Blueprint Medicines Corp. is a precision therapy company, which engages in the invention of medicines for people with cancer and blood disorders. The company was founded by Chris Varma, Nicholas B. Lydon, Brian Druker, and Alexis Borisy on October 14, 2008 and is headquartered in Cambridge, MA.
Clinical Trials
86
45 active
Approvals
9
Total approvals
Agencies
3
Regulatory bodies
Founded
N/A
Active, not recruiting
44
51.2%
Approved For Marketing
2
2.3%
Completed
20
23.3%
No Longer Available
1
1.2%
Not yet recruiting
1
1.2%
Recruiting
12
13.9%
Terminated
5
5.8%
Unknown
1
1.2%
- AbCellera has appointed Dr. Lynn Seely, an experienced biopharmaceutical executive, to its Board of Directors as an independent director. - Dr. Seely previously led the clinical development of Xtandi (enzalutamide) at Medivation through global approvals, culminating in a $14 billion acquisition by Pfizer. - As CEO of Myovant Sciences, she successfully commercialized Myfembree and Orgovyx, demonstrating expertise in endocrinology and women's health. - Dr. Seely currently serves as president and CEO of Lyell Immunopharma, advancing next-generation CAR T-cell therapies for hematologic malignancies and solid tumors.
- Ayvakit (avapritinib) is an FDA-approved kinase inhibitor for PDGFRA exon 18-mutant GIST, advanced systemic mastocytosis, and indolent systemic mastocytosis, with clinical trials demonstrating high response rates and improved quality of life. - The drug carries serious safety warnings including intracranial hemorrhage, cognitive changes, and photosensitivity, requiring close monitoring of platelet levels and neurological status throughout treatment. - Ayvakit costs approximately $42,000 for a 30-day supply, but most commercial insurance and Medicare plans cover it, and the YourBlueprint patient support program can reduce out-of-pocket costs to $0 for eligible patients. - As the only drug highly effective against the PDGFRA D842V mutation in GIST, Ayvakit addresses a critical unmet need where standard kinase inhibitors like imatinib typically fail.
- NICE has recommended Ayvakyt (avapritinib) for NHS use in England and Wales for advanced systemic mastocytosis, including first-line treatment. - The recommendation covers aggressive SM, SM with associated haematological neoplasm, and mast cell leukaemia, offering an alternative to current therapies like midostaurin. - Clinical trials showed the majority of evaluable patients responded to avapritinib, with some achieving complete remissions with haematologic recovery. - Ayvakyt generated $128 million in Q3 global sales, predominantly from the US, with analysts projecting potential blockbuster status above $1 billion.
- Proxygen has appointed Chiara Conti, Ph.D., as Chief Scientific Officer to lead the company's transition from discovery to clinical-stage execution of its molecular glue degrader platform. - Dr. Conti brings extensive experience from Blueprint Medicines, where she identified four development candidates in seven years and established the company's targeted protein degradation platform. - The appointment comes as Proxygen expands beyond degradation into broader induced proximity therapeutics, aiming to address high-value targets across oncology and other therapeutic areas. - Proxygen has built partnerships with major pharmaceutical companies including Merck & Co, demonstrating industry interest in proximity-based therapeutic approaches.
- Daiichi Sankyo has appointed John Tsai, MD as its new Global Head of R&D, effective April 1, 2026, replacing Ken Takeshita who is stepping down after five years. - Tsai brings over 25 years of leadership experience, having previously served as Chief Medical Officer at Novartis where he led development of 160 new projects and 500 clinical trials resulting in 15 new medicine approvals. - The appointment comes as Daiichi Sankyo prepares to execute its next five-year business plan, building on its industry-leading antibody-drug conjugate portfolio including blockbuster drug Enhertu. - Takeshita is credited with transforming Daiichi Sankyo's R&D organization into a globally integrated innovation engine and expanding the company's ADC portfolio through strategic partnerships.
- GC Therapeutics unveiled its initial pipeline programs targeting demyelinating neurological disorders including multiple sclerosis and a regenerative cell therapy for Type 1 diabetes using its TFome™ platform. - The company completed its first Cell State Cookbook™ mapping over 300 human cell states and testing two million transcription factor combinations to enable precision cell state engineering. - Both the FDA and EMA endorsed GCTx's TFome™-enabled development model and supported a streamlined regulatory pathway for the company's pipeline programs. - The TFome™ platform achieves single-step, four-day stem cell differentiation with greater than 90% efficiency, potentially accelerating cell therapy development up to 100 times faster than conventional methods.
- Galecto appointed Sherwin Sattarzadeh as Chief Operating Officer and Dr. Becker Hewes as Chief Medical Officer, both former Blueprint Medicines executives with extensive hematology/oncology drug development experience. - The company's lead asset DMR-001, a potentially best-in-class monoclonal antibody targeting mutant calreticulin, remains on track for IND submission in mid-2026 with subcutaneous administration planned. - With $285 million raised in November 2025 PIPE financing, Galecto has financial runway into 2029 to support multiple data milestones, including Phase 1 clinical proof-of-concept data for DMR-001 anticipated in 2027. - DMR-001 demonstrates highly potent activity against both Type 1 and Type 2 mutCALR-driven preclinical models, positioning it to address the full spectrum of CALR mutations in essential thrombocythemia and myelofibrosis.
- Blueprint Medicines presented multi-year clinical data at ASH 2025 showing AYVAKIT (avapritinib) maintains sustained efficacy across both indolent and advanced systemic mastocytosis with durable symptom control. - In advanced systemic mastocytosis first-line treatment, AYVAKIT achieved an 87% overall response rate with median overall survival not yet reached after four years of follow-up. - Long-term safety data spanning over three years demonstrated a favorable tolerability profile with only 3% discontinuation rate due to treatment-related adverse events in indolent systemic mastocytosis patients. - The drug showed significant bone health improvements across both disease subtypes, reflecting potential disease-modifying effects beyond symptom management.
- Rigel Pharmaceuticals' lead product Tavalisse generated $68.5 million in sales during the first half of 2025, representing a 44% year-over-year increase. - The company's second FDA-approved drug Rezlidhia showed strong momentum with 31% year-over-year growth to $13.1 million in sales. - Rigel raised its 2025 revenue guidance to $270-$280 million from the previous expectation of $200-$210 million due to strong commercial performance. - The company is advancing R289, a dual IRAK1/IRAK4 inhibitor, in Phase Ib trials for myelodysplastic syndrome with dose expansion planned for the second half of 2025.
- The first half of 2025 witnessed unprecedented M&A activity with major deals including Merck's $3.9 billion SpringWorks acquisition, Sanofi's $9.5 billion Blueprint Medicines purchase, and Johnson & Johnson's $14.6 billion Intra-Cellular Therapies acquisition. - A landmark U.S. court ruling in the Pomalyst case established that most-favored-entry clauses in pharmaceutical patent settlements can withstand antitrust scrutiny when properly structured, providing clearer guidance for industry settlements. - UK and EU regulators set new enforcement precedents with coordinated action against pharmaceutical disparagement, while the UK's enhanced merger control powers target potential "killer acquisitions" in early-stage biotech deals. - The Court of Appeal upheld a £51.9 million fine against Advanz Pharma for excessive pricing of thyroid drug liothyronine, reinforcing regulatory authority over pharmaceutical pricing abuses in post-Brexit UK.
