myelodysplastic anemia

Novo Nordisk has ended one of the multiple trials testing the lead oral drug from its $1.1 billion Forma Therapeutics buyout , according to an update to the US federal trials database this week. The Danish drug giant terminated a Phase 2 trial of etavopivat for anemia in adults with various forms of myelodysplastic syndromes, or MDS, according to an update to the US clinical trials site on Thursday. In patients with MDS, red blood cells don’t mature or become healthy ones, and the cancer can progress into leukemia. The drug is still being tested in mid – and late-stage trials for patients with sickle cell disease and thalassemia. Novo made the decision after a “futility assessment concluded low likelihood of the trial showing meaningful patient benefit,” according to an update to the Fortitude trial registry. “This decision was not taken lightly and reflects a judgement of low likelihood that the Fortitude trial would generate benefit for patients with MDS,” a Novo spokesperson said in an emailed statement to Endpoints News . MDS wasn’t highlighted in Novo’s deal announcement in fall 2022, but it was nonetheless another patient population that the pharmaceutical company had hoped to treat with the selective pyruvate kinase-R activator. Patients with sickle cell disease and thalassemia are the main populations for which Novo is developing the once-daily medicine. “We remain confident in etavopivat’s promise and are committed to its continued development in sickle cell disease (SCD) and thalassemia,” the spokesperson said. “The termination of the Fortitude trial does not diminish our belief in etavopivat’s potential to make a significant impact on patients’ lives.” Novo highlighted the asset’s potential in MDS in its annual report in January. Agios Pharmaceuticals is also testing a PK activator, dubbed tebapivat, for MDS-associated anemia. Agios’ more advanced PK activator, mitapivat, is approved for adult PK deficiency and marketed as Pyrukynd. Bristol Myers Squibb’s Reblozyl is indicated for MDS-related anemia. Since the acquisition, Novo has offloaded some of the pipeline at Forma, a drug developer that had touted “ the science of giving a damn. ” It sold a preclinical cancer candidate to Debiopharm and a Phase 1 prostate cancer drug to Pathos AI .

– Reported Positive Topline Data from Phase 3 ENERGIZE-T Study; Expect to File sNDA Based on ENERGIZE and ENERGIZE-T Studies Encompassing All Thalassemia Subtypes by End of 2024 – – Announced $905 Million Purchase Agreement for Vorasidenib Royalty with Royalty Pharma; Agios to Receive a Total of $1.1 Billion in Payments Upon FDA Approval of Vorasidenib – – Reported Results from Phase 3 ACTIVATE-KidsT Study of Mitapivat in Children with Pyruvate Kinase (PK) Deficiency Who are Regularly Transfused – – PYRUKYND® (Mitapivat) Net Revenue of $8.6 Million in Q2; Cash, Cash Equivalents and Marketable Securities of $645.3 Million as of June 30, 2024 – CAMBRIDGE, Mass., Aug. 01, 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 second quarter ended June 30, 2024. “Based on the positive data generated in the Phase 3 ENERGIZE and ENERGIZE-T studies, mitapivat is the first therapy to demonstrate efficacy in all subtypes of thalassemia, and we look forward to filing for FDA review by the end of the year,” said Brian Goff, chief executive officer at Agios. “This morning, we were pleased to report topline data in the Phase 3 ACTIVATE-KidsT study of mitapivat, which is the first study to report safety and efficacy data in children with PK deficiency. We continue to make significant progress toward our vision of becoming a leading rare disease company with a potential multi-billion-dollar franchise in PK activation. Finally, we were pleased to bolster our cash position through a purchase agreement with Royalty Pharma for our vorasidenib royalty, with Agios now positioned to receive a total of $1.1 billion in payments upon FDA approval of vorasidenib.” Second Quarter 2024 and Recent Highlights PYRUKYND® Revenues: Generated $8.6 million in net revenue for the second quarter of 2024, a 5 percent sequential increase from the first quarter of 2024, primarily driven by increased patient demand. A total of 201 unique patients have completed prescription enrollment forms, representing an increase of 7 percent over the first quarter of 2024. A total of 128 patients are on PYRUKYND® therapy, a 7 percent increase from the first quarter of 2024.Thalassemia: Met the primary and all key secondary endpoints in the Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia.Presented positive results from the Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent thalassemia in a plenary session at the European Hematology Association 2024 (EHA2024) Hybrid Congress. Pediatric PK Deficiency: Announced topline data from the Phase 3 ACTIVATE-KidsT study of mitapivat in children with PK deficiency who are regularly transfused. Agios plans to present a more detailed analyses of the results at an upcoming medical meeting.Completed enrollment of the Phase 3 ACTIVATE-Kids study of mitapivat in children with PK deficiency who are not regularly transfused. Topline data from this study are expected in 2025. Corporate Development: Announced a $905 million purchase agreement with Royalty Pharma for Agios’ rights to its vorasidenib royalty. Under the agreement, Agios will receive a payment of $905 million upon approval of vorasidenib by the FDA and Royalty Pharma will receive the entirety of the 15% royalty on annual U.S. net sales of vorasidenib up to $1 billion, and a 12% royalty on annual U.S. net sales greater than $1 billion. Agios retains a 3% royalty on annual U.S. net sales greater than $1 billion. Agios retains rights to a $200 million milestone payment from Servier upon FDA approval of vorasidenib.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. Key Upcoming Milestones & Priorities Agios expects to execute on the following additional key milestones and priorities 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 trials (year-end).Sickle Cell Disease: Complete enrollment in the Phase 3 portion of the RISE UP study of mitapivat (year-end).Lower-risk Myelodysplastic Syndromes: Dose first patient in Phase 2b study of tebapivat (AG-946) (mid-year).Other: Potential approval of Servier’s vorasidenib for the treatment of IDH-mutant diffuse glioma. The FDA has assigned a PDUFA action date of August 20, 2024. Second Quarter 2024 Financial Results Revenue: Net product revenue from sales of PYRUKYND® for the second quarter of 2024 was $8.6 million, compared to $6.7 million for the second quarter of 2023. Cost of Sales: Cost of sales for the second quarter of 2024 was $1.5 million. Research and Development (R&D) Expenses: R&D expenses were $77.4 million for the second quarter of 2024, compared to $68.9 million for the second quarter of 2023. The year-over-year increase was primarily attributable to an increase in costs associated with the in-licensed siRNA TMPRSS6 program for polycythemia vera. Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $35.5 million for the second quarter of 2024 compared to $30.4 million for the second quarter of 2023. The year-over-year increase was primarily attributable to an increase in commercial-related activities as we prepare for the potential approval of PYRUKYND® in thalassemia. Net Loss: Net loss was $96.1 million for the second quarter of 2024 compared to $83.8 million for the second quarter of 2023. Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2024, were $645.3 million compared to $806.4 million as of December 31, 2023. Agios expects that its cash, cash equivalents and marketable securities together with anticipated product revenue, interest income and payments upon FDA approval of vorasidenib, will provide the financial independence to prepare for potential PYRUKYND® launches in thalassemia and sickle cell disease, and to opportunistically expand our pipeline through both internally and externally discovered assets. Conference Call Information Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second 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, 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®, AG-946 (tebapivat) and AG-181; the potential FDA approval of vorasidenib; 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 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 milestone or royalty payments related to the sale of its oncology business or 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) June 30, 2024 December 31, 2023Cash, cash equivalents, and marketable securities $645,296 $806,363Accounts receivable, net 3,762 2,810Inventory 23,937 19,076Total assets 773,063 937,118Stockholders' equity 660,510 811,019 Consolidated Statements of Operations Data(in thousands, except share and per share data)(Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Revenues: Product revenue, net$8,615 $6,712 $16,804 $12,321 Total revenue 8,615 6,712 16,804 12,321 Operating expenses: Cost of sales$1,495 $1,108 $2,122 $1,662 Research and development 77,401 68,895 146,021 136,196 Selling, general and administrative 35,536 30,409 66,550 58,776 Total operating expenses 114,432 100,412 214,693 196,634 Loss from operations (105,817) (93,700) (197,889) (184,313)Interest income, net 8,120 8,254 17,009 16,345 Other income, net 1,579 1,640 3,213 3,144 Net loss$(96,118) $(83,806) $(177,667) $(164,824)Net loss per share - basic and diluted$(1.69) $(1.51) $(3.14) $(2.97)Weighted-average number of common shares used in computing net loss per share – basic and diluted 56,802,546 55,604,330 56,593,011 55,435,796 Contacts: Investor ContactChris Taylor, VP Investor Relations and Corporate CommunicationsAgios PharmaceuticalsIR@agios.com Media ContactDan Budwick1ABdan@1abmedia.com

– ACTIVATE-KidsT is Agios’ First Pediatric Data Readout;Safety Results Consistent with Safety Profile for Mitapivat Previously Observed in Adultswith PK Deficiency Who are Regularly Transfused – – Prespecified Statistical Criterion for the Primary Endpoint in ACTIVATE-KidsT Was Not Met; Results Were Clinically Meaningful, with Observed Response Rates Higher for Mitapivat than Placebo for the Primary Endpoint of Transfusion Reduction Response and for the Secondary Endpoints of Transfusion-Free Response and Normal Hemoglobin Response – – Enrollment Completed for Phase 3 ACTIVATE-Kids Study of Mitapivat in Children with PK Deficiency Who Are Not Regularly Transfused; Topline Data Expected in 2025 – CAMBRIDGE, Mass., Aug. 01, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced topline results from the global Phase 3 ACTIVATE-KidsT study of mitapivat in children aged 1 to <18 years with PK deficiency who are regularly transfused. Using Bayesian methodology, the prespecified statistical criterion for the primary endpoint in ACTIVATE-KidsT was not met using low or moderate borrowing of data from the ACTIVATE-T study in adults. In the ACTIVATE-KidsT study, 28.1% of patients in the mitapivat arm achieved the primary endpoint of transfusion reduction response, compared to 11.8% of patients in the placebo arm. Transfusion-free response and normal hemoglobin response were secondary endpoints in this study and only observed in patients in the mitapivat arm. In the 32-week double-blind treatment period, mitapivat was generally safe and well-tolerated, with safety results consistent with the safety profile for mitapivat previously observed in adults with PK deficiency who are regularly transfused. “After years of working with the PK deficiency community and caregivers whose children have no disease-modifying therapies, it is gratifying to share encouraging efficacy and safety data that may support the potential of a first-ever pediatric treatment for this rare blood disorder,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. “We look forward to completing our pediatric PK deficiency clinical development program next year with the readout of the ACTIVATE-Kids study of mitapivat in children who are not regularly transfused. More broadly, the ACTIVATE-KidsT study represents Agios’ first pediatric data readout. With our focus on lifelong, debilitating rare diseases, we hope that this study will be the first of several pediatric studies to make a positive impact in the lives of children facing rare hemolytic anemias, including PK deficiency, thalassemia and sickle cell disease.” “Children with PK deficiency can experience significant disease burden, including fatigue, the need for blood transfusions, and the risk of iron overload. Symptoms of PK deficiency, disease complications, and common supportive therapies can interfere with regular childhood activities,” said Rachael F. Grace, M.D., MMSc; Dana-Farber/Boston Children’s Cancer and Blood Disorder Center, Harvard Medical School, Boston, an investigator in the ACTIVATE-KidsT study. “The ACTIVATE-KidsT trial is the first study of mitapivat in children who are regularly transfused and demonstrates the potential for meaningful clinical benefit, resolving the anemia and need for transfusions in a subset of children.” In addition, Agios has completed enrollment in the ACTIVATE-Kids study of mitapivat in children with PK deficiency who are not regularly transfused, and expects to report topline data in 2025. Topline results for the Phase 3 ACTIVATE-KidsT study were as follows: A total of 49 patients aged 1 to <18 years were enrolled in the study, with 32 randomized to mitapivat twice-daily and 17 randomized to matched placebo. 30 patients (93.8%) in the mitapivat arm and 16 (94.1%) in the placebo arm completed the 32-week double-blind period of the study.The primary endpoint of the study was transfusion reduction response (TRR), defined as ≥33% reduction in the total red blood cell transfusion volume from Week 9 through Week 32 of the double-blind period normalized by weight and actual study drug duration compared with the historical transfusion volume standardized by weight and to 24 weeks. The analysis of the primary endpoint was based on Bayesian statistical methodology whereby the TRR data from the adult ACTIVATE-T study inform and contribute to the analysis of TRR in the ACTIVATE-KidsT study. The analysis was performed using a range of relative weights of borrowing from the adult ACTIVATE-T study, representing the prior degree of belief in the similarity of the treatment effect in the pediatric and adult populations. The prespecified statistical criterion for the primary endpoint in ACTIVATE-KidsT was not met with low or moderate borrowing weights; however, the results were clinically meaningful.28.1% (9/32) of patients in the mitapivat arm achieved a transfusion reduction response, compared to 11.8% (2/17) of patients in the placebo arm. In addition, a higher proportion of patients in the mitapivat arm compared to the placebo arm achieved the secondary endpoints of transfusion-free response and normal hemoglobin response: 6 patients (18.8%) in the mitapivat arm compared to 0 in the placebo arm had a transfusion-free response, defined as no red blood cell transfusions from Week 9 through Week 32 of the double-blind period.4 patients (12.5%) in the mitapivat arm compared to 0 in the placebo arm achieved a normal hemoglobin response, defined as hemoglobin concentrations within normal limits at least once, 8 weeks or more after a transfusion, from Week 9 through Week 32 of the double-blind period. In the 32-week double-blind treatment period of the study, a similar proportion of patients had adverse events (AEs) in the mitapivat and placebo arms and there were no discontinuations of study treatment due to AEs.Safety results in this pediatric study were consistent with the safety profile for mitapivat previously observed for adult patients with PK deficiency who are regularly transfused. Agios plans to present a more detailed analysis of the Phase 3 ACTIVATE-KidsT data at an upcoming medical meeting. About PYRUKYND® (mitapivat)PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the United States, and for the treatment of PK deficiency in adult patients in the European Union. IMPORTANT SAFETY INFORMATIONAcute Hemolysis: Acute hemolysis with subsequent anemia has been observed following abrupt interruption or discontinuation of PYRUKYND in a dose-ranging study. Avoid abruptly discontinuing PYRUKYND. Gradually taper the dose of PYRUKYND to discontinue treatment if possible. When discontinuing treatment, monitor patients for signs of acute hemolysis and anemia including jaundice, scleral icterus, dark urine, dizziness, confusion, fatigue, or shortness of breath. Adverse Reactions: Serious adverse reactions occurred in 10% of adult patients receiving PYRUKYND in the ACTIVATE trial, including atrial fibrillation, gastroenteritis, rib fracture, and musculoskeletal pain, each of which occurred in 1 patient. In the ACTIVATE trial, the most common adverse reactions including laboratory abnormalities (≥10%) in patients with PK deficiency were estrone decreased (males), increased urate, back pain, estradiol decreased (males), and arthralgia. The adverse reactions reported in the population of adult patients who were regularly transfused (ACTIVATE-T) were consistent with that seen in ACTIVATE. Drug Interactions: Strong CYP3A Inhibitors and Inducers: Avoid concomitant use.Moderate CYP3A Inhibitors: Do not titrate PYRUKYND beyond 20 mg twice daily.Moderate CYP3A Inducers: Consider alternatives that are not moderate inducers. If there are no alternatives, adjust PYRUKYND dosage.Sensitive CYP3A, CYP2B6, CYP2C Substrates Including Hormonal Contraceptives: Avoid concomitant use with substrates that have narrow therapeutic index.UGT1A1 Substrates: Avoid concomitant use with substrates that have narrow therapeutic index.P-gp Substrates: Avoid concomitant use with substrates that have narrow therapeutic index. Hepatic Impairment: Avoid use of PYRUKYND in patients with moderate and severe hepatic impairment. Please see full Prescribing Information and Summary of Product Characteristics for PYRUKYND. About AgiosAgios 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 and MDS-associated anemia. In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera, and a preclinical PAH stabilizer as a potential treatment for phenylketonuria (PKU). For more information, please visit the company’s website at www.agios.com. 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 mitapivat; Agios’ plans for the future clinical development of mitapivat in pyruvate kinase deficiency; and Agios’ strategic plans and prospects. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “would,” “could,” “potential,” “possible,” “hope” 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 or its collaborators 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. Moreover, there can be no guarantee that any medicines ultimately commercialized by Agios will receive commercial acceptance. 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; 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 milestone or royalty payments related to the sale of Agios’ oncology business or 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 proceeds from the transaction with Servier; competitive factors; 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 ContactChris Taylor, VP Investor Relations and Corporate CommunicationsAgios PharmaceuticalsIR@agios.com Media Contact Dan Budwick1AB dan@1abmedia.com