Blueprint Medicines Outlines 2027 Blueprint to Achieve Precision Medicine at Scale and Reports Third Quarter 2022 Financial Results at Investor Day 2022 - Yahoo Finance

-- On way to taxable supplemental caller cause exertion for AYVAKIT® (avapritinib) successful non-advanced SM successful Q4 2022, with an anticipated U.S. motorboat successful mid-2023 --

-- Reports updated information from the SYMPHONY proceedings of BLU-945 demonstrating objective enactment and a differentiated information profile, supporting operation improvement successful first-line EGFR-mutant non-small compartment lung crab --

-- Expects to execute the high-end of full gross guidance of $180 million to $200 million for full-year 2022 --

-- Blueprint Medicines to webcast Investor Day 2022 lawsuit contiguous astatine 8:30 a.m. ET --

, /PRNewswire/ -- Blueprint Medicines (NASDAQ: BPMC) contiguous announced its 2027 Blueprint to execute precision medicine astatine scale, a five-year concern strategy to grow the company's scope to broader diligent populations by leveraging its technological leadership, proven improvement capableness and integrated business. The institution plans to item this concern strategy, including commercialized plans to bring AYVAKIT to patients with non-advanced systemic mastocytosis (SM), astatine its Investor Day lawsuit today. In addition, Blueprint Medicines contiguous reported fiscal results and provided a concern update for the 3rd 4th ended September 30, 2022.

"The accidental to bring AYVAKIT to patients with non-advanced SM, based connected the affirmative results of the PIONEER study, volition alteration america to standard our interaction and code the needs of a importantly larger diligent colonisation successful the adjacent term," said Kate Haviland, Chief Executive Officer of Blueprint Medicines. "This greeting astatine our Investor Day, we volition item our 2027 Blueprint strategy to treble our interaction successful 5 years crossed aggregate metrics of portfolio strength. We program to execute this standard with the imaginable motorboat of AYVAKIT successful non-advanced SM, aggregate advancing objective improvement programs for EGFR-mutant lung crab and CDK2-vulnerable bosom cancer, and an expansive precision therapy probe pipeline, each of which physique connected our R&D achievements to day and leverage our afloat integrated planetary infrastructure."

"In addition, contiguous we are reaffirming full gross guidance for full-year 2022, portion we little merchandise gross guidance based connected show successful the 3rd 4th and our near-term expectations for growth. Over the past year, we person established AYVAKIT arsenic the modular of attraction successful patients who are being actively treated for their precocious SM, and we are present focusing connected expanding attraction rates successful patients with SM and an associated hematologic neoplasm, wherever determination has been little adoption. In parallel, we are prioritizing efforts to bring AYVAKIT to patients with non-advanced SM, which, if approved, would correspond a 15-fold oregon larger accidental based connected the fig of patients with moderate-to-severe non-advanced SM who are diagnosed, being treated for their SM, and observable successful U.S. claims information today."

2027 Blueprint Global Business Strategy

Building connected Blueprint Medicines' important achievements implicit the past decade, including the regulatory support of 2 internally discovered precision therapies wrong the company's archetypal decade, the five-year 2027 Blueprint strategy aims to treble the company's interaction crossed aggregate measures of portfolio spot successful astir fractional the time.

Blueprint Medicines aims to execute the pursuing by the extremity of 2027:

• Products: 4+ marketed products for oncology, hematology, oregon mast compartment disorders (versus 2 today)

• Portfolio: 3+ illness enactment areas (versus 1, mast compartment disease, today)

• Clinical: 4+ late-stage objective programs (versus 2 today)

• Research: 25+ cumulative improvement candidates nominated (versus 14 today) derived from 2 probe platforms (versus 1, kinase inhibitor platform, today)

Investor Day Presentation Highlights

At the Investor Day event, Blueprint Medicines plans to:

• Review the go-to-market program for AYVAKIT successful non-advanced SM and facilitate a sheet treatment with illness experts connected perceptions of the registration-enabling PIONEER proceedings data, the existent authorities of SM care, and the imaginable relation of a caller disease-modifying therapy. Participants volition include:

• Highlight objective advancement informing improvement strategies for the company's EGFR portfolio therapies, including updated Phase 1/2 SYMPHONY proceedings dose escalation information supporting plans to prioritize improvement of BLU-945 successful operation with osimertinib successful first-line EGFR L858R-positive non-small compartment lung crab (NSCLC).

• Introduce a caller probe programme targeting wild-type KIT, which aims to physique connected the company's KIT people enactment to beforehand a best-in-class oral precision therapy for communal mast compartment diseases adjacent to systemic mastocytosis, including chronic urticaria.

Third Quarter 2022 Highlights and Recent Progress

AYVAKIT®/AYVAKYT® (avapritinib): SM and PDGFRA gastrointestinal stromal tumor

• Reported planetary nett merchandise revenues of $28.6 million for the 3rd 4th of 2022.

• Announced affirmative top-line results from the registration-enabling Part 2 of the PIONEER proceedings of AYVAKIT successful patients with non-advanced SM, demonstrating clinically meaningful and highly important improvements crossed the superior and each cardinal secondary endpoints, including patient-reported symptoms and nonsubjective measures of mast compartment burden. AYVAKIT had a favorable information illustration compared to the power arm, supporting the imaginable for semipermanent treatment. Read the property merchandise here.

• Published results from the TouchStone survey of diligent and healthcare supplier perceptions of SM illness burden, which highlighted that SM is associated with terrible and burdensome symptoms including anaphylactic events, predominant exigency section visits, usage of aggregate symptom-directed medications, reduced quality to work, impaired carnal functioning and mediocre prime of life. Read the property merchandise here.

GAVRETO® (pralsetinib): RET-altered cancers

• As antecedently recorded and reported by Roche, GAVRETO planetary merchandise income were 20 cardinal CHF twelvemonth to day which excludes income successful the Greater China territory driven by CStone Pharmaceuticals.

BLU-945, BLU-701, BLU-525, and BLU-451: EGFR-mutant NSCLC

• Based connected emerging objective and preclinical data, Blueprint Medicines plans to prioritize improvement of BLU-525, a back-up EGFR inhibitor candidate, and deprioritize further improvement of BLU-701. Compared to BLU-701, BLU-525 has a chiseled chemic operation with improved kinome selectivity and differentiated metabolism, and equivalent EGFR mutation coverage, wild-type EGFR selectivity, and cardinal tense strategy penetration. The institution plans to taxable an investigational caller cause exertion to the U.S. Food and Drug Administration (FDA) for BLU-525 successful the archetypal 4th of 2023.

• Blueprint Medicines, with Guardant Health, presented real-world information from patients with EGFR-mutant NSCLC identifying the EGFR C797X mutation arsenic the astir communal absorption mechanics to osimertinib astatine the International Association for the Study of Lung Cancer 2022 World Conference connected Lung Cancer. Read the property merchandise here.

Key Upcoming Milestones

Blueprint Medicines expects to execute the pursuing near-term milestones:

• Submit a supplemental New Drug Application to the FDA for AYVAKIT for non-advanced SM successful the 4th fourth of 2022.

• Present elaborate information from the registration-enabling Part 2 of the PIONEER proceedings of AYVAKIT successful non-advanced SM astatine a aesculapian legislature successful precocious 2022 oregon aboriginal 2023.

• Report top-line 12-week information from Part 1 of the HARBOR proceedings of BLU-263 successful non-advanced SM successful the 4th fourth of 2022.

• Submit an IND to the FDA for BLU-525 for the attraction of EGFR-mutant NSCLC successful the archetypal 4th of 2023.

• Present archetypal information from the Phase 1/2 CONCERTO survey of BLU-451 successful patients with EGFR-mutant NSCLC successful the archetypal fractional of 2023.

• Present archetypal information from the Phase 1/2 VELA proceedings of BLU-222 successful CDK2-vulnerable cancers successful the archetypal fractional of 2023.

Third Quarter 2022 Results

• Revenues: Revenues were $66.0 million for the 3rd 4th of 2022, including $28.6 million of nett merchandise revenues from income of AYVAKIT/AYVAKYT, $9.8 million in collaboration and licence revenues, and $27.5 million successful licence revenues- related party. Blueprint Medicines recorded revenues of $24.2 million in the 3rd 4th of 2021, including $17.3 million of nett merchandise revenues from income of AYVAKIT/AYVAKIT and $6.9 million in collaboration and licence revenues.

• Cost of Sales: Cost of income was $3.0 cardinal for the 3rd 4th of 2022, arsenic compared to $3.8 cardinal for the 3rd 4th of 2021.

• R&D Expenses: Research and improvement expenses were $128.0 cardinal for the 3rd 4th of 2022, arsenic compared to $84.4 cardinal for the 3rd 4th of 2021. This summation was chiefly owed to increased objective proviso manufacturing and objective improvement activities owed to the progression and enlargement of our objective trials and accrued costs related to aboriginal find effort. Research and improvement expenses included $10.0 cardinal successful stock-based compensation expenses for the 3rd 4th of 2022.

• SG&A Expenses: Selling, wide and administrative expenses were $57.6 cardinal for the 3rd 4th of 2022, arsenic compared to $49.8 cardinal for the 3rd 4th of 2021. This summation was chiefly owed to accrued costs associated with expanding our commercialized infrastructure for commercialization of AYVAKIT/AYVAKYT. General and administrative expenses included $14.1 cardinal successful stock-based compensation expenses for the 3rd 4th of 2022.

• Net Loss: Net nonaccomplishment was $133.2 cardinal for the 3rd 4th of 2022, oregon a nett nonaccomplishment per stock of $2.23, arsenic compared to a nett nonaccomplishment of $117.2 cardinal for the 3rd 4th of 2021, oregon a nett nonaccomplishment per stock of $2.00.

• Cash Position: As of September 30, 2022, cash, currency equivalents and investments were $1,192.6 million, arsenic compared to $1,034.6 cardinal arsenic of December 31, 2021.

Financial Guidance

Blueprint Medicines anticipates it volition execute the precocious extremity of antecedently provided gross guidance for full-year 2022 of astir $180 million to $200 million, including astir $108 million to $111 million successful AYVAKIT nett merchandise revenues. The institution continues to expect that its existing cash, currency equivalents and investments, unneurotic with anticipated aboriginal merchandise revenues, volition supply capable superior to alteration the institution to execute a self-sustainable fiscal profile.

Conference Call Information

Blueprint Medicines will big a unrecorded league telephone and webcast at 8:30 a.m. ET today for Investor Day and to sermon 3rd 4th 2022 fiscal results. The unrecorded webcast of the lawsuit volition beryllium disposable nether "Events and Presentations" successful the Investors & Media conception of Blueprint Medicines' website astatine http://ir.blueprintmedicines.com/. A replay of the webcast volition beryllium archived connected Blueprint Medicines' website for 90 days pursuing the event.

About Blueprint Medicines

Blueprint Medicines is simply a planetary precision therapy institution that invents life-changing therapies for radical with crab and humor disorders. Applying an attack that is some precise and agile, we make medicines that selectively people familial drivers, with the extremity of staying 1 measurement up crossed stages of disease. Since 2011, we person leveraged our probe platform, including expertise successful molecular targeting and world-class cause plan capabilities, to rapidly and reproducibly construe subject into a wide pipeline of precision therapies. Today, we are delivering approved medicines straight to patients successful the United States and Europe, and we are globally advancing aggregate programs for systemic mastocytosis, lung crab and different genomically defined cancers, and crab immunotherapy. For much information, sojourn www.BlueprintMedicines.com and travel america connected Twitter (@BlueprintMeds) and LinkedIn.

Cautionary Note Regarding Forward-Looking Statements

This property merchandise contains forward-looking statements wrong the meaning of the Private Securities Litigation Reform Act of 1995, arsenic amended, including, without limitation, statements regarding plans, strategies, timelines and expectations for interactions with the FDA and different regulatory authorities; to taxable a supplemental New Drug Application to the FDA for AYVAKIT successful non-advanced SM, with a consequent submission of a benignant II saltation selling authorization exertion to the European Medicines Agency; plans and timing for presenting elaborate information from the PIONEER proceedings of AYVAKIT successful patients with non-advanced SM, and, expectations regarding the imaginable benefits of AYVAKIT successful treating patients with non-advanced SM; statements regarding plans and expectations for Blueprint Medicines' existent oregon aboriginal approved drugs and cause candidates; the imaginable benefits of immoderate of Blueprint Medicines' existent oregon aboriginal approved drugs oregon cause candidates successful treating patients; and Blueprint Medicines' financial performance, strategy, goals and anticipated milestones, concern plans and focus. The words "aim," "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target" and akin expressions are intended to place forward-looking statements, though not each forward-looking statements incorporate these identifying words. Any forward-looking statements successful this property merchandise are based connected management's existent expectations and beliefs and are taxable to a fig of risks, uncertainties and important factors that whitethorn origin existent events oregon results to disagree materially from those expressed oregon implied by immoderate forward-looking statements contained successful this property release, including, without limitation, risks and uncertainties related to the interaction of the COVID-19 pandemic to Blueprint Medicines' business, operations, strategy, goals and anticipated milestones, including Blueprint Medicines' ongoing and planned probe and find activities, quality to behaviour ongoing and planned objective trials, objective proviso of existent oregon aboriginal cause candidates, commercialized proviso of existent oregon aboriginal approved products, and launching, selling and selling existent oregon aboriginal approved products; Blueprint Medicines' quality and plans successful continuing to found and grow a commercialized infrastructure, and successfully launching, selling and selling existent oregon aboriginal approved products; Blueprint Medicines' quality to successfully grow the approved indications for AYVAKIT/AYVAKYT and GAVRETO oregon get selling support for AYVAKIT/AYVAKYT successful further geographies successful the future; the hold of immoderate existent oregon planned objective trials oregon the improvement of Blueprint Medicines' existent oregon aboriginal cause candidates; Blueprint Medicines' advancement of aggregate early-stage efforts; Blueprint Medicines' quality to successfully show the information and efficacy of its cause candidates and summation support of its cause candidates connected a timely basis, if astatine all; the preclinical and objective results for Blueprint Medicines' cause candidates, which whitethorn not enactment further improvement of specified cause candidates either arsenic monotherapies oregon successful operation with different agents oregon whitethorn interaction the anticipated timing of information oregon regulatory submissions; the timing of the initiation of objective trials and proceedings cohorts astatine objective proceedings sites and diligent enrollment rates; actions of regulatory agencies, which whitethorn impact the initiation, timing and advancement of objective trials; Blueprint Medicines' quality to obtain, support and enforce patent and different intelligence spot extortion for AYVAKIT/AYVAKYT, GAVRETO oregon immoderate cause candidates it is developing; Blueprint Medicines' quality to make and commercialize companion diagnostic tests for AYVAKIT/AYVAKYT, GAVRETO oregon immoderate of its existent and aboriginal cause candidates; Blueprint Medicines' ability to successfully grow its operations, probe level and portfolio of therapeutic candidates, and the timing and costs thereof; Blueprint Medicines' quality to recognize the anticipated benefits of its enforcement enactment modulation plan; and the occurrence of Blueprint Medicines' existent and aboriginal collaborations, financing arrangements, partnerships oregon licensing arrangements. These and different risks and uncertainties are described successful greater item successful the conception entitled "Risk Factors" successful Blueprint Medicines' filings with the Securities and Exchange Commission (SEC), including Blueprint Medicines' astir caller Annual Report connected Form 10-K, arsenic supplemented by its astir caller Quarterly Report connected Form 10-Q and immoderate different filings that Blueprint Medicines has made oregon whitethorn marque with the SEC successful the future. Any forward-looking statements contained successful this property merchandise correspond Blueprint Medicines' views lone arsenic of the day hereof and should not beryllium relied upon arsenic representing its views arsenic of immoderate consequent date. Except arsenic required by law, Blueprint Medicines explicitly disclaims immoderate work to update immoderate forward-looking statements.

Trademarks

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

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SOURCE Blueprint Medicines Corporation