Idorsia Company Profile

Headquartered in – a biotech-hub of Europe – Idorsia is a high-potential company, specialized in the discovery, development and commercialization of innovative small molecules, with the aim of transforming the horizon of therapeutic options. The company has an experienced team of over 1,000 highly qualified professionals, a full R&D pipeline, state-of-the-art facilities, and a strong balance sheet – the ideal constellation to bringing R&D efforts to business success.

Company Financial overview First Half Profile in CHF millions, except EPS (CHF) and US GAAP Non-GAAP* > Financial number of shares (millions) 2021 2020 2021 2020 Overview The full financial statements can be found in Revenues 14 58 14 58 the Financial Report available on our corporate Company website. Strategy Operating expenses (265) (236) (248) (193) Operating income (loss) (252) (178) (234) (134) * Idorsia measures, reports and issues guidance on Clinical non-GAAP operating performance. Idorsia believes Net income (loss) (243) (189) (223) (138) Development that these non-GAAP financial measurements Pipeline Basic EPS (1.46) (1.41) (1.34) (1.03) more accurately reflect the underlying business performance and therefore provide useful Basic weighted average number of shares 166.9 133.8 166.9 133.8 Partnerships supplementary information to investors. These Diluted EPS (1.46) (1.41) (1.34) (1.03) non-GAAP measures are reported in addition Company to, not as a substitute for, US GAAP financial Diluted weighted average number of shares 166.9 133.8 166.9 133.8 Milestones performance. Share Information Company Strategy

Idorsia was incorporated in March 2017 We will develop Idorsia into one of Europe’s Bring Idorsia to sustainable profitability and listed on the SIX Swiss Exchange (ticker leading biopharmaceutical companies, with We are building Idorsia with a long-term symbol: IDIA) in June 2017. a strong scientific core. We have identified focus and ambitious aspirations. By five key strategic priorities to ensure the advancing our development pipeline and Idorsia Ltd is part of the following indices: company’s success in the mid-term: commercial readiness, we aim to bring SPI, SPIEX, SPI ESG, SXSLI, SXI Life Sciences, Idorsia to sustainable profitability as soon as SXI Bio+Medtech, and SSIRT. Deliver at least three products to market possible. We believe that our development Idorsia is traded under the following compounds have the potential to Fuel our pipeline with new discoveries symbols: significantly change treatment in their While building up our commercial operations Reuters IDIA.S / Bloomberg IDIA:SW. target diseases, resulting in medicines with and developing our late-stage clinical substantial commercial potential. pipeline so as to bring innovative therapies to patients, we also continue to discover Build a world-class commercial new compounds. organization In order to bring pioneering therapies to Utilize state-of-the-art technologies to patients and to maximize the value of our drive innovation innovations, we plan to continue building As we wish to remain at the cutting edge of and integrating our global commercial science, it is vital that we consider innovative organization. approaches and utilize state-of-the-art Company technologies at each stage of the process, Profile from bench to bedside. Financial Overview

> Company Strategy

Clinical Development Pipeline

Partnerships

Company Milestones Clinical Development Pipeline

Compound Mechanism of Action Target Indication Status

Daridorexant Dual orexin receptor antagonist Insomnia Under review with health authorities

Aprocitentan* Dual endothelin receptor antagonist Resistant hypertension management Phase 3 recruitment complete

Clazosentan Endothelin receptor antagonist Cerebral vasospasm associated with aneurysmal Japan: NDA submitted subarachnoid hemorrhage Global: Phase 3 ongoing

Lucerastat Glucosylceramide synthase inhibitor Fabry disease Phase 3 recruitment complete

Selatogrel P2Y12 receptor antagonist Suspected acute myocardial infarction Phase 3

Cenerimod S1P1 receptor modulator Systemic lupus erythematosus Phase 2b recruitment complete

ACT-539313 Selective orexin 1 receptor antagonist Binge eating disorder Phase 2

Sinbaglustat GBA2/GCS inhibitor Rare lysosomal storage disorders Phase 1 complete

ACT-1004-1239 CXCR7 antagonist Immunology Phase 1

ACT-1014-6470 - Immunology Phase 1 Company Profile ACT-777991 - Immunology Phase 1 Financial Overview * In collaboration with to jointly develop aprocitentan, Janssen Biotech has sole commercialization rights worldwide

Company Neurocrine Biosciences has a global license to develop and commercialize ACT-709478 (NBI-827104), Idorsia's novel T-type calcium channel blocker. ACT-709478 is Strategy currently investigated in two Phase 2 studies for the treatment of a rare form of pediatric epilepsy and essential tremor.

> Clinical Development Pipeline

Partnerships

Company Milestones Partnerships

Collaboration Agreement with Janssen Revenue Sharing Agreement with License agreement with Neurocrine Biotech, Inc. Johnson & Johnson In 2020, Idorsia entered into a global license In 2017, Idorsia entered into a collaboration In 2017, Idorsia and Actelion agreement with Neurocrine Biosciences for agreement with Janssen Biotech, one of Pharmaceuticals Ltd, one of the Janssen the development and commercialization the Janssen Pharmaceutical Companies Pharmaceutical Companies of Johnson of ACT-709478, Idorsia’s potent, selective, of Johnson & Johnson, to jointly develop & Johnson, have entered into a revenue- orally active and brain-penetrating T-type aprocitentan and any of its derivative sharing agreement in respect of ponesimod. calcium channel blocker, for the treatment compounds or products. Janssen Biotech of a rare pediatric epilepsy, and a research has sole commercialization rights worldwide. Under the terms of the revenue-sharing collaboration to discover, identify and agreement, Idorsia is entitled to receive develop additional novel T-type calcium quarterly payments of 8% of the net sales of channel blockers. ponesimod products from Actelion. Further Collaborations Idorsia also has agreements in place with Antares, Mochida, Santhera, and Syneos. For more information visit our corporate website.

Company Profile

Financial Overview

Company Strategy

Clinical Development Pipeline

> Partnerships

Company Milestones Company milestones 2021 2019 2018

June December June Initiation of Phase 3 registration study License Agreement with Mochida for the Initiation of Phase 3 registration study with with selatogrel for the treatment of acute supply, co-development and co-marketing of aprocitentan for resistant hypertension myocardial infarction daridorexant in Japan management

March November June Marketing authorisation application (MAA) Collaboration Agreement with Antares Establishment of Idorsia Pharmaceuticals for daridorexant submitted to the EMA for the development of a novel self- Japan administered drug-device product for March selatogrel May New drug application (NDA) for clazosentan Initiation of Phase 3 registration study with submitted to the Japanese PMDA July lucerastat for patients with Fabry disease Janssen reported positive top-line Phase 3 January results for ponesimod in adults with New drug application (NDA) for daridorexant relapsing multiple sclerosis 2017 submitted to the US FDA February December Initiation of Phase 3 registration program Collaboration Agreement with Janssen 2020 with clazosentan for patients with cerebral Biotech Inc. to jointly develop and vasospasm associated with aSAH commercialize aprocitentan Company September Profile Idorsia selects Syneos Health as January June Financial commercialization partner to launch Initiation of multipledose efficacy and safety Idorsia opens its doors and is listed on SIX Overview daridorexant in the United States study with cenerimod for the treatment of Swiss Stock Exchange

Company systemic lupus erythematosus Strategy May Global license agreement with Neurocrine Clinical Biosciences for the development and Development Pipeline commercialization of ACT-709478

Partnerships

> Company Milestones Idorsia is an independent biopharmaceutical company based on science and innovation. The company is specialized in the discovery and development of small molecules, to transform the horizon of therapeutic options. It is headquartered in Allschwil/, Switzerland and is quoted on the SIX Swiss Exchange (tickersymbol: IDIA). All trademarks are legally protected by their respective owners.

Disclaimer This fact sheet has the sole purpose to provide members of the public with general information about the activities of Idorsia. The forward-looking statements in this fact sheet are based on current expectations and belief of company management, which are subject to numerous risks and uncertainties.

Idorsia Pharmaceuticals Ltd Hegenheimermattweg 91 4123 Allschwil Switzerland

[email protected] Phone +41 58 844 10 10 www.idorsia.com

Company Profile

Latest update: July 2021 Copyright © 2021 Idorsia