Company Announcements

Allschwil, Switzerland – October 16, 2024

Idorsia Ltd (SIX: IDIA) today announced that new data on aprocitentan, Idorsia’s endothelin receptor antagonist, will be presented at the American Society of Nephrology (ASN) Kidney Week 2024, taking place in San Diego, CA, October 23–27, 2024.

The following oral and poster presentations will be held, highlighting the promising results of using aprocitentan for patients with hypertension not adequately controlled with other medications, with positive effects on blood pressure and proteinuria measures, in patients with co-morbid chronic kidney disease (CKD) stage 3 and 4, and its favorable tolerability profile including the absence of aggravation of renal function and hyperkalemia.

• Bakris G. L., et al. “Aprocitentan in patients with chronic kidney disease (CKD): sub-group analysis of the PRECISION trial”, oral presentation by Markus Schlaich, abstract #: FR-OR48, session ‘Hypertension, CVD, and the Kidneys: Clinical Studies [OR1602], room 5 (Convention Center), Oct 25, 16:50 – 17:00.
• Bakris G. L., et al. “Incidence and prevalence of edema and the effect of aprocitentan treatment strategy in the PRECISION study”, poster presentation by Markus Schlaich, poster number SA-PO356, Exhibit Halls A-C, Oct 26, 10:00 – 12:00.

Idorsia US will also be present at ASN Kidney Week with a TRYVIO (aprocitentan) commercial and medical information booth. Visit the team at booth #1705.

In addition, data from the Phase 3 program with lucerastat, Idorsia’s glucosylceramide synthase inhibitor, will be presented as a poster:

• Wallace E. L., et al. “Lucerastat effect on kidney function in patients with Fabry Disease: Results from the Phase 3 clinical program”, poster presentation by Eric Wallace, poster number SA-PO654, Exhibit Halls A-C, Oct 26, 10:00 – 12:00.
  
  

The abstracts will be published in the Journal of American Society of Nephrology and can be found in the ASN Kidney Week abstract supplement.

Notes to the editor

About aprocitentan
Aprocitentan is Idorsia’s once-daily, orally active, dual endothelin receptor antagonist, which inhibits the binding of ET-1 to ET_A and ET_B receptors. In May 2022, Idorsia announced positive top-line results of the Phase 3 PRECISION study with aprocitentan for the treatment of patients with resistant hypertension. Detailed results were published in The Lancet and presented as a Late-Breaking Science presentation during the American Heart Association (AHA) Scientific Sessions in November 2022. More details and commentary can be found in the dedicated press release and an investor webcast featuring Prof. Markus Schlaich, an investigator in PRECISION. On March 19, 2024, aprocitentan was approved as TRYVIO™ in the US. On June 27, 2024, the European Commission granted market authorization for JERAYGO™ (aprocitentan).

About lucerastat
Lucerastat is Idorsia’s 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. In October 2021, the company reported that lucerastat 1000 mg b.i.d. did not meet the primary endpoint of reducing neuropathic pain during 6 months of treatment versus placebo. However, Lucerastat demonstrated a substantial reduction in levels of the Fabry disease biomarker plasma Gb3 during the treatment period, with a decrease of approximately 50% observed in plasma Gb3 in the lucerastat treatment group compared to an increase of 12% in the placebo group. Furthermore, results suggested a treatment effect on kidney function. Lucerastat was well tolerated. Analysis of the ongoing open-label extension (OLE) of the Phase 3 study corroborated the long-term effect on plasma Gb3 levels and a potential positive long-term effect on kidney function. The analysis also showed a safety and tolerability profile consistent with that observed during the 6-month randomized treatment period. The company is conducting a kidney biopsy substudy within a subset of patients currently participating in the OLE study in order to steer further development in Fabry disease. In parallel, Idorsia is working with regulatory authorities to design the next Phase 3 study to evaluate the effect of lucerastat on renal function.

About Idorsia
Idorsia Ltd is reaching out for more – We have more ideas, we see more opportunities and we want to help more patients. In order to achieve this, we will develop Idorsia into a leading biopharmaceutical company, with a strong scientific core.

Headquartered near Basel, Switzerland – a European biotech-hub – Idorsia is specialized in the discovery, development and commercialization of small molecules to transform the horizon of therapeutic options. Idorsia has a 25-year heritage of drug discovery, a broad portfolio of innovative drugs in the pipeline, an experienced team of professionals covering all disciplines from bench to bedside, and commercial operations in Europe and North America – the ideal constellation for bringing innovative medicines to patients.

Idorsia was listed on the SIX Swiss Exchange (ticker symbol: IDIA) in June 2017 and has over 750 highly qualified specialists dedicated to realizing our ambitious targets.

For further information, please contact
Andrew C. Weiss
Senior Vice President, Head of Investor Relations & Corporate Communications
Idorsia Pharmaceuticals Ltd, Hegenheimermattweg 91, CH-4123 Allschwil
+41 58 844 10 10
investor.relations@idorsia.com
media.relations@idorsia.com
www.idorsia.com

The above 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", "pending" or "anticipates" or similar expressions, or by discussions of strategy, plans or intentions. Such statements include descriptions of the company's investment and research and development programs and anticipated expenditures in connection therewith, descriptions of new products expected to be introduced by the company and anticipated customer demand for such products and products in the company's existing portfolio. Such statements reflect the current views of the company with respect to future events and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements of the company to be materially different from any future results, performances or achievements that may be expressed or implied by such forward-looking statements. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described herein as anticipated, believed, estimated or expected.

Attachment

• Press Release PDF