Tebapivat

iStock, f11photo Biopharma companies—including AstraZeneca, BioNTech and Agios—peered farther into the future on the second day of JPM, setting both revenue and R&D targets through the end of the decade. The J.P. Morgan Healthcare Conference is widely known as an annual starting gun for the biopharma industry, giving companies a platform to lay out their strategies for the coming year. On day two, however, companies looked farther afield, giving near-term roadmaps for their businesses and setting sales and clinical targets through the end of the decade. Some takeaways: AstraZeneca Doubles Down on 2030 $80B Goal At JPM, AstraZeneca’s Chief Financial Officer Aradhana Sarin said the company’s goal of hitting $80 billion in total revenue by 2030 is “very much within reach.” To get there, the pharma expects to launch a clutch of high-value products in the near term, including its hypertension drug baxdrostat, for which a new drug application was accepted last month. AstraZeneca’s breast cancer candidate camizestrant and myasthenia gravis drug gefurulimab are also approaching the market. Meanwhile, the pharma also draws confidence from its pipeline, particularly in cancer, for which it is testing eight wholly-owned clinical-stage antibody-drug conjugates (ADCs), as well as two cell therapies with potentially $5 billion in peak revenues. Read more about AstraZeneca at JPM 2026 here . BioNTech Continues Pivot From COVID to Cancer, With PD-1/VEGF at the Core BioNTech continues to move away from COVID-19 and return to its cancer roots. At a company event on Tuesday, the pharma laid out its strategic goals for this year: accelerate its late-stage pipeline, focus on combination therapies and move from a platform-centered to a tumor-driven approach. In a note to investors Tuesday evening, analysts at BMO Capital Markets said that pumitamig, BioNTech’s PD-1/VEGF bispecific antibody, “is at the heart” of the company’s 2026 strategy. Indeed, pumitamig is being studied both as a monotherapy—for breast cancer, hepatocellular carcinoma, colorectal cancer and different types of lung cancers—and as part of a combination regimen with ADCs. BioNTech is expecting a host of early- and late-stage readouts across its pumitamig program this year, as per a Monday release . Among the potential pumitamig indications, “focus remains on the near-term Ph 3 1L [triple-negative breast cancer] China readout,” BMO said. Data from this trial, the analysts added, will help determine if pumitamig could become “a next-generation IO backbone.” For BioNTech, a lot is riding on pumitamig’s success. The drug is under a June 2025 partnership with Bristol Myers Squibb, which has promised up to $7.6 billion in milestones. The PD-1/VEGF space is quickly filling up with rivals, including AbbVie, which put nearly $5 billion on the line this week for ex-China rights to a candidate from RemeGen. As for BioNTech, “With 8 pivotal studies across oncology indications, we could see the company shift into a commercial oncology company within the decade,” BMO said. Cancer AbbVie Bets Nearly $5B on RemeGen To Vie for Position in Hot Bispecific Market The deal, which sees AbbVie paying RemeGen $650 million upfront, gives the pharma ex-China rights to the biotech’s PD-1/VEGF bispecific antibody—a modality being targeted by companies including BMS, Merck and Pfizer. January 12, 2026 · 2 min read · Nick Paul Taylor Agios Eyes $10B Market Potential By 2030 Like AstraZeneca, Agios Pharmaceuticals announced a market target for itself: Unlock a market opportunity of more than $10 billion by 2030. Unlike the big pharma, however, Agios will build toward that opportunity by “evolving from an ultra-rare focus into a broader rare disease portfolio,” analysts at Truist Securities said in a Tuesday note. Indeed, during its company presentation , Agios named its early-stage assets AG-181 for phenylketonuria and AG-236 polycythemia vera as key to hitting its 2030 goal, with each contributing upwards of $1 billion in estimated sales. “Future growth will be driven by assets like AG-236, which extend the core concept of iron dysregulation and hepcidin feedback loops into larger disease populations,” Truist said. The biotech is also working on sickle cell disease, a $3 billion opportunity for which it is developing its pyruvate kinase activators mitapivat and tebapivat. Additionally, Agios is trialing tebapivat for lower-risk myelodyplastic syndromes, which it anticipates will open a $4.5 billion opportunity. This year, Agios will focus its efforts on setting itself up to hit its 2030 target. Key priorities for 2026 include launching its thalassemia drug Aqvesme, approved last month , as well as advancing its early-stage portfolio. Acadia Looks Beyond Analyst Expectations, Stands Against MFN Also looking ahead to the future is Acadia Pharmaceuticals, which announced that by 2028, it expects to report around $1 billion in sales for its antipsychotic Nuplazid and $700 million for the Rett syndrome drug Daybue. Such guidance far outstrips what analysts have forecast for these drugs, according to BMO Capital Markets, which highlighted the “disconnect between Street and internal expectations” in a note on Tuesday. Analysts were expecting $834 million in Nuplazid revenue and $621 million for Daybue. Helping the biotech reach its projected goal is the planned launch of the newly approved STIX formulation of Daybue, an oral solution that is free of dyes and preservatives. The company expects to make Daybue STIX available “on a limited basis” in the first quarter, followed by a broader market debut in the second quarter. Acadia is also planning to expand its Daybue franchise globally—a move that BMO said “may take time,” but will also be “one of the greatest drivers of long-term revenue growth.” Also at JPM, Acadia CEO Catherine Owen Adams said she has formed a group with other small biotechs to lobby for exemptions from the Trump administration’s Most Favored Nation pricing scheme and similar policies that could arise down the line. “I don’t think the administration is out to target small pharma or small biotech particularly. And what we’re trying to do is make sure that they understand that a peanut butter blanket approach to MFN probably would not be the best for U.S. innovation,” Owen Adams told BioSpace . Policy Acadia CEO Bands With Peers To Lobby Against MFN Drug Pricing for Small Biotech Acadia Pharma’s Catherine Owen Adams has formed a group of small- to mid-cap biotechs to advocate against a ‘peanut butter blanket’ approach to drug pricing for small companies. January 13, 2026 · 3 min read · Annalee Armstrong

AQVESME™ (mitapivat) U.S. commercial launch in thalassemia underway following December 2025 FDA approval Pre-sNDA meeting with FDA for mitapivat in sickle cell disease anticipated in first quarter of 2026, with planned U.S. regulatory submission to follow Company progressing early- and mid-stage pipeline in multiple high-value indications Clear path to profitability through the company’s existing commercial presence in thalassemia and PK deficiency, with potential to achieve over $1 billion in peak global sales CAMBRIDGE, Mass. , Jan. 12, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced its 2026 strategic priorities and key milestones anticipated during the year. Members of the company’s management team will present this update at the 44th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14, 2026 , at 8:15 a.m. PT / 11:15 a.m. ET . “In 2025, Agios delivered another year of strong and consistent execution across our portfolio, marking meaningful progress toward our goal of becoming a sustainable and diversified rare disease company,” said Brian Goff , Chief Executive Officer, Agios. “Last year culminated in the historic U.S. approval of AQVESME™ (mitapivat), our pyruvate kinase (PK) activator and the only medicine approved to treat anemia in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. This approval brings a new, disease-modifying oral option to people living with this debilitating and deadly rare blood disorder. “Entering 2026, the company is at an important inflection point. We will deliver a high-impact U.S. launch of AQVESME in thalassemia, seek to expand our PK activation franchise into additional high-value indications such as sickle cell disease and lower-risk myelodysplastic syndromes, and advance our promising early-stage pipeline with the potential to further diversify across hematologic and rare diseases. We also remain focused on disciplined capital allocation and operational efficiency to support our long-term sustainability. With strong momentum and a clear roadmap, Agios enters the year positioned to deliver transformative innovation and meaningful impact for patients living with rare diseases,” Mr. Goff added. Anticipated 2026 MilestonesThalassemia In December 2025 , the U.S. Food and Drug Administration (FDA) approved AQVESME for the treatment of anemia in adults with alpha- or beta-thalassemia. AQVESME is the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. Agios expects AQVESME to become available in the U.S. in late January 2026 following implementation of the AQVESME Risk Evaluation and Mitigation Strategy (REMS) program. Commercial launch activities are already underway and will continue throughout the year. Sickle Cell Disease Topline results from the RISE UP Phase 3 trial of mitapivat in sickle cell disease were reported in November 2025 . Agios anticipates having a pre-supplemental New Drug Application (sNDA) meeting with the FDA in the first quarter of 2026, and plans to submit a U.S. marketing application for mitapivat in sickle cell disease following that engagement. Enrollment in the Phase 2 sickle cell disease trial of tebapivat, Agios’ more potent, once-daily oral PK activator, was initiated in 2025. Agios expects to report topline results from this trial in the second half of 2026. Lower-Risk Myelodysplastic Syndromes (LR-MDS) Enrollment in the Phase 2b LR-MDS trial of tebapivat was completed in 2025. Agios expects to report topline results from this trial in the first half of 2026. Polycythemia Vera (PV) Agios expects to report topline results from a Phase 1 healthy volunteer trial of AG-236, a small interfering RNA (siRNA) targeting TMPRSS6 as a potential treatment for PV, in the first half of 2026. Phenylketonuria (PKU) With dosing completed in the Phase 1 single- and multiple-ascending-dose trial of AG-181, a phenylalanine hydroxylase (PAH) stabilizer, in healthy volunteers, Agios expects to initiate a Phase 1b proof-of-mechanism trial of AG-181 in patients with PKU in the first half of 2026 and to confirm proof of mechanism in the second half of 2026. Presentation at 44th Annual J.P. Morgan Healthcare ConferenceMembers of Agios’ management team will present at the 44th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14, 2026, at 8:15 a.m. PT / 11:15 a.m. ET. 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 archived on the company’s website for at least two weeks following the presentation. About AQVESME™ (mitapivat) U.S. INDICATIONAQVESME is indicated for the treatment of anemia in adults with alpha- or beta-thalassemia. U.S. IMPORTANT SAFETY INFORMATIONBOXED WARNING: HEPATOCELLULAR INJURY AQVESME can cause serious hepatocellular injury. Measure liver laboratory tests (ALT, AST, alkaline phosphatase and total bilirubin with fractionation) at baseline and every 4 weeks for 24 weeks and then as clinically indicated. Avoid use of AQVESME in patients with cirrhosis. Discontinue AQVESME if hepatic injury is suspected.Because of the risk of hepatocellular injury, AQVESME is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the AQVESME REMS. WARNINGS AND PRECAUTIONS Hepatocellular Injury AQVESME can cause hepatocellular injury. Avoid use of AQVESME in patients with cirrhosis. In patients with thalassemia treated with AQVESME, liver injury with and without jaundice has been observed within the first 6 months of exposure. Obtain liver tests (including ALT, AST, alkaline phosphatase, total bilirubin with fractionation) prior to the initiation of AQVESME, then every 4 weeks for the first 24 weeks, and as clinically indicated thereafter. Interrupt AQVESME if clinically significant increases in liver tests are observed or alanine aminotransferase is >5 times the upper limit of normal (ULN). Complete a comprehensive evaluation to rule out other causes of liver injury when drug-induced liver injury is suspected. Discontinue AQVESME if hepatocellular injury due to AQVESME is suspected. Symptoms and signs of early liver injury may mimic those of thalassemia. Advise patients to report new or worsening symptoms of loss of appetite, nausea, right-upper-quadrant abdominal pain, vomiting, scleral icterus, jaundice, or dark urine while on AQVESME treatment. During the double-blind period, 2 of 301 patients (0.66%) with thalassemia treated with AQVESME experienced adverse reactions suggestive of hepatocellular injury. Three additional patients experienced adverse reactions suggestive of hepatocellular injury during the open-label extension periods after switching from placebo to AQVESME. Of these 5 patients, 2 had serious liver injury requiring hospitalization, including 1 patient who developed jaundice (peak bilirubin 32 mg/dL). Another patient developed jaundice (peak bilirubin 4 mg/dL) without requiring hospitalization. These reactions were characterized by a time to onset within the first 6 months of treatment with peak elevations of alanine aminotransferase of >5×ULN with or without jaundice. All patients discontinued treatment with AQVESME, and these reactions improved upon treatment discontinuation. AQVESME REMSAQVESME is available only through a restricted program under a REMS called the AQVESME REMS because of the risk of hepatocellular injury. ADVERSE REACTIONSThe most common adverse reactions among patients taking AQVESME were headache and insomnia. DRUG INTERACTIONS Strong CYP3A Inhibitors and Inducers: Avoid concomitant use. Moderate CYP3A Inhibitors: Avoid concomitant use. Moderate CYP3A Inducers: Consider alternatives that are not moderate inducers. If there are no alternatives, see full Prescribing Information for recommended dosage for drug interactions with moderate CYP3A inducers. Sensitive CYP3A Substrates, including hormonal contraceptives: Avoid concomitant use with substrates that have narrow therapeutic index. CYP2B6, CYP2C, and UGT1A1 Substrates: Monitor patients for efficacy of the substrates with narrow therapeutic index. P-gp Substrates: Monitor patients for adverse reactions of the substrates with narrow therapeutic index. HEPATIC IMPAIRMENT Avoid use of AQVESME in patients with cirrhosis (Child-Pugh Class A, B, or C). Please see full Prescribing Information for AQVESME, including Boxed Warning. 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 StatementsThis 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 Agios’ products, including AQVESME™; Agios’ plans for future meetings with, or submissions to, regulators, including the FDA; its plans for the development of mitapivat, tebapivat, AG-236 and AG-181, and its strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “intend,” “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: 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’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; 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’ 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. Contacts: Investor Contact Morgan Sanford , Vice President, Investor Relations Agios Pharmaceuticals morgan.sanford@agios.com Media Contact Eamonn Nolan , Senior Director, Corporate Communications Agios Pharmaceuticals eamonn.nolan@agios.com Source: Agios Pharmaceuticals, Inc.