Building an Orphan Drug Company - Proactiveinvestors UK · 2018. 12. 8. · market or through a professional intermediary, ... ***Excluding generics . 4 ... Global Healthcare Research

Bui ld ing an Orphan Drug Company

A p r i l 2 0 1 6

2

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Amryt Pharma is focused

on Rare/Orphan Diseases

3

•Amryt is committed to the development and commercialisation of medicines to treat patients with rare/orphan diseases

•Lead product has already been approved in Europe* and will be developed as a treatment for Epidermolysis Bullosa with earlier stage opportunities in Acromegaly and Cushing's disease

Singular Strategic Focus

•Worldwide orphan drug sales forecast to total $176bn (CAGR 2014 to 2020:+10.5%)**

•Orphan drugs set to be 19.1% of worldwide prescription sales by 2020***

Orphan Disease Represents a Major Market Opportunity

•Focus on orphan areas with large and unmet medical need with aim of radically improving patient lives

•Develop diversified pipeline of therapies from earlier stage to late stage/commercial

Develop Portfolio of High Value Therapies to Patients, Physicians and Payers

•Acquired two attractive orphan drug companies with an already approved product

•Completed IPO H1 2016 via RTO of Fastnet Equity Plc (AIM: FAST)

•Further acquisitions will be evaluated on an ongoing basis

Create value for Amryt Shareholders

*Marketing authorisation granted by the European Commission

14th January 2016

**Evaluate Pharma as of 17th July 2015

***Excluding generics

4

Led by an Experienced Team

Senior Management

Joseph Wiley – CEO

• 20+ years in healthcare and private equity

• Opened and led Sofinnova Ventures European office

• Previously Medical Director at Astellas Pharma

• Currently Non-executive director of Innocoll AG

(NASDAQ:INNL)

Rory Nealon – COO/CFO

• CFO/COO of Trinity Biotech

• Oversaw the acquisition and integration of 12

companies in 5 countries.

• Previously CFO of Conduit plc, an Irish telecoms

company

• Previously associate director within structured finance

team in AIB

Operational Team

Michele Bellandi – CCO

• Head of Commercial Europe at Shire

• Responsible for the entire portfolio representing $1 billion

revenues and approx. 600 employees

• Previously Global Marketing Director for the Neurology

franchise at Serono

• Previously Cymbalta Global Marketing Manager at Eli

Lilly

Dr. Tobias Zahn – Head of Clinical Operations

Dr Alan Harris – Head of Scientific Advisory

Board

Non-Executive Directors Harry Stratford, Chairman

• Founder of Shire Pharmaceuticals

• Founder, CEO and Chairman of Prostrakan

James Culverwell

• Partner, Sudbrook Associates

• FVP, Global Healthcare Research at Merrill Lynch

CMO – To Be Announced

• Strong background in clinical trials and regulatory

submissions

Raymond Stafford

• Former Chief Executive, Europe and Executive Vice

President, Global Marketing at Forest Laboratories

• Sold Tosara Group, the maker of Sudocrem, to Forest

in 1986

• Joined Birken in 2009 and was responsible for the

development of Episalvan

• Previously a consultant at The Boston Consulting Group

• Founder of SomPharmaceuticals

• At Novartis, headed Clinical Development of octreotide,

approved for the treatment of Acromegaly ($1.65bn

sales in 2014)

Cathal Friel

• Managing Director, Raglan Capital

• Founder and Chairman of Fastnet Equity plc

5

Orphan Drugs are a Uniquely

Attractive Market Segment

Source: Evaluate Pharma as of 17th July 2015

6

Driven by the Benefits of

Developing Orphan Treatments


*Subject to conditions

*

7

That is Reflected in the Growth

of Orphan Drug Sales

6.3%

19.1%

0%

5%

10%

15%

20%

25%

0

20

40

60

80

100

120

140

160

180

200

2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 2020

Orp

han

sale

s a

s a

% o

f P

rescri

pti

on

Sale

s e

xclu

din

g

gen

eri

cs

Orp

han

Dru

g S

ale

s (

$ B

n)

Global Orphan Drug Sales & Share of Prescription Drug Market

Orphan Sales Orphan as % of RX

+10.5% CAGR 2014-20


8

Also Reflected in Orphan Drug

Company Valuations Company Market Cap ($m) Lead Orphan Indication

Marketed

343,030 Atypical hemolytic uremic syndrome

13,690 Phenylketonuria, mucopolysaccharidosis VI and Lambert-Eaton

myasthenic syndrome (LEMS)

1,790 Dravet Syndrome and Lennox-Gastaut Syndrome

Phase III Sage Therapeutics

1,180 Super-Refractory Status Epilepticus (IV)

Phase I/II Bellicum Pharmaceuticals

303 Orphan inherited blood disorders

Marinus Pharmaceuticals 119 Fragile X syndrome

Pre-clinical Omeros Corporation

568 Orphan GPCR platform

Fibrocell Science 122 Recessive Dystrophic Epidermolysis Bullosa

Source: Capital IQ as of 18th April 2016

9

And Comparable Orphan Drug

M&A Transactions

Year Target Acquirer Value ($m)* Lead Orphan Indication

Phase III / Filed

2015 8,394 Lysosomal acid lipase deficiency and

mucopolysaccharidosis (MPS)

2015 3,500 Chorea associated with Huntington’s disease

2015 842 Epidermolysis Bullosa

Phase I/II

2014 260 Rare hepatic diseases and non-alcoholic steatohepatitis

2015 245 Rare GI disease product

Pre-clinical

2013 325 Recessive Dystrophic Epidermolysis Bullosa

2013 130 Recombinant human acid-alpha glucosidase & Pompe

disease

2010 97 Rare, genetically defined cancers

Source: Capital IQ as of 18th April 2016

*some of which are partly conditional on

milestones

Amryt’s Products are Targeting

Multiple Orphan Diseases

10

Product

Candidate Indication Preclinical Phase I Phase II Phase III Approved*

Est.

Market

Entry

Total Est.

Market Size

Episalvan***

(AP101)

Partial

Thickness

Wounds 2016 US$150+

million**

Episalvan

(AP101)

Epidermolysis

Bullosa (EB) 2019 US$1.5+ billion

AP102*** Cushing’s

Disease 2023

US$500+

million

AP102*** Resistant

Acromegaly 2024

US$650+

million

**EU Only

***Subject to securing additional capital

*Marketing authorisation granted by the European

Commission 14th January 2016

Episalvan Approved for PTW in Europe* with

Future Opportunity in Epidermolysis Bullosa

11

Current Approval Partial

Thickness Wounds (PTW) Orphan Opportunity**

Burns

STSG

Dermatological Procedures

Aesthetic Procedures

Decubitus

Toxic Epidermal Necrolysis (TEN)

Radiation and Drug Side

Effects

Epidermolysis Bullosa

$1.5 billion+ Market Opportunity

ODD granted in

EU & USA

Episalvan

*Marketing authorisation granted by the European Commission 14th January 2016

**Management intends to seek label extension to existing EU approval leveraging

off validation to date and to seek further approval in US

12

Episalvan Heals by Accelerating Migration

of Skin Cells Across Wound Surface

Episalvan Promotes cell migration of primary keratinocytes And also supports formation of a tight skin barrier

by enhancing keratinocyte differentiation

Split-thickness skin graft donor site

wounds

Intra-individual difference in time to

wound closure between the wound

halves

Mean acceleration of wound

healing with Episalvan was

approximately 2 days*

Median time to wound closure: 14

days

Episalvan

N=219

13

Episalvan Approval based

on Positive Phase 3 Data

Characteristics

Primary Endpoint

Patients

Results

Representative photo series:

Results show significant improvement

SOC

0 4 11 Day

*Investigator assessment average time interval of -2.3 days (CI: -3.0, -1.7)

faster wound closure with Episalvan, p<0.0001

2nd degree burns

Percentage of patients with earlier

wound healing vs. standard of care

(SOC)

85.7% of healed wound halves

treated Episalvan (p<0.0001)

Mean acceleration in wound

healing = 1.6 days by direct

investigator assessment

N=61

14

Episalvan Approval based

on Positive Phase 3 Data

Characteristics

Primary Endpoint

Patients

Results

Representative photo series:

Results show significant improvement

SOC Episalvan

15

With Episalvan Rated Superior

over Standard of Care

60%

33%

7%

56%

37%

7%

0%

10%

20%

30%

40%

50%

60%

70%

Episalvan betteror much better

equal SOC better ormuch better

STSG - Efficacy assessed at end of treatment

Investigatorassessment

Patient assessment

45%

53%

2%

46% 50%

4%

0%

10%

20%

30%

40%

50%

60%



STSG - Tolerability assessed at end of treatment


Patient assessment

88%

10% 2%

85%

15%

0% 0%

20%

40%

60%

80%

100%



Burns - Efficacy assessed at end of treatment


Patient assessment

77%

23%

0%

77%

21%

2% 0%

20%

40%

60%

80%

100%



Burns - Tolerability assessed at end of treatment


Patient assessment

16

And Long Term Outcomes Were

Also Better with Episalvan

51

67

50

19 19 13

0

10

20

30

40

50

60

70

80

Redness Pigmentation Texture

Pa

tie

nts

(n

)

Episalvan

SoC

STSG 3-Month*

19

35

20

6

13

4

0

5

10

15

20

25

30

35

40


Pa

tie

nts

(n

)

Episalvan

SOC

STSG 12-Month*

9

14

11

1 0

1

0

2

4

6

8

10

12

14

16


Pa

tie

nts

(n

)

Episalvan

SOC

Burns 3-Month*

3

5

4

0 0 0 0

1

2

3

4

5

6


Pa

tie

nts

(n

)

Episalvan

SOC

Burns 12-Month*

*Which of the former wound halves is more similar in

appearance to the surrounding healthy skin?

Episalvan Providing Hope to Young

Children with this Distressing Disorder

17

Junctional • Most severe

• Extensive blistering all over body

• Often fatal in early childhood

Dystrophic • More severe

• Fusion of fingers and toes

• Possibility of skin cancer

Simplex • Mildest form

• Episodic blistering

Mortality

Risk

NO

TREATMENT

OPTIONS

TODAY

18

Defined Path to Orphan Drug

Approval and Commercialisation

Product Activity

2016 2017 2018

H1 H2 H1 H2 H1 H2

Episalvan

(AP101)

EU Partial Thickness

Approved

3 month tox

US/EU Phase III Study

EB

Submission for US/EU

Approval

19

Currently We have a Range of Various

Imlan Products

Imlan is sold as a dermaceutical and marketed as a treatment for “sensitive ,

allergy-prone and dry skin, also recommended for basic care for eczema or

psoriasis”

Year 2014 2015

Revenue €883 €933k

20

Earlier Stage Pipeline of Products are Targeting

Acromegaly & Cushing’s Disease Headache, Supraorbital Bulging, Malocclusion,

Vision Defect (~15%), Sleep Apnea (~70%),

Thyroid Hypertrophy

Cardiomyopathy

Hypertension (~40%)

Enlarged Colon and

Colon Polyps (~45%)

Hepatomegaly

Nephromegaly

Splenomegaly

Enlarged Hands

Carpal Tunnel Syndrome (~28%)

Skin Tags

Hypogonadism (~50%)

Arthropathy (~75%)

Enlarged Feet

Psychosis, Impaired Memory, Sleep

Disturbance, Major Depression and Anxiety

Disorder (55%-80%), Facial Fat

Accumulation

Hypertension (60%-80%)

Dyslipidemia (40%-70%)

Overweight / Obesity (up to 90%)

Abdominal Fat Accumulation

Impaired Glucose Tolerance /

Diabetes (up to 65%)

Muscle and Skin Atrophy

Osteoporosis

Cushing’s disease increases

mortality rate by up to 5x normal

Acromegaly increases mortality

rate by up to 2.3x normal

21

AP102 Market Potential

Estimated Market Size US$650M

Acromegaly

Octreotide generates US$1.65B in annual

sales.

However, up to 70% of patients are resistant

to Octreotide

The current second-line treatment,

Pasireotide, is complicated by causing

diabetes.

Pasireotide currently sells for US$175,000 in

the U.S. per patient per year.

AP102 is a next generation somatostatin

analogue therapy to treat resistant

Acromegaly and Cushing’s disease without

causing diabetes.

22

Amryt Highlights

Overview

Already

Generating

Revenues

Risk Mitigation

Late stage European orphan drug company

Part of an exclusive group on AIM with an approved drug

Strong management team and board with excellent track record

Existing revenue generating commercial stage dermaceutical range

with partnering opportunities providing revenue upside

Potential to launch recently approved Episalvan product in EU

which would further drive revenue growth

Transformational Episalvan opportunity in est. $1.5 billion EB

market with phase 3 data anticipated in early 2018

Earlier stage opportunities in Acromegaly and Cushing's disease

Experienced team with significant experience in orphan diseases

Sustainable core business with already approved product

Episalvan approval reduces the risk in getting approval in EB that

unlocks significant upside

Multiple products for multiple indications diversifies product risk

23

Appendix

24

Amryt Pharma: Advisors

Dr Jonathan Wilkin

• Previous Founding Director of Dermatology & Dental Products at FDA

Michelle Hefley

• President and CEO at Orphic Therapeutics

Shlomo Melmed

• World leading expert in Acromegaly and Cushing’s Disease at Cedars-Sinai

Nan Wu

• Partner in Patent Counselling and Prosecution at Cooley

25

Amryt is a New Orphan Drug Company

Criteria for Potential

Third Acquisition

100% owned

Subsidiaries

Based in Germany

€54m invested to date by Software-

AG Stiftung

Orphan Drug Designation in US and

EU

Software-AG Stiftung retain 20%

equity stake in Amryt pre-RTO

Product: Episalvan (AP101) approved

in Europe – January 2016

Based in Switzerland

Founded by lead developer of

Sandostatin® at Novartis - $1.65

billion orphan disease drug

Developing next generation therapy

for acromegaly and Cushing’s

Disease

Product: AP102

Screening criteria for acquisitions Rare disease

Devastating disorder

Significant orphan patient population

Meaningful clinical benefit for patients

100% owned

Subsidiaries

100% owned

Subsidiaries

Epidermolysis bullosa (EB) is a distressing and painful genetic skin condition that

causes the skin layers and internal body linings to separate

EB is characterised by extreme fragility of the skin from birth

Prevalence: 25,000 – 35,000 in U.S; 30,000 – 41,000 in EU*

What is Epidermolysis Bullosa?

26

1:17,000

RARE GENETIC ANYONE NO CURE

1 in 17,000 live

births affected

Hereditary, but

parents may not

know they’re carriers

Equally affects

both genders and

every ethnicity

No current treatment

*The Dystrophic Epidermolysis Bullosa Research Association (DEBRA)

*Stanford School of Medicine, “Epidermolysis Bullosa Clinic”

Epidermolysis Dystrophic and

Recessive subtypes

Blinded assessment of wound

reduction by third party reviewer

12 wounds assessed in 10 patients

8/12 faster healing with Episalvan;

0/12 with SOC

4 undecided

10 patients ages 6 to 48 years

• 7 male

• 3 female

27

Episalvan Shown to be Effective in EB

Characteristics

Primary Endpoint

Patients

Results

Wound reduction

Episalvan

+ wound

dressing

Wound

dressing

only

Undecided

28

Episalvan Shows Consistency of

Data Across All Phase 3 Studies

BBW-11

Burn Wounds Grade 2a

61 patients

BSH-12

Split-thickness-skin-graft

donor site wounds

107 patients

BSG-12

Split-thickness-skin-graft

donor site wounds

112 patients

BEB-10

EB Study

10 patients

29

And Has Robust IP Protection in

Addition to Orphan Exclusivity

Technology Scope of Protection Geographical Coverage Patent Status

Extraction

Method

Device and method for continuously

extracting extract materials from solid

materials, for washing solid materials and

recrystallizing

Europe: AT, BE CH, DE, ES, FR, GB, IE, IT LU Filling Date: 14/08/2003

Expiry Date: 2023

Emulsion Emulsion containing a triterpene containing

plant extract, method for producing said

emulsion and obtaining a plant extract

Americas: US

Europe: AT, BE, CH, CY, DE, DK, EA, EE, ES,

FI, FR, GB, GR, IE, IT, LU, MC, NL, NO, PL,

PT, SE, TR

Asia: IN, JP, CN, MN

Filling Date: 26/03/2001

Expiry Date: 2021

Formulation Triterpene-containing gel-forming agent,

triterpene-containing gel and method for

producing triterpene-containing gel

Americas: CA, MX, US

Europe: AT, BA, BE, BG, CH, CY, CZ, DE, DK,

EE, ES, FI, FR, GB, GR, HU, IE, IL, IS, IT, LT,

LU, LV, MC, ME, NL, PL, PT, RO, RS, RU, SE,

SI, SK, TR, UA

Asia/Oceania: AU, CN, ID, IN, JP, KR, NZ, SG

Africa: ZA


Expiry Date: 2025

(USA: 2026)

Method of Use

for Wound

Healing

Use of a gel containing triterpene for

healing wounds

Europe

US

Africa: ZA


Expiry Date: 2030

Pending: CA

Pending: BR, NO

Pending: all others

30

Launching in EB Will Require a

Small Sales Force Footprint

31

Like Pasireotide, AP102 Binds to

SSTR2 & SSTR5

Compound SSTR1 SSTR2 SSTR3 SSTR4 SSTR5

Somatostatin-14 - 2.3 0.2 1.4 1.8 0.9

Octreotide >1000 0.6 34.5 >1000 7.0

Lanreotide >1000 0.8 107 >1000 5.2

Somatoprim >1000 3.0 >100 7.0 6.0

Pasireotide 9.3 1.0 1.5 >100 0.2

AP102 >1000 0.6 >1000 N/A 0.7

IC50 nmol

0

100

200

300

400

500

600

Ctrl AP102 Pasireotide Octreotide

GH

[u

g/L

]

0

10

20

30

40

50


GH

[u

g/L

]

32

With Efficacy Equivalent to or Better Than

Current Treatment

Human pituitary Growth Hormone (GH) secreting adenoma Biopsies

- In vitro tissue culture media ELISA results

- Tissue biopsy of human adenoma by patient consent

* * *

* * *

*P<0.05 cf. control

* * *

0

10

20

30

40

50

60

70

80


GH

[u

g/L

]

33

However, Unlike Pasireotide, AP102 Does

Not Result in Increase in Glucose

Blood Glucose Levels

Glu

co

se (

mg

/dL

)

Vehicle l

Pasireotide 1 µg/kg k

Pasireotide 10 µg/kg g

Pasireotide 30 µg/kg h

AP102 1 µg/kg h

AP102 10 µg/kg u

AP102 30 µg/kg

Time (h)

300

200

100

0

0 1 2 3 4 5 6

Documents

Building an Orphan Drug Company - Proactiveinvestors UK · 2018. 12. 8. · market or through a professional intermediary, ... ***Excluding generics . 4 ... Global Healthcare Research