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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
More knowledge –
Powered by science
“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
Hover over each phase each phase in the drug discovery and development lifecycle to learn more
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.
Hover over the steps involved in US 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.
More potential – Navigating regulatory review
“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
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.”
— Antonio Olivieri
Senior Vice President, Chief Medical Officer & Head of Global Medical Affairs
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.
More hope – Changing the treatment paradigm
Hover over the steps involved in the European Medicines Agency review process
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.
Years -5 to 0
Drug discovery phase
Year 0
Patenting the molecule
Preclinical studies
Drug development
Years 0 to 5
Clinical studies
Years 5 to 10
Regulatory submission
Years 10 to 12
Product launch
From year 12
A discovery program aims at discovering molecules which need to be progressively optimized for activity against a biological target and for desired physicochemical, pharmacokinetic, and other properties. Their pharmacological activity and their safety need to confirm their potential in pathological situations.
Drug discovery phase
Patenting the molecule
Patents are filed for the most promising compounds. This protection provides 20 years of exclusive commercial use – the clock starts!
Preclinical studies
The effects and toxicity of drugs are assessed in silico (with computer programs), in vitro (in test tubes), and in vivo (in animals).
Drug development
The processes to manufacture research-grade molecules are transformed through chemical and pharmaceutical development to produce pharmaceutical-grade drugs, compliant with health authority guidelines, for administration to patients.
Phase 1
assessment of the safety and efficacy of the compound in a limited number of patients, with the aim of finding the optimal dose for large-scale studies.
Phase 2
Phase 3
assessment of the safety and efficacy of a future medicine, most often compared to placebo, in a large group of patients.
assessment of tolerability or side effects in a small group of healthy volunteers.
Efficacy and safety of future drugs are assessed in humans.
Before a drug can be placed on the market, it must first be approved by local regulatory authorities. A comprehensive dossier is submitted for review and approval.
Regulatory submission
Product launch
Once approved by the local health authorities, a drug enters the market and physicians may prescribe it to patients. Patient access to a drug is often determined by the drug reimbursement system and payor decisions about the treatment.
Note: The timeline can be influenced by many factors, such as the indication for which a drug is being studied.
Drug discovery and development lifecycle
Primary review
FDA sends questions to Idorsia on ongoing basis
Month
Day 0 Submit New Drug Application (NDA)
Day 60 FDA filing decision
By day 74 FDA issues application filing confirmation decision
Month 5 Mid-cycle meeting
Month 9 Late cycle meeting
Month 12 PDUFA date, or deadline for the FDA to review New Drug Application
In some cases: 90 days after PDUFA date
US Drug Enforcement Administration (DEA) publishes scheduling.
US regulatory standard review – FDA
Teams from across Idorsia collaborate to develop a robust and comprehensive dossier for submission to the health authorities. The data included is wide-reaching: preclinical research, such as compound screening, animal models and all pharmacology, pharmacokinetics and toxicology data; technical descriptions of the properties and chemical synthesis of the drug substance, as well as quality controls and procedures for pharmaceutical manufacturing; and complete results and analysis of each of the clinical studies and safety data collected over the course of clinical phases – in other words, the story of each molecule from bench to bedside.
Scheduling depends upon the drug's acceptable medical use and the drug's abuse or dependency potential.
Day 0 Submission of Marketing Authorisation Application (MAA)
Day
Day 14 Administrative
validation of dossier
Day 19 Start date
Day 120
List of questions to Idorsia
Day 121
Clock starts when Idorsia responds to question list
“Clock stop” ~3 months to respond to questions
Day 180
List of outstanding questions to Idorsia
Day 181
Clock starts
when Idorsia responds
“Clock stop”
Day 210
Committee for Medicinal Products for Human Use (CHMP) opinion
Day 277 European Commission decision granting Marketing Authorisation
Teams from across Idorsia collaborate to develop a robust and comprehensive dossier for submission to the health authorities. The data included is wide-reaching: preclinical research, such as compound screening, animal models and all pharmacology, pharmacokinetics and toxicology data; technical descriptions of the properties and chemical synthesis of the drug substance, as well as quality controls and procedures for pharmaceutical manufacturing; and complete results and analysis of each of the clinical studies and safety data collected over the course of clinical phases – in other words, the story of each molecule from bench to bedside.
EU regulatory standard review – EMA
Key countries
Discovery and Development Teams
Value & Access
Medical Affairs
Marketing
Global Product Strategy
Value & Access is responsible for demonstrating the value of our products – which is more important than ever, given increasing budgetary constraints in healthcare systems across the world. As an engaged member of the healthcare ecosystem, Idorsia understands its responsibility to help find solutions to the high cost of healthcare, and we are committed to playing our part in supporting patient access to our medicines. The prices of our medicines will reflect the value that our innovations deliver, generating revenues to fuel the discovery and development of future molecules. To demonstrate meaningful innovation, we develop a value proposition, underpinned by our science and clinical data, to help payors determine the value offered by our treatments compared to existing options. Our ultimate goal is to help patients gain access to our treatments through reimbursement or other coverage arrangements.
Value & Access
Marketing
Global Marketing generates deep insights from patients and healthcare professionals, which help us to gain a holistic understanding of our customers’ needs. This helps us to address unmet needs in the marketplace and to clearly differentiate our brands. We also focus our marketing efforts on raising awareness among patients, healthcare professionals, and other key stakeholders (e.g. policymakers) of the impact of the conditions targeted by our products.
Idorsia’s Global Medical Affairs team is responsible for communicating to the healthcare community our science, the data on our products, and the key differences from other treatments. To inform and develop our global strategy, we also seek medical insights regarding how our products’ core data resonates with physicians. Our medical and clinical development teams continue to generate new evidence for approved products – with real world evidence in high demand among payors and physicians alike. Importantly, this team also manages Idorsia’s repository of medical information and has developed an intelligent digital platform, providing 24/7 self-service access to scientifically robust, balanced, and easily digestible information.
Medical Affairs
While our product strategies are global, our country teams own the execution of their local launches and customer relationships, and they tailor the global strategies to their markets. Working closely together, our affiliates and global teams all play a role in ensuring a successful launch and thus maximizing the value of Idorsia’s innovation.
Key
countries
Discovery and Development Teams
The commercial organization teams up with people from across Idorsia to gather insights from the discovery and development phases and ensure that science is at the core of our global product strategies. For example, our development team members share learnings about the needs of patients and healthcare professionals from our clinical trials, and also partner with our medical affairs group to shape how we continue to gather data about our treatments.
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Drug discovery phase
At first, we seek to enhance the potency of the compound’s effects on the target protein, but as we advance we look at other activities, which may cause side effects. The aim is to ensure that the compound’s overall properties allow it to become a drug.
For example, our electrophysiologists screen drugs for side effects by monitoring electrical activity in the heart or brain. Here, electrical communication depends on ion channels in the cell membrane; if a drug blocks some of these ion channels, it can have serious adverse effects.
A discovery program aims at discovering molecules which need to be progressively optimized for activity against a biological target and for desired physicochemical, pharmacokinetic and other properties. Their pharmacological activity and their safety need to confirm their potential in pathological situations.
Patenting the molecule
Patents are filed for the most promising compounds. This protection provides 20 years of exclusive commercial use – the clock starts!
Drug development
A discovery program aims at discovering molecules which need to be progressively optimized for activity against a biological target and for desired physicochemical, pharmacokinetic and other properties. Their pharmacological activity and their safety need to confirm their potential in pathological situations.
Preclinical studies
The effects and toxicity of drugs are assessed in silico (with computer programs), in vitro (in cell cultures), and in vivo (in animals).
Clinical studies
Phase 1
assessment of the safety and efficacy of the compound in a limited number of patients, with the aim of finding the optimal dose for large-scale studies.
Phase 2
Phase 3
assessment of the safety and efficacy of a future medicine, most often compared to placebo, in a large group of patients.
assessment of tolerability or side effects in a small group of healthy volunteers.
The effects and toxicity of drugs are assessed in silico (with computer programs), in vitro (in cell cultures), and in vivo (in animals).
Regulatory submission
Before a drug can be placed on the market, it must first be approved by local regulatory authorities.
A comprehensive dossier is submitted for review
and approval.
Product launch
Month 9 Late cycle meeting
Month 12 PDUFA date, or deadline for the FDA to review New Drug Application
By day 74 FDA issues application filing confirmation decision
Month 5
Mid-cycle meeting
Primary review
FDA sends questions to Idorsia on ongoing basis
Day 60 FDA filing decision
Month
In some cases: 90 days after PDUFA date US Drug Enforcement Administration (DEA) publishes scheduling.
Day 0 Submit New Drug Application (NDA)
Day 0
Teams from across Idorsia collaborate to develop a robust and comprehensive dossier for submission to the health authorities. The data included is wide-reaching: preclinical research, such as compound screening, animal models and all pharmacology, pharmacokinetics and toxicology data; technical descriptions of the properties and chemical synthesis of the drug substance, as well as quality controls and procedures for pharmaceutical manufacturing; and complete results and analysis of each of the clinical studies and safety data collected over the course of clinical phases – in other words, the story of each molecule from bench to bedside.
Day 90
Scheduling depends upon the drug's acceptable medical use and the drug's abuse or dependency potential.
Day
Day 14 Administrative
validation of dossier
Day 19 Start date
Day 120
List of questions to Idorsia
“Clock stop” ~3 months to respond to questions
Day 121
Clock starts when Idorsia responds to question list
Day 180
List of outstanding questions to Idorsia
“Clock stop”
Day 181
Clock starts when Idorsia responds
Day 210
CHMP opinion
Day 277 European Commission decision granting Marketing Authorisation
Day 0 Submission of Marketing Authorization Application (MAA)
Day 0
Teams from across Idorsia collaborate to develop a robust and comprehensive dossier for submission to the health authorities. The data included is wide-reaching: preclinical research, such as compound screening, animal models and all pharmacology, pharmacokinetics and toxicology data; technical descriptions of the properties and chemical synthesis of the drug substance, as well as quality controls and procedures for pharmaceutical manufacturing; and complete results and analysis of each of the clinical studies and safety data collected over the course of clinical phases – in other words, the story of each molecule from bench to bedside.
Once approved by the local health authorities, a drug enters the market and physicians may prescribe it to patients. Patient access to a drug is often determined by the drug reimbursement system and payor decisions about the treatment.
Value & Access
Value & Access is responsible for demonstrating the value of our products – which is more important than ever, given increasing budgetary constraints in healthcare systems across the world. As an engaged member of the healthcare ecosystem, Idorsia understands its role to help find solutions to the high cost of healthcare and is committed to doing our part in supporting patient access to our medicines. The prices of our medicines will reflect the value that our innovations deliver, which generates revenues to fuel the discovery and development of future molecules. To demonstrate meaningful innovation, we develop a value proposition, underpinned by our science and clinical data, to help payors determine the value offered by our treatments compared to existing options. Our ultimate goal is to help patients gain access to our treatments through reimbursement or other coverage arrangements.
Marketing
Global Marketing generates deep insights from patients and healthcare professionals, which help us to gain a holistic understanding of our customers’ needs. This helps us to address unmet needs in the marketplace and to clearly differentiate our brands. We also focus our marketing efforts on raising awareness among patients, healthcare professionals and other key stakeholders (e.g. policymakers) of the impact of the conditions targeted by our products.
Medical Affairs
Idorsia’s Global Medical Affairs team is responsible for communicating to the healthcare community our science, the data on our products, and the key differences from other treatments. To inform and develop our global strategy, we also seek medical insights regarding how our products’ core data resonates with physicians. Our medical and clinical development teams continue to generate new evidence for approved products – with real-world evidence in high demand among payors and physicians alike. Importantly, this team also manages Idorsia’s repository of medical information and has developed an intelligent digital platform, providing 24/7 self-service access to scientifically robust, balanced, and easily digestible information.
Key countries
While our product strategies are global, our country teams own the execution of their local launches and customer relationships, and they tailor the global strategies to their markets. Working closely together, our affiliates and global teams all play a role in ensuring a successful launch and thus maximizing the value of Idorsia’s innovation.
Discovery and Development Teams
Targets
Hits
Lead Structure
Improved Leads
Compound Library
Preclinical Development
Drug Development
Clinical Development
Registration
Launch
Molecular Biology
(Target Finding)
Molecular Biology
(Target Finding)
Biochemistry
(High Throughput Screening)
Biochemistry
(High Throughput Screening)
Structural Biology
and Molecular Modeling
Structural Biology
and Molecular Modeling
Research
Information
Management
Research
Information
Management
Medicinal
Chemistry
Medicinal
Chemistry
Pharmacokinetics & Metabolism
Pharmacokinetics & Metabolism
Chemistry
Process R&D
Chemistry
Process R&D
Pharmacology
Pharmacology
Molecular Biology
(Target Finding)
Our work in the lab begins with the target. This may be a particular protein which, when its activity is modulated, can normalize a biological process in the body – with beneficial effects for patients. To see whether we can affect the protein’s activity, we first need to be able to measure it.
We produce, or “express”, the target in large quantities and measure its natural activity in assays. The assay needs to be sensitive, accurate, and highly reliable.
Plus, in order to perform hundreds of thousands of measurements, it needs to be automated, using robotic equipment.
Biochemistry
(High Throughput Screening)
But there are two sides to the discovery process – a target and a compound.
Compounds are substances which, we hope, will modify the activity of a target involved in a pathological process and which can be developed into a drug
for patients.
At Idorsia, we maintain a library consisting of hundreds of thousands of different compounds. To begin our hunt for drugs, we test the entire library on the target,
in the hope that one of these compounds will modify the activity of the protein.
This process is called high-throughput screening; if it’s a simple assay, we can test the whole library within a matter of weeks, with the goal of identifying compounds which exhibit activity on the target.
Structural Biology and
Molecular Modeling
Target and compound needs to fit together like a lock and a key. Our experts in structural biology and molecular modeling analyze these compounds – using techniques and tools such as computer-aided drug design – to aid
the optimization process by looking
at 3D shapes, interactions, and properties of the molecules.
Research Information Management
Throughout the drug discovery process, huge amounts of data are generated, and powerful IT tools are required to extract the knowledge we need. To really understand the data, we visualize it and study the relationship between chemical structures and biological properties.
Medicinal chemistry involves the use of chemistry’s tools to design molecules that are potential drugs. Based on the knowledge gained from literature, high-throughput screening results, initial studies, and molecular modeling, we form a hypothesis for analysis. We then enter drug design with the goal of finding the ideal compound – which must not only be potent but have
the right properties such as solubility, stability, bioavailability and selectivity.
The molecules we design are synthesized and then sent back to our biologists or pharmacologists for testing in an iterative process. With each cycle, the compound is further optimized to ultimately deliver a potent, effective and safe drug for development.
Medicinal Chemistry
Pharmacokinetics & Metabolism
At this point, we also study how the drug substance moves through the body. This is vital to understand key factors such as the duration of the treatment effect and intensity, which helps determine the appropriate dose.
Chemistry Process R&D
Small-scale testing for initial assays requires only milligram quantities; for subsequent testing, however, much more material is needed. This is where our process research teams come into the picture. They are responsible for scaling up from milligram to gram quantities, and finally to the kilogram batch which is used for preclinical testing.
It’s no good having a potent compound which is destroyed by the body before it has a chance to do its job. Our chemical and drug product specialists take the optimized compound and develop the most robust, safe, and cost-efficient formulation to deliver it to the patient.
Once reproducible processes have been elaborated to produce large quantities of the active compound and the formulated drug product, our technical project teams manage the production of the drug with partner companies. They secure drug supplies for clinical development and, when appropriate, for commercial launch and beyond.
Pharmacology
Once we have identified a target with good potency and a reasonable distribution profile, we test them in disease models which reproduce most of the clinical manifestations of the disease. These experiments allow us to evaluate how our compounds perform in disease models and possibly benchmark their activity against that of reference compounds. In the disease models,
we not only seek to improve the efficacy of our compounds, but also investigate
any possible side effects in dedicated safety studies.
Our drug discovery process
Targets
Compound Library
Hits
Lead Structure
Improved Leads
Preclinical Development
Drug Development
Clinical Development
Registration
Launch
Molecular Biology
(Target Finding)
Biochemistry (High Throughput Screening)
Structural Biology and Molecular Modeling
Research
Information
Management
Medicinal
Chemistry
Pharmacokinetics & Metabolism
Chemistry
Process R&D
Pharmacology
Our work in the lab begins with the target. This may be a particular protein which, when its activity is modulated, can normalize a biological process in
the body – with beneficial effects for patients. To see whether we can affect the protein’s activity, we first need to be able to measure it.
We produce, or “express”, the target in large quantities and measure its natural activity in assays. The assay needs to be sensitive, accurate and highly reliable. Plus, in order to perform hundreds of thousands of measurements, it needs to be automated, using robotic equipment.
Molecular Biology
(Target Finding)
But there are two sides to the discovery process –
a target and a compound.
Compounds are substances which, we hope,
will modify the activity of a target involved in
a pathological process and which can be developed into a drug for patients.
At Idorsia, we maintain a library consisting of hundreds of thousands of different compounds. To begin our hunt for drugs, we test the entire library on the target, in the hope that one of these compounds will modify the activity of the protein. This process is called high-throughput screening; if it’s a simple assay, we can test the whole library within a matter of weeks, with the goal of identifying compounds which exhibit activity on the target.
Biochemistry (High Throughput Screening)
Structural Biology and
Molecular Modeling
Target and compound needs to fit together like
a lock and a key. Our experts in structural biology and molecular modeling analyze these compounds – using techniques and tools such as computer-aided drug design – to aid the optimization process by looking at 3D shapes, interactions,
and properties of the molecules.
Research Information Management
Throughout the drug discovery process, huge amounts of data are generated, and powerful IT tools are required to extract the knowledge we need. To really understand the data, we visualize it and study the relationship between chemical structures and biological properties.
Medicinal Chemistry
Medicinal chemistry involves the use of chemistry’s tools to design molecules that are potential drugs. Based on the knowledge gained from literature,
high-throughput screening results, initial studies,
and molecular modeling, we form a hypothesis for analysis. We then enter drug design with the goal
of finding the ideal compound – which must not only be potent but have the right properties such as solubility, stability, bioavailability and selectivity.
The molecules we design are synthesized and then sent back to our biologists or pharmacologists for testing in an iterative process. With each cycle,
the compound is further optimized to ultimately deliver a potent, effective and safe drug
for development.
Pharmacokinetics & Metabolism
At this point, we also study how the drug substance moves through the body. This is vital to understand key factors such as the duration of the treatment effect and intensity, which helps determine the appropriate dose.
Chemistry Process R&D
Small-scale testing for initial assays requires only milligram quantities; for subsequent testing, however, much more material is needed. This is where our process research teams come into the picture. They are responsible for scaling up from milligram to gram quantities, and finally to the kilogram batch which is used for preclinical testing.
It’s no good having a potent compound which is destroyed by the body before it has a chance to do its job. Our chemical and drug product specialists take a compound which has been the optimized by the chemists compound and develop the most robust, safe, and cost-efficient processes for the drug substance, the formulation, and packaging materials to deliver it to the patient.
Once reproducible processes have been elaborated to produce large quantities of the active compound and the formulated drug product, our technical project teams manage the production of the drug with partner companies. They secure drug supplies for clinical development and, when appropriate, for commercial launch and beyond.
Pharmacology
Once we have identified a target with good potency and a reasonable distribution profile in animals, we test them in animal models which reproduce most of the clinical manifestations of the disease. These experiments allow us to evaluate how our compounds perform in disease models and possibly benchmark their activity against that of reference compounds. In the disease models, we not only seek to improve the efficacy of our compounds, but also investigate any possible side effects in dedicated safety studies.
Our drug discovery process
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.
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.