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Idorsia Reaching out for more Idorsia - Investor Webcast - 6 - PowerPoint PPT Presentation

Idorsia Reaching out for more Idorsia - Investor Webcast - 6 February 2018 The following information contains certain forward-looking statements, relating to the companys business, which can be identified by the use of


  1. Idorsia – Reaching out for more Idorsia - Investor Webcast - 6 February 2018

  2. 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 Full Year Financial Reporting

  3. More novel drugs – Inspiring potential Jean-Paul Clozel, CEO

  4. Highlights in 2017 Pipeline Results Substantial progress with the pipeline – productive discussions with health authorities Idorsia is born Idorsia created and listed – pipeline advanced without missing a beat Partnerships Development partnership with Janssen Biotech to advance aprocitentan Research collaboration with Roche in the field of cancer immunotherapy 4 Full Year Financial Reporting

  5. Idorsia Today >650 Highly qualified 11 professionals Compounds in the pipeline, with four progressing to late-stage development in the year ahead >1 bn > 550 Swiss francs in cash State-of-the-art laboratory workspaces 5 Full Year Financial Reporting

  6. Our Strategic Priorities 1 Deliver at least three products to market 5 key priorities to ensure the 2 company’s success Build a commercial organization over the next 5 years 3 Bring Idorsia to profitability in a sustainable manner 4 Create a pipeline with a sales potential of CHF 5 billion 5 Utilize state-of-the-art technologies 6 Full Year Financial Reporting

  7. More in the pipeline – Resistant Fabry disease hypertension Promising compounds Lucerastat Management Glucosylceramide Vasospasm associated Aprocitentan synthase inhibitor with aneurysmal Dual endothelin Status: Advancing to subarachnoid Diversified and balanced pipeline: CNS, receptor antagonist Phase 3 hemorrhage (aSAH) Insomnia Status: Advancing to cardiovascular and immunological Phase 3 Clazosentan disorders & orphan diseases ACT-541468 Endothelin receptor Dual orexin receptor antagonist antagonist Status: Advancing to Status: Advancing to Phase 3 Phase 3 Systemic lupus Duchenne muscular erythematosus dystrophy Cenerimod Vamorolone S1P 1 receptor Dissociative steroid modulator Acute Coronary Status: Phase 2 Status: Phase 2 Syndrome Idorsia has an exclusive option to the worldwide rights for ReveraGen's vamorolone ACT-246475 P2Y12 receptor antagonist Status: Phase 2 Asthma and allergy Orphan CNS disorders diseases ACT-774312 Anxiety ACT-519276 CRTH2 receptor GBA2/GCS inhibitor ACT-539313 antagonist Epilepsy Selective orexin 1 Status: Phase 1 Status: Phase 1 receptor antagonist ACT-709478 Status: Phase 1 T-type calcium channel blocker Status: Phase 1 7 Full Year Financial Reporting

  8. More in the pipeline – Resistant Fabry disease hypertension Promising compounds Lucerastat Management Glucosylceramide Vasospasm associated Aprocitentan synthase inhibitor with aneurysmal Dual endothelin Status: Advancing to subarachnoid Diversified and balanced pipeline: CNS, receptor antagonist Phase 3 hemorrhage (aSAH) Insomnia Status: Advancing to cardiovascular and immunological Phase 3 Clazosentan disorders & orphan diseases ACT-541468 Endothelin receptor Dual orexin receptor antagonist antagonist Status: Advancing to Status: Advancing to Phase 3 Phase 3 8 Full Year Financial Reporting

  9. Lucerastat for Fabry disease • Fabry disease is a rare genetic disorder involving deficiency or Fabry disease dysfunction of an enzyme that breaks down fatty waste products Compound: Lucerastat in body • Lucerastat oral monotherapy offering new treatment approach Mechanism of action: with potential to alleviate symptoms of Fabry disease Glucosylceramide synthase inhibition • Phase 3 study to assess effects of compound on neuropathic pain Status: Advancing to Phase 3 and gastrointestinal symptoms and safety and tolerability – to start in first half of 2018 • Lucerastat has orphan drug status in US and EU Lucerastat is investigational, in development and not approved or marketed in any country. 9 Full Year Financial Reporting

  10. ACT-541469 for insomnia • Potential for fast onset of sleep and duration of action not Insomnia exceeding a normal night , while maintaining natural sleep Compound: ACT-541468 architecture Mechanism of action: • Phase 3 program consisting of three studies to evaluate time to sleep onset and duration of action in patients with insomnia – Dual orexin expected to start in first half of 2018 receptor antagonism Status: Advancing to Phase 3 Registration program based on feedback from health authorities • ACT-541468 is investigational, in development and not approved or marketed in any country. 10 Full Year Financial Reporting

  11. Aprocitentan for resistant hypertension management • Aprocitentan has great potential in resistant hypertension Resistant hypertension management as an oral, once daily treatment with potent, long management lasting hypertensive effects Compound: Aprocitentan Mechanism of action: Dual • In collaboration with Janssen Biotech to jointly develop and commercialize product endothelin receptor antagonism • Phase 3 study to evaluate initial and long-term effects on systolic Status: Advancing to Phase 3 and diastolic blood pressure in patients requiring resistant hypertension management – expected to start in first half 2018 • Registration program based on feedback from health authorities Aprocitentan is investigational, in development and not approved or marketed in any country. 11 Full Year Financial Reporting

  12. Clazosentan for cerebral vasospasm associated with aSAH* • Developed as intravenous infusion for prevention and treatment Cerebral vasospasm of cerebral vasopasms in patients who have suffered an aSAH Compound: Clazosentan Has potential to prevent ischemic complications of cerebral • vasoconstriction and to decrease the need for invasive Mechanism of action: intervention Endothelin receptor antagonism • Registration studies in Japan expected to complete in second half Status: Advancing to Phase 3 of 2018 Phase 3 study evaluating the safety and efficacy of clazosentan in • an enriched aSAH population to start later in 2018 * aneurysmal subarachnoid hemorrhage Clazosentan is investigational, in development and not approved or marketed in any country. 12 Full Year Financial Reporting

  13. More in the pipeline – Promising compounds Diversified and balanced pipeline: CNS, cardiovascular and immunological disorders & orphan diseases Systemic lupus Duchenne muscular erythematosus dystrophy Cenerimod Vamorolone S1P 1 receptor Dissociative steroid modulator Acute Coronary Status: Phase 2 Status: Phase 2 Syndrome Idorsia has an exclusive option to the worldwide rights for ReveraGen's vamorolone ACT-246475 P2Y12 receptor antagonist Status: Phase 2 Asthma and allergy Orphan CNS disorders diseases ACT-774312 Anxiety ACT-519276 CRTH2 receptor GBA2/GCS inhibitor ACT-539313 antagonist Epilepsy Selective orexin 1 Status: Phase 1 Status: Phase 1 receptor antagonist ACT-709478 Status: Phase 1 T-type calcium channel blocker Status: Phase 1 13 Full Year Financial Reporting

  14. More energy – Growing and delivering André Muller, CFO

  15. Operating results -48 Non-GAAP US-GAAP 158 -75 -27 -10 8 -6 -8 Revenues Research Development G&A Non-GAAP D&A SBC US-GAAP Operating Operating results results Financial results as of Dec 31, 2017 15 Full Year Financial Reporting

  16. Net results Non-GAAP US-GAAP 8 5 -8 -14 -3 -7 Operating results Financial Income Tax Non-Controlling Net results results results Interests Financial results as of Dec 31, 2017 16 Full Year Financial Reporting

  17. Cash flow 14 227 -150 1'091 1'000 Cash @ OPEX J&J Capex Liquidity @ Demerger (excl. milestone) milestone & WCR Dec 31, 2017 Financial results as of Dec 31, 2017 17 Full Year Financial Reporting

  18. Liquidity 1’091 250 Cash deposits 218 long-term Cash deposits short-term 1'000 Cash and Cash Equivalents 622 Liquidity @ Liquidity @ Demerger Dec 31, 2017 Financial results as of Dec 31, 2017 18 Full Year Financial Reporting

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