Single-center, Double-blind Trial to Assess the Pharmacokinetics, the Pharmacodynamics, and the Tolerability of a Single 16 mg Dose of Selatogrel (ACT-246475) in Healthy Japanese and Caucasian Participants

This study is for research purposes only and is not intended to treat any medical condition. The purpose of this study is to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and tolerability of selatogrel following a single dose in healthy Japanese and Caucasian participants. Pharmacokinetics is the study of the absorption and breakdown of the study drug in the body. Pharmacodynamics is the study of the effect of the study drug on the body. There will be 2 groups in the study: 16 Japanese participants in one group and 16 Caucasian participants in the other group.
The duration of participation in this study is approximately 70 days from screening to the end of follow-up. A screening visit is required within 28 days prior to the start of the study to determine whether the volunteer qualifies and is willing to participate in this research study. This study requires in-patient stay in the research clinic of 3 or 4 days (2 or 3 nights), an end-of-trial (EOT) examination at least 36 hours after study drug administration, and a post-trial safety follow-up telephone call or site visit 30-40 days after the EOT examination.

Start Date07 Oct 2025

Sponsor / Collaborator

Viatris, Inc.

NCT04957719

/ Enrolling by invitationPhase 3

Multi-center, Double-blind, Randomized, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Self-administered Subcutaneous Selatogrel for Prevention of All-cause Death and Treatment of Acute Myocardial Infarction in Subjects With a Recent History of Acute Myocardial Infarction

This study will randomize patients recently discharged from the hospital with a confirmed diagnosis of type 1 acute myocardial infarction (Thygesen et al. 2018) and having additional cardiovascular risk factors.

Start Date14 Aug 2021

Sponsor / Collaborator

Viatris, Inc.

NCT04557280

/ CompletedPhase 1

A Randomized, Open-label, 3-period Cross-over Study to Assess the Pharmacokinetics of Selatogrel (ACT-246475) in Healthy Subjects After Subcutaneous Administration by Syringe and Auto-injector

The main purpose is to study the pharmacokinetics of selatogrel (ACT-246475) using different administration modes and formulations. The clinical pharmacology data will be used to support demonstration of bioequivalence and interchangeability of the different formulations.

Start Date06 Nov 2020

Sponsor / Collaborator

Viatris, Inc.

100 Clinical Results associated with Selatogrel

100 Translational Medicine associated with Selatogrel

100 Patents (Medical) associated with Selatogrel

Literatures (Medical) associated with Selatogrel

01 Nov 2025·British Journal of Pharmacology

Selatogrel: Potential to redefine timely anti‐platelet intervention

Review

Author: Zalzal, Rudy N. ; Eid, Ali H. ; Dakroub, Ali H. ; Salem, Peter P.

Acute coronary syndrome (ACS) encompasses a number of heart diseases that cause a sudden decrease in coronary perfusion, precipitating cardiomyocyte necrosis or heightened risk thereof. This pathology is a major burden of cardiovascular disease. The etiopathogenesis and clinical manifestation of ACS are predominantly attributable to myocardial hypoperfusion consequent, to coronary vessel occlusion, typically resulting from atherosclerotic plaque rupture and subsequent thrombosis. Dual antiplatelet therapy (DAPT), comprising aspirin and a P2Y 12 receptor antagonist, has long been the mainstay of ACS management. Notwithstanding, limitations in the pharmacokinetic (PK) and pharmacodynamic (PD) profiles of conventional DAPT agents persist. Selatogrel (ACT‐246475), a novel P2Y 12 antagonist currently undergoing Phase III clinical trial, is poised to revolutionise ACS treatment. This highly selective and potent 2‐phenylpyrimidine‐4‐carboxamide analogue is administered subcutaneously. As such, it affords immediate intervention in ACS patients. Importantly, selatogrel has a remarkably rapid onset of action and a favourable safety profile. These advantages render selatogrel a promising candidate for pre‐hospital, self‐administered ACS treatment, potentially optimising the reduction of total ischaemic time. Having successfully completed several Phase I and Phase II trials, selatogrel is currently undergoing Phase III evaluation to further elucidate its safety and efficacy. Subsequent investigations will serve to support or refine its therapeutic attributes.

01 Nov 2024·Journal of Computational Biophysics and Chemistry

Molecular Docking, Quantum Mechanics and Molecular Dynamics Simulation of Anti-CAD Drugs Against High-Risk Xanthine Dehydrogenase Variants Associated with Oxidative Stress Pathways

Author: Ahmed, Shiek S. S. J. ; Sudhan, M. ; Emran, Talha Bin ; Attia, Sabry M. ; Ahmad, Sheikh F. ; Janakiraman, V.

Xanthine dehydrogenase (XDH) contributes significantly to generating reactive oxygen species in coronary artery disease (CAD). XDH has been proposed as a therapeutic target, but its genetic variants could affect protein structure and drug response. We aimed to assess protein structure modification occur due to genetic variants and to screen 215 CAD drugs for their utility in personalized CAD treatment against the XDH variants. A series of computational methods were implemented to identify pathogenic variants that cause XDH structure instability localized at the con served regions contributing to functional significance. Then, the XDH structures with the pathogenic variants were modeled using two different approaches to select the best models for docking with the CAD drugs. Finally, the stability of the docked complexes and their ability to transfer electrons were evaluated using molecular dynamics (MD) simulations and quantum mechanics/molecular mechanics (QM/MM) calculation. Among 751 variants examined; R149C and Q919R showed high pathogenicity, localized in conserved regions could alter protein structure and function. Further, docking of CAD drugs against XDH (native, R149C and Q919R) showed vericiguat with higher affinity, ranging from −7.95 kcal/mol to −10.41 kcal/mol, than the well-known XDH inhibitor (febuxostat, −5.73 kcal/mol to −8.35 kcal/mol). This indicates that vericiguat will be effective in CAD treatment, regardless of the XDH variants. Additionally, MD simulation and QM/MM confirmed vericiguat stability and electron transfer ability to form hydrogen bonds with the XDH protein. In conclusion, vericiguat will be beneficial for the personalized treatment of CAD by inhibiting XDH variants. Additional clinical studies are necessary to confirm our findings.

Journal of thrombosis and thrombolysis

Comparison of the effects of the GPIIb-IIIa antagonist Zalunfiban and the P2Y12 antagonist Selatogrel on Platelet Aggregation

Article

Author: Rikken, A O F Sem ; Ten Berg, Jurriën M ; van 't Hof, Arnoud W J ; Gibson, C Michael ; Jennings, Lisa K ; Granger, Christopher B ; Curry, Benjamin J

Abstract:

Understanding the pharmacodynamic effects of platelet inhibitors is standard for developing more effective antithrombotic therapies. An example is the antithrombotic treatment of acute coronary syndrome (ACS), in particular ST-elevated myocardial infarction (STEMI) patients who are in need for rapid acting strong antithrombotic therapy despite the use of aspirin and oral P2Y12-inhibitors. In this study, we evaluated two injectable platelet inhibitors under clinical development (the P2Y12 antagonist selatogrel and the GPIIb-IIIa antagonist zalunfiban) that may be amenable to pre-hospital treatment of STEMI patients. Platelet reactivity was assessed at inhibitor concentrations that represent clinically relevant levels of platelet inhibition (IC20-50%, 1/2Cmax, and Cmax). Light transmission aggregometry (LTA), was used to evaluate the initial rate of aggregation (primary slope, PS) and maximal aggregation (MA). Both adenosine diphosphate (ADP) and thrombin receptor agonist peptide (TRAP) were used as agonists. Zalunfiban demonstrated similar inhibition of platelet aggregation when blood was collected in PPACK or TSC, whereas selatogrel demonstrated greater inhibition in PPACK. In this study, using PPACK anticoagulant, selatogrel and zalunfiban affected PS in response to ADP equivalently at all drug concentrations tested. In contrast, zalunfiban had significantly greater potency at its Cmax concentration compared to selatogrel using TRAP as agonist. Upon evaluation of MA responses at lower doses, selatogrel had greater inhibition of MA in response to ADP than zalunfiban; however, at concentrations that represent Cmax, the drugs were equivalent. Zalunfiban also had greater inhibition of MA in response to TRAP at the Cmax dose. These data suggest that zalunfiban may provide greater protection in reducing thrombus formation than selatogrel, especially since thrombin is an early, key primary agonist in the pathophysiology of thrombotic events.

News (Medical) associated with Selatogrel

17 Feb 2026

·www.prnewswire.com

HALOZYME REPORTS FULL YEAR 2025 RECORD REVENUE OF $1.4 BILLION AND REITERATES STRONG 2026 FINANCIAL GUIDANCE

Full Year 2025 Total Revenue Increased 38% YOY to Record $1.397 billion Full Year 2025 Royalty Revenue Increased 52% YOY to Record $868 million Completed Acquisitions of Elektrofi's Hypercon™ Technology and Surf Bio's Hyperconcentration Technology Reiterating 2026 Financial Guidance Ranges: Total Revenue of $1.710 - $1.810 billion, YOY Growth of 22% - 30% Adjusted EBITDA of $1.125 - $1.205 billion, YOY Growth of 71% - 83%1 Non-GAAP Diluted EPS of $7.75 - $8.25, YOY Growth of 87% - 99%1 SAN DIEGO, Feb. 17, 2026 /PRNewswire/ -- Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme" or the "Company") today reported its financial and operating results for the full year and fourth quarter ended December 31, 2025, and provided an update on its recent corporate activities. "2025 was a pivotal year for Halozyme as we delivered record total revenue of $1.4 billion, which was the result of continued growth in our ENHANZE business. In addition, we expanded our drug delivery technology portfolio with two acquisitions. Three ENHANZE‑enabled blockbusters, DARZALEX SC, Phesgo and VYVGART Hytrulo, drove royalty revenue growth of 52%, reaching a record $868 million in 2025. In the year, we also expanded our ENHANZE opportunities, adding three new collaboration and licensing agreements with Takeda, Merus and Skye Bioscience and gained one new target nomination from Roche. Furthermore, Janssen expanded the global reach of ENHANZE with approvals in the U.S., China and Japan for Rybrevant SC and new indication approvals in front line settings for DARZALEX Faspro. In parallel, we broadened our drug delivery portfolio with the acquisitions of the Hypercon technology and Surf Bio's hyperconcentration technology, which meaningfully expand, diversify and extend our long‑term royalty opportunity into the mid-2040s," said Dr. Helen Torley, President and Chief Executive Officer. Dr. Torley continued, "As we look ahead, our long-term outlook reflects the strong momentum and opportunity we have built through a broader drug delivery portfolio and offering to the biopharma industry. With royalty revenue projected to exceed $1 billion in 2026 and a clear line of sight to more than $2 billion in total revenue by 2028, Halozyme is entering its next phase of growth with strong conviction. Our projected 22% to 30% total revenue growth in 2026, combined with an expanding development pipeline with six projected new ENHANZE and two Hypercon development program starts, plus plans to sign three or more ENHANZE and Hypercon partnerships in 2026, all reinforce the durability of our revenue. With our strong cash generation and a diversified set of high‑value royalty drivers, we remain focused on delivering sustained long‑term value for shareholders." Fourth Quarter and Recent Corporate Highlights: In December 2025, Halozyme completed the acquisition of Surf Bio, Inc. ("Surf Bio"), subsequently renamed Halozyme Surf Bio, Inc., resulting in an expansion of Halozyme's drug delivery technology portfolio and the potential for future growth through new collaboration agreements. In December 2025, Halozyme announced that a German court had granted Halozyme's request for a preliminary injunction ordering Merck Sharp & Dohme Corp. ("Merck") to refrain from distributing and offering Keytruda® SC in Germany. In November 2025, Halozyme completed the acquisition of Elektrofi Inc. ("Elektrofi"), subsequently renamed Halozyme Hypercon, Inc., resulting in an expansion of Halozyme's drug delivery technology portfolio and the potential for future growth through new collaboration agreements. In November 2025, Halozyme completed the sale of $750.0 million aggregate principal amount of the 2031 Convertible Notes and $750.0 million aggregate principal amount of the 2032 Convertible Notes. The Company used a portion of the net proceeds to fund the cost of entering into the 2031 Capped Call Transactions and the 2032 Capped Call Transactions. The Company also used a portion of the net proceeds to enter into privately negotiated agreements with certain holders of its outstanding 2027 Convertible Notes and 2028 Convertible Notes to repurchase their 2027 Convertible Notes and 2028 Convertible Notes for cash through privately negotiated transactions entered into concurrently with or shortly after the offering. In November 2025, Halozyme entered into an amendment to the Company's credit agreement that, among other things extended the maturity date and increased the borrowing capacity of the Company's existing revolving credit facility from $575.0 million to $750.0 million. Fourth Quarter and Recent Partner Highlights: In January 2026, Janssen announced the U.S. Food and Drug Administration ("FDA") approved DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in combination with bortezomib, lenalidomide and dexamethasone ("D-VRd") for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant. In December 2025, Halozyme and Skye Bioscience entered into a non-exclusive global collaboration and license agreement that provides Skye Bioscience access to ENHANZE® for the development and potential commercialization of an SC formulation of nimacimab for the treatment of obesity. In December 2025, Halozyme and Takeda entered into a new global collaboration and exclusive license agreement which provides Takeda with access to ENHANZE® for use with vedolizumab, marketed globally as ENTYVIO®, for the treatment of adults with moderately to severely active Crohns' disease or ulcerative colitis, which are the two main forms of inflammatory bowel disease. In December 2025, Roche nominated a new undisclosed non-exclusive target to be studied using ENHANZE®. In the fourth quarter of 2025, the ongoing argenx ARGX-121 Phase 1 program was expanded to include an SC-arm evaluating ARGX-121 with ENHANZE® in healthy adults. In December 2025, Janssen announced the FDA approved RYBREVANT FASPRO™ (amivantamab and hyaluronidase-lpuj) for the treatment of patients with epidermal growth factor receptor ("EGFR")-mutated locally advanced or metastatic non-small cell lung cancer ("NSCLC"). In December 2025, Janssen received approval from the National Medical Products Administration in China for RYBREVANT™ FASPRO for the first-line treatment of adult patients with advanced NSCLC. In December 2025, Janssen received approval from the Ministry of Health, Labour and Welfare in Japan for RYBROFAZ® (amivantamab) with ENHANZE® for the first-line treatment of adult patients with advanced NSCLC. In December 2025, Halozyme entered into a commercial license and supply agreement with Viatris under which Halozyme licenses and supplies an auto-injector product for self-administered SC selatogrel for the treatment of acute myocardial infarction in adult patients. In November 2025, Janssen announced the FDA approved DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) co-formulated with ENHANZE®, as single treatment of adult patients with high-risk smoldering multiple myeloma. In November 2025, Halozyme and Merus entered into a non-exclusive global collaboration and license agreement that provides Merus access to ENHANZE® technology for a single target. Merus intends to explore development and potential commercialization of SC administration of petosemtamab, an EGFR and leucine-rich repeat-containing G-protein coupled receptor 5 bispecific antibody, for the treatment of head and neck cancer. Full Year and Fourth Quarter 2025 Financial Highlights: Total revenue for the full year was $1,396.6 million, compared to $1,015.3 million in 2024. The 38% year-over-year increase was primarily driven by royalty revenue growth and an increase in product sales. Revenue included $867.8 million in royalties, an increase of 52% compared to $571.0 million in 2024, primarily driven by continued sales uptake of ENHANZE® partner products that have launched since 2020, predominantly by VYVGART® Hytrulo by argenx, DARZALEX® SC by Janssen and Phesgo® by Roche in all geographies. Cost of sales for the full year was $228.8 million, compared to $159.4 million in 2024. The increase in cost of sales was primarily due to an increase in product sales and labor allocation initiatives. Amortization of intangibles expense for the full year was $76.7 million, compared to $71.0 million in 2024. The increase in amortization of intangibles expense was due to the acquisition of Elektrofi in November 2025. Research and development expense for the full year was $81.5 million, compared to $79.0 million in 2024. The increase was primarily due to the acquisition of Elektrofi and Surf Bio, partially offset by lower compensation expense driven by resource optimization, labor allocation initiatives, and timing of planned investments in ENHANZE® related to the development of our new high-yield rHuPH20 manufacturing process. Selling, general and administrative expense for the full year was $207.1 million, compared to $154.3 million in 2024. The increase was primarily due to an increase in consulting and professional service fees, including litigation costs incurred in connection with a patent infringement litigation case, diligence and transaction-related costs incurred in support of the acquisition of Elektrofi and Surf Bio, and an increase in compensation expense. Net income for the full year was $316.9 million, compared to $444.1 million in 2024. Net Income included acquired in-process research and development ("IPR&D") expense of $284.9 million related to the Surf Bio acquisition in the fourth quarter of 2025. Adjusted EBITDA for the full year was $657.6 million, compared to $632.2 million in 2024.1,2 Adjusted EBITDA included acquired IPR&D expense of $284.9 million related to the Surf Bio acquisition in the fourth quarter of 2025. GAAP diluted earnings per share for the full year was $2.56, compared to $3.43 in 2024. Non-GAAP diluted earnings per share was $4.15, compared to $4.23 in 2024.1,2 GAAP and non-GAAP diluted earnings per share included an unfavorable impact of approximately $2.30 per share related to acquired IPR&D expense for the Surf Bio acquisition in the fourth quarter of 2025. Total revenue in the fourth quarter was $451.8 million, compared to $298.0 million in the fourth quarter of 2024. The 52% year-over-year increase was primarily driven by royalty revenue growth and an increase in product sales. Revenue included $258.0 million in royalties, an increase of 51% compared to $170.4 million in the fourth quarter of 2024, primarily driven by continued sales uptake of ENHANZE® partner products that have launched since 2020, predominantly by VYVGART® Hytrulo by argenx, DARZALEX® SC by Janssen and Phesgo® by Roche in all geographies. Net loss in the fourth quarter was $141.6 million, compared to net income of $137.0 million in the fourth quarter of 2024. Net loss included acquired IPR&D expense of $284.9 million related to the Surf Bio acquisition in the fourth quarter of 2025. Adjusted EBITDA in the fourth quarter was $21.9 million, compared to $195.8 million in the fourth quarter of 2024.1,2 Adjusted EBITDA included acquired IPR&D expense of $284.9 million related to the Surf Bio acquisition in the fourth quarter of 2025. GAAP diluted loss per share in the fourth quarter was $1.20, compared to GAAP diluted earnings per share $1.06 in the fourth quarter of 2024. Non-GAAP diluted loss per share was $0.24 compared to Non-GAAP diluted earnings per share of $1.26 in the fourth quarter of 2024.1,2 GAAP and non-GAAP diluted loss per share in the fourth quarter of 2025 included an unfavorable impact of $2.42 per share related to acquired IPR&D expense for the Surf Bio acquisition in the fourth quarter of 2025. Cash, cash equivalents, restricted cash and marketable securities were $145.4 million on December 31, 2025, compared to $596.1 million on December 31, 2024. The decrease was primarily driven by cash used for the Elektrofi and Surf Bio acquisitions and share repurchases, partially offset by the net proceeds from issuance of convertible notes and cash generated from operations. Financial Outlook for 2026 The Company is reiterating its 2026 financial guidance ranges, which were last updated on January 28, 2026. For the full year 2026, the Company expects: Total revenue of $1.710 billion to $1.810 billion, representing growth of 22% to 30% over 2025 total revenue, primarily driven by increases in royalty revenue and product sales from API. Revenue from royalties of $1.130 billion to $1.170 billion, representing growth of 30% to 35% over 2025. Adjusted EBITDA of $1.125 billion to $1.205 billion, representing growth of 71% to 83% over 2025, including new Hypercon™ and Surf Bio investment of approximately $60 million. Non-GAAP diluted earnings per share of $7.75 to $8.25, representing growth of 87% to 99% over 2025. The Company's earnings per share guidance includes new Hypercon™ and Surf Bio investment of approximately $60 million and does not consider the impact of potential future share repurchases. Table 1. 2026 Financial Guidance Webcast and Conference Call Halozyme will host its Quarterly Update Conference Call for the fourth quarter and full year ended December 31, 2025 today, Tuesday, February 17, 2026, at 1:30 p.m. PT/4:30 p.m. ET. The conference call may be accessed live with pre-registration via link: . The call will also be webcast live through the "Investors" section of Halozyme's corporate website and a recording will be made available following the close of the call. To access the webcast and additional documents related to the call, please visit Halozyme.com. About Halozyme Halozyme is a biopharmaceutical company advancing disruptive solutions to improve patient experiences and outcomes for emerging and established therapies. As the innovators of ENHANZE® drug delivery technology with the proprietary enzyme rHuPH20, Halozyme's commercially-validated solution facilitates the subcutaneous delivery of injected drugs and fluids, reducing treatment burden and improving convenience. ENHANZE® has touched more than one million patient lives through ten commercialized products across over 100 global markets and is licensed to leading pharmaceutical and biotechnology companies including Roche, Takeda, Pfizer, Janssen, AbbVie, Eli Lilly, Bristol-Myers Squibb, argenx, ViiV Healthcare, Chugai Pharmaceutical, Acumen Pharmaceuticals, Merus N.V. and Skye Bioscience. Halozyme expanded its drug delivery technology portfolio to develop partner products using Hypercon™ and the Surf Bio hyperconcentration technology. Hypercon™ is an innovative microparticle technology expected to set a new standard in hyperconcentration of drugs and biologics by reducing injection volume for the same dosage and enabling administration in at‑home and healthcare‑provider settings. The development of the Surf Bio polymer‑based hyperconcentration technology further broadens the range of biologics that can be delivered subcutaneously, meaningfully expanding the scope of opportunities across therapeutic modalities. The Hypercon™ technology has been licensed to leading biopharmaceutical partners, including Janssen, Eli Lilly and argenx. Halozyme also develops, manufactures and commercializes drug-device combination products using advanced auto-injector technologies designed to improve convenience, reliability and tolerability, enhancing patient comfort and adherence. The Company has two proprietary commercial products, Hylenex® and XYOSTED®, partnered commercial products and ongoing development programs with Teva Pharmaceuticals and McDermott Laboratories Limited, an affiliate of Viatris Inc. Halozyme is headquartered in San Diego, CA, with offices in Ewing, NJ; Minnetonka, MN; and Boston, MA. Minnetonka is also the site of its operations facility. For more information, visit and connect with us on LinkedIn. Note Regarding Use of Non-GAAP Financial Measures In addition to disclosing financial measures prepared in accordance with U.S. generally accepted accounting principles ("GAAP"), this press release and the accompanying tables contain certain Non-GAAP financial measures. The Company reports earnings before interest, taxes, depreciation, and amortization ("EBITDA"), adjusted EBITDA, Non-GAAP diluted earnings per share, Non-GAAP diluted shares, and guidance with respect to those measures, in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company calculates Non-GAAP diluted earnings per share excluding share-based compensation expense, amortization of debt discounts, intangible asset amortization, one-time items, if any, such as changes in contingent liabilities, inventory adjustments, impairment charges, transaction costs for business combinations, severance and share-based compensation acceleration expenses, intellectual property litigation costs, inducement expenses related to convertible notes, and certain adjustments to income tax expense. The Company calculates Non-GAAP diluted shares excluding the dilutive impact of convertible notes which is used in calculating Non-GAAP diluted earnings. The Company calculates EBITDA excluding interest, taxes, depreciation and amortization. The Company calculates adjusted EBITDA excluding one-time items, if any, such as changes in contingent liabilities, inventory adjustments, impairment charges, transaction costs for business combinations, severance and share-based compensation acceleration expenses and intellectual property litigation costs. Reconciliations between GAAP and Non-GAAP financial measures are included at the end of this press release. The Company does not provide reconciliations of forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in share-based compensation expense and the effects of any discrete income tax items. For the same reasons, the Company is unable to address the probable significance of the unavailable information. The Company provides Non-GAAP financial measures that it believes will be achieved; however, it cannot accurately predict all of the components of the adjusted calculations and the GAAP measures may be materially different than the Non-GAAP measures. The Company evaluates other items of income and expense on an individual basis for potential inclusion in the calculation of Non-GAAP financial measures and considers both the quantitative and qualitative aspects of the item, including (i) its size and nature, (ii) whether or not it relates to the Company's ongoing business operations and (iii) whether or not the Company expects it to occur as part of the Company's normal business on a regular basis. Non-GAAP financial measures do not have any standardized meaning and are therefore unlikely to be comparable to similarly titled measures presented by other companies. These Non-GAAP financial measures are not meant to be considered in isolation and should be read in conjunction with the Company's consolidated financial statements prepared in accordance with GAAP, and are not prepared under any comprehensive set of accounting rules or principles. In addition, from time to time in the future there may be other items that the Company may exclude for purposes of its Non-GAAP financial measures, and the Company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. The Company considers these Non-GAAP financial measures to be important because they provide useful measures of the operating performance of the Company, exclusive of factors that do not directly affect what the Company considers to be its core operating performance, as well as unusual events. The Non-GAAP measures also allow investors and analysts to make additional comparisons of the operating activities of the Company's core business over time and with respect to other companies, as well as assessing trends and future expectations. The Company uses Non-GAAP financial information in assessing what it believes is a meaningful and comparable set of financial performance measures to evaluate operating trends, as well as in establishing portions of our performance-based incentive compensation programs. Safe Harbor Statement In addition to historical information, the statements set forth in this press release include forward-looking statements including, without limitation, statements concerning the Company's financial performance (including the Company's expected financial outlook for 2026) and expectations for future growth, profitability, revenue durability, total revenue, royalty revenue, royalty revenue duration, EBITDA, Adjusted EBITDA, and non-GAAP diluted earnings-per-share, and shareholder value. Forward-looking statements also include future plans, objectives, expectations and intentions related to the acquisitions of Elektrofi and Surf Bio, such acquisitions' expected impact and contributions to the Company's and combined group's operations and financial results (including potential development and commercialization of partnered products and timing related to these events), as well as the expected benefits of the acquisitions. Forward-looking statements related to Elektrofi's and Surf Bio's intellectual property include expectations for length of patent terms and patent expirations and the expected impact such patents may have on the duration, durability and amounts of future royalty payments the Company may receive from licensing such intellectual property. Forward-looking statements regarding the Company's ENHANZE® drug delivery technology may include the possible benefits and attributes of ENHANZE®, its potential application to aid in the dispersion and absorption of other injected therapeutic drugs and facilitating more rapid delivery and administration of higher volumes of injectable medications through subcutaneous delivery. Forward-looking statements regarding the Company's business may include potential growth and receipt of royalty and milestone payments driven by our partners' development and commercialization efforts, potential new clinical trial study starts and advancement of partnered development programs, regulatory submissions and product launches, the size and growth prospects of our partners' drug franchises, potential new or expanded collaborations and collaborative targets, and potential approvals of new partnered or proprietary products, and the potential timing of these events. These forward-looking statements are typically, but not always, identified through use of the words "expect," "believe," "enable," "may," "will," "could," "intends," "estimate," "anticipate," "plan," "predict," "probable," "potential," "preliminary," "possible," "should," "continue," and other words of similar meaning and involve risk and uncertainties that could cause actual results to differ materially from those in the forward-looking statements. Actual results could differ materially from the expectations contained in these forward-looking statements as a result of several factors, including uncertainties concerning future matters such as market conditions, changes in domestic and foreign business, changes in the competitive environment in which the Company operates, the expected benefits of its acquisitions of Elektrofi and Surf Bio, unexpected early expiration or termination of the patent terms for the Company's drug delivery technologies, unexpected levels of revenues, expenditures and costs, unexpected results or delays in the growth of the Company's business, or in the development, regulatory review or commercialization of the Company's partnered or proprietary products, regulatory approval requirements, unexpected adverse events or patient outcomes and competitive conditions. These and other factors that may result in differences are discussed in greater detail in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, including under the headings "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". Except as required by law, the Company undertakes no duty to update forward-looking statements to reflect events after the date of this release. Contacts: Tram Bui VP, Investor Relations and Corporate Communications 609-333-7668 [email protected] Sydney Charlton Teneo 917-972-8407 [email protected] SOURCE Halozyme Therapeutics, Inc. 21% more press release views with Request a Demo

Phase 1License out/inDrug ApprovalAcquisition

15 Oct 2025

·www.prnewswire.com

Viatris Completes Acquisition of Aculys Pharma Including Exclusive Rights to Pitolisant in Japan and to Spydia® in Japan and Certain Other Markets in the Asia-Pacific Region

Strengthens Viatris' Presence in Japan With the Addition of Two Innovative Assets Targeting Areas of Significant Unmet Medical Need Leverages Existing Infrastructure and Expertise in Central Nervous System Therapy Area Aligned with Strategy to Target Accretive Regional Business Development Opportunities PITTSBURGH, Oct. 15, 2025 /PRNewswire/ -- Viatris Inc. (Nasdaq: VTRS), a global healthcare company, today announced it has acquired Aculys Pharma, Inc., a clinical stage biopharmaceutical company focused on commercializing innovative treatments for neurological conditions. Viatris received rights to develop and commercialize pitolisant and Spydia®, two assets in the Central Nervous System (CNS) therapy area, further expanding Viatris' portfolio of innovative products in Japan. As part of the transaction, Viatris has acquired exclusive development and commercialization rights in Japan for pitolisant, a selective/inverse agonist of the histamine H3 receptor. Based on the strength of recent Phase 3 clinical trial results in Japanese patients and the positive benefit-risk profile established globally, Viatris is on track to file for marketing approval from the Ministry of Health, Labour and Welfare (MHLW) of Japan for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of excessive daytime sleepiness associated with obstructive sleep apnea syndrome (OSAS) by the end of 2025. The transaction also includes exclusive rights in Japan and certain other markets in the Asia-Pacific region for Spydia Nasal Spray, which was approved in Japan in June 2025 for the treatment of status epilepticus. "The acquisition of Aculys Pharma leverages our deep commercial infrastructure in Japan and longstanding expertise in CNS, positioning us to bring these innovative treatments to more patients in need," said Corinne Le Goff, Chief Commercial Officer, Viatris. "The addition of pitolisant and Spydia to our portfolio of innovative products is strategically aligned with our commitment to grow in areas where we can make the greatest impact and is a great example of our business development strategy designed to complement our core strengths in markets across the world." This acquisition further expands Viatris' portfolio of innovative products in Japan which includes Effexor for the treatment of generalized anxiety disorder (GAD) which is under regulatory review, selatogrel in Acute MI, Nefecon in IgA nephropathy, and cenerimod in systemic lupus erythematosus (SLE), all of which have pivotal Phase 3 trials currently on going and Tyrvaya in dry eye disease for which a Phase 3 trial is anticipated to start in 2026. Terms of the Transaction Under the terms of the acquisition agreement, Viatris has made an upfront payment to Aculys Pharma shareholders as consideration for the acquisition, with additional consideration contingent upon the achievement of specified regulatory and commercial milestones, and royalties on net sales. About Pitolisant Pitolisant is an antagonist/inverse agonist that selectively binds to the histamine H3 receptor, an autoreceptor located in the presynaptic region of the histamine-containing neurons in the human brain that plays a critical role in regulating sleep and wake rhythm. The drug was approved by the European Medicines Agency (EMA) for the treatment of narcolepsy with or without cataplexy in 2016 and for the treatment of excessive daytime sleepiness associated with OSAS in 2021. In 2019, the U.S. Food and Drug Administration (FDA) approved pitolisant under the brand name Wakix® for the treatment of excessive daytime sleepiness associated with narcolepsy and cataplexy associated with narcolepsy in 2020. As of the end of 2023, pitolisant had obtained regulatory approval in 38 countries including the U.S. and the EU for the treatment of narcolepsy, and in 29 countries in the EU for the treatment of OSAS. Positive Pivotal study results in Japanese patients were recently achieved in both narcolepsy and OSAS. In the narcolepsy Phase 3 trial, the primary endpoint of improvement in excessive daytime sleepiness (EDS) compared to a placebo group using the Epworth Sleepiness Scale (ESS) was met. Statistically significant difference in ESS was observed between the two groups. Furthermore, the key secondary endpoint of the frequency of cataplexy attacks showed a suppression effect comparable to that observed in prior global Phase 3 trials. No serious adverse events were noted, and the safety and tolerability results were consistent with global clinical trials. The OSAS Phase 3 trial evaluated the effect of pitolisant in Japanese patients with OSAS who were experiencing residual EDS despite treatment with CPAP therapy. At the end of the 12-week treatment period, patients receiving pitolisant scored lower on the ESS used to measure EDS compared to those in the placebo group and this difference was statistically significant (p=0.007). Additionally, safety and tolerability were consistent with results from global clinical studies. About Spydia (diazepam) In Japan, Aculys Pharma received marketing approval for Spydia Nasal Spray 5 mg, 7.5 mg, and 10 mg for the treatment of status epilepticus in patients 2 years or older in June 2025. This is the first intranasal anti-seizure medication approved in Japan for the treatment of status epilepticus or seizures with potential progression to status epilepticus. It is also the first rescue medication approved for adults for out-of-hospital use. This drug was developed by Neurelis, Inc. in the US and Aculys Pharma obtained exclusive development and commercialization rights in Japan and certain markets in the Asia-Pacific region, including Australia, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, New Zealand, Philippines, South Korea, Thailand and Vietnam. In 2020, the FDA approved diazepam nasal spray under the brand name Valtoco® for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity that are distinct from a usual seizure pattern in patients with epilepsy aged 6 years and older. In April 2025, the FDA extended the indication to include patients aged 2 years and older. Diazepam has been used for approximately 60 years in Japan, primarily in injectable form as a treatment for epileptic seizures. About Viatris Viatris Inc. (Nasdaq: VTRS) is a global healthcare company uniquely positioned to bridge the traditional divide between generics and brands, combining the best of both to more holistically address healthcare needs globally. With a mission to empower people worldwide to live healthier at every stage of life, we provide access at scale, currently supplying high-quality medicines to approximately 1 billion patients around the world annually and touching all of life's moments, from birth to the end of life, acute conditions to chronic diseases. With our exceptionally extensive and diverse portfolio of medicines, a one-of-a-kind global supply chain designed to reach more people when and where they need them, and the scientific expertise to address some of the world's most enduring health challenges, access takes on deep meaning at Viatris. We are headquartered in the U.S., with global centers in Pittsburgh, Shanghai and Hyderabad, India. Learn more at viatris.com and investor.viatris.com, and connect with us on LinkedIn, Instagram, YouTube and X. About Aculys Pharma, Inc. Aculys Pharma is a clinical stage biopharmaceutical company that is pioneering ways to eliminate drug lag/drug loss in Japan and is working to resolve social issues related to neurological and psychiatry diseases. Its corporate name was created from the philosophy of serving as a "Catalyst to Access". Aiming to act as a bridge for innovative medical care in the field of neuropsychiatry, Aculys Pharma develops and commercializes novel pharmaceuticals introduced from the US and European countries and provides innovations for better medical care to patients, their families, healthcare professionals, and society. Forward-Looking Statements This press release includes statements that constitute "forward-looking statements." These statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may include statements regarding Viatris completes acquisition of Aculys Pharma including exclusive rights to pitolisant in Japan and to Spydia® in Japan and certain other markets in the Asia-Pacific region; strengthens Viatris' presence in Japan with the addition of two innovative assets targeting areas of significant unmet medical need; leverages existing infrastructure and expertise in Central Nervous System therapy area; aligned with strategy to target accretive regional business development opportunities; based on the strength of recent Phase 3 clinical trial results in Japanese patients and the positive benefit-risk profile established globally, Viatris is on track to file for marketing approval from the Ministry of Health, Labour and Welfare (MHLW) of Japan for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of excessive daytime sleepiness associated with obstructive sleep apnea syndrome (OSAS) by the end of 2025; the acquisition of Aculys Pharma leverages our deep commercial infrastructure in Japan and longstanding expertise in CNS, positioning us to bring these innovative treatments to more patients in need; the addition of pitolisant and Spydia to our portfolio of innovative products is strategically aligned with our commitment to grow in areas where we can make the greatest impact and is a great example of our business development strategy designed to complement our core strengths in markets across the world; this acquisition further expands Viatris' portfolio of innovative products in Japan which includes Effexor for the treatment of generalized anxiety disorder (GAD) which is under regulatory review, selatogrel in Acute MI, Nefecon in IgA nephropathy, and cenerimod in systemic lupus erythematosus (SLE), all of which have pivotal Phase 3 trials currently on going and Tyrvaya in dry eye disease for which a Phase 3 trial is anticipated to start in 2026; under the terms of the acquisition agreement, Viatris has made an upfront payment to Aculys Pharma shareholders as consideration for the acquisition, with additional consideration contingent upon the achievement of specified regulatory and commercial milestones, and royalties on net sales; positive Pivotal study results in Japanese patients were recently achieved in both narcolepsy and OSAS; in the narcolepsy Phase 3 trial, the primary endpoint of improvement in excessive daytime sleepiness (EDS) compared to a placebo group using the Epworth Sleepiness Scale (ESS) was met; statistically significant difference in ESS was observed between the two groups; furthermore, the key secondary endpoint of the frequency of cataplexy attacks showed a suppression effect comparable to that observed in prior global Phase 3 trials; no serious adverse events were noted, and the safety and tolerability results were consistent with global clinical trials; the OSAS Phase 3 trial evaluated the effect of pitolisant in Japanese patients with OSAS who were experiencing residual EDS despite treatment with CPAP therapy; at the end of the 12-week treatment period, patients receiving pitolisant scored lower on the ESS used to measure EDS compared to those in the placebo group and this difference was statistically significant (p=0.007); additionally, safety and tolerability were consistent with results from global clinical studies. Because forward-looking statements inherently involve risks and uncertainties, actual future results may differ materially from those expressed or implied by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to: Viatris not realizing the anticipated benefits of the acquisition; the uncertainties inherent in research and development, including the outcomes of clinical trials; the ability to meet anticipated clinical endpoints; the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from clinical studies; actions and decisions of healthcare and pharmaceutical regulators; our ability to comply with applicable laws and regulations; changes in healthcare and pharmaceutical laws and regulations in the U.S. and abroad; any regulatory, legal or other impediments to Viatris' ability to bring new products to market; products in development and/or that receive regulatory approval may not achieve expected levels of market acceptance, efficacy or safety; longer review, response and approval times as a result of evolving regulatory priorities and reductions in personnel at health agencies; Viatris' or its partners' ability to develop, manufacture, and commercialize products; the scope, timing and outcome of any ongoing legal proceedings, and the impact of any such proceedings on Viatris; Viatris' failure to achieve expected or targeted future financial and operating performance and results; goodwill or impairment charges or other losses; any changes in or difficulties with Viatris' manufacturing facilities; risks associated with international operations; changes in third-party relationships; the effect of any changes in Viatris' or its partners' customer and supplier relationships and customer purchasing patterns; the impacts of competition; changes in the economic and financial conditions of Viatris or its partners; uncertainties regarding future demand, pricing and reimbursement for Viatris' products; uncertainties and matters beyond the control of management, including but not limited to general political and economic conditions, potential adverse impacts from future tariffs and trade restrictions, inflation rates and global exchange rates; and the other risks described in Viatris' filings with the Securities and Exchange Commission ("SEC"). Viatris routinely uses its website as a means of disclosing material information to the public in a broad, non-exclusionary manner for purposes of the SEC's Regulation Fair Disclosure (Reg FD). Viatris undertakes no obligation to update these statements for revisions or changes after the date of this press release other than as required by law. SOURCE Viatris Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Clinical ResultDrug ApprovalAcquisition

21 May 2025

·www.idorsia.com

New funds secured – allowing the commercial ramp-up of QUVIVIQ to accelerate Idorsia’s path to profitability

Ad hoc announcement pursuant to Art. 53 LR CHF 150 million new money facility from bondholders now implemented and providing cash runway to mid-2026 Holistic convertible debt restructuring progressing with bondholder meeting to be held in the coming weeks – ~90% of bondholders have already agreed to amend the terms and to exchange bonds for newly created notes in Idorsia Investments SARL Idorsia re-activating collaboration discussions for aprocitentan following the removal of the TRYVIO REMS and positive prescriber experience Guidance for 2025 upgraded due to successful commercial ramp-up of QUVIVIQ Financial outlook – company continues to target commercial profitability in 2026 and overall profitability in 2027 Cash flow outlook – company targets positive operating cash flow in 2028 and will therefore need to raise funding for ongoing operations and to bridge to sustainable profitability Allschwil, Switzerland – May 21, 2025Idorsia Ltd (SIX: IDIA) today announced the implementation of the CHF 150 million new money facility previously agreed (press release) with holders of its convertible bond debt. In addition, the company provides an update on the holistic restructuring of the convertible bond debt and an overview of its plans for investment moving forward. The company also upgraded its guidance for 2025 and gave a positive financial outlook beyond. New money facilityA new money facility for a net amount of CHF 150 million to extend Idorsia’s cash runway to mid-2026 has now been signed and the company intends to draw down the first tranche of funds in the coming days. This new money facility has a maturity of 24 months and is fully backstopped by a bondholder group. André C. Muller, CEO of Idorsia, commented: “Securing both the new money facility and the restructuring of the convertible debt overhang allows us to fully concentrate on driving the commercial ramp-up of QUVIVIQ in Europe and Canada which will bring the company to profitability in the near-term, with an upside in the US should the DORA class be descheduled. We will also continue to invest in our R&D portfolio in an efficient and responsible manner to advance selected compounds from our very innovative pipeline to the next inflection points.” Commercial ramp-up of QUVIVIQThe company is working to elevate the conversation around chronic insomnia so that it is established as an independent medical disorder and that the short- and long-term burden of the condition is well understood. The medical, access and commercial teams are tackling long entrenched behaviors to ensure patient access to QUVIVIQ, to help healthcare professionals understand the broad patient population that can benefit from the product’s differentiated profile, and to support patients to get the best out of their QUVIVIQ treatment. As a result of these efforts, QUVIVIQ is taking off in Europe and Canada (EUCAN region) as shown in the first quarter of 2025 (press release) with a 50% growth quarter on quarter, translating into 10 million QUVIVIQ tablets being prescribed in the first quarter of 2025 compared to 15 million tablets in the whole of 2024. Growth is driven by countries where reimbursement has been secured: France, Germany, the UK, Canada (for the private market), and most recently Austria (from June 1, 2025). The company continues to work towards reimbursement in Spain, Canada, and the Nordic region. The successful commercial approach of implementing co-promotion partnerships for the GP market in France and Germany will also be taken in other markets, and the company will look for collaborations with potential partners to expand the European footprint and other geographies such as LATAM and Middle East. In the US, a streamlined, focused, and more cost-efficient commercialization approach for QUVIVIQ has been implemented to maintain sales until the potential descheduling of the dual orexin receptor antagonist (DORA) class can be achieved. Should this occur, a major barrier to prescription will be removed and there is every reason to believe that the true potential of QUVIVIQ can be unlocked. Based on the feedback at medical congress and symposia, the positive experience from prescribers and patients alike, and the commercial ramp-up across the EUCAN region, Idorsia is confident to reach commercial profitability in 2026. Activating the clinical development pipelineIdorsia has a very strong track-record in discovering first- or best-in-class drugs with significant potential to change the treatment paradigms in the target indications. This is matched by the design of efficient development programs that generate the safety and efficacy data that allow prescribers to practice evidence-based medicine. In the short history of the company, Idorsia has secured three product approvals in different geographies. Based on this outstanding success and expertise, the company has prioritized assets in the pipeline that have the greatest potential to create value in the mid-term. The result is a portfolio of potential blockbusters. Further details including the current status of each project in our portfolio can be found in our innovation fact sheet. Holistic convertible bond debt restructuringFollowing a successful completion of the ongoing restructuring, Idorsia will have removed its convertible debt overhang through the creation of an asset-backed new entity where the debt will be sequestered together with Idorsia’s rights for three of the company’s assets. The newly established Luxembourg entity, Idorsia Investments SARL (previously referred to as the “SPV”), will facilitate the holistic restructuring of Idorsia’s convertible bond debt. As of today, bondholders have committed to exchange at least CHF 710 million of the Idorsia convertible bond debt, for newly created notes expected to be issued by Idorsia Investments SARL in the coming weeks, in each case subject to the agreement with such bondholders, which would leave Idorsia Ltd with a maximum of CHF 90 million of convertible bond debt maturing in 2034/2038. Idorsia Pharmaceuticals Ltd will contribute its rights for aprocitentan, selatogrel, and cenerimod, to Idorsia Investments SARL to allow the repayment of the newly created notes. Once the notes have been fully repaid, rights to all three products sequestered to Idorsia Investments SARL will return to Idorsia, delivering long-term value creation to Idorsia shareholders. For more details see the press release issued in February 2025. André Muller concluded: “Following the removal of the TRYVIO REMS and positive prescriber experience in the US, we are excited to have the opportunity to strike a deal for aprocitentan that recognizes this new value proposition. Based on our forecasts for aprocitentan, and the forecasts of our partner Viatris for selatogrel and cenerimod, the three assets sequestered to Idorsia Investments SARL have the potential to not only allow the repayment of the newly created Notes in the short- to mid-term, but to also deliver significant long-term growth for Idorsia once the rights have been returned.” Financial guidance for 2025 For the Idorsia-led portfolio in 2025, the company expects a continued acceleration of QUVIVIQ with net sales of around CHF 130 million, COGS of around CHF 15 million, SG&A expenses of around CHF 200 million, and R&D expense of around CHF 90 million, leading to non-GAAP operating expenses of around CHF 305 million. This performance would result in an Idorsia-led business non-GAAP operating loss of around CHF 175 million and US-GAAP operating loss of around CHF 220 million. The company expects US-GAAP EBIT for the partnered business of around CHF 135 million and mainly driven by the amended deal with Viatris. This would result in a US-GAAP loss for the global business of around CHF 85 million. All amounts exclude unforeseen events and potential revenue related to additional business development activities. Arno Groenewoud, Chief Financial Officer, commented:“With 4 months of QUVIVIQ sales behind us, the new co-promotion collaboration in Germany reaching out to GPs, and the new commercial approach stabilizing in the US, we are clearly seeing the successful commercial ramp-up of QUVIVIQ in action. As a result, I am now confident that we will substantially exceed the sales target by around 20%, compared to our expectations at the beginning of 2025.” Financial outlookIdorsia aims to reach sustainable commercial profitability in 2026 and overall sustainable profitability by the end of 2027. Sustainable profitability only includes income generated from Idorsia’s portfolio of assets. This implies Idorsia-led QUVIVIQ sales ramp-up to around CHF 210 million in 2026 growing to around CHF 270 million in 2027, both at an approximate 80% gross margin. The ramp-up will be predominantly driven by the EUCAN region with a potential upside in the US (depending on DEA descheduling of the DORA class). During this period, non-GAAP OPEX (including marketing and selling, R&D and G&A) will remain stable with annual non-GAAP OPEX of around CHF 285 million. Beyond 2027, Idorsia aims to continue the sales trajectory of QUVIVIQ with a stable OPEX, leading to higher sustainable profitability. This could benefit from additional income from new collaborations from the existing pipeline assets and from the return of sequestered products following the repayment of Idorsia Investments SARL notes. Cash-flow outlookThe company expects to achieve a positive operating cash flow from 2028 onwards. To further extend the cash runway into early 2027, the company has committed to the new money facility lenders to raise CHF 50 million via a new equity line but may also look to other avenues to strengthen its balance sheet. The company will then explore all options to fully finance operations and repay the new money facility at maturity. Notes to the editor Idorsia’s portfolioIdorsia intends to develop certain assets to the next inflection point before partnering, or when feasible and appropriate, developing further with in-house expertise. Other assets are already being prepared for out-licensing. LucerastatLucerastat is an oral inhibitor of glucosylceramide synthase, offering a potential new treatment approach for all patients living with Fabry disease, irrespective of the mutation type of the GLA gene. During a Phase 3 open-label study, with patients treated for up to 6 years, lucerastat has shown a long-term effect on plasma Gb3 levels and improved kidney function compared to historical data. The effect of lucerastat on reducing the eGFR decline is seen across all subgroups of patients with a particularly large effect observed in patients with renal impairment at baseline, and in patients with anti-drug antibody to enzyme replacement therapy, two groups of patients with a high medical need. The analysis also showed a safety and tolerability profile consistent with that observed during the 6-month randomized treatment period. The company is conducting a pilot kidney biopsy sub-study within a subset of patients currently participating in the OLE study. The regulatory pathway will then be further discussed with the US FDA. ACKR3 (CXCR7) receptor antagonistIdorsia's ACKR3 (CXCR7) receptor antagonist, a first-in-class compound that offers a unique combination of re-myelination and anti-inflammatory effects A proof-of-concept study is in preparation for patients with progressive multiple sclerosis. CCR6 receptor antagonistIdorsia's selective CCR6 receptor antagonist is a first-in-class, oral therapy for the treatment of T helper 17 driven immuno-dermatology and autoimmune disorders. A proof-of-concept study is in preparation for patients with psoriasis. CXCR3 receptor antagonistIdorsia's first-in-class oral CXCR3 receptor antagonist allows dual targeting of CD8+ CXCR3+ T cells and melanocytes and offers potential as the first targeted systemic therapy for vitiligo and other immuno-dermatology and autoimmune disorders. There is currently no systemic treatment approved for the treatment of vitiligo. A proof-of-concept study is in preparation for patients with vitiligo. Other early-stage assetsIdorsia also has a synthetic glycan vaccine platform, with vaccines targeting Clostridium difficile, Klebsiella pneumonia, and Neisseria gonorrhea infections, as well as other undisclosed pathogens alone or combined. The results from the lead program, a Phase 1 study in Clostridium difficile infection which will test the immune response of the vaccine and evaluate its safety and tolerability, are expected in in Q2 2025. If positive, Idorsia will seek a partner for the platform or individual vaccines. Further details including the current status of each project in our portfolio can be found in our innovation fact sheet. Idorsia Investments SARL portfolio TRYVIO™/JERAYGO™ (aprocitentan)TRYVIO™ (aprocitentan) is approved in the US for the treatment of hypertension in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs. Lowering blood pressure reduces the risk of fatal and non-fatal cardiovascular events, primarily strokes and myocardial infarctions. TRYVIO is the first and only new treatment to decrease systemic blood pressure on top of existing therapies by blocking the endothelin pathway via both endothelin receptors and has the potential to preserve or improve renal function. TRYVIO is ready to launch with a comprehensive collection of FDA-approved product positioning, branding, websites, materials, training and educational platforms, and field sales force and MSL coverage plans. TRYVIO was made available for prescription in October 2024 and there is ongoing engagement with hypertension experts at major cardiovascular and nephrology congresses and encouraging discussions with payors. In March 2025, the US FDA fully released TRYVIO from its REMS (Risk Evaluation and Mitigation Strategy) requirement to minimize the burden on the healthcare delivery system of complying with the REMS. As a result, a rapid transition from specialty pharmacy to a wide retail pharmacy distribution model is underway. Funding for a field sales force and promotional activities continues to be dependent on a partnership deal. JERAYGO™ (aprocitentan) is approved in the EU and UK for the treatment of resistant hypertension in adult patients in combination with at least three antihypertensive medicinal products. Viatris DealIn 2024, Idorsia entered into a global research and development collaboration with Viatris, for the global development and commercialization rights to selatogrel and cenerimod. Idorsia is entitled to potential development and regulatory milestone payments, and certain contingent payments of additional sales milestone payments and tiered royalties in the mid-single to low-double digit percentages on annual net sales. SelatogrelAcute Myocardial Infarction (AMI) accounts for ~1/3 of all deaths in developed nations and there is a dire need for early intervention, as ~30% of deaths occur prior to hospital admission. Selatogrel has the potential to shift the treatment paradigm in AMI as a potent, reversible and highly selective P2Y12 receptor antagonist, with rapid uptake & fast onset of action and short duration. In the Phase 2 trial, > 90% of participants had > 80% inhibition of platelet aggregation within 15 minutes after dosing, there was reduced off-target interference of hemostasis compared to other P2Y12 inhibitors and no difference in major bleeds compared to placebo on top of standard of care of dual anti-platelet therapy. Comprehensive Phase 3 study design with Special Protocol Assessment was agreed to with FDA and includes a fast-track designation. Currently in Phase 3, on schedule for full enrollment / study expected to read out in 2026. (source: “Selatogrel (AMI) Overview & Market Opportunity” Viatris presentation provided in November 2024). CenerimodCenerimod is a first-in-class oral therapy with a novel mechanism of action and potential for highly differentiated benefit-risk profile in Systemic Lupus Erythematosus (SLE). During the Phase 2 CARE study with over 400 patients, cenerimod 4mg met its primary endpoint, demonstrating statistically significant and clinically meaningful reduction in mSLEDAI-2K1 with a differentiated safety profile vs. existing SLE treatments. FDA fast-track designation, two comprehensive Phase 3 studies ongoing which reflect the learnings from Phase 2, including higher enrollment of INF-1 High patients. Currently in Phase 3, on schedule for full enrollment in 2025 / study expected to readout in 2026. (source: “Cenerimod (SLE) Overview & Market Opportunity” Viatris presentation provided in November 2024). About IdorsiaIdorsia Ltd is reaching out for more – we have more passion for science, we see more opportunities, and we want to help more patients. The purpose of Idorsia is to challenge accepted medical paradigms, answering the questions that matter most. To achieve this, we will discover, develop, and commercialize transformative medicines – either with in-house capabilities or together with partners – and evolve Idorsia into a leading biopharmaceutical company, with a strong scientific core. Headquartered near Basel, Switzerland – a European biotech hub – Idorsia has a highly experienced team of dedicated professionals, covering all disciplines from bench to bedside; QUVIVIQ™ (daridorexant), a different kind of insomnia treatment with the potential to revolutionize this mounting public health concern; strong partners to maximize the value of our portfolio; a promising in-house development pipeline; and a specialized drug discovery engine focused on small-molecule drugs that can change the treatment paradigm for many patients. Idorsia is listed on the SIX Swiss Exchange (ticker symbol: IDIA). For further information, please contact:Investor & Media RelationsIdorsia Pharmaceuticals Ltd, Hegenheimermattweg 91, CH-4123 Allschwil+41 58 844 10 10investor.relations@idorsia.com – media.relations@idorsia.com – www.idorsia.com The above information contains certain "forward-looking statements", relating to the company's business, which can be identified by the use of forward-looking terminology such as "estimates", "believes", "expects", "may", "are expected to", "will", "will continue", "should", "would be", "seeks", "pending" or "anticipates" or similar expressions, or by discussions of strategy, plans or intentions. Such statements include descriptions of the company's investment and research and development programs and anticipated expenditures in connection therewith, descriptions of new products expected to be introduced by the company and anticipated customer demand for such products and products in the company's existing portfolio. Such statements reflect the current views of the company with respect to future events and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements of the company to be materially different from any future results, performances or achievements that may be expressed or implied by such forward-looking statements. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described herein as anticipated, believed, estimated or expected. Attachment Press Release PDF Press Release PDF

Clinical ResultPhase 3Drug ApprovalPhase 2Phase 1

100 Deals associated with Selatogrel

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Indication	Highest Phase	Country/Location	Organization	Date
Acute myocardial infarction	Phase 3	Denmark	Idorsia Pharmaceuticals Ltd.	19 Jul 2021
Coronary Artery Disease	Phase 2	United States	Viatris, Inc.	24 Jan 2018
Coronary Artery Disease	Phase 2	Canada	Viatris, Inc.	24 Jan 2018
Coronary Artery Disease	Phase 2	Denmark	Viatris, Inc.	24 Jan 2018
Coronary Artery Disease	Phase 2	Germany	Viatris, Inc.	24 Jan 2018
Coronary Artery Disease	Phase 2	Netherlands	Viatris, Inc.	24 Jan 2018
Coronary Artery Disease	Phase 2	Singapore	Viatris, Inc.	24 Jan 2018
Coronary Artery Disease	Phase 2	Sweden	Viatris, Inc.	24 Jan 2018
Coronary Artery Disease	Phase 2	United Kingdom	Viatris, Inc.	24 Jan 2018
Coronary Stenosis	Phase 1	China	Idorsia Pharmaceuticals Ltd.	09 Mar 2026

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04557280 (Pubmed \| Clin Pharmacokinet)	Phase 1	-	24	Phase III liquid formulation by autoinjector	afvxtbqlqk(uturkobrxv) = cdldfcmzrj jmokcypjhc (kxyfwmsygg )	-	28 Dec 2021
				Phase I/II reconstituted lyophilizate-based formulation by syringe	afvxtbqlqk(uturkobrxv) = okxngdxpyh jmokcypjhc (kxyfwmsygg )
NCT03487445 (CTgov)	Phase 2	Acute myocardial infarction	48	Selatogrel 8 mg (Selatogrel 8 mg)	pxytjkzufn = nelnjzhqjz azcukjtwvv (tnaagjpxrw, oezwymkgcj - vowwrghlmi) View more	-	25 Aug 2021
				Selatogrel 16 mg (Selatogrel 16 mg)	pxytjkzufn = nmladxnfxe azcukjtwvv (tnaagjpxrw, uowadnhsfd - pkhiwdogyx) View more
NCT03487445 (Pubmed \| J Am Coll Cardiol) Manual	Phase 2	Acute myocardial infarction	47	Selatogrel (a single subcutaneous dose of selatogrel of 8mg)	nbgrhcejdu(qflbuqovzy) = qymecijkeb zlchlpazmf (friyyiylfr, 80 - 100) View more	Positive	26 May 2020
				Selatogrel (a single subcutaneous dose of selatogrel of 16mg)	nbgrhcejdu(qflbuqovzy) = xjgmewbiys zlchlpazmf (friyyiylfr, 87 - 100) View more
NCT03593278 (Pubmed \| Xenobiotica) Manual	Phase 1	-	6	selatogrel	dzauywodvv(fxuqdiklba) = 94.9%, of which 92.5% was recovered in faeces and 2.4% in urine mjjqsnmttn (vmdgndtijg )	Positive	01 Apr 2020
NCT03384966 (Pubmed \| Eur Heart J)	Phase 2	-	345	Selatogrel 8mg	nftntjstzb(vejkzrgvjo) = Transient dyspnoea occurring overall in 7% (16/229) of patients tarlykpaye (tjskyaxnoa )	-	14 Nov 2019
				Selatogrel 16mg

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