QUESTCOR PHARMACEUTICALS INC - FORM 8-K - EX-99.1

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8-K - 8-K - QUESTCOR PHARMACEUTICALS INC	d658915d8k.htm

NASDAQ:

QCOR

J.P. Morgan

Healthcare Conference

January 16, 2014

Exhibit 99.1

Safe Harbor Statement

Note: Except for the historical information contained herein, this press release contains forward-looking statements that have been made pursuant to the

Private Securities Litigation Reform Act of 1995. These statements relate to future events or our future financial performance. These statements are only

predictions. Actual events or results may differ materially. Factors that could cause or contribute to such differences include, but are not limited to, the

following: Our reliance on Acthar for substantially all of our net sales and profits; Reductions in vials used per prescription resulting from changes in

treatment regimens by physicians or patient compliance with physician recommendations; Our ability to receive high reimbursement levels from third party

payers; The complex nature of our manufacturing process and the potential for supply disruptions or other business disruptions; The lack of patent

protection for Acthar and the possible FDA approval and market introduction of competitive products; Our ability to continue to generate revenue from

sales of Acthar to treat on-label indications associated with NS, MS, IS or rheumatology-related conditions, and our ability to develop other therapeutic

uses for Acthar; Research and development risks, including risks associated with Questcor's work in the area of NS and Lupus, our efforts to develop and

obtain FDA approval of Synacthen, our reliance on third-parties to conduct research and development and the ability of research and development to

generate successful results; The results of any pending or future litigation, investigations or claims, including government investigations and private

securities litigation; Our ability to comply with federal and state regulations, including regulations relating to pharmaceutical sales and marketing practices;

Regulatory changes or other policy actions by governmental authorities and other third parties in connection with U.S. health care reform or efforts to

reduce federal and state government deficits; An increase in the proportion of our Acthar unit sales comprised of Medicaid-eligible patients and

government entities; Our ability to estimate reserves required for Acthar used by government entities and Medicaid-eligible patients and the impact that

unforeseen invoicing of historical Medicaid prescriptions may have upon our results; Our ability to effectively manage our growth, including the expansion

of our sales forces, planned international expansion, and our reliance on key personnel; Our ability to integrate the BioVectra business with our business

and to manage, and grow, a contract manufacturing business; Our ability to comply with foreign regulations related to the operating of BioVectra's

business and the international sales of Synacthen; The impact to our business caused by economic conditions; Our ability to protect our proprietary rights;

The risk of product liability lawsuits; Our ability to successfully enter into, and operate in, international markets; The risk of unfavorable changes in currency

exchange rates; Unforeseen business interruptions and security breaches; Volatility in Questcor's Acthar shipments, estimated channel inventory, and

end-user demand, as well as volatility in our stock price; Our ability and willingness to continue to pay our quarterly dividend or make future increases in

our quarterly dividend; and Other risks discussed in Questcor's annual report on Form 10-K for the year ended December 31, 2012 as filed with the

Securities and Exchange Commission, or SEC, on February 27, 2013, and other documents filed with the SEC.

The risk factors and other information contained in these documents should be considered in evaluating Questcor's prospects

and future financial performance.

Questcor

A biopharmaceutical company focused on the treatment

of patients with serious, difficult-to-treat autoimmune

and inflammatory disorders

Flagship product H.P. Acthar Gel has a unique

therapeutic role and sustainable competitive advantages

Acthar is approved for 19 indications, many in markets

with sizable unmet need

Market penetration remains modest;

sales have increased rapidly

Increasing investment in R&D to grow the

body of evidence and diversify

Profitable, strong cash flow and balance sheet; returned

cash to shareholders through buybacks and dividends

Investment Highlights

3-Year Net Sales

Quarterly Net Sales ($M)

$29.3

$46.0

$75.5

$112.5

$160.5

$184.6

$236.3

$31.3

$36.8

$59.8

$96.0

$140.3

$135.1

Q3 '10

Q4 '10

Q1 '11

Q2 '11

Q3 '11

Q4 '11

Q1 '12

Q2 '12

Q3 '12

Q4 '12

Q1 '13

Q2 '13

Q3 '13

Acthar Business Diversification Continues

Rheumatology

20%-25%

Nephrology

30%-35%

Infantile Spasms

7%-12%

Other

~2%

25%-30%

Acthar Estimated % Net Sales by

Therapeutic Area (Q3 2013)

Net Sales

($M)

TTM 2013: Trailing twelve months through September 30, 2013

$88

$115

$218

$509

$717

2009

2010

2011

2012

TTM 2013

Expanding Acthar Sales

Across Therapeutic Areas

Acthar Net Sales ($M)

$88

$115

$218

$509

$717

*Numbers

the

bar

graph

represent

actual

Acthar

net

sales.

The

underlying

allocation

Acthar

net

sales

based

Management’s internal estimates;

TTM 2013: Trailing twelve months through September 30, 2013

Infantile Spasms

Multiple Sclerosis

Nephrology

Rheumology

Other

2009

2010

2011

2012

TTM 2013

Net Income Growth

Non-GAAP

Net

Income

GAAP

Net

Income

TTM

2013:

Trailing

twelve

months

through

September

30,

2013;

See

reconciliation

table

Appendix

CAGR

77%

CAGR

79%

$27

$35

$80

$198

$265

$29

$39

$84

$210

$299

2009

2010

2011

2012

TTM 2013

2009

2010

2011

2012

TTM 2013

($M)

Earnings Growth

Non-GAAP Diluted EPS

GAAP Diluted EPS

TTM

2013:

Trailing

twelve

months

through

September

30,

2013;

See

reconciliation

table

Appendix

CAGR

81%

CAGR

83%

2009

2010

2011

2012

TTM 2013

$0.40

$0.54

$1.21

$3.14

$4.34

2009

2010

2011

2012

TTM 2013

$0.44

$0.60

$1.27

$3.33

$4.89

Q3-2013 Financial Results

* Includes $75 million in restricted cash. See reconciliation table in Appendix

Q3 –

2012

Change

Net Sales ($M)

$140.3

68%

Fully Diluted, GAAP EPS

$1.52

$0.91

$1.68

$0.97

73%

Investments ($M)

$281.1*

Cash Flow from Operations ($M)

$108.9

Diluted Shares Outstanding

62.1

61.4

Fully Diluted, Non-GAAP EPS

Cash and Short Term

$236.3

Q3 –

2013

$111.9

$51.2

67%

Recent Updates and Trends

Total Paid Acthar Rxs

•

$0.30 Dividend declared Q413

–

Record date: 1/17/14

–

Distribution date: 1/24/14

•

960K shares repurchased in Q413

•

$320M in Cash and ST Investments*

•

Board level Science and Strategic

Advisory Committees formed

–

To assist in ongoing evaluation of

factors contributing to the persistent

and significant discount in the

Company’s valuation relative to its

peer companies

–

Will also assess potential strategic

alternatives to address these

factors

*As of December 31, 2013 (includes $75M of restricted cash)

500

1,000

1,500

2,000

2,500

3,000

Q3-11

Q4-11

Q1-12

Q2-12

Q3-12

Q4-12

Q1-13

Q2-13

Q3-13

Q4-13

Flagship Product:

•

19 approved indications

Key Therapeutic Areas:

•

Nephrotic Syndrome, Multiple Sclerosis Relapse, Infantile Spasms,

Rheumatology Indications, Symptomatic Sarcoidosis

•

Significant areas of unmet need; large growth potential

Strategy:

•

Expand awareness, appropriate use of Acthar in key specialties

•

Develop Rheumatology and other on-label indications

Acthar Overview

*In

this

presentation,

the

terms

“Nephrotic

Syndrome,”

“Multiple

Sclerosis

Relapse,”

“Infantile

Spasms,”

“Rheumatology

Indications,”

and

“Symptomatic

Sarcoidosis,”

and

their

abbreviations,

refer

on-label

indications

for

Acthar

associated

with

such

conditions.

Investors should refer to the FDA approved Acthar label, which can be found at http://www.acthar.com/files/Acthar-PI.pdf

Nephrotic Syndrome

iMN, FSGS,

IgA, LN,

MPGN, MCD

Sub-Nephrotic

~90,000

Nephrotic

Population

~35K

~35,000

Addressable NS Patients

(US)

•

Characterized by excessive spilling of

protein from the kidneys into the urine

(nephrotic-range proteinuria)

•

Caused by a number of underlying

types of kidney disease

(eg, iMN, FSGS, IgA nephropathy, etc.)

•

Can result in end-stage renal disease

(ESRD), dialysis, transplant

•

Significant unmet need;

few treatment options

Multiple Sclerosis (MS) Relapse

Adequately

Treated Relapses

~70K

Addressable

Patient

Population

~30K

•

MS is a neurodegenerative disease

occurring in about 400,000 patients

in the US

•

Estimated >100,000 relapses/year

•

Relapses range from mild to severe

and can cause a range of symptoms

–

Loss of sensation in the extremities

–

Loss of vision

–

Loss of ability to walk

•

Relapses can have a measurable

and sustained effect on disability in

MS patients

~30,000 Addressable

Annual Relapses (US)

•

Devastating, ultra-rare form of childhood epilepsy

•

Can cause permanent developmental disabilities, increased mortality

•

Acthar

often

considered

the

“gold

standard”

and

currently

used to treat ~50% of IS patients

Infantile Spasms (IS)

Acthar

Penetration

~780 Pts

~1,500 Patients (US)

Rheumatology

Acthar’s Largest Market Opportunity

DM/PM

jRA

Lupus

PsA

*See http://www.acthar.com/files/Acthar-PI.pdf for specific label information.

Approximately 250,000 combined patients

(US) are believed to be in need of additional

treatment options

•

Rheumatology-related indications

on the Acthar label*

–

Dermatomyositis/Polymyositis

–

Systemic lupus erythematosus

–

Rheumatoid

arthritis

adjunctive

therapy

or in selected cases low-dose

maintenance therapy

–

Psoriatic

arthritis

–

short

term

adjunctive

–

Ankylosing

spondylitis

–

short

term

adjunctive

•

Each can pose a serious health risk if not

adequately controlled

•

Some cases difficult to manage;

Acthar is an additional FDA-approved

treatment option

•

Positive initial uptake; expanded Rheum

Sales Force from 12 to 62 reps

Initiating a Pilot Commercialization Effort

in Pulmonology

•

Acthar is approved for the

treatment of respiratory

manifestations of symptomatic

sarcoidosis

–

A systemic inflammatory disease

where cell nodules or granulomas

can manifest in multiple organs, most

often in the lungs

–

Cause is unknown

–

May be asymptomatic or

chronic and may cause death;

difficult to treat

Asymptomatic

or Adequately

Treated

~130K

Potential

Acthar Patients

~20K

~20,000 Addressable Patients (US)

Indication

Potential Acthar

Patient Population

% Acthar Penetration

(estimated)

Large Addressable Markets with

Growth Potential (U.S. Only)

Note: Amounts and percentages based on internal company estimates.

Indication

Potential Acthar

Patient Population

% Acthar Penetration

(estimated)

NEUROLOGY

Infantile Spasms

1,500

52%

Large Addressable Markets with

Growth Potential (U.S. Only)

Note: Amounts and percentages based on internal company estimates.

Indication

Potential Acthar

Patient Population

% Acthar Penetration

(estimated)

NEUROLOGY

Infantile Spasms

1,500

52%

Multiple Sclerosis Flares

30,000

18%

Large Addressable Markets with

Growth Potential (U.S. Only)

Note: Amounts and percentages based on internal company estimates.

Large Addressable Markets with

Growth Potential (U.S. Only)

Note:

Amounts

and

percentages

based

internal

company

estimates.

Indication

Potential Acthar

Patient Population

% Acthar Penetration

(estimated)

NEUROLOGY

Infantile Spasms

Multiple Sclerosis Flares

NEPHROLOGY

Nephrotic Syndrome

1,500

30,000

35,000

52%

18%

Indication

Potential Acthar

Patient Population

% Acthar Penetration

(estimated)

NEUROLOGY

Infantile Spasms

1,500

52%

Multiple Sclerosis Flares

30,000

18%

NEPHROLOGY

Nephrotic Syndrome

35,000

RHEUMATOLOGY

PM/DM

20,000

Rheumatoid Arthritis

80,000

0.5%

Lupus

70,000

0.4%

Ankylosing spondylitis

50,000

0.04%

Psoriatic arthritis

30,000

0.2%

Large Addressable Markets with

Growth Potential (U.S. Only)

Note: Amounts and percentages based on internal company estimates.

Indication

Potential Acthar

Patient Population

% Acthar Penetration

(estimated)

NEUROLOGY

Infantile Spasms

1,500

52%

Multiple Sclerosis Flares

30,000

18%

NEPHROLOGY

Nephrotic Syndrome

35,000

RHEUMATOLOGY

PM/DM

20,000

Rheumatoid Arthritis

80,000

0.5%

Lupus

70,000

0.4%

Ankylosing spondylitis

50,000

0.04%

Psoriatic arthritis

30,000

0.2%

PULMONOLOGY

Sarcoidosis

20,000

0.2%

Large Addressable Markets with

Growth Potential (U.S. Only)

Note: Amounts and percentages based on internal company estimates.

Synacthen (tetracosactide) Overview

•

Acquired license to develop and commercialize Synacthen and

Synacthen Depot in U.S.

•

Rights to develop and commercialize in over three dozen countries*

–

Opportunity to reinvigorate Synacthen and provides platform for

potential international growth

•

Expands presence in inflammatory and autoimmune disorders

–

Provides foundation for next generation melanocortin peptide therapeutics

•

Further diversifies business

*Subject to certain closing conditions

Advancing Our Understanding of Acthar

and Melanocortin Peptides

•

One of 9 families of hormones produced by the pituitary,

the “master gland”

•

Believed to modulate the immune system and associated

inflammatory process through binding to 5 melanocortin receptors

–

MC1R, MC2R, MC3R, MC4R, and MC5R

–

Differences in chemical structure influence binding affinity

•

Questcor currently has two distinct melanocortin-peptide

based products

–

Acthar (porcine ACTH 1-39); Synacthen (tetracosactide)

Acthar Mechanism of Action

•

Clinical observations

–

Acthar has been shown to have increased

efficacy vs. corticosteroids in infantile spasms

–

Acthar has been successfully used to induce

remission of proteinuria in nephrotic syndrome

without uremia of the idopathic type

(e.g., iMN, FSGS, IgA nephropathy)

•

Preclinical observations demonstrate

steroid-independent

anti-inflammatory,

immuno-modulatory properties of

ACTH & other MC peptides

1: Bomback et al, Am J Nephrol 2012;36:58–67

2: Gong et al, Kidney International, 83, January 2013

Implications of Acthar and Other Melanocortins

(Synacthen and new MCs)

•

We now believe Acthar and other melanocortin peptides impact

–

Immune system

–

Inflammatory process

–

Some cell function

–

Homeostasis

•

Dozens of moderate-to-severe medical conditions may benefit from

Acthar or other melanocortin peptide therapeutics

Significantly Increasing Investment in R&D

•

Have funded or have approved

funding for studies covering

both on label and potential

new indications

–

Company sponsored pre-clinical

and clinical studies

–

Independent physician sponsored

studies

•

Investigating potential biological

properties of Acthar

–

Direct effect on biochemical

pathways, cells and tissues

–

Immunomodulation and

anti-inflammatory properties

R&D Investment ($M)

TTM 2013: Trailing twelve months through September 30, 2013

$11

$10

$11

$17

$34

$52

2008

2009

2010

2011

2012

TTM

2013

Expanding the Body of Evidence for

On-Label and New Indications/Targets

IDIOPATHIC MEMBRANOUS NEPHROPATHY

SYSTEMIC LUPUS ERYTHEMATOSUS

DIABETIC NEPHROPATHY

AMYOTROPHIC LATERAL SCLEROSIS

ACUTE RESPIRATORY DISTRESS

SYNDROME

Pre-Clinical

Phase 1

Phase 2

Phase 4

Phase 3

ALS Phase 2 Open-Label Safety Study for

Acthar

•

Goals

–

Assess short-term safety and tolerability of Acthar in ALS

–

Inform dosage selection for future studies

•

Study Design

–

Enroll up to 40 patients at multiple sites in U.S.

–

8-week treatment, plus optional 28-week open label extension

–

Patients randomized to one of four dosing regimens

Findings to Drive Design for Pivotal Efficacy Study

ARDS Phase 2 Safety and Efficacy Study for

Acthar

•

Goals

–

Determine if Acthar increases number of

ventilator-free days during 28-day

treatment period

–

Assess if Acthar reduces mortality,

organ failure, length of hospital or ICU stay

–

Inform dosage selection for future studies

•

Study Design

–

4-week randomized, placebo controlled trial

–

Enroll up to 210 patients at up to 40 sites in U.S.

–

Patients randomized to one of six dosing regimens

Findings to Drive Design for Pivotal Efficacy Study

Strong Platform for Growth

•

Increasing penetration of current Acthar markets and expanding sales into

new, approved indications

–

NS and MS market penetration remains modest

–

Rheumatology

new

Acthar

market

very

early

development;

growing

rapidly

–

Pilot selling effort in pulmonology for symptomatic sarcoidosis

–

Possible

Acthar

role

dermatology

and

ophthalmology

indications

being

evaluated for commercial potential

•

Untapped international market opportunities

–

Developing international markets for Synacthen and Acthar

•

Developing

new

indications

for

Acthar,

Synacthen

and

potentially

other

melanocortin therapeutics

•

Strong free cash flow generation enables possible product

acquisitions/partnering

Growing Cash Flow

TTM 2013: Trailing twelve months through September 30, 2013

Operating Cash Flow ($M)

$40

$37

$86

$219

$316

2010

2011

2012

2013TTM

2009

Committed to Creating Long Term Value

for Shareholders

•

Continued stewardship of Acthar and Synacthen

•

Demonstrated ability to execute

•

Long

term

investment

R&D

doubled

R&D

spending

2012

and

2013

(projected)

•

Highly selective, strategic diversification

•

Have returned $465 million to shareholders through share repurchases

and dividends

–

23.1 million shares repurchased

–

5.3

million

remain

available

for

repurchase

under

repurchase

program*

•

Quarterly dividend increased twice during 2013; $0.30/share

NASDAQ:

QCOR

January 2014

Net income per share –

basic and diluted may not foot

due to rounding

Use of Non-GAAP Financial Measures

Our “non-GAAP adjusted net income”

excludes

the

following items from

GAAP net income:

1. Share-based compensation expense

3. Interest expense associated with the net present value adjustment on

our contingent consideration

4. Interest expense associated with the net present value adjustment on

the R&D liability in conjunction with acquisition of Synacthen

5. Compensation expense associated with the BV Trust agreement

6. Foreign currency transaction loss

9. Impairment of purchased technology related to our

acquisition of Doral

Reconciliation of Non-GAAP Adjusted

Financial Disclosure

Three Months

Ended

September 30,

Nine Months

Ended

September 30,

2013

2012

2013

2012

Adjusted net income

$104,36

$59,427

$233,32

$143,94

Share-based compensation expense (1)

(5,269)

(2,855)

(13,807)

(6,908)

Depreciation and amortization expense (2)

(3,127)

(226)

(6,253)

(638)

Interest expense associated with contingent consideration (3)

(188)

(572)

Interest expense associated with R&D liability in conjunction with

(1,141)

(1,140)

Compensation expense associated with BV Trust (5)

(202)

(534)

Foreign currency transaction loss (6)

(329)

(7,751)

BioVectra

purchase price adjustment (8)

169

Impairment of purchased technology (9)

(659)

(485)

(662)

Net

income

–

GAAP

$94,441

$55,687

$202,62

$135,73

Adjusted

net

income

per

basic

$1.77

$1.01

$4.00

$2.36

Share-based compensation expense (1)

(0.09)

(0.05)

(0.24)

(0.11)

Depreciation and amortization expense (2)

(0.05)

0.00

(0.11)

(0.01)

Interest expense associated with contingent consideration (3)

0.00

—

(0.01)

—

Interest expense associated with R&D liability in conjunction with

(0.02)

Compensation expense associated with BV Trust (5)

0.00

—

(0.01)

—

Foreign currency transaction loss (6)

—

(0.01)

—

(0.13)

—

BioVectra purchase price adjustment (8)

—

0.00

—

Impairment of purchased technology (9)

—

(0.01)

Net

income

per

–

basic

$1.60

$0.95

$3.47

$2.23

Adjusted

net

income

per

diluted

$1.68

$0.97

$3.82

$2.25

Share-based compensation expense (1)

(0.08)

(0.05)

(0.23)

(0.11)

Depreciation and amortization expense (2)

(0.05)

0.00

(0.10)

(0.01)

Interest expense associated with contingent consideration (3)

0.00

—

(0.01)

—

Interest expense associated with R&D liability in conjunction with

(0.02)

Compensation expense associated with BV Trust (5)

0.00

—

(0.01)

—

Foreign currency transaction loss (6)

—

(0.01)

—

(0.13)

—

BioVectra purchase price adjustment (8)

—

0.00

—

Impairment of purchased technology (9)

—

(0.01)

Net

income

per

–

diluted

$1.52

$0.91

$3.32

$2.12

Net

sales

–

Questcor

$227,29

$140,33

$531,11

$348,76

Net sales -

BioVectra

9,050

24,935

Consolidated net sales

236,346

140,339

556,048

348,760

Medicaid adjustment

11,500

Adjusted consolidated net sales

$236,34

$140,33

$567,54

$348,76

2. Depreciation and amortization expense, including amortization

expense on our purchased intangibles

8. BioVectra purchase price adjustment related to a labor rebate

received in the second quarter 2013

Notes to Reconciliation of Non-GAAP Adjusted Financial

Disclosure

acquisition of Synacthen

(4)

7. Medicaid

adjustment

for

prior

period

2002

2009

Medicaid

adjustment

for

2002

2009

(7)

acquisition

Synacthen

(4)

acquisition of Synacthen

(4)

Medicaid

adjustment

for

2002

2009

(7)

Medicaid

adjustment

for

2002

2009

(7)

Reconciliation of Non-GAAP Adjusted

Financial Disclosure

2009

2010

2011

2012

TTM 2013

Adjusted

Net

Income

$29,242

$38,988

$83,956

$209,644

$299,033

Stock-based

Compensation

(2,310)

(2,649)

(5,128)

(10,502)

(17,396)

Depreciation

Amortization

Expense

(303)

(352)

(731)

(811)

(6,428)

Other

non-cash

expense

(income)

acquisition

BioVectra

(1,266)

Other

non-cash

expense

(income)

acquisition

Synacthen

(1,140)

Medicaid

adjustment

for

2002-2009

(7,751)

Tax

adjustments

(916)

1,702

Impairment

goodwill

(209)

(656)

(485)

GAAP

Net

Income

$26,629

$35,071

$79,591

$197,675

$264,566

Adjusted

Net

Income

per

Basic

0.46

0.63

1.34

3.48

5.12

Stock-based

Compensation

(0.04)

(0.08)

(0.17)

(0.30)

Depreciation

Amortization

Expense

(0.00)

(0.01)

(0.11)

Other

non-cash

expense

(income)

acquisition

BioVectra

(0.02)

Other

non-cash

expense

(income)

acquisition

Synacthen

(0.02)

Medicaid

adjustment

for

2002-2009

(0.13)

Ohio

CAT

(0.01)

0.03

Impairment

goodwill

(0.00)

(0.01)

GAAP

Net

Income

per

Basic

0.41

0.56

1.27

3.28

4.54

Adjusted

Net

Income

per

Diluted

0.44

0.60

1.27

3.33

4.89

Strong Platform for Growth

•

1.3 Rheumatic Disorders:

–

As adjunctive therapy for short-term administration

(to tide the patient over an acute episode or exacerbation)

in: Psoriatic arthritis; Rheumatoid arthritis, including

juvenile rheumatoid arthritis (selected cases may require

low-dose maintenance therapy);

Ankylosing spondylitis.

•

1.4 Collagen Diseases:

–

During an exacerbation or as maintenance therapy in

selected cases of: systemic lupus erythematosus,

systemic dermatomyositis (polymyositis).

How Does Acthar Work?

•

Treats autoimmune/inflammatory process associated with the

pathophysiology

•

By binding to melanocortin receptors, may modulate the immune

system and associated inflammatory process

1,5-9

•

Triggers the production of cortisol and other adrenal compounds

through binding to MC2R receptors found in the adrenal cortex

•

Properties extend beyond steroidogenesis

1,5-9

•

Acthar

components

have

yet

fully

characterized

Arnason

al.

Mult

Sclerosis

2012;

Arya

al.

Child

Neuro

2012;

Bomback

al.

Amer

Neph

2012;

Levine,

Drug

Design,

Dev

Therapy,

2012.

Catania, et al. Pharmacol Rev. 2004;

Stafstrom, et al. J Child Neuro 2011;

Manna SK, J Immunol. 1998;

Gong R. Nat Rev Nephrol. 2011;

Bohm et al. Endocrine Reviews 2012; 10H.P. Acthar Gel package insert. Questcor Pharmaceuticals, Inc., 2011.

Binds to melanocortin receptors found on immune cells

Binds

cells

many

types

tissues

(e.g.,

kidney

podocytes)

1,3,5,6,8,9

ACTH is believed to be the primary active component in Acthar,

but there may be others

1-4

Melanocortin Peptides May Activate Up to

Five Known Melanocortin Receptors

Steroid-dependent

anti-inflammatory

effects (indirect)

MC2R

Corticosteroids

Adrenal cortical cells

MC1R

MC3R

MC4R

MC5R

Altered tissue/organ & cell

function

Direct effect on organ-specific

cells

(eg, CNS, kidney, muscle)

Effects on immune cells

Immune cells, CNS cells, kidney cells, muscle cells,

many other cells

Acthar

Steroid-independent anti-inflammatory/immunomodulatory effects

MCR

Tissue/Cell Expression

Potential biologic activity

MC1R

Podocytes

Renal Mesangial Cells

Endothelial Cells (Glomerular,

Tubular, Vascular)

Macrophages, Monocytes, Neutrophils

Melanocytes

Keratinocytes

Central Nervous System

Chondrocytes

Respiratory tract

GI tract

•

Immunomodulation (including modulation of

antigen presentation; immune cell adhesion and

trafficking;

dampen

autoimmunity;

NF-

sequestration)

•

Cytoprotection (reduction of oxidative stress)

•

Ischemia-reperfusion

protection

Protection

from

LPS

-induced systemic inflammatory response

•

Cytoskeletal effects (regulate expression of

collagen, vimentin, podocyte specific proteins)

MC2R

Adrenal Cortex, Adipocytes, Testis

•

Steroidogenesis

MC3R

Central Nervous System

Macrophages

•

Immunomodulation

•

Protection from ischemia

MC4R

Podocytes

Renal Mesangial Cells

Central Nervous System

•

Regulation of

neuroinflammation

•

Metabolic control

MC5R

Central Nervous System

Exocrine Glands

Lymphocytes

•

Immunomodulation

•

B cell signaling

•

Exocrine secretion

•

Ocular immunity

•

Lipid regulation

Cerebral ischemic protection

Endothelial Cells (Glomerular, Tubular)

Includes

prescriptions

covered

commercial

carriers,

Medicare,

Medicaid

and

Tricare

in all

periods regardless of the rebate percentage applicable in those periods.

Total number of prescriptions includes all paid prescriptions.

Based on internal company estimates

New Paid Acthar Prescriptions

by Therapeutic Area*

Paid Rx

Comparison

Q3 –

2013

Q3 –

2012

Q2 –

2013

370 -

380

Rheumatology

1,370 -

1,400

225 -

230

450 -

460

33%

N/M

Total

2,450 -

2,500**

30%

10%

43%

Biosimilar Pathways Highly Challenging

•

Complex formulation and pharmacology, with multiple receptor

binding properties

–

Possibly multiple active peptides

–

Slow release gel formulation

–

Complex and not well characterized (research is ongoing)

•

Formulation and manufacturing trade secrets inherent with Acthar

•

Future synthetic versions of ACTH might be possible, but in a

specific indication

–

Clinical trial(s) and other development work likely required

–

Multi-year pathway; challenging IP landscape

NASDAQ:

QCOR

January 2014

QUESTCOR PHARMACEUTICALS INC - FORM 8-K - EX-99.1 - January 13, 2014

Attached files

QUESTCOR PHARMACEUTICALS INC Reports