Agios Pharmaceuticals Touts 2026 Growth Plan at JPM: Acvezmi Launch, SCD Data, Expense Discipline

Key Points

• Agios secured FDA approval for Acvezmi in thalassemia (Dec. 23) and is launching in the U.S. targeting ~4,000 adult patients with a list price of $425,000/year, supported by a ~40-person sales force, a REMS for thalassemia, and an initial prescription written before the end of 2025.
• The Phase 3 RISE UP study in sickle cell disease showed a strong anti‑hemolytic profile with about 41% hemoglobin responders and high uptake into the open‑label extension; Agios plans a Q1 pre‑sNDA meeting with the FDA and intends to pursue full approval pending regulatory discussions.
• Agios is pacing growth with multiple 2026 readouts (tevapivat in SCD H2, MDS Phase 2b H1, and early data for AG‑236 and AG‑181) while guiding to keep 2026 operating expenses flat versus 2025 and gating SCD investment until regulatory clarity, aiming for a path to profitability from its PKD and thalassemia franchise.

Agios Pharmaceuticals NASDAQ: AGIO used its presentation at the 44th Annual J.P. Morgan Healthcare Conference to outline its outlook for 2026, highlighting a new thalassemia launch, continued expansion of its pyruvate kinase (PK) activator franchise, multiple clinical readouts, and an operating plan aimed at maintaining financial discipline while moving toward profitability.

Management frames a “growth inflection point” built on PK activation

Chief Executive Officer Brian Goff said the company is at a pivotal moment driven by three components: the emergence of its PK activator franchise as a potential standard of care across hemolytic anemias, advancement of early- and mid-stage pipeline programs, and a clearer path to profitability supported by its current commercial portfolio.

Goff described the PK activation platform as anchored by mitapivat, approved for pyruvate kinase deficiency (PKD) and, more recently, thalassemia. The company is also pursuing sickle cell disease (SCD) and developing a second PK activator, tevapivat, for both SCD and low-risk myelodysplastic syndromes (MDS). Earlier-stage pipeline programs include AG-181 for phenylketonuria (PKU) and AG-236, a TMPRSS6 inhibitor being developed for polycythemia vera.

FDA approval of Acvezmi in thalassemia sets up a high-impact launch

Goff said Agios received FDA approval on Dec. 23 for Acvezmi in thalassemia, calling it a “historic approval” for a community that has been waiting for new options. He emphasized that Acvezmi is a separate brand from Pyrukynd, allowing the company to isolate a risk evaluation and mitigation strategy (REMS) for thalassemia while noting there is no REMS for Pyrukynd in PKD.

Management highlighted several “firsts” associated with the thalassemia label, including coverage of both alpha and beta thalassemia regardless of transfusion burden, and positioning as the first oral medicine approved in the indication with twice-daily dosing. The company also pointed to fatigue improvement in the ENERGIZE trial for non-transfusion-dependent patients and durable transfusion reductions lasting up to 36 weeks in the ENERGIZE-T study.

Chief Commercial Officer Tsveta Milanova said there are about 4,000 addressable adult patients in the U.S. at launch (out of roughly 6,000 adult patients), supported by established ICD coding and validated through claims data and account profiling. Agios’ thalassemia effort is supported by an approximately 40-person sales force and the myAgios Patient Support Program, which was established during the Pyrukynd PKD launch.

Goff noted the company generated its first Acvezmi prescription before the close of 2025. The U.S. list price was described as $425,000 per patient per year, which management said reflects the value shown in the clinical studies.

On launch execution, Milanova said near-term priorities include demand generation and ensuring drug availability by the end of January, which she tied to finalizing remaining REMS elements. She characterized physician and patient interest as strong, citing inbound requests to learn more about Acvezmi.

• Initial target patients: Milanova said early adoption is expected to skew toward transfusion-dependent patients, given their frequent contact with the healthcare system.
• REMS education: She said the main education burden is expected to be around REMS paperwork, while clinically, monthly liver monitoring for the first six months aligns with what clinicians would typically do when initiating new thalassemia therapy.
• Launch trajectory: Management said it does not expect an immediate “bolus” of patient starts, describing a more typical rare-disease adoption curve.

Sickle cell disease: Phase 3 data and upcoming FDA engagement

Agios discussed results from the RISE UP Phase 3 study of mitapivat in SCD reported in November. Goff said the trial demonstrated a strong anti-hemolytic profile, including nearly 41% of patients achieving a pre-specified hemoglobin improvement response, along with improvements in other hemolysis markers. He also said the company did not see hepatocellular injury cases matching a pattern observed in thalassemia, and noted that more than 99% of eligible patients opted into the open-label extension.

In the Q&A, Head of R&D and Chief Medical Officer Sarah Gheuens said the trial again showed a consistent anti-hemolytic profile and that, while the pain crisis endpoint was not statistically observed in the overall population, hemoglobin responders showed clinically meaningful benefits across pain crisis-related endpoints and fatigue.

Gheuens said Agios plans to meet with the FDA in a “classic pre-sNDA meeting” in the first quarter to discuss the data package. She added that, while the FDA has noted hemoglobin can be a surrogate endpoint in SCD that may support accelerated approval pathways, Agios intends to pursue a full approval based on the RISE UP dataset and will provide an update on the regulatory path after discussions with the agency.

Tevapivat readouts, earlier pipeline programs, and disciplined spending

Agios also highlighted tevapivat as a more potent PK activator that may offer longer and stronger binding to PKR and PKM2, lower drug-drug interaction risk, and potential once-daily dosing due to a longer half-life. The company is studying tevapivat in SCD (Phase 2, three dose levels) with data expected in the second half of the year.

For low-risk MDS, Goff cited a late-2023 Phase 2a readout showing a 40% response rate in low transfusion burden patients achieving transfusion independence. He said exposure levels at a 5 mg dose were lower than modeled, prompting Agios to test higher doses (10 mg, 15 mg, 20 mg daily) in a fully enrolled Phase 2b study, with results expected in the first half of the year.

In earlier-stage development, Goff said Agios expects Phase 1 healthy volunteer data in the first half of the year for AG-236, a GalNAc-based TMPRSS6 inhibitor in-licensed from Alnylam for polycythemia vera, with a goal of durable hematocrit control and “Q6 month dosing.” He also said the company aims to dose the first patient in a Phase 1b trial for AG-181 in PKU, with proof-of-mechanism data expected in the second half of the year.

On finances, Goff said Agios is guiding to keep 2026 operating expenses flat versus 2025, while remaining fully funded for the Acvezmi launch. He said the company is “gating” investment in SCD until it gains regulatory clarity and is focused on operating model efficiencies. Goff added that the company sees a clear path to profitability based on its current commercial opportunities in PKD and thalassemia.

During the Q&A, management also said it remains open to business development opportunities that fit Agios’ hematology capabilities and differentiation bar, while maintaining discipline by comparing external opportunities against internal pipeline priorities.

About Agios Pharmaceuticals NASDAQ: AGIO

Agios Pharmaceuticals, Inc is a biopharmaceutical company founded in 2008 as a spin-out from research at Dana-Farber Cancer Institute and the Broad Institute. Headquartered in Cambridge, Massachusetts, Agios focuses on understanding and targeting cellular metabolism to develop novel therapies for cancer and rare genetic diseases. The company's scientific platform integrates genomic discovery, metabolic profiling and precision medicine approaches to identify and advance small-molecule candidates that correct or exploit metabolic dysfunction.

Agios's lead products are IDH (isocitrate dehydrogenase) inhibitors that target specific cancer mutations.

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