From bench to bedside… from compounds to commercialization… no matter how you put it, Idorsia’s ambition is clear: to discover great molecules, build evidence in creative clinical studies, and successfully bring new treatments to patients, creating a sustainable business based on innovation.
More knowledge – Powered by science
Our innovation starts with a brilliant idea and culminates, we hope, in a new drug that can change the treatment paradigm in the target indication.
Idorsia’s drug discovery focuses on families of proteins, characterized by the way they work. We pursue innovative programs involving proteins which have not been targeted up to now, so as to develop drugs with novel mechanisms of action. We are also constantly looking for ways to integrate new technologies and approaches to drug design, such as the use of artificial intelligence (AI) tools.
The drug discovery process starts with an idea from our scientists. We scour the literature to see what others have not yet discovered, to generate ideas and then translate them into a concept which can lead to new treatments for patients.
“We need creativity to be innovative, so we need a brilliant idea and a deep understanding of the disease, to translate it into a molecular mechanism, and to try to find a drug to treat that disease.”
— John Gatfield
Associate Director, Principal Scientist
“It’s very exciting to be able to discover innovative new treatments and really help patients, while at the same time contributing to the growth and value generation of our company."
— Naomi Tidten
Director, Team Leader Computer-Aided Drug Design
More experience – Building the clinical evidence
Following the drug discovery phase, the selected molecule must be comprehensively studied to demonstrate clinical safety and efficacy.
Idorsia aims to deliver new products with the potential to significantly change the treatment options for the target diseases. We want to bring new perspectives to the development of innovative compounds, challenging accepted paradigms to answer the questions that matter most. Our key assets have the potential to transform treatment in the target indications.
When we have decided to target a specific disease, we research to understand the characteristics of the affected patient population (e.g., gender, age, race, concomitant diseases). We design our clinical studies to include participants whose diversity reflects - to the extent possible - that observed in the real world. Idorsia intends to ensure a high level of diversity and inclusion in our clinical trial programs across all therapeutic areas and to follow the measurement of trial diversity using the ICER scoring system. For example, in PRECISION, the Phase 3 study with aprocitentan, we achieved a score of 18 out of 21 points.
Idorsia’s clinical development function comprises a broad spectrum of expertise clustered within multiple departments: therapy area units, strategic development, clinical pharmacology, biostatistics and data management, drug safety, drug regulatory affairs, clinical operations, and life cycle management. Life cycle cross-functional teams – under the direction of a life cycle leader – bring expertise from preclinical development, clinical development, and technical operations to the efficient development of new medicines. They steer the compounds from entry into human studies through to submission of the dossier to health authorities, approval, and maintenance of the license during the commercialization phase up to the loss of the medicine’s exclusivity in the major markets and beyond. Idorsia’s clinical development function manages clinical programs in accordance with the appropriate ethical, scientific, medical, and operational standards, so as to generate the information required by regulatory health authorities worldwide.
“We tailor the target indication to characteristics of the compound. We always try to find the disease, spectrum of diseases, or subset of medical conditions where the molecule will fit best from an efficacy and safety perspective, and where it addresses a medically important need.”
— Sara Mangialaio
Senior Vice President, Head of Clinical Science & Operations
More potential – Navigating regulatory review
With successful clinical studies demonstrating a compound’s safety and efficacy in hand, we must then navigate the regulatory review and approval process.
From the first-in-human study of a drug through market approval and for as long as it remains on the market, we maintain an ongoing dialogue with health authorities in every country where we operate. We ensure that our development plans meet the regulators’ expectations and that we generate the types of data that are required to support registration of the product.
Once our products reach the registration phase, we embark on the regulatory review process.
“For each of the dossiers that we submit to health authorities, we highlight the science-based approach that Idorsia has taken to address patients’ unmet needs. Our robust data tells an amazing story.”
— Brian Schlag
Senior Vice President, Head of Global Drug Regulatory Affairs
More hope – Changing the treatment paradigm
Regulatory approval is a key milestone, but our treatments can only reach patients if our products are successfully launched by our commercial organization – completing the journey from bench to bedside.
Our approach to launch starts long before approval, with the global product strategy – a roadmap to accelerate our affiliates’ efforts to successfully launch our products, while also providing a consistent foundation across the world.
To bring our commercially available products to patients, three functions – Marketing, Medical Affairs, and Value & Access – are responsible for the global product strategy, in close collaboration with key country leaders and our discovery and development teams.
“We ensure that the global product strategy is truly built on scientific evidence and the needs of patients and healthcare professionals.”