UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
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Item 1.01 Entry into a Material Definitive Agreement.
Amendment – Roche Immunotherapy Collaboration
On January 8, 2021, Blueprint Medicines Corporation (the “Company”) entered into a ninth amendment to its collaboration and license agreement, as amended, with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. (collectively, “Roche”), related to the discovery, development and commercialization of small molecule therapeutics targeting kinases in cancer immunotherapy. Pursuant to the amendment, the Company and Roche agreed to modify certain time periods related to Roche’s option rights for one of the collaboration programs and agreed to terminate two of the other collaboration programs, including certain mechanics related to the wind-down of activities for such terminated targets. As a result of the amendment, the parties are currently conducting activities for up to two programs under the collaboration, including the previously announced program for the kinase target MAP4K1, which is believed to play a role in T cell regulation. Subject to the terms of the agreement, as amended, in addition to upfront and milestone payments previously received, the Company is eligible to receive up to approximately $323 million in contingent option fees and milestone payments related to specified research, pre-clinical, clinical, regulatory and sales-based milestones.
The foregoing description of the material terms of the ninth amendment to the collaboration and license agreement with Roche is qualified in its entirety by reference to the complete text of such amendment, which the Company intends to file, with confidential terms redacted, with the Securities and Exchange Commission (“SEC”) as an exhibit to the Company’s Annual Report on Form 10-K for the year ended December 31, 2020.
Item 5.02 Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officers.
Transition of Anthony L. Boral, M.D., Ph.D. from Chief Medical Officer to Executive Vice President, Clinical Development
Effective as of January 11, 2021, the Company entered into a first amendment to employment agreement with Anthony L. Boral, M.D., Ph.D. pursuant to which Dr. Boral will transition from the role of Chief Medical Officer into the role of Executive Vice President, Clinical Development. In this role, Dr. Boral will advise on clinical development, regulatory and business development strategy across the Company’s portfolio. Pursuant to the terms of the amendment, Dr. Boral will devote 60% of his full working time and efforts to the business and affairs of the Company, Dr. Boral’s annual base salary will be reduced to $286,196, and he will be eligible for an annual performance bonus targeted at 35% of his annualized base salary.
The foregoing description of the first amendment to employment agreement with Dr. Boral is qualified in its entirety by reference to the complete text of such amendment, a copy of which is attached as Exhibit 10.1 to this Current Report on Form 8-K.
Appointment of Becker Hewes, M.D. as Chief Medical Officer
Effective as of January 11, 2021, the board of directors of the Company promoted Becker Hewes, M.D., the Company’s Senior Vice President, Clinical Development, to succeed Dr. Boral and serve as Chief Medical Officer, and the Company entered into an amended and restated employment agreement with Dr. Hewes, which provides for “at will” employment. As Chief Medical Officer, Dr. Hewes will be responsible for clinical development, clinical operations, pharmacovigilance, translational medicine and biostatistics.
Dr. Hewes brings 20 years of industry and clinical experience in oncology and hematology, including achieving the approval of three tyrosine kinase inhibitors. Dr. Hewes, age 55, previously served as Senior Vice President, Clinical Development, of the Company from May 2020 to January 2021. Prior to joining the Company, Dr. Hewes served as Chief Medical Officer of Repertoire Immune Medicines (formerly Torque Therapeutics) (“Repertoire”) from February 2017 to May 2020, where he built Repertoire’s multidisciplinary clinical and biomarker team and advanced its lead immuno-oncology programs into clinical development. From June 2013 to February 2017, Dr. Hewes served as Executive Director of Translational Clinical Oncology at the Novartis Institutes for BioMedical Research where he led clinical development and translational medicine efforts for multiple early-stage oncology programs through clinical proof-of-concept, including Kisqali® (ribociclib), a targeted therapy approved to treat breast cancer, and other programs combining novel therapies. Prior to that, he held roles of increasing responsibility related to clinical development in oncology and hematology within AstraZeneca PLC, Genzyme Corporation and Wyeth Pharmaceuticals, including leading registration programs for Bosulif® (bosutinib) and Torisel® (temsirolimus) for chronic myelogenous leukemia and mantle cell lymphoma, respectively. Before joining industry, he conducted immuno-oncology research at the Emory Vaccine Center while treating patients as a pediatric oncologist at Children’s Healthcare of Atlanta. Dr. Hewes holds an M.D. from the Georgetown University School of Medicine and a B.S. from Vanderbilt University.
Pursuant to the terms of his amended and restated employment agreement, Dr. Hewes is entitled to an annual base salary of $462,000, effective as of January 1, 2021. Dr. Hewes is also eligible for an annual performance bonus targeted at 45% of his annualized base salary. In addition, pursuant to the terms of his amended and restated employment agreement,
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if Dr. Hewes’ employment is terminated by the Company without cause or by Dr. Hewes for good reason, and subject to Dr. Hewes’ execution of a release of potential claims against the Company, Dr. Hewes will be entitled to receive: (i) a lump sum in cash in an amount equal to 12 months of base salary and (ii) a monthly cash payment for 12 months for medical and dental benefits or Dr. Hewes’ COBRA health continuation period, whichever ends earlier. However, in the event that Dr. Hewes’ employment is terminated by the Company without cause, or Dr. Hewes terminates his employment with the Company for good reason, in either case within 12 months following the occurrence of a sale event (as defined in his amended and restated employment agreement), in lieu of the severance payments and benefits described in the preceding sentence and subject to Dr. Hewes’ execution of a release of potential claims against the Company, Dr. Hewes will be entitled to receive: (i) a lump sum in cash in an amount equal to the sum of 12 months of Dr. Hewes’ base salary then in effect plus Dr. Hewes’ target annual incentive compensation for the year in which the termination occurs, (ii) a monthly cash payment for 12 months for medical and dental benefits or Dr. Hewes’ COBRA health continuation period, whichever ends earlier, and (iii) full and immediate vesting and exercisability of all time-based stock options and other time-based stock-based awards held by Dr. Hewes.
In connection with Dr. Hewes’ appointment as Chief Medical Officer, Dr. Hewes entered into the Company’s standard form of indemnification agreement, a copy of which was filed as Exhibit 10.12 to the Company’s Registration Statement on Form S-1 (File No. 333-202938) filed with the Securities and Exchange Commission on March 23, 2015. Pursuant to the terms of the indemnification agreement, the Company may be required, among other things, to indemnify Dr. Hewes for some expenses, including attorneys’ fees, judgments, fines and settlement amounts incurred by his in any action or proceeding arising out of his service as one of our officers. Dr. Hewes has also previously entered into a confidentiality, assignment and non-competition agreement that contains, among other things, non-competition and non-solicitation provisions that apply during the term of Dr. Hewes’ employment and for 12 months thereafter.
Dr. Hewes has no family relationship with any of the executive officers or directors of the Company. There are no arrangements or understandings between Dr. Hewes and any other person pursuant to which he was appointed as an officer of the Company.
The foregoing description of the amended and restated employment agreement with Dr. Hewes is qualified in its entirety by reference to the complete text of such agreement, a copy of which is attached as Exhibit 10.2 to this Current Report on Form 8-K.
Item 7.01 Regulation FD.
From time to time, the Company presents and/or distributes to the investment community at various industry and other conferences slide presentations to provide updates and summaries of its business. The Company is posting to the “Investors & Media” portion of its website at http://ir.blueprintmedicines.com/ a copy of its current corporate slide presentation. 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 January 11, 2021, the Company issued a press release announcing its corporate goals for 2021 and certain other business updates. 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.
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Exhibit No. |
| Description |
10.1# |
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10.2# |
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99.1 |
| Corporate slide presentation of Blueprint Medicines Corporation dated January 11, 2021 |
99.2 |
| Press release issued by Blueprint Medicines Corporation on January 11, 2021 |
104 |
| Cover Page Interactive Data File (embedded within the Inline XBRL document and incorporated as Exhibit 101) |
# Indicates management contract or compensatory plan or arrangement.
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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.
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| BLUEPRINT MEDICINES CORPORATION | |
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Date: January 11, 2021 | By: | /s/ Jeffrey W. Albers |
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| Jeffrey W. Albers |
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| Chief Executive Officer |
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Exhibit 10.1
FIRST AMENDMENT TO EMPLOYMENT AGREEMENT
This First Amendment to Employment Agreement (the “First Amendment”) between Blueprint Medicines Corporation, a Delaware corporation (the “Company”), and Anthony L. Boral (the “Executive”), is effective as of January 11, 2021 (the “Amendment Effective Date”). Capitalized terms used and not defined herein shall have the meanings ascribed to such terms in the Employment Agreement (as defined below).
WHEREAS, the Company and the Executive are parties to the Employment Agreement dated as of November 6, 2015 (the “Employment Agreement”); and
WHEREAS, the Company and the Executive desire to amend the Employment Agreement as set forth below in connection with the mutually agreed upon reduction in the amount of the Executive’s time that will be devoted to the Company;
NOW, THEREFORE, for good and valuable consideration, the receipt of which is hereby confirmed, the Company and the Executive agree that the Employment Agreement is amended effective as of the Amendment Effective Date, as follows:
“Position and Duties. The Executive shall serve as Executive Vice President, Clinical Development, of the Company. As Executive Vice President, Clinical Development, the Executive shall report to, and perform services for the Company as determined by, the Company’s President, Research and Development (“President, R&D”) or another executive designated by the Company. The Executive acknowledges and agrees that his role as Executive Vice President, Clinical Development, may change from time to time, and such changes shall not constitute “Good Reason” as defined herein unless they are made without his consent and constitute a material diminution in the Executive’s responsibilities, authority or duties, in the aggregate, as Executive Vice President, Clinical Development. The Executive will devote sixty percent (60%) of his full working time and efforts to the business and affairs of the Company. The Executive may engage in outside professional activities including by serving on other boards of directors, provided such activities do not pose a conflict of interest and are approved in advance by the Board of Directors of the Company (the “Board”). The Executive may also engage in religious, charitable, or other community activities as long as such services and activities do not materially interfere with the performance of his duties to the Company as provided in this Agreement.”
2. Section 2(a) of the Employment Agreement shall be replaced in its entirety with the following:
“Base Salary. The Executive’s annualized base salary shall be $286,196. The Executive’s annualized base salary shall be reviewed annually and may be subject to increase but not decrease (other than for any mutually agreed-upon reduction in the amount of the Executive’s time that will be devoted to the Company) while serving in the
role as Executive Vice President, Clinical Development. The annualized base salary in effect at any given time is referred to herein as “Base Salary.” The Base Salary shall be payable in a manner that is consistent with the Company’s usual payroll practices for senior executives.”
“Incentive Compensation. During the Term, the Executive shall be eligible to earn cash incentive compensation as determined by the Board or the Compensation Committee of the Board from time to time. Beginning with the performance period for the year ending December 31, 2021, Executive’s target annual incentive compensation shall be 35% of his Base Salary (the “Target Incentive Compensation”), and the Board shall weigh its bonus determination as follows: 60% on Company performance and 40% on Executive’s individual performance. For the avoidance of doubt, for the year ended December 31, 2020, the Executive shall remain eligible to earn incentive compensation based on Executive’s previously determined target annual incentive compensation for such year, which is 45% of his Base Salary, with the Board weighing the bonus determination as 75% on Company performance and 25% on Executive’s individual performance. To earn any incentive compensation, the Executive must be employed by the Company on the day such incentive compensation is paid.”
“Other Benefits. The Executive shall be eligible to participate in or receive benefits under the Company’s employee benefit plans in effect from time to time to the extent such plans apply to part-time employees working a 60% schedule, subject to the plans’ respective terms and conditions.”
“Vacation. The Executive shall be entitled to accrue paid vacation during the Term in accordance with the Company’s applicable policy, pro-rated based on his part-time schedule.
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“a material diminution in the Executive’s responsibilities, authority or duties, in the aggregate, without the Executive’s consent.”
[Signature page follows.]
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IN WITNESS WHEREOF, the undersigned have executed this First Amendment as of the Amendment Effective Date.
| BLUEPRINT MEDICINES CORPORATION | |
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| By: | /s/ Jeffrey W. Albers |
| Name: | Jeffrey W. Albers |
| Title: | President and Chief Executive Officer |
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| EXECUTIVE | |
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| /s/ Anthony L. Boral | |
| Anthony L. Boral | |
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Exhibit 10.2
AMENDED AND RESTATED
EMPLOYMENT AGREEMENT
This amended and restated employment agreement (“Agreement”) is between Blueprint Medicines Corporation, a Delaware corporation (the “Company”), and L. Becker Hewes, M.D. (the “Executive”) and is effective as of January 11, 2021 (the “Effective Date”).
WHEREAS, the Company and the Executive are parties to an employment agreement, effective as of May 20, 2020 (the “Original Employment Agreement”);
WHEREAS, the Company and the Executive desire to enter into this Agreement to replace the Original Employment Agreement; and
WHEREAS, the Company desires to employ the Executive and the Executive desires to be employed by the Company on the terms and conditions contained herein.
NOW, THEREFORE, in consideration of the mutual covenants and agreements herein contained and other good and valuable consideration, the receipt and sufficiency of which is hereby acknowledged, the parties agree as follows:
| 1. | Employment. |
| 2. | Compensation and Related Matters. |
| 4. | Compensation Upon Termination. |
The amounts payable under this Section 4(b) shall be paid out in substantially equal installments in accordance with the Company’s payroll practice over twelve (12) months commencing within 60 days after the Date of Termination; provided, however, that if the 60-day period begins in one calendar year and ends in a second calendar year, the Severance Amount shall begin to be paid in the second calendar year by the last day of such 60-day period; provided, further, that the initial payment shall include a catch-up payment to cover amounts retroactive to the day immediately following the Date of Termination. Each payment pursuant to this Agreement is intended to constitute a separate payment for purposes of Treasury Regulation Section l.409A-2(b)(2).
The receipt of severance payments and benefits pursuant to Section 4 will be subject to Executive not violating the Restrictive Covenants Agreement and the Separation Agreement and Release. In the event Executive breaches any of the provisions of either such agreement, in addition to all other available legal and equitable remedies, the Company shall have the right to terminate or suspend all continuing payments and benefits to which Executive may otherwise be entitled pursuant to Section 4 (including without limitation the Severance Amount) without affecting the Executive’s release or Executive’s obligations under the Separation Agreement and Release.
The amounts payable under Section 5(a)(i) and (ii) shall be paid or commence to be paid within 60 days after the Date of Termination; provided however, that if the 60-day period begins in one calendar year and ends in a second calendar year, such payment shall be paid or commence to be paid in the second calendar year by the last day of such 60-day period.
Notwithstanding the foregoing, a “Sale Event” shall not be deemed to have occurred for purposes of the foregoing clauses (ii) and (iv) solely as the result of an acquisition of securities by the Company which, by reducing the number of shares of voting securities outstanding, increases the proportionate number of voting securities beneficially owned by any person to 50% or more of the combined voting power of all of the then outstanding voting securities; provided, however, that if any person referred to in this sentence shall thereafter become the beneficial owner of any additional shares of voting securities (other than pursuant to a stock split, stock dividend, or similar transaction or as a result of an acquisition of securities directly from the Company) and immediately thereafter beneficially owns 50% or more of the combined voting power of all of the then outstanding voting securities, then a “Sale Event” shall be deemed to have occurred for purposes of the foregoing clauses (ii) and (iv).
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| 6. | Section 409A. |
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[Signature page follows.]
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IN WITNESS WHEREOF, the parties have executed this Agreement as of the Effective Date.
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| BLUEPRINT MEDICINES CORPORATION | |
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| By: | /s/ Jeffrey Albers |
| Name: | Jeffrey Albers |
| Title: | President and Chief Executive Officer |
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| EXECUTIVE | |
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| /s/ L. Becker Hewes | |
| Name: | L. Becker Hewes, M.D. |
Signature Page – Amended and Restated
Employment Agreement
Exhibit 99.1
| © 2021 Blueprint Medicines Corporation R.S., living with systemic mastocytosis PRECISION THAT MOVES™ Staying one step ahead of disease JANUARY 2021 COMPANY OVERVIEW |
| Forward-looking statements 2 This presentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. 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. In this presentation, forward-looking statements include, without limitation, statements regarding 2021 goals and anticipated milestones for Blueprint Medicines Corporation (the “Company”); plans, strategies, timelines and expectations for the Company’s current or future approved drugs and drug candidates, including timelines for marketing applications and approvals, the initiation of clinical trials, or results of ongoing and planned clinical trials; the potential benefits of any of the Company’s current or future approved drugs or drug candidates in treating patients; and the Company’s strategy, goals and anticipated milestones, business plans and focus. The Company has based these forward-looking statements on management's current expectations, assumptions, estimates and projections. While the Company believes these expectations, assumptions, estimates and projections are reasonable, such forward-looking statements are only predictions and involve known and unknown risks, uncertainties and other important factors, many of which are beyond the Company's control and may cause actual results, performance or achievements to differ materially from those expressed or implied by any forward-looking statements. These risks and uncertainties include, without limitation, risks and uncertainties related to the impact of the COVID-19 pandemic to the Company's business, operations, strategy, goals and anticipated milestones, including the Company's 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 drugs, and launching, marketing and selling current or future approved drugs; the Company’s ability and plans in establishing a commercial infrastructure, and successfully launching, marketing and selling current or future approved products; the Company’s ability to successfully expand the approved indications for AYVAKIT™/AYVAKYT® (avapritinib) and GAVRETO™(pralsetinib) 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 the Company's drug candidates or the licensed drug candidate; the Company's advancement of multiple early-stage efforts; the Company's ability to successfully demonstrate the efficacy and safety of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; the preclinical and clinical results for the Company's drug candidates, which may not support further development of such drug candidates; actions or decisions of regulatory agencies or authorities, which may affect the initiation, timing and progress of clinical trials or marketing applications; the Company's ability to obtain, maintain and enforce patent and other intellectual property protection for AYVAKIT/AYVAKYT, GAVRETO or any drug candidates it is developing; the Company's ability to develop and commercialize companion diagnostic tests for any of the Company's current or future approved drugs or drug candidates; and the success of the Company's current and future collaborations, partnerships and licenses. These and other risks and uncertainties are described in greater detail under "Risk Factors" in the Company's filings with the Securities and Exchange Commission ("SEC"), including its most recent Annual Report on Form 10-K, as supplemented by its most recent Quarterly Report on Form 10-Q, and any other filings it has made or may make with the SEC in the future. The Company cannot guarantee future results, outcomes, levels of activity, performance, developments, or achievements, and there can be no assurance that its expectations, intentions, anticipations, beliefs, or projections will result or be achieved or accomplished. The forward-looking statements in this presentation are made only as of the date hereof, and except as required by law, the Company undertakes no obligation to update any forward-looking statements contained in this presentation as a result of new information, future events or otherwise. This presentation also contains estimates, projections and other statistical data made by independent parties and by the Company relating to market size and growth and other data about the Company's industry. These data involve a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections, assumptions and estimates of the Company's future performance and the future performance of the markets in which the Company operates are necessarily subject to a high degree of uncertainty and risk. Blueprint Medicines, AYVAKIT, AYVAKYT, GAVRETO and associated logos are trademarks of Blueprint Medicines Corporation. |
| Hopeful foundation A new precision therapy platform 2010 Hopeful reality ~2,600 patients treated with an approved or investigational Blueprint Medicines therapy 2021 Rob T. Living with GIST |
| 2020: a transformational year for Blueprint Medicines 4 Build commercial momentum Advance registration program for SM Strengthen pipeline with new programs ✓ AYAVKIT™ / AYVAKYT® (avapritinib) approved for PDGFRA-driven GIST in the U.S. and Europe1 ✓ GAVRETO™ (pralsetinib) approved for RET-altered NSCLC, MTC and other thyroid cancers in the U.S.2 ✓ Initiated transformational global collaboration with Roche to develop and commercialize GAVRETO ✓ Submitted sNDA to FDA for AYVAKIT for the treatment of advanced systemic mastocytosis (SM) ✓ Initiated global enrollment of registration-enabling Part 2 of PIONEER trial of AYVAKIT in non-advanced SM ✓ Received FDA breakthrough therapy designation for AYVAKIT for moderate to severe indolent SM ✓ Nominated four new development candidates since Q4 2019 • BLU-263, a next-generation KIT inhibitor, for non-advanced SM and other KIT-driven disorders • BLU-945, a triple-mutant EGFR inhibitor, for treatment-resistant EGFR-driven NSCLC • Double-mutant EGFR inhibitor, for treatment-resistant EGFR-driven NSCLC • MAP4K1 inhibitor, under our cancer immunotherapy collaboration with Roche 1. AYVAKIT is approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. AVAKYT is approved in Europe for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA D842V mutation. 2. GAVRETO is approved in the U.S. for adults with metastatic RET fusion-positive NSCLC, adult and pediatric patients with advanced or metastatic RET-mutant MTC 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; MTC, medullary thyroid cancer; NSCLC, non-small cell lung cancer; sNDA, supplemental new drug application. ~$1.36B IN CASH, CASH EQUIVALENTS AND INVESTMENTS AT END OF Q3 2 020 Not for promotional use. |
| Blueprint Medicines’ core mission and foundational principles 5 We aim to make real the promise of precision therapy to improve and extend life for as many people with cancer and hematologic disorders as possible TRANSFORMATIVE BENEFIT PRECISION ADAPTIVE ABILITY RELENTLESS DRIVE FOCUS ON URGENT PATIENT NEEDS HIGHLY POTENT AND SELECTIVE INHIBITORS PREVENT AND OVERCOME RESISTANCE FULLY INTEGRATED GLOBAL BIOPHARMACEUTICAL COMPANY Not for promotional use. SCALABLE PLATFORM & COMMERCIALIZATION |
| A leader in precision oncology and hematology 6 ONCOLOGY HEMATOLOGY RARE DISEASES PRIMARY FOCUS OPPORTUNISTIC PORTFOLIO AREAS OF FOCUS SYSTEMIC MASTOCYTOSIS LUNG CANCER THERAPEUTIC AREA LEADERSHIP Not for promotional use. |
| 1. Unresectable or metastatic disease. 2. CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib, pralsetinib and fisogatinib in Mainland China, Hong Kong, Macau and Taiwan. 3. Approved in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Received conditional marketing authorization in Europe under the brand name AVYAKYT® for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. 4. In collaboration with Roche. Blueprint Medicines and Roche have co-exclusive rights to develop and commercialize pralsetinib in the U.S., and Roche has exclusive rights to develop and commercialize pralsetinib outside the U.S., excluding the CStone territory. 5. Received accelerated approval in the U.S. for the treatment of adults with metastatic RET fusion-positive NSCLC. Continued approval may be contingent on a confirmatory trial. The proposed indication for the MAA is locally advanced or metastatic RET fusion-positive NSCLC previously treated with platinum- based chemotherapy. 6. Received accelerated approval in the U.S. for the treatment of patients with advanced or metastatic RET-mutant medullary thyroid cancer and RET fusion-positive thyroid cancer. Continued approval may be contingent on confirmatory trials. 7. In collaboration with Roche. For one of the programs, Blueprint Medicines has U.S. commercial rights and Roche has ex-U.S. commercialization rights. For one of the programs, Roche has worldwide commercialization rights. GIST, gastrointestinal stromal tumors; HCC, hepatocellular carcinoma; MAA, marketing authorization application; MTC, medullary thyroid cancer; NDA, new drug application; NSCLC, non-small cell lung cancer; SM, systemic mastocytosis. DISCOVERY EARLY-STAGE DEVELOPMENT LATE-STAGE DEVELOPMENT REGULATORY SUBMISSION APPROVED U.S., Europe PDGFRA GIST1,2,3 Non-advanced SM2 AYVAKIT™ (avapritinib) (PDGFRA & KIT) Fisogatinib (FGFR4) EGFR+ NSCLC1 BLU-945 (EGFR+ triple mutant) EGFR+ NSCLC1 (EGFR+ double mutant) (3 undisclosed targets) (MAP4K1)7 (1 undisclosed immunokinase target)7 Non-advanced SM BLU-263 (KIT) ongoing or completed planned Advanced HCC (+/- sugemalimab)2 GAVRETO™ (pralsetinib) (RET) Updated as of January 11, 2021 Other RET-altered solid tumors1,2,4 RET+ MTC1,2,4,6 U.S. MAA Not for promotional use. Blueprint Medicines, AYVAKIT, AYVAKYT, GAVRETO and associated logos are trademarks of Blueprint Medicines Corporation. NDA Advanced SM2 MAA RET+ thyroid cancer1,2,4,6 U.S. MAA RET+ NSCLC1,2,4,5 U.S. MAA |
| 2021 roadmap for precision medicine leadership 8 Accelerate global adoption of AYVAKIT and GAVRETO Advance a new wave of therapeutic candidates toward clinical proof-of-concept Further expand the company’s precision therapy pipeline Not for promotional use. |
| 2021 roadmap for precision medicine leadership 9 Accelerate global adoption of AYVAKIT and GAVRETO Advance a new wave of therapeutic candidates toward clinical proof-of-concept Further expand the company’s precision therapy pipeline Not for promotional use. |
| Two precision therapies first approved in 2020 with clear pathways for growth 10 • Approved for unresectable or metastatic PDGFRA exon 18 mutant GIST • Approved for advanced or metastatic RET-altered NSCLC, MTC and other thyroid cancers CORE VALUE OPPORTUNITY • sNDA submitted to FDA for advanced SM in Q4 2020 • Plan to submit MAA to EMA for advanced SM in Q1 2021 • Registrational PIONEER trial in non-advanced SM enrolling • FDA breakthrough therapy designations granted for advanced SM and moderate to severe indolent SM GROWTH OPPORTUNITY • Transformative global collaboration with Roche – Ongoing co-commercialization in the U.S. – MAA for RET fusion+ NSCLC under review by EMA – Plan to submit marketing applications across multiple additional global geographies – Plan to develop in additional treatment settings Not for promotional use. |
| Systemic mastocytosis is driven by KIT D816V Mild symptoms Non-Advanced SM Severe symptoms Indolent SM (ISM) • Some progression to advanced disease (4%)2 • Long-term morbidity, including life- threatening anaphylaxis, frequent GI upset, and debilitating fatigue3 Smoldering SM (SSM) • Increased organ infiltration1,4 • Increased progression to advanced disease (9%)2 AML, acute myeloid leukemia; ASM, aggressive systemic mastocytosis; GI, gastrointestinal; MCL, mast cell leukemia; SM-AHN, systemic mastocytosis with associated hematologic neoplasm. 1. Pardanani A. Am J Hematol. 2016;91(11):1146-1159. 2. Sperr WR et al. Lancet Haematol. 2019;6(12):e638-e649. 3. Jennings SV et al. Immunol Allergy Clin North Am. 2018;38(3):505-525. 4. Swerdlow SH et al, eds. WHO Classification of Tumours of Haematopoietic and Lymphoid Tissues. Revised 4th ed. Lyon, France: International Agency for Research on Cancer; 2017. 5. Shomali W, Gotlib J. Hematology Am Soc Hematol Educ Program. 2018;2018(1):127-136. 6. Desmond DH, Carmichael MG. Hawaii J Med Public Health. 2018;77(2):27-29. Minimal organ damage4 Advanced SM (ASM, SM-AHN, MCL) • Extensive organ infiltration and damage1 • Historical overall survival (OS) <6 mo to <5 years1,5,6 Significant Organ damage4 11 Not for promotional use. |
| Significant initial target patient population with additional growth potential Major markets include U.S., France, Germany, Italy, Spain, the United Kingdom and Japan. 1. Cohen S et al Br J Haematol (2014) 166(4):521-8 and World Bank Population estimates. 12 ~75,000 PATIENTS IN MAJOR MARKETS ~1/3 DIAGNOSED INITIAL OPPORTUNITY GROWTH OPPORTUNITY Estimated addressable patients (extrapolated from U.S. claims data and market research analyses) Estimated prevalence1 • ~5% advanced SM • ~95% non-advanced SM ~1/3 MISDIAGNOSED CUTANEOUS MASTOCYTOSIS ~1/3 UNDIAGNOSED NON-ADVANCED Not for promotional use. |
| Pursuing a range of testing initiatives to facilitate SM patient identification 13 ENHANCE TESTING INFRASTRUCTURE CHANGE TESTING BEHAVIOR DATA AGREEMENTS SUPPORT TO STAND UP TESTING SPONSORED TESTING INITIATIVES AWARENESS & EDUCATION Expand testing footprint Optimize testing protocols Support access to testing Empower patients DATA SHOW HIGHLY SENSITIVE DDPCR TESTING DETECTS KIT D816V IN 95% OF PATIENTS 1 Anticipate highly sensitive ddPCR KIT D816V testing to be widely available in 2021 at laboratories currently testing ~80% of SM patients in U.S.2 1. Data in patients with non-advanced SM presented at the American Society of Hematology Annual Meeting in December 2020. 2. Based on internal market research. Not for promotional use. |
| AVYAKIT registration program in advanced systemic mastocytosis 14 mIWG, modified International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) & European Competence Network on Mastocytosis (ECNM) response criteria. AdvSM, advanced systemic mastocytosis; QD, once daily. AdvSM Open-label N= ~80 AdvSM Open-label N= ~100 30-400 mg QD 300 mg QD 200 mg QD 200 mg QD DOSE ESCALATION REGISTRATIONAL PHASE 1 TRIAL PHASE 2 TRIAL PRIMARY ENDPOINT FOR APPROVAL: OVERALL RESPONSE RATE PER mIWG Not for promotional use. REGISTRATIONAL |
| Consistently high ORRs and prolonged duration of response across trials Top-line EXPLORER and PATHFINDER data presented in September 2020. Data cutoff: May 27, 2020 for EXPLORER and June 23, 2020 for PATHFINDER, with response assessments per central review completed in September 2020. CR, complete remission; CRh, CR with partial hematologic recovery; CI, clinical improvement; mDOR, median duration of response; mOS, median overall survival; NE, not evaluable; ORR, overall response rate; PR, partial remission. 15 ORR (CR+CRh+PR+CI) 75.5% (61.7- 86.2) 75.0 (56.6 – 88.5) CR+CRh 35.8% 18.8% mDOR (months) 38.3 (21.7 - NE) NE (NE - NE) mOS (months) NE (46.9 - NE) NE 68.2% 18.2% PATHFINDER INTERIM ANALYSIS WAS POSITIVE (P-VALUE=0.0000000016) Median follow up: 27.3 months Median follow up: 10.4 months Median follow up: 10.4 months 2 0 0 M G Q D POOLED GROUP Not for promotional use. |
| Deep reductions in mast cell burden and resolution of organ damage 16 -100 -80 -60 -40 -20 0 20 M a x i m u m P e r c e n t C h a n g e f r o m B a s e l i n e BLU-285-2202 BLU-285-2101 Study SERUM TRYPTASE1 -100 -80 -60 -40 -20 0 20 40 60 M a x i m u m P e r c e n t C h a n g e f r o m B a s e l i n e BLU-285-2202 BLU-285-2101 Study BONE MARROW MAST CELLS 1 -100 -80 -60 -40 -20 0 20 M a x i m u m P e r c e n t C h a n g e f r o m B a s e l i n e BLU-285-2202 BLU-285-2101 Study KIT D816V ALLELE FRACTION 1 Explorer Pathfinder 1. Top-line EXPLORER and PATHFINDER data presented in September 2020. Data cutoff: May 27, 2020 for EXPLORER and June 23, 2020 for PATHFINDER, with response assessments per central review completed in September 2020. 2. EXPLORER patient case presented at ASH 2018 annual meeting in December 2018. Baseline Cycle 6 Day 1 RESOLUTION OF ORGAN DAMAGE (C-FINDINGS)2 • Weight loss of >50 pounds • Hypoalbuminemia (2.3 mg/dL) • Ascites with paracentesis (15 L/week) • All weight gained back • Albumin normalized • Ascites resolved Patient with SM - AHN Not for promotional use. |
| AYVAKIT demonstrated improved tolerability at 200 mg QD 17 Treatment Emergent AEs ≥ 20%, All Grades* 200 mg n=81 (%) All doses N=148 (%) Peripheral Edema 39 ( 48.1) 65 ( 43.9) Periorbital Edema 32 ( 39.5) 81 ( 54.7) Thrombocytopenia 28 ( 34.6) 55 ( 37.2) Anemia 26 ( 32.1) 65 ( 43.9) Diarrhea 23 ( 28.4) 53 ( 35.8) Nausea 20 ( 24.7) 49 ( 33.1) Fatigue 15 ( 18.5) 44 ( 29.7) Vomiting 15 ( 18.5) 42 ( 28.4) • Overall, 8.1% of patients discontinued treatment due to treatment-related AEs • ICB risk mitigations implemented o Starting dose of 200 mg QD o Exclusion criteria for pre-existing severe thrombocytopenia o Increased platelet monitoring o Mandatory dose interruption for severe thrombocytopenia • ICB events in patients without pre-existing severe thrombocytopenia o Pooled 200 mg group (n=76): 2 (2.6%)† o PATHFINDER (n=57): 0‡ Cognitive effects 10 ( 12.3) 37 ( 25.0) ≥Grade 2 2 (2.5) 13 (8.8) Top-line EXPLORER and PATHFINDER data presented in September 2020. Data cutoff: May 27, 2020 for EXPLORER and June 23, 2020 for PATHFINDER, with response assessments per central review completed in September 2020. † Both ICB events in EXPLORER patients were Grade 1 and asymptomatic. ‡ 1 ICB event occurred in a PATHFINDER patient with pre-existing severe thrombocytopenia prior to exclusion of such patients for 1/62 (1.6%) overall. AE, adverse event; ICB, intracranial bleed. * Most common AEs in patients treated at 200mg in EXPLORER and PATHFINDER Not for promotional use. |
| Plan to complete enrollment of registrational Part 2 of PIONEER trial of AYVAKIT in non-advanced SM in mid-2021 18 ISM, indolent system mastocytosis; ISM-SAF, indolent systemic mastocytosis – symptom assessment form; RP2D, recommended phase 2 dose. Non-advanced SM RP2D 25 mg QD PART 1 DOSE ESCALATION REGISTRATIONAL PART 2 PHASE 2 TRIAL PRIMARY ENDPOINT FOR APPROVAL: CHANGE IN ISM-SAF TOTAL SYMPTOM SCORE PIONEER REGISTRATION-ENABLING PART 2 Design: Randomized, double-blind, placebo-controlled treatment period, followed by open-label expansion Key endpoints: Response rate defined as ≥30% reduction in ISM-SAF total symptom score (primary), measures of mast cell burden, quality of life, concomitant medications Duration: 24 weeks Enrolling N= ~200 Not for promotional use. |
| PIONEER Part 1 data showed AYVAKIT 25 mg QD reduces symptoms and mast cell burden in non-advanced SM Presented at EAACI Virtual 2020 Congress in June 2020. Data cutoff: March 31, 2020. *24 weeks or last assessment before, if 24 weeks not available. EAACI, European Academy of Allergy and Clinical Immunology. 19 Part 2 primary endpoint ≥30% reduction in ISM-SAF Total Symptom Score at 24 weeks 60% 0% Response rate: Part 2 first key secondary endpoint ≥50% tryptase reduction at 24 weeks* 70% 0% Not for promotional use. |
| Safety results for AYVAKIT 25mg QD are similar to placebo at 16 weeks1 1. Data presented in March 2020 at AAAAI annual meeting. Data cutoff: December 27, 2019. 2. Data cutoff: March 31, 2020. 20 AE in >15% of placebo or avapritinib arms avapritinib Preferred term Placebo n=9 25 mg n=10 % of subjects with ≥1 AE any grade grade 3 any grade grade 3 89 22 100 0 Nausea 22 0 10 0 Dizziness 22 0 30 0 Headache 11 0 30 0 Diarrhea 11 0 0 0 Fatigue 11 0 40 0 Face edema 0 0 10 0 Peripheral edema 0 0 10 0 Periorbital edema 0 0 0 0 Bone Pain 22 0 0 0 AVAPRITINIB 25 MG QD • No patients had serious AEs o 2 patients treated with placebo had serious AEs, 1 with psychogenic seizure and 1 with diffuse cutaneous mastocytosis • No patients had dose modifications • No patients discontinued due to AEs FOLLOW UP AT 24 WEEKS SHOWED NO ≥GRADE 3 AES OR DISCONTINUATIONS DUE TO AES FOR 25 MG QD2 Not for promotional use. |
| AYVAKIT is the only clinically validated, highly potent inhibitor of KIT D816V, the genetic driver of SM 1. Top-line EXPLORER and PATHFINDER data presented in September 2020. Data cutoff: May 27, 2020 for EXPLORER and June 23, 2020 for PATHFINDER, with response assessments per central review completed in September 2020. 2. Data reported at AAAAI Annual Meeting in March 2020. Data cutoff: December 27, 2019. 21 REDUCE MAST CELL BURDEN IMPROVE DISEASE SYMPTOMS INDUCE DEEP AND DURABLE RESPONSES -100 -80 -60 -40 -20 0 20 M a x i m u m P e r c e n t C h a n g e f r o m B a s e l i n e BLU-285-2202 BLU-285-2101 Study -5 0 -4 0 -3 0 -2 0 -1 0 0 1 0 W e e k s I S M - S A F T o t a l S y m p t o m S c o r e m e a n % c h a n g e f r o m b a s e l i n e 0 4 8 12 16 a v a p ritin ib 2 5 m g (n = 1 0 ) p la c e b o (n = 9 ) NON-ADVANCED SM2 ADVANCED SM1 Safety profile enables tailored dosing based on patient need ADVANCED SM1 NON-ADVANCED SM2 Not for promotional use. |
| 2021 roadmap for precision medicine leadership 22 Accelerate global adoption of AYVAKIT and GAVRETO Advance a new wave of therapeutic candidates toward clinical proof-of-concept Further expand the company’s precision therapy pipeline Not for promotional use. |
| Multiple additional opportunities for transformative medicines 23 PROGRAM (TARGET) DESCRIPTION / STATUS BLU-263 (KIT D816V) Non-advanced SM and other mast cell disorders • Well-tolerated in Phase 1 healthy volunteer trial • Plan to initiate Phase 2 trial in non-advanced SM in mid-2021 BLU-945 (triple-mutant EGFR) Treatment-resistant EGFR-driven NSCLC • Presented foundational preclinical data at ESMO 2020 • Plan to initiate Phase 1 trial in 1H 2021 (Double-mutant EGFR) Treatment-resistant EGFR-driven NSCLC • Plan to present foundational preclinical data in 1H 2021 • Plan to initiate Phase 1 trial by the end of 2021 (MAP4K1) Cancer immunotherapy, under collaboration with Roche • Plan to present foundational preclinical data in 1H 2021 10 precision oncology IPOs in 2020 4 had no clinical assets at time of IPO $2.3B mean market capitalization1 Not for promotional use. 4 DEVELOPMENT CANDIDATES NOMINATED SINCE Q4 2019 1. Estimated market capitalization at close of market on January 7, 2021. INDUSTRY BENCHMARK |
| Our vision for transforming treatment of EGFR+ NSCLC 1. Girard N. Future Oncol. 2018;14(11):1117–1132. 2. Leonetti, A et al. British Journal of Cancer. 2019;121:725–737. 1L, first-line treatment; 2L, second-line treatment; 3L, third-line treatment. 24 1ST GEN EGFR TKI OSIMERTINIB TRIPLE MUTANT INHIBITOR (BLU-945) T790M C797S OSIMERTINIB DOUBLE MUTANT INHIBITOR C797S DOUBLE MUTANT INHIBITOR TRIPLE MUTANT INHIBITOR (BLU-945) 1L TRANSFORMATIVE PREVENTIVE COMBO 2L SINGLE AGENT OR COMBO WITH EXISTING TKIs 3L SINGLE AGENT OR COMBO WITH EXISTING TKIs POTENTIAL FOR PROLONGED CLINICAL BENEFIT WITH TRANSFORMATIVE 1L PREVENTIVE COMBO • Primary EGFR mutation frequency in NSCLC: ~10-15% in the U.S. and Europe; ~40-50% in Asia1 • While current therapies have revolutionized care, treatment resistance is a significant, emerging medical need • T790M and C797S are most common on-target resistance mutations to 1st generation EGFR inhibitors and osimertinib2 Not for promotional use. |
| Foundational BLU-945 preclinical data presented at ESMO 2020 support initiation of clinical development in 1H 2021 Data presented at ESMO 2020 virtual conference in September 2020. 25 SUBNANOMOLAR POTENCY EXCELLENT SELECTIVITY ROBUST SINGLE AGENT ACTIVITY COMBINATION POTENTIAL PRECLINICAL CNS ACTIVITY L858R/ T790M/C797S ex19del/ T790M/C797S BLU-945 0.5 0.8 Gefitinib 3921.8 1219.7 Osimertinib 5461.6 649.9 BIOCHEMICAL IC 50 EGFR wild-type (A431 cell line) BLU-945 544.4 Gefitinib 16.5 Osimertinib 115.9 CELLULAR IC 50 Not for promotional use. |
| 2021 roadmap for precision medicine leadership 26 Accelerate global adoption of AYVAKIT and GAVRETO Advance a new wave of therapeutic candidates toward clinical proof-of-concept Further expand the company’s precision therapy pipeline Not for promotional use. |
| Constant expansion of highly productive research platform 27 PLAN TO EXPAND PIPELINE WITH ONE OR MORE DEVELOPMENT CANDIDATES WORLD-CLASS EXPERTISE IN CATALYTIC KINASE INHIBITION MOLECULAR TARGETING THERAPEUTIC DESIGN Deep biological insights across core areas of focus Vast genomic datasets and computational power Proprietary library of fully annotated compounds Sophisticated structure-based design capability EXPANDED INTERNAL CAPABILITY ACROSS ADDITIONAL PRECISION THERAPY MODALITIES POTENTIAL COMPLEMENTARY EXTERNAL INNOVATION PLANNED FUTURE Not for promotional use. |
| 2021 roadmap for precision medicine leadership: strategies and key goals 28 Accelerate global adoption of AYVAKIT and GAVRETO Advance a new wave of therapeutic candidates toward clinical proof-of-concept Further expand the company’s precision therapy pipeline • Obtain FDA approval and launch AYVAKIT for advanced SM in the U.S. in 2H 2020 • Submit MAA to EMA for AYAVKIT for advanced SM in Q1 2021 • Present registrational PATHINDER trial data for AYVAKIT in advanced SM in 1H 2021 • Complete enrollment of registration-enabling PIONEER trial in mid-2021 • Obtain EMA approval and launch GAVRETO for RET fusion-positive NSCLC in 1H 2021 • Submit MAA to EMA for GAVRETO for RET-altered thyroid cancers in 2H 2021 • Initiate GAVRETO cohort in Roche’s TAPISTRY tumor-agnostic platform trial in 2H 2021 • Submit multiple marketing applications for GAVRETO across multiple additional geographies • Initiate Phase 2 HARBOR trial of BLU-263 in non-advanced SM in mid-2021 • Initiate Phase 1 trial of BLU-945 in EGFR-driven NSCLC in 1H 2021 • Initiate Phase 1 trial of double-mutant EGFR inhibitor in EGFR-driven NSCLC by the end of 2021 • Present preclinical data for double-mutant EGFR and MAP4K1 inhibitors in 1H 2021 • Present preclinical data for combo of BLU-945 and double-mutant EGFR inhibitor in 2H 2021 • Expand pipeline with one or more development candidates. • Pursue external opportunities to complement the company’s precision medicine pipeline. Not for promotional use. |
| Blueprint Medicines is in the strongest financial position in our history 1. Includes stock-based compensation expense of $8.6M in 2020 and $7.7M in 2019. 2. Includes stock-based compensation expense of $11.0M in 2020 and $7.3M in 2019. 29 Statement of Operations (unaudited) Three Months Ended 9/30/2020 Three Months Ended 9/30/2019 Total revenue $745.1M $9.1M Collaboration revenue Net product sales $738.8M $6.3M $9.1M -- Cost of sales $0.1M -- Research & development expense1 $74.2M $81.5M Selling, general & administrative expense2 $37.4M $25.6M Net income (loss) $634.0M $(94.3)M Balance Sheet (unaudited) 9/30/2020 12/31/2019 Cash, cash equivalents and investments $1,355.9M $548.0M Based on current operating plans, expect existing cash balance, with anticipated product revenues, to enable self-sustainable financial profile |
Exhibit 99.2
Blueprint Medicines Reports Portfolio Milestones and Outlines 2021 Roadmap for Precision Medicine Leadership
-- AYVAKIT™ (avapritinib) granted FDA breakthrough therapy designation for the treatment of moderate to severe indolent systemic mastocytosis --
-- Positive top-line results from Phase 1 healthy volunteer trial of BLU-263 support plans to initiate Phase 2 HARBOR trial in non-advanced systemic mastocytosis in mid-2021 --
-- Nominated potential first-in-class development candidate targeting double-mutant EGFR, deepening leadership in lung cancer --
-- Nominated potential best-in-class development candidate targeting MAP4K1 under cancer immunotherapy collaboration with Roche --
CAMBRIDGE, Mass., Jan. 11, 2021 /PR Newswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC) today provided an update on key portfolio milestones and outlined a strategic roadmap to become the world’s leading precision therapy company.
“For the first time, we enter a new year as a fully integrated, global biopharmaceutical company, with four regulatory approvals in the United States and Europe in 2020, a pipeline of eight wholly owned or partnered precision therapies, and the strongest financial position since our inception,” said Jeff Albers, Chief Executive Officer of Blueprint Medicines. “With this solid foundation, we are now scaling our ambition and aim to make real the promise of precision medicine to improve and extend life for as many people with cancer and hematologic disorders as possible. We will do this by bringing our medicines to more patients globally, rapidly advancing a wave of new therapeutic candidates to clinical proof-of-concept, and further expanding our platform-enabled research pipeline.”
In addition, Blueprint Medicines today announced the achievement of several portfolio milestones:
| ● | AYVAKIT received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of moderate to severe indolent systemic mastocytosis (SM), which encompasses the majority of patients with SM, highlighting the medical need in this population as well as the clinical potential of AYVAKIT to demonstrate substantial improvement over the current standard of care. |
| ● | Positive top-line results from a Phase 1 trial in healthy volunteers showed BLU-263 was well-tolerated across a range of single- and multiple-ascending doses predicted to potently inhibit D816V mutant KIT, the underlying SM disease driver. These data support development of BLU-263 as a potential treatment for patients with SM and other mast cell disorders. |
| ● | The company nominated a selective, brain-penetrant development candidate for treatment-resistant double-mutant EGFR-driven non-small cell lung cancer (NSCLC), with the potential to be first-in-class, showing potent activity against the activating L858R or exon 19 deletion mutations and the acquired C797S mutation, the most common on-target resistance mutation to osimertinib. |
| ● | The company nominated a development candidate targeting MAP4K1, a kinase believed to play a role in T-cell regulation, with the potential to be best-in-class. The program was developed under the company’s cancer immunotherapy collaboration with Roche. In addition, Blueprint Medicines and Roche have amended their agreement to focus on MAP4K1 and one additional undisclosed target, collectively identified as the most promising targets of the collaboration to date. |
Entering 2021, the company’s key strategies and goals include:
1. Accelerate global adoption of AYVAKIT and GAVRETO™ (pralsetinib)
AYVAKIT, a selective KIT and PDGFRA inhibitor, is approved in the U.S. and Europe for the treatment of patients with unresectable or metastatic gastrointestinal stromal tumor driven by certain PDGFRA mutations.
| ● | Obtain FDA approval and launch AYVAKIT in advanced SM in the U.S. in the second half of 2021. |
| ● | Submit a Type II variation marketing authorization application (MAA) to the European Medicines Agency (EMA) for AYVAKYT® (avapritinib) for advanced SM in the first quarter of 2021. |
| ● | Present registrational data from the PATHFINDER trial of AYVAKIT in advanced SM in the first half of 2021. |
| ● | Complete enrollment of the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced SM in mid-2021. |
GAVRETO, a selective RET inhibitor, is approved in the U.S. for the treatment of patients with certain advanced or metastatic RET fusion-positive NSCLC, RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer. Under a global collaboration, Blueprint Medicines and Roche are developing and commercializing GAVRETO for the treatment of RET-altered cancers.
| ● | Obtain regulatory approval from the European Commission and launch GAVRETO in RET fusion-positive NSCLC in Europe in the first half of 2021. |
| ● | Submit a Type II variation MAA to the EMA for GAVRETO for RET-altered thyroid cancers in the second half of 2021. |
| ● | Initiate a GAVRETO cohort in Roche’s TAPISTRY tumor-agnostic platform trial in the second half of 2021. |
| ● | Submit marketing applications for GAVRETO for RET-altered NSCLC and thyroid cancers across multiple additional global geographies in 2021. |
2. Advance a new wave of innovative therapeutic candidates into clinical development, with plans to achieve rapid proof-of-concept and regulatory approval.
BLU-263, a next-generation selective KIT inhibitor
| ● | Initiate the Phase 2 HARBOR trial of BLU-263 in patients with non-advanced SM in mid-2021. |
Development candidates for treatment-resistant EGFR-driven NSCLC
| ● | Initiate a Phase 1 trial of BLU-945, a triple-mutant EGFR inhibitor, in patients with treatment-resistant EGFR-driven NSCLC in the first half of 2021. |
| ● | Initiate a Phase 1 trial of the company’s double-mutant EGFR inhibitor in patients with treatment-resistant EGFR-driven NSCLC by the end of 2021. |
| ● | Present foundational preclinical data for the company’s double-mutant EGFR inhibitor in the first half of 2021. |
| ● | Present preclinical data supporting combination of the company’s wholly owned double- and triple-mutant EGFR inhibitors in treatment-naïve EGFR-driven NSCLC in the second half of 2021. |
Development candidate targeting MAP4K1, under the cancer immunotherapy collaboration with Roche
| ● | Present foundational preclinical data in the first half of 2021. |
3. Further expand the company’s precision medicine pipeline with a focus on delivering transformational benefit to patients with cancer and hematologic disorders.
| ● | Expand pipeline with one or more development candidates in 2021. |
| ● | Pursue external opportunities to complement the company’s precision medicine pipeline. |
Financial Guidance
Based on its current operating plans, Blueprint Medicines continues to anticipate its existing cash, cash equivalents and investments, together with anticipated future product revenues, will provide sufficient capital to enable the company to achieve a self-sustainable financial profile.
About Blueprint Medicines
Blueprint Medicines is a global precision therapy company that invents life-changing therapies for people with cancer and hematologic 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
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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 genomically defined cancers, systemic mastocytosis, 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 Blueprint Medicines’ 2021 goals and anticipated milestones; plans, strategies, timelines and expectations for Blueprint Medicines’ current or future approved drugs and drug candidates, including timelines for marketing applications and approvals, the initiation of clinical trials or the results of ongoing and planned clinical trials; 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 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 establishing a commercial infrastructure, and successfully launching, marketing and selling current or future approved products, including AYVAKIT and GAVRETO; Blueprint Medicines’ ability to successfully expand the approved indications for AYVAKIT and GAVRETO or obtain marketing approval for AYVAKIT and GAVRETO 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 develop and commercialize companion diagnostic tests for its current and future drug candidates; and the success of Blueprint Medicines’ current and future collaborations, partnerships or licensing arrangements, including Blueprint Medicines’ global collaboration with Roche for the development and commercialization of GAVRETO. 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.
Trademarks
Blueprint Medicines, AYVAKIT, AYVAKYT, GAVRETO and associated logos are trademarks of Blueprint Medicines Corporation.
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Media Contact
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media@blueprintmedicines.com
Investor Contact
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ir@blueprintmedicines.com
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