Idorsia Ltd. · SIX:IDIA
Idorsia — Technical Presentation (July 2020)
21 slides from Idorsia's technical presentation, Jul 6, 2020
Slide 1: Daridorexant – Successful second pivotal study
Daridorexant – Successful second pivotal study Investor Webcast – July 2020
Slide 2: Forward-looking statements
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”,
Slide 3: Quote from Head of Global Clinical Development
“I am delighted to see the replicated effect of 25 mg of daridorexant in this large confirmatory study. The consistency of the treatment effect across both studies is remarkable.” Head of Global ClinicalGuyDevelopment Braunstein 3 Daridorexant – Successful second pivotal study | 06 Jul 2020
Slide 4: Daridorexant registration program
Daridorexant registration program Robust program in adult and elderly insomnia patients Following completion of Phase 2 studies, two similar pivotal studies of 3-month duration in moderate and severe insomnia Efficacy • Objective and subject sleep parameters (onset and maintenance) by polysomnograph
Slide 5: Study design
Study design 2nd pivotal study Screening 20-31days Treatment Period 84 days Safety Follow-up 30 days Single-blind Placebo or 25 mg or 10 mg daridorexant Single- placebo run-in Double-blind blind placebo run-out Randomization 1st month 2nd month 3rd month EOT EOS V1 V2 V3 V4 V5 V6 V7 EODBT V8 V9 V10
Slide 6: Study objectives
Study objectives 2nd pivotal study Primary objective • To evaluate the efficacy of 25 mg and 10 mg daridorexant on objective sleep parameters in patients with insomnia. Secondary objective • To evaluate the efficacy of 25 mg and 10 mg daridorexant on subjective sleep parameters and daytime functioni
Slide 7: Primary and secondary endpoints and analysis
Primary and secondary endpoints and analysis 2nd pivotal study Study-wise type 1 error controlled at 0.05 (across 16 comparisons to placebo) Primary endpoints (night) Two dose levels • Wakening after sleep onset by PSG • 10 mg vs. placebo • Latency to persistent sleep by PSG • 25 mg vs. placebo Seco
Slide 8: Statistical design and hypothesis testing
Statistical design and hypothesis testing α /2 α = 0.05 α/2 H1 H2 WASO LPS H9 H10 H3 WASO LPS 1/2 1/2 sTST High dose H11 vs placebo 1/2 sTST 1/2 Low dose 1/2 1/2 1/2 Month 1 vs 1/4 1/4 1/2 1/2 placebo 1/2 H4 1/4 1/4 Month 1 IDSIQ H12 IDSIQ 1/2 1/2 H5 H6 1/2 1/2 WASO H7 LPS H13 H14 1/2 1/2 WASO LPS s
Slide 9: Study patient disposition
Study patient disposition 2nd pivotal study Entry 3683 screened Sleep diary & polysomnography 25% placebo run-in Subjective & objective confirmation of chronic insomnia 924 randomized 93% 856 completed 837 completed 414 enrolled in long-term treatment placebo run-out double blind extension 9 Daridor
Slide 10: Objective sleep parameters
Objective sleep parameters At 25 mg vs. placebo: highly consistent effect with that of the first pivotal study The study demonstrated the efficacy of daridorexant in objective sleep parameters 50 mg vs 25 mg vs 25 mg vs 10 mg vs 1 st pivotal study placebo placebo 2 nd pivotal study placebo placebo s
Slide 11: Subjective sleep parameters
Subjective sleep parameters SDQ The study demonstrated the efficacy of daridorexant in increasing patient’s assessed total sleep time 50 mg vs 25 mg vs 25 mg vs 10 mg vs 1 st pivotal study placebo placebo 2 nd pivotal study placebo placebo significant significant significant numerical At 1 month sTS
Slide 12: Daytime functioning
Daytime functioning IDSIQ sleepiness domain The study showed numerical improvement of patient’s daytime functioning 50 mg vs 25 mg vs 25 mg vs 10 mg vs 1 st pivotal study placebo placebo 2 nd pivotal study placebo placebo significant numerical numerical numerical At 1 month IDSIQ At 1 month IDSIQ im
Slide 13: Overview of treatment emergent adverse events
Overview of treatment emergent adverse events 1st pivotal study 2nd pivotal study Daridorexant Placebo Daridorexant Placebo 50 mg 25 mg 25 mg 10 mg n = 308 n = 310 n = 309 n = 308 n = 306 n = 306 Subjects with at least one event n (%) n (%) n (%) n (%) n (%) n (%) AE during the double-blind study pe
Slide 14: Most frequent adverse events
Most frequent adverse events* 1st pivotal study 2nd pivotal study Daridorexant Placebo Daridorexant Placebo 50 mg 25 mg 25 mg 10 mg n = 308 n = 310 n = 309 n = 308 n = 306 n = 306 Preferred Term n (%) n (%) n (%) n (%) n (%) n (%) Subjects with at least one event 117 (37.7) 116 (37.7) 105 (34.0) 121
Slide 15: Clinically relevant adverse events
Clinically relevant adverse events* 1st pivotal study 2nd pivotal study Daridorexant Placebo Daridorexant Placebo 50 mg 25 mg 25 mg 10 mg n = 308 n = 310 n = 309 n = 308 n = 306 n = 306 Adjudicated by Independent Safety Board n (%) n (%) n (%) n (%) n (%) n (%) Subjects with at least one event 2 (0.
Slide 16: Two positive pivotal studies
Two positive pivotal studies Remarkable consistency between studies 1st pivotal study 2nd pivotal study Daridorexant 50 mg Daridorexant 25 mg Daridorexant 25 mg Daridorexant 10 mg 1 month 3 months 1 month 3 months 1 month 3 months 1 month 3 months Primary WASO √ √ √ √ √ √ NS* NS* endpoints LPS √ √ √
Slide 17: CEO quote on NDA filing and launch
“The whole company is united in the effort to file the NDA with the US FDA around the end of this year and to prepare for a successful launch.” Jean-Paul Clozel Chief Executive Officer 17 Daridorexant – Successful second pivotal study | 06 Jul 2020 Daridorexant is investigational, in development and
Slide 18: Roadmap to a successful launch
Roadmap to a successful launch The second NDA to be filed pivotal study around the end of confirms the the year – meeting findings of the with FDA set for first Autumn We have a unique The whole company is drug for patients preparing for the with insomnia commercial launch of daridorexant 18 Daridor
Slide 19: Idorsia's strengths
Daridorexant illustrates: Ability to innovate Idorsia’s strengths Drug discovery capability Patient-focused Understanding of what drug development is required for commercial success 19 Daridorexant – Successful second pivotal study | 06 Jul 2020 Daridorexant is investigational, in development and no
Slide 20: Our Strategic Priorities
Our Strategic Priorities Daridorexant is a 2 Build a commercial key component of organization our strategy for 3 Bring Idorsia to value creation profitability in a sustainable manner 1 Deliver at least three products to market 4 Create a pipeline with a sales potential of CHF 5 billion 5 Utilize sta
Slide 21: Program conclusion
Program conclusion The program with daridorexant demonstrated statistically significant and clinically meaningful improvements at month 1 and at month 3 Efficacy during the night and the day Safety and tolerability profile consistent between both pivotal studies Sleep • No dose-dependent treatment e