Agios Pharmaceuticals

Phase 3 RISE UP study enrollment completed for mitapivat, an oral therapy for sickle cell disease (SCD), with results expected by end of 2025. Mitapivat, approved as Pyrukynd for pyruvate kinase deficiency, aims to boost red blood cell health and prevent sickling by activating pyruvate kinase and reducing 2,3-DPG levels. Phase 2 results showed increased hemoglobin and reduced pain crises; Phase 3 will assess similar outcomes over a year. Participants can opt for an open-label extension phase.

Agios completes enrollment for Phase 3 RISE UP study evaluating mitapivat in sickle cell disease, expecting topline results in late 2025.

Pfizer's withdrawal of Oxbryta from global markets due to increased risk of deaths and complications has left the sickle cell disease community reeling. Despite recent setbacks, hope for SCD treatment lies in next-gen transplantation and gene therapy, with St. Jude developing its own gene therapy. Pfizer's inclacumab and osivelotor, Agios Pharmaceuticals' mitapivat, Novo Nordisk's etavopivat, and Fulcrum Therapeutics' pociredir are among investigational therapies in the SCD pipeline.

DelveInsight's Alpha Thalassemia Market Insights report forecasts significant market growth (2020-2034) driven by rising prevalence, genetic research advancements, and emerging therapies from companies like Agios Pharmaceuticals, Forma Therapeutics, and Novartis Pharmaceuticals.

Pfizer withdraws Oxbryta, its sickle cell disease therapy, from worldwide markets due to higher risk of deaths and complications, leaving patients and doctors seeking alternatives.

Pfizer withdraws sickle cell drug Oxbryta from global market due to safety concerns, leaving patients and advocates scrambling. The decision follows EMA review highlighting increased vaso-occlusive crisis cases. Pfizer pauses ongoing clinical trials and shuts down expanded access programs. The FDA advises against prescribing Oxbryta and recommends alternative treatments.

Pfizer's withdrawal of Oxbryta due to death risk may accelerate trials of rivals like Agios' mitapivat and Fulcrum's pociredir. Oxbryta's suspension leaves mild-moderate patients reliant on hydroxyurea. Gene therapies Lyfgenia and Casgevy target severe cases. Pfizer's stock dips amid broader challenges.

The FDA granted orphan drug designation to tebapivat (AG-946) for treating myelodysplastic syndromes (MDS). Tebapivat is being studied in a phase 2 trial for anemia in lower-risk MDS, with phase 2a data showing 4 out of 10 low transfusion burden patients achieving transfusion independence. The phase 2b study will continue to evaluate tebapivat in patients with lower-risk MDS, focusing on transfusion independence as the primary endpoint.

Agios Pharmaceuticals received orphan drug designation (ODD) from the FDA for tebapivat to treat myelodysplastic syndromes (MDS). The designation offers benefits like tax credits, fee exemptions, and market exclusivity. Agios aims to deliver the first oral therapy addressing anaemia in lower-risk MDS, affecting around 75,000-80,000 patients in key markets. The company focuses on developing therapies for rare diseases, with lead asset mitapivat, a PK activator, previously receiving ODD for PK deficiency, thalassaemia, and sickle cell disease.