017 2018 2019 2020 2021 CDK4/6 INHIBITOR GLOBAL SALES ($, BILLIONS) 2 ER+/HER2- BREAST CANCER 0% 10% 20% 30% 40% Breast Lung Uterine Esophageal Stomach Ovarian Uterine FREQUENCY OF CCNE1 AMPLIFICATION 3 CCNE1-AMPLIFIED TUMORS BLU-222 WILL ADDRESS THE SPECTRUM OF OPPORTUNITY IN CDK2 -VULNERABLE CANCERS |
| BLU-222 is advancing toward clinical proof-of-concept CCNE1, cyclin E; CDK4/6i, CDK4/6 inhibitor; ER, estrogen receptor. 18 Multiple dose cohorts* RP2D Combo with ER antagonist – ER+/HER2- breast Combo with CDK4/6i + ER antagonist – ER+/HER2- breast Monotherapy – CCNE1 tumors PHASE 2 EXPANSION (PLANNED) PHASE 1 DOSE ESCALATION (NOW ENROLLING) Combo with chemotherapy – CCNE1 tumors PHASE 1/2 VELA TRIAL OF BLU-222 INITIATED IN Q1 2022 AND FIRST PATIENT DOSED • Safety • Preliminary clinical activity • Patient selection strategy PHASE 1/2 TRIAL OF BLU-222 IN CDK2 VULNERABLE CANCERS Not for promotional use. *Includes monotherapy and combination regimens Monotherapy – multiple other CCNE1 tumors (basket cohort) |
| Anticipated clinical data milestones over the next year POC, proof-of-concept 19 Topline results, with additional detail presented at a medical conference at a later date Late summer 2022 2H 2022 1H 2023 Clinical data for BLU-222 Clinical data for BLU-451 Early clinical data for BLU-945+osimertinib, BLU-701 monotherapy Part 1 data for BLU-263 Not for promotional use. |
| Leading Precision Medicines Drug Discovery Platform Diane L. Lung cancer patient Not for promotional use. |
| Research platform expansion to drive innovation & expanded productivity 21 KINASE PLATFORM KINASE PLATFORM NEW PLATFORMS INTEGRATED PLATFORM CURRENT CAPABILITIES TARGETED PROTEIN DEGRADATION UNPARALLELED PRODUCTIVITY COMPLEMENTARY & SCALABLE NEW TARGET CLASSES LIBRARY & NEW TARGET DISCOVERY EXPANSION Consolidate precision medicine platforms with combination of internal and external innovation YESTERDAY TODAY FUTURE Not for promotional use. EXPANSION AIMS TO DOUBLE THE HISTORIC OUTPUT OF OUR DISCOVERY ENGINE BY 2025 |
| Business development plays a key role in our company's value creation and long-term portfolio growth 22 Global Strategic Partnerships Access complementary expertise to maximize program value and global impact on patients Geographic Expansion Pioneering early development partnerships in China; expanding to additional key markets through regional distributors Buy-Side Transactions Tap into external innovations to complement organic portfolio build Program Partnerships Tap into external resources & expertise to enhance internal programs Over $1.1B earned to-date inclusive of upfront, milestones and royalties * Fibrodysplasia ossificans progressiva Not for promotional use. 2015: Discovery collaboration on FOP* 2016: Global I/O research collaboration 2019: WW out-license of BLU-782 for FOP* 2020: Global collaboration of GAVRETO® 2018: Greater China partnering on 3 clinical programs 2021: Greater China partnering on 2 preclinical programs 2022: Acquisition for its EGFR Exon20 program 2022: Research collaboration on targeted protein degradation 2022: Supply agreement for osimertinib 2022: Translational research collaboration on CDK2 2021: Distribution agreement for AYVAKITTM in Israel 2022: Distribution agreement for AYVAKITTM in 14 Central Eastern European countries |
| Strong financial position with total revenues currently estimated for 2022 between $180 and $200 million 1. Includes stock-based compensation expense of $10.0M and $8.9M in the three months ended 3/31/22 and 3/31/21, respectively. 2. Includes stock-based compensation expense of $13.4M and $11.7M in the three months ended 3/31/22 and 3/31/21, respectively. 23 Statement of Operations (unaudited) Three Months Ended 3/31/2022 Three Months Ended 3/31/2021 Total revenue $62.7M $21.6M Net product sales Collaboration revenue $23.8M $38.9M $9.0M $12.6M Cost of sales $5.1M $0.1M Collaboration loss sharing $3.3M -- Research & development expense1 $103.1M $79.7M Selling, general & admin expense2 $57.1M $42.0M Net loss $(106.0)M $(99.7)M Balance Sheet (unaudited) 3/31/2022 12/31/2021 Cash, cash equivalents and investments $893.4M $1,034.6M Not for promotional use. ON TRACK TO ACHIEVE $115 TO $130 MILLION IN AYVAKIT NET PRODUCT REVENUES IN 2022 |
| Blueprint is uniquely positioned with a diversity of significant growth drivers Ongoing global collaboration with Roche and Genentech for the development and commercialization of GAVRETO. FDA, U.S. Food and Drug Administration; EC, European Commission; PDGFRA, platelet-derived growth factor receptor alpha; GIST, gastrointestinal stromal tumor; SM, systemic mastocytosis; sNDA, supplemental new drug application; R&D, research and development; POC, proof-of-concept; EGFR, epidermal growth factor receptor; NSCLC, non-small cell lung cancer; CDK2, cyclin-dependent kinase 2 24 • Topline results expected late summer '22 • sNDA and launch in non-advanced SM, if approved Not for promotional use. • Global commercial expansion • $115 - $130M in AYVAKIT product revenue in '22 • BLU-222 in breast cancer and other CDK2-vulnerable tumors • First clinical data expected 1H '23 • R&D day 2H 2022 • Two new development candidates by end of 2022 GLOBAL COMMERCIAL EXECUTION CLINICAL STAGE GROWTH LEADING PRECISION MEDICINE DISCOVERY PLATFORM • BLU-945+osi early clinical data 2H '22 • BLU-701 first clinical data expected 2H '22 • BLU-451 first clinical data expected 1H '23 |
Exhibit 99.2
Blueprint Medicines Reports Continued Progress Toward Goal of Transforming Treatment of Systemic Mastocytosis
-- New analyses showing AYVAKIT® (avapritinib) significantly improved overall survival in advanced SM, when indirectly compared to real-world data for prior standard therapies, to be presented at EHA 2022 Congress --
-- Primary endpoint of PIONEER trial of AYVAKIT in non-advanced SM to be updated to mean change in total symptom score, previously a key secondary endpoint, based on U.S. FDA recommendation --
-- On track to report top-line registrational data from PIONEER trial in late summer 2022 --
-- Kate Haviland, Chief Executive Officer, to present at Jefferies Healthcare Conference today at 1:30 p.m. EDT --
CAMBRIDGE, Mass., June 9, 2022 -- Blueprint Medicines Corporation (NASDAQ: BPMC) today announced updates on the AYVAKIT®/AYVAKYT® (avapritinib) development program in systemic mastocytosis (SM):
| • | New analyses, which will be presented this week at the European Hematology Association (EHA) 2022 Congress, add to the growing body of clinical evidence supporting AYVAKIT as the standard of care for patients with advanced SM. Findings showed AYVAKIT improved overall survival (OS), as well as other clinical outcomes, in patients with advanced SM, when indirectly compared to real-world data for prior best available therapies. Based on these analyses, patients treated with AYVAKIT had a 41 percent reduction in the risk of death compared to patients treated with midostaurin and a 68 percent reduction in the risk of death compared to patients treated with cladribine. In total, Blueprint Medicines is supporting the presentation of seven abstracts at the EHA 2022 Congress, highlighting the company’s leadership in SM. |
| • | In addition, Blueprint Medicines plans to update the primary endpoint of the registrational PIONEER trial of AYVAKIT in patients with non-advanced SM, based on a written recommendation from the U.S. Food and Drug Administration (FDA) on statistical considerations ahead of the planned database lock. The mean absolute change in total symptom score (TSS), previously a key secondary endpoint, will be the primary endpoint and the proportion of patients with a 30 percent or greater decrease in TSS, previously the primary endpoint, will be a key secondary endpoint. Both analyses were previously defined as key endpoints that the PIONEER trial was powered to assess. In addition, both endpoints are based on the Indolent SM Symptom Assessment Form, a patient-reported outcomes tool that has been developed and validated in collaboration with the SM community and global regulatory authorities. Blueprint Medicines continues to plan to report top-line data from the PIONEER trial in late summer 2022 and submit a supplemental new drug application to the FDA for AYVAKIT for non-advanced SM by the end of 2022. |
“Based on the recommendation of the FDA, we have re-ordered pre-planned efficacy analyses in Part 2 of the PIONEER trial, elevating mean change in total symptom score, which characterizes clinical benefit across all patients,” said Becker Hewes, M.D., Chief Medical Officer of Blueprint Medicines. “Consistent with Part 1 data, we believe the mean change in total symptom score, together with other measures of improvement in clinical outcomes, quality of life and mast cell burden, will paint a compelling picture of AYVAKIT clinical benefit, including its ability to modify the disease biology and provide meaningful relief to patients living with debilitating symptoms of non-advanced SM.”
Highlights from AYVAKIT Presentations at the EHA 2022 Congress
Three presentations at the EHA 2022 Congress will highlight results from a study (NCT04695431) indirectly comparing clinical outcomes in advanced SM patients receiving AYVAKIT in the registrational EXPLORER and PATHFINDER trials, versus patients treated with best available therapy in real-world clinical practice. Results showed that AYVAKIT improved clinical outcomes when retrospectively compared to best available therapies, including the two other most common treatments (midostaurin, cladribine) identified in the real-world study cohort.
“These rigorous, retrospective analyses highlight the prolonged survival, extended duration of treatment and observed reduction in mast cell burden shown by avapritinib in patients with advanced systemic mastocytosis,” said Prof. Dr. Andreas Reiter, M.D., University Medical Centre, Heidelberg University, Mannheim, Germany. “Notably, in an indirect comparative analysis of patients with SM and an associated hematologic neoplasm, those treated in avapritinib clinical trials had a significantly lower risk of death than those receiving prior best available therapy in the real-world setting. These results are highly meaningful for this patient population, which has a significant disease burden and limited treatment options.”
For this study, patients treated in real-world clinical practice were identified in a retrospective medical chart review at six centers with similar patient eligibility criteria as EXPLORER and PATHFINDER. Retrospective data were collected and analyzed using methods to balance key baseline variables; however, the study may have limitations due to inherent differences between data collected from prospective trials and real-world experience. No prospective, randomized, controlled head-to-head studies have been conducted comparing AYVAKIT to other therapies in patients with advanced SM. EXPLORER and PATHFINDER results were reported as of an April 20, 2021 cutoff date.
Overall Survival Data in Advanced SM
| AYVAKIT1 | Midostaurin | Cladribine | |
| Number of patients | 176 | 94 | 44 |
| Median in months (95% CI) | NR (46.9, NE) | 28.6 (18.2, 44.6) | 23.4 (14.8, 40.6) |
| Hazard ratio (95% CI)2 | --- | HR: 0.59 (0.36, 0.97) | HR: 0.32 (0.15, 0.67) |
| P-value2 | --- | p<0.001 | p=0.003 |
1. Pooled data from EXPLORER and PATHFINDER clinical trials of AYVAKIT in advanced SM (all doses).
2. Comparative analyses used inverse-probability-of-treatment-weighting to balance differences in key baseline covariates; HR<1 favors AYVAKIT.
Additional weighted, indirect comparison analyses showed:
| • | AYVAKIT (all doses) reduced the risk of death by 58 percent compared to best available therapy (p<0.001) in patients with SM with an associated hematological neoplasm (SM-AHN). |
| • | AYVAKIT (≤200 mg once-daily dose) reduced the risk of treatment discontinuation by 64 percent compared to best available therapy (p<0.001) in advanced SM patients. |
| • | The maximum percent reduction in serum tryptase levels for AYVAKIT (≤200 mg once-daily dose) was 85 percent, compared to 9 percent for best available therapy (p<0.001), in advanced SM patients. |
Regulatory approvals in the U.S. and EU were based on results from the EXPLORER and PATHFINDER trials. In the U.S., AYVAKIT is indicated for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM-AHN and mast cell leukemia (MCL). AYVAKIT is not recommended for the treatment of patients with advanced SM with low platelet counts (less than 50,000/µL). Warnings and precautions include intracranial hemorrhage, cognitive effects and embryo-fetal toxicity. The most common adverse reactions were edema, diarrhea, nausea and fatigue/asthenia.
Further details from these analyses, along with additional SM data, will be reported in multiple presentations at the EHA 2022 Congress:
| • | Overall survival in patients with advanced systemic mastocytosis receiving avapritinib versus midostaurin or cladribine (Abstract P1014) |
| • | Overall survival in patients with systemic mastocytosis with associated hematologic neoplasm treated with avapritinib versus best available therapy (Abstract P1013) |
| • | Duration of treatment and reduction in serum tryptase levels in patients with advanced systemic mastocytosis treated with avapritinib versus best available therapy (Abstract P1015) |
| • | Responses to avapritinib in patients with advanced systemic mastocytosis: histopathologic analyses from EXPLORER and PATHFINDER clinical studies (Abstract P1027) |
| • | Clinicopathologic and molecular correlates of organ damage across the spectrum of advanced systemic mastocytosis (Abstract P1038) |
| • | Utility of KIT p.D816 in myeloid neoplasm without documented systemic mastocytosis to detect hidden mast cells in bone marrow (Abstract P996) |
| • | HARBOR: A phase 2/3 study of BLU-263 in patients with indolent systemic mastocytosis and monoclonal mast cell activation syndrome (Trial-in-progress abstract P1017) |
Tomorrow, June 10, copies of the posters will be available on the EHA congress website and in the “Science—Publications and Presentations” section of Blueprint Medicines’ website.
Upcoming Investor Conference
Kate Haviland, Chief Executive Officer at Blueprint Medicines, will present a company overview at the Jefferies Healthcare Conference today at 1:30 p.m. EDT. A live webcast of the presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at http://ir.blueprintmedicines.com. A replay of the webcast will be archived on Blueprint Medicines’ website for 30 days following the presentation.
About AYVAKIT (avapritinib)
AYVAKIT (avapritinib) is a kinase inhibitor approved by the FDA for the treatment of adults with Advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and 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. Under the brand name AYVAKYT (avapritinib), this medicine is approved by the European Commission for the treatment of adults with ASM, SM-AHN or MCL, after at least one systemic therapy, as well as adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.
AYVAKIT/AYVAKYT is not approved for the treatment of any other indication in Europe and the U.S.
Blueprint Medicines is developing AYVAKIT globally for the treatment of advanced and non-advanced SM. The FDA granted breakthrough therapy designation to AYVAKIT for the treatment of moderate to severe indolent SM. The European Commission granted orphan medicinal product designation for AYVAKYT for the treatment of GIST and mastocytosis.
To learn about ongoing or planned clinical trials, contact Blueprint Medicines in the U.S. at medinfo@blueprintmedicines.com or +1 888-258-7768, or in Europe at medinfoeurope@blueprintmedicines.com or +31 85 064 4001. Additional information is available at blueprintclinicaltrials.com and clinicaltrials.gov.
Please click here to see the full U.S. Prescribing Information for AYVAKIT, and click here to see the European Summary of Product Characteristics for AYVAKYT.
Important Safety Information
Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. Overall, ICH (eg, subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT. In Advanced SM patients who received AYVAKIT at 200 mg daily, ICH occurred in 2 of 75 patients (2.7%) who had platelet counts ≥50 x 109/L prior to initiation of therapy and in 3 of 80 patients (3.8%) regardless of platelet counts. Monitor patients closely for risk of ICH including those with thrombocytopenia, vascular aneurysm or a history of ICH or cerebrovascular accident within the prior year. Permanently discontinue AYVAKIT if ICH of any grade occurs. A platelet count must be performed prior to initiating therapy. AYVAKIT is not recommended in Advanced SM patients with platelet counts <50 x 109/L. Following treatment initiation, platelet counts must be performed every 2 weeks for the first 8 weeks. After 8 weeks of treatment, monitor platelet counts every 2 weeks or as clinically indicated based on platelet counts. Manage platelet counts of <50 x 109/L by treatment interruption or dose reduction.
Cognitive adverse reactions can occur in patients receiving AYVAKIT. Cognitive adverse reactions occurred in 39% of 749 patients and in 28% of 148 SM patients (3% were Grade >3). Memory impairment occurred in 16% of patients; all events were Grade 1 or 2. Cognitive disorder occurred in 10% of patients; <1% of these events were Grade 3. Confusional state occurred in 6% of patients; <1% of these events were Grade 3. Other events occurred in <2% of patients. Depending on the severity, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue.
AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise 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 2 weeks after the final dose.
The most common adverse reactions (≥20%) at all doses were edema, diarrhea, nausea, and fatigue/asthenia.
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.
To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please click here to see the full Prescribing Information for AYVAKIT.
About Systemic Mastocytosis
Systemic mastocytosis (SM) is a rare disease primarily 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 ASM, SM-AHN and MCL. In addition to mast cell activation symptoms, advanced SM is associated with organ damage due to mast cell infiltration and poor survival. Across advanced SM subtypes, the median OS is approximately 3.5 years in ASM, approximately two years in SM-AHN and less than six months in MCL.i In Europe, there are about 40,000 patients with SM, and advanced SM represents about 5 to 10 percent of this patient population.ii
Debilitating symptoms, including anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue and bone pain, often persist across all forms of SM despite treatment with a number of symptomatic therapies. Patients often live in fear of severe, unexpected symptoms, have limited ability to work or perform daily activities, and isolate themselves to protect against unpredictable triggers. Historically, there had been no approved therapies for the treatment of SM that selectively inhibit D816V mutant KIT.iii,iv
About Blueprint Medicines
Blueprint Medicines is a global precision therapy company that invents life-changing therapies for people with cancer and blood disorders. Applying an approach that is both precise and agile, we create medicines 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 approved medicines directly to patients in the United States and Europe, and we are globally advancing multiple programs for systemic mastocytosis, lung cancer and other genomically defined cancers, and cancer immunotherapy. 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, timelines and expectations for interactions with the FDA and other regulatory authorities; plans and timelines to update the primary endpoint of the registrational PIONEER trial of AYVAKIT in patients with non-advanced SM; expectations regarding the potential benefits of AYVAKIT in treating patients with non-advanced SM and advanced SM; 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 report 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 report, 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 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 either as monotherapies or in combination with other agents or may impact the anticipated timing of data or regulatory submissions; the timing of the initiation of clinical trials and trial cohorts at clinical trial sites and patient enrollment rates; 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; Blueprint Medicines’ ability to successfully expand its operations, research platform and portfolio of therapeutic candidates, and the timing and costs thereof; Blueprint Medicines’ ability to realize the anticipated benefits of its executive leadership transition plan; and the success of Blueprint Medicines’ current and future collaborations, acquisitions, 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 report 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.
Trademarks
Blueprint Medicines, AYVAKIT, AYVAKYT, GAVRETO and associated logos are trademarks of Blueprint Medicines Corporation.
Media Contact
Sarah Mena Guerrero
+1 (617)-714-6684
media@blueprintmedicines.com
Investor Relations Contact
Kristin Hodous
+1 (617)-714-6674
ir@blueprintmedicines.com
References
i Sperr WR, Kundi M, Alvarez-Twose I, et al. International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study. Lancet Haematol. 2019;6(12):e638-e649.
ii Estimated SM prevalence and patient subtypes based on internal claims analysis and epidemiology reported in Orphanet (orpha.net) and Cohen SS, Skovbo S, Vestergaard H, et al. Epidemiology of systemic mastocytosis in Denmark. Br J Haemotol. 2014;166(4):521-528.
iii Jennings SV, Slee VM, Zack RM, et al. Patient perceptions in mast cell disorders. Immunol Allergy Clin North Am. 2018;38(3):505-525.
iv Mesa RA, Sullivan EM, Dubinski D, et al. Patient reported outcomes among systemic mastocytosis (SM) patients in routine clinical practice: results from the TouchStone Survey. Blood. 2020;136(1):37.