Agios Pharmaceuticals : Q1 2026 Financial Results and Business Highlights

Published: 2026-04-29 07:05:11 ET AGIO

Published on 04/29/2026 at 07:05 am EDT

Ǫ1 2026

Financial Results

and Business Highlights

Conference call for investors and analysts 29 April 2026

Ǫ1 2026 earnings call agenda

1 Introduction Morgan Sanford, VP Investor Relations

2 CEO Opening Remarks

Brian Goff, Chief Executive Officer

3 Financial Results

Cecilia Jones, Chief Financial Officer

4 Commercial Highlights

Tsveta Milanova, Chief Commercial Officer

5 RGD Highlights

Sarah Gheuens, MD, PhD, Chief Medical Officer, Head of RCD

6 CEO Closing Remarks and ǪGA

CEO Opening Remarks

Brian Goff, Chief Executive Officer

Continued focus on delivering on 2026 strategic priorities

Ǫ1 2026 - early progress toward growth inflection

•

Ǫ1 2026 Net Revenues $20.7M, $8.7M in Ǫ1 2025

2026 OpEx expected to be approximately flat compared to prior year

Strong cash position - over $1B

Strong initial AǪVESME thalassemia launch progress - 242 prescriptions1

Mitapivat sNDA filing for U.S. accelerated approval in sickle cell disease in Ǫ2 2026

Agios - positioned for near-term growth inflection and sustained longer-term value creation

AǪVESME launch execution

Mitapivat sNDA filing in sickle cell disease

Tebapivat - upcoming catalysts

LR-MDS Phase 2b topline data H1 2026

Sickle cell disease Phase 2 topline data H2 2026

Progress early-stage programs

Expansion in rare hematology and other rare diseases

Near-term value Mid-term value Long-term value

Financial Results

Cecilia Jones, Chief Financial Officer

Ǫ1 2026 Financial Results

Statement of Operations ($M)

Ǫ1 2026

Ǫ1 2025

Mitapivat Net Revenue

$20.7

$8.7

US Net Revenue

$18.8

$8.7

Ex-US Net Revenue

$1.9

Cost of Sales

$1.3

$1.1

Research C Development Expense

$81.1

$72.7

Selling, General C Administrative Expense

$48.3

$41.5

Net (Loss) Income

($99.1)

($89.3)

Balance Sheet

Ǫ1 2026

Ǫ4 2025

Cash, Cash Equivalents and Marketable Securities

$1.0B

$1.2B

Disciplined capital allocation to drive sustained long-term value creation

Focused investment to maximize AǪVESME U.S. launch in thalassemia

Operating expense management aligned with driving sustained long-term growth

Strategic pipeline diversification through disciplined internal and external innovation

Focused on delivering 2026 strategic priorities balanced against driving long-term growth

Commercial Highlights

Tsveta Milanova, Chief Commercial Officer

Ǫ1 2026 - robust performance driven by strong commercial execution

Mitapivat Net Revenues - Ǫuarterly ($M)

$20.0

$20.7

$4.0

$1.9

$18.8

$12.5

$12.9

$16.0

$8.7

$18.8M U.S. Net Revenues

Strong initial AǪVESME U.S. launch execution in thalassemia

$1.9M ex-U.S. Net Revenues

Driven by thalassemia uptake in Saudi Arabia, reflects early market access dynamics

Continued quarter-on-quarter variability due to ordering patterns, inventory dynamics and GtN

Ǫ1 2025 Ǫ2 2025 Ǫ3 2025 Ǫ4 2025 Ǫ1 2026

$20.7M Ǫ1 2026 Net Revenue, +138% vs prior year

- early U.S. launch reflects high-quality demand

242 prescriptions1

as of March 31st

Strong early adoption among highly-engaged patients, exiting Ǫ1 with growing NTDT patients

Broadening community-based prescriber uptake

Shorter-than-expected time to treatment, reflecting patient engagement and readiness

- Early in launch, still anticipate 10-12 weeks on average time-to-treatment initiation

- early U.S. launch unlocking path to sustained adoption

Compelling clinical profile and broad indication statement driving early adoption

Efficient REMS onboarding, after operationalization in late January

Comprehensive patient services supporting continuity of treatment

Expand prescriber breadth across community and academic practices

Broaden adoption into NTD patients

(~2/3 of adult diagnosed population in U.S.)

Payer engagement and education to further shape coverage policy

R&D Highlights

Sarah Gheuens, MD, PhD,

Chief Medical Officer, Head of R&D

EARLY-STAGE

LATE-STAGE

REGULATORY

SUBMISSION

APPROVAL

Continued pipeline momentum

PRECLINICAL

Approved U.S., EU, UK

First-in-class PK activator

Phase 3 completed1 ACTIVATE-Kids/KidsT

Approved U.S., KSA, UAE; filing under review in EU2

Phase 3 ENERGIZE-Kids/KidsT to initiate H2 2026

Phase 3 completed1; Planned sNDA filing in Ǫ2 2026

Sickle Cell Disease

AǪVESME (U.S.)

PYRUKYND® (KSA, UAE)

Thalassemia

PYRUKYND®

PK Deficiency

Potential best-in-class PK activator

Phase 2b ongoing

Phase 2 ongoing

Sickle Cell Disease

LR-MDS

PAH stabilizer

Phase 1b initiate H1 2026

Phenylketonuria

siRNA TMPRSS6

Phase 1 HV ongoing

Polycythemia Vera

1. Defined as completion of double-blind randomized portion of the trial. 2. Positive Committee for Medicinal Products for Human Use (CHMP) opinion disclosed 17 October 2025. PK = pyruvate kinase; EU = European Union;

UK = United Kingdom; KSA = Kingdom of Saudi Arabia; UAE = United Arab Emirates; LR-MDS = lower-risk myelodysplastic syndrome; PAH = Phenylalanine Hydroxylase; siRNA = small interfering RNA; TMPRSS6 =

Mitapivat advancing toward U.S. accelerated approval in sickle cell disease

Pre-sNDA meeting completed

Confirmatory trial designed to demonstrate clinical benefit, balancing operationally feasible design and execution

Plan to share additional RISE UP data at upcoming medical congress

U.S. accelerated approval pathway regulatory process1

Pre-sNDA

meeting

sNDA

submission

sNDA regulatory

decision

FDA alignment on

confirmatory trial

sNDA

acceptance

Pleased with progress of FDA engagement and now expect to file sNDA in Ǫ2 2026

Tebapivat - next-generation PK activator - distinct design supporting potential for differentiated clinical profile

Next-generation PK activator

Sickle Cell Disease

Studied in healthy volunteers and sickle cell

disease patients (Phase 1)

Long half-life (~87-93 h), enabling once-daily dosing1

• +1.9 g/dL mean Hb increase at 5 mg ǪD in SCD1

Dose-dependent ↓2,3-DPG / ↑ATP with PD

durability up to 4 weeks post-dose1

Potent PKM2 activation with antifibrotic effects (preclinical)2

Lower Risk-MDS

≥1.5 g/dL mean Hb increase achieved in one

patient (Weeks 8-16) 3

40% of low-transfusion-burden patients achieved transfusion independence3

~50% lower exposure vs NH, supporting evaluation of higher doses in broader LR-MDS population3

Ex vivo RBC data show ↑PK activity, ↑ATP, and

improved red cell function4

Structurally differentiated, next-generation PK activator

Potent dual PKR/PKM2 activator

PK/PD properties support ǪD dosing without taper

Phase 2b LR-MDS topline data in H1 2026; Phase 2 Sickle Cell Disease topline data in H2 2026

1. Xu JZ et al. Results from a Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Tebapivat (AG-946) in Patients with Sickle Cell Disease. 2. Nguyen et al., Blood (ASH 2024), Abstract #1107. Preclinical mouse models of nephritis (anti-GBM) demonstrating PKM2-mediated antifibrotic effects of PK activation. 3. Phase 2a LR-MDS trial, data on file. 4. Fattizzo et al. American Journal of Hematology (2024). Glycolytic

activity and in vitro effect of the pyruvate kinase activator AG-946 in red blood cells from low-risk myelodysplastic syndromes patients. DOI: 10.1002/ajh.27300. PK = pyruvate kinase; PK/PD = pharmacokinetic/pharmacodynamic;

CEO Closing Remarks

Brian Goff, Chief Executive Officer

Strong catalyst flow across pipeline in 2026

H1 2026

Sickle Cell Disease Mitapivat

Pre-sNDA meeting Ǫ1

Lower-Risk MDS tebapivat

Phase 2b topline data

Polycythemia Vera AG-236 (TMPRSS6i)

Phase 1 HV topline data

H2 2026

Sickle Cell Disease tebapivat

Phase 2 topline data

Phenylketonuria AG-181 (PAH stabilizer)

Phase 1b PoM data

AǪVESME (mitapivat) U.S. launch underway in thalassemia

PK deficiency

Thalassemia

Sickle Cell

Disease

PKU

Polycythemia

Vera

LR-MDS

Agios - pipeline advancement driving leadership in rare hematology and other rare diseases

Foundation in rare hematology

Other rare

PYRUKYND

AǪVESME/ PYRUKYND1

Mitapivat/ Tebapivat

Tebapivat

AG-236

AG-181

Mechanism of action

PK activator

TMPRSS6i

siRNA

PAH stabilizer

Development stage

Approved

Registration/ Phase 2

Phase 2b

Phase 1 HV

Phase 1b in PKU patients

Total market opportunity by 2030

Ultra-rare

$1B+

$3B+

$4.5B+

$1B+

Agios pipeline >$10B in potential PYS opportunity in 2030

*EvaluatePharma forecasted U.S. market value in 2030; LR-MDS represents a subset of the provided market size and prevalence is estimated ~70% of total MDS patients. 1. Mitapivat approved and marketed in U.S.

Ǫ&A session

Appendix

Primary Endpoint

Proportion of participants with transfusion independence for at least 8 consecutive weeks5 during the core 24 week treatment period

Appendix - tebapivat Phase 2b Lower Risk-MDS trial

10mg ǪD

N=60

15mg ǪD

Extension

Period (tebapivat 10mg, 15mg, 20mg)

20mg ǪD

24 weeks Open-label period2

156 weeks

Phase 2b topline data expected

H1 2026

Key Inclusion Criteria

•

Lower-risk MDS3 Transfusion dependent4 Hb <10.0 g/dL

Up to 2 prior therapies, including ESAs and/or luspatercept

Key Exclusion Criteria

Known history or AML or secondary MDS

Prior exposure to a PK activator, IDH inhibitors, IST, stem cell transplant

Currently receiving imetelstat, iMiDs, HMAs

Secondary Endpoints

Change in hemoglobin

Transfusion independence for 12 weeks

Additional measures of anemia

PK/PD biomarkers

Safety

1. Full trial details, including participation criteria and study plan, can be found at clinicaltrials.gov NCT05490446; 2. Enrollment completion of one cohort triggers opening of enrollment in the next cohort; 3. Risk score:

Primary Endpoint

Hb response3 at baseline, and from Weeks 10 - 12

Key Exclusion Criteria

>10 SCPCs in the past 12 months

Receiving treatment with voxelotor, crizanlizumab, L-glutamine, or hematopoietic stimulating agents within

90 days before randomization

Secondary Endpoints

Average change from baseline in Hb concentration

Average change from baseline in markers of hemolysis and erythropoiesis

PROs: PROMIS Fatigue, PROMIS Pain, ASCǪ-Me

Safety

Appendix - tebapivat Phase 2 Sickle Cell Disease trial

Phase 2 topline data expected

H2 2026

Key Inclusion Criteria

•

Hb ≥5.5 - ≤10.5 g/dL

HU use permitted, if dose has been stable for at least 90 days before randomization2

Disclaimer

Agios Pharmaceuticals Inc. published this content on April 29, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on April 29, 2026 at 11:04 UTC.