RSC Chemical Information and Computer Applications Group Measurement

RSC - Chemical Information and Computer Applications Group Measurement, Information and Innovation: Digital Disruption in the Chemical Sciences Tuesday 20 th October 2015, RSC, Burlington House, Piccadilly, London John Wise, Executive Director Business Development, Pistoia Alliance

Challenges in a Changing Landscape The Challenge for Pharma Drug Efficacy is not the same as Drug Effectiveness The terms “efficacy” and “effectiveness” have very different meanings. Efficacy refers to the extent to which a drug does more good than harm in clinical trials where patients are carefully selected and monitored Effectiveness refers to the extent to which a drug does more good than harm in real life where patients are not so narrowly selected and often not closely monitored. Hans-Georg Eichler, M. D. , M. Sc. Senior Medical Officer at the European Medicines Agency in London, United Kingdom The Tale of Health Care Reform - DIA Global Forum December 2010 p 20 © Pistoia Alliance [Today] “Pharma is developing drugs that bring incremental benefits, but at a premium price. This has given rise to the debate between the providers and payers—what is the value of the extra benefit? ”

3 © Pistoia Alliance Escaping the sword of Damocles: Toward a new future for pharmaceutical R&D (Mc. Kinsey)

How Much Does Pharmaceutical Innovation Cost? A Look At 100 Companies Blue Line – The number of NMEs / BLAs per company in 10 years Brown Line – Average cost to develop each NME / BLA over 10 years Blue Arrow – Member of the Pistoia Alliance 4 http: //www. forbes. com/sites/matthewherper/2013/08/11/the-cost-of-inventing-a-new-drug-98 -companies-ranked/

A rapidly evolving ecosystem Yesterday Tomorrow Big Life Science Company Yesterday Tomorrow Innovation Model Innovation inside Searching for Innovation Heterogeneity of collaborations. Part of the wider ecosystem IT Internal apps & data Struggling with change Security and Trust Cloud/Services Data Mostly inside Inside Outside Distributed Portfolio Internally driven and owned Partially shared Shared portfolio

Strategic Alliance in Life Sciences “Currently there is a lack of adequate alliance setup and first-hand experience of alliance management which will most likely only serve to inhibit the industry’s ability to capture the full value of strategic alliances. To realise the true potential of strategic alliances an increase in the number of alliance engagements as well as a high commitment – be it financial or not – is required. ”

Racing to define pharmaceutical R&D external innovation models Liangsu Wang, Andrew Plump, Michael Ringel Drug Discovery Today, March 2015

Racing to define pharmaceutical R&D external innovation models Liangsu Wang, Andrew Plump, Michael Ringel Drug Discovery Today, March 2015

The Pistoia Alliance

Pistoia Alliance Lowering the barriers to innovation in life science R&D by improving inter-operability of business processes © Pistoia Alliance through pre-competitive collaboration

Current Pistoia Alliance members 11 © Pistoia Alliance 25 th Nov 2014

IP 3 encourages open innovation • Collection of all PA project ideas • Highlights PA active and funding portfolios • Discussion around ideas • Progress updates and status reports • Fully open to everyone • Please contribute! https: //main. qmarkets. org/live/pistoia/ 12

Develop new insights and new contacts via the Pistoia Alliance webinar programme 13

Current Pistoia Alliance projects – Chemists need to be aware of the potential hazards of their experiments. – However, such information is not always to hand where chemists need it most - at the bench. – Ontologies are critical to manage life sciences data. – Within each domain we often find multiple ontologies being applied – a better understanding of the ontologies that are used, and ways to interchange between these ontologies. – Identify industry associations, alliances, and initiatives, and identify the domain and scope of each. – Create pictures and tools to allow the industry to identify who it should contact for its needs. – Enable alliances to identify their partners and competitors and how best to support each other and collaborate towards the common goal of improving science.

Recent Pistoia Alliance successes • Controlled Substance Compliance Services (CSCS) – Big pharma and compound vendors alike benefit from standardised commercial tools to interpret regulations • • Sequence Services – establishing secure, cloud-based implementations of gene sequence analysis Sequence Squeeze - developed a number of new, faster, better compression algorithms for NGS data tran. SMART - established an independent foundation to maintain and support the code, community and continuity of this popular translational research tool Hierarchical Editing Language for Macromolecule (HELM) – Exchangeable and consistent notation allows for easier sharing and representation of complex molecules, such as antibodies – Won the Bio-IT Best Practices Award 2014

Pistoia Alliance HELM Project Setting the Standard for Biomolecular Data Exchange Pistoia Alliance Project Leader: Sergio H. Rotstein, Ph. D. Pistoia Alliance Project Manager: Claire Bellamy, MBA

What is a “Biomolecule” ASOs si. RNAs Peptides Therapeutic Proteins Antibodies ADCs Vaccines © Pistoia Alliance Biomolecule: Anything that is not a small molecule

Stuck in the middle… Small Molecules Sequence-Based Tools Sequences Biomolecules © Pistoia Alliance Small Molecule Tools G A P

Pistoia HELM Project Goal Transition HELM technology from Pfizer proprietary to Open Source © Pistoia Alliance • Provide an industry-wide standard for data exchange within and between organizations • Reduce software development costs and time by minimizing the need for companies to develop similar functionality

Open Source HELM Editor API https: //github. com/Pistoia. HELM Code for • HELM Toolkit • HELM Editor • HELM Antibody Editor Permissive MIT licence © Pistoia Alliance HELM Notation Toolkit

HELM Evolution Open. HELM Released 2012 2013 Pistoia project conceived Pistoia project started UNIVERSITY OF CAMBRIDGE Andreas Bender Group Inline Search HELM Prototype HAb. E Released 2014 Exchangeable HELM 2015 Ch. EMBL 20 with HELM © Pistoia Alliance Paper published 21

How do you take the HELM? Biomolecule Data Exchange Mechanism Level of Adoption 22 © Pistoia Alliance Foundation for your biomolecule informatics infrastructure • Registration • Visualization • Analysis and design • Workflows

The HELM Ecosystem • Pharma / Biotech / Institutes – BMS, GSK, Lundbeck, Merck, Novartis, Pfizer, Roche • Software vendors – ACD/Labs, Arxspan, Biochemfusion, Bio. Max, Biovia, Chem. Axon, Next. Move, Scilligence • Content / Service Providers • Active discussions on-going with others © Pistoia Alliance – EBI (Ch. EMBL), e. Molecules, quattro research,

HELM Phase 2 - Ambiguity Systems need to handle molecules that are not always fully defined 24 © Pistoia Alliance • A design for the representation of ambiguity has been drafted • RFP Issued and bid selected • Development work starting soon

IDMP The implementation guide for ISO 11238: Health Informatics - Identification of medicinal products will include HELM as an acceptable format. 25 © Pistoia Alliance Working with the FDA to include HELM as a format within GIn. AS. • GIn. AS (Global Ingredient Archival System) will provide a common global identifier for all substances used in medicinal products or active substances under clinical investigation

HELM Team Members Active Team Members: • Roland Knispel (Chem. Axon) • Matthias Nolte (BMS) • Jan Holst Jensen (Chembiofusion) • Thomas Gan (Merck) • Stefan Klostermann (Roche) • Sven Neumeyer (Novartis) • Yohann Potier (Novartis) • Tianhong Zhang (Pfizer) Steering Committee Members: • John Wise (Pistoia Alliance) • Margret Assfalg (Roche) • Leah O'Brien (GSK) • Ramesh Durvasula (BMS) • Sergio Rotstein (Pfizer) • Alex Drijver (Chem. Axon) • Chris Waller (Merck) • Quan Yang (Novartis) Pfizer Team • Peter Henstock • David Klatte • Christine Lawrence • Frank Loganzo • Hongli Li • Sergio Rotstein • Simone Sciabola • Rob Stanton • Nathan Tumey • Simon Xi • Tianhong Zhang © Pistoia Alliance The Pistoia Alliance HELM Project Team, especially: • Sergio Rotstein (Pfizer) – Domain Lead • Claire Bellamy (Pistoia Alliance) – Project Manager