Agios Pharmaceuticals

Avanzanite Bioscience has secured exclusive European commercialization rights for PYRUKYND® (mitapivat), a first-in-class pyruvate kinase activator approved for treating adults with PK deficiency.

DelveInsight's 2025 assessment reveals a robust sickle cell disease pipeline with 55+ companies developing 60+ therapeutic candidates across various clinical development stages.

• Dr. Eyal Attar joins Halda Therapeutics as Chief Medical Officer, bringing over 25 years of biotechnology R&D and clinical trial experience to advance the company's novel RIPTAC cancer therapy platform. • Halda is currently conducting a Phase 1/2 clinical trial of HLD-0915, their lead candidate targeting metastatic castration-resistant prostate cancer (mCRPC) through a unique "hold and kill" mechanism designed to overcome resistance. • The RIPTAC platform represents a new therapeutic modality that creates neomorphic protein-protein interactions to selectively target cancer cells, with programs in development for prostate cancer, breast cancer, and other serious diseases.

Mitapivat demonstrated significant efficacy in the ACTIVATE-Kids Phase 3 trial, becoming the first oral therapy to show positive results in children with non-transfusion-dependent PK deficiency.

Agios Pharmaceuticals is developing Tebapivat (AG-946), an oral PKR activator, targeting multiple hemolytic anemic conditions including sickle cell disease and PK deficiency.

The European Commission has granted orphan medicinal product designation to mitapivat for the treatment of sickle cell disease, offering benefits like reduced fees and market exclusivity.

Updated data from the phase 3 INDIGO study demonstrates vorasidenib's continued efficacy in patients with IDH1/2-mutated diffuse glioma.

• The sickle cell disease treatment landscape is facing challenges, including access barriers to gene therapies and the withdrawal of Pfizer's Oxbryta due to safety concerns. • Gene therapies like Vertex and CRISPR Therapeutics' Casgevy and bluebird bio's Lyfgenia, while potentially curative, are limited by high costs, complex administration, and toxicity. • Companies like Cellarity are developing alternative oral drugs to induce fetal hemoglobin production, aiming for a more accessible and scalable treatment option. • Despite setbacks, the sickle cell disease market remains attractive, with multiple companies developing new therapies and gene editing technologies.

Agios Pharmaceuticals reported $9 million in net Pyrukynd revenue for Q3 2024, a 22% increase compared to Q3 2023, alongside a strong cash position of $1.7 billion.

Agios Pharmaceuticals' Phase 3 RISE UP study, evaluating mitapivat for sickle cell disease (SCD), has completed enrollment of over 200 patients aged 16 and older.