Idorsia (IDIA) investor relations material

Innovation and Mechanism of Action

• TRYVIO (aprocitentan) is the first FDA-approved dual endothelin receptor antagonist for systemic hypertension, offering a novel mechanism for patients with difficult-to-control hypertension after decades of limited progress.

• It targets both ETA and ETB receptors, providing efficacy in upregulated endothelin systems and a strong safety and tolerability profile.

• The endothelin pathway is particularly relevant for high-risk groups, including Black patients, elderly, postmenopausal women, obese individuals, and those with CKD, diabetes, heart failure, or sleep apnea.

• TRYVIO is included in updated ACC/AHA guidelines and is nominated for the 2025 Prix Galien USA Awards.

Clinical Evidence and Patient Populations

• Clinical trials demonstrated rapid, significant, and sustained blood pressure reductions, including in high-risk and resistant hypertension subgroups, with effects seen as early as two weeks and maintained up to 48 weeks.

• TRYVIO showed unique efficacy in reducing nighttime blood pressure and urine albumin-to-creatinine ratio, with stable eGFR in CKD stage 3/4 patients.

• Blood pressure-lowering effects were consistent across age, sex, race, BMI, kidney function, and diabetes status.

• The FDA-approved label covers a broader population than the trial, including adults inadequately controlled on other antihypertensives and highlights cardiovascular risk reduction.

• TRYVIO demonstrated good tolerability, minimal side effects, no drug-drug interactions, and no signals for hyperkalemia or hypotension, supporting its use in patients on multiple therapies.

Market Opportunity and Commercial Strategy

• Nearly 26 million US patients are inadequately controlled on other drugs and eligible for TRYVIO, with 30–50% of treated patients falling into this category.

• An initial focus is on 7 million easily identifiable patients, especially those with CKD, who have limited options and for whom TRYVIO is approved down to EGFR 15.

• Over 1,000 physician interactions confirm strong recognition of TRYVIO's efficacy, safety, and unique mechanism, with favorable payer responses and a WACC set at $775/month.

• Geographic expansion opportunities exist in the EU, UK, Japan, China, and further value is possible through IP extension, fixed-dose combinations, and new indications.

• TRYVIO is approved and commercially available in the US since October 2024, also approved as JERAYGO in the EU and UK, with applications under review in Canada and Switzerland.