Entrepreneurial Track, “Developing Your Product”

Target Product Profiles Medical & Development Considerations and Challenges

Barbara Wirostko, M.D.,Jade Therapeutics, Inc2013 BioUtah Life Science SymposiumNovember 6, 2013

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• Co Founder and Chief Scientific Officer of Jade

Therapeutics, Inco Based at Echelon, University of Utah Research Park, Salt

lake City

• Adjunct Clinical Associate Professor; Moran Eye

Center, University of Utah, Salt Lake City, Utah

• Consult for various drug delivery companies

focused in ophthalmology

Disclosures

• Medical need in Ophthalmology

• What is a Target Product Profile (TPP)?

• What is the purpose of the TPP?

• How did we use it?

Objectives

• Chronic diseases + aging population = attractive area to be in

• Aging population o chronic ocular pathologies: glaucoma, diabetic retinopathy, and

macular degeneration,

o many require chronic therapy

• Daily self application is difficult

• The ability to develop new drugs and deliver them through

novel technology is attractive

Recognized unmet medical need

Ophthalmology – $ 23 Billion

A lot of different deliveries in development

Topical eye drops

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This can’t always be easy….

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• Where is the unmet need?

• What is the unmet need?

• Confirm it….

• Nail it then Scale it. The Entrepreneur’s Guide to Creating and

Managing Breakthrough Innovation• Nathan Furr & Paul Ahlstrom

o “Entrepreneurs don’t necessarily invent – they innovate”

o Go out into the field

o Don’t propose the solution

o BUT ask the questions

• Is there a need?• Could we solve it ?• What would the solutions look like?• Would this solution work?

Identify the Unmet Need

• The Target Product Profile (TPP) is the cornerstone

of pharmaceutical product development.o Represents the envisioned product

o Think about what will the eventual label be

• Help guide development to get to this label• Living document

Help you to fulfill that unmet need of the

customer!!

Develop the TPP

• Traditional TPPs have served as an R & D tool used to

guide the clinical program

o Patient population

o Endpoints

o Safety and efficacy

o Phase 3 comparator

Also serves as a business tool for determining asset

value and capital commitment.

Target Product profile for XXXcompound

compound

Active Ingredient

Indication

Target population

Mechanism of Action

Dosage

Route of Administration

Safety

Efficacy

Competitive Advantage over Other Therapies

Jade’s unmet need: Sustained Delivery of Antibiotics for Bacterial Keratitis

A corneal-blinding condition (scarring/perforation)

Most common cause in US is contact lens wear

Standard of care is topical antibiotics

dosed as often as hourly

No marketed/ approved drug product in U.S. Likely orphan drug indication

U.S. Army interest

NSF Phase I SBIR funded grant

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• No current eye drop for this indicationo Off label use and or compounding of approved antibiotics

• Difficult to administer topical drops every hour x 2-3

days

• Rapidly blinding condition

o Ocular emergency

• Compliance and Adherence are NOT GOOD

o Poor compliance with topical drops

Confirmed with end users & customers

Is there an unmet need?

Initial Target Product Profile(s)

4-7 day drug-eluting strip/film Use of approved ocular antibiotics to treat bacterial keratitis

(also known as corneal ulcers)

Applied in-office by M.D.

Local topical placement Inferior Cul de sac

under bandage contact lens

Improved efficacy and safety over

“Off label” compounded drops (SOC)

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Key Messages

Focus on an attractive area Ophthalmology

Orphan diseases

Address a critical unmet need Increased compliance/drug exposure Improved outcomes

Mitigate risk Focus on local administration of already-approved drugs (505(b)2)

Proprietary polymer-based platform for novel delivery (strong IP)15

Thank You!

“It is Fun to do the impossible”

Walt Disney

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