Certara, Inc. · NASDAQ:CERT
Certara — Technical Presentation (2011)
36 slides from Certara's technical presentation, 2011
Slide 1: The Emerging Role of Translational Science as Part of a Model Based Drug Development Paradigm
The Emerging Role of Translational Science as Part of a Model Based Drug Development Paradigm: Building an Infrastructure for the Future Daniel Weiner, PhD © Certara, L.P. 2011 All Rights Reserved
Slide 2: Presentation Topics for Today
Presentation Topics for Today 1. Background – The business case for model based drug development 2. Translational Science 3. Criteria for a successful informatics infrastructure 4. Implementation Issues and conclusions 2 © Certara, L.P. 2011 All Rights Reserved
Slide 3: Only 21 New Drugs Were Approved by the FDA in 2010
Only 21 New Drugs Were Approved by the FDA in 2010, and Only A Few of These Were First in Class or Represented A Significant Therapeutic Advance Only 1 in 10,000+ molecules gets approved, and of that small percentage, only 30% of drugs that are approved have a positive ROI Ref: Mullard , A. (2011) N
Slide 4: The Lack of Consistent Success is Adversely Affecting Pharmaceutical Stock Prices
The Lack of Consistent Success is Adversely Affecting Pharmaceutical Stock Prices Pharmaceutical Index (^DRG) This capitalization-weighted index is designed to represent a cross-section of widely held, highly capitalized companies involved in various phases of the pharmaceutical industry. ~20% 4 © C
Slide 5: Companies are Trying to Cope In Part By Raising Prices
Companies are Trying to Cope In Part By Raising Prices 5 © Certara, L.P. 2011 All Rights Reserved
Slide 6: But Price Controls Will Eventually Occur in the U.S.
But Price Controls Will Eventually Occur in the U.S. 6 © Certara, L.P. 2011 All Rights Reserved
Slide 7: A Perfect Storm …
A Perfect Storm … Source: BioIT World 7 © Certara, L.P. 2011 All Rights Reserved
Slide 8: When Profits Go Down …
When Profits Go Down … 1. There are three basic ways to address this 2. Mergers and acquisitions – e.g., Pfizer (Wyeth), Merck (Schering Plough, many others). These are rarely synergistic and ―value‖ comes from eliminating redundant expenses. By most measures while having some short term positive im
Slide 9: A Major Problem is that Bad Drugs Need to Fail Sooner
A Major Problem is that “Bad” Drugs Need to Fail Sooner Failure rate is 50% in phase 3 for drugs completing phase 2B Presented at Kitasato-Harvard Conference, Sept. 2008 © Certara, L.P. 2011 All Rights Reserved
Slide 10: The Prior Slides Beg the Question…
The Prior Slides Beg the Question… Why are these drugs failing so late in development (as opposed to earlier in development)? Are these (late stage) failures occurring because of • Poor formulations? • Unexpected toxicity? • Unacceptable benefit/risk ratio? • Inability to determine an optimal dose o
Slide 11: Comments on Historical Approaches
Comments on Historical Approaches Challenges • What Biomarkers could have predicted efficacy and safety issues earlier? • Can we learn this from current experiments? • What are the population variables with this therapeutic issue? • Safety, Efficacy, Dosing regimen • Which clinical trial population?
Slide 12: Motivation for Change
Motivation for Change The current Pharma model, which is fraught with increasing costs year on year while approvals are declining, is not sustainable. Consensus: Companies need to develop better lead candidates (molecules) while at the same time reducing the risk of developing them. To achieve this
Slide 13: Model Based Drug Development – Is a Prerequisite for Improvement
Model Based Drug Development – Is a Prerequisite for Improvement FDA’s characterization: the development and application of pharmaco-statistical models of drug efficacy and safety from preclinical and available clinical data to improve drug development knowledge management and decision making. If im
Slide 14: Increasing Reliance on Biotech to Drive Growth
Increasing Reliance on Biotech to Drive Growth 14 © Certara, L.P. 2011 All Rights Reserved
Slide 15: The Translational Science Imperative
The Translational Science Imperative Fully loaded cost for developing a single novel therapeutic with ―blockbuster potential‖ > $800M (the number is ~ double this if you include amortized failures) A major component of estimated expenditure is the cost of failure • > 90% chance that an agent that en
Slide 16: What is Translational Science?
What is Translational Science? It means different things to different people. Most definitions involve the translation of science to clinical practice. In common use it has been applied to everything from drug discovery, to preclinical (e.g., allometric scaling) to medical treatment (patient individ
Slide 17: Need for Improved Collaboration Reinforced …
Need for Improved Collaboration Reinforced … Clinical and Translational Science: A New Discipline to Catalyze the Transfer of Information. Arthur M. Feldman, MD, PhD 1 1 Editor-in-Chief, CTS. Volume 1, Issue 1, 2008. … This concept of "translating" novel discoveries found at the laboratory bench int
Slide 18: What Does it Involve?
What Does it Involve? Clinical and Translational Science: A New Discipline to Catalyze the Transfer of Information. Arthur M. Feldman, MD, PhD 1 1 Editor-in- Chief, CTS. Volume 1, Issue 1, 2008. … Furthermore, a variety of impediments, including … the need to integrate clinical and translational sci
Slide 19: Translational vs. Clinical Pharmacology Approaches
Translational vs. Clinical Pharmacology Approaches Clinical Pharmacology Translational Science/Medicine The primary focus is the drug. The primary focus is the disease, and how to target drugs to alter the natural progression of the disease. 19 © Certara, L.P. 2011 All Rights Reserved
Slide 20: Translational Science Takes Root
Translational Science Takes Root Translational Medicine groups have been established both in academia and industry with the mandate of improving the translation of the wealth of new data into new medicines for the treatment of human disease. • At least 50 pharma and biotech companies have establishe
Slide 21: Companies are Starting to Reorganize Along Translational Science Lines
Companies are Starting to Reorganize Along Translational Science Lines, and … One concept of Translational Science is to break down the barriers between Discovery and Development. Several companies have reorganized along these lines. Translational Science Discovery Development Cheminformatics Bioinf
Slide 22: Need an Informatics Infrastructure to Facilitate Data Sharing and Feedback
… Need an Informatics Infrastructure to Facilitate Data Sharing and Feedback Shared Data from Research and Development Post Marketing • SAR • EC50 • IC50 • Biomarkers • AUC • ADRs • PK/PD t1/2 • Drug-drug • Binding Mode • Genomics • PK/PD t1/2 • Toxicity interactions •Chemical • Pathways • Clearance
Slide 23: Key Barriers to Implementation of Translational Approaches
Key Barriers to Implementation of Translational Approaches Organizational—budgets, priorities, reward systems are set by multiple different departmental managers in large organizations. This may lead to sub-optimization of the entire system. Scientific Silos—scientists see problems from their own pe
Slide 24: Personal Observations on the Organizational/Silo Issue
Personal Observations on the Organizational/Silo Issue Here are two examples of political/control issues getting in the way of making progress in implementing effective translational approaches. • I know of multiple companies where one M&S team existed with limited success, and the company is trying
Slide 25: Conquering Barriers to the TM Vision
Conquering Barriers to the TM Vision Organizational • Organizational issues can be overcome in 2 years or so by motivated pharma and biotech companies Scientific Silos • Scientific silos will take 5 or more years to break down and implement new approaches • Move to a Translational based organization
Slide 26: Enablers – Standards and Processes
Enablers – Standards and Processes Process Changes 1. Automate and standardize data collection, handling and reporting 2. Establish central proprietary databases (that are domain specific) 3. Utilize all available information to generate knowledge and optimize development at compound and portfolio l
Slide 27: How Might a Translational Query Work?
How Might a ―Translational Query‖ Work? Data Networks Across Multiple Domains of Data A drug is demonstrating liver toxicity in clinical studies. TS query example: • Is this toxicity related to increased exposure? • Are there any preclinical biomarkers that could have predicted this? • Are there any
Slide 28: A Typical Researcher's Desktop
A Typical Researcher’s Desktop Many applications, little integration! Where? How? What? Chemical inventory Chemical Chemistry sketchers Research data access reporting Scientific Discovery literature search information portal Data manipulation Electronic & analysis notebook Structure visualization Bi
Slide 29: Effective Translational Approaches Require …
Effective Translational Approaches Require … An informatics infrastructure with 3 key, high level capabilities: 1. Data Access • Users can ask any question of the data appropriate to their task and understanding • Data is presented in a manner that the user understands 2. Data Analysis • Data is tra
Slide 30: Where Should Such a Solution Be Applied?
Where Should Such a Solution Be Applied? ALL Phases of Drug Development Early Drug Discovery • Standard project data views through complex data mining • Users from chemistry, biology, management… • Applicable to both large and small organizations Preclinical • Animal PK/PD data • Safety Data (tox, p
Slide 31: How Should Queries Be Facilitated?
How Should Queries Be Facilitated? Most companies will want to keep data sources federated • Relational databases, web services, files… • ―Complete‖ Data warehouse not a requirement A Queryable Data Network is created through logical data relationships that is comprehensible to users • The solution
Slide 32: Queryable Data Networks for Safety Data
Queryable Data Networks for Safety Data Clinical/preclinical scientists can use such a solution to perform study or group-centric searches that return related subject, treatment, and observation information • Queryable data networks relate data in scientifically meaningful ways • The same data can b
Slide 33: D360 User's Desktop: A Hub for Data Access & Analysis
D360 User’s Desktop: A Hub for Data Access & Analysis Research Chemistry sketchers reporting Chemical Data inventory manipulation & analysis Discovery information portal Data analysis Electronic notebook Structure visualization Scientific literature search 33 © Certara, L.P. 2011 All Rights Reserved
Slide 34: High Level Architecture of TS Solution (D360)
High Level Architecture of TS Solution (D360) 34 © Certara, L.P. 2011 All Rights Reserved
Slide 35: Benefits of a Translation Support Platform
Benefits of a Translation Support Platform Link data ―Islands‖ • Provide support for translational approaches to research • e.g. discovery, non-clinical safety, PK-PD, clinical data Serve the needs of the scientist: allow the user to easily go from Question -> Action • Automatically present data in
Slide 36: Summary and Conclusions
Summary and Conclusions Companies need to move to a true Translational development model to improve profitability. • The move to a Translational-based organization needs to be driven top-down. • Reorganizing is not enough – there needs to be an informatics platform in place to support collaboration