Transcript PharmAdvisors Capabilities

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Pharmaceutical Advisors
– Core staff with average 20 years industry experience
– Network of over 100 Advisors and growing
– Covering every discipline for pharmaceutical development
and commercialization
– Founded in 2001, currently more than 75 clients
• Small and mid-sized companies
• Emerging pre-clinical and clinical stage companies
• Top 5 pharmaceutical companies
• Suppliers to the Life Sciences
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The Issue We Address
Everyone can pull in the various experts needed …
Chemistry
ADME
Tox
Process
Development
Formulation
Project
Management
Quality
Engineering
Regulatory
Clinical
Manufacturing
Marketing
Drug Substance
Drug Safety
Drug Product
Clinical
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The Issue We Address
…linking broad and deep perspective early is key to
anticipating issues, accelerating progress, managing risk and…
unlocking value!
Chemistry
ADME
Formulation
Tox
Clinical
Process
Development Manufacturing
Marketing
Project
Management
Quality
Engineering
Drug Substance
Drug Safety
Drug Product
Clinical
Regulatory
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Advisors Industry Focus
Pharmaceuticals
Transaction
Valuation and
Licensing
Suppliers to the
Life Sciences
Animal
Health
Technology
Platforms
Medical Devices
Venture Capital
Biotechnology
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Advisors Functional Focus
Integrating Research, Development Engineering and
Commercialization Expertise
Click on Any Hexagon for Examples
Rapid,
Interactive
Expert Panels
Resolve Issues
Build Skills
Chemistry, Formulation
Drug Delivery
Safety & Efficacy
ADME
Practical
Quality
Systems &
Regulatory
Integrated
Development
View
Linking
CMC, Tox, Clinical
Portfolio
Decisions,
Due Diligence
& Valuation
Operational
Input to
Capital Plans
Scale-up &
Sourcing
Decisions
Supporting
Development, Operations, Strategy and Portfolio Management
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What Do We Do Best?
What + How = Delivery Assurance
Expert
Content
Efficient
Process
Delivery
Assurance
Professional
Management
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Inverting the Consulting Model…
Industry Experts
Experienced
Consultant
Managers
Unique Client
Situation
Proven Delivery
Insight-Driven
Customized Resourcing
Integrated, Experienced Functional Perspective
Hands-On Problem-Solvers
Cost-effective, Flexible, Independent
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With Experienced Advisors…
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Associate Director, Venture Mgmt
Asst Director for Technical Affairs
Chief Executive Officer
Chief Operating Officer
Dept. Head of Pharmaceutical Engineering
Director of Fermentation and Biocatalysis
Development
Director of Formulation Development
Director of Pharmaceutics R&D
Director of Quality Control
Director of Sourcing & Planning of Chemical
Development
Director, Clinical Operations, Medical Affairs
Director, Experimental Pathology
Director, Global Medical Operations
Director, Planning and Business Support, U.S.
Medical and Regulatory Affairs
Director, Production of Therapeutic and Diagnostic
Proteins
Exec Director of Manufacturing
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Executive Vice President, Global Drug Safety,
Metabolism and Safety Pharmacology
Group Director of Clinical Research
Group Director of Process R&D
Group Director, Preclinical Safety Assessment
Manager of Organizational Effectiveness
Mgr, Radiopharmaceutical Manufacturing
President
President of Technical Operations
Senior Director Operations Planning - Worldwide
Pharmaceutical Group
Senior Director, North American Clinical Affairs
Senior Manager, Clinical Research
Senior Vice-President and Chief Scientific Officer
Senior Director of Process Chemistry
Senior VP of Pharmaceutical R&D
Senior VP, Global Drug Development
Vice President of Business Development
Vice President Information Technology
Vice President of Drug Development
Vice President of Process Development
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Who’ve Run Companies…
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AAI, Inc.
American Cyanamid/Lederle Labs
American Home Product
Amylin Pharmaceuticals
Applied Immune Services
Arthur D. Little
Astra Pharmaceuticals
Aventis
Axxima Pharmaceuticals AG
Baxter Healthcare Corp.
Benzon Pharmaceuticals
BioReliance Corporation
BMS
Boehringer Ingelheim
Boots Pharmaceuticals
Bristol-Myers Squibb
BSCI, Inc.
Cambrian Associates
Cambrex
Cancer Research Center
Cardinal Health
Centocor
Ciba-Geigy Corporation
Corixa
Coulter Pharmaceutical
Dentsply
Discovery Partners
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Eli Lilly & Company
Environmental Toxicological, Int’l
US FDA Center CBER & CDER
FEI Technologies
Fisons
Fluor Daniel
Genentech
Genovo
GSK
GX BioSystems
Hoechst Marion Roussel
Hybridon
ICI Pharmaceuticals
IMBIC
Immunex
Immunogenetics
Immunomedics
IMRE Corporation
Isolation Technologies
J&J
Knoll Pharmaceuticals, Nottingham
Matthew Hall Engineering
Merck
Merck, Sharpe & Dohme
Mettler Toledo
MicroProbe Corporation
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Millennium
Monsanto
Napp Researche Centre
National Cancer Institute
Neose
Norcliff-Thayer
Novartis
Ortho Biotech
PA Consulting Group
Pfizer
Pharmacia
Response Biomedical
Richardson-Vicks
Salmedix
Schering Corporation
Schering-Plough
Searle
Sepracor Inc.
Sigma-Aldrich Corporation
SmithKline Beecham
SUGEN
Tektagen
The Life Sciences Group
Upjohn Ltd.
US Environmental Protection Agency
Wyeth-Ayerst
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With Broad Functional Expertise
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Advisory Board s
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Analytical Development
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Analytical Methods
Development/Validation
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Analytical Operations
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Biologic Manufacturing
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Bio-Pharmaceutical Development •
Bulk Active Ingredients
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Bulk Clinical Supply Planning
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Bulk Drug Substance
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Bulk Manufacturing
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Business Development
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Capital Raising
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Chemical Synthesis
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Chemotherapeutic Agents
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Clinical Pharmacology
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Clinical Research
Clinical Supplies Mgmt & Release •
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CMC of IND/NDAs
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Commercial Product Profiling
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Corporate Inventories
Cost Evaluation/Budget Prep & •
Control
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CRO, Supplier Auditing & Mgmt •
Diabetes & Metabolic Diseases •
Documentation
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Drug Delivery Technology
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Drug Development Drug
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Interaction
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Drug Metabolism
Drug Product
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Endocrinology & Metabolism
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Engineering
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Environmental Impact Analysis •
Expert Reports, Dossiers, DMFs
Facility Design and/or Operation •
FDA Regulations & Inspections •
(Technical Data Pkgs.)
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Fermentation
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Filling & Packaging Operations •
Finance
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Formulation Development
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Good Clinical Practices
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Hematology & Internal Medicine •
Immunochemistry Research
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In Vitro Diagnostics
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In-Process Control Activities
Intellectual Property Management•
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Investments
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Just-In-Time Methodologies
Laboratory Operations
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Life-Cycle Strategy
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Lipid Consultant
Long-Term Supply Agreements •
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Manufacturing
Materials Resource Planning New
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Bulk Substances
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Novel Formulations
Oncology Clinical Research
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Operations
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Operations Research &
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Management
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Organizational Analysis
Parenteral/Oral Dosage Forms •
Patents & Procedures
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Performance Audits & Validation•
Pharmaceutical Formulation
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Pharmaceutics Research
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Pharmacodynamics
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Pharmacokinetics
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Phase III/Commercial Needs
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Portfolio Management Strategies
(Evaluation)
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Preformulation Studies
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Preparative Chromatography •
Process & Lab Automation
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Process and/or Equipment
Validation
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Process Development
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Product Development
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Product Launches
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Product Licensing
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Project Implementation
Project Management
(Multi-national)
Purchasing
Quality Assurance
Quality Control
QC Strategies &
Assessment/QA
Quality Systems
R&D
Radiolabeled Antibodies
Regulatory Affairs
Rx to OTC Opportunities
Safety Evaluations
Sales and/or Marketing
Scale-Up from R&D to
Manufacturing
SOP Creation
Start-Ups
Supply Chain
Synthetic & Semi-Synthetic
Drug Candidates
Target Identification
Technology Profile
Technology Transfer
Toxicology-Pathology
Transdermal Technology
...That is Growing
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Our Strengths to Help You
• HOW we support our clients
– Speed & flexibility
– Content expertise & general management perspective
– Business decisions & technical expertise
• Use our approach to solve problems and address issues
– The RIGHT expertise based on client needs
– We efficiently identify and involve what’s needed
• We effectively deploy and integrate the RIGHT resources
– Insight about implications on other functions and processes
– Multiple perspectives within a specialty where needed
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Some Relevant Case Studies
1. Support of Critical Manufacturing Issues for a Complex Biologic
2. High Powered Expert Panel on a Deal- Breaker Issue
3. Package/ Shipping Validation to Launch Key Products
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Recent Case Study (1)
Support Critical Manufacturing Issues for a Complex Biologic
The Issue
• Our client is a small molecule drug discovery and development company that acquired a
commercially launched biologic pharmaceutical. Process variability in the fermentation of the
drug was creating N-terminal variants, preventing the product from being manufactured reliably.
What We Did
• Assembled a cross-functional team of Bio-Analytical, Fermentation, and Bio-Manufacturing
experts. We supported the client through the investigation, experimentation, and
implementation phases of the project by:
– Assessing the process and existing data for root cause of process variability
– Selecting and directing the outsourced supplier to identify weaknesses of the existing
process and analytical methods supporting the process
– Identifying the most likely sources of variability
– Establishing vendors for new analytical methods and fermentation development
– Designing and executing an experimental protocol to definitively assign root cause
– Designing cost-effective scale-up plans to manufacture the revised process at scale
• Providing the client with:
– The root cause of variability in the face of a complex web of confounding factors
– A cost-effective solution to product variability, avoiding product withdrawal
Protected a key source of revenue during a critical phase of growth
for a young and growing Bio-Pharmaceutical Client
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The Team
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John Apathy, Trained in Biochemistry and Immunology. 20 years experience developing
strategies and leading pharmaceutical product development teams within a major
pharmaceutical company and as senior managing consultant with a major pharmaceutical
consultancy.
David Dodds, PhD, Former Director of Fermentation and Bio-catalysis Development with
BMS
Kent Iverson, formerly with Immunex and Coulter Pharmaceuticals, has led the Process
Development and Materials Management departments of all products from pre-clinical
development through commercialization.
John McEntire, PhD Former Corporate Vice President of BioReliance, with expertise in
biopharmaceutical development analytical and formulation.
Tim Noonan, PhD. Trained as a medicinal chemist, and a former VP of manufacturing,
Tim has broad manufacturing, operations and quality systems experience, was instrumental
in the formation of a successful contract oligonucleotide manufacturing firm. Dr. Noonan
also worked as a consultant to venture capital firms performing technology due diligence.
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Recent Case Study (2)
High Powered Expert Panel on a Deal- Breaker Issue
The Issue
• Our client identified a degredant as a known mutagen. The client needed to understand the
implications for development and the value of the compound for a partner meeting. They
also needed the right regulatory strategy, as well as an understanding of the trade-offs
between salt forms to pursue appropriate development programs.
What We Did
• In less than 2 weeks assembled a team that addressed their issues
– 2 Gentox experts that wrote the ICH guidelines
– 1 Gentox expert that convinced the FDA to act favorably with a similar situation
– CMC expertise with polymorph expertise
– Pharmacology
– Tox expertise in early and late development strategies
• Providing the client with:
– Answers to the right questions to show their partner that they had control and a plan
– Awareness of questions they had not considered
– Understanding of what was NOT an issue
– Insight into the right development strategies to manage risk
– Confidence that they had explored all the alternatives
– One place to go for follow-up and coaching
No one got on a plane, stayed overnight, or was wined and dined
No one had to assemble and assimilate the input
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The Team
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Jane S. Allen, Ph.D., D.A.B.T., former Director of Toxicology, GSK, RTP with expertise in genetic
toxicology. Member of US PhRMA committee that established the ICH genetic toxicology guidelines.
Michael Berman, PhD, is a former biotech CEO with broad experience in pre-clinical development
for Phase I studies with a focus on biologicals and peptides as well as small molecules. Dr. Berman
has taken a number of novel biologicals into Phase I.
Robert L. Brent, MD, Ph.D. Distinguished Professor of Pediatrics, Radiology and Pathology and
Louis and Bess Stein Professor of Pediatrics, Jefferson Medical College, Philadelphia. Former advisor
to Aventis for ARAVATM, with expertise in clinical medicine related to genetic toxicology.
Charles E. Piper, PhD, former Executive Director, Clinical Research Strategic Partnering, Clinical
Development, and former Executive Director, Product Safety Assessment, Preclinical Development,
Searle. Member of US PhRMA committee that established the ICH genetic toxicology guidelines.
Thomas Crawford, PhD, former Group Director of Process R&D with Pfizer where he was
responsible for the development of chemical technology and the manufacture of supplies for drug
candidates from nomination to transfer to Pfizer's Global Manufacturing Group.
William D. Kerns DVM, MS, DACVP former Executive Vice President, Global Drug Safety,
Metabolism and Safety Pharmacology, Eisai and SmithKlineBeecham.
Guy Paulus, PhD. Served most recently as Senior Vice President Drug Safety Evaluation Pfizer
Central Research Groton U.S.
Robert Ronfeld, Ph.D. who led Pfizer’s Clinical Pharmacokinetics and Pharmacodynamics group
with particular expertise in Drug Metabolism and Development Strategy.
Richard S. Woodward, Ph.D. Biotechnology and pharmaceutical executive with multidisciplinary
international experience. Formerly with Pharmacia, Ajinomoto USA, Inc. and Quality Biotech, Inc.
Tim Noonan, PhD. Trained as a medicinal chemist, and a former VP of manufacturing, Tim has
broad manufacturing, operations and quality systems experience, was instrumental in the
formation of a successful contract oligonucleotide manufacturing firm. Dr. Noonan also worked as a
consultant to venture capital firms performing technology due diligence.
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Recent Case Studies (3)
Complex Package/ Shipping Validation to Launch Key Products
The Issue
• Our client is extending its growth strategy requiring an enhanced capability in packaging / package
design, logistics, shipping validation, quality, and management of vendors beyond current resources
and internal capabilities.
What We Did
• Assembled a cross-functional team of Logistics, Quality, Packaging and Manufacturing / Operations
experts. We are supporting the client through the investigation, planning & experimentation, and
implementation phases of the project by helping to determine:
– when and how to utilize engineering resources from package design and testing firms vs.
independent engineering resources,
– the cumulative effect of expected Time /Temp excursions in the context of stability / shelf life
and possible variations in packaging and logistics
– where to utilize off-the-shelf secondary packaging solutions
– where custom packaging solution are appropriate, trading off cost of manufacture and design
with logistics and other costs.
– efficiencies anywhere in the distribution chain, based on decisions in packaging or other areas
– with complex, issues such as this, any key activities or decisions not assigned or staged.
• Providing the client with:
– Roadmap for the packaging, shipping and shipping validation project, maintaining the timeline
– Key program management
Enabling progress in a critical area without the need to scale-up resources
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The Team
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John Apathy, Trained in Biochemistry and Immunology. 20 years experience developing
strategies and leading pharmaceutical product development teams within a major
pharmaceutical company and as senior managing consultant with a major pharmaceutical
consultancy.
William Gierke, former manager of Package Development with Eli Lilly and a member of
the Drug & Pharmaceutical Packaging Committee
Kent Iverson, former Vice President of Manufacturing & Process Development at Corixa,
Coulter and Immunex. Kent has broad operational expertise and has implemented practical
solutions to complex controlled temperature shipping issues for injectables and has
developed successful supply plans for numerous 2-8C products.
John Pock, former head of Performance Engineering & Worldwide Quality as well as
manager of International Distribution at Eli Lilly.
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Other Recent Projects
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Rapid due diligence of compounds for critical decisions
Manufacturing and Outsourcing strategy including determination of
number of locations and in- source / outsource mix
Integrated early and full drug development plans
Quality Systems planning and implementation for developing company
cGMP Quality System Gap Analysis for an established company
Process development to address issues on a marketed product
Site Audits with regulatory and practical operations perspective
Manufacturing readiness assessment
Sourcing - identification of sources and review / selection
Candidate nomination process and portfolio review
Formulation and drug delivery choices
Market studies by simulating a pharma development group
Deal valuation support
cGMP Supplier Management
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Why do clients look to us?
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Trust and confidence that we will get it done
We apply world-class expertise that exactly meets the need
Efficiency and effectiveness of our structured approach
Avoid time consuming management and coordination of
challenging resources and diverse inputs to complex issues
Speed of results
Accountability at a single, rather than multiple points
We ensure a cross-functional and downstream view
Fixed predictable budget vs. multiple experts with “ the meters
running at different rates”
Delivery Assurance
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What is keeping you up at night?
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Practical Quality Systems
& Regulatory Help
What we do and how it’s different:
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Operations oriented Quality perspective, not just “ex-FDA…”
Practical approaches to regulatory compliance
Helps avoid costly reactive remedial consultants
Cost-effective, proactive compliance
Examples of recent projects:
• Establishment of appropriate cGMP systems for a research
based company moving to commercial development
• GAP Analysis and Action Plans following FDA Warning Letter
• Vendor Audits that strengthen the vendor / client relationship
• CGMP Gap Analysis and Action Plan
• Pre-Approval Inspection Readiness Assessment
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Operational Input to Capital Plans
What we do and how it’s different:
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Application of functional operations AND engineering expertise to
complement client resources
Helps client identify operational implications and issues with a
"downstream” perspective
Avoids costly change orders and “feature creep”, and supports sound
decisions, early in the process
Examples of recent projects:
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Development of a URS ( User requirement Specification ) for $200M
Small and Large Molecule development facility
“Future Proofing” of current pharmaceutical process through process
and equipment reviews
Process Validation, Equipment Qualification and Operational Efficiency
input to design of new vessels for aseptic processing
Development of downstream processing technology requirements to
meet future development needs.
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Resolve Issues and Build Skills
What we do and how it’s different:
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Focused and efficient application of functional expertise
Integrated development perspective
Multi-company perspectives
Access to the latest thinking
Integration of inputs without costly coordination
Examples of Recent Projects:
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Expert oversight of challenging outsourced cytotoxic fermentation
Dosage form strategy and action plan to obtain IP protection
Dosage form strategy to overcome half life deficiency
Micro Lab productivity analysis and recommendations to improve
efficiency and handle greater workload without adding staff
Operational Cost Model for research stage company transitioning to a
product stage company
Informed Tox, CMC development and risk reduction plans to accelerate
progress
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Rapid & Interactive Expert Panels
What we do and how it’ s different:
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Expert teams, custom assembled to address critical issues
Focused assignments for panel members ensure efficient process
Individual and group discussions, across-functions and within functions
Interactive panel discussions via telecon with the client
Real, actionable answers, action plans
Completed in 1-3 weeks
Examples of recent projects:
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A rapid due diligence review of targets
Implications and path forward for gen- tox issue to support out-license
Review and selection of appropriate animal models
Go / No-Go review of existing efficacy data
Manufacturability & scalability of synthetic routes
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Scale-up and Sourcing Decisions
What we do and how it’s different:
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Capitalize on multiple advisors experience in development, sourcing, and vendor
management
Rapid identification of technical requirements, identification of possible vendors,
screening & qualification, support development of contracting, quality
agreements etc.
Drug Substance/API, Intermediates, Formulation, Drug Product, Fill-Finish
Examples of Recent Projects:
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Identified and qualified sources for hard-to-locate emulsion capability in Europe
Identified and qualified US sources of peptide manufacture for a European
company
API Development, Scale-Up and route filing strategy
Scalability / Manufacturability review
Vendor Management Strategy and Process for an emerging large Biotech
Identification and Qualification of Controlled Release Drug Product
manufacturers for an established Biotech
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Integrated Development View
The Core of Advisors’ Approach
• Leverage understanding of the interrelationship between CMC,
Clinical and Safety development to create and protect value
• Multi-company and cross-functional perspectives
• Insight-driven anticipation of Advisors Functional Focus issues
for improved risk management
• Advisors who have been accountable for all aspects of
development
• Application to specific issues and overall program management
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Portfolio Decisions,
Due Diligence and Valuation
What we do and how it’s different:
• Concurrent technical and business perspective
• Portfolio management decision experience across a wide range
of technologies and therapeutic areas
• Rapid inclusion of focused subject matter expertise rather than
generalist research
Examples of recent projects:
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Due Diligence of compounds in various therapeutic areas
Due Diligence of company business models
Valuation of compounds in support of out-licensing decision
Assembly of a “ Virtual Pharmaceutical Company” team of users
to help a supplier understand the value of their technologies
resulting in successful new business strategy
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