Agios Reports Second Quarter 2025 Financial Results and Provides Business Update
Agios Pharmaceuticals (NASDAQ: AGIO) reported Q2 2025 financial results, highlighting $12.5 million in PYRUKYND net revenues, a significant increase from $8.6 million in Q2 2024. The company maintains a strong financial position with $1.3 billion in cash and equivalents.
Key developments include the pending FDA review of PYRUKYND's sNDA for thalassemia with a PDUFA date of September 7, 2025, and anticipated topline results from the RISE UP Phase 3 trial in sickle cell disease by year-end. The company reported a net loss of $112.0 million for Q2 2025, compared to $96.1 million in Q2 2024, with increased R&D expenses of $91.9 million and SG&A expenses of $45.9 million.
Notable operational progress includes 248 unique patients completing prescription enrollment forms and 142 patients currently on PYRUKYND therapy. The company also secured a European distribution agreement with Avanzanite Bioscience and received IND clearance for AG-236 in polycythemia vera treatment.
Agios Pharmaceuticals (NASDAQ: AGIO) ha comunicato i risultati finanziari del secondo trimestre 2025, evidenziando ricavi netti di 12,5 milioni di dollari da PYRUKYND, un aumento significativo rispetto agli 8,6 milioni di dollari del secondo trimestre 2024. L'azienda mantiene una solida posizione finanziaria con 1,3 miliardi di dollari in liquidit脿 e equivalenti.
Tra gli sviluppi chiave si segnala la revisione FDA in corso della sNDA di PYRUKYND per la talassemia, con una data PDUFA fissata per il 7 settembre 2025, e i risultati principali attesi entro fine anno dallo studio di fase 3 RISE UP sulla malattia falciforme. L'azienda ha registrato una perdita netta di 112,0 milioni di dollari nel secondo trimestre 2025, rispetto ai 96,1 milioni dello stesso periodo del 2024, con un aumento delle spese di R&S a 91,9 milioni e delle spese SG&A a 45,9 milioni.
Tra i progressi operativi rilevanti, 248 pazienti unici hanno completato i moduli di iscrizione alla prescrizione e 142 pazienti sono attualmente in terapia con PYRUKYND. La societ脿 ha inoltre siglato un accordo di distribuzione europeo con Avanzanite Bioscience e ottenuto l'autorizzazione IND per AG-236 nel trattamento della policitemia vera.
Agios Pharmaceuticals (NASDAQ: AGIO) inform贸 los resultados financieros del segundo trimestre de 2025, destacando ingresos netos de 12,5 millones de d贸lares por PYRUKYND, un aumento significativo respecto a los 8,6 millones del segundo trimestre de 2024. La empresa mantiene una posici贸n financiera s贸lida con 1,3 mil millones de d贸lares en efectivo y equivalentes.
Entre los avances clave se encuentra la revisi贸n pendiente por la FDA de la sNDA de PYRUKYND para talasemia, con una fecha PDUFA el 7 de septiembre de 2025, y se esperan los resultados principales del ensayo de fase 3 RISE UP en enfermedad de c茅lulas falciformes para finales de a帽o. La compa帽铆a report贸 una p茅rdida neta de 112,0 millones de d贸lares en el segundo trimestre de 2025, frente a los 96,1 millones del mismo trimestre de 2024, con un aumento en gastos de I+D de 91,9 millones y gastos SG&A de 45,9 millones.
Entre los avances operativos notables, 248 pacientes 煤nicos completaron formularios de inscripci贸n para prescripci贸n y 142 pacientes est谩n actualmente en terapia con PYRUKYND. La empresa tambi茅n asegur贸 un acuerdo de distribuci贸n europeo con Avanzanite Bioscience y recibi贸 la aprobaci贸n IND para AG-236 en el tratamiento de policitemia vera.
Agios Pharmaceuticals (NASDAQ: AGIO)電� 2025雲� 2攵勱赴 鞛 鞁れ爜鞚� 氚滍憸頃橂┌ PYRUKYND 靾滊Г於滌暋鞚� 1,250毵� 雼煬搿� 2024雲� 2攵勱赴 860毵� 雼煬鞐愳劀 韥矊 歃濌皜頄堧嫟瓿� 氚濏様鞀惦媹雼�. 須岇偓電� 順勱笀 氚� 順勱笀靹� 鞛愳偘鞙茧 13鞏� 雼煬鞚� 韮勴儎頃� 鞛 靸來儨毳� 鞙犾頃橁碃 鞛堨姷雼堧嫟.
欤检殧 臧滊皽 靷暛鞙茧電� PYRUKYND鞚� 韮堧澕靹鸽鞎勳毄 sNDA鞐� 雽�頃� FDA 瓴�韱犼皜 歆勴枆 欷戩澊氅�, 2025雲� 9鞗� 7鞚� PDUFA 雮犾臧� 鞓堨爼霅橃柎 鞛堦碃, 鞐半旯岇 瓴胳儊鞝來槇甑硲鞐� 雽�頃� RISE UP 3靸� 鞁滍棙鞚� 欤检殧 瓴瓣臣臧� 旮半寑霅橁碃 鞛堨姷雼堧嫟. 須岇偓電� 2025雲� 2攵勱赴鞐� 1鞏� 1,200毵� 雼煬鞚� 靾滌啇鞁れ潉 氤搓碃頄堨溂氅�, 鞚措姅 2024雲� 2攵勱赴鞚� 9,610毵� 雼煬氤措嫟 歃濌皜頃� 靾橃箻鞛呺媹雼�. 鞐瓣惮臧滊皽牍勲姅 9,190毵� 雼煬, 韺愲Г甏�毽箘電� 4,590毵� 雼煬搿� 歃濌皜頄堨姷雼堧嫟.
鞖挫榿靸� 欤茧頃� 毵岉暅 歆勳爠鞙茧電� 248氇呾潣 瓿犾湢 頇橃瀽臧� 觳橂癌 霌彪 鞏戩嫕鞚� 鞕勲頄堨溂氅�, 142氇呾潣 頇橃瀽臧� 順勳灛 PYRUKYND 旃橂毳� 氚涥碃 鞛堨姷雼堧嫟. 霕愴暅 須岇偓電� Avanzanite Bioscience鞕� 鞙犽熃 鞙犿喌 瓿勳暯鞚� 觳搓舶頄堨溂氅�, 雼ろ槇甑 旃橂鞝� AG-236鞐� 雽�頃� IND 鞀轨澑鞚� 氚涭晿鞀惦媹雼�.
Agios Pharmaceuticals (NASDAQ : AGIO) a publi茅 ses r茅sultats financiers du deuxi猫me trimestre 2025, mettant en avant des revenus nets de 12,5 millions de dollars provenant de PYRUKYND, une augmentation significative par rapport 脿 8,6 millions de dollars au deuxi猫me trimestre 2024. L'entreprise conserve une solide position financi猫re avec 1,3 milliard de dollars en liquidit茅s et 茅quivalents.
Les d茅veloppements cl茅s incluent l'examen en attente par la FDA de la sNDA de PYRUKYND pour la thalass茅mie, avec une date PDUFA fix茅e au 7 septembre 2025, et les r茅sultats principaux attendus de l'essai de phase 3 RISE UP sur la dr茅panocytose d'ici la fin de l'ann茅e. L'entreprise a enregistr茅 une perte nette de 112,0 millions de dollars pour le deuxi猫me trimestre 2025, contre 96,1 millions au deuxi猫me trimestre 2024, avec une augmentation des d茅penses en R&D 脿 91,9 millions et des frais SG&A 脿 45,9 millions.
Parmi les progr猫s op茅rationnels notables, 248 patients uniques ont rempli les formulaires d'inscription 脿 la prescription et 142 patients sont actuellement sous traitement PYRUKYND. La soci茅t茅 a 茅galement conclu un accord de distribution europ茅en avec Avanzanite Bioscience et obtenu une autorisation IND pour AG-236 dans le traitement de la polycyth茅mie vraie.
Agios Pharmaceuticals (NASDAQ: AGIO) berichtete 眉ber die Finanzergebnisse des zweiten Quartals 2025 und hob 12,5 Millionen US-Dollar Nettoumsatz mit PYRUKYND hervor, was eine deutliche Steigerung gegen眉ber 8,6 Millionen US-Dollar im zweiten Quartal 2024 darstellt. Das Unternehmen verf眉gt 眉ber eine starke Finanzlage mit 1,3 Milliarden US-Dollar an liquiden Mitteln und 脛quivalenten.
Wesentliche Entwicklungen umfassen die ausstehende FDA-Pr眉fung der sNDA von PYRUKYND f眉r Thalass盲mie mit einem PDUFA-Datum am 7. September 2025 sowie die erwarteten Hauptergebnisse der Phase-3-Studie RISE UP zur Sichelzellenkrankheit bis Jahresende. Das Unternehmen meldete einen Nettogewinnverlust von 112,0 Millionen US-Dollar im zweiten Quartal 2025 im Vergleich zu 96,1 Millionen US-Dollar im zweiten Quartal 2024, mit gestiegenen F&E-Ausgaben von 91,9 Millionen und SG&A-Ausgaben von 45,9 Millionen US-Dollar.
Erhebliche operative Fortschritte umfassen 248 einzigartige Patienten, die Anmeldeformulare f眉r Verschreibungen ausgef眉llt haben, sowie 142 Patienten, die derzeit mit PYRUKYND behandelt werden. Das Unternehmen sicherte sich zudem eine europ盲ische Vertriebsvereinbarung mit Avanzanite Bioscience und erhielt die IND-Freigabe f眉r AG-236 zur Behandlung der Polyzyth盲mia vera.
- PYRUKYND revenue increased 45% year-over-year to $12.5 million in Q2 2025
- Strong cash position of $1.3 billion provides financial independence for future launches and pipeline development
- Patient enrollment growing with 248 unique prescription forms (+6% QoQ) and 142 active patients (+4% QoQ)
- Expanded European presence through distribution agreement with Avanzanite Bioscience
- Multiple pipeline advancements including IND clearance for AG-236 and first patient dosed in tebapivat Phase 2 trial
- Net loss increased to $112.0 million from $96.1 million year-over-year
- R&D expenses increased 19% to $91.9 million year-over-year
- SG&A expenses rose 29% to $45.9 million compared to Q2 2024
- Cash position decreased from $1.5 billion to $1.3 billion since December 2024
Insights
Agios shows modest PYRUKYND revenue growth while advancing pipeline; upcoming regulatory decisions and late-stage trial results will drive future value.
Agios reported
The near-term catalyst hierarchy is clear: First, the September 7th FDA decision for PYRUKYND in thalassemia represents the most immediate opportunity to expand the drug's approved indications. Second, year-end topline results from the RISE UP Phase 3 trial in sickle cell disease could potentially support a 2026 commercial launch in a substantially larger market.
The increased R&D expenses (
Two early-stage developments warrant attention: the first patient dosed with tebapivat in sickle cell disease and IND clearance for AG-236 in polycythemia vera. These programs diversify Agios's portfolio beyond mitapivat and provide longer-term growth opportunities.
The European distribution agreement with Avanzanite Bioscience represents a capital-efficient approach to market expansion without building extensive infrastructure, though revenue sharing terms weren't disclosed.
While current PYRUKYND revenue remains modest, the
$12.5 million in second quarter PYRUKYND庐 (mitapivat) net revenues; ended second quarter with$1.3 billion dollars in cash, cash equivalents and marketable securities- PYRUKYND sNDA for thalassemia under active review, with FDA PDUFA goal date of September 7, 2025
- Topline results from RISE UP Phase 3 trial of mitapivat in sickle cell disease on track by year-end with potential U.S. commercial launch in 2026
- Dosed first patient in the tebapivat Phase 2 sickle cell disease trial and received IND clearance for AG-236
CAMBRIDGE, Mass., July 31, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced financial results and updates for the second quarter ended June 30, 2025.
鈥淲ith fewer than 40 days to our PDUFA goal date, our commercial team is prepared for the potential U.S. approval of PYRUKYND for thalassemia,鈥� said Brian Goff, Chief Executive Officer, Agios. 鈥淚n the second quarter, we made progress advancing our early- and mid-stage pipeline and remain on track to deliver topline results of the RISE UP Phase 3 trial for PYRUKYND in sickle cell disease by the end of the year. Collectively, our progress reflects our continued focus on delivering innovative medicines听with the potential to transform the lives of those affected by rare diseases and deliver long-term shareholder value.鈥�
Second Quarter 2025 and Recent Corporate Highlights
Commercial Performance 鈥� PYRUKYND庐 (mitapivat)
- Generated
$12.5 million in net revenue for the second quarter of 2025, compared to$8.6 million in the second quarter of 2024.- 248 unique patients completed prescription enrollment forms, representing an increase of 6 percent over the first quarter of 2025.
- 142 patients are on PYRUKYND therapy, inclusive of new starts and continued therapy, representing an increase of 4 percent over the first quarter of 2025.
- Entered into a distribution agreement with Avanzanite Bioscience B.V., a rapidly growing European specialty pharmaceutical company focused on rare diseases, to distribute and commercialize PYRUKYND across the European Economic Area, the United Kingdom and Switzerland.
R&D Highlights
- PYRUKYND (mitapivat)
- Thalassemia 鈥�
- Launch preparations underway ahead of U.S. PDUFA goal date of September 7, 2025. The sNDA for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia remains under active review by the U.S. Food and Drug Administration (FDA).
- Other regulatory applications remain under review by health authorities in Saudi Arabia, United Arab Emirates, and the European Union.
- Sickle Cell Disease 鈥�
- Topline results from RISE UP Phase 3 trial of mitapivat in sickle cell disease on track by year-end with potential U.S. commercial launch in 2026.
- Thalassemia 鈥�
- Tebapivat
- Sickle Cell Disease 鈥�
- Dosed the first patient in the Phase 2 trial investigating tebapivat in sickle cell disease. The trial is enrolling across three dose cohorts (2.5mg, 5mg, 7.5mg) and placebo and the primary endpoint will measure hemoglobin response, defined as a 鈮�1g/dL increase in hemoglobin concentration from week 10 to week 12, compared to baseline.
- Lower-risk Myelodysplastic Syndromes (LR-MDS) 鈥�
- Continue to progress patient enrollment in the Phase 2b trial for tebapivat in LR-MDS with target enrollment completion by the end of 2025.
- Sickle Cell Disease 鈥�
- Early Pipeline
- Investigational New Drug (IND) clearance received for AG-236, an siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera (PV).
- Presented new data on mitapivat and tebapivat at the . A total of 14 presentations and publications, led by Agios and external collaborators, were shared, covering sickle cell disease, thalassemia, PK deficiency, and MDS.
Second Quarter 2025 Financial Results
For the quarter ended June 30, 2025, net loss was
- Net product revenue from sales of PYRUKYND for the second quarter of 2025 was
$12.5 million , compared to$8.6 million for the second quarter of 2024. - Cost of sales for the second quarter of 2025 was
$1.7 million . - Research and Development (R&D) Expenses were
$91.9 million for the second quarter of 2025, compared to$77.4 million for the second quarter of 2024. The year-over-year increase was primarily attributed to a$10.0 million regulatory milestone payment to Alnylam associated with our agreement to develop and commercialize AG-236, an siRNA targeting TMPRSS6, intended for the treatment of polycythemia vera. - Selling, General and Administrative (SG&A) Expenses were
$45.9 million for the second quarter of 2025 compared to$35.5 million for the second quarter of 2024. The year-over-year increase was primarily attributable to an increase in commercial-related activities, including headcount, as the company prepares for the potential approval of PYRUKYND in thalassemia. - Cash, cash equivalents and marketable securities as of June 30, 2025, were
$1.3 billion compared to$1.5 billion as of December 31, 2024. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND launches in thalassemia and sickle cell disease, advance existing programs, and opportunistically expand its pipeline through both internally and externally discovered assets.
Conference Call Information
Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company鈥檚 second quarter 2025 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company's website () under the 鈥淓vents & Presentations鈥� tab. A replay of the webcast will be available on the company鈥檚 website approximately two hours after the event.
About Agios: Fueled by Connections to Transform Rare Diseases
At Agios, our vision is to redefine the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities 鈥� collaborating to develop and deliver innovative medicines that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts. To learn more, visit听 and follow us on and .
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND庐 (mitapivat), tebapivat, AG-236 and AG-181; Agios鈥� plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND庐, tebapivat, AG-236 and AG-181; Agios鈥� use of proceeds from the transaction with Royalty Pharma; potential U.S. net sales of vorasidenib and potential future royalty payments; Agios鈥� strategic vision and goals, including its key milestones for 2025; and the potential benefits of Agios鈥� strategic plans and focus. The words 鈥渁nticipate,鈥� 鈥渆xpect,鈥� 鈥済oal,鈥� 鈥渉ope,鈥� 鈥渕ilestone,鈥� 鈥減lan,鈥� 鈥減otential,鈥� 鈥減ossible,鈥� 鈥渟trategy,鈥� 鈥渨ill,鈥� 鈥渧ision,鈥� and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios鈥� current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios鈥� product candidates will successfully continue. There can be no guarantee that any positive developments in Agios鈥� business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios鈥� business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios鈥� results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios鈥� ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios鈥� ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios鈥� cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios鈥� public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
| Consolidated Balance Sheet Data | ||||||||
| (in thousands) | ||||||||
| (Unaudited) | ||||||||
| 听 | 听 | 听 | 听 | 听 | 听 | 听 | ||
| 听 | 听 | 听 | 听 | June 30, 2025 | 听 | December 31, 2024 | ||
| Cash, cash equivalents, and marketable securities | 听 | 听 | 听 | $ | 1,339,404 | 听 | $ | 1,532,031 |
| Accounts receivable, net | 听 | 听 | 听 | 听 | 4,986 | 听 | 听 | 4,109 |
| Inventory | 听 | 听 | 听 | 听 | 30,848 | 听 | 听 | 27,616 |
| Total assets | 听 | 听 | 听 | 听 | 1,471,237 | 听 | 听 | 1,663,199 |
| Stockholders' equity | 听 | 听 | 听 | 听 | 1,369,555 | 听 | 听 | 1,540,956 |
| Consolidated Statements of Operations Data | |||||||||||||||
| (in thousands, except share and per share data) | |||||||||||||||
| (Unaudited) | |||||||||||||||
| 听 | 听 | 听 | 听 | 听 | 听 | 听 | 听 | ||||||||
| 听 | Three Months Ended June 30, | 听 | Six Months Ended June 30, | ||||||||||||
| 听 | 听 | 2025 | 听 | 听 | 听 | 2024 | 听 | 听 | 听 | 2025 | 听 | 听 | 听 | 2024 | 听 |
| Revenues: | 听 | 听 | 听 | 听 | 听 | 听 | 听 | ||||||||
| Product revenue, net | $ | 12,455 | 听 | 听 | $ | 8,615 | 听 | 听 | $ | 21,181 | 听 | 听 | $ | 16,804 | 听 |
| Total revenue | 听 | 12,455 | 听 | 听 | 听 | 8,615 | 听 | 听 | 听 | 21,181 | 听 | 听 | 听 | 16,804 | 听 |
| Operating expenses: | 听 | 听 | 听 | 听 | 听 | 听 | 听 | ||||||||
| Cost of sales | $ | 1,702 | 听 | 听 | $ | 1,495 | 听 | 听 | $ | 2,787 | 听 | 听 | $ | 2,122 | 听 |
| Research and development | 听 | 91,940 | 听 | 听 | 听 | 77,401 | 听 | 听 | 听 | 164,683 | 听 | 听 | 听 | 146,021 | 听 |
| Selling, general and administrative | 听 | 45,869 | 听 | 听 | 听 | 35,536 | 听 | 听 | 听 | 87,396 | 听 | 听 | 听 | 66,550 | 听 |
| Total operating expenses | 听 | 139,511 | 听 | 听 | 听 | 114,432 | 听 | 听 | 听 | 254,866 | 听 | 听 | 听 | 214,693 | 听 |
| Loss from operations | 听 | (127,056 | ) | 听 | 听 | (105,817 | ) | 听 | 听 | (233,685 | ) | 听 | 听 | (197,889 | ) |
| Interest income, net | 听 | 14,513 | 听 | 听 | 听 | 8,120 | 听 | 听 | 听 | 30,600 | 听 | 听 | 听 | 17,009 | 听 |
| Other income, net | 听 | 523 | 听 | 听 | 听 | 1,579 | 听 | 听 | 听 | 1,776 | 听 | 听 | 听 | 3,213 | 听 |
| Net loss | $ | (112,020 | ) | 听 | $ | (96,118 | ) | 听 | $ | (201,309 | ) | 听 | $ | (177,667 | ) |
| Net loss per share - basic and diluted | $ | (1.93 | ) | 听 | $ | (1.69 | ) | 听 | $ | (3.49 | ) | 听 | $ | (3.14 | ) |
| Weighted-average number of common shares used in computing net loss per share 鈥� basic and diluted | 听 | 57,932,576 | 听 | 听 | 听 | 56,802,546 | 听 | 听 | 听 | 57,697,193 | 听 | 听 | 听 | 56,593,011 | 听 |
Contacts:
Investor Contact
Morgan Sanford, VP, Investor Relations
Agios Pharmaceuticals
[email protected]
Media Contact
Eamonn Nolan, Senior Director, Corporate Communications
Agios Pharmaceuticals
[email protected]
FAQ
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