Vorasidenib

$12.5 million in second quarter PYRUKYND® (mitapivat) net revenues; ended second quarter with $1.3 billion dollars in cash, cash equivalents and marketable securitiesPYRUKYND sNDA for thalassemia under active review, with FDA PDUFA goal date of September 7, 2025Topline 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 2026Dosed 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. “With fewer than 40 days to our PDUFA goal date, our commercial team is prepared for the potential U.S. approval of PYRUKYND for thalassemia,” said Brian Goff, Chief Executive Officer, Agios. “In 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 with the potential to transform the lives of those affected by rare diseases and deliver long-term shareholder value.” Second Quarter 2025 and Recent Corporate Highlights Commercial Performance – PYRUKYND® (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 – 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 – 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. Tebapivat Sickle Cell Disease – 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 ≥1g/dL increase in hemoglobin concentration from week 10 to week 12, compared to baseline. Lower-risk Myelodysplastic Syndromes (LR-MDS) – Continue to progress patient enrollment in the Phase 2b trial for tebapivat in LR-MDS with target enrollment completion by the end of 2025. 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 30th European Hematology Association Congress. 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 $112.0 million dollars, compared to a net loss of $96.1 million dollars for the second quarter ended June 30, 2024. 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’s second quarter 2025 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company's website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast will be available on the company’s 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 – 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 www.agios.com and follow us on LinkedIn and X. 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-236 and AG-181; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, tebapivat, AG-236 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 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 AG-236, 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) June 30, 2025 December 31, 2024Cash, cash equivalents, and marketable securities $1,339,404 $1,532,031Accounts receivable, net 4,986 4,109Inventory 30,848 27,616Total assets 1,471,237 1,663,199Stockholders' equity 1,369,555 1,540,956 Consolidated Statements of Operations Data(in thousands, except share and per share data)(Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024 Revenues: Product revenue, net$12,455 $8,615 $21,181 $16,804 Total revenue 12,455 8,615 21,181 16,804 Operating expenses: Cost of sales$1,702 $1,495 $2,787 $2,122 Research and development 91,940 77,401 164,683 146,021 Selling, general and administrative 45,869 35,536 87,396 66,550 Total operating expenses 139,511 114,432 254,866 214,693 Loss from operations (127,056) (105,817) (233,685) (197,889)Interest income, net 14,513 8,120 30,600 17,009 Other income, net 523 1,579 1,776 3,213 Net loss$(112,020) $(96,118) $(201,309) $(177,667)Net loss per share - basic and diluted$(1.93) $(1.69) $(3.49) $(3.14)Weighted-average number of common shares used in computing net loss per share – basic and diluted 57,932,576 56,802,546 57,697,193 56,593,011 Contacts: Investor ContactMorgan Sanford, VP, Investor Relations Agios PharmaceuticalsMorgan.Sanford@agios.com Media ContactEamonn Nolan, Senior Director, Corporate CommunicationsAgios PharmaceuticalsEamonn.Nolan@agios.com

Europe’s Committee for Medicinal Products for Human Use has reversed its decision on Eli Lilly's Alzheimer's disease treatment Kisunla, giving it a positive opinion in a limited patient population. Four months after giving Eli Lilly’s Alzheimer’s disease treatment a thumbs-down, Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended it for approval, albeit in a restricted patient population.The positive opinion covers Kisunla's use in patients with early Alzheimer’s symptoms who have one or no copies of the apolipoprotein E ε4 (ApoE4) gene. The drug is not recommended for individuals carrying two copies of the ApoE4 gene. Those patients are more susceptible to amyloid-related imaging abnormalities (ARIA), which are a primary side effect of currently approved Alzheimer’s drugs.The restriction is the same as the one applied by European regulators when it granted marketing authorization to Eisai and Biogen’s Alzheimer’s drug Leqembi three months ago. Those companies went through the same rejection/appeal process as Lilly did with European regulators.Lilly went through the same go-round with regulators in the U.S. before it secured approval of Kisunla in July of last year. Then, earlier this month, the FDA signed off on a new dosing regimen for the treatment, featuring a more gradual titration, which leads to a significantly lower rate of ARIA-related brain swelling. The new dosing regimen also applies to Thursday’s thumbs-up from the CHMP. Endorsement for Gilead’s PrEP injection  The CHMP has also provided a positive opinion on Gilead Sciences' HIV pre-exposure prophylaxis (PrEP) Yeytuo (lenacapavir), a twice-a-year subcutaneous injection which has been hailed as one of the most important breakthroughs in the effort to end the epidemic.The FDA blessed the shot last month under its commercial name Yeztugo.The CHMP recommendation applies to adults and adolescents and came under an accelerated pathway, “because it is considered to be of major public health interest in the EU and the rest of the world,” the regulator said.It was also evaluated under the EU-Medicines for all program, which included participation from the World Health Organization and regulators from several African and Asian nations. The program helps speed approvals where “regulatory capacity may be limited,” the CHMP said.“Lenacapavir for PrEP has the potential to become a critical tool for public health, helping to expand prevention options for people who face the highest barriers to care,” Dietmar Berger, M.D., Ph.D., Gilead’s chief medical officer, said in a release. Other CHMP decisions A month after Supernus Pharmaceuticals made a $561 million bet on Zurzuvae, buying out its developer Sage Therapeutics, the CHMP has registered a positive opinion on the treatment for postpartum depression. The Biogen-partnered treatment was endorsed by the FDA in August 2023, along with a rejection from the regulator in a much larger patient population, major depressive disorder.The European regulator has also signed off on rare disease specialist IntraBio’s Aqneursa for the treatment of the lysosomal storage disorder Niemann-Pick disease type C (NPC). The endorsement comes 10 months after the FDA approved two treatments for NPC in consecutive weeks, Aqneursa and Zevra Therapeutics’ Miplyffa, which has yet to win a nod in Europe.Less than three weeks after the FDA approved KalVista Pharmaceuticals' Ekterly for acute attacks of hereditary angioedema (HAE), the CHMP has followed suit. This is the first oral treatment for HAE recommended for approval in Europe, reducing the burden of injection on patients and speeding up the time between onset of attack and administration of the medicine, the CHMP said. Ono Pharmaceuticals has won a CHMP nod for Romvimza, for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT), a condition where the tissue surrounding the joints and tendons expands abnormally, forming outgrowths of the joint. The drug, which was acquired last year in Ono’s $2.4 billion buyout of Deciphera Pharmaceuticals, was endorsed by the FDA in February of this year, joining Daiichi Sankyo’s Turalio as the only other marketed treatment for TGCT.Ionis Pharmaceuticals' Tryngolza, for the treatment of adults with familial chylomicronemia syndrome, has gotten a thumbs-up from CHMP. The rare, autosomal recessive inherited disorder is characterized by elevated levels of triglycerides in the blood. The FDA approved the treatment in December of last year.Also receiving a CHMP nod is Servier’s Voranigo for low-grade astrocytoma or oligodendroglioma, two rare malignant brain tumors. The FDA blessed the treatment in August of last year.