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Blueprint Medicines Reports Third Quarter 2024 Results and Raises AYVAKIT®/AYVAKYT® (avapritinib) Full Year Revenue Guidance

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Blueprint Medicines (BPMC) reported strong Q3 2024 financial results, achieving $128.2 million in AYVAKIT net product revenues, representing 136% growth year-over-year. The company raised its full-year 2024 AYVAKIT revenue guidance to $475-480 million, up from previous guidance of $435-450 million. Q3 net loss was $56.3 million, improved from $133.7 million in Q3 2023. Cash position strengthened to $882.4 million as of September 30, 2024. The company expects declining operating expenses and cash burn in 2024 compared to 2023, while maintaining focus on long-term growth.

Blueprint Medicines (BPMC) ha riportato risultati finanziari solidi per il terzo trimestre del 2024, registrando $128.2 milioni di ricavi netti da prodotto AYVAKIT, con una crescita del 136% rispetto all'anno precedente. L'azienda ha alzato le stime sui ricavi annuali per AYVAKIT a $475-480 milioni, rispetto alle precedenti stime di $435-450 milioni. La perdita netta del Q3 è stata di $56.3 milioni, in miglioramento rispetto ai $133.7 milioni del Q3 2023. La posizione di liquidità è stata rafforzata a $882.4 milioni al 30 settembre 2024. L'azienda prevede una diminuzione delle spese operative e del burn rate di cassa nel 2024 rispetto al 2023, mantenendo comunque un focus sulla crescita a lungo termine.

Blueprint Medicines (BPMC) reportó resultados financieros sólidos para el tercer trimestre de 2024, alcanzando $128.2 millones en ingresos netos de productos AYVAKIT, lo que representa un crecimiento del 136% en comparación con el año anterior. La compañía elevó su guía de ingresos para AYVAKIT para todo el año 2024 a $475-480 millones, desde la guía anterior de $435-450 millones. La pérdida neta del Q3 fue de $56.3 millones, mejorando desde los $133.7 millones en el Q3 2023. La posición de efectivo se fortaleció a $882.4 millones al 30 de septiembre de 2024. La compañía espera una disminución de los gastos operativos y el consumo de efectivo en 2024 en comparación con 2023, manteniendo el enfoque en el crecimiento a largo plazo.

블루프린트 의약품 (BPMC)은 2024년 3분기 강력한 재무 결과를 보고했으며, 1억 2,820만 달러의 AYVAKIT 순 제품 수익을 달성하여 전년 대비 136% 성장했습니다. 회사는 2024년 AYVAKIT 연간 수익 가이던스를 4억 7,500-4억 8,000만 달러로 상향 조정했으며, 이는 이전의 $4억 3,500-4억 5,000만 달러에서 증가한 것입니다. 3분기 순손실은 5,630만 달러로, 2023년 3분기의 1억 3,370만 달러에서 개선되었습니다. 2024년 9월 30일 기준 현금 보유액은 8억 8,240만 달러로 강화되었습니다. 회사는 2023년에 비해 2024년에 운영 비용과 현금 소모가 감소할 것으로 예상하고 있으며, 장기 성장에 대한 집중을 유지하고 있습니다.

Blueprint Medicines (BPMC) a annoncé de bons résultats financiers pour le troisième trimestre 2024, avec des revenus nets de produit AYVAKIT s'élevant à 128,2 millions USD, représentant une croissance de 136 % par rapport à l'année précédente. La société a relevé ses prévisions de revenus pour AYVAKIT pour l'année entière 2024 à 475-480 millions USD, contre des prévisions précédentes de 435-450 millions USD. La perte nette du T3 s'élevait à 56,3 millions USD, en amélioration par rapport à 133,7 millions USD au T3 2023. La position de trésorerie a été renforcée à 882,4 millions USD au 30 septembre 2024. La société s'attend à une baisse des dépenses d'exploitation et de la consommation de trésorerie en 2024 par rapport à 2023, tout en maintenant son accent sur la croissance à long terme.

Blueprint Medicines (BPMC) meldete starke Finanzergebnisse für das 3. Quartal 2024 und erzielte 128,2 Millionen USD an Nettoverkaufserlösen für AYVAKIT, was einem Wachstum von 136 % im Vergleich zum Vorjahr entspricht. Das Unternehmen erhöhte seine Umsatzprognose für AYVAKIT auf 475-480 Millionen USD, nachdem die vorherige Prognose bei 435-450 Millionen USD lag. Der Nettoverlust im 3. Quartal betrug 56,3 Millionen USD, was eine Verbesserung gegenüber 133,7 Millionen USD im 3. Quartal 2023 darstellt. Die Liquiditätsposition wurde zum 30. September 2024 auf 882,4 Millionen USD gestärkt. Das Unternehmen erwartet im Vergleich zu 2023 sinkende Betriebskosten und Cash-Burn im Jahr 2024, während es weiterhin auf langfristiges Wachstum fokussiert bleibt.

Positive
  • AYVAKIT revenue grew 136% YoY to $128.2M in Q3 2024
  • Raised full-year revenue guidance to $475-480M from $435-450M
  • Net loss improved significantly to $56.3M from $133.7M YoY
  • Strong cash position of $882.4M, up from $767.2M at end of 2023
  • Expecting decreased operating expenses and cash burn in 2024
Negative
  • R&D expenses remained high at $85.3M despite decrease from prior year
  • SG&A expenses increased to $89.9M from $70.7M YoY
  • Company still operating at a net loss

Insights

Blueprint Medicines delivered exceptional Q3 results with $128.2M in AYVAKIT revenue, showing remarkable 136% year-over-year growth. The raised full-year guidance to $475-480M from $435-450M indicates strong commercial execution and market penetration. The 26% sequential quarterly growth demonstrates accelerating momentum.

Operating metrics show improved efficiency with R&D expenses down 22.7% YoY to $85.3M. While SG&A increased to support commercialization, the net loss narrowed significantly to $56.3M from $133.7M. With $882.4M in cash and growing revenue, the path to profitability is becoming clearer.

AYVAKIT's strong performance in treating indolent systemic mastocytosis (ISM) validates its disease-modifying potential. The expansion into both moderate and severe ISM patients broadens the addressable market. The advancement of BLU-808 for chronic urticaria represents a strategic expansion in the mast cell disorder space, targeting a larger patient population.

The upcoming HARBOR Part 2 study for elenestinib could further solidify Blueprint's leadership in mast cell disorders. The company's focused pipeline strategy, emphasizing high-value opportunities while maintaining cost discipline, positions it well for sustainable growth in the rare disease space.

-- Achieved $128.2 million in AYVAKIT net product revenues in the third quarter 2024 --

-- Raising AYVAKIT net product revenue guidance to $475 million to $480 million for 2024 --

--  On track to initiate the registration-enabling HARBOR Part 2 study of elenestinib in ISM by year end --

CAMBRIDGE, Mass., Oct. 30, 2024 /PRNewswire/ -- Blueprint Medicines Corporation (Nasdaq: BPMC) today reported financial results, provided a business update for the third quarter ended Sept 30, 2024, and provided updated financial guidance.

"With another strong quarter of AYVAKIT revenue performance, our year-to-date results provide the foundation to drive significant growth and long-term shareholder value creation in 2025 and beyond. AYVAKIT's sales momentum has given us the confidence to raise our revenue expectations again this year, estimating that we will end the year between $475 million and $480 million dollars. Ending our first full year of launch at nearly half a billion dollars in revenue positions AYVAKIT to be one of the most successful rare disease launches to-date," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "AYVAKIT has demonstrated tangible benefits to patients globally, with long-term data further reinforcing the clinically meaningful benefits and well-tolerated profile of a disease-modifying treatment. AYVAKIT's strong revenue ramp coupled with disciplined investment in our most compelling product opportunities, places Blueprint Medicines in a position to realize the significant decline in cash burn this year, while maintaining our focus on long-term growth and value creation."

Third Quarter 2024 Highlights and Recent Progress

Mast cell disorders

  • Achieved AYVAKIT net product revenues of $128.2 million for third quarter of 2024, including $113.1 million in the US and $15.1 million ex-US, representing 136 percent growth year-over-year.
  • Advanced the Phase 1 single ascending dose/multiple ascending dose healthy volunteer study of BLU-808, a highly selective and potent investigational oral wild-type KIT inhibitor, with best-in-class potential for chronic urticaria and other mast cell disorders. Data from the healthy volunteer study, including safety, pharmacokinetic and pharmacodynamic markers, are anticipated in early 2025.
  • Presented data at the American College of Allergy, Asthma and Immunology (ACAAI) conference demonstrating the efficacy of AYVAKIT in patients with moderate and severe indolent SM (ISM) at baseline, and showing the significant disease burden of SM, highlighting the urgency to diagnose and treat. Read the presentations here.
  • Hosting scientific seminar on mast cell franchise development with leading allergy and asthma expert Dr. Paul O'Byrne on Thursday, November 14, 2024.

2024 Financial Guidance

Blueprint Medicines now anticipates approximately $475 million to $480 million in global AYVAKIT net product revenues in 2024, an increase from the previous range of $435 million to $450 million. This updated guidance is based on continued growth in patients on therapy, continued favorability in compliance and other performance factors, and stronger than expected performance outside of the U.S. The company continues to expect that full-year operating expenses and cash burn will decline in 2024 as compared to 2023, and that its existing cash, cash equivalents and investments, together with anticipated product revenues, will enable the company to maintain a durable capital position to achieve a self-sustainable financial profile.

Key Upcoming Milestones

The company plans to achieve the following remaining milestones by the end of 2024:

Mast cell disorders

  • Initiate registration-enabling Part 2 of the HARBOR trial of elenestinib in ISM by the end of this year.

Cell cycle inhibition

  • Continue strategic business development discussions.
  • Complete Phase 1 combination dose escalation for BLU-222 by end of year to inform registration plans.

Third Quarter 2024 Results

  • Revenues: Revenues were $128.2 million for the third quarter of 2024 generated from the net product sales of AYVAKIT/AYVAKYT. Revenues were $56.6 million in the third quarter of 2023, including $54.2 million of net product revenues from sales of AYVAKIT/AYVAKYT and $2.4 million in collaboration, license and other revenues.
  • Cost of Sales: Cost of sales was $1.9 million for the third quarter of 2024, as compared to $2.8 million for the third quarter of 2023. The decrease was primarily due to lower sales to our collaboration partner in greater China.
  • R&D Expenses: Research and development expenses were $85.3 million for the third quarter of 2024, as compared to $110.3 million for the third quarter of 2023. This decrease was primarily due to operational efficiency across our portfolio as we execute across our top priority programs and the timing of manufacturing of clinical trial materials. Research and development expenses included $12.6 million in stock-based compensation expenses for the third quarter of 2024.
  • SG&A Expenses: Selling, general and administrative expenses were $89.9 million for the third quarter of 2024, as compared to $70.7 million for the third quarter of 2023. This increase was primarily due to an increase in activities supporting the commercialization of AYVAKIT/AYVAKYT. Selling, general, and administrative expenses included $15.7 million in stock-based compensation expenses for the third quarter of 2024.
  • Net Loss: Net loss was $56.3 million for the third quarter of 2024, as compared to a net loss of $133.7 million for the third quarter of 2023.
  • Cash Position: As of September 30, 2024, cash, cash equivalents and investments were $882.4 million, as compared to $767.2 million as of December 31, 2023. Blueprint Medicines' cash and investments provide a durable capital position which, together with anticipated product revenues, the company believes will enable it to reach a self-sustainable financial profile.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:00 a.m. ET today to discuss third quarter 2024 financial results and recent business activities. The conference call may be accessed by dialing 833-470-1428 (domestic) or 404-975-4839 (international) and referring conference ID 387547. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at http://ir.blueprintmedicines.com/. The archived webcast will be available on Blueprint Medicines' website approximately two hours after the conference call and will be available for 30 days following the call.

Upcoming Investor Conferences 

Blueprint Medicines will participate in three upcoming investor conferences:

  • Guggenheim Inaugural Healthcare Innovation Conference on Tuesday, November 12, 2024 at 3:30 p.m. ET.
  • Stifel 2024 Healthcare Conference on Monday, November 18, 2024 at 10:20 a.m. ET.
  • Jefferies London Healthcare Conference on Thursday, November 21, 2024 at 12:00 noon GMT.

Scientific Webinar Series

  • Blueprint Medicines will host the second event in its scientific seminar series, focused on mast cell diseases, on Thursday, November 14th at 10:00 a.m. ET.

A live webcast of the above presentations and any related slides will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at http://ir.blueprintmedicines.com. A replay of the webcasts will be archived on the Blueprint Medicines website following the events.

About Blueprint Medicines

Blueprint Medicines is a global, fully integrated biopharmaceutical company that invents life-changing medicines. We seek to alleviate human suffering by solving important medical problems in two core focus areas: allergy/inflammation and oncology/hematology. Our approach begins by targeting the root causes of disease, using deep scientific knowledge in our core focus areas and drug discovery expertise across multiple therapeutic modalities. We have a track record of success with two approved medicines, including AYVAKIT®/AYVAKYT® (avapritinib) which we are bringing to patients with systemic mastocytosis (SM) in the U.S. and Europe. Leveraging our established research, development, and commercial capability and infrastructure, we now aim to significantly scale our impact by advancing a broad pipeline of programs ranging from early science to advanced clinical trials in mast cell diseases including SM and chronic urticaria, breast cancer and other solid tumors. For more information, visit www.BlueprintMedicines.com and follow us on X (formerly 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, strategies, timelines and expectations for Blueprint Medicines' operations, including its expectations regarding growth and net product revenue in 2024; statements regarding its current or future approved drugs and drug candidates and operations, including plans to initiate registration-enabling Part 2 of the HARBOR trial of elenestinib in ISM in 2024, complete Phase 1 combination dose escalation for BLU-222 to inform registration plans and continue strategic business development discussions by the end of 2024, and present healthy volunteer data from its Phase 1 trial for BLU-808 in early 2025; expectations related to the markets for current or future approved drugs and drug candidates, including expectations regarding the size or scale of patient opportunities that future approved drugs and drug candidates could address; the potential benefits of any of its current or future approved drugs or drug candidates in treating patients; statements related to liquidity and capital position, including expectations that its cash, cash equivalents and investments will provide a durable capital position which, together with anticipated product revenues, will enable Blueprint Medicines to reach a self-sustainable financial profile; and its financial performance, strategy, goals and anticipated milestones, business plans and focus, including expectations regarding its revenue ramp and continued decline in operating expenses and cash burn. 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: the risk that the marketing and sale of AYVAKIT/ AYVAKYT or any future approved drugs may be unsuccessful or less successful than anticipated, or that AYVAKIT/ AYVAKYT may not gain market acceptance by physicians, patients, third-party payors and others in the medical community; the risk that the market opportunities for AYVAKIT/ AYVAKYT or Blueprint Medicines' drug candidates are smaller than we estimate or that any approval we obtain may be based on a narrower definition of the patient population that we anticipate; the risk of delay of any current or planned clinical trials or the development of Blueprint Medicines' current or future drug candidates; risks related to 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; preclinical and clinical results for Blueprint Medicines' drug candidates 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 may be delayed or slower than anticipated; actions of regulatory agencies may affect its approved drugs or its current or future drug candidates, including affecting the initiation, timing and progress of clinical trials; risks related to Blueprint Medicines' ability to obtain, maintain and enforce patent and other intellectual property protection for its products and current or future drug candidates it is developing; the success of Blueprint Medicines' current and future collaborations, financing arrangements, partnerships or licensing and other arrangements; risks related to its liquidity and financial position, including the risk that Blueprint Medicines may be unable to generate sufficient future product revenues to achieve and maintain a self-sustainable financial profile; and the accuracy of its estimates of revenues, expenses and capital requirements. 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 and associated logos are trademarks of Blueprint Medicines Corporation.

Blueprint Medicines Corporation

Selected Condensed Consolidated Balance Sheet Data

(in thousands)

(unaudited)









September 30,


December 31,



2024


2023

Cash, cash equivalents and investments

$

882,353


$

767,171

Working capital (1)


597,187



593,470

Total assets


1,199,649



1,049,250

Deferred revenue (2)


12,034



5,604

Liability related to the sale of future royalties and revenues (2)


261,207



441,625

Term loan (2)


386,569



238,813

Total liabilities


886,501



918,641

Total stockholders' equity


313,148



130,609


(1)   Blueprint Medicines defines working capital as current assets less current liabilities.

(2)   Includes both current and long-term portions of the balance.

 

Blueprint Medicines Corporation

Condensed Consolidated Statements of Operations Data

(in thousands, except per share data)

(unaudited)




Three Months Ended


Nine Months Ended



September 30,


September 30,



2024


2023


2024


2023

Revenues:













Product revenue, net


$

128,184


$

54,228


$

334,825


$

133,173

Collaboration, license and other revenue





2,338



27,633



44,250

Total revenues



128,184



56,566



362,458



177,423

Cost and operating expenses:













Cost of sales



1,932



2,782



12,716



8,280

Collaboration loss sharing





1,771





4,301

Research and development



85,300



110,252



257,761



330,184

Selling, general and administrative



89,926



70,741



262,822



215,826

Total cost and operating expenses



177,158



185,546



533,299



558,591

Other income (expense):













Interest expense, net



(7,616)



(3,808)



(20,376)



(13,624)

Other income (expense), net



587



(728)



962



(369)

Debt extinguishment gain







173,676



Total other income (expense), net



(7,029)



(4,536)



154,262



(13,993)

Loss before income taxes



(56,003)



(133,516)



(16,579)



(395,161)

Income tax expense



273



197



554



907

Net Loss


$

(56,276)


$

(133,713)


$

(17,133)


$

(396,068)

Net Loss per share — basic and diluted


$

(0.89)


$

(2.20)


$

(0.27)


$

(6.55)

Weighted-average number of common
shares used in net loss per share —
basic and diluted



63,381



60,688



62,608



60,445

 

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SOURCE Blueprint Medicines Corporation

FAQ

What was Blueprint Medicines (BPMC) Q3 2024 revenue?

Blueprint Medicines reported $128.2 million in AYVAKIT net product revenues for Q3 2024, with $113.1 million from US sales and $15.1 million from ex-US sales.

What is BPMC's updated revenue guidance for 2024?

Blueprint Medicines raised its full-year 2024 AYVAKIT revenue guidance to $475-480 million, increased from the previous guidance of $435-450 million.

What was Blueprint Medicines' net loss in Q3 2024?

Blueprint Medicines reported a net loss of $56.3 million for Q3 2024, improved from a net loss of $133.7 million in Q3 2023.

How much cash does BPMC have as of Q3 2024?

As of September 30, 2024, Blueprint Medicines had $882.4 million in cash, cash equivalents and investments.

Blueprint Medicines Corporation

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