Agios Set to Revolutionize Rare Disease Treatment: 2023-2026 Catalysts to Drive Improved Patient Outcomes

We are excited to announce five pivotal trials in the field of rare hematological diseases, with strong clinical data to support them. Our goal is to obtain FDA approval for two additional indications with PYRUKYND®, one in thalassemia in 2025 and one in sickle cell disease in 2026. We have a solid cash position to ensure the completion of our current programs and the expansion of our pipeline, bringing us to a point of cash-flow positivity.

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO) is excited to announce its upcoming 2023 milestones and value-driving catalysts that will revolutionize patient outcomes in rare diseases. Join us as we present at the 41st Annual J.P. Morgan Healthcare Conference on Wednesday, January 11th, 2023 at 7:30 AM PT. You can stream the event live from our website to find out how we plan to make a lasting impact on rare diseases through 2026.

Agios is on the brink of major expansion with the potential of two additional PYRUKYND® approvals by 2026, and is well-equipped to further its existing clinical pipeline and broaden the scope within its core areas of expertise. As the leading innovator in PK activation, with over seven years of clinical experience with PYRUKYND®, Agios has generated a strong body of consistent and impressive data across rare hematological diseases. Additionally, the company has launched its first rare disease U.S. product in adult PK deficiency, offering the first disease-modifying therapy for this patient community, and is poised for success with expected launches in larger patient populations.

Recent Highlights

  • PYRUKYND® has recently been granted marketing authorization in the European Union and Great Britain for the treatment of adults with an inherited disorder called pyruvate kinase (PK) deficiency. This is exciting news for those living with this rare disorder, as PYRUKYND® provides a new option to help manage their condition.
  • Approximately half of patients with thalassemia have enrolled in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND® to explore the potential effectiveness of the treatment in not regularly transfused and regularly transfused adults respectively. This is a great step forward for those in the Thalassemia community who are seeking more effective treatments.
  • In December, the RISE UP study of PYRUKYND® closed its screening phase for adults with sickle cell disease, and the study is expected to be completed by January. This study presents an exciting opportunity to explore the potential of PYRUKYND® in the treatment of sickle cell disease, and to provide hope to those living with the condition.
  • At the 64th American Society of Hematology (ASH) Annual Meeting & Exposition, we shared a comprehensive range of clinical and translational data regarding the long-term effects of PYRUKYND® in adults with non-transfusion-dependent thalassemia and adults with PK deficiency. Our presentation offered an in-depth exploration of the potential of this innovative new treatment.
  • Tsveta Milanova, who brings two decades of extensive experience in rare disease commercial strategy and global market access, has been appointed to the role of chief commercial officer at [company name]. Her impressive track record in this field and her in-depth knowledge of the industry makes her the perfect candidate to help [company name] continue to grow and succeed.

Anticipated 2023 Milestones

  • We are thrilled to announce that enrollment for the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND® for the treatment of Thalassemia is expected to be completed by mid-year. This marks an exciting milestone in the journey towards finding a potential solution for this debilitating condition.
  • This year, we have the chance to make history and enroll at least half of patients with Pediatric PK Deficiency in the groundbreaking Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND®. Let’s seize this opportunity and make a difference in the lives of these children.
  • We are excited to announce the data readout from the Phase 2 portion of the RISE UP study of PYRUKUND®, and look forward to making a go/no-go decision to progress to Phase 3 by mid-year. This is a major milestone in the research of Sickle Cell Disease and we have high hopes that PYRUKUND® will provide a positive outcome for those affected.
  • At year-end, we anticipate the completion of enrollment for the Phase 2a study of the novel PK activator AG-946, which is aimed at treating Lower-risk Myelodysplastic Syndromes (LR-MDS). This groundbreaking research offers a glimmer of hope for patients struggling with this complex disease.
  • By the end of the year, we will be filing an Investigational New Drug (IND) application for a PAH Stabilizer, which could potentially be used as a treatment for Phenylketonuria (PKU). Let’s work together to make this revolutionary breakthrough a reality!

Agios 2026 Vision

Agios has an ambitious vision for 2026: the establishment of a classical hematology franchise with PYRUKYND® approvals across PK deficiency, thalassemia and sickle cell disease, the advancement of AG-946 and the preclinical pipeline, and disciplined business development all aligned with the company’s core therapeutic focus areas and capabilities – all culminating in cash-flow positivity. To reach this goal, Agios has provided a roadmap of additional catalysts between 2024 and 2026 – a journey that could be a game-changer for the company and its patients.


We eagerly await the data readout from the Phase 3 ENERGIZE study of PYRUKYND® in adults with non-transfusion-dependent thalassemia, which is expected in the first half of 2024, as well as from the Phase 3 ENERGIZE-T study of PYRUKYND® in adults with transfusion-dependent thalassemia, which is expected in the second half of 2024. We are also looking forward to the data readout from the Phase 2a study of AG-946 in LR-MDS.


PYRUKYND® is on the cusp of potentially receiving approval from the FDA for its use in treating thalassemia. Furthermore, the results of its Phase 3 RISE UP study in sickle cell disease will be available in 2023, when a go/no-go decision will be made. Lastly, the Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND® in pediatric PK deficiency are providing promising data readouts.


Reach for the stars and unlock the potential of PYRUKYND®: with a potential FDA approval for use in sickle cell disease and pediatric PK deficiency, we could be one step closer to achieving cash-flow positivity.

Presentation at 41st Annual J.P. Morgan Healthcare Conference

Agios will be hosting a corporate presentation at the 41st Annual J.P. Morgan Healthcare Conference on Wednesday, January 11 at 7:30 a.m. PT. All interested parties can tune in to the live webcast of the presentation via the Investors section of the company’s website. Plus, the presentation will be archived on the Agios website for two weeks following the event, so you don’t miss out!

About Agios

At Agios, our mission is to develop and deliver therapies for rare diseases and empower patient communities, healthcare professionals, and partners. Our team is driven by a passion to make a difference and cultivate powerful relationships to uncover innovative treatments. We are proud to offer the first-in-class pyruvate kinase (PK) activator in the U.S. for adults with PK deficiency, the first disease-modifying therapy for this lifelong, debilitating hemolytic anemia. In addition, our clinical pipeline of investigational medicines is advancing treatments for alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency and MDS-associated anemia. With numerous investigational therapies in preclinical development and extensive expertise in classical hematology, Agios is dedicated to improving the lives of those living with rare diseases.

Cautionary Note Regarding Forward-Looking Statements

At Agios, we are excited about the potential benefits of PYRUKYND® (mitapivat), AG-946 and its PAH stabilizer. We are committed to advancing the preclinical, clinical and commercial development of these drug candidates, and our strategic vision and goals include key milestones for 2023 and potential catalysts through 2026. Our plans and focus pledge to deliver a range of potential benefits to our partners and stakeholders. However, our progress is subject to numerous factors, risks and uncertainties, including the potential impact of the COVID-19 pandemic on our operations and clinical trials, regulatory approvals, and patent and intellectual property protection. We remain committed to our mission and look forward to continued progress.

Leave a Comment