Idorsia Ltd. · SIX:IDIA
Idorsia — Technical Presentation (April 2020)
25 slides from Idorsia's technical presentation, Apr 20, 2020
Slide 1: Daridorexant – Successful first pivotal study
Daridorexant – Successful first pivotal study Investor Webcast – April 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: CEO quote on the results
“While we designed daridorexant to have the optimal profile for a sleep medicine, I am none-the-less stunned by the results.” Jean-Paul Clozel Chief Executive Officer 3 Daridorexant – Successful first pivotal study | 20 Apr 2020 Daridorexant is investigational, in development and not approved or mar
Slide 4: CSO quote on the results
“The results have made working for over 20 years on the project completely worthwhile and prove that we were right to persevere.” Martine Clozel Chief Scientific Officer 4 Daridorexant is investigational, in development and not approved or marketed in any country.
Slide 5: The optimization process
The optimization process Potent DUAL OX1 and OX2 receptor blockade in vitro No major drug- drug interaction High brain penetration Optimal in Optimized duration of vivo efficacy action (fast onset, no next-day hangover) > 25,000 1,361 236 83 12 Daridorexant compounds synthesized in the Human orexin
Slide 6: Head of Global Clinical Development quote
“The results from this pivotal study are truly remarkable… this is the first study to demonstrate an insomnia product can improve how the patient feels during the day.” Guy Braunstein Head of Global Clinical Development 6 Daridorexant – Successful first pivotal study | 20 Apr 2020
Slide 7: Insomnia: A disease of the night and the day
Insomnia: A disease of the night and the day ≥ 3 nights/week ≥ 3 months Difficulty staying asleep Insomnia patients’ desire from a Difficulty pharmaceutical intervention: falling asleep Waking too early 1 - Increase in total sleep time 2 - Improvement in daily performance Impaired daytime performanc
Slide 8: Key assessments in daridorexant Phase 3 studies
Key assessments in daridorexant Phase 3 studies Objective sleep Patient’s oriented outcome assessment daily recorded Objective measures of night Subjective measures of night Assessment of impact of parameters by parameters by the sleep diary insomnia during the day by the polysomnography (PSG) in qu
Slide 9: Objective sleep assessments
Objective sleep assessments Repeated polysomnography recordings in a sleep lab in all patients • Assess insomnia objectively • Ensure well-characterized insomnia patients are randomized Sensors • Establish solid baseline during placebo run-in measure brain activity, eye • Measure primary endpoint at
Slide 10: Sleep diary questionnaire (SDQ) daily recording
Sleep diary questionnaire (SDQ) daily recording Morning questionnaire Evening questionnaire • 10 questions related to medication, • 2 questions related to napping quantification of sleep, awakenings • 2 visual analog scales related to • 3 visual analog scales related to alertness and ability to perf
Slide 11: Subjective assessment of daytime performance
Subjective assessment of daytime performance Insomnia Daytime Symptoms and Impacts Questionnaire (IDSIQ) validated according to FDA guidelines Measures 19:30 1 Clear-Headed 2 Concentrate “Alert/cognition” 3 Forgetful domain score 4 Worried 11. How mentally tired did you feel today ? 5 Frustrated 6 I
Slide 12: 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 PSG and SDQ •
Slide 13: Study design
Study design Screening 20-31days Treatment Period 84 days Safety Follow-up 30 days Single-blind Placebo or 25 mg or 50 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 V11 25 mg 50 mg Pl
Slide 14: Study objectives
Study objectives Primary objective • To evaluate the efficacy of 25 mg and 50 mg daridorexant on objective sleep parameters in patients with insomnia. Secondary objective • To evaluate the efficacy of 25 mg and 50 mg daridorexant on subjective sleep parameters and daytime performance in patients wit
Slide 15: Primary and secondary endpoints and analysis
Primary and secondary endpoints and analysis 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 − 50 mg vs. placebo − Latency to persistent sleep by PSG − 25 mg vs. placebo • Secondary endpoi
Slide 16: Study patient disposition
Study patient disposition Entry 3326 screened Sleep diary & polysomnography 28% placebo run-in Subjective & objective confirmation of chronic insomnia 930 randomized 92% 853 completed 847 completed 392 enrolled in long-term treatment placebo run-out double blind extension 16 Daridorexant – Successfu
Slide 17: Demographics and baseline characteristics
Demographics and baseline characteristics N = 930 Demographic characteristic The study was conducted at 75 hospitals and Female, n (%) 624 (67.1) sleep centers in 10 countries (Germany, Australia, Mean age, years (SD) 55.4 (15.3) Canada, Denmark, Italy, Poland, Serbia, Spain, BMI Switzerland and the
Slide 18: Objective sleep parameters
Objective sleep parameters At both doses of 50 and 25 mg vs. placebo: highly consistent effect Primary Endpoints The study demonstrated the efficacy of daridorexant in inducing and maintaining sleep • Statistically significant improvement in LPS at 1 month • Statistically significant improvement in
Slide 19: Subjective sleep parameter
Subjective sleep parameter SDQ Secondary Endpoints The study demonstrated the efficacy of daridorexant in increasing patient’s assessed total sleep time at both doses of 50 and 25 mg vs. placebo • Statistically significant improvement in sTST at 1 month • Statistically significant improvement in sTS
Slide 20: Daytime performance
Daytime performance IDSIQ Secondary Endpoints The study demonstrated the efficacy of daridorexant in improving patient’s daytime performance 50 mg • Statistically significant improvement in IDSIQ sleepiness domain at 1 month • Statistically significant improvement in IDSIQ sleepiness domain at 3 mon
Slide 21: Overview of treatment emergent adverse events
Overview of treatment emergent adverse events Daridorexant 25 mg Daridorexant 50 mg Placebo n = 310 n = 308 n = 309 n (%) n (%) n (%) Subjects with at least one: AE during the double-blind study period 117 (37.7) 116 (37.7) 105 (34.0) AEs leading to premature discontinuation of double-blind study tr
Slide 22: Adverse events during the DB study period
Adverse events during the DB study period Daridorexant 25 mg Daridorexant 50 mg Placebo N = 310 N = 308 N = 309 Preferred Term n (%) n (%) n (%) Subjects with at least one event 117 (37.7) 116 (37.7) 105 (34.0) Most frequent adverse events (based on 50 mg daridorexant) Nasopharyngitis 21 ( 6.8) 20 (
Slide 23: Study conclusion
Study conclusion Daridorexant 25mg and 50 mg Consistency of efficacy across Safety and tolerability profile at the tested doses • Objective and subjective endpoints • Doses • Rate of AE similar between placebo and daridorexant at both treatment • Timepoints Daridorexant 25 mg Daridorexant 50 mg dose
Slide 24: Idorsia is entering a new era
Idorsia is entering a new era 24 Daridorexant – Successful first pivotal study | 20 Apr 2020 Daridorexant is investigational, in development and not approved or marketed in any country.
Slide 25: Study conclusion: Daridorexant 25mg and 50 mg
Study conclusion: Daridorexant 25mg and 50 mg Treatment 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 at the tested doses Sleep • Rate of AE similar between placebo and mai