Agios Reports Business Highlights and Second Quarter 2024 Financial Results

Core Insights - Agios Pharmaceuticals reported positive topline data from the Phase 3 ENERGIZE-T study, indicating mitapivat's efficacy across all thalassemia subtypes, with plans to file a supplemental New Drug Application (sNDA) by the end of 2024 [1][2] - The company announced a $905 million purchase agreement with Royalty Pharma for vorasidenib royalty rights, expecting to receive a total of $1.1 billion upon FDA approval of vorasidenib [1][2] - Agios reported net revenue of $8.6 million for PYRUKYND® (mitapivat) in Q2 2024, reflecting a 5% sequential increase from Q1 2024, with a total of 201 unique patients enrolled [3][5] Thalassemia Developments - The Phase 3 ENERGIZE-T study met primary and key secondary endpoints for mitapivat in adults with transfusion-dependent thalassemia [3] - Positive results from the Phase 3 ENERGIZE study were presented at the European Hematology Association 2024 Congress, demonstrating mitapivat's efficacy in non-transfusion-dependent thalassemia [3] Pediatric PK Deficiency - Topline data from the Phase 3 ACTIVATE-KidsT study of mitapivat in children with PK deficiency was announced, marking the first study to report safety and efficacy data in this demographic [3][4] - Enrollment for the Phase 3 ACTIVATE-Kids study in children not regularly transfused has been completed, with topline data expected in 2025 [3][4] Financial Performance - For Q2 2024, Agios reported a net loss of $96.1 million, compared to a net loss of $83.8 million in Q2 2023 [5][6] - Research and Development (R&D) expenses increased to $77.4 million in Q2 2024 from $68.9 million in Q2 2023, primarily due to costs associated with the siRNA TMPRSS6 program [5][6] - Cash, cash equivalents, and marketable securities totaled $645.3 million as of June 30, 2024, down from $806.4 million at the end of 2023 [6][12] Corporate Development - Agios entered a distribution agreement with NewBridge Pharmaceuticals to commercialize PYRUKYND® in the Gulf Cooperation Council (GCC) region [3] - The company anticipates filing an sNDA for mitapivat in thalassemia and completing enrollment in the Phase 3 RISE UP study for sickle cell disease by the end of 2024 [4]