Idorsia Ltd. · SIX:IDIA
Idorsia — H1 2019 Earnings Presentation
37 slides from Idorsia's earnings presentation, Jul 23, 2019
Slide 1: Half Year 2019 Financial Reporting
Financial Reporting 20 HALF YEAR 19
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: running our studies, news flow approaching
“This half year has been about running our studies and getting ready for the wave of results and news flow approaching soon.” Jean-Paul Clozel Chief Executive Officer 3 Half Year 2019 Financial Reporting | 23 Jul 2019
Slide 4: More in the pipeline – Promising compounds
Insomnia More in the pipeline – Daridorexant Fabry disease Vasospasm Promising compounds (ACT-541468) Dual orexin Lucerastat Glucosylceramide associated with receptor antagonist synthase inhibitor aneurysmal Diversified and balanced pipeline: Status: Phase 3 Resistant Status: Phase 3 subarachnoid CN
Slide 5: CFO quote: recruiting patients for late-stage trials
“We fully focus on recruiting patients for our late-stage clinical trials and shaping our commercial strategy.” André C. Muller Chief Financial Officer 5 Half Year 2019 Financial Reporting | 23 Jul 2019
Slide 6: Operating results
Operating results 20 HALF YEAR in CHF millions, rounding differences may occur 19 Non-GAAP US-GAAP Financial results as of Jun 30, 2019 6 Half Year 2019 Financial Reporting | 23 Jul 2019
Slide 7: Net results
Net results 20 HALF YEAR in CHF millions, rounding differences may occur 19 -221 -222 Non-GAAP US-GAAP -18 -1 8 -1 -232 -239 Operating results Financial results Income Tax results Non-Controlling Net results Interests Financial results as of Jun 30, 2019 7 Half Year 2019 Financial Reporting | 23 Jul
Slide 8: Liquidity
Liquidity 20 HALF YEAR in CHF millions, rounding differences may occur 19 1,220 Δ - 108 1,111 Δ - 108 298 1,004 300 Cash deposits 123 > 12 months 318 94 Cash deposits < 12 months 219 Cash and Cash equivalents 799 718 467 Liquidity @ Liquidity @ Liquidity @ Dec 31, 2018 Mar 31, 2019 Jun 30, 2019 Fina
Slide 9: Cash flow
Cash flow 20 HALF YEAR in CHF millions, rounding differences may occur 19 Q1 2019 Q2 2019 HY 2019 HY 2018 5 5 5 19 3 14 -113 -122 -138 Contract revenue Working capital -235 requirements -108 -5 -5 Funds from -108 operations -141 Capex / other -5 -216 Financial results as of Jun 30, 2019 9 Half Year
Slide 10: Financial Guidance for 2019
Financial Guidance 20 for 2019 HALF YEAR 19 Unforeseen events excluded, US-GAAP operating expenses of around CHF 570 million and Non-GAAP operating expenses of around CHF 530 million (both measures excluding any potential milestone payments). + + + ++ + 10 Half Year 2019 Financial Reporting | 23 Jul
Slide 11: Quote: Phase 3 program with daridorexant on track
“I'm very pleased that the global Phase 3 program with daridorexant is on track to report 3-month efficacy results in the first half of 2020.” Guy Braunstein Head of Global Clinical Development 11 Half Year 2019 Financial Reporting | 23 Jul 2019
Slide 12: Daridorexant (ACT-541468) – Phase 2 results
Daridorexant (ACT-541468) – Phase 2 results presented at SLEEP 2019 Daridorexant is investigational, in development and not approved or marketed in any country. 12 Half Year 2019 Financial Reporting | 23 Jul 2019
Slide 13: Chronic insomnia disorder
Chronic insomnia disorder Objectively measured by Night Polysomnography (PSG) ≥ 3 nights/week ≥ 3 months • Latency to persistent sleep (LPS) Difficulty • Wake after sleep onset (WASO) falling asleep • Total sleep time (TST) Difficulty Waking too early staying asleep Impaired daytime Patient reported
Slide 14: Rationale for development of daridorexant
Rationale for development of daridorexant • There remains a need for effective and safe treatments for chronic insomnia disorder. • Accumulating evidence for the role of the orexin (OX) system to regulate wake drive has led to the development of new treatments for insomnia disorder that inhibit OX s
Slide 15: Two Phase 2 studies completed
Two Phase 2 studies completed Adult study: parallel group design Elderly study: cross over design • 4-week treatment to assess effect after single • 2-night treatment dose and durability of effect over time • 4 dose levels identical to adults • Short off treatment period at the end to • Placebo-cont
Slide 16: Adult study design
Adult study design 14–28 days 29 days 30 days Screening Double-blind treatment Safety follow-up PSG measurements Sleep diary Zolpidem 10 mg Placebo Daridorexant 5 mg Daridorexant 10 mg Screening Run-in period period Daridorexant 25 mg Single-blind placebo run-in (2 nights) Daridorexant 50 mg On trea
Slide 17: Adult study patient disposition
Adult study patient disposition Entry 1005 screened Sleep diary Subjective confirmation of 664 enrolled in chronic insomnia 36% placebo PSG run-in Polysomnography Objective confirmation of 360 randomized chronic insomnia (359 treated) 344 completed treatment Daridorexant is investigational, in devel
Slide 18: Demographics and baseline characteristics
Demographics and baseline characteristics Adult study (N = 359) Demographic characteristic Baseline sleep parameters Mean (SD) Female, n (%) 230 (64) WASO, min 97.5 (38.6) Mean age, years (SD) 45 (11) LPS, min 71.8 (39.3) Mean BMI, kg/m2 (SD) 25 (3) TST, min 318.5 (56.6) White race, n (%) 321 (89) s
Slide 19: Wake after sleep onset (WASO)
Wake after sleep onset (WASO) Adult study 0 Days 1 & 2 Days 28 & 29 -10 P<0.001 -20 Change from baseline in WASO (min) -30 -40 -50 P=0.050 -60 Placebo 5 10 25 50 Zolpidem 10 mg Daridorexant dose (mg) Data shown as mean (SE) Daridorexant is investigational, in development and not approved or marketed
Slide 20: Latency to persistent sleep (LPS)
Latency to persistent sleep (LPS) Adult study 0 Days 1 & 2 Days 28 & 29 P<0.001 -10 -20 Change from baseline in LPS (min) -30 -40 -50 P=0.042 -60 Placebo 5 10 25 50 Zolpidem 10 mg Daridorexant dose (mg) Data shown as mean (SE) Daridorexant is investigational, in development and not approved or marke
Slide 21: Subjective WASO & LSO
Subjective WASO & LSO Adult study sWASO sLSO Change from baseline in sWASO (min) Change from baseline in sLSO (min) 0 Week 2 0 Week 2 Week 4 P=0.547 Week 4 -10 -10 P=0.074 -20 -20 P=0.107 -30 -30 P=0.045 -40 -40 Zolpidem Zolpidem Placebo 5 10 25 50 Placebo 5 10 25 50 10 mg 10 mg Daridorexant dose (m
Slide 22: Total sleep time (TST) & subjective TST
Total sleep time (TST) & subjective TST Adult study TST sTST Change from baseline in sTST (min) Change from baseline in TST (min) P<0.001 80 80 P=0.006 60 60 40 40 P<0.001 P=0.069 20 20 Days 1 & 2 Week 2 Days 28 & 29 Week 4 0 0 Zolpidem Zolpidem Placebo 5 10 25 50 Placebo 5 10 25 50 10 mg 10 mg Dari
Slide 23: Treatment emergent adverse events
Treatment emergent adverse events Adult study Daridorexant Zolpidem Placebo 5 mg 10 mg 25 mg 50 mg 10 mg n (%) (n=60) (n=60) (n=58) (n=60) (n=61) (n=60) Participants with ≥1 TEAE 18 21 22 23 21 24 AEs leading to treatment discontinuation Angioedema 0 0 0 0 1 0 Anxiety 0 0 0 0 0 1 Arthralgia 0 0 1 0
Slide 24: Karolinska sleepiness scale (morning assessment)
Karolinska sleepiness scale (morning assessment) Adult study Extremely sleepy, 9 fighting sleep 8 Mean KSS score (±SD)* 7 6 Baseline *9-point scale; Days 1 & 2 5 = Neither alert nor sleepy Lower scores indicate less 5 Days 15 & 16 sleepiness 4 Days 28 & 29 3 2 Extremely alert 1 Placebo 5 10 25 50 zo
Slide 25: Elderly study design
Elderly study design Screening Double-blind treatment Safety follow-up Treatment periods 1 2 3 4 5 Washout Washout Washout Washout Screening Placebo Randomization period nights Two consecutive polysomnography nights Daridorexant is investigational, in development and not approved or marketed in any
Slide 26: Elderly study patient disposition
Elderly study patient disposition Entry 149 screened Sleep diary Subjective confirmation of 101 enrolled in chronic insomnia 39% placebo PSG run-in Polysomnography Objective confirmation of chronic insomnia 58 randomized 54 completed treatment Daridorexant is investigational, in development and not
Slide 27: Demographics and baseline characteristics
Demographics and baseline characteristics Elderly study (N = 58) Demographic characteristic Baseline sleep parameters Mean (SD) Median age (range), years 69 (65-85) WASO, min 116.9 (40.2) Sex, n (%) – Male 19 (33) LPS, min 75.1 (50.0) Sex, n (%) – Female 39 (67) TST, min 295.8 (57.6) Mean BMI (SD),
Slide 28: Wake after sleep onset (WASO)
Wake after sleep onset (WASO) Elderly study 0 -10 -20 p ≤ 0.0001 Change from baseline -30 in WASO (min) -40 -50 -60 -70 Placebo 5 10 25 50 Daridorexant dose (mg) Dots: LSMean changes from baseline; bars: 95% CIs; dotted line = 95% CI of curve Daridorexant is investigational, in development and not a
Slide 29: WASO by quarter of the night
WASO by quarter of the night Elderly study 5 Dose-dependent effects were observed for WASO 0 throughout the night -5 Change from baseline -10 in WASO (min) -15 Placebo -20 Daridorexant 5 mg Daridorexant 10 mg -25 Daridorexant 25 mg -30 Daridorexant 50 mg 1st Quarter 2nd Quarter 3rd Quarter 4th Quart
Slide 30: Latency to persistent sleep (LPS)
Latency to persistent sleep (LPS) Elderly study 0 -10 -20 p ≤ 0.025 Change from baseline in LPS (min) -30 -40 -50 -60 Placebo 5 10 25 50 Daridorexant dose (mg) Dots: LSMean changes from baseline; bars: 95% CIs; dotted line = 95% CI of curve Daridorexant is investigational, in development and not app
Slide 31: Subjective WASO & LSO
Subjective WASO & LSO Elderly study 20 sWASO 0 sLSO Baseline mean (overall) = 99.6 min Baseline mean (overall) = 65.7 min Mean change from baseline 0 -10 (min, 95% CI) -20 -20 -40 -30 -60 -40 Placebo 5 10 25 50 Placebo 5 10 25 50 Daridorexant dose (mg) Daridorexant dose (mg) Daridorexant is investig
Slide 32: Total sleep time (TST) & subjective TST
Total sleep time (TST) & subjective TST Elderly study 150 100 TST sTST Baseline mean (overall) = 295.8 min Baseline mean (overall) = 301.7 min 80 Mean change from baseline 100 (min, 95% CI) 60 40 50 20 0 0 Placebo 5 10 25 50 Placebo 5 10 25 50 Daridorexant dose (mg) Daridorexant dose (mg) Daridorexa
Slide 33: Safety results
Safety results Elderly study SB- Daridorexant Placebo Placebo 5 mg 10 mg 25 mg 50 mg n, (%) (N=58) (N=54) (N=56) (N=54) (N=55) (N=56) • No SAE, no deaths Participants with ≥1 6 8 13 12 10 16 adverse event • No narcolepsy-like events Adverse event for ≥2 participants in • No suicidal ideation any dos
Slide 34: Karolinska sleepiness score (morning assessment)
Karolinska sleepiness score (morning assessment) Elderly study Extremely sleepy, 9 fighting sleep 8 7 Mean KSS score (SD) 9-point scale: 6 5 = Neither alert nor sleepy. Lower score indicates Baseline 5 improvement. Next morning after 4 treatment 3 2 Extremely alert 1 Placebo 5 10 25 50 Daridorexant
Slide 35: Conclusion from Phase 2
Conclusion from Phase 2 • In adult and elderly patients with chronic insomnia o daridorexant dose-dependently improved sleep onset, sleep maintenance, and total sleep time o daridorexant was well tolerated o no residual next-morning effect was observed at any dose • Three doses were selected for Pha
Slide 36: From Phase 2 to Phase 3
From Phase 2 to Phase 3 Night Sleep onset Concept • 1,800 adult and elderly chronic insomnia patients in 2 studies Sleep Total sleep time maintenance • Objective and subjective sleep assessment • Assessment of impact on patient’s functioning during the day Daytime • Long-term efficacy and safety (up
Slide 37: Be prepared for more
Be prepared for more 37 Half Year 2019 Financial Reporting | 23 Jul 2019