IDORSIA LTD --------------------------- ------------ ------------ ------------ Long-term deposits 196 192 180 --------------------------- ------------ ------------ ------------ Total liquidity* 1,065 1,200 632 --------------------------- ------------ ------------ ------------ Indebtedness -------------------------- ------------ ------------ ------------ Convertible loan 390 388 382 --------------------------- ------------ ------------ ------------ Convertible bond 199 199 199 --------------------------- ------------ ------------ ------------ Other financial debt - - - -------------------------- ------------ ------------ ------------ Total indebtedness 589 587 581 --------------------------- ------------ ------------ ------------ *rounding differences may occur Clinical Development Idorsia has a diversified and balanced clinical development pipeline covering multiple therapeutic areas, including CNS, cardiovascular and immunological disorders, as well as orphan diseases. In April and July of 2020, Idorsia reported positive results in each of the two pivotal Phase 3 studies of daridorexant in patients with insomnia. The Phase 3 registration program demonstrated efficacy of daridorexant on objective and subjective sleep parameters, and an improvement in daytime functioning, while maintaining a favorable safety profile. More details and commentary can be found in the dedicated press releases (first study release https://www.idorsia.com/media/news-details?newsId=2284972 ), (second study release https://www.idorsia.com/media/news-details?newsId=2329316 ) and the investor webcasts (first study webcast https://www.idorsia.com/investors/news-and-events/webcast-daridorexant ), (second study webcast https://www.idorsia.com/investors/news-and-events/webcast-daridorexant-302-results ) which are available for replay on Idorsia's corporate website. This week, the final results of the 40-week extension study with daridorexant became available. The study collected information on the safety of long-term treatment as well as allowing an exploratory analysis of the maintenance of efficacy. There were no new emerging safety findings. Moreover, the efficacy on sleep and daytime functioning appeared to be maintained over the longer treatment duration. The Phase 3 program is therefore concluded. A New Drug Application (NDA) was submitted to the US FDA on January 8, 2021 and a Marketing Authorization Application (MAA) to the European Union EMA on March 2, 2021 and to Switzerland health authority, Swissmedic, on April 20, 2021. Should approval be received, the company anticipates launch in the US in the first half of 2022, followed by other regions thereafter. Guy Braunstein, MD and Head of Global Clinical Development of Idorsia, commented: "I'm pleased to report that the Phase 3 development program with daridorexant has been successfully concluded. The final analyses of the 40-week extension study with daridorexant are aligned to the interim results submitted to the health authorities. The totality of the data supports the chronic use of daridorexant in insomnia." In November of 2020, Idorsia reported positive results in each of the two Japanese registration studies of clazosentan assessing the efficacy and safety of clazosentan in reducing vasospasm-related morbidity and all-cause mortality in patients following aneurysmal subarachnoid hemorrhage. More details can be found in the dedicated press release https://www.idorsia.com/media/news-details?newsId=2410833 . A New Drug Application (NDA) to the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) for clazosentan was submitted on March 1, 2021. Recruitment into the global REACT study of clazosentan has been impacted by the coronavirus pandemic but is steadily progressing. The MODIFY Phase 3 study of lucerastat in Fabry disease was fully recruited in February 2021, with 118 patients in the Phase 3 study. Results are expected in the fourth quarter of this year, should all patients continue into the open label extension study. Full recruitment has also just been achieved for PRECISION, a Phase 3 study to demonstrate the antihypertensive effect of aprocitentan when added to standard of care in patients with resistant hypertension, with 730 patients randomized. This 12-month study should deliver results in mid-2022. The CARE study, a large Phase 2b multiple-dose, efficacy and safety study with cenerimod, for the treatment of systemic lupus erythematosus completed randomization at the end of February 2021, with 427 patients enrolled. The results are targeted for the fourth quarter of 2021. A Phase 2 proof-of-concept study with ACT-539313, a selective orexin 1 receptor antagonist, in binge eating disorder has begun recruitment. Binge eating disorder is the most common eating disorder characterized by repeated episodes of eating unusually large portions of food in a short period of time (within any 2-hour period). It is associated with a sense of lack of control over what is being eaten and there is often an absence of pleasure in what has been eaten. Preclinical studies have shown that orexins play an important role in driving compulsive binge-like consumption and that orexin receptor antagonists have reduced binge-like eating behavior in animal models. This is the first study of orexin 1 receptor antagonism as a new mechanism of action for patients with binge eating disorder. Idorsia's clinical development pipeline Mechanism of Target Indication Status Compound Action ------------- ---------------- ---------------------------------------- -------------------- Daridorexant Dual orexin Insomnia Under review with receptor FDA/EMA antagonist ------------- ---------------- ---------------------------------------- -------------------- Aprocitentan* Dual endothelin Resistant hypertension management Phase 3 recruitment receptor complete antagonist ------------- ---------------- ---------------------------------------- -------------------- Clazosentan Endothelin Cerebral vasospasm assoc. Japan: NDA submitted receptor with aneurysmal subarachnoid hemorrhage Global: Phase 3 antagonist ------------- ---------------- ---------------------------------------- -------------------- Lucerastat Glucosylceramide Fabry disease Phase 3 recruitment synthase complete inhibitor ------------- ---------------- ---------------------------------------- -------------------- Selatogrel P2Y(12) receptor Suspected acute myocardial infarction Phase 3 in antagonist preparation ------------- ---------------- ---------------------------------------- -------------------- Cenerimod S1P(1) receptor Systemic lupus erythematosus Phase 2b recruitment modulator complete ------------- ---------------- ---------------------------------------- -------------------- ACT-539313 Selective orexin Binge eating disorder Phase 2 1 receptor antagonist ------------- ---------------- ---------------------------------------- -------------------- Sinbaglustat GBA2/GCS Rare lysosomal storage disorders Phase 1 complete inhibitor ------------- ---------------- ---------------------------------------- -------------------- ACT-1004-1239 CXCR7 antagonist Immunology Phase 1 ------------- ---------------- ---------------------------------------- -------------------- ACT-1014-6470 - Immunology Phase 1 ------------- ---------------- ---------------------------------------- -------------------- ACT-541478 - CNS Phase 1 ------------- ---------------- ---------------------------------------- -------------------- ACT-777991 - Immunology Phase 1 ------------- ---------------- ---------------------------------------- -------------------- * In collaboration with Janssen Biotech to jointly develop aprocitentan, Janssen Biotech has sole commercialization rights worldwide Neurocrine Biosciences has a global license to develop and commercialize ACT-709478, a novel T-type calcium channel blocker. In November 2020, Neurocrine announced it had initiated a Phase 2 study investigating ACT-709478 for the treatment of a rare form of pediatric epilepsy. Further details including the current status of each project in the pipeline can be found in our clinical development fact sheet: https://www.idorsia.com/documents/com/fact-sheets-presentations/fs-clinical-development.pdf . Collaboration update In 2017, Idorsia entered into a research collaboration with Roche in the field of cancer immunotherapy. In the first quarter of 2021, the agreement was terminated. Idorsia continues drug discovery efforts on this promising molecular target. About the Revenue Sharing Agreement for ponesimod Idorsia and Actelion Pharmaceuticals Ltd, one of the Janssen Pharmaceutical Companies of Johnson & Johnson, have entered into a
(MORE TO FOLLOW) Dow Jones Newswires
April 22, 2021 01:00 ET (05:00 GMT)
