“Innovation in the Biopharmaceutical

Pipeline: A Multidimensional View”

John Jeffrey JonesRegis University Class of 2016

jjones004@regis.edu

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Topics• Continuing Need for New Medicines• R&D Pipeline

• Total Number of Medicines in Development, by Therapeutic Area

• Potential First-in-Class Medicines in Development• Orphan Diseases• Therapies Targeting Diseases with No Recently

Approved Therapies• Personalized Medicines in Development• Novel Scientific Strategies

• Conclusion

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Introductiono U.S. leads the world in development

of new medicines

o Improving the length and QOL and reduced disease burden for individuals and society

o Focus: NME Clinical testing stage in human volunteers

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Challenges of Drug Discovery

• Long/Complex Process – risk of failure at each step

• $1.2 billion cost to yield a single FDA-approved drug (includes cost of development failures)1

• 10-15 year span from R&D + FDA approval process time2

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Annual & Cumulative New Drug Approvals Since 20003

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Distribution of Products & Projects by Phase3

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Distribution of Products and Projects by Therapeutic

Area/Phase3

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Potential First-in-Class Medicines in Development, by Phase3

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Potential First-in-Class Medicines in Development, by

Therapeutic Area3

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Spotlight: DM II & Schizophrenia3

New Approach to Controlling Type II

Diabetes

Difficult-to-Treat Symptoms of

Schizophrenia

O Compound that controls blood sugar levels, or glycemia, independent of insulin pathways.

O Sodium glucose cotransporter-2 inhibitor

O Glycine reuptake inhibitor – could help normalize transmission of glutamate

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Orphan Disease Designation by the FDA Over Time3

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Spotlight: Idiopathic Pulmonary Fibrosis (IPF) 3

O Patients experience progressive difficulty breathing due to scarring of the lungs.

O There are currently no effective treatment options available, and the average patient with IPF dies within 3 years of diagnosis.

O A medicine in development targets connective tissue growth factor, which is elevated in the lungs of IPF patients.

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Projects for Selected Diseases/Conditions with No

Approvals in 10 Years3

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Spotlight on Therapies Targeting Diseases with No Recently Approved Therapies: Recent Progress

Against Lupus3

O Lupus: Complex, potentially fatal autoimmune disease that attacks healthy tissues from MULTIPLE organ systems

O At least 7 drugs in the last several years have suffered setbacks in clinical trials

O As of December 2011, there were 58 other projects in development to treat lupus or various aspects of lupus such as serious kidney complications.

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Personalized Medicine Trials by Condition & Therapeutic Area, 1993-

20083

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Distribution of Personalized Medicine Clinical Trials by

Therapeutic Area & Growth Over Time, 1993 - 20083

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Spotlight: Melanoma, Lung Cancer, & Cystic Fibrosis3

O Vemurafenib (Zelboraf®) – Melanoma

O Crizotinib (Xalkori®) - Lung cancer

O Ivacaftor (Kalydeco®) - Cystic fibrosis

O Each is indicated for patients expressing a specific genetic mutation where the drug is known to be effective.

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Examples of Selected Breakthrough Scientific Strategies3

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Spotlight: RNAi & Gene Therapy3

RNAi Targeted Therapy Approach for Duchenne

Muscular Dystrophy (DMD)

Gene Therapy as a Possible Approach for Parkinson’s Disease

O DMD: most severe genetic disorderO Affects 1 in 3,500

newborn boysO targeted

restoration of the function of dystrophin

O Use of adeno-associated virus (AAV) as a vector to deliver neurturin to restore cells; protection from further degeneration

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SummaryO Diverse clinical research programs

across many different therapeutic areas

O New treatments address both common conditions like cardiovascular disease and rare diseases like cystic fibrosis

O New hope for current and future patients

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ReferencesO 1. J.A. DiMasi and H.G. Grabowski. (2007) The

cost of biopharmaceutical R&D: is biotech different?. Managerial & Decision Economics. 28:469-479.

O 2. DiMasi, J.A., Hansen, R.W., and Grabowski, H.G. (2003) The price of innovation: new estimates of drug development costs. Journal of Health Economics. 22:151-185.

O 3. G. Long and J. Works. (2013) Innovation in the Biopharmaceutical Pipeline: A Multidimensional View. Analysis Group, Inc. 1-29.