- Phase 1 results showed Agios Pharmaceuticals' (NASDAQ:AGIO) AG-120 was well-tolerated in patients with IDH1 mutation-positive glioma and chondrosarcoma. The glioma data were presented at the Society for Neuro-Oncology Annual Meeting in Scottsdale, AZ and the chondrosarcoma data were presented in Lisbon, Portugal at the Connective Tissue Oncology Society meeting.
- In the glioma study, as of August 1, 66 patients had been treated with single-agent AG-120 and 28 remained on treatment. No dose-limiting toxicities were observed and the majority of adverse events (AEs) were mild to moderate. The most common AEs were headache, nausea, diarrhea and vomiting. There were 11 patients with serious adverse events, none related to AG-120.
- In 65 response-evaluable patients, two (3%) patients experienced minor responses and 41 (63%) had stable disease over a median treatment duration of 8.1 months (glioma).
- The chondrosarcoma study involved 21 patients as of September 23, 12 in the dose-escalation phase and nine in the expansion phase, with seven remaining on treatment. No dose-limiting toxicities were noted. The majority of AEs were mild to moderate with the most common being diarrhea, nausea, decreased appetite, QT prolongation and fatigue. One serious AE, hypophosphatemia (low blood phosphorus) was possibly treatment-related.
- O 20 response-evaluable patients, 11 (58%) had stable disease. Three-month progression-free survival was 58%.
- AG-120 is an inhibitor of the mutated form of the metabolic enzyme IDH1 (isocitrate dehydrogenase 1) which is present in a wide range of cancers.
Original article