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Idorsia Business Report 2020 Business Report 20 ANNUAL REPORT 20 Idorsia – Reaching out for more The purpose of Idorsia is to discover, develop and commercialize innovative medicines to help more patients. We have more ideas, we see more opportunities and we want to transform the horizon of therapeutic options. More science – For a better future 4 Further parts of the Idorsia Annual Report 2020 Contents Be prepared Financial for more Report 20 ANNUAL REPORT 20 Curious to learn more? Reach out to us. 6 Idorsia today Investor Relations Idorsia Pharmaceuticals Ltd Hegenheimermattweg 91 4123 Allschwil Switzerland Phone +41 58 844 10 10 [email protected] © Idorsia Pharmaceuticals Ltd 2020 www.idorsia.com 8 Milestones in 2020 Idorsia – 10 Letter from the Chairman of the Board Reaching out 12 Letter from the Chief Executive Officer 16 Our strategic priorities Be prepared Governance for more Report 2 for more 22 Q&A with the Chief Commercial Officer 20 ANNUAL REPORT 20 Curious to learn more? Reach out to us. Investor Relations Idorsia Pharmaceuticals Ltd Hegenheimermattweg 91 4123 Allschwil Switzerland Phone +41 58 844 10 10 [email protected] © Idorsia Pharmaceuticals Ltd 2020 www.idorsia.com 30 Q&A with the Chief Scientific Officer 36 Clinical development pipeline Be prepared Compensation 38 Daridorexant for insomnia for more Report 46 Aprocitentan for resistant hypertension 20 ANNUAL REPORT 20 52 Clazosentan for cerebral vasospasm Curious to learn more? Reach out to us. Investor Relations Idorsia Pharmaceuticals Ltd Hegenheimermattweg 91 4123 Allschwil Switzerland Phone +41 58 844 10 10 58 Lucerastat for Fabry disease [email protected] © Idorsia Pharmaceuticals Ltd 2020 www.idorsia.com Our Research & 64 Selatogrel for acute myocardial infarction 70 Cenerimod for systemic lupus erythematosus 26 Development 76 Partnerships Contents navigation Our 80 Working at Idorsia > Contents 78 People 88 The Idorsia Executive Committee Idorsia – Reaching out for more Our 91 Compliance, ethics and quality Our Research & 96 Sustainability reporting strategy Development 90 Responsibilities Our People Our Responsibilities 5 >900 Highly qualified professionals 12 Compounds in the pipeline, with six in late-stage development Strong balance sheet >550 State-of-the-art laboratory workspaces 6 More science – Bursting with ideas Idorsia is a high-potential biopharmaceutical Idorsia’s key numbers (non-GAAP* results) company, with an experienced team of over in CHF millions, except EPS (CHF) and 2020 2019 900 highly qualified professionals, a full R&D number of shares (millions) pipeline, state-of-the-art facilities, and a Revenues 72 24 strong balance sheet – the ideal constellation Operating expenses (444) (470) Operating income (loss) (372) (446) for bringing successful medicines to the Net income (loss) (392) (448) market. Basic EPS (2.75) (3.41) Basic weighted average number of shares 142.8 131.2 We began our operations after demerging from Actelion Diluted EPS (2.75) (3.41) following its acquisition by Johnson & Johnson in 2017. At that Diluted weighted average number of shares 142.8 131.2 time, approximately 650 talented and engaged employees were transferred to Idorsia, together with the discovery pipeline and * Idorsia measures, reports and issues guidance on non-GAAP operating performance. Idorsia believes that these non-GAAP financial measurements more accurately reflect early-stage clinical assets. the underlying business performance and therefore provide useful supplementary information for investors. These non-GAAP measures are reported in addition to, not Contents Idorsia is specialized in the discovery and development of small as a substitute for, US GAAP financial performance. The full financial statements can navigation be found in the 2020 Financial Report. molecules, with the aim of transforming the horizon of therapeutic Contents options. We have a broad, diversified and balanced development pipeline, covering multiple therapeutic areas. Our clinical pipeline “2020 was an outstanding year for Idorsia. We will invest during > Idorsia – Reaching out comprises 12 assets, 6 of which are in late-stage development. While 2021 to ensure commercial success of daridorexant in the US and for more we do not yet have any marketed products, we expect to see two clazosentan in Japan, both anticipated to launch in 2022, following product launches in major markets in the first half of 2022, subject market authorization. As a result, our spend will increase in 2021, Our Research & to regulatory approval. With this in mind, we have built a commercial with US GAAP operating expenses of around CHF 685 million and Development organization to bring our products to patients. non-GAAP operating expenses of around CHF 640 million, both Our measures include an inventory build of around CHF 35 million and People Idorsia is headed by Chief Executive Officer Jean-Paul Clozel; he and exclude unforeseen events.” Chief Scientific Officer Martine Clozel (who co-founded Actelion) Our André Muller Responsibilities now hold almost 30 % of Idorsia’s shares. Executive Vice President, Chief Financial Officer 7 Milestones in 2020 Idorsia started the year with 12 compounds in development – 4 in the final stage before filing for May 2020 Successful offering of marketing authorization. 2020 has been an incredible 11 million new shares year for Idorsia – positive Phase 3 results, preparation secured CHF 330 million of funding to prepare for the of our first new drug application, and market launch of daridorexant and preparation for our first products. to develop our diversified pipeline 2020 March 2020 April 2020 May 2020 July 2020 Janssen submitted a new Positive results in the Neurocrine Biosciences Positive results of the drug application to the first Phase 3 study entered into a license second Phase 3 study US FDA and a marketing of daridorexant, agreement for the with daridorexant in authorization application to demonstrating improved development and insomnia, confirming and the EMA for ponesimod for overall sleep and daytime commercialization of reinforcing the efficacy the treatment of adults with functioning of patients Idorsia’s novel T-type and safety profile from relapsing multiple sclerosis* with insomnia calcium channel blocker the first pivotal study * Idorsia and Janssen have a revenue-sharing agreement in respect of ponesimod 8 August 2020 Daridorexant Phase 3 October 2020 July 2020 results in insomnia Successful capital increase January 2021 US commercial operations presented at SLEEP 2020 – secured CHF 535 million New drug application established, with the world’s largest meeting of funding to prepare for for daridorexant leadership team in place devoted entirely to clinical the launch of daridorexant submitted to the US to prepare for the launch sleep medicine, and sleep and to develop our FDA for the treatment of daridorexant and circadian research diversified pipeline of insomnia August 2020 September 2020 November 2020 Idorsia Japan confirmed Syneos Health selected as Positive results in the daridorexant dose commercialization partner Japanese registration response in Japanese to build the salesforce for program for clazosentan, Contents patients with insomnia – the launch of daridorexant demonstrating a reduction navigation preparations for a local in the US in vasospasm-related Contents registration program morbidity and all-cause underway mortality > Idorsia – Reaching out for more Our Research & Development Our People Our Responsibilities 9 The purpose of Idorsia is to discover, develop and commercialize innovative medicines to help more patients. Mathieu Simon Despite the devastation all around us, I am Chairman of the Board very proud and excited to report that Idorsia has not only weathered the storm this year, but gone from strength to strength. Thanks to the investments made during Idorsia’s first few years, our technology platforms are state-of-the-art and scalable. As a result, when the call went out for office-based staff to work from home Dear Shareholders and for those who need to work on-site It is my great honor to write to you as to be protected, our workforce was able Chairman of your company for the first to transition seamlessly. Our laboratory- time. I am only sorry that we have not yet based staff willingly turned up every day, had the opportunity to interact personally. motivated to move the company forward. Our virtual Annual General Meeting was just one of the many disruptions caused by The resilience and determination of our the COVID-19 pandemic – a crisis which has workforce contributed to an incredibly impacted every area of our lives in 2020, successful year: the company has delivered bringing misery and hardship to countless on every one of the ambitious goals set by millions. In our industry, COVID-19 has the management and Board at the end of upended supply chains and clinical trial 2019. Jean-Paul Clozel’s update on these timelines, threatening longer waiting times achievements (see the CEO’s letter) makes for much-needed treatments. for very enjoyable reading. As you reflect 10 “Our workforce’s resilience and determination contributed to an incredibly successful year.” Mathieu Simon Chairman of the Board on these successes, please bear in mind that science and quality, together with a strong Of course, if Idorsia is to become it is very unusual for a clinical pipeline to governance framework, means that the sustainable, we now need to bring our advance so positively. For a young company company has sound foundations
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