AFREZZA - cdn.ymaws.com › › resource › co… · Analysis Questions Outside In Market Competition Technology Stakeholders Inside Out ... Global Attitudes of Patients and Physicians

©2014 MannKind Corporation. All rights reserved. No copying or distribution of this material may be made without written consent of MannKind Corporation. 1

AFREZZA®

A Journey of Strategic, Regulatory & Commercial Challenges

- Hakan Edstrom

President, COO & Director

May 13, 2014


Our focus is patients,our passion is innovation®

Nasdaq: MNKD

3©2014 MannKind Corporation. All rights reserved. No copying or distribution of this material may be made without written consent of MannKind Corporation.

The first and only ultra rapid-acting insulin that

closely mimics natural physiologic insulin kinetics


THE AFREZZA® JOURNEY

Strategic Planning in the Face of a Regulated Environment


Situation Analysis

The Issue What are we going to consider and why?

Reporting What do we know about the “Issue”?

History?

Current Situation?

Future Expectations?


The Issue

Process Questions What are we going to consider and make decisions about?

What are our priorities?

What are we not doing?

Analysis Questions What decisions have been made?

What is the most important situation we are facing?

What is the greatest opportunity?

What is the greatest challenge?

What do we want to accomplish?

Who is responsible?


MannKind Business History

Oncology – CTL Immunotherapie’s

Allergy – Allecure

Diabetes – PDC - Danbury


MannKind Diabetes

Business Rationale

Significant Market Opportunity

Significant Unmet Needs

Disease Area Experience & History

Business Insight

Market Credibility

Funding Opportunities

Clear Purpose


Reporting

Process Questions What has happened?

Where are we today?

What do we believe about the future?

Analysis Questions Outside In

Market

Competition

Technology

Stakeholders

Inside Out

Our product and services

Competitive situation

Financials

Our future opportunity and why?


OUTSIDE IN & INSIDE OUT

We must think and act Outside In and Inside Out


Outside In

Opportunities


Global EpidemicPrevalence of Diabetes 2013-2035

Source: International Diabetes Federation Diabetes Atlas, 2013

36.7

50.4

24.1

38.5

56.3

68.9

19.8

41.4

72.1

123.0

201.8

138.2

34.6

67.9

2013

2035


The Number of Patients on Insulin Continues to Increase

Source: 2013 Roper US Diabetes Patient Market Study (Gfk Healthcare 2013)


CURRENT INSULIN

TREATMENTS

Long duration of action

risks hypoglycemia

Hypoglycemia fear may

lead to poor compliance

and/or disease management

Late onset of action

leads to poor PPG control

Undesired weight gain

Need for injection

AFREZZA ADVANTAGES

Less risk of hypoglycemia

PPG control

Less weight gain

No need for injection

Short duration of action

AFREZZA Addresses Current Insulin Treatment Challenges


Physicians Confirm Need for A Faster Insulin

“We need insulins that work faster than current

rapid-acting analogs.”

94%

4%

0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

% o

f Phys

icia

ns

Source: Close Concerns, Diabetes Close Up; HCP Survey ADA 2010

Yes No


Outside In

Challenges

Changing Health Care Environment

The Obama Health Care Plan

Pricing & Reimbursement Challenges

Pharmaceutical Pricing and Reimbursement Process in US, Europe,

Japan, Australia and China

New Improved Treatment Options

DPP4 - Inhibitors

SGLT2 - Inhibitors

GLP1 - Agonists

Oligopolistic Insulin Market

Novo

Sanofi

Lilly

Obama Care


Inside Out

New Technology

Clinical Benefits


AFREZZA Product Description

pH < 6

FDKP Particle Formation

Insulin Adsorption Onto Particle

pH > 6

Rapid Dissolution

Insulin + FDKP


Recent survey revealed 1/3 of patients fail to take insulin as prescribed.

67% of patients are concerned about hypoglycemia events.

74% of physicians would treat to target if no fear of hypoglycemia events.

Simplicity and ease of use = Better Compliance

Source: Global Attitudes of Patients and Physicians in Insulin Therapy (GAPP) Survey, Novo Nordisk, 2010


Inside Out

Clinical Benefits

Preferred Patient Experience

Less Hypoglycemia

Less/No Weight Gain

Better Glucose Control

Better Compliance


Small, discreet, easy-to-use inhaler

No injection required

No cleaning required

Breath powered

Efficient delivery to deep lung

Minimal training needed

Disposed after 15 days of use

Preferred Patient Experience


One-third of patients failed to take insulin as prescribed

Insulin Treatment Challenges:

‒ 67% patients concerned about hypoglycemia

‒ 74% physicians would treat to target if no fear of hypoglycemia events

Limitations of current insulin regimens include:

‒ Hypoglycemia

‒ Slow Absorption

‒ Weight Gain

‒ Inconvenience of Injections

Current Insulin Treatment Challenges

Survey: Global Attitudes of Patients and Physicians in Insulin Therapy

Source: GAPP Survey (Global Attitudes of Patients and Physicians in Insulin Therapy), 2010, Novo Nordisk.


Conclusion

Having carefully penetrated the “Issue” and as

part of the “Reported” findings, we determined

that there are unmet needs that the company

had the technologies to address. The next

step was to begin the Strategic Planning

Process.


MannKind Strategic Planning Model

Objectives

Activities

Personnel & Organization

Reporting

Success

Factors

Purpose

Process Steps

Process Questions

Analysis Questions


Process Question What will we do for whom and why?

Analysis Questions Who will use our product and why?

Who decides the need of our product?

Who pays for the product?

Who are the key stakeholders and what are their needs?

What businesses are we running today?

What business will we run tomorrow?

What part of our business will we carry out with partners?

Purpose


Success factors are what we choose to be recognized by, in order

to be appreciated and successful compared to alternative choices.

Process Question How will we be recognized in order to be successful?

Analysis Questions What decision criteria will customers apply?

What are the alternatives?

What are our SWOT’s?

What are the emotional decision criteria?

What are the regulatory requirements or impediments?

What is the cost involved?

How will it be reimbursed?

Success Factors


Excellent Control Less Risk

AFREZZA®Changing the Way Diabetes is Treated

Ideal PK/PD Profile

Meal synchronization

Simple, easy to use,

patient-friendly

More Physiologic

Less Hypoglycemia,

Less Weight Gain

Improved

compliance


Objectives are a “State of Affairs” or a position we intend to be in

by a particular time.

The can be Long-Term Strategic Objectives or Short Term

Operational Objectives.

Process Question What are we going to achieve over the long and the short

terms?

Analysis Questions

Long Term - Short Term -

What is our vision? What do we mean by Short Term?

How do we define Long Term? What is the budget?

What do our customers expect? What is realistic?

What ROI do we plan on?

Objectives


Strategic Objectives Start of Phase 3 Trials Q2/2006

FDA Submission Q3/2009

Partnership Q1/2010

FDA Approval Q2/2010

Commercialization Q1/2011

Operational Objectives Company Structure and Management 2005

IPO Q3/2007

Insulin Long Term Supply Contract 2003

Product Portfolio 2003

Organizational Structure Q4/2002

Planning Objectives


Regulatory Objectives

March 2009 – NDA Submission (with MedTone)

• Four efficacy studies: type 1 - 009, type 2 – 014, 102, 103

• One long-term safety study: 030

March 2010 – Complete Response Letter

• Human factors validation

• Clinical utility

June 2010 – Resubmission

• MedTone – Gen2 bioequivalence

• Study 117 – type 1

January 2011 – Complete Response Letter

• Cannot bridge devices based on in vitro performance

data and clinical pharmacology

October 2013 – Resubmission (with Gen2)

• Two efficacy studies: type 1 - 171, type 2 – 175

• Ad Com

• PDUFA July 15th, 2014

Gen2 Inhaler

MedTone Inhaler

Bioequivalence


Secure Insulin Supply, API

Build Manufacturing Capability / Capacity

Source Commercial FDKP

Begin Partnering Due Diligence

Build IP Estate

Afrezza Business Objectives


All T1 Patients

12%

All T2 Patients

13%

T2: Insulin

Other (i.e., NPH,

pre-mix)

20%

T2: Long-acting

(only insulin)

18%

T2: Rapid-

acting insulin

(any regimen)

18%

T2: Insulin Naïve

9%

Q21.In the list below, please indicate whether the following medications are part of this patient’s current anti-diabetes regimen.

Q32.For the patient(s) listed below, you indicated you would change to a regimen that includes Product X. We are very interested in how the current regimen for each patient will change with the addition of Product X?

Please indicate below which other treatment(s) in addition to Product X will be part of the overall anti-diabetic drug regimen for each patient

T1 Patients

(% of patients)T2 Patients (% of patients)

18% share in

insulin experienced

Patient Level Data

Summary of AFREZZA Share Across Patient Types

Source: US Opportunity Assessment (2013 Blue Print Research Group)


Conservative Market Penetration Yields Significant Revenues

Forecast assumes peak penetration of 8% for type 1 and 5% in type 2

2015 2020 2025

US Net Revenue ($mm) (1) $ 294 $ 2,733 $ 4,951

Type 1 Net Revenue ($mm) $ 28 $ 225 $ 366

Type 2 Net Revenue ($mm) $ 266 $ 2,509 $ 4,585

Type 1 AFREZZ Market Penetration:

AFREZZA / Treated Patients 0.9% 5.6% 7.9%

Type 2 AFREZZ Market Penetration:

AFREZZA / Treated Patients 0.5% 3.5% 4.9%

AFREZZA / Insulin Treated Patients 1.7% 9.9% 13.5%

(1) Assumes 3% price increases annually from 2014 through 2025


Multiple Patent Families

>200 issued utility patents worldwide

24 of these are U.S. Orange Book listable patents

>40 issued design patents

>300 pending patent applications worldwide

Categories of direct protection of AFREZZA:

Formulation technology, including optimized particles

Inhalation Systems

Methods of Treatment

Additional applications relate to manufacture and

testing, and to training patients to use inhaler


Process Questions What are we going to continue doing, begin doing and stop

doing to achieve our objectives?

What are the financial consequences?

Analysis Questions How will we structure our activities?

When and how will we perform the activity?

Who will be responsible for the activity?

What will we do within each activity?

What will the financial consequences be, cost and revenue?

Activities


Afrezza Key Activities Organizational Layout Structure & Management

Pre-Clinical Studies Safety Focus

Clinical Trials Safety & Efficacy

Manufacturing Build-Out Technology Decision

IPO Preparation Funding

Regulatory Application NDA

I.P. Asset Management & Preservation

Partnership Planning

Regulatory Submission

Commercial Planning

Activities


Based on the issue and reporting within the set purpose, the chosen

success factors, the set objectives and the activities we must make

decisions about relevant personnel and organizational structure.

Process Questions Who will be responsible for what and how will we interact?

How do we recruit the right competence?

Analysis Questions What competence do we have in-house?

How is reporting organized?

What authority will be delegated?

What is included in each area of responsibility?

How do we interact?

What will our Culture be? (values, morals, ethics)



Company Structure

Danbury, Connecticut - Technical Operations Team and the following functions:

• Manufacturing

• Engineering

• Quality Assurance & Quality Control

Paramus, New Jersey - Clinical Research & Development Groups and the following functions:

• Clinical Assessment & Quality Systems Management

• Biometrics and Data Management

• Clinical Outsourcing Regulatory Affairs and Compliance

• Marketing Business Development

• Supply Chain & Procurement

Valencia, California - Headquarters and the following functions:

• Drug Discovery

• Pharmacology & Toxicology

• Analytical & Exploratory Formulation/Development

• Molecular & Cell Biology, Peptides, and Translational Medicine


Process Question What must we follow up on and how, in order to achieve our

objectives?

Analysis Questions What?

When?

How?

By Whom?

To Whom?

Reporting


Org Chart

Alfred MannChairman

Chief Executive OfficerMannKind Corporation

Hakan EdstromPresident

Chief Operating OfficerMannKind Corporation

Juergen MartensCorporate Vice President

Pharmaceutical Development

Technical Operations540

(Danbury)

Diane PalumboCorporate Vice President

Human Resources717

Matt Pfeffer Corporate Vice President

& CFO705

David Thomson Corporate VP & General

Counsel727

Hakan EdstromActing Chief

Commercial Officer[740]


AFREZZA® TAKEAWAYS


AFREZZA® Takeaways

• Large and growing patient population

• Poorly met medical need

Blockbuster potential

• Ultra rapid-acting insulin that mimics physiology

• Offers glycemic control without increased risk of hypoglycemia

and weight gain

• Delivered through discreet and easy-to-use inhaler

A new class of insulin therapy

• FDA confirmation of protocols using new, advanced inhaler

• Completed two clinical trials – achieved primary endpoints

PDUFA date: July 15, 2014


Danbury, CT facility dedicated to manufacturing, engineering, quality assurance and quality control.

328,000 sq.ft.

Set to produce AFFREZZA® for up to 450,000 patients at launch.

Additional capacity can be brought online at facility to increase production to 2 million patients.

Production Capabilities


Product Image –Commercial Pack


MannKind Strategic Planning Model

Objectives

Activities


Reporting

Success

Factors

Purpose

Process Steps

Process Questions

Analysis Questions


The first and only ultra rapid-acting insulin

Documents

AFREZZA - cdn.ymaws.com › › resource › co… · Analysis Questions Outside In Market Competition Technology Stakeholders Inside Out ... Global Attitudes of Patients and Physicians