Clinical Trials

Date: 2015-04-19

Type of information: Presentation of results at a congress

phase: preclinical

Announcement: presentation of results at the American Association for Cancer Research (AACR)

Company: Blueprint Medicines (USA - MA)

Product: avapritinib (BLU-285)

Action mechanism:

kinase inhibitor. BLU-285 is a highly targeted drug that potently and selectively inhibits PDGFR? D842V and KIT Exon 17 mutants, which are receptor tyrosine kinase mutants known to be key drivers in treatment-resistant and metastatic GIST. Because BLU-285 binds to regions of the PDGFR? and KIT kinases that are structurally similar, it is able to simultaneously inhibit PDGFR? D842V and KIT Exon 17 mutants with minimal inhibition of other kinases, potentially limiting the off-target side effects associated with many cancer treatments.

Disease:

gastrointestinal stromal tumors (GIST)

Therapeutic area: Cancer - Oncology

Country:

Trial details:

Latest news:

• On April 19, 2015, Blueprint Medicines announced new preclinical data demonstrating that its drug candidate BLU-285 has significant anti-tumor activity in treatment-resistant models of gastrointestinal stromal tumors (GIST) and achieved and maintained complete tumor regression in all mice treated at the highest dose level. These data were presented at the American Association for Cancer Research (AACR) Annual Meeting 2015. The new preclinical data presented at AACR show that BLU-285 induces significant tumor regression in a patient-derived in vivo model of GIST that is refractory to treatment with imatinib, an approved first-line treatment for GIST. Key findings include:
• BLU-285 achieved and maintained complete tumor remission in 100 percent of mice treated with an oral, once daily dose of 30 mg/kg or 100 mg/kg for 28 days
• Complete tumor regression was maintained during a 28-day observation period following treatment at the 100 mg/kg dose
Blueprint Medicines is on track to file an Investigational New Drug (IND) Application and begin a Phase 1 clinical trial of BLU-285 in mid-2015 that will include GIST patients whose cancer is driven by PDGFR? D842V and KIT Exon 17 mutants. The Company also plans to file an IND and begin a Phase 1 clinical trial in mid-2015 for BLU-285 in patients with systemic mastocytosis, another disease with significant unmet need in which a KIT Exon 17 mutant plays a key role.