Updated Pyrukynd data of long-term safety profile and durable improvement in hemoglobin and markers of hemolysis and ineffective erythropoiesis in non-transfusion-dependent alpha- and beta-thalassemia

Data from the study were featured in a poster presentation (abstract #1030) at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition, hosted Dec. 10-13, 2022, in New Orleans.

Consistent with previously reported data, durable improvements in hemoglobin concentration and markers of hemolysis and ineffective erythropoiesis were observed for up to 72 weeks of treatment in both alpha- and beta-thalassemia patients. Additionally, markers of iron homeostasis remained stable or improved through Week 72. Pyrukynd was well tolerated, and the safety profile was consistent with previous studies.

“The data presented today continue to underscore the potential of PK activation to address multiple aspects of the complex underlying pathophysiology of alpha - and beta-thalassemia, including hallmarks of the disease: hemolysis and ineffective erythropoiesis,” said Kevin Kuo, M.D., hematologist at University of Toronto, Toronto General Hospital, and an investigator in the study. “Thalassemia is a rare, debilitating lifelong blood disorder, and there are no currently approved treatment options for those with alpha-thalassemia and options are limited for those with beta -thalassemia. These data, along with long-term extension study data from ongoing studies of the treatment in pyruvate kinase deficiency, demonstrate the potential clinical benefits of Pyrukynd for a broad spectrum of hemolytic anemias and support its continued investigation in thalassemia.”

Agios also presented data at ASH further elucidating the burden of disease and unmet needs in alpha- and beta-thalassemia; 1."Characterizing the Clinical, Health-related Quality of Life and Economic Burden of Alpha-thalassemia: A Systematic Literature Review and Evidence Gaps Assessment (Abstract #1036)"- In a first-of-its-kind systemic literature review investigating clinical, health-related quality of life and economic burden associated with alpha-thalassemia, results underscore the need for further research to fully characterize the burden of disease. Where reported, adult patients with deletional and non-deletional alpha -thalassemia experience clinical complications across a range of conditions, including moderate-to-severe iron overload (31%), iron overload of unspecified severity (66%) and advanced liver fibrosis (20%). Complications were significantly higher in adults with non-deletional alpha-thalassemia. Generally, children and adolescents with alpha-thalassemia experience similar health-related quality of life scores, across psychological, emotional, social and school functioning parameters, as those with beta-thalassemia.

2."Clinical Burden of Alpha- and Beta-thalassemia Compared to Matched Controls in the Real-world Setting (Abstract #2351)"-In a poster presentation reviewing claims data for patients and controls from commercial and government databases, an analysis showed that serious comorbidities and unmet needs persist for patients with thalassemia, even in thalassemia types that have historically been considered less severe, such as non-transfusion dependent thalassemia. Both alpha and beta-non-transfusion dependent thalassemia had significantly higher clinical burden than matched controls including endocrinopathies, cardiovascular disease, liver disease and pulmonary hypertension – conditions associated with considerable morbidity and mortality. Additional therapies are needed to address the underlying cause of the disease and for prevention of these serious complications.