Idorsia Reaching out for more Idorsia - Investor Webcast - 24 - PowerPoint PPT Presentation

Idorsia – Reaching out for more Idorsia - Investor Webcast - 24 October 2017

The following 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. 2 Idorsia - Investor Webcast | October 2017

We are off to an excellent start Jean-Paul Clozel, CEO

A new venture 1 Innovative deal with J & J is the basis for good start 2 Excellent collaboration with Actelion / J & J 3 Fully functional since day 1 4 Establishing strong foundation for successful future 4 Idorsia - Investor Webcast | October 2017

Idorsia’s strengths The crucial elements for bringing R&D to successful medicines An experienced A full team of research and State-of-the-art CHF 1 billion highly development facilities in cash qualified pipeline professionals 5 Idorsia - Investor Webcast | October 2017

Idorsia’s Clinical Development Pipeline Status Compound Mechanism of Action Target indications Aprocitentan* Endothelin receptor antagonist Resistant hypertension ACT-541468 Dual orexin receptor antagonist Insomnia Phase 2 Cenerimod S1P 1 receptor modulator Systemic lupus erythematosus Clazosentan** Endothelin receptor antagonist Vasospasm associated with aneurysmal subarachnoid hemorrhage Vamorolone*** Non-hormonal steroid modulator Duchenne muscular dystrophy Phase 1b Lucerastat Glucosylceramide synthase inhibitor Fabry disease ACT-246475 P2Y12 receptor antagonist Acute coronary syndrome ACT-774312 CRTH2 receptor antagonist Asthma & allergy disorders Phase 1 ACT-539313 Selective orexin 1 receptor antagonist Anxiety ACT-709478 T-type calcium channel blocker Epilepsy *Johnson and Johnson has option to jointly develop and solely commercialize Aprocitentan worldwide **In Japan a Phase 2 study was completed in 2017 and market registration trials have started ***Idorsia has exclusive option to worldwide rights to ReveraGen’s Vamorolone 6 Idorsia - Investor Webcast | October 2017

Aprocitentan Results from study of oral, potent, once-a-day drug for control of blood pressure • The effect of aprocitentan observed is clinically relevant • The effect of aprocitentan covers the 24 h period • Aprocitentan was well tolerated across all four doses in this patient population • The overall frequency of adverse events was similar on aprocitentan to placebo • It is anticipated that the results can be extrapolated to resistant hypertension • The study provides the necessary information for moving into pivotal registration program Aprocitentan is investigational, in development and not approved or marketed in any country. 7 Idorsia - Investor Webcast | October 2017

ACT-541468 (DORA) Novel dual orexin receptor antagonist for treatment of insomnia Phase 2 program overview: • Two dose-response studies to evaluate the safety and efficacy of DORA in adult and elderly patients with insomnia • Adult study with zolpidem as an active reference − Study 1: 360 adult insomnia patients − Study 2: 58 elderly insomnia patients ACT-541468 is investigational, in development and not approved or marketed in any country. 8 Idorsia - Investor Webcast | October 2017

ACT-541468 (DORA) • Idorsia has significant expertise in the discovery and development of DORAs • DORAs have the potential to promote sleep and maintain a natural sleep architecture • PK/PD profile of ACT-541468 suggests an optimal combination of effect on the CNS and low residual concentration next-day • Both Phase 2 studies, in adult and elderly patients, meet their primary endpoints • Results show desired effect on sleep maintenance and onset – significant dose-response relationship • Idorsia to advance ACT-541468 into pivotal registration program ACT-541468 is investigational, in development and not approved or marketed in any country. 9 Idorsia - Investor Webcast | October 2017

Idorsia’s Clinical Development Pipeline Status Compound Mechanism of Action Target indications Aprocitentan* Endothelin receptor antagonist Resistant hypertension ACT-541468 Dual orexin receptor antagonist Insomnia Phase 2 Cenerimod S1P 1 receptor modulator Systemic lupus erythematosus Clazosentan** Endothelin receptor antagonist Vasospasm associated with aneurysmal subarachnoid hemorrhage Vamorolone*** Non-hormonal steroid modulator Duchenne muscular dystrophy Phase 1b Lucerastat Glucosylceramide synthase inhibitor Fabry disease ACT-246475 P2Y12 receptor antagonist Acute coronary syndrome ACT-774312 CRTH2 receptor antagonist Asthma & allergy disorders Phase 1 ACT-539313 Selective orexin 1 receptor antagonist Anxiety ACT-709478 T-type calcium channel blocker Epilepsy *Johnson and Johnson has option to jointly develop and solely commercialize Aprocitentan worldwide **In Japan a Phase 2 study was completed in 2017 and market registration trials have started ***Idorsia has exclusive option to worldwide rights to ReveraGen’s Vamorolone 10 Idorsia - Investor Webcast | October 2017

Financial Update André Muller, CFO

Operating results Financial results as of 30 September 2017 Non-GAAP US-GAAP -34 -71 -79 -23 -14 -5 -3 Revenues Research Development G&A Non-GAAP D&A SBC US-GAAP Operating Operating results results 12 Idorsia - Investor Webcast | October 2017

Net results Financial results as of 30 September 2017 Non-GAAP US-GAAP -71 -73 -2 +1 -79 -4 -82 Operating results Financial Income Tax Non-Controlling Interests Net results results results 13 Idorsia - Investor Webcast | October 2017

Cash flow Financial results as of 30 September 2017 +22 +2 - 72 1,000 952 Cash at Funds from Working Capital Capex / Liquidity as of Demerger Operations Requirements Other 30 Sep 2017 14 Idorsia - Investor Webcast | October 2017

Liquidity Financial results as of 30 September 2017 952 250 Cash deposits > 3 months Cash deposits 150 < 3 months 1,000 Cash and Cash Equivalents 552 Liquidity at Liquidity as of demerger 30 Sep 2017 15 Idorsia - Investor Webcast | October 2017

Convertible bond Financial results as of 30 September 2017 580 445 135 Equity component 82 Debt component 363 445 Convertible Bond Convertible Bond at demerger as of 30 Sep 2017 16 Idorsia - Investor Webcast | October 2017

Potential issued shares Financial results as of 30 September 2017 Issued common shares = 119.1 million Potential issued shares = 162.7 million 162.7 157.8 4.9 38.7 Equity instruments 50.5 Equity derivatives 119.1 Issued common 107.3 shares Potential shares Potential shares at demerger as of 30 Sep 2017 17 Idorsia - Investor Webcast | October 2017

Financial Guidance for 2017 As of 24 October 2017 Non-GAAP Operating Expenses = -10 • CHF -160 / -170 million -61 2017 = 6 ½ months activity since • demerger from Actelion -89 -99 -160 -170 Q2 2017 Q3 2017 Q4 2017 FY 2017 (Actual) (Actual) (Estimate) (Estimate) 18 Idorsia - Investor Webcast | October 2017

Execution becomes the strategy 19 Idorsia - Investor Webcast | October 2017