Agios Pharmaceuticals

The IDH market is set to grow rapidly due to expanded indications for approved therapies and increased R&D. Key players like Servier and Bayer are developing novel IDH inhibitors, with IDHIFA (enasidenib) being the first-in-class oral targeted inhibitor for IDH2-mutated AML. IDH inhibitors, including TIBSOVO (ivosidenib) and REZLIDA (enasidenib), are FDA and EMA-approved for various cancers. Vorasidenib, in advanced development, aims to treat IDH-mutant diffuse glioma. The market growth is driven by advancements in research and expanding pipeline of IDH inhibitors.

Agios announces positive Phase 3 ENERGIZE-T results for mitapivat in transfusion-dependent alpha- or beta-thalassemia, with 30.4% response rate vs 12.6% placebo, and files regulatory applications in U.S., EU, Saudi Arabia, and UAE.

ENERGIZE-T Phase 3 study showed mitapivat, an oral PK activator, significantly reduced transfusion burden in adults with transfusion-dependent alpha- or beta-thalassemia, meeting primary and key secondary endpoints. Agios filed for regulatory approval of mitapivat for both transfusion-dependent and non-transfusion-dependent thalassemia in the U.S., EU, Saudi Arabia, and UAE.

Agios Pharmaceuticals presented positive Phase 3 ENERGIZE-T study results for mitapivat, an oral PK activator, in transfusion-dependent alpha- or beta-thalassemia patients, achieving primary and key secondary endpoints. The company filed for regulatory approval of mitapivat in the U.S., EU, Saudi Arabia, and UAE. A live and webcast investor event will be hosted on December 9.

The Oncology Market is critical in global healthcare, driven by cancer prevalence, technological advancements, and strategic collaborations. Key players like Daiichi Sankyo, Bayer AG, and others innovate in diagnostics, treatments, and precision medicine. Market growth is fueled by R&D investments, sustainability focus, and consumer awareness. Challenges include regulatory constraints and talent shortages. Technological integrations like AI and IoT enhance patient care and operational efficiency.

Sanofi's rilzabrutinib shows promise in treating immune thrombocytopenia, while Novo Nordisk's etavopivat reduces sickle cell pain crises. Beam Therapeutics' CRISPR therapy for sickle cell yields consistent results, though fertility preservation remains a concern.

Crescent Biopharma appoints Susan Moran, MD, MSCE, and Alexandra Balcom, MBA, CPA, to its Board of Directors, strengthening its foundation for growth in oncology therapeutics.

Two new gene editing medications for sickle cell disease, including the first FDA-approved CRISPR-based drug, face barriers like high costs and complex delivery processes. Pfizer withdrew its sickle cell drug Oxbryta due to safety concerns, and patient uptake for new gene therapies has been slow. Despite these challenges, the sickle cell treatment market is expected to grow, with over 40 companies developing more than 50 treatments. Cellarity is developing an oral drug to induce fetal hemoglobin, aiming to provide a more accessible treatment option.

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.