The Journey from Branded Generic Company to CNS Specialty Pharma

1
The Journey from Branded Generic Company to CNS
Specialty Pharma

• Beth A-S. Brown, Ph.D., R.Ph., Director,
  Pharmaceutical Development

2
Agenda

• Overview of Upsher-Smith Laboratories
• Strategic Vision in CNS
• USL/Proximagen Partnership

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Overview of Upsher-Smith Laboratories
4
USL Locations
5
A Brief Historical Timeline
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An Integrated Pharmaceutical Company

• Pre-Clinical Development
• Pharmaceutical Sciences Drug Delivery
• Project Management
• Clinical Research
• Drug Safety
• Regulatory Affairs
• Corporate Compliance QC/QA
• Manufacturing Packaging
• Medical Education Communication
• Sales Marketing

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Established Brand Market Leader

• Klor-Con
• Slo-Niacin
• Pacerone
• Clenia
• Jantoven
• Fortical
• Amlactin
• Vandazole
• Divigel
• PreNexa

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Strategic Vision in CNS
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Vision Statement
To become a leader in providing therapies that
empower people suffering from central nervous
system (CNS) diseases to lead healthy, productive
lives
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Strategic Intent

• Focus
• Innovative, NDA products within key therapeutic
  categories
• Epilepsy
• Parkinsons disease
• Strategy
• Building a CNS pipeline via three complementary
  strategies
• Alliances and co-development agreements
• Internally developed programs that leverage core
  competencies
• Licensing and acquisitions

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USL/Proximagen Partnership
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• PROXIMAGEN SIGNS 232 MILLION LICENSING AGREEMENT
  WITH UPSHER-SMITH TO
• DEVELOP PRX1 PROGRAMME

London, UK - Proximagen Neuroscience plc (AIM
PRX), the drug discovery and development company
focused on neurodegenerative diseases, is today
pleased to announce it has entered into a
worldwide licensing agreement ("the Agreement")
with Upsher-Smith Laboratories Inc.
("Upsher-Smith"), the Minnesota-based company
which develops, manufactures and markets
pharmaceuticals in a number of therapeutic areas.
The Agreement covers the development and
commercialisation of Proximagen's proprietary
PRX1 programme for the symptomatic treatment of
Parkinson's disease (PD). Proximagen will receive
an upfront payment and milestone payments
totalling up to 232 million (117 million),
plus royalties on global product sales.
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Unmet Needs in Parkinsons Disease Therapy
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The Gold Standard Treatment L-DOPA Plasma
Concentrations at Typical Therapeutic Dose
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Partnership Roles for Discovery Phase

• Proximagen
• Design the molecules
• Pharmacodynamic models
• Upsher-Smith Laboratories (PharmSci)
• Preformulation
• Form screening and selection
• Formulation for FIH
• Shared
• Preclinical work (IND-enabling)
• Manage contract API manufacture

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Hit to Lead Process
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Desired PK Profile
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Desired PD Profile MPTP Model
Brain (2008), 131, 3380-3394
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Preliminary CMC Screen

• Solution Stability
• Solid State Stability

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Developability Screen

• Form Screening and Selection
• Free base
• Salt
• Co-crystal
• Physicochemical Properties
• pKa, logP
• Water Uptake
• Thermal Analysis
• Physical form(s)
• Solubility
• Solution and Solid-State Stability

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Summary

• Upsher-Smith Laboratories is progressing the
  development of a CNS portfolio of products
• Creation of business and technical processes to
  support the transition
• Leveraging and continued development of internal
  competency and external capacity
• Building a CNS pipeline through co-development
  partnerships, internal development, and licensing
  activities

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Acknowledgments

• Steve Berge Pharmaceutical Sciences
• Steve Elrod Technical Leadership
• Chris Evenstad - Marketing
• Rich Fisher Strategic Portfolio Management
• Lori Freese Strategic Portfolio
  ManagementLeslie Helou Strategic Portfolio
  Management
• Mike McBride Business Development
• Brent Miller Pharmaceutical Sciences
• Troy Windt Business Development
• Roger Zanon Pharmaceutical Sciences

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