Profile

Blueprint Medicines is a global, fully integrated biopharmaceutical company that invents life-changing medicines. The company seeks to alleviate human suffering by solving important medical problems in two core focus areas: allergy/inflammation and oncology/hematology. Their approach begins by targeting the root causes of disease, using deep scientific knowledge in their core focus areas and drug discovery expertise across multiple therapeutic modalities. They have a track record of success with two approved medicines, including AYVAKIT®/AYVAKYT® (avapritinib), which they are bringing to patients with systemic mastocytosis (SM) in the U.S. and Europe. Leveraging their established research, development, and commercial capability and infrastructure, they now aim to significantly scale their 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.

Key Products

Ayvakit (avapritinib)

• Approved by the FDA for the treatment of three indications: adults with indolent systemic mastocytosis (ISM; May 2023), adults with advanced systemic mastocytosis (advanced SM; June 2021), and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations (January 2020)


• Designed to potently and selectively inhibit KIT D816V, the primary underlying driver of ISM and advanced SM


• Designed to potently and selectively inhibit PDGFRA exon 18 mutations, which are implicated in a subset of patients with GIST


• For more information, visit www.AYVAKIT.com

Elenestinib

• Next-generation KIT D816V inhibitor in clinical development for the treatment of ISM and other mast cell disorders.


• Currently being evaluated in the Phase 2/3 HARBOR clinical trial of patients with ISM.

BLU-808

• Potent and selective wild-type KIT inhibitor that was developed leveraging Blueprint Medicines’ expertise in KIT and mast cell biology.


• Wild-type KIT plays a central role in mast cell activation, which is implicated in a broad range of inflammatory diseases including chronic urticaria.


• Plan to submit an investigational new drug application (IND) for BLU-808 in the second quarter of 2024.

BLU-222

• Potent and selective CDK2 inhibitor that is currently being evaluated in the Phase 1/2 VELA clinical trial.


• CDK2 is a cell cycle regulator and an important therapeutic target across multiple cancers, including hormone-receptor-positive, HER2-negative breast cancer.

Customer Insights

• Healthcare providers in oncology/hematology, allergy/immunology.

Statistics

• 2 approved medicines


• About 650 employees in the US and Europe


• 5 Breakthrough therapy designations granted by the FDA


• 4 years: Average time from investigational new drug application to first approval


• Focus areas: allergy/inflammation and oncology/hematology


• Company launch: 2011


• Global Headquarters: Cambridge, Massachusetts


• EU Office: Zug, Switzerland

Key Executives

The executive team includes Kate Haviland (CEO), Christy Rossi (COO) and Fouad Namouni (President, Research & Development), among others. Philina Lee (CCO) is also a member of the executive team.

To view their executive page, click here.

Organizational Insights

Our Mission: At Blueprint Medicines, we seek to alleviate human suffering with life-changing medicines.

Our Core Values define how we work — as an organization deeply committed to our employees and patients, driven by the pursuit of innovation and the passion to make an impact.

• Patients First: We maintain intense focus on improving patients’ lives.


• Thoughtfulness: We explore creative approaches, daring to make well-thought-out decisions and owning the outcomes.


• Urgency: We solve complex problems rapidly, with attention and care.


• Trust: We collaborate to build and maintain a cohesive team that leads with integrity as well as mutual respect of lived experiences, viewpoints and diverse talents


• Optimism: We pursue transformative therapies that we believe will make a difference

Board Members

• Jeffrey Albers, Venture Partner, Atlas Venture


• Daniella Beckman, Chief Financial Officer, Tango Therapeutics


• Alexis Borisy, Co-Founder and Operating Chairman, Curie.Bio


• Lonnel Coats, Chief Executive Officer, Lexicon Pharmaceuticals


• Habib Dable, Part-time Venture Partner, RA Capital Management, L.P.


• Mark Goldberg, M.D., Lecturer in Medicine, Harvard Medical School and Faculty Member, Hematology Division, Brigham and Women’s Hospital


• Kate Haviland, President and Chief Executive Officer, Blueprint Medicines


• Nick Lydon, Scientific Founder, Blueprint Medicines


• Lynn Seely, President and Chief Executive Officer, Lyell Immunopharma and Lead Independent Director, Blueprint Medicines


• John Tsai, Chief Executive Officer, Forcefield Therapeutics

To view their executive page, click here.

Key Milestones

• 2011: Company launch


• 2015: Launched initial public offering on the Nasdaq stock exchange under the ticker BPMC


• 2020: Received FDA approval of Ayvakit for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.


• 2020: Received FDA approval of Gavreto (pralsetinib) for the treatment of adults with metastatic RET fusion-positive non-small cell lung cancer, and patients with advanced or metastatic RET fusion-positive thyroid cancer


• 2021: Received FDA approval of Ayvakit for the treatment of adults with advanced systemic mastocytosis


• 2023: Received FDA approval of Ayvakit for the treatment of adults with indolent systemic mastocytosis


• 2024: Announced a definitive agreement with Rigel Pharmaceuticals granting Rigel the U.S. rights for Gavreto.

News and Press Releases

• Feb 2024: Blueprint Medicines Reports Fourth Quarter and Full Year 2023 Results (Read).


• Feb 2024: Blueprint Medicines Highlights AYVAKIT® (avapritinib) Long-Term Efficacy and Safety Data and Advances in Mast Cell Disease Research at 2024 AAAAI Annual Meeting (Read).


• Dec 2023: Blueprint Medicines’ AYVAKYT® (avapritinib) Receives European Commission Approval as the First and Only Treatment for Indolent Systemic Mastocytosis (Read).


• May 2023: FDA Approves AYVAKIT® (avapritinib) as the First and Only Treatment for Indolent Systemic Mastocytosis (Read).

To view their full press release, click here.

Corporate Responsibility

At Blueprint Medicines, they believe that when they work together, the impact of their collective efforts can make a profound difference for patients, their employees, and the communities where they live and work. They are grounded in integrity, lead with their core values, and are inspired to make the world a better place.

to know more about Blueprint Medicines, click here.