Core Viewpoint - Idorsia Ltd is positioned for profitability and growth with two products entering the value-acceleration phase and a promising pipeline of innovative medicines [2][3] Highlights in 2025 - Idorsia achieved strong operational progress, including the global expansion of QUVIVIQ, which is on track to meet sales guidance of approximately CHF 130 million [7] - The company completed debt restructuring and secured new financing, extending its cash runway into 2028 [7] - Clinical validation for its synthetic glycan vaccine technology and initiation of a proof-of-concept trial with a CCR6 antagonist were also notable achievements [7] Areas of Focus in 2026 - The company aims to make QUVIVIQ the standard of care for insomnia and maximize the value of TRYVIO/JERAYGO for uncontrolled hypertension [3][7] - Plans include advancing lucerastat for Fabry Disease registration and progressing the Phase 2 proof-of-concept immunology portfolio [7] - Idorsia will continue to explore partnerships and new sales models for QUVIVIQ, as well as support potential descheduling in the US [7]

Core Insights - Idorsia Ltd has published results from the pivotal Phase 3 MODIFY study and its open-label extension, highlighting the potential of lucerastat as an oral substrate reduction therapy for Fabry disease, particularly in patients with renal impairment [1][3]. Study Overview - The MODIFY study was a multicenter, double-blind, randomized, placebo-controlled trial involving 118 patients, aimed at assessing the efficacy and safety of lucerastat as an oral monotherapy for adult patients with Fabry disease [2]. - The study's primary endpoint of reducing neuropathic pain over six months was not met; however, lucerastat showed significant reductions in plasma and urinary Gb3 levels compared to placebo, with sustained effects observed in the open-label extension [3][17]. Renal Function Insights - An interim analysis of the open-label extension indicated a notable improvement in renal function trajectory, with a reduced rate of eGFR decline in patients treated with lucerastat compared to their eGFR slope prior to enrollment [4][9]. - In patients with impaired renal function or fast-deteriorating eGFR at baseline, lucerastat was associated with a significant reduction in kidney function loss, suggesting a potential disease-modifying effect [4][17]. Long-term Treatment and Tolerability - The open-label extension has collected data from patients treated with lucerastat for over 42 months, with some receiving treatment for more than 6 years, demonstrating good tolerability and no serious treatment-related adverse events [5][8]. - A kidney biopsy sub-study was conducted to evaluate Gb3 inclusions in kidney cells of male participants with classic Fabry disease treated for over 3 years with lucerastat [5]. Future Directions - The company is collaborating with the US FDA to design a new Phase 3 program to ensure a regulatory pathway for lucerastat's approval [6][10]. - A post-trial access program is being established to ensure continuity of care for participants still receiving lucerastat at the study's closure [7].

Core Viewpoint - Idorsia Ltd has initiated a Phase 2 proof-of-concept trial for IDOR-1117-2520, an oral selective CCR6 receptor antagonist aimed at treating moderate-to-severe psoriasis, with potential implications for other autoimmune conditions associated with CCR6 and Th17 pathways [2][10]. Company Overview - Idorsia is focused on developing transformative medicines and aims to evolve into a leading biopharmaceutical company with a strong scientific foundation [13]. - The company is headquartered near Basel, Switzerland, and has a dedicated team covering all aspects of drug development [14]. Product Details - IDOR-1117-2520 is a first-in-class, oral small-molecule drug designed to block the CCL20-driven recruitment of pathogenic CCR6-expressing immune cells, showing efficacy comparable to existing IL-17 and IL-23 inhibitors in preclinical models [9]. - The drug is currently in a Phase 2 trial, with results expected in the first quarter of 2027 [6]. Clinical Trial Information - The Phase 2 trial is a multicenter, double-blind, randomized, placebo-controlled study involving approximately 30 participants, evaluating the efficacy and safety of two dosages of IDOR-1117-2520 over 12 weeks [6]. - Primary efficacy will be measured by the change in Psoriasis Area and Severity Index (PASI) score from baseline to Week 12 [6]. Mechanism of Action - The CCR6 antagonism targets a key receptor that directs pathogenic immune cells to inflamed tissues, potentially modulating inflammation without broad immunosuppression [8]. - The Th17 pathway, which is implicated in various autoimmune diseases, is a primary focus for IDOR-1117-2520, aligning with Idorsia's strategic goals [4][11].

Core Viewpoint - Idorsia Ltd has received the inaugural Prix Galien Bridges Award for its innovative treatment for insomnia disorder, highlighting the company's commitment to advancing patient care through scientific innovation [1][8]. Company Overview - Idorsia is focused on developing transformative medicines and aims to evolve into a leading biopharmaceutical company with a strong scientific foundation [13][14]. - The company is headquartered near Basel, Switzerland, and is listed on the SIX Swiss Exchange under the ticker symbol IDIA [14]. Product Details - The awarded treatment is a dual orexin receptor antagonist, recognized as the first and only drug of its kind approved in Europe for insomnia disorder [8][11]. - The development process involved synthesizing and characterizing over 25,000 compounds over a span of more than 10 years to achieve a drug that effectively improves both nighttime sleep and daytime functioning [3][11]. Recognition and Awards - The Prix Galien Awards, established in 1970, are considered the equivalent of the Nobel Prize for the life sciences industry, recognizing outstanding innovation and scientific advancement [3][5]. - The award received by Idorsia is particularly significant as it is the first of its kind in the Nordics, underscoring the company's innovative contributions to healthcare [2]. Insomnia Disorder Context - Insomnia disorder affects approximately 9.2% of the working-age population in Switzerland, characterized by difficulty in initiating or maintaining sleep, leading to significant daytime impairment [7][6]. - The orexin system plays a crucial role in regulating wakefulness, and its antagonism is key to restoring natural sleep architecture in patients with insomnia [10][11].

Idorsia’s dual orexin receptor antagonist, the first and only drug of its kind approved in Europe for the treatment of insomnia disorder, has been awarded the inaugural Prix Galien Bridges Award in the category of “Best Biotechnology & Pharmaceutical Product”Inaugural winners are honored for breakthrough innovations in life sciences Allschwil, Switzerland – December 9, 2025Idorsia Ltd (SIX: IDIA) announces that its novel treatment for insomnia disorder has been awarded the inaugural Prix Galien Bridges Awar ...

Core Insights - New analysis confirms that aprocitentan significantly reduces blood pressure and albuminuria in patients with true resistant hypertension and high cardiovascular risk, including those with chronic kidney disease [1][3] Company Overview - Idorsia Ltd is set to present new findings from the Phase 3 PRECISION study of aprocitentan at the American Society of Nephrology Kidney Week 2025 in Houston, TX, from November 5 to 9, 2025 [2] - Aprocitentan is a once-daily, orally active dual endothelin receptor antagonist, approved as TRYVIO in the US for systemic hypertension treatment in combination with other antihypertensives since October 2024 [5] - The drug is also approved as JERAYGO for resistant hypertension treatment in the European Union, UK, and Switzerland, with a marketing authorization application under review in Canada [5] Clinical Findings - A poster presentation titled "Is Aprocitentan's Effect on Albuminuria Merely Due to Lowered BP? Subgroup Analysis of the PRECISION Trial" will take place on November 6, highlighting significant reductions in systolic blood pressure and Urine Albumin-to-Creatinine Ratio (UACR) compared to placebo across all subgroups [3] - The analysis indicates that the reductions in UACR are not solely due to lowered blood pressure, suggesting additional renal-protective mechanisms of aprocitentan, such as decreased glomerular permeability and functional tubular changes [3] Upcoming Events - Idorsia will also participate in the American Heart Association Scientific Sessions 2025 in New Orleans, LA, from November 7 to 10, with a symposium discussing the next era in hypertension treatment [4]

Core Insights - Idorsia Ltd has received marketing authorization from Swissmedic for JERAYGO (aprocitentan) to treat resistant hypertension in adults, marking it as the first and only endothelin receptor antagonist for this condition [1][8] - Resistant hypertension affects approximately 10% of hypertensive patients, highlighting the need for more effective treatment options [3] - JERAYGO has shown significant blood pressure reduction in diverse patient populations, including those with obesity, chronic kidney disease, or type 2 diabetes [4] Company Overview - Idorsia is focused on developing transformative medicines and has a strong scientific foundation, with JERAYGO representing their second product approval in Switzerland [4][15] - The company is in discussions with potential partners to expand the availability of JERAYGO across Switzerland and Europe [4] Product Details - JERAYGO is administered orally at a recommended starting dose of 12.5 mg once daily, which can be increased to 25 mg for patients needing tighter blood pressure control [1] - Aprocitentan, the active ingredient in JERAYGO, is a dual endothelin receptor antagonist that has been commercially available in the US as TRYVIO™ since October 2024 [12] Clinical Study Findings - The Phase 3 PRECISION study demonstrated that both 12.5 mg and 25 mg doses of aprocitentan significantly reduced sitting systolic blood pressure compared to placebo [10] - The treatment effect was consistent across various demographics, including age, sex, and race, and was evident within the first two weeks of treatment [10] Patient Demographics - The study included 730 patients with a mean age of 61.7 years, with a significant portion having comorbidities such as type 2 diabetes (54.1%) and chronic kidney disease (22.2%) [7][9] Safety Profile - Common adverse reactions to aprocitentan included edema/fluid retention and decreased hemoglobin levels, with specific contraindications for pregnant women and those with severe hepatic impairment [11]

Core Viewpoint - Idorsia Ltd, in collaboration with Stanford Hypertension Center and Duke Heart Center, has launched the IMPACT-HTN program aimed at modernizing the management of difficult-to-control hypertension through a multi-phase initiative that incorporates AI technology and real-world evidence [2][3][4]. Group 1: Program Overview - The IMPACT-HTN program consists of three phases designed to standardize treatment protocols and improve patient outcomes for those with difficult-to-control hypertension [2]. - The initiative will generate real-world evidence and develop scalable AI-powered tools to assist in identifying patients who may benefit from innovative therapies, including Idorsia's TRYVIO™ [2][5]. Group 2: Collaboration and Goals - The program is led by Dr. Vivek Bhalla from Stanford and Dr. Sreekanth Vemulapalli from Duke, emphasizing a cross-institutional collaboration to enhance care coordination and access to new therapies [3][4]. - The goal is to standardize evaluation and management practices for hypertension, ultimately improving patient outcomes and addressing the global health issue of hypertension, which affects approximately 50% of adults in the U.S. [3][4]. Group 3: Tools and Innovations - The program will introduce a digital care algorithm to standardize assessment and management of difficult-to-control hypertension, utilizing interactive tools such as AI chatbots and patient-facing algorithms [6]. - A personalized hypertension risk score will be developed to better identify patients at risk for negative cardiovascular outcomes, allowing for timely intervention [6]. Group 4: TRYVIO™ Overview - TRYVIO, a dual endothelin receptor antagonist, is now available for prescription and is indicated for patients whose hypertension is not adequately controlled with other medications [7][18]. - It has been included in the new clinical practice guidelines from the American College of Cardiology and the American Heart Association, highlighting its significance in the treatment landscape [7].

Core Insights - Idorsia Ltd is actively participating in World Sleep 2025, showcasing its commitment to advancing sleep science and insomnia treatment through various presentations and symposiums [1][6]. Company Activities - Idorsia will present eight scientific posters at World Sleep 2025, focusing on the abuse potential of insomnia therapies and the efficacy of daridorexant [1][2][3]. - The company will host an educational symposium on September 9, featuring experts discussing the relationship between insomnia and comorbid neurological or psychiatric conditions, along with new data on daridorexant [5]. Research Highlights - A significant study will analyze real-world data on the abuse potential of insomnia medications, utilizing the FDA Adverse Event Reporting System [1]. - The largest clinical characterization of insomnia patients to date will be presented, based on data from over 5,000 patients, providing insights into the disorder's impact on symptoms and functioning [4]. Industry Context - World Sleep 2025 serves as a global platform for sleep professionals to discuss advancements in sleep science and treatment, fostering collaboration among various stakeholders in the field [6][8].

Core Viewpoint - Idorsia Ltd has successfully completed a repurchase offer for its convertible bonds, significantly reducing its outstanding debt and restructuring its financial obligations [1][4]. Group 1: Repurchase Offer Details - The repurchase offer for the CHF 204 million convertible bonds maturing in 2025 (CB 2025) saw bondholders accept an aggregate nominal value of CHF 187,476,000, which is 91.90% of the total issued nominal value [2]. - For the CHF 600 million convertible bonds maturing in 2028 (CB 2028), bondholders accepted an aggregate nominal value of CHF 567,200,000, corresponding to 94.53% of the total issued nominal value [2]. - The settlement involved the issuance of A1 Notes (CHF 120,037,805), A2 Notes (CHF 254,962,195), and B Notes (CHF 379,676,000), along with 8,040,000 Exchange Shares and 8,040,000 Exchange Warrants [3]. Group 2: Financial Restructuring - The completion of the repurchase offer is part of a broader restructuring plan agreed upon with a majority of bondholders, initially announced on February 26, 2025 [4]. - A total nominal value of CHF 761,779,000 in A1, A2, and B Notes has been issued by Idorsia Investments SARL as part of this restructuring [4]. - The company plans to establish an equity line for up to 20,000,000 shares, set to expire at the end of February 2026, to extend its cash runway into 2027 [5]. Group 3: Company Overview - Idorsia aims to challenge established medical paradigms by discovering, developing, and commercializing transformative medicines, positioning itself as a leading biopharmaceutical company [6]. - The company is headquartered near Basel, Switzerland, and has a strong focus on small-molecule drugs, with a promising in-house development pipeline [7]. - Idorsia is listed on the SIX Swiss Exchange under the ticker symbol IDIA [8].