Blueprint Medicines : 2024 Annual Report (a8aebb)

Published: 2025-04-24 16:57:06 ET BPMC

2024

Annual Report

Turning innovation into impact

Nasdaq: BPMC

Clouds Will Pass by PC Elliott

Acrylic on Board, Inspired by Sue, living with SM

BLUEPRINT MEDICINES 2024 ANNUAL REPORT

Kate Haviland

President, Chief Executive

Officer and Board Director

AYVAKIT is one of the most

successful rare disease

launches to date, supported

by a compelling benefit- risk profile and our proven commercial capability.

Dear Stockholders,

2024 was a remarkable year of achievement for Blueprint Medicines in our continued effort to drive growth and innovation with operational excellence.

Last year, we significantly advanced the launch of AYVAKIT®/ AYVAKYT® (avapritinib), as we brought this important medicine to many more patients with systemic mastocytosis (SM) and achieved global net product revenues of $479 million, representing approximately 135 percent year-over-year growth. Today, AYVAKIT is one of the most successful rare disease launches to date, supported by a compelling benefit-risk profile and our proven commercial capability. AYVAKIT is now approved and reimbursed for at least one indication in 16 countries, with plans for additional country launches in indolent SM (ISM). In 2025, we expect to drive further AYVAKIT growth, with product revenue guidance of $680- $710 million, on path to achieve our goal of $2 billion in revenue by 2030.

REVENUE

135%

$479

GROWTH

$, MILLIONS

$204

$111

$53

2021

2022

2023

2024

The SM opportunity is larger than we first thought and growing faster than we expected

As the first therapy approved to treat ISM, AYVAKIT is proving to be a catalyst for diagnosis, treatment and coordination of patient care in this new rare disease market. In fact, over the last five years, we have seen a compound annual growth rate of 25 percent in the number of diagnosed SM patients observable in U.S. claims data, with more than 25,000 diagnosed patients in the U.S. today. Combined with recently published independent epidemiology data showing SM prevalence may be double than prior estimates, we believe there is a $4 billion peak opportunity for our SM franchise.

And we have made important progress in advancing our franchise lifecycle strategy. In 2024, we initiated the registration-directed Phase 3 HARBOR trial of elenestinib, our next-generation KIT D816V inhibitor. Through strategic design of the HARBOR trial, we expect to drive further innovation in the treatment of ISM, with novel endpoints assessing bone health, anaphylaxis and markers of inflammation designed with input from the U.S. Food and Drug Administration (FDA). With our approach, we expect to clinically differentiate elenestinib, raising the bar from symptom control to disease modification, and deliver significant value to the growing SM market for many years into the future.

MEDICINES 2024 ANNUAL REPORT

LARGE, GROWING MARKET OPPORTUNITY

GLOBAL SM REVENUE POTENTIAL

Increase in diagnosed SM patients

$4B

~25K patients observable in U.S. claims data1

Widening lens on who is an AYVAKIT patient

Supported by growing body of long-term safety & efficacy data

estimated SM franchise

Higher estimated SM prevalence

peak revenue

2x prior estimate, based on recent large independent study2

BLUEPRINT

OBSERVABLE SM PATIENTS IN U.S. CLAIMS DATA1

~25k

2019

2020

2021

2022

2023

2024

$2B

estimated annual AYVAKIT

revenue by 2030

1 Internal Blueprint Medicines analyses of U.S. claims data (as of 2024). 2 Bergstrom et al; Acta Oncologica (2024).

BLU-808, our next potential driver of significant growth

In 2024, we also advanced our broader mast cell therapy portfolio, with the rapid completion of a Phase 1 healthy volunteer study of BLU-808, our oral, highly potent and selective wild-type KIT inhibitor. We designed BLU-808 to enable a differentiated clinical profile, with tunable dosing that can dial-in clinical activity and manage on-target adverse events to optimize a favorable benefit-risk profile that supports chronic treatment.

UNIVERSE OF ALLERGIC &

INFLAMMATORY DISEASES

Skin

• Chronic urticaria

• Psoriasis

• Atopic dermatitis

Respiratory

• Allergic asthma

• Allergic rhinitis

• Allergic conjunctivitis

• Nasal polyps

• COPD

Gastrointestinal

• Eosinophilic disorders

• Irritable bowel syndrome

• Food allergy

Multi-system

• Mast cell activation

syndrome (MCAS)

PLUS OTHERS...

Supporting this approach, BLU-808 healthy volunteer data presented early this year showed BLU-808 was well- tolerated, had dose-dependent pharmacokinetics with multiple doses providing clear target coverage, and rapid, robust and sustained reductions in serum tryptase,

a pharmacodynamic marker of mast cell activity. Based on these data, we are now initiating multiple clinical proof- of-concept trials to further characterize the broad potential of BLU-808 across multiple allergic and inflammatory diseases.

BLU-808 PROOF-OF-CONCEPT STRATEGY

Move rapidly into areas where targeting KIT has been de-risked

Explore other biology across organ systems to unlock broader potential

BLUEPRINT MEDICINES 2024 ANNUAL REPORT

Continued disciplined financial stewardship

In 2024, we continued to prioritize investment in our most compelling programs with discipline. As we drove significant AYVAKIT revenue growth and advanced our pipeline of promising mast cell therapies, we also reduced our operating cash burn by more than 50 percent, and we expect further reductions in 2025. We ended the year with approximately $863.9 million in cash, with an additional $78.7 million received in the first quarter of 2025 from proceeds of our equity stake in IDRx, which was acquired by GSK. Together, we expect our disciplined approach to capital allocation and durable cash position to enable us to continue to sustainably invest in strategic growth opportunities.

Achievement fueled by a talented team and an award-winning culture

Blueprint Medicines was again recognized as an employer-of-choice in 2024. We were named to USA Today's "Top Places to Work" list; to The Boston Globe's "Top Places to Work in Massachusetts" list; as a Fortune "Future 50" honoree; and as a top employer in Switzerland by Top Employers Institute. Our achievements are a testament to the hard work and dedication of our almost 650 team members and I thank each of them for their unwavering commitment to the patients and communities we serve.

Today our future is brighter than ever before. We have the right people, portfolio, capabilities and vision to bring forward medicines that solve important medical challenges, dramatically improve patient lives and create significant value for our stockholders in 2025 and beyond.

On behalf of our board of directors, team members, patients and healthcare partners, we thank you for your continued support and investment in Blueprint Medicines.

Sincerely,

Kate Haviland

President, Chief Executive Officer and Board Director

On Behalf of the Board of Directors

Cambridge, Massachusetts

April 24, 2025

2024 ANNUAL REPORT

Colors of SM - From Experience and Expression to Innovation and Impact

BLUEPRINT MEDICINES

Sue and PC Elliott at Colors of SM inaugural event

It's often said a blank canvas holds unlimited possibilities and a picture is worth a thousand words. But for people living with SM, words often fall short. The life-altering symptoms - rashes, bone pain, diarrhea, brain fog and more - aren't just chronic; they are unpredictable and unrelenting disruptions that profoundly impact people's quality of life. And yet, because these experiences can be so difficult to describe, too often they remain unheard and misunderstood.

At Blueprint Medicines, we believe to truly advance care, every patient's story must be seen, shared and understood. Throughout 2024, we partnered with Twist Out Cancer - a non-profit organization dedicated to supporting individuals touched by diseases through creative arts, fostering healing, connection and resilience. Together, we launched a first-of-its-kind program Colors of SM: Expressions of Life with Systemic Mastocytosis, designed to bring visibility, validation and voice to people living with ISM through art and storytelling, creating connection and driving education and awareness to shorten the diagnostic odyssey and improve quality of care.

Each participant - the Inspiration - was paired with a volunteer artist. Over six months, the Inspiration shared their journey: the symptoms they endure, the road to diagnosis and what it truly means to live with SM. In turn, the artist interpreted their story into a one-of-a-kind work of art reflecting the Inspiration's experience, resilience and hope.

"The road to my diagnosis with SM has been

lifelong. After a lifetime of on-again, off-again health problems, personal battles and my stubborn refusal to stop looking for answers, I was formally diagnosed in 2020. My 'twist' on SM is to be the voice of encouragement for the person who is still struggling

to find a diagnosis! If I had stopped pushing for an

answer at every closed door, I wouldn't be here today."

- Sue, Inspiration living with SM

For more than 14 years, our teams have listened to and learned from the mast cell disease community

At Blueprint Medicines, our mission is to alleviate human suffering by solving serious medical challenges through the development of life-changing medicines. Our work in SM is a clear example

of this mission in action. Programs like Colors of SM demonstrate our ongoing commitment to elevating patient voices and building a community of belonging, where those impacted by mast cell disease feel seen, heard and empowered.

As we look ahead, we remain steadfast in translating insights into innovation. What we've learned from SM is guiding our efforts across a broader spectrum of allergic and inflammatory diseases - expanding the impact of our science while staying rooted in the experiences of the patients we serve. Every step forward is driven by a shared goal: to bring forward life-changing therapies that improve lives and reflect the needs of those at the heart of it all.

To learn more about the lives of people affected by SM, visit ColorsofSM.com

More artwork and stories to be revealed in 2025.

BLUEPRINT MEDICINES 2024 ANNUAL REPORT

Blueprint Medicines Pipeline

Mast cell disorders

DISCOVERY

EARLY STAGE

LATE STAGE

COMMERCIAL

DEVELOPMENT

AYVAKIT® (avapritinib): KIT D816V

» Indolent SM1,2

» Advanced SM1,3

Elenestinib (next gen): KIT D816V » Indolent SM

BLU-808: Wild-type KIT » Mast cell disorders

Undisclosed mast cell targets/ modalities

Solid tumors

AYVAKIT (avapritinib): PDGFRA

» PDGFRA GIST1,4

BLU-956 (next gen): CDK2

Targeted protein degrader: CDK2

Targeted protein degrader: CDK4 » HR+, HER2- breast cancer

Additional research

Programs undisclosed

Updated as of February 13, 2025

1CStone Pharmaceuticals has exclusive rights to develop and commercialize avapritinib in Greater China.

2Approved in the U.S. for adults with indolent SM. Approved in Europe (AYVAKYT®) for adults with indolent SM with moderate to severe symptoms inadequately controlled on symptomatic treatment.

3Approved in the U.S. for adults with advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). Approved in Europe (AYVAKYT) for adults with ASM, SM-AHN or MCL, after at least one systemic therapy.

4Approved in the U.S. for adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Approved in Europe (AYVAKYT) for adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.

GIST = gastrointestinal stromal tumors. SM = systemic mastocytosis.

Blueprint Medicines, AYVAKIT, AYVAKYT and associated logos are trademarks of Blueprint Medicines Corporation.

Not for promotional use.

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, DC 20549

Form 10-K

(Mark One)

For the transition period from

Commission File Number: 001-37359

BLUEPRINT MEDICINES CORPORATION

(Exact name of registrant as specified in its charter)

Delaware

26-3632015

(State or other jurisdiction of

(IRS Employer

incorporation or organization)

Identification No.)

45 Sidney Street

Cambridge, MA

02139

(Address of principal executive offices)

(Zip Code)

Registrant's telephone number, including area code: (617) 374-7580

Securities registered pursuant to Section 12(b) of the Act:

Title of Class

Trading Symbols

Name of Exchange on Which Registered

Common Stock, par value $0.001 per share

BPMC

Nasdaq Global Select Market

Securities registered pursuant to Section 12(g) of the Act: None

☐

Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes ☑

Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. Yes ☐

☑

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.

Yes ☑ No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☑ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See definitions of "large accelerated filer," "accelerated filer," "smaller reporting company" and "emerging growth company" in Rule 12b-2 of the Exchange Act.

Large accelerated filer

☑

Accelerated filer ☐

Non-accelerated filer

☐

Smaller reporting company ☐

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Indicate by check mark whether the registrant has filed a report on and attestation to its management's assessment of the effectiveness of its internal control over financial reporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the registered public accounting firm that prepared or issued its audit report.

Yes ☑ No ☐

If securities are registered pursuant to Section 12(b) of the Act, indicate by check mark whether the financial statements of the registrant included in the filing reflect the correction of an error to previously issued financial statements. ☐

Indicate by check mark whether any of those error corrections are restatements that required a recovery analysis of incentive-based compensation received by any of the registrant's executive officers during the relevant recovery period pursuant to §240.10D-1(b). ☐

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Act). Yes ☐ No ☑

As of June 30, 2024, the aggregate market value of the voting and non-voting common equity held by non-affiliates of the registrant, based on the last reported sales price for the registrant's common stock, par value $0.001 per share, on the Nasdaq Global Select Market on such date, was approximately $6,747,459,120.

Number of shares of the registrant's common stock, par value $0.001 per share, outstanding on February 11, 2025: 63,906,011

DOCUMENTS INCORPORATED BY REFERENCE

Portions of the registrant's definitive proxy statement for its 2025 Annual Meeting of Stockholders, which the registrant intends to file with the Securities and Exchange Commission pursuant to Regulation 14A within 120 days after the end of the registrant's fiscal year ended December 31, 2024, are incorporated by reference into Part III of this Annual Report on Form 10-K.

Auditor Firm Id:

Auditor Name:

Ernst & Young LLP

Auditor Location:

Boston, Massachusetts, United States

TABLE OF CONTENTS

PART I

Page

Item 1.

Business

Item1A.

Risk Factors

Item 1B.

Unresolved Staff Comments

Item 1C.

Cybersecurity

Item 2.

Properties

Item 3.

Legal Proceedings

Item 4.

Mine Safety Disclosures

PART II

Item 5.

Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of

Equity Securities

Item 6.

Reserved

Item 7.

Management's Discussion and Analysis of Financial Condition and Results of Operations

Item 7A.

Quantitative and Qualitative Disclosures About Market Risk

108

Item 8.

Financial Statements and Supplementary Data

109

Item 9.

Change in and Disagreements with Accountants on Accounting and Financial Disclosure

109

Item 9A.

Controls and Procedures

109

Item 9B.

Other Information

111

Item 9C.

Disclosure Regarding Foreign Jurisdictions that Prevent Inspections

112

PART III

Item 10.

Directors, Executive Officers and Corporate Governance

112

Item 11.

Executive Compensation

112

Item 12.

Security Ownership of Certain Beneficial Owners and Management and Related Stockholder

Matters

112

Item 13.

Certain Relationships and Related Transactions, and Director Independence

112

Item 14.

Principal Accounting Fees and Services

112

PART IV

Item 15.

Exhibits and Financial Statement Schedules

113

Item 16.

Form 10-K Summary

122

Unless otherwise stated, all references to "us," "our," "Blueprint," "Blueprint Medicines," "we," the

"Company" and similar designations in this Annual Report on Form 10-K refer to Blueprint Medicines Corporation and its consolidated subsidiaries. Blueprint Medicines, AYVAKIT®, AYVAKYT® and associated logos are trademarks of Blueprint Medicines Corporation. Other brands, names and trademarks contained in this Annual Report on Form 10-K are the property of their respective owners.

RISK FACTOR SUMMARY

Below is a summary of the material risks to our business, operations and the investment in our common stock. This summary does not address all of the risks that we face. Risks and uncertainties not presently known to us or that we presently deem less significant may also impair our business operations. Additional discussion of the risks summarized in this risk factor summary, and other risks that we face, can be found below under the heading "Risk Factors" and should be carefully considered, together with other information in this Annual Report on Form 10-K in its entirety before making investment decisions regarding our common stock.

receives accelerated approval. In addition, our current or future approved drugs could be subject to labeling and other restrictions and market withdrawal and we may be subject to penalties if we fail to comply with regulatory requirements or experience unanticipated problems with our drugs.

Disclaimer

Blueprint Medicines Corporation published this content on April 23, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on April 24, 2025 at 20:55 UTC.