PATIENT GROUPS & CLINICAL TRIALS CASE STUDYJeffrey W. Sherman, M.D., FACP

Past President – Inaugural Fellow Chief Medical Officer – Executive Vice President

February 1, 2017

DisclaimerThe views and opinions expressed in this presentation are those of the individual presenter and do not necessarily reflect the views of their employer, professional association affiliation, or Clinical Trials Transformation Initiative.

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Why Adopt CTTI Recommendations?Expedite development of potential products for unmet medical needs Over 7000 rare diseases of which only 5% have

treatments. Patient groups have the knowledge of the disease to expedite clinical trials

Best to start engagement as early as possible Facilitate planning and implementation of development

activities

Continue engagement throughout the R&D continuum Greatly increases chances for success (new treatment) Saves time, money, and resources - ROE

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Patient Groups & Clinical TrialsQuestions to be Answered

How did you operationalize engagement?

What were challenges?

How did you manage the challenges?

What were specific successes?

How are you applying your learning from this experience to other engagement opportunities?

Were there things that didn’t work well that you won’t try to replicate, or modify a future approach?

Key Consideration – Patients Come First

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Drug Information Association -Fostering Innovation to Improve Health and Well-Being WorldwideBringing health care product development professionals together in a trusted, global, neutral environment to share insights and drive action in health care product development and life cycle management Academia Government Regulators Industry Patient Groups Payers

Over a 50-year history of driving collaboration worldwide as a non-profit professional association

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Horizon PharmaFocused Commitment to Community

Four PillarsTherapeutic Areas STEM/Education Innovation Children’s Healthcare

Company Confidential – Not for Distribution

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Pharmaceutical Interferon ProductsInterferon (IFNs) – Proteins made and released by host cells in an immune response to the presence of pathogens such as viruses, bacteria, parasites, or tumor cells.

Treatment targets appear to not overlap• Hepatitis C (soon to be historical use as non-interferon agents continue to emerge)

• Interferon alpha 2a - Roferon A• Pegylated Interferon alpha 2a - Pegasys• Interferon alpha 2b - Intron A• Pegylated Interferon alpha 2b - PegIntron

• Multiple Sclerosis• Interferon beta 1a - Rebif (liquid); Avonex (lyophilized)• Interferon beta 1b - Betaseron

‒ Chronic Granulomatous Disease (CGD); Severe Malignant Osteopetrosis (SMO)• Interferon gamma 1b ‐ ACTIMMUNE®

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ACTIMMUNE® (Interferon gamma-1b)Orphan Diseases – Modulating Genetic Deficiencies

Chronic GranulomatousDisease (CGD)

Severe Malignant Osteopetrosis

(SMO)Friedreich’s ataxia

(FA)

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Why FARA?Preclinical Models of Disease

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Why FARA?Clinical Trial Infrastructure

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FARA and Horizon EmbracingBest Practices in Effective Engagement

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ACTIMMUNE Phase 3 FA Clinical StudyFARA and Horizon

Randomized, Multicenter, Double-Blind, Placebo-Controlled, Efficacy, Safety, and Pharmacokinetic Study of ACTIMMUNE® (interferon γ-1b) in Children and

Young Adults with Friedreich’s Ataxia

Short title:

Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich’s Ataxia Study

Clinicaltrials.gov: NCT0241512715

Patient Groups & Clinical TrialsQuestions Answered – Patients Come First

How did you operationalize engagement? Follow best practices – Make engagement a priority

What were challenges? Minimize challenges by building trust upfront for a long-term relationship

How did you manage the challenges? Have frequent interactions particularly early on

What were specific successes? Completed enrollment in a Phase 3 trial on time and with no screen failures

How are you applying your learning from this experience to other engagement opportunities? Apply the same success factors as above

Were there things that didn’t work well that you won’t try to replicate, or modify a future approach? Important again to build trust upfront

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Industry & Patient Group Interactions:Participate, Listen, and Learn

RideAtaxia IllinoisRideAtaxia Atlanta

NORD Rare Impact Awards