Heme Arginate in Transplantation Study - a Multi-centre Blinded Parallel-group Randomised Trial of Heme Arginate Versus Placebo to Reduce Delayed Graft Function in Kidney Transplant Recipients

Organ shortage for transplant has necessitated use of kidneys from older donors, increasing the chance that the kidney will not work immediately or for as long as expected. The investigators gave the drug heme arginate (HA) to 20 kidney transplant patients in the first 24 hours after transplant, and showed that it may reduce kidney injury and is safe. The investigators plan to conduct a large study recruiting 600 patients to determine whether HA treatment increases the number of kidney transplants that work immediately. If successful, HA may be introduced into clinical practice at the end of this study.
Patients will be invited to take part in the study once listed for a kidney transplant. Further discussions will be had with them when admitted for transplant, and they will be offered the opportunity to participate. Consent will not be taken until the patient is admitted for transplantation. Following consent, participants will be randomised to receive either 2 doses of the study drug, HA, or a salt water solution, one at the time of transplant, and one approximately 24 hours later. Otherwise, treatment will be the same as any other patient undergoing a kidney transplant. Information about recovery from surgery, and specifically about kidney function, will be collected, but will not require additional blood tests. The study period ends after the first 7 days post-transplant, although longer term data will be collected from routine follow up appointments. Participants will be asked to complete a simple quality of life questionnaire 3 times: just before transplant, at approximately one week and three months after transplant.

Start Date12 Mar 2019

Sponsor / Collaborator

The University of Edinburgh

NCT02314780

/ CompletedPhase 2IIT

Pilot Trial: The Effects of Intravenous Heme Arginate on HO-1 Expression and Oxidative Stress in the Human Heart

Ischemia reperfusion injury may be attenuated by HO-1 induction. Our previous data confirmed strong HO-1 induction in peripheral blood cells following heme arginate infusion in healthy humans. Furthermore, we could demonstrate the amelioration of experimental ischemia reperfusion injury in the calf musculature by heme arginate in healthy subjects as measured by functional MRI.
Therefore, we propose that HO-1 induction in the human heart may be a suitable target to mitigate cardiac ischemia-reperfusion injury.
The HO-1 induction will be assessed in a clinical trial by myocardial biopsy prior to and after aortic cross-clamping in subjects with or without preceding heme arginate treatment in two different dosages. The HO-1 expression will also be measured in the clinical trials in peripheral blood mononuclear cells. As additional outcome, levels of myoglobin, creatine-kinase and troponin T and reactive oxygen species will be measured in plasma according to standard laboratory procedures.

Start Date29 Mar 2015

Sponsor / Collaborator

Medical University of Vienna

[+1]

NCT02142699

/ CompletedPhase 2IIT

Phase II Study of Heme Arginate in Patients Planned for Cardiac Surgery

A recent analysis of over 4500 cardiac surgical patients at the Royal Infirmary of Edinburgh has confirmed that acute kidney injury (AKI) is not only a relatively common post-operative complication but is associated with prolonged hospital stay, and increased risk of death. There is currently no specific therapy available except supportive care.
In laboratory studies, heme arginate (HA), a drug licensed for human use, has been shown to upregulate the anti-inflammatory enzyme hemeoxygenase-1 (HO-1) and protect aged mice from acute kidney injury.
This study will bring this research into the human arena. It will aim to evaluate the minimum effective dose of HA and verify its safety in this specific group of patients. This will be the next step in investigating if HA could be a potential protective treatment for reducing AKI in patients about to have cardiac surgery.
Patients who are due to have cardiac surgery and are aged 60 or above will be approached for inclusion in the study. If agreeable, they will be randomly assigned to receive either HA at a dose of 1mg/kg or 3mg/kg. There will be 10 patients in each group.
Blood tests will be taken just before the study drug is given, at 6 hours, 24 hours and 7 days post dose. These samples will be used to examine the effect of HA on HO-1 at different doses, and will verify drug safety. Any adverse effects of the drug will be evaluated, although HA has an excellent safety profile when used as it is currently licensed for acute porphyria. Urine samples will also be collected to assess inflammation and quantify urinary biomarkers of AKI. This will set the scene for a randomised clinical trial of HA in cardiac surgical patients at high risk of AKI.

Start Date17 Oct 2014

Sponsor / Collaborator

The University of Edinburgh

[+2]

100 Clinical Results associated with Hemin

100 Translational Medicine associated with Hemin

100 Patents (Medical) associated with Hemin

2,984

Literatures (Medical) associated with Hemin

01 Nov 2026·FREE RADICAL BIOLOGY AND MEDICINE

Heme oxygenase-1/CO signaling suppresses NLRP3 inflammasome–mediated stromal fibrosis in primary myelofibrosis

Article

Author: Broggi, Giuseppe ; Giallongo, Sebastiano ; Ferrigno, Jessica ; Caltabiano, Rosario ; Duminuco, Andrea ; La Spina, Enrico ; Di Rosa, Michelino ; Marsala, Gabriella ; Tibullo, Daniele ; Del Fabro, Vittorio ; Li Volti, Giovanni ; Longhitano, Lucia ; Nicolò Conti, Giuseppe ; Giallongo, Cesarina ; Pricoco, Elisabetta P ; Orlando, Laura ; Palumbo, Giuseppe Alberto ; Barbagallo, Ignazio Alberto ; Calabrese, Vittorio

Inflammation and oxidative stress are critical drivers of bone marrow (BM) fibrosis in primary myelofibrosis (MF). Here, we investigated the interplay between the heme oxygenase-1 (HO-1)/carbon monoxide (CO) pathway and NLRP3 inflammasome activation in MF microenvironment. Bioinformatic analysis of patient datasets (GSE53482 and GSE41812) revealed an approximately two-fold reduction of HO-1 expression in CD34⁺ peripheral blood cells compared with healthy controls (p < 0.01). Consistently, JAK2V617F-mutated cell lines (HEL, SET2) displayed respectively lower HO-1 and higher NLRP3 expression than JAK2 wild-type UT7 cells. Ruxolitinib, a potent pharmacological inhibitor of JAK/STAT pathway, restored HO-1 (≈1.7-fold increase) and reduced NLRP3 by ∼50% (p < 0.01). Similarly, in mesenchymal stromal cells (HS-5), tumor-conditioned medium induced a two-fold increase in NLRP3 and Caspase-1 and a ∼40% decrease in HO-1. Pharmacological induction of HO-1 by hemin (10 μM) or CO-releasing molecule CORM-A1 (25 μM) restored HO-1 (∼1.8-fold) and reduced NLRP3 activation (∼50%), limiting αSMA expression and collagen deposition by ∼55-60%. In a TPO^high zebrafish model, nlrp3, caspase-a, and il1β were upregulated ≈2.5-fold, while hmox1a/b decreased by ∼50%; CORM-A1 co-treatment reversed these effects, increasing hmox1a (∼1.7-fold) and decreasing cd41 and col1a1 (∼45%). NLRP3 inhibition by MCC950 (1 μM) produced similar results. Consistently, BM biopsies from MF patients showed ∼65% lower HO-1 expression (p < 0.001) and nuclear BACH1 localization in megakaryocytes. In conclusion, our results demonstrate that HO-1/CO signaling suppresses NLRP3 inflammasome activation and fibrotic remodeling, suggesting that this pathway may represent a potential therapeutic target in MF.

01 Nov 2026·EXPERIMENTAL NEUROLOGY

The LncRNA CRNDE/miR-152-3p/ROCK1 axis aggravates neurological deficits and neuroinflammation in intracerebral hemorrhage

Article

Author: Xiao, Junli ; Zuo, Haiyan ; Yan, Weichen ; Wang, Ming

OBJECTIVE:

Intracerebral hemorrhage (ICH) is a fatal cerebrovascular disease. This study explored the intrinsic relationship between ICH and lncRNA CRNDE.

METHODS:

Injecting the lentiviral interference vector (sh-CRNDE) into the ICH mice (induced by collagenase). Subsequently, the neurological deficits were evaluated through Garcia scoring, the corner turn test, and the water maze test. The levels of CRNDE and inflammatory factors in the brain tissue were detected by RT-qPCR and ELISA, respectively. In vitro experiments, microglia and neuronal cells were respectively cultured with hemoglobin and Hemin. Subsequently, the level of M1 polarization markers was detected by RT-qPCR. In neuronal cells, the proliferation activity was evaluated by CCK-8. The apoptosis level was comprehensively assessed based on the results of flow cytometry and the level of LDH. Finally, the effects of inhibiting the CRNDE/miR-152-3p axis on ROCK1 and the phenotype of ICH were re-verified in animal models and cell models.

RESULTS:

In ICH mice, inhibition of CRNDE prevented the neurological deficits and neuroinflammation. Blocking CRNDE also significantly alleviated the M1 polarization of microglia and the apoptosis of neuronal cells. CRNDE sponges miR-152-3p through the ceRNA mechanism and regulates ROCK1. Inhibition of miR-152-3p or overexpression of ROCK1 weakens the inhibitory effect of sh-CRNDE on neurological deficits, M1 polarization, and the apoptosis of neurons. Moreover, in cell models, inhibition of ROCK1 showed the same protective effect as CRNDE downregulation.

CONCLUSION:

In ICH, CRNDE upregulates ROCK1 by adsorbing miR-152-3p. This regulatory axis exacerbates neuroinflammation and neurofunctional disorders.

01 Nov 2026·FREE RADICAL BIOLOGY AND MEDICINE

CKLF1 upregulation exacerbates secondary brain injury after intracerebral hemorrhage by promoting neuronal ferroptosis and oxidative stress

Article

Author: Chen, Qianxue ; Tian, Yihao ; Yang, Shangwen ; Zhang, Jianwen ; Hua, Qiuwei ; Liu, Yupei ; Cai, Qiang ; Jiang, Hongxiang ; Zhang, Xinyi

BACKGROUND:

Secondary brain injury (SBI) following spontaneous intracerebral hemorrhage (ICH) is a critical determinant of neurological outcome. It is closely associated with neuronal death, and inflammatory responses. However, current research on SBI after ICH remains limited, and effective therapeutic targets are still lacking.

METHODS:

In this study, a collagenase-induced intracerebral hemorrhage (ICH) model was employed, and experiments were conducted 24 h after hemorrhage induction. For in vitro experiments, differentiated PC12 cells were stimulated with hemin and the CKLF1 agonist peptide C27 to evaluate the potential of CKLF1 to induce neuronal ferroptosis. Mechanistically, the CKLF1 antagonist peptide C19, the CCR5 inhibitor Maraviroc (MVC), and a p38 MAPK inhibitor were applied, in combination with immunoprecipitation (Co-IP) assays and overexpression of p53 site mutation, to elucidate the molecular mechanisms underlying CKLF1-induced ferroptosis in PC12 cells. In vivo, the effects of CKLF1 knockdown on secondary brain injury after ICH were assessed using quantitative real-time PCR (qPCR), Western blotting (WB), immunohistochemistry (IHC), and immunofluorescence (IF) analyses.

RESULTS:

Mechanistic studies revealed that CKLF1 binds to its receptor CCR5 to activate p38 MAPK, which promotes ferroptosis by regulating p53 phosphorylation and nuclear translocation, while the mutation of p53 partially reversed the effect. This signaling cascade leads to the downregulation of ferroptosis-suppressing proteins (SLC7A11 and GPX4) and the upregulation of pro-ferroptotic proteins (COX2 and ACSL4), thereby exacerbating lipid peroxidation and oxidative stress. Conversely, AAV-mediated knockdown of CKLF1 significantly suppressed this ferroptotic signaling pathway in vivo/vitro, reduced iron deposition and neuronal ferroptosis in perihematomal tissue, improved blood-brain barrier (BBB) integrity, inhibited microglial activation, and enhanced both neurological function scores and 7-day survival rates in ICH rats.

CONCLUSION:

The present study identifies CKLF1 as a previously unrecognized regulator of neuronal ferroptosis following ICH through the CCR5/p38/p53 signaling pathway. These findings provide new mechanistic insights into ICH pathogenesis and highlight CKLF1 as a promising therapeutic target for precision treatment of hemorrhagic stroke.

News (Medical) associated with Hemin

30 Jul 2026

·www.biospace.com

Alnylam Pharmaceuticals Reports Second Quarter 2026 Financial Results and Highlights Recent Period Progress

− Achieved Second Quarter 2026 Global Net Product Revenues of $1,172 Million (74% Growth Compared with Q2 2025), Driven Primarily by Total TTR Revenues of $1,030 Million (89% Growth Compared with Q2 2025) – − Revises Full-Year 2026 TTR Net Product Revenue Guidance from $4,400 to $4,700 Million to $4,200 to $4,500 Million (75% Growth Compared with 2025 at Revised Midpoint) – − Presented New Data from HELIOS-B at Heart Failure 2026 Demonstrating Vutrisiran's Consistent Clinical Benefit Across Patient Populations – − Advanced Pipeline with Phase 2 Initiations of ALN-6400 in Von Willebrand Disease and Mivelsiran in Down Syndrome-Associated Alzheimer's Disease; Results from Phase 1 Trial of ALN-HTT02 in Patients with Huntington's Disease to be Presented at EHDN – ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); − Accelerated Integration of AI Across Alnylam by Establishing Strategic Collaborations with Inceptive to Transform RNAi Discovery and a Large Health Care System in California to Support Earlier Identification of ATTR-CM in Routine Care, as well as Expanding Partnership with Komodo Health to Scale Commercial Intelligence – − Entered Into an Exclusive Agreement with BeOne Medicines for Commercialization of AMVUTTRA in China – CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the second quarter ended June 30, 2026, and reviewed recent business highlights. “During the first half of 2026, we continued to meaningfully advance our business, generating over $1 billion in quarterly product revenues for the first time in our history during the first quarter and, building on that momentum, over $1 billion in TTR revenues during the second quarter. These results underscore the growing leadership and global impact of our TTR franchise in transforming outcomes for patients with ATTR amyloidosis, with AMVUTTRA being the only product approved for the full spectrum of the disease. We have lowered our TTR product sales guidance for full-year 2026 to reflect learnings from the initial phase of our launch in the evolving ATTR-CM market, in particular the normalization of growth in second line volume after satisfying pent-up demand from patients waiting for a new therapy. Given the strong foundation we have established and continued growth in ATTR-CM diagnosis and treatment, we remain confident in the trajectory of our ongoing ATTR-CM launch and are continuing to invest robustly in this franchise, as we bring AMVUTTRA to more patients and establish it as a foundational therapy,” said Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam. “During the second quarter, we also continued to advance our high-value pipeline with the initiation of two Phase 2 studies, ALN-6400 in von Willebrand disease and mivelsiran in Down syndrome-associated Alzheimer’s disease, while progressing multiple additional programs toward important clinical readouts later this year. Together, these achievements demonstrate our continued progress against our Alnylam 2030 strategy and our commitment to creating long-term value through scientific innovation and patient impact.” Second Quarter 2026 and Recent Significant Business Highlights Total TTR: AMVUTTRA ® (vutrisiran) & ONPATTRO ® (patisiran) Achieved global net product revenues for AMVUTTRA and ONPATTRO for the second quarter of $1,012 million and $18 million, respectively, together representing $1,030 million in total TTR net product revenues and 89% total TTR growth compared to Q2 2025. U.S. TTR net product revenues increased $106 million compared with Q1 2026 with the growth driven by a $129 million increase in demand, partially offset by approximately $20 million in inventory impact and a modest reduction in net price. The growth in demand in the quarter was more than double the growth in demand in Q1 2026 compared with Q4 2025. International TTR net product revenues increased $14 million compared with Q1 2026 driven primarily by increased demand in both hATTR-PN and ATTR-CM across international markets. Announced a collaboration with a large health care system in California to support their study, DETECT-ATTR, evaluating Invision Precision Cardiac Amyloid, an AI-enabled, FDA-cleared, echocardiography-based screening approach for the detection of cardiac amyloidosis. DETECT-ATTR will be conducted within one of the nation's largest integrated healthcare systems, serving more than 4.5 million members, with the intention of addressing underdiagnosis and improving disease recognition in clinical practice. Presented new analyses from the HELIOS-B Phase 3 clinical trial of vutrisiran in patients with ATTR-CM at Heart Failure 2026, the annual congress of the Heart Failure Association of the European Society of Cardiology: Reductions in all-cause mortality and recurrent cardiovascular events were maintained across key subgroups of patients taking a broad range of heart failure therapies. A pooled analysis of over 25,000 patient-years of experience with TTR-silencing RNAi therapies shows a consistent safety profile, including no clinical meaningful ocular effects of vitamin A lowering. Shared the design and rationale of the DemonsTTRate study, a global, prospective, observational study evaluating real-world outcomes in patients with ATTR-CM. The study is expected to enroll more than 2,000 patients and follow them for up to five years, generating longitudinal data on clinical outcomes, treatment patterns and healthcare utilization across routine clinical practice. Continued to expand the global reach of AMVUTTRA with a recent launch in Spain, and a new commercial partnership with BeOne Medicines to distribute AMVUTTRA in mainland China and Macao, subject to AMVUTTRA receiving marketing authorization. Total Rare: GIVLAARI ® (givosiran) & OXLUMO ® (lumasiran) Achieved global net product revenues for GIVLAARI and OXLUMO for the second quarter of $90 million and $52 million, respectively, together representing $142 million in total Rare net product revenues and 11% total Rare growth compared to Q2 2025. Other Highlights Initiated a Phase 2 clinical trial of ALN-6400 , an investigational RNAi therapeutic targeting plasminogen, in adult and adolescent female patients with von Willebrand disease and heavy menstrual bleeding. Initiated a Phase 1 clinical trial of ALN-6222 , an investigational RNAi therapeutic targeting inhibin E (INHBE), in adult patients with obesity. Advanced mivelsiran, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) for the treatment of cerebral amyloid angiopathy (CAA) and Alzheimer’s disease. Completed enrollment in the cAPPricorn-1 Phase 2 clinical trial in patients with CAA. Initiated a Phase 2 clinical trial in patients with Down syndrome-associated Alzheimer's disease. Shared additional Phase 1 data in early-onset Alzheimer's disease at the Alzheimer's Association International Conference (AAIC) 2026. An analysis of safety data from single- and multiple-doses of mivelsiran showed no evidence of increased risk of amyloid-related imaging abnormality (ARIA) events. Results also showed robust, durable reductions in cerebrospinal fluid (CSF) soluble amyloid beta precursor protein (sAPPβ) and amyloid beta 42 (Aβ42), with up to 30 months of treatment exposure. The most common adverse events (AEs) were procedural pain and procedural headache, and no serious or severe AEs were deemed related to study drug. Presented preclinical data and Phase 1 design details at AAIC 2026 for ALN-5288 , an investigational RNAi therapeutic targeting microtubule-associated protein tau (MAPT) for Alzheimer's disease and tauopathies. Our collaboration partner, Regeneron Pharmaceuticals, Inc., announced that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have accepted regulatory applications for cemdisiran to treat adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. Regeneron indicated that the FDA will review the New Drug Application (NDA) under Priority Review with a target action date in November 2026, following use of a Priority Review Voucher, and that a decision from the European Commission is anticipated in the second half of 2027. Additional Business Updates Announced a strategic collaboration agreement with Inceptive Nucleics, Inc. designed to increase the pace of RNAi therapeutic innovation. The alliance pairs Alnylam's RNAi platform and 20+ years of proprietary data with Inceptive's foundation models and AI expertise to catalyze progress beyond rational drug design. Expanded the Company's strategic partnership with Komodo Health to scale Marmot, Komodo's analytics AI platform, across key enterprise functions at Alnylam. Appointed Benjamin Franklin Cravatt III, Ph.D., to Alnylam's Board of Directors. Published 2025 Corporate Responsibility Report. Key Upcoming Events Alnylam announces today that it will present initial results from the Phase 1 clinical trial of ALN-HTT02 in patients with Huntington's disease at the European Huntington's Disease Network (EHDN) Clinical Research Congress on Friday, October 23, 2026, in Kraków, Poland. The Company continues to host its 10 th RNAi Roundtable series this year, during which Alnylam R&D leaders, as well as medical thought leaders, will discuss the progress and opportunity across key pipeline programs of investigational RNAi therapeutics. Upcoming RNAi Roundtables include: Zilebesiran : Targeting Angiotensinogen to Achieve Continuous Control of Blood Pressure Thursday, September 17, 10:30 am ET ALN-HTT02 : Targeting Exon 1 of the Huntington Gene to Reduce Progression of Huntington's Disease Monday, October 26, 10:00 am ET In the second half of 2026, Alnylam expects to announce clinical data from additional pipeline programs, including: Results from Phase 1 and Phase 2 clinical trials of ALN-6400 in healthy volunteers and patients with hereditary hemorrhagic telangiectasia (HHT), respectively. Results from a Phase 1 clinical trial of ALN-2232 in obesity and weight management. Second Quarter 2026 Financial Results Three Months Ended June 30, % Change (In thousands, except per share amounts and percentages) 2026 2025 Total revenues $ 1,290,948 $ 773,689 67 % GAAP Income (loss) from operations $ 231,441 $ (16,199 ) ** Non-GAAP Income from operations $ 318,066 $ 95,481 233 % GAAP Net income (loss) $ 164,494 $ (72,228 ) ** Non-GAAP Net income $ 251,801 $ 38,171 * GAAP Net income (loss) per common share — basic $ 1.23 $ (0.55 ) ** GAAP Net income (loss) per common share — diluted $ 1.21 $ (0.55 ) ** Non-GAAP Net income per common share — basic $ 1.88 $ 0.29 * Non-GAAP Net income per common share — diluted $ 1.84 $ 0.28 * * Indicates the percentage change period over period is greater than 500% ** Not meaningful For an explanation of our use of non-GAAP financial measures, refer to the “Use of Non-GAAP Financial Measures” section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measure, see the tables at the end of this press release. Revenue Summary Three Months Ended June 30, % Change % Change at CER* (In thousands, except percentages) 2026 2025 Net product revenues: AMVUTTRA $ 1,011,762 $ 491,953 106 % 106 % ONPATTRO 18,461 52,538 (65 )% (65 )% Total TTR net product revenues 1,030,223 544,491 89 % 89 % GIVLAARI 89,764 80,849 11 % 10 % OXLUMO 52,122 46,872 11 % 9 % Total Rare net product revenues 141,886 127,721 11 % 10 % Total net product revenues 1,172,109 672,212 74 % 74 % Net revenues from collaborations: Roche 41,888 18,267 129 % 129 % Regeneron Pharmaceuticals 5,020 32,542 (85 )% (85 )% Other 257 10,687 (98 )% (98 )% Total net revenues from collaborations 47,165 61,496 (23 )% (23 )% Royalty revenue 71,674 39,981 79 % 79 % Total revenues $ 1,290,948 $ 773,689 67 % 67 % * Change at constant exchange rates, or CER, represents growth calculated as if exchange rates had remained unchanged from those used during the three months ended June 30, 2025. CER is a non-GAAP financial measure. Total Net Product Revenues Total net product revenues increased 74%, both at actual currency and at CER during the three months ended June 30, 2026, compared to the same period in 2025. The increase was primarily due to growth from AMVUTTRA revenues driven by increased patient demand, mainly in patients with ATTR-CM in the U.S., and growth from an increased number of patients on GIVLAARI and OXLUMO, which was partially offset by a decreased number of patients on ONPATTRO. Net Revenues from Collaborations Net revenues from collaborations decreased during the three months ended June 30, 2026, as compared to the same period in 2025, due to lower revenue recognized under our Regeneron collaboration, partially offset by increased revenue under our Roche collaboration driven by higher reimbursable development activities related to the ZENITH Phase 3 clinical trial of zilebesiran. Royalty Revenue Royalty revenue increased during the three months ended June 30, 2026, as compared to the same period in 2025, due to increased volume and rate of royalties earned from global net sales of Leqvio by Novartis. Operating Expense Summary Three Months Ended June 30, % Change (In thousands, except percentages) 2026 2025 Cost of goods sold $ 298,261 $ 142,029 110 % % of net product revenues 25.4 % 21.1 % Cost of collaborations and royalties $ 190 $ 924 (79 )% GAAP Research and development expenses $ 413,134 $ 323,621 28 % Non-GAAP Research and development expenses $ 377,240 $ 274,069 38 % GAAP Selling, general and administrative expenses $ 347,922 $ 323,314 8 % Non-GAAP Selling, general and administrative expenses $ 297,191 $ 261,186 14 % Cost of Goods Sold Cost of goods sold as a percentage of net product revenues increased during the three months ended June 30, 2026, as compared to the same period in 2025, primarily as a result of increased sales of AMVUTTRA and an associated increase in the blended royalty rate payable on net sales of AMVUTTRA. Research & Development (R&D) Expenses GAAP and non-GAAP R&D expenses for the three months ended June 30, 2026 increased as compared to the same period in 2025, primarily due to increased clinical trial expenses for the ZENITH Phase 3 clinical trial of zilebesiran, the TRITON-CM Phase 3 clinical trial of nucresiran in patients with ATTR-CM and the TRITON-PN Phase 3 clinical trial of nucresiran in patients with hATTR-PN.. Selling, General & Administrative (SG&A) Expenses GAAP and non-GAAP SG&A expenses for the three months ended June 30, 2026 increased as compared to the same period in 2025, primarily due to increased marketing investment associated with the ongoing global commercial launch of AMVUTTRA in ATTR-CM. Other Financial Highlights Interest expense Interest expense for the three months ended June 30, 2026 of $82 million included interest of $53 million attributed to the liability related to the sale of future Leqvio royalties and $26 million attributed to the liabilities related to the vutrisiran and zilebesiran development funding. Provision for income taxes During the three months ended June 30, 2026, we recorded a provision for income taxes of $13 million, primarily related to U.S. state income taxes, utilization of Switzerland net deferred tax assets, as well as taxable income from jurisdictions in which we are subject to tax. We will utilize deferred tax assets in Switzerland to offset current cash tax liabilities and will continue to maintain a full valuation allowance against our net deferred tax assets in the U.S. and certain deferred tax assets in Switzerland. Financial position Cash, cash equivalents and marketable securities were $3.3 billion as of June 30, 2026, as compared to $2.9 billion as of December 31, 2025, with the increase primarily driven by net cash inflows from operating activities. Net cash provided by operating activities for the three months ended June 30, 2026 included $26 million of payments associated with the liability related to the sale of future Leqvio royalties recorded to interest expense, as well as $33 million of payments associated with the liabilities related to vutrisiran and zilebesiran development funding recorded to interest expense. A reconciliation of our GAAP to non-GAAP financial results is included in the tables at the end of this press release. 2026 Financial Guidance Full-year 2026 financial guidance is updated and consists of the following: Item Prior FY 2026 Guidance Updated FY 2026 Guidance Total TTR net product revenues (AMVUTTRA, ONPATTRO) 1 $4,400 million - $4,700 million $4,200 million - $4,500 million Total Rare net product revenues (GIVLAARI, OXLUMO) 1 $500 million - $600 million Reiterate Total net product revenues 1 $4,900 million - $5,300 million $4,700 million - $5,100 million Net product revenues growth vs. 2025 at currency exchange rates as of June 30, 2026 1 64% to 77% 57% to 71% Net product revenues growth vs. 2025 at constant exchange rates 2 64% to 77% 57% to 70% Net revenues from collaborations and royalties $400 million - $500 million $575 million - $625 million Non-GAAP R&D and SG&A expenses 3 $2,700 million - $2,800 million Reiterate 1 Full-year 2026 guidance utilizing currency exchange rates as of June 30, 2026: 1 EUR = 1.14 USD and 1 USD = 162 JPY 2 Representing growth calculated as if the exchange rates had remained unchanged from those used in 2025, which is a non-GAAP financial measure 3 Excludes $300 million - $400 million of stock-based compensation expense from estimated GAAP R&D and SG&A expenses in the prior FY 2026 guidance and $330 million - $380 million of stock-based compensation expense in the updated FY 2026 guidance The change in the Company’s TTR net product revenue guidance reflects an updated outlook for AMVUTTRA in the second line segment of the U.S. market based on learnings as the ATTR-CM launch has progressed. Specifically, growth in second line demand for AMVUTTRA moderated in early 2026 to what the Company now believes is a normalized level, following an early launch period that, with hindsight, benefited from pent-up demand from patients progressing on stabilizers who had been waiting for a new treatment option. Use of Non-GAAP Financial Measures This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains or losses outside the ordinary course of the Company’s business. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies. The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release are stock-based compensation expenses, and realized and unrealized losses on marketable equity securities. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the impact of the realized and unrealized losses on marketable equity securities because the Company does not believe these adjustments accurately reflect the performance of the Company’s ongoing operations for the period in which such gains or losses are reported, as their sole purpose is to adjust amounts on the balance sheet. Percentage changes in revenue growth at CER are presented excluding the impact of changes in foreign currency exchange rates for investors to understand the underlying business performance. The current period’s foreign currency revenue values are converted into U.S. dollars using the average exchange rates from the prior period. The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release. Conference Call Information Management will provide an update on the Company and discuss second quarter 2026 results as well as expectations for the future via conference call on Thursday, July 30, 2026, at 8:30 am ET. A live audio webcast of the call will be available on the Investors section of the Company’s website at . An archived webcast will be available on the Alnylam website approximately two hours after the event. About AMVUTTRA ® (vutrisiran) AMVUTTRA ® (vutrisiran) is a transthyretin (TTR) silencer that delivers rapid knockdown of TTR at the source to address the underlying cause of transthyretin amyloidosis (ATTR). In a clinical study, AMVUTTRA rapidly knocked down TTR in as early as six weeks and decreased TTR levels by 87% with two and a half years of treatment. It is approved as a treatment for the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and for the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in various countries, globally. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only silencer approved for the treatment of ATTR-CM and hATTR-PN. For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit AMVUTTRA.com . About ONPATTRO ® (patisiran) ONPATTRO is an RNAi therapeutic that is approved in the United States and Canada for the treatment of adults with hATTR amyloidosis with polyneuropathy. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby reducing the production of TTR protein before it is made. Reducing the pathogenic protein leads to a reduction in amyloid deposits in tissues. For more information about ONPATTRO, including full Prescribing Information, visit ONPATTRO.com . About GIVLAARI ® (givosiran) GIVLAARI (givosiran) is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved in the United States and Brazil for the treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is also approved in the European Union for the treatment of AHP in adults and adolescents aged 12 years and older. In the pivotal trial, GIVLAARI was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or intravenous hemin administration at home compared to placebo. Contacts Alnylam Pharmaceuticals, Inc. Bo Piela (Media) 508-308-9783 Josh Brodsky (Investors) 617-551-8276 Read full story here

13 May 2026

·www.biospace.com

RECORDATI: STRONG MOMENTUM OF THE GROUP CONTINUES INTO THE FIRST QUARTER OF 2026 NET REVENUE +4.9%, EBITDA(1) +5.0%, ADJUSTED NET INCOME(2) +7.2%

Consolidated net revenue of € 713.4 million in the first quarter of 2026, +4.9% or +8.7% on a like-for-like basis (3) at constant exchange rates (CER); adverse FX impact of € 29.1 million (-4.3%) EBITDA (1) of € 283.6 million, +5.0%, margin on net revenue of 39.7% Adjusted net income (2) of € 188.1 million, +7.2%, margin on net revenue of 26.4% Net income of € 153.1 million, +22.4% Free cash flow (4) of € 92.1 million, -€ 66.7 million vs prior year Net debt (5) at € 1,985.2 million, just below 2.0x EBITDA FY 2026 financial targets confirmed Primary endpoint met in pasireotide (Signifor ® ) Phase 2 trial for the treatment of post-bariatric hypoglycemia (PBH); FDA meeting scheduled to discuss next steps Sutimlimab (Enjaymo ® ) to advance into a Phase 3 trial for the treatment of chronic immune thrombocytopenia (ITP) Performance share plan 2023-2025: assessment of the level of achievement of the performance targets for the first cycle (2023 grant) Performance share plan 2026-2028: assignment of rights provided under the first cycle (subject to certain conditions) Milan, 12 th May 2026 – The Board of Directors of Recordati S.p.A. approved the Group’s Interim Report as of 31 st March 2026, representing additional voluntary financial reporting ( 6 ) . The report was prepared using the assessment, measurement and recognition criteria prescribed by international accounting standards (IFRS). The financial statements as of 31 st March 2026 will be available by 15 th May 2026 at the company’s offices and on the company’s website ( ) and can also be viewed on the authorised storage system 1Info ( ). Rob Koremans, Chief Executive Officer of Recordati, commented: “Recordati has delivered a strong start to the year, driven by excellent momentum in Rare Diseases and resilient in-market growth of the Specialty & Primary Care promoted portfolio. We are pleased to report further progress in advancing our R&D pipeline. We see significant opportunity for pasireotide in PBH following positive Phase 2 results and are progressing sutimlimab into a Phase 3 study in ITP. Isturisa ® continues to perform very well, with ongoing expansion in the U.S. across both overt and non-overt Cushing’s syndrome populations. Overall, we remain well positioned to deliver on our strategic and financial objectives for 2026 and beyond.” Q1 2026 Financial highlights Consolidated net revenue for the first quarter of 2026 was € 713.4 million, up 4.9% or 8.7% on a like-for-like ( 3) basis at CER (+7.9% excluding Türkiye) versus the first quarter of 2025. This was driven, in particular, by the strong momentum from the Rare Diseases business. The adverse FX impact for the first quarter of 2026 was € 29.1 million (-4.3%), mainly driven by the U.S. dollar and Turkish lira devaluation. Rare Diseases revenue was € 292.4 million for the first quarter of 2026, up 14.8%, or 22.4% at CER as compared to the first quarter of 2025, driven by strong volume growth across the Endocrinology and Hemo-Oncology franchises. The Endocrinology franchise achieved net revenue of € 120.7 million, an increase of 38.1%, reflecting continued growth of Isturisa ® (€ 86.3 million, +56.8%), driven mostly by strong new patient uptake across geographies, particularly in the U.S., and growth of Signifor ® (€ 34.4 million, +6.3%). The Hema-Oncology franchise achieved net revenue of € 113.2 million, growing by 18.2%, reflecting the strong momentum of Enjaymo ® across geographies (€ 43.9 million, +37.6%) and Qarziba ® (€ 42.9 million, +13.9%), as well as growth of Sylvant ® (€ 22.8, million +1.8%). The Metabolic franchise achieved net revenue of € 58.5 million, a decrease of 18.3%, reflecting phasing of Carbaglu ® across geographies and slightly lower demand of Panhematin ® in the U.S. against a strong performance in the first quarter of 2025. Specialty & Primary Care revenue was € 404.4 million for the first quarter of 2026, down 1.0% or up 0.2% on a like-for-like basis (3) at CER as compared to the first quarter of 2025 ( 7 ) , reflecting continued in-market growth of the promoted portfolio (+5% (8) ) and some expected one-off headwinds. In particular, the Cardiovascular franchise achieved net revenue of € 113.7 million, an increase of 1.5%, and the Gastrointestinal franchise achieved net revenue of € 69.4 million, an increase of 2.1%, with continued good in-market performance of key products in both therapeutic areas. The Urology franchise achieved net revenue of € 104.4 million, a decrease of 4.3%, due to a high prior-year base following a product relaunch in Russia in 2025, and the Cough & Cold franchise achieved net revenue of € 29.6 million, a decrease of 12.8% due to a weaker season in key markets. EBITDA ( 1) was € 283.6 million for the first quarter of 2026, up 5.0% compared to the first quarter of 2025, with margin of 39.7% of net revenue. Strong revenue performance and the positive mix effect at gross profit level was partially offset by a higher level of investments to support the U.S. expansion, primarily for Isturisa ® , the continued development of Enjaymo ® , ongoing geographic expansion in Rare Diseases as well as the launch of Vazkepa ® in Specialty & Primary Care. Adjusted operating income ( 9 ) was €231.1 million in the first quarter of 2026, an increase of 5.4% versus the first quarter of 2025. This represents 32.4% of net revenue, compared with 32.2% in the prior year, supported by strong operating performance. Operating income was € 229.6 million in the first quarter of 2026, up 17.3% over the first quarter of 2025, reflecting gross margin-related non-cash charges of € 22.4 million in 2025, arising mostly from the unwind of the fair value step up of the acquired Enjaymo ® inventory. Non-recurring costs were € 1.5 million versus € 1.1 million in the first quarter of 2025. Financial expenses were € 28.9 million, down by € 2.1 million compared to the previous year. Net exchange losses over the period were € 1.9 million, slightly higher as compared to losses of € 1.8 million in the first quarter of 2025. The impact of hyperinflation was negative € 2.0 million, the same as in the first quarter of 2025. Adjusted net income (2) was € 188.1 million, 26.4% of net revenues, up by 7.2% compared to the same period of 2025, benefitting from higher adjusted operating income, lower financial expenses and a lower tax rate resulting from a positive country mix. Net income was € 153.1 million, 21.5% of net revenue, an increase of 22.4% versus the prior year, reflecting positive operating income, lower financial expenses and a lower income tax rate versus the first quarter of 2025. Free cash flow ( 4) was € 92.1 million for the first quarter of 2026, a decrease of € 66.7 million versus the first quarter of 2025, reflecting higher EBITDA which was offset by higher working capital absorption and income taxes paid. Net debt (5) as of March 31, 2026 was € 1,985.2 million, or leverage of just below 2.0x EBITDA, compared to net debt of € 2,037.3 million on December 31, 2025. Shareholders’ equity was € 2,060.3 million. Pipeline Development The Phase 2 trial evaluating pasireotide for the treatment of post-bariatric hypoglycemia met its primary endpoint with a dose-dependent and significant increase in glucose levels during a standardized meal test (p<0.02) (10) . This was associated with a lowering of level 2 and 3 hypoglycemia (not significant), particularly in patients with higher baseline hypoglycemia rates (post-hoc analysis). Recordati is scheduled to meet with the FDA to discuss potential next steps. Immune thrombocytopenia (ITP) is a rare autoimmune disease, characterized by increased platelet destruction and decreased platelet production/release. Main symptoms include an increased risk of bleeding events, fatigue, decreased quality of life, increased risk of thrombosis (increased morbidity and mortality). Refractory ITP represents a significant unmet need, with around 20-30% failing several lines of therapy. On the basis of encouraging FDA feedback as well as early encouraging clinical evidence showing that sutimlimab, by targeting the classical complement pathway, can lead to a rapid and sustained platelet response in patients refractory to multiple lines of treatment, Recordati has decided to advance sutimlimab into a pivotal registrational Phase 3 trial for the treatment of chronic ITP. The other lifecycle management programs are progressing in line with plans. Corporate Development On January 29, 2026, Recordati announced a collaboration and license agreement with Moderna to develop and commercialize worldwide mRNA-3927, an investigational product for the treatment of propionic acidemia (PA). Under the terms of the agreement, Moderna will continue to lead the development of mRNA-3927, in collaboration with Recordati, and if approved, Recordati will lead global commercialization. mRNA-3927 is a post proof-of-concept, investigational product aimed to restore propionyl-CoA carboxylase (PCC) enzyme activity in patients with propionic acidemia. If approved, this could be the first disease-modifying treatment option on the market for this severe disease. mRNA-3927 is currently being evaluated in a potential registrational clinical study. The target patient enrollment has been reached, with a potential data readout expected by the end of 2026. Business outlook The Group confirms its financial targets for full year 2026 as follows: Net revenue between € 2,730 and € 2,800 million with FX headwind of ~-3.5% EBITDA (1) between € 995 and € 1,030 million; margin of +/- 36.5% with FX headwind of ~-4.0% Adjusted net income (2) between € 655 and € 685 million; margin of +/- 24.0% The full year 2027 targets ( 1 1 ) remain unchanged, with strong organic growth complemented by bolt-on business development and M&A. Additional resolutions Performance share plan 2023-2025: assessment of the level of achievement of the performance targets for the first cycle (2023 grant) With reference to the incentive plan of Recordati S.p.A. (“ Recordati ” or the “ Company ”) named the “ 2023–2025 Performance Share Plan ” (the “ Plan ”), approved by the Shareholders’ Meeting of the Company held on 21 st April 2023, and to the related first award cycle resolved upon by the Board of Directors on 27 th June 2023, notice is hereby given that, on today’s date, the Board of Directors of the Company, acting upon the proposal of the Remuneration and Appointments Committee, to the extent within its remit, and in accordance with the provisions of the relevant rules, has assessed the level of achievement of the performance targets relating to the first cycle of the Plan. On 27 th June 2026, being the date on which the vesting period will be completed, the beneficiaries’ entitlement to receive up to a maximum aggregate number of 388,630 ordinary shares of Recordati, granted free of charge pursuant to the Plan, will vest. Such number may be reduced in accordance with the terms and conditions of the Plan, including in the event of termination of the relationship with the Recordati Group prior to such date. Within the above maximum aggregate number, the shares attributable to the Chief Executive Officer of Recordati amount to no. 30,485. For further information on the Plan (12) , reference should be made to the relevant Information Document, which is available on the Company’s website in the “Governance” section, under “Remunerations”. Performance share plan 2026-2028: assignment of rights provided under the first cycle (subject to certain conditions) With reference to the “2026–2028 Performance Share Plan” (the “ Plan ”) of Recordati approved by its shareholders’ meeting on 29 th April 2026, notice is hereby given that today the Board of Directors of the Company, upon proposal of the Company’s Remuneration and Nominations Committee – within the scope of its competence and in accordance with the provisions of the relevant regulations – resolved to grant to the beneficiaries of the Plan a total of no. 528,913 rights (the “ Rights ”) to receive ordinary shares of the Company, which fall within the first allocation cycle of the Plan. The Chief Executive Officer of Recordati has been assigned no. 24,251 Rights. The grant of the Rights will cease to have any effect in case the potential voluntary tender offer on all ordinary shares of the Company aimed at its delisting - communicated on 25 March 2026 (13) by CVC to the Company by means of a conditional non-binding indication of interest - is launched and the delisting is completed by the date of the Annual General Meeting approving the 2026 financial statements. For further information on the Plan (1 2 ) , reference should be made to the relevant Information Document, which is available on the Company’s website in the “Governance” section, under “Remunerations”. (1) Net income before income taxes, financial income and expenses, depreciation, amortization and write-downs of property, plant and equipment, intangible assets and goodwill, non-recurring items and non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS . (2) Net income excluding amortization and write-downs of intangible assets (except software) and goodwill, non-recurring items, non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3, monetary net gains/losses from hyperinflation (IAS 29), net of tax effects . (3) Proforma growth calculated excluding revenue of Vazkepa® for Q1 2026 and Cardicor ® for Q1 2025 and Q1 2026 (Specialty & Primary Care) . (4) Total cash flow excluding financing items, milestones, dividends, purchases of treasury shares net of proceeds from exercise of stock options. (5) Cash and cash equivalents, less bank debts and loans, which include the measurement at fair value of hedging derivatives. ( 6 ) Italian Legislative Decree 25/2016, which implements Directive 2013/50/EU, no longer stipulates the submission of an interim management report, which was previously required in terms of paragraph 5 of Art. 154-ter of Legislative Decree 58/1998 (7) The 2025 figures have been restated to reflect the reclassification of certain brands from Other Therapeutic a reas to Cardiovascular and Gastrointestinal areas in 2026. The amount of reclassification for Q1 2025 is as follows: €2.5 million from Other Therap eu tic a reas to Cardiovascular and €4.4 million from Other Therapeutic a reas to Gastrointestinal . (8) IQVIA Feb RQ -2026 vs Feb RQ -2025 . ( 9 ) Net income before income taxes, financial income and expenses and non-recurring items, non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3. (10) p=0.0106 (50 µg s.c. pasireotide vs placebo); p=0.0010 (100 µg s.c. pasireotide vs. placebo); p< 0.0001 (200 µg s.c. pasireotide vs placebo) . (1 1 ) FY 2027 targets: Net Revenue €3,000 - €3,200 million, EBITDA €1,140 - €1,225 million, Adjusted Net Income €770- €820 million, excluding potential impact from tariffs and/or most favored nation pricing policies in the U.S. (12) Table no. 1 referred to in paragraph 4.24 of Annex 3A, Form 7, to the Issuers’ Regulation no. 11971/1999, will be published in the manner and within the timeframe set out in Article 84-bis, paragraph 5, letter a), of the same Issuers’ Regulation. (13) Please refer to the press release issued by the Company on this regard on 26th March 2026. Conference Call Recordati will host a conference call on May 13 th , at 2:00 p.m. CET ( 1:00 p.m. GMT+1 ) to present the results for the first quarter of 2026. Please find the pre-registration link here with all the dial-in details and a calendar invitation to follow. Alternatively, if not pre-registered, the dial-in numbers for the conference call are: Italy + 39 02 802 09 11, toll free 800 231 525 UK + 44 1 212818004, toll free (44) 0 800 0156371 USA +1 718 7058796, toll free (1) 1 855 2656958 France +33 1 70918704 Germany +49 6917415712 Participants are invited to dial in 10 minutes before the start of the conference call. If operator assistance is required to connect, please dial *0. The slides that will be referenced during the call will be available at under Investors/Company Presentations. Recordati is an international pharmaceutical Group listed on the Italian Stock Exchange (XMIL: REC), with roots dating back to a family-run pharmacy in Northern Italy in the 1920s. We are uniquely structured to provide treatments across specialty and primary care and rare diseases. Our fully integrated operations span clinical development, manufacturing of active ingredients and finished products, commerciali z ation and licensing. We operate in approximately 150 countries across EMEA, the Americas and APAC , with around 4, 700 employees. We believe that health is a fundamental right, not a privilege. Today, our purpose “Unlocking the full potential of life” aims to empower individuals to live life to the fullest, from common conditions to the rarest. Investor Relations Eugenia Litz Eugenia.Litz@recordati.com Gianluca Saletta saletta.g@recordati.it Media Relations Laura Conti conti.l@recordati.it Rebecca Kerr rebecca.kerr@recordati.com This document contains forward-looking statements relating to future events and future operating, economic and financial results of the Recordati group. By their nature, forward-looking statements involve risk and uncertainty because they depend on the occurrence of future events and circumstances. Actual results may therefore differ materially from those forecast for a variety of reasons, most of which are beyond the Recordati group ’ s control. The information on the pharmaceutical specialties and other products of the Recordati group contained in this document is intended solely as information on the activities of the Recordati Group, and, as such, it is not intended as a medical scientific indication or recommendation, or as advertising . MAIN PRODUCTS NET REVENUE (€ thousands) First quarter 2026 First quarter 2025 Change % Specialty & Primary Care 404,387 408,638 (1.0) Zanidip ® (lercanidipine) and Zanipress ® (lercanidipine+enalapril) 1 53,702 57,737 (7.0) Eligard ® (leuprorelin acetate) 33,604 33,038 1.7 Avodart ® (dutasteride) and Combodart ® /Duodart ® (dutasteride/tamsulosin) 2 26,455 24,529 7.9 Seloken ® /Seloken ® ZOK/Logimax ® (metoprolol/metoprolol+felodipine) 26,426 28,233 (6.4) Urorec ® (silodosin) 21,921 23,065 (5.0) Livazo ® (pitavastatin) 17,830 14,901 19.7 Vazkepa ® (ethyl-icosapent) 5,182 - n.a. Rare Diseases 292,419 254,808 14.8 Isturisa ® (osilodrostat) 86,233 55,009 56.8 Signifor ® / Signifor LAR (pasireotide) 34,442 32,399 6.3 Enjaymo ® (sutimlimab) 43,910 31,913 37.6 Qarziba ® (dinutuximab beta) 42,863 37,644 13.9 Sylvant ® (siltuximab) 22,790 22,393 1.8 (1) of which Zanidip ® € 45.3 million in Q1 2026 and € 50.0 million in Q1 2025 ( 2 ) Trademarks are owned by or licensed to the GSK group of companies RECORDATI GROUP Summary of the consolidated results, prepared in accordance with International Financial Reporting Standards (IFRS) (€ thousands) INCOME STATEMENT First quarter 2026 First quarter 2025 Change % NET REVENUE 713,424 679,960 4.9 Cost of sales (202,598) (221,188) (8.4) GROSS PROFIT 510,826 458,772 11.3 Selling expenses (147,827) (139,742) 5.8 Research and development expenses (86,337) (80,117) 7.8 General and administrative expenses (44,934) (41,648) 7.9 Other income/(expenses), net (2,125) (1,499) 41.8 OPERATING INCOME 229,603 195,766 17.3 Financial income/(expenses), net (28,852) (30,906) (6.6) PRE-TAX INCOME 200,751 164,860 21.8 Income taxes (47,690) (39,819) 19.8 NET INCOME 153,061 125,041 22.4 Adjusted gross profit (1) 510,826 481,165 6.2 Adjusted operating income (2) 231,113 219,236 5.4 Adjusted net income (3) 188,139 175,464 7.2 EBITDA (4) 283,575 270,158 5.0 Net income attributable to: Equity holders of the Parent 153,061 125,041 22.4 Non-controlling interests 0 0 n.s. EARNINGS PER SHARE (euro) Basic (5) 0.750 0.606 23.8 Diluted (6) 0.732 0.598 22.4 (1) Gross profit adjusted from impact of non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3 . (2) Net income before income taxes, financial income and expenses, non-recurring items and non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3 . (3) Net income excluding amortization and write-downs of intangible assets (except software) and goodwill, non-recurring items, non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3 , monetary net gains/losses from hyperinflation (IAS 29), net of tax effects. (4) Net income before income taxes, financial income and expenses, depreciation, amortization and write-downs of property, plant and equipment, intangible assets and goodwill, non-recurring items and non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3 . (5 ) Earnings per share (EPS) are based on average shares outstanding during the respective period, 20 4 . 07 3 . 709 in 202 6 and 20 6 . 355 . 324 in 202 5 . These amounts are calculated deducting treasury shares in the portfolio, the average of which was 5 . 538 . 003 shares in 202 6 and 3. 041 . 684 shares in 202 5 . (6) Diluted earnings per share is calculated by taking into account rights granted to employees. COMPOSITION OF NET REVENUE First quarter 2026 First quarter 2025 Change % Total revenue 713,424 679,960 4.9 Italy 96,750 95,960 0.8 International 616,674 584,000 5.6 RECORDATI GROUP (€ thousands) Reconciliation of Net income to EBITDA (1) First quarter 2026 First quarter 2025 Net income 153,061 125,041 Income taxes 47,690 39,819 Financial income/(expenses), net 28,852 30,906 Non-recurring expenses 1,510 1,077 Non-cash charges from PPA inventory uplift 0 22,393 Adjusted operating income (2) 231,113 219,236 Depreciation, amortization and write-downs 52,462 50,922 EBITDA (1) 283,575 270,158 Reconciliation of Net income to Adjusted Net income (3) First quarter 2026 First quarter 2025 Net income 153,061 125,041 Amortization and write-downs of intangible assets (excluding software) 41,566 40,988 Tax effect (9,607) (9,692) Non-recurring operating expenses 1,510 1,077 Tax effect (386) (286) Non-cash charges from PPA inventory uplift 0 22,393 Tax effect 0 (5,599) Monetary net (gain)/losses from hyperinflation (IAS29) 1,995 2,029 Tax effect 0 (487) Non-recurring tax (income)/expense, net 0 0 Adjusted net income (3) 188,139 175,464 (1) Net income before income taxes, financial income and expenses, depreciation, amortization and write-downs of property, plant and equipment, intangible assets and goodwill, non-recurring items and non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3 . (2) Net income before income taxes, financial income and expenses, non-recurring items and non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3 . (3) Net income excluding amortization and write-downs of intangible assets (except software) and goodwill, non-recurring items, non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3 , monetary net gains/losses from hyperinflation (IAS 29), net of tax effects. RECORDATI GROUP Summary of the consolidated results, prepared in accordance with International Financial Reporting Standards (IFRS) (€ thousands) ASSETS 31.03.2026 31.12.2025 Property, plant and equipment 229,165 222,324 Intangible assets 2,406,550 2,393,448 Goodwill 802,801 795,680 Other equity investments and securities 14,497 16,244 Other non-current assets 10,685 10,259 Deferred tax assets 142,657 136,415 TOTAL NON-CURRRENT ASSETS 3,606,355 3,574,370 Inventories 545,822 539,804 Trade receivables 659,052 570,154 Other receivables 105,676 106,458 Other current assets 29,747 24,591 Derivative instruments measured at fair value 7,423 8,074 Cash and cash equivalents 380,466 428,824 TOTAL CURRENT ASSETS 1,728,186 1,677,905 TOTAL ASSETS 5,334,541 5,252,275 RECORDATI GROUP Summary of the consolidated results, prepared in accordance with International Financial Reporting Standards (IFRS) (€ thousands) EQUITY AND LIABILITIES 31.03.2026 31.12.2025 Share capital 26,141 26,141 Share premium reserve 83,719 83,719 Treasury shares (282,983) (239,379) Reserve for derivative instruments 3,258 (17) Translation reserve (340,729) (348,362) Other reserves 76,777 73,822 Profits carried forward 2,469,827 2,009,007 Net income 153,061 443,624 Interim dividend (128,783) (128,783) Shareholders’ equity attributable to equity holders of the Parent 2,060,288 1,919,772 Shareholders’ equity attributable to non-controlling interests 0 0 TOTAL SHAREHOLDERS’ EQUITY 2,060,288 1,919,772 Loans - due after one year 1,757,101 2,130,296 Provisions for employee benefits 20,614 19,838 Deferred tax liabilities 128,347 129,687 TOTAL NON-CURRENT LIABILITIES 1,906,062 2,279,821 Trade payables 306,896 345,183 Other payables 308,018 257,244 Tax liabilities 116,498 80,572 Other current liabilities 1,685 8,479 Provisions for risks and charges 20,114 19,152 Derivative instruments measured at fair value 5,120 4,862 Loans - due within one year 593,303 313,341 Short-term debts to banks and other lenders 16,557 23,849 TOTAL CURRENT LIABILITIES 1,368,191 1,052,682 TOTAL SHAREHOLDERS’ EQUITY AND LIABILITIES 5,334,541 5,252,275 RECORDATI GROUP Summary of consolidated results prepared in accordance with International Financial Reporting Standards (IFRS) (€ thousands) CASH FLOW STATEMENT First quarter 2026 First quarter 2025 OPERATING ACTIVITIES Net income 153,061 125,041 Income taxes 47,691 39,819 Net interest 22,459 25,370 Depreciation of property, plant and equipment 9,483 8,885 Amortization of intangible assets 42,979 42,037 Write-downs 0 0 Equity-settled share-based payment transactions 4,760 3,981 Other non-monetary components 7,488 28,594 Change in other assets and other liabilities (4,712) (12,745) Cash flow generated/(used) by operating activities before change in working capital 283,209 260,982 Change in: - inventories 1,224 (11,992) - trade receivables (93,277) (79,420) - trade payables (36,307) 39,141 Change in working capital (128,360) (52,271) Interest received 1,045 1,305 Interest paid (31,871) (32,878) Income taxes paid (21,153) (12,349) Cash flow generated/(used) by operating activities 102,870 164,789 INVESTMENT ACTIVITIES Investments in property, plant and equipment (11,172) (5,958) Disposals of property, plant and equipment 418 11 Investments in intangible assets (6,390) (2,574) Disposals of intangible assets 930 150 Sale of non-current assets held for sale 239 0 Acquisition of holdings in other companies 0 0 Cash flow generated/(used) by investment activities (15,975) (8,371) FINANCING ACTIVITIES Opening of loans 0 4 Repayment of loans (96,409) (117,600) Payment of lease liabilities (3,044) (3,185) Change in short-term debts to banks and other lenders 1,132 2,204 Dividends paid (214) (1,086) Purchase of treasury shares (43,952) (49,061) Sale of treasury shares 328 24,671 Cash flow generated/(used) by financing activities (142,159) (144,053) Change in cash and cash equivalents (55,264) 12,365 Opening cash and cash equivalents 428,824 322,423 Currency translation effect 6,906 (1,744) Closing cash and cash equivalents 380,466 333,044 DECLARATION BY THE FINANCIAL REPORTING OFFICER The Financial Reporting Officer, Niccolò Giovannini, declares, pursuant to paragraph 2 of Article 154- bis of the Consolidated Law on Finance, that the accounting information contained in this press release corresponds to the Company’s documentation, books and accounting records.

License out/inPhase 3Financial StatementAcquisitionPhase 2

30 Apr 2026

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Alnylam Pharmaceuticals Reports First Quarter 2026 Financial Results and Highlights Recent Period Progress

− Achieved First Quarter 2026 Global Net Product Revenues of $1,036 Million (121% Growth Compared with Q1 2025), Driven Primarily by Total TTR Revenues of $910 Million (153% Growth Compared with Q1 2025) – − Reiterates 2026 Financial Guidance, Including Combined Net Product Revenues of $4,900 Million to $5,300 Million, and Total TTR Net Product Revenues of $4,400 to $4,700 Million – − New Data Presented at ACC.26 Further Support Use of Vutrisiran as First-Line Treatment for ATTR-CM – − Established Collaborations with Viz.ai and AHA Focused on Earlier Diagnosis, Coordinated Care and Long-Term Patient Impact in ATTR-CM – − Deep Pipeline Progressing Toward Multiple Clinical Readouts During the Second Half of 2026 Expected to Support Long-Term Growth – CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter ended March 31, 2026, and reviewed recent business highlights. “2026 is off to a strong start for Alnylam with the end of the first quarter marking one year since the U.S. launch of AMVUTTRA for ATTR-CM, and also representing a significant financial milestone with the achievement of over $1 billion in quarterly product revenues for the first time in our history,” said Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam. “At the same time, we continued to advance our deep and high-value pipeline of RNAi therapeutics, progressing three ongoing Phase 3 trials, and initiating a Phase 1 study for our first adipose-targeted program for obesity and weight management. In addition, multiple programs across our pipeline are advancing toward key clinical readouts this year. Together, this represents important initial progress toward our Alnylam 2030 goals of achieving global TTR leadership, driving growth through innovation, and scaling with financial discipline as we work to deliver substantial patient impact and create long-term value.” First Quarter 2026 and Recent Significant Business Highlights Total TTR: AMVUTTRA ® (vutrisiran) & ONPATTRO ® (patisiran) Achieved global net product revenues for AMVUTTRA and ONPATTRO for the first quarter of $890 million and $20 million, respectively, together representing $910 million in total TTR net product revenues and 153% total TTR growth compared to Q1 2025. Today announced an update to the TRITON-CM Phase 3 study of nucresiran, an investigational next-generation TTR silencer, in patients with ATTR-CM. Enrollment in the study is proceeding faster than anticipated. Consequently, the Company has decided to utilize a pre-specified protocol option to expand target enrollment from 1,250 to approximately 1,750 patients. While Alnylam is increasing the number of patients who will be enrolled in the trial, given the rapid pace of enrollment, the Company still expects to launch nucresiran, assuming positive data and regulatory approval, in ATTR-CM by 2030. Presented new analyses from the HELIOS-B Phase 3 clinical trial of vutrisiran in patients with ATTR-CM at the American College of Cardiology's Annual Scientific Session and Expo. Vutrisiran improved health-related quality-of-life in patients with ATTR-CM relative to placebo, with the treatment effect corresponding to the difference observed in ATTR-CM patients more than 10 years apart in age. Consistent treatment effects of vutrisiran were observed across the ATTR-CM disease spectrum, including patients with the most advanced disease and diastolic dysfunction. Real-world data demonstrated high adherence to and persistence of quarterly HCP-administered dosing with vutrisiran. Announced new strategic efforts aimed at facilitating earlier diagnosis, coordinated care, and long-term patient impact in ATTR-CM. Alnylam is partnering with Viz.ai to develop an AI-enabled ATTR-CM care pathway designed to assist in the identification of patients earlier in the course of disease and guide appropriate diagnostic evaluation and referral. Alnylam is also supporting a national effort led by the American Heart Association which consists of a three-year initiative that will convene a 10-site cohort of multidisciplinary health systems in a national learning collaborative designed to identify gaps in care, share best practices, and scale effective models for diagnosing and managing ATTR-CM. Hosted a TTR Investor Webinar highlighting Alnylam's progress delivering for patients with ATTR-CM as well as the long-term growth and durability of the Company's flagship TTR franchise. A replay is available here . Total Rare: GIVLAARI ® (givosiran) & OXLUMO ® (lumasiran) Achieved global net product revenues for GIVLAARI and OXLUMO for the first quarter of $74 million and $51 million, respectively, together representing $126 million in total Rare net product revenues and 15% total Rare growth compared to Q1 2025. Other Highlights Presented additional Phase 2 results for zilebesiran , in development for cardiovascular risk reduction in hypertension patients, including assessing the ability to provide continuous control of blood pressure, at the American College of Cardiology's Annual Scientific Session and Expo. A comprehensive analysis of safety across the Phase 2 KARDIA program demonstrated an acceptable safety pro zilebesiran, both as a monotherapy and in combination with standard-of-care antihypertensives, across patients with mild-to-moderate hypertension, those at high CV risk, or with lower eGFR at baseline. Initiated a Phase 1 clinical trial of ALN-2232 , Alnylam's first adipose-targeted RNAi therapeutic, targeting ACVR1C for obesity and weight management. Alnylam's collaboration partner, Regeneron Pharmaceuticals, Inc., announced the submission of a New Drug Application (NDA) to the FDA for cemdisiran, an investigational RNAi therapeutic for adults with generalized myasthenia gravis. Additional global filings are planned for 2026. Additional Business Updates Entered into a research collaboration with Tenaya Therapeutics to discover novel human genetic targets for the potential development of disease-modifying treatments for cardiovascular diseases. Announced a new multi-year agreement with Helix to drive precision medicine development, in which Alnylam will gain access to Helix's deeply phenotyped GenoSphere TM cohorts consisting of comprehensive genomic and longitudinal clinical data, and medical and pharmacy claims that cover a wide range of disease areas. Key Upcoming Events Alnylam will host its 10 th RNAi Roundtable series later this year, at which Alnylam scientists and program leaders, as well as medical thought leaders, will discuss the progress and opportunity across key pipeline programs. A detailed schedule and registration information will be announced separately. In the first half of 2026, Alnylam expects to: Complete enrollment in the cAPPricorn-1 Phase 2 clinical trial of mivelsiran in patients with cerebral amyloid angiopathy. Initiate a Phase 2 trial of mivelsiran in patients with Alzheimer’s disease. Initiate a Phase 2 clinical trial of ALN-6400 in a second bleeding disorder. In the second half of 2026, Alnylam expects to announce clinical data from several pipeline programs, including: Results from Phase 1 and Phase 2 clinical trials of ALN-6400 in healthy volunteers and patients with hereditary hemorrhagic telangiectasia (HHT), respectively. Results from a Phase 1 clinical trial of ALN-HTT02 in patients with Huntington's disease. Results from a Phase 1 clinical trial of ALN-2232 in obesity and weight management. Financial Results for the Quarter Ended March 31, 2026 Three Months Ended March 31, % Change (In thousands, except per share amounts and percentages) 2026 2025 Total revenues $ 1,167,175 $ 594,189 96 % GAAP Income from operations $ 268,636 $ 18,077 * Non-GAAP Income from operations $ 338,790 $ 74,789 353 % GAAP Net income (loss) $ 205,991 $ (18,251) ** Non-GAAP Net income $ 273,038 $ 37,941 * GAAP Net income (loss) per common share — basic $ 1.55 $ (0.14) ** GAAP Net income (loss) per common share — diluted $ 1.51 $ (0.14) ** Non-GAAP Net income per common share — basic $ 2.05 $ 0.29 * Non-GAAP Net income per common share — diluted $ 1.99 $ 0.29 * * Indicates the percentage change period over period is greater than 500% ** Not meaningful For an explanation of our use of non-GAAP financial measures, refer to the “Use of Non-GAAP Financial Measures” section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measure, see the tables at the end of this press release. Revenue Summary Three Months Ended March 31, % Change % Change at CER* (In thousands, except percentages) 2026 2025 Net product revenues: AMVUTTRA $ 889,931 $ 309,992 187 % 183 % ONPATTRO 20,481 49,489 (59 )% (61 )% Total TTR net product revenues 910,412 359,481 153 % 150 % GIVLAARI 74,394 66,968 11 % 7 % OXLUMO 51,321 42,089 22 % 13 % Total Rare net product revenues 125,715 109,057 15 % 10 % Total net product revenues 1,036,127 468,538 121 % 117 % Net revenues from collaborations: Roche 35,641 17,056 109 % 109 % Regeneron Pharmaceuticals 46,336 51,039 (9 )% (9 )% Other 98 31,090 (100 )% (100 )% Total net revenues from collaborations 82,075 99,185 (17 )% (17 )% Royalty revenue 48,973 26,466 85 % 85 % Total revenues $ 1,167,175 $ 594,189 96 % 93 % * Change at constant exchange rates, or CER, represents growth calculated as if exchange rates had remained unchanged from those used during the three months ended March 31, 2025. CER is a non-GAAP financial measure. Total Net Product Revenues Total net product revenues increased 121% and 117% at actual currency and CER, respectively, during the three months ended March 31, 2026, as compared to the same period in 2025, primarily due to growth from AMVUTTRA revenues driven by increased patient demand, mainly in patients with ATTR-CM in the U.S., which was partially offset by a decreased number of patients on ONPATTRO, and due to growth from an increased number of patients on GIVLAARI and OXLUMO. Net Revenues from Collaborations Net revenues from collaborations decreased during the three months ended March 31, 2026, as compared to the same period in 2025, primarily driven by recognition of a $30 million payment in connection with the amendment to our agreement with Vir Biotechnology, Inc. in March 2025. Royalty Revenue Royalty revenue increased during the three months ended March 31, 2026, as compared to the same period in 2025, due to increased volume and rate of royalties earned from global net sales of Leqvio by Novartis. Operating Expense Summary Three Months Ended March 31, % Change (In thousands, except percentages) 2026 2025 Cost of goods sold $ 207,520 $ 70,183 196 % % of net product revenues 20.0 % 15.0 % Cost of collaborations and royalties $ 3,602 $ 858 320 % GAAP Research and development expenses $ 364,866 $ 265,122 38 % Non-GAAP Research and development expenses $ 334,754 $ 241,324 39 % GAAP Selling, general and administrative expenses $ 322,551 $ 239,949 34 % Non-GAAP Selling, general and administrative expenses $ 282,509 $ 207,035 36 % Cost of Goods Sold Cost of goods sold as a percentage of net product revenues increased to 20% during the three months ended March 31, 2026, as compared to the same period in 2025, primarily as a result of increased sales of AMVUTTRA and an associated increase in the blended royalty rate payable on net sales of AMVUTTRA. Research & Development (R&D) Expenses GAAP and non-GAAP R&D expenses for the three months ended March 31, 2026 increased as compared to the same period in 2025, primarily due to increased clinical trial expenses for the ZENITH Phase 3 clinical trial of zilebesiran, the TRITON-CM Phase 3 clinical trial of nucresiran in patients with ATTR-CM and the TRITON-PN Phase 3 clinical trial of nucresiran in patients with hATTR-PN. Selling, General & Administrative (SG&A) Expenses GAAP and non-GAAP SG&A expenses for the three months ended March 31, 2026 increased as compared to the same period in 2025, primarily due to higher employee compensation costs and increased marketing investment associated with the ongoing global commercial launch of AMVUTTRA in ATTR-CM. Other Financial Highlights Interest expense Interest expense for the three months ended March 31, 2026 of $69 million included interest of $40 million attributed to the liability related to the sale of future Leqvio royalties and $26 million attributed to the liabilities related to the vutrisiran and zilebesiran development funding. Provision for income taxes During the three months ended March 31, 2026, we recorded a provision for income taxes of $16 million primarily related to U.S. state income taxes, utilization of Switzerland net deferred tax assets, as well as taxable income from jurisdictions in which we are subject to tax. We will utilize deferred tax assets in Switzerland to offset current cash tax liabilities and will continue to maintain a full valuation allowance against our net deferred tax assets in the U.S. and certain deferred tax assets in Switzerland. Financial position Cash, cash equivalents and marketable securities were $3.0 billion as of March 31, 2026, as compared to $2.9 billion as of December 31, 2025, with the increase primarily driven by net cash inflows from operating activities. Net cash provided by operating activities for the three months ended March 31, 2026 included $30 million of payments associated with the liability related to the sale of future Leqvio royalties recorded to interest expense, as well as $32 million of payments associated with the liabilities related to vutrisiran and zilebesiran development funding recorded to interest expense. A reconciliation of our GAAP to non-GAAP financial results is included in the tables at the end of this press release. 2026 Financial Guidance Full-year 2026 financial guidance is reiterated and consists of the following: Total TTR net product revenues (AMVUTTRA, ONPATTRO) 1 $4,400 million - $4,700 million Total Rare net product revenues (GIVLAARI, OXLUMO) 1 $500 million - $600 million Total net product revenues 1 $4,900 million - $5,300 million Net product revenues growth vs. 2025 at currency exchange rates as of December 31, 2025 2 64% to 77% Net product revenues growth vs. 2025 at constant exchange rates 2 64% to 77% Net revenues from collaborations and royalties $400 million - $500 million Non-GAAP R&D and SG&A expenses 3 $2,700 million - $2,800 million 1 Full-year 2026 guidance utilizing currency exchange rates as of December 31, 2025: 1 EUR = 1.17 USD and 1 USD = 157 JPY 2 Representing growth calculated as if the exchange rates had remained unchanged from those used in 2025, which is a non-GAAP financial measure 3 Excludes $300 million - $400 million of stock-based compensation expense from estimated GAAP R&D and SG&A expenses Use of Non-GAAP Financial Measures This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains or losses outside the ordinary course of the Company’s business. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies. The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release are stock-based compensation expenses, and realized and unrealized gains or losses on marketable equity securities. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the impact of the realized and unrealized gains or losses on marketable equity securities because the Company does not believe these adjustments accurately reflect the performance of the Company’s ongoing operations for the period in which such gains or losses are reported, as their sole purpose is to adjust amounts on the balance sheet. Percentage changes in revenue growth at CER are presented excluding the impact of changes in foreign currency exchange rates for investors to understand the underlying business performance. The current period’s foreign currency revenue values are converted into U.S. dollars using the average exchange rates from the prior period. The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release. Conference Call Information Management will provide an update on the Company and discuss first quarter 2026 results as well as expectations for the future via conference call on Thursday, April 30, 2026, at 8:30 am ET. A live audio webcast of the call will be available on the Investors section of the Company’s website at . An archived webcast will be available on the Alnylam website approximately two hours after the event. About AMVUTTRA ® (vutrisiran) AMVUTTRA ® (vutrisiran) is a transthyretin (TTR) silencer that delivers rapid knockdown of TTR at the source to address the underlying cause of transthyretin amyloidosis (ATTR). In a clinical study, AMVUTTRA rapidly knocked down TTR in as early as six weeks and decreased TTR levels by 87% with two and a half years of treatment. It is approved as a treatment for the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and for the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in various countries, globally. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only silencer approved for the treatment of ATTR-CM and hATTR-PN. For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit AMVUTTRA.com . About ONPATTRO ® (patisiran) ONPATTRO is an RNAi therapeutic that is approved in the United States and Canada for the treatment of adults with hATTR amyloidosis with polyneuropathy. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby reducing the production of TTR protein before it is made. Reducing the pathogenic protein leads to a reduction in amyloid deposits in tissues. For more information about ONPATTRO, including full Prescribing Information, visit ONPATTRO.com . About GIVLAARI ® (givosiran) GIVLAARI (givosiran) is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved in the United States and Brazil for the treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is also approved in the European Union for the treatment of AHP in adults and adolescents aged 12 years and older. In the pivotal trial, GIVLAARI was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam’s first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare professional. GIVLAARI works by specifically reducing elevated levels of ALAS1 messenger RNA (mRNA), leading to reduction of toxins associated with attacks and other disease manifestations of AHP. For more information about GIVLAARI, including the full U.S. Prescribing Information, visit GIVLAARI.com . About OXLUMO ® (lumasiran) OXLUMO (lumasiran) is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1). HAO1 encodes glycolate oxidase (GO). Thus, by silencing HAO1 and depleting the GO enzyme, OXLUMO inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. OXLUMO utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. OXLUMO has received regulatory approvals from the U.S. Food and Drug Administration (FDA) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients and from the European Medicines Agency (EMA) for the treatment of PH1 in all age groups. In the pivotal ILLUMINATE-A trial, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. In the ILLUMINATE-B pediatric Phase 3 trial, OXLUMO demonstrated an efficacy and safety pro to that observed in ILLUMINATE-A. In the ILLUMINATE-C trial, OXLUMO resulted in substantial reductions in plasma oxalate in patients with advanced PH1. Across all three studies, injection site reactions (ISRs) were the most common drug-related adverse reaction. OXLUMO is administered via subcutaneous injection once monthly for three months, then once quarterly beginning one month after the last loading dose at a dose based on actual body weight. For patients who weigh less than 10 kg, ongoing dosing remains monthly. OXLUMO should be administered by a healthcare professional. For more information about OXLUMO, including the full U.S. Prescribing Information, visit OXLUMO.com . About LNP Technology Alnylam has licenses to Arbutus Biopharma lipid nanoparticle (LNP) intellectual property for use in RNAi therapeutic products using LNP technology. About RNAi RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. Contacts Alnylam Pharmaceuticals, Inc. Christine Akinc (Investors and Media) 617-682-4340 Josh Brodsky (Investors) 617-551-8276 Read full story here

Clinical ResultFinancial StatementPhase 2Phase 1Phase 3

100 Deals associated with Hemin

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KEGG	Wiki	ATC	Drug Bank
D10003	Hemin	B06AB01	DB03404

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Porphyria, Acute Intermittent	Japan	CMIC Holdings Co., Ltd.	25 Mar 2013
Porphyrias	South Korea	Sam OH Pharm Co. Ltd.	16 Dec 2011
Porphyrias, Hepatic	France	Orphan Europe SARL	31 Dec 1995

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Myelodysplastic Syndromes	Phase 2	United States	H. Lundbeck A/S	01 May 2007

Clinical Result

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02922413 (CTgov)	Phase 2	Porphyria, Variegate \| Porphyria, Acute Intermittent \| Coproporphyria, Hereditary	13	Hemin for injection (Hemin for Injection)	fsovkejrci = igafjujejx xcwxgzisyv (bawqgfzujp, stvobmtbqu - zgavjsngej) View more	-	30 Jan 2026
				Placebo (Placebo)	fsovkejrci = qzlduueqey xcwxgzisyv (bawqgfzujp, pkxkbqujrl - atpvkamdiu) View more
NCT02180412 (CTgov)	Phase 2	Porphyria, Acute Intermittent	20	Glucose+Panhematin (Panhematin)	rbnlfnzhsp(wncelrebhg) = khnvicrohc mrszdbjabx (logteduyef, 2.2) View more	-	03 Apr 2025
				Glucose (Placebo)	rbnlfnzhsp(wncelrebhg) = eduxtwjwwx mrszdbjabx (logteduyef, 1.9) View more
NCT01855841 (Pubmed \| Pancreatology)	Phase 2	Post-ERCP acute pancreatitis	294	Hemin (4 mg/kg)	egvnkaonoa(xileglaclt) = douryryvbf oqemcsmxlq (zolmafpgfj )	Negative	01 May 2024
				Placebo	egvnkaonoa(xileglaclt) = pquwzxfiwt oqemcsmxlq (zolmafpgfj )
NCT02314780 (CTgov)	Phase 2	stress oxidative \| Myocardial Ischemia	31	Heme arginate 3mg/kg (Heme Arginate (High Dose))	cyykchejzm(akspxvnxxa) = sdrovulwyp ocacwxgyad (oiqetadixg, 195.7) View more	-	15 Feb 2021
				Heme arginate 1mg/kg (Heme Arginate (Low Dose))	cyykchejzm(akspxvnxxa) = zlzieszotm ocacwxgyad (oiqetadixg, 173.1) View more
NCT01206582 (CTgov)	Phase 2	Gastroparesis \| Diabetic Gastroparesis	20	Hemin (Hemin)	lvpqmzzwom(uidwdmlvfv) = hoxgsrhxuz vimhemtufi (niwcxbwsan, 0.3) View more	-	04 Feb 2016
				Albumin (Albumin)	lvpqmzzwom(uidwdmlvfv) = fexqlwuano vimhemtufi (niwcxbwsan, 0.1) View more
NCT01430156 (Heme Oxygenase-1 in renal Transplantation, Pubmed \| Transplantation)	Phase 3	-	40	Heme arginate (HA)	pqkrbagbez(ikglygvsvu) = utnehokazo yorryaiilx (rnryefkkld ) View more	Positive	01 Jan 2016
				Placebo (0.9% NaCl)	pqkrbagbez(ikglygvsvu) = sanyhgexbm yorryaiilx (rnryefkkld ) View more
NCT00467610 (CTgov)	Phase 2	Myelodysplastic Syndromes	6	Panhematin	aijktbugec = qzboishdwi pokzgexjul (tfplrczfjq, lvanqcdauj - nvrtgyljsq) View more	-	24 Oct 2014
NCT01461512 (Pubmed \| J Cardiovasc Magn Reson)	Phase 2	Ischemia	12	Heme arginate	vxusyreues(uicjbzectd) = rloraiitcp ifpponbooc (ukkikeaxwz ) View more	-	02 Aug 2012
				Placebo	vxusyreues(uicjbzectd) = akmmivsqhk ifpponbooc (ukkikeaxwz ) View more

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