Agios Reports Business Highlights and Third Quarter 2024 Financial Results
Agios Pharmaceuticals reported Q3 2024 financial results and business highlights. The company achieved $9.0 million in PYRUKYND® net revenue, a 4% increase from Q2. Significant developments include completing enrollment for Phase 3 RISE UP study in sickle cell disease and receiving $1.1 billion in payments following FDA approval of vorasidenib. The company reported net income of $947.9 million and ended the quarter with $1.7 billion in cash and equivalents. R&D expenses decreased to $72.5 million, while SG&A expenses increased to $38.5 million.
Agios Pharmaceuticals ha riportato i risultati finanziari e i punti salienti aziendali del terzo trimestre 2024. L'azienda ha raggiunto 9,0 milioni di dollari di ricavi netti da PYRUKYND®, con un aumento del 4% rispetto al secondo trimestre. Sviluppi significativi includono il completamento dell'arruolamento per lo studio di Fase 3 RISE UP sulla malattia falciforme e la ricezione di 1,1 miliardi di dollari in pagamenti a seguito dell'approvazione da parte della FDA di vorasidenib. L'azienda ha riportato un reddito netto di 947,9 milioni di dollari e ha chiuso il trimestre con 1,7 miliardi di dollari in contante e equivalenti. Le spese R&D sono diminuite a 72,5 milioni di dollari, mentre le spese SG&A sono aumentate a 38,5 milioni di dollari.
Agios Pharmaceuticals reportó los resultados financieros y los aspectos destacados del negocio del tercer trimestre de 2024. La empresa logró 9,0 millones de dólares en ingresos netos de PYRUKYND®, un aumento del 4% con respecto al segundo trimestre. Los desarrollos significativos incluyen la finalización de la inscripción para el estudio de Fase 3 RISE UP en enfermedad de células falciformes y la recepción de 1,1 mil millones de dólares en pagos tras la aprobación de la FDA de vorasidenib. La compañía reportó un ingreso neto de 947,9 millones de dólares y cerró el trimestre con 1,7 mil millones de dólares en efectivo y equivalentes. Los gastos de I+D disminuyeron a 72,5 millones de dólares, mientras que los gastos de SG&A aumentaron a 38,5 millones de dólares.
Agios Pharmaceuticals는 2024년 3분기 재무 결과 및 비즈니스 하이라이트를 보고했습니다. 회사는 PYRUKYND®에서 900만 달러의 순 수익을 달성했으며, 이는 2분기 대비 4% 증가한 수치입니다. 주요 발전사항으로는 세포 겸상병에 대한 3상 RISE UP 연구의 등록 완료와 vorasidenib의 FDA 승인 이후 11억 달러의 지급을 받았다는 점이 있습니다. 회사는 9억 4천790만 달러의 순이익을 기록하였고, 분기를 17억 달러의 현금 및 현금성 자산으로 마무리했습니다. 연구개발(R&D) 비용은 7천250만 달러로 감소하였고, SG&A 비용은 3천850만 달러로 증가하였습니다.
Agios Pharmaceuticals a publié les résultats financiers et les points saillants de l'entreprise pour le troisième trimestre 2024. L'entreprise a réalisé un chiffre d'affaires net de 9,0 millions de dollars avec PYRUKYND®, soit une augmentation de 4 % par rapport au deuxième trimestre. Les développements importants incluent la finalisation de l'inscription à l'étude de Phase 3 RISE UP sur la maladie de cellules falciformes et la réception de 1,1 milliard de dollars de paiements suite à l'approbation de la FDA pour le vorasidenib. L'entreprise a déclaré un revenu net de 947,9 millions de dollars et a terminé le trimestre avec 1,7 milliard de dollars en liquidités et équivalents. Les dépenses de R&D ont diminué à 72,5 millions de dollars, tandis que les dépenses SG&A ont augmenté à 38,5 millions de dollars.
Agios Pharmaceuticals berichtete über die finanziellen Ergebnisse und Geschäftshighlights des dritten Quartals 2024. Das Unternehmen erzielte 9,0 Millionen Dollar an Nettoumsätzen mit PYRUKYND®, was einem Anstieg von 4 % im Vergleich zum zweiten Quartal entspricht. Zu den bedeutenden Entwicklungen gehört der Abschluss der Rekrutierung für die Phase-3-Studie RISE UP zu Sichelzellenkrankheit und der Erhalt von 1,1 Milliarden Dollar an Zahlungen nach der FDA-Zulassung von vorasidenib. Das Unternehmen meldete einen Nettogewinn von 947,9 Millionen Dollar und schloss das Quartal mit 1,7 Milliarden Dollar an Bargeld und Äquivalenten ab. Die F&E-Ausgaben sanken auf 72,5 Millionen Dollar, während die SG&A-Ausgaben auf 38,5 Millionen Dollar stiegen.
- Received $1.1 billion in payments from Royalty Pharma and Servier
- Net income of $947.9 million in Q3 2024
- Strong cash position of $1.7 billion
- PYRUKYND® revenue increased 4% quarter-over-quarter to $9.0 million
- R&D expenses decreased from $81.8M to $72.5M year-over-year
- SG&A expenses increased by 49% year-over-year to $38.5 million
- Phase 3 ACTIVATE-KidsT trial did not meet prespecified statistical criterion
Insights
The Q3 results reveal significant financial strengthening with the receipt of
The net income of
The completion of Phase 3 RISE UP study enrollment for mitapivat in sickle cell disease represents a important milestone, with topline data expected in late 2025. The initiation of tebapivat's Phase 2b study in lower-risk MDS, coupled with FDA Orphan Drug Designation, strengthens the company's rare disease portfolio.
The ACTIVATE-KidsT trial results, while not meeting statistical significance, showed clinically meaningful transfusion reduction responses. The expanding patient base for PYRUKYND®, with 211 unique patients enrolled and 127 on therapy, demonstrates growing market acceptance. The upcoming sNDA filing for thalassemia indication could significantly expand the drug's market potential.
– Completed Enrollment of the Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease; Topline Data from 52-Week Study Expected in Late 2025 –
– Received
of Vorasidenib –
– Commenced Enrollment of the Phase 2b Study of Tebapivat in Lower-Risk Myelodysplastic Syndromes (LR-MDS); Granted FDA Orphan Drug Designation for Treatment of MDS –
– PYRUKYND® (Mitapivat) Net Revenue of
CAMBRIDGE, Mass., Oct. 31, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today reported business highlights and financial results for the third quarter ended September 30, 2024.
“We had a strong quarter, marked by several important advancements across our pipeline. We completed enrollment of our Phase 3 RISE UP study of mitapivat in sickle cell disease, on our way to sharing topline results in late 2025. Our Phase 2b study of tebapivat in lower-risk MDS was initiated, and we received orphan drug designation from the FDA to support the development of tebapivat in this indication,” said Brian Goff, chief executive officer of Agios. “Our cash position was further strengthened by the receipt of
Third Quarter 2024 and Recent Highlights
- PYRUKYND® Revenues: Generated
$9.0 million in net revenue for the third quarter of 2024, a 4 percent increase from the second quarter of 2024, primarily driven by increased patient demand. A total of 211 unique patients have completed prescription enrollment forms, representing an increase of 5 percent over the second quarter of 2024. A total of 127 patients are on PYRUKYND® therapy, inclusive of new prescriptions and continued therapy. - Sickle Cell Disease: Completed enrollment of the 52-week Phase 3 RISE UP study of mitapivat in sickle cell disease, with more than 200 patients enrolled worldwide, and topline data expected in late 2025.
- Lower-risk Myelodysplastic Syndromes:
- Initiated patient enrollment in the Phase 2b study of tebapivat (AG-946).
- Granted Orphan Drug Designation from the FDA to tebapivat for the treatment of myelodysplastic syndromes (MDS).
- Presented topline data from Agios’ Phase 3 ACTIVATE-KidsT trial of mitapivat in pediatric patients with PKD.
- Observed transfusion reduction response rates were higher in the mitapivat arm compared to placebo and were clinically meaningful despite not meeting prespecified statistical criterion for the primary endpoint; secondary endpoints of transfusion-free response and normal hemoglobin response were observed only in the mitapivat arm.
- Safety was consistent with the profile observed in adults with PK deficiency who are regularly transfused.
- Presented data from the Phase 3 ENERGIZE trial as an encore session at the 19th Annual Academy for Sickle Cell and Thalassemia (ASCAT) Conference.
- Corporate Development:
- The FDA approved Servier’s vorasidenib for the treatment of IDH-mutant diffuse glioma. As a result, Agios received a
$200 million milestone payment from Servier and a$905 million payment from Royalty Pharma in connection with the purchase agreement announced in May 2024. - Entered into a distribution agreement with NewBridge Pharmaceuticals to advance commercialization of PYRUKYND® in the Gulf Cooperation Council (GCC) region. NewBridge, a leading specialty company headquartered in Dubai, will commercialize PYRUKYND® in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia and the United Arab Emirates.
- The FDA approved Servier’s vorasidenib for the treatment of IDH-mutant diffuse glioma. As a result, Agios received a
Key Upcoming Milestones & Priorities
Agios expects to achieve the following key milestones by the end of 2024:
- Thalassemia: File sNDA for mitapivat in thalassemia based on the positive results from the Phase 3 ENERGIZE and ENERGIZE-T clinical trials.
- Present additional clinical data at the 66th Annual American Society of Hematology (ASH) Congress, taking place December 7-10, 2024, in San Diego.
Third Quarter 2024 Financial Results
Revenue: Net product revenue from sales of PYRUKYND® for the third quarter of 2024 was
Cost of Sales: Cost of sales for the third quarter of 2024 was
Research and Development (R&D) Expenses: R&D expenses were
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were
Net Income (Loss): Net income was
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of September 30, 2024, were
Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss third quarter 2024 financial results and recent business highlights. The live webcast can be accessed under “Events & Presentations” in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company's website beginning approximately two hours after the event.
About Agios
Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company's deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndromes (MDS)-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera. For more information, please visit the company’s website at www.agios.com.
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® (mitapivat), tebapivat (AG-946), TMPRSS6 siRNA and AG-181, Agios’ PAH stabilizer; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, tebapivat, and AG-181; Agios’ use of proceeds from the transaction with Royalty Pharma; potential U.S. net sales of vorasidenib and potential future royalty payments; Agios’ strategic vision and goals, including its key milestones for 2024 and 2025; and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” 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’ 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’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ 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’ 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’ 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’ 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’ 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 TMPRSS6 siRNA, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ 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’ 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) | |||||||||
| September 30, 2024 | December 31, 2023 | ||||||||
| Cash, cash equivalents, and marketable securities | $ | 1,660,646 | $ | 806,363 | |||||
| Accounts receivable, net | 3,118 | 2,810 | |||||||
| Inventory | 26,429 | 19,076 | |||||||
| Total assets | 1,791,794 | 937,118 | |||||||
| Stockholders' equity | 1,626,672 | 811,019 | |||||||
| Consolidated Statements of Operations Data | |||||||||||||||
| (in thousands, except share and per share data) | |||||||||||||||
| (Unaudited) | |||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Revenues: | |||||||||||||||
| Product revenue, net | $ | 8,964 | $ | 7,399 | $ | 25,768 | $ | 19,720 | |||||||
| Total revenue | 8,964 | 7,399 | 25,768 | 19,720 | |||||||||||
| Operating expenses: | |||||||||||||||
| Cost of sales | $ | 783 | $ | 633 | $ | 2,905 | $ | 2,295 | |||||||
| Research and development | 72,455 | 81,841 | 218,476 | 218,037 | |||||||||||
| Selling, general and administrative | 38,537 | 25,822 | 105,087 | 84,598 | |||||||||||
| Total operating expenses | 111,775 | 108,296 | 326,468 | 304,930 | |||||||||||
| Loss from operations | (102,811 | ) | (100,897 | ) | (300,700 | ) | (285,210 | ) | |||||||
| Gain on sale of contingent payments | 889,136 | — | 889,136 | — | |||||||||||
| Milestone payment from gain on sale of oncology business | 200,000 | — | 200,000 | — | |||||||||||
| Interest income, net | 13,059 | 8,375 | 30,068 | 24,720 | |||||||||||
| Other income, net | 1,651 | 1,198 | 4,864 | 4,342 | |||||||||||
| Net income (loss) before taxes | 1,001,035 | (91,324 | ) | 823,368 | — | (256,148 | ) | ||||||||
| Income tax expense | 53,120 | — | 53,120 | — | |||||||||||
| Net income (loss) | $ | 947,915 | $ | (91,324 | ) | $ | 770,248 | $ | (256,148 | ) | |||||
| Net income (loss) per share - basic | $ | 16.65 | $ | (1.64 | ) | $ | 13.58 | $ | (4.61 | ) | |||||
| Net income (loss) per share - diluted | $ | 16.22 | $ | (1.64 | ) | $ | 13.38 | $ | (4.61 | ) | |||||
| Weighted-average number of common shares used in computing net income (loss) per share – basic | 56,939,403 | 55,803,663 | 56,709,318 | 55,559,766 | |||||||||||
| Weighted-average number of common shares used in computing net income (loss) per share – diluted | 58,432,796 | 55,803,663 | 57,581,382 | 55,559,766 | |||||||||||
Contacts:
Investor Contact
Chris Taylor, VP, Investor Relations and Corporate Communications
Agios Pharmaceuticals
IR@agios.com
Media Contact
Eamonn Nolan, Senior Director, Corporate Communications
Agios Pharmaceuticals
media@agios.com
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