Idorsia Ltd. · SIX:IDIA
Idorsia — Fact Sheet (July 2025)
23 slides from Idorsia's fact sheet, Jul 30, 2025
Slide 1: Focused innovation – from bench to bedside
Focused innovation – from bench to bedside Idorsia aims to deliver new products with the potential to significantly change the treatment options in their target diseases. We want to bring new perspectives to the discovery, development, and commercialization of innovative treatments, challenging acce
Slide 2: Key portfolio assets
Key portfolio assets Compound Target indication Mechanism of action P1 P2 P3 R C Status QUVIVIQ™ Insomnia Dual orexin receptor Commercially available in the US, Germany, Italy, Switzerland, Spain, (daridorexant) antagonist the UK, Canada, Austria, France, Sweden and Finland; approved throughout the
Slide 3: Partner-led portfolio
Partner-led portfolio Compound Target indication Mechanism of action Partner P1 P2 P3 R C Status Terms TRYVIO™ Systemic hypertension in Dual endothelin receptor In partnership discussions: Commercially available in (aprocitentan)* combination with other antagonist worldwide development and the US an
Slide 4: Daridorexant
Daridorexant Daridorexant is a dual orexin receptor antagonist (DORA) which blocks the binding of the wake- promoting orexin neuropeptides. Rather than inducing sleep through broad inhibition of brain activity, daridorexant selectively blocks the activation of orexin receptors. Consequently, daridor
Slide 5: Daridorexant pivotal trial results
in a sleep lab by polysomnography and A major focus of the trials was to evaluate observed upon abrupt discontinuation subjectively with a daily patient diary at the impact of daridorexant on daytime of treatment. Across treatment groups, home. The impact of insomnia on patients’ functioning in pati
Slide 6: Daridorexant sleep parameters over time
Wake W akafter time e tim e afonset sleep ter sleep onset Latency to persistent sleep Wake time after sleep onset Latency to persistent sleep Latency to persistent sleep 120 120 80 placebo 25 mg 50 mg 80 placebo 25 mg 50 mg Mean of observed values (min) 100 Mean of observed values (min) 100 Mean of
Slide 7: Daridorexant total sleep time and daytime functioning
Subjective total sleep time IDSIQ sleepiness domain SuS bujebcjteicvteivteottaoltsallesele petpimtieme SubSjIe ID D IQcStIsiQ vle esetlp oietnapelisnsse e led sesopd mtoaim inaein 400400 placebo25 mg placebo 25 mg50 mg 50 mg 24 24400 placebo 25 mg 50 mg 2 p<0.0001 p<0.0001 p<0.0001 Mean of observed
Slide 8: Phase 4 study in patients with chronic insomnia and nocturia
Phase 4 study in patients with chronic the US in May 2022. For more information Sweden in September 2024, and Finland in insomnia and nocturia about QUVIVIQ in the US, see the June 2025. In March 2025, data from a Phase 4 study Full Prescribing Information. with daridorexant in patients with chronic
Slide 9: Current status in global clinical development
Current status in global clinical Analysis of the primary endpoint Beyond sleep, the results suggest that development demonstrated a statistically orexin signaling may play a broader role In March 2026, Idorsia announced positive significant (p=0.0185) dose‑dependent in children with neurodevelopmen
Slide 10: Milestones and key scientific literature
Milestones Key scientific literature 2026 Positive Phase 2 study in children with insomnia • Dauvilliers Y et al. Sleep Medicine. 2025. doi: disorder 10.1016/j.sleep.2025.106523. 2025 QUVIVIQ launched by Simcere in China and • Lederer K et al. Journal of Sleep Research. 2025. Hong Kong doi: 10.1111/
Slide 11: Aprocitentan
Aprocitentan Aprocitentan is a once-daily, orally active, dual endothelin receptor antagonist, which inhibits the binding of ET-1 to ETA and ETB receptors. Aprocitentan has a low potential for drug-drug interaction and a mechanism of action that is ideally suited for lowering blood pressure in adult
Slide 12: Aprocitentan sitting systolic blood pressure efficacy
Aprocitentan has significant and sustained efficacy Part 1 Part 2 Part 3 Double blind Single blind Double blind withdrawal 155 Sitting systolic blood pressure (mmHg) 150 Placebo Aprocitentan 12.5 mg 145 Aprocitentan 25 mg † * *Primary endpoint 140 P=0.0042 for aprocitentan 12.5 mg vs placebo P=0.00
Slide 13: Ambulatory BP monitoring at Week 4
Efficacy confirmed by Ambulatory BP monitoring at Week 4 150 Daytime Night-time 145 0 0 systolic blood pressure (mmHg) 140 blood pressure (mmHg) -2 -2 Ambulatory systolic Change in ambulatory 135 -4 -4 130 -6 -6 125 * -8 -8 ** 120 -10 -10 † 115 -12 -12 ** 0 6 12 18 24 No. of patients 179 175 182 17
Slide 14: Aprocitentan unattended office SBP results
in unattended office SBP from baseline to and decreased within the first 2 weeks of ≥ 75 years), sex, race (including patients week 4 and from withdrawal baseline to Part 2 before stabilizing in those previously with Black or African American origin), BMI, week 40, respectively. Secondary endpoints
Slide 15: Current status in the US
Current status in the US tolerating the 12.5 mg dose and in need of Milestones tighter blood pressure (BP) control. 2025 Approved as JERAYGO (aprocitentan) by Health Canada For more information on the marketing 2025 Approved as JERAYGO (aprocitentan) by authorization of JERAYGO in the European Swiss
Slide 16: Lucerastat
Lucerastat Lucerastat is an oral inhibitor of glucosylceramide synthase, offering a potential new treatment approach for all patients living with Fabry disease, irrespective of the mutation type of the GLA gene. Fabry disease is a rare, X-linked lysosomal Lucerastat, an oral inhibitor of While the M
Slide 17: Lucerastat current status
loss in patients with severe disease course, ERT naïve or pseudo-naïve and 2 switched Current status such as classic males with Fabry disease from ERT. Median kidney Gb3 BLISS score Idorsia has aligned with the US FDA (historical: -4.32; Lucerastat: -2.05), was 1.7 (range: 0.7–4.5; mean (SD): 2.41 o
Slide 18: Milestones and key scientific literature
Milestones 2026 New data and evaluation of long-term treatment with lucerastat lucerastat presented at WORLDSymposium™ 2026 2021 Phase 3 open label extension study continues 2021 Phase 3 study completed – primary endpoint not met 2018 Phase 3 study initiated 2016 Phase 1b study completed Key scienti
Slide 19: Early-stage clinical pipeline
Early-stage clinical pipeline Idorsia has developed platforms of expertise in families of molecular targets which allow high productivity in the generation of innovative compounds potentially addressing a wide range of high unmet medical needs. IDOR-1117-2520 – CCR6 antagonist ACT-777991 – CXCR3 ant
Slide 20: Preclinical pipeline
Preclinical pipeline Idorsia has developed platforms of expertise in families of molecular targets which allow high productivity in the generation of innovative compounds potentially addressing a wide range of high unmet medical needs. ACT-1016-0707 – LPA 1 receptor antagonist Immune-mediated and fi
Slide 21: Partner-led portfolio
Partner-led portfolio For Idorsia, sophisticated partnerships are a way of gaining strategic access to technologies or products and fully exploiting our discovery engine and clinical pipeline. We seek suitable external project partners to maximize the value of internal innovation. TRYVIO/JERAYGO (in
Slide 22: Daridorexant (DOD) and ACT-1002-4391
Daridorexant (DOD) Idorsia is supporting a clinical study sponsored by the US Department of Defense (DOD) to develop new therapies for posttraumatic stress disorder (PTSD). The Phase 2 study will evaluate the safety, tolerability, and efficacy of potential therapeutic interventions, including darido
Slide 23: About Idorsia and contact
Idorsia is an independent biopharmaceutical company based on science and innovation. The company is specialized in the discovery, development, and commercialization of innovative small molecules, with the aim of transforming the horizon of therapeutic options. It is headquartered in Allschwil/Basel,