Agios Pharmaceuticals (AGIO) IR Breakout at EHA 2024 summary

Key data presentations and study results

• Seven abstracts were presented at EHA 2024, including two on the phase 3 Energize study of mitapivat in non-transfusion-dependent thalassemia.

• Both Energize and Energize-T phase 3 studies met primary and key secondary endpoints, showing efficacy in all thalassemia subtypes.

• In the Energize study, 42% of mitapivat patients achieved a ≥1 g/dL hemoglobin increase vs. 1.6% for placebo; improvements were seen across all subgroups.

• Statistically significant improvements in fatigue and 6-minute walk test were observed, with mitapivat exceeding clinically meaningful thresholds.

• Mitapivat was generally well tolerated, with adverse events similar to placebo and low discontinuation rates.

Regulatory and commercial plans

• A marketing application for mitapivat in thalassemia, covering both Energize and Energize-T data, is planned for FDA submission by year-end.

• Global regulatory filings are prioritized, with the Gulf region and Europe identified as key markets following the U.S.

• Launch preparations are underway, with a focus on market education and partner selection in high-prevalence regions.

Patient impact and clinical perspectives

• Mitapivat is positioned as the first oral disease-modifying therapy for both alpha and beta thalassemia, addressing a major unmet need.

• Physicians indicated most non-transfusion-dependent thalassemia patients with hemoglobin <10 g/dL could benefit, with therapy to be personalized.

• The majority of patients are continuing into the open-label extension, reflecting satisfaction and perceived benefit.

• Oral administration is seen as a major advantage over current injectable or transfusion-based therapies, improving compliance and quality of life.