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Amryt Pharma – Acquisition of Aegerion Building a Global Leader in Rare and Orphan Diseases STRICTLY PRIVATE & CONFIDENTIAL August/September 2019

Amryt Pharma Acquisition of Aegerion Building a Global ... · This presentation has been prepared by Amryt Pharma plc (the “Company”). “Presentation”means this document, any

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Amryt Pharma – Acquisition of Aegerion

Building a Global Leader in Rare and Orphan DiseasesSTRICTLY PRIVATE & CONFIDENTIAL

August/September 2019

Disclaimer

2

This presentation has been prepared by Amryt Pharma plc (the “Company”). “Presentation” means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed duringthe meeting. By receiving this presentation and/or attending the meeting where this presentation is made, or by reading the presentation slides, you agree to be bound by the following limitations. This presentation and its contents areconfidential and you agree that you may not, nor are you authorised to, copy or reproduce this presentation in whole or in part in any manner whatsoever or distribute, publish or forward this presentation or disclose any of itscontents to any person. Failure to comply with these undertakings may constitute a violation of applicable securities law. This document contains an indicative summary of the terms and conditions of a potential transaction (the“Transaction”) and this document, and such terms and conditions, may be amended, suspended or replaced, or the Transaction may not proceed at all. This presentation is being made only to, and is directed only at, (i) persons havingprofessional experience in matters relating to investments who fall within the definition of "investment professionals" in Article 19(5) of the Financial Services and Markets Act 2000 (Financial Promotion) Order 2005 (as amended fromtime to time) (the “Order”); (ii) high net worth bodies corporate, unincorporated associations, partnerships and trustees of high value trusts as described in Article 49(2)(a)-(d) of the Order; (iii) “overseas recipients” as defined in Article 12of the Order; (iv) persons in member states of the European Economic Area (the “EEA”) who are qualified investors within the meaning of Article 86(7) of the Prospectus Regulation (EU 2017/1129), including any relevant implementingmeasure in each member state of the EEA which has implemented this Prospectus Regulation; (v) qualified institutional buyers in the United States as defined in Rule 144A of the United States Securities Act of 1933; or (vi) persons towhom it would otherwise be lawful to distribute it (all such persons being “Relevant Persons”). Persons who receive this communication (other than Relevant Persons) should not rely on or act upon the contents of this presentation andshould return this document immediately. This presentation is being directed only at Relevant Persons and any investment or investment activity to which this presentation relates will be engaged in only with Relevant Persons. Thispresentation does not constitute or form part of any offer to sell or issue, or invitation to purchase or subscribe for, or any solicitation of any offer to purchase or subscribe for, any securities of the Company or any of its subsidiaries(together the “Group”) or in any other entity, nor shall this presentation or any part of it, or the fact of its presentation, form the basis of, or be relied on in connection with, any contract or investment activity (including within themeaning specified in section 21 of the Order), nor does it constitute a recommendation regarding the securities of the Group. 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Neither this presentation nor any information contained in this presentation should be transmitted into,distributed in or otherwise made available in whole or in part by the recipients of the presentation to any other person in the United States, Canada, Australia, Japan or any other jurisdiction which prohibits or restricts the same except incompliance with or as permitted by law or regulation. Recipients of this presentation are required to inform themselves of and comply with all restrictions or prohibitions in such jurisdictions. Accordingly, by requesting to receive andreviewing this document you represent that you are able to receive this document without contravention of any legal or regulatory restrictions applicable to you. 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The Company’s shares are currently admitted to trading on public exchanges and, therefore, certain of the information in this presentation could constitute inside information and/or price sensitive information. By accepting thispresentation, you agree to use and maintain any such information in accordance with your contractual obligations (including, without limitation, pursuant to the confidentiality undertakings you have entered into with Phoenix) andapplicable laws, including all applicable securities laws. The information contained in this presentation has not been independently verified. The information in this presentation includes forward-looking statements, made in good faith,which are based on the Group’s or, as appropriate, the Group’s directors' current expectations and projections about future events. 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In light of these risks, uncertainties and assumptions, the events or circumstances referred to in the forward-looking statements maydiffer materially from those indicated in these statements. Forward-looking statements may, and often do, materially differ from actual results. Thus, these forward-looking statements should be treated with caution and the recipients ofthe presentation should not place undue reliance on any forward-looking statements. None of the future projections, expectations, estimates or prospects or any other statements contained in this presentation should be taken asforecasts or promises nor should they be taken as implying any indication, assurance or guarantee that the assumptions on which such future projections, expectations, estimates or prospects have been prepared are correct orexhaustive or, in the case of the assumptions, fully stated in the presentation. Nothing contained in this presentation shall be deemed to be a forecast, projection or estimate of the Company’s future financial performance. TheCompany’s past performance cannot be relied on as a guide to future performance. Any forward looking-statements are made as of the date hereof.The information and opinions contained in this presentation and any other materialdiscussed verbally are provided as at the date of this presentation and are subject to verification, completion and change without notice. The delivery of this presentation shall not give rise to any implication that there have been nochanges to the information and opinions contained in this presentation since the time specified. Subject to obligations under the AIM Rules for Companies published by the London Stock Exchange plc and the Market Abuse Regulation(Regulation 596/2014) (each as amended from time to time), none of the Company, the Group, their affiliates and advisers and their respective directors, officers, partners, representatives, employees and agents, undertakes to publiclyupdate or revise any such information or opinions, including without limitation, any forward-looking statement or any other statements contained in this presentation, whether as a result of new information, future events or otherwise.In giving this presentation none of the Company, the Group, their affiliates and advisers and their respective directors, officers, partners, representatives, employees and agents, undertakes any obligation to provide the recipient withaccess to any additional information or to update any additional information or to correct any inaccuracies in any such information which may become apparent. Certain industry and market data contained in this presentation has beenobtained from third party sources. Third party industry publications, studies and surveys generally state that the data contained therein have been obtained from sources believed to be reliable, but that there is no guarantee of theaccuracy or completeness of such data. While the Company believes that each of these publications, studies or surveys has been prepared by a reputable source, the Company has not independently verified the data contained therein. Inaddition, certain of the industry, scientific and market data contained in this presentation comes from the Company’s own internal case studies, research and estimates based on the knowledge and experience of the Company’smanagement in the market in which it operates. While the Company believes that such research, estimates and results from its case studies are reasonable and reliable, they, and their underlying methodology and assumptions, have notbeen verified by any independent source for accuracy or completeness unless otherwise stated and are subject to change without notice. This presentation may include certain combined or pro forma financial information for AegerionPharmaceuticals Inc and the Company. Such combined or pro forma financial information is preliminary in nature, only represents current estimates of the potential impact of the Transaction on the Company, remains subject to changeand is provided solely for illustrative purposes. No reliance should be placed on the combined or pro forma financial information contained in this presentation. By requesting to receive and reviewing this document, you agree to bebound by the foregoing limitations and conditions and, in particular, will be deemed to have represented, warranted and undertaken that you have read and agree to comply with the contents of this disclaimer including, withoutlimitation, the obligation to keep this document and its contents confidential and that you are a Relevant Person.

3

Creating a Global Leader in Rare & Orphan Diseases

• Revenue generating rare & orphan drug company

• Strong portfolio of commercial & development assets

• Lojuxta (Juxtapid in US) in-licenced by Amryt (Dec 16) in EMEA - 2018 Sales: $16.1m

• Strong management team with unique knowledge of Aegerion assets

• Upside through clinical Epidermolysis Bullosa (‘EB’)franchise

• AP101 - potential mkt. opportunity >$1bn

• AP103 - novel gene therapy platform

• Rare & orphan disease focus with a global footprint

• Two commercial products with significant growth potential:

• Juxtapid 2018 Sales: $47.9m US & RoW (Lojuxta in Europe)

• Metreleptin 2018 Sales: $71.4m US, Europe & RoW

• Pipeline opportunities for Myaleptin PL and Juxtapid/Lojuxta in FCS

• Established US infrastructure in place

• Strong patent & regulatory exclusivity protection

Emerging global leader in rare & orphan diseases with diversified,

commercial portfolio

Delivers sustainable revenue, pipeline and market growth

Creates a rare disease business with two approved products – lomitapide(Lojuxta® / Juxtapid®) and metreleptin (Myalept® / Myalepta®)

$136.5m of 2018 built-in revenues, multiple growth opportunities, and a robust pipeline for value creation

Reunites the lomitapide franchise and transforms Amryt into a global player in the orphan disease market

Capitalizes on Amryt management’s unique knowledge of Aegerion’s assets and European commercialization capabilities

Presents the opportunity for meaningful expense synergies - $25m-$40m in 2020*

Establishes an appropriate capital structure and liquidity profile to drive growth and create value

Transaction already endorsed by 34.3% of Amryt’s shareholders and 67% of Aegerion’s bondholders

4

Strong Strategic Rationale for Combination

Creating a global player in rare & orphan diseases*Directors belief based on work undertaken to date

Enhanced scale of combined group expected to drive revenue growth and future profitability

Expected to deliver meaningful operational synergies over the medium term

Amryt’s deep knowledge of Aegerion products is key to driving growth

Reunification of lomitapide brands provides potential to replicate success of Lojuxta in Europe with Juxtapid in the US

Opportunity to grow Myalepta revenues with broader reach across EU to accelerate recent launch

Delivers a ready-made commercial US infrastructure in advance of anticipated launch of AP101

Recapitalized business well-positioned to drive pipeline value

Planned short-term NASDAQ listing to drive liquidity and investor reach

Opportunity for corporate restructuring to drive additional value

5

Value Creation Opportunity Driven by Execution on Plan

Delivering superior returns & driving shareholder value

6

Amryt Leadership Team

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 AIB

Dr Mark Sumeray – CMO

20 years’ experience in the pharmaceutical, medical

devices and biotech sectors

Chief Medical Officer at Aegerion Pharmaceuticals

Previously VP Cardiovascular Metabolics US Medical

at Bristol-Myers Squibb

Dr Helen Phillips – Head of Medical Affairs

20+ years in large pharma and small biotech

companies

Previously European VP of Medical Affairs in

Aegerion Pharmaceuticals

Dr Joe Wiley – CEO

Founded Amryt in 2015

20+ years in healthcare and private equity

Opened and led Sofinnova Ventures European office

Senior investment roles at Aberdeen Asset Managers,

Inventages Venture Capital

Previously Medical Director at Astellas Pharma

David Allmond – CCO

25 years’ experience in the pharmaceutical industry

in commercial roles

President EMEA at Aegerion Pharmaceuticals

Previously Corporate Vice President of Global

Marketing for Celgene Corporation

Kieran Rooney – VP, Strategic Alliances & Licensing

25+ years in business/corporate development

in pharmaceutical and biotech industry

Previously VP Business Development at Amakem

Therapeutics and corporate/management consultant to

multiple pharma/biotech/professional service companies including PwC

Derval O’ Carroll – Head of Regulatory Affairs

25 years in pharmaceutical regulatory affairs

companies

Previously Senior Director of Regulatory Affairs at

Retrophin Inc

11 years consulting experience in regulatory with clients

such as Daiichi Sankyo and Shionogi

Julie Eastwood – Head of HR

25 years experience in HR

Fellow CIPD

Previously HR Director at Totalmobile, TV3

Oxigen and Readymix.

Stephen Joyce – VP Global Marketing15+ years in the pharmaceutical industry

Held multiple commercial roles, with a large focus on

launch, access and commercial excellence

Previously General Manger, Switzerland, Senior Director Area

Marketing & Market Access at Astellas Pharma

Gerard Gilligan – VP Manufacturing, Supply Chain 25+ years on Pharmaceutical Manufacturing & Supply Chain

Previously Held various role in Leo Pharma including Site,

Operations and Manufacturing Lead, Chemistry and

Lean/Black Belt

Team in place to scale and grow our business

7

Amryt + Aegerion - Global Presence

7

Geographically complementary and providing new market access

Pending markets

Out-licensed

Current markets

8

Overlap in combined group brings significant synergies including head office.Amryt brings additional reach and expertise in MENA, CEE and Russia

MENATURKEYUKSPAINITALYFRANCEGERMANY

+++++++++++++++++

++++++++++++++++++

A perfect fit delivering a stronger combined platform for growth

Complementary Fit - Global Commercial Footprint

US

+

++++

Product Candidate Indication Preclinical Phase 1 Phase 2 Phase 3 Approved

9

Amryt + Aegerion - Rich Commercial & Development Pipeline

Myalept/Myalepta GL US/EU

Myalepta PL EU

Juxtapid (lomitapide) HoFH (adult) US/LATAM

Lojuxta (lomitapide) HoFH (adult) EU/MENA

AP101 EB (DEB/JEB) GLOBAL

Myalept PL US

Lomitapide FCS US/EU

AP101 SJS/TEN, Rad. Ind Derm GLOBAL

AP103 EB (RDEB) GLOBAL

AP103 Various/undisclosed GLOBAL

Lojuxta (lomitapide) HoFH (paediatrics) EU

9

Augments existing pipeline and delivers growth opportunities

Aegerion Joint Amryt

US/EU

EU

US/LATAM

EU/MENA

H1H1 H1H2H1 H2H1

KJB I

H2 H2H2H1

LA C F GE HD

A. Metreleptin GL/PL Filing in Other Countries

B. US PL FDA meeting

C. Lojuxta top line PoC data for FCS

D. First Quarterly Report as Combined Company

E. AP101 top line data

F. First Annual Report as Combined Company

G. AP101 Approval (FDA & EMA) and Launch

H. Lojuxta Phase 3 topline data for FCS

I. Lojuxta EU pediatric approval for HoFH

J. AP103 Clinical PoC data

K. Lojuxta launch in FCS

L. US PL launch

2020

10

2022

Building a global leader in rare & orphan diseases

2019 2021 2023 2024

2019

2019

2019

2020

2021

2021

2021

2022

2022

2023

2023

2024

H2

Note: Clinical milestones are estimates and subject to change.

Compelling Equity Story With Significant Upcoming Newsflow

11

Lojuxta / Juxtapid▲Reunifying the global lomitapide brands

▲Lojuxta / Juxtapid

▲ Approved in both Europe (Jul 2013) and the US (Dec 2012) to treat adults with HoFH

▲ Reduces LDL-C in adult HoFH patients▲ Patent protection in US to mid 2027 and 2028 in EU*

▲ 2018 Global Revenues: $64m**

▲Amryt successfully commercializing Lojuxta in Europe since 2016

▲Market potential for adult HoFH patients in the US

▲Deploy proven strategy for Lojuxta in Europe to drive Juxtapidgrowth in the US

▲ Lifecycle opportunities in pediatric HoFH and Familial Chylomicronemia Syndrome (‘FCS’)

Demonstrable track record in commercializing Lojuxta/Juxtapid

What is HoFH ? HoFH is a potentially life threatening disorder that impairs the body’s ability to remove LDL ‘bad’ cholesterol from the blood. Typically results in extremely high blood LDL cholesterol levels leading to aggressive and premature blocking of arterial blood vessels.

*SPC Granted in 4 of 5 major markets; pending in UK. **Excluding Japan revenues; including Japan royalties

Lojuxta Performance & Growth Trajectory In EMEA

23% increase in revenues in 2018

Forecast to grow a further 31% by end 2019*

Performance driven by lean & talented team

– Repositioned product

– Optimized market access - successfully

delivered positive reimbursement in key

markets such as UK and FR

– Broadened geographic reach

12

Opportunity to replicate this success globally

* Consensus estimates

Myalept / Myalepta (metreleptin)

14

Myalept / Myalepta - A Significant Global Opportunity for Growth

▲Myalept / Myalepta▲ Approved in the US (Feb 2014) to treat Generalized Lipodystrophy

(‘GL’)▲ Approved in Europe (July 2018) to treat both GL and Partial

Lipodystrophy (‘PL’)▲ Treats the complications of leptin deficiency in patients ▲ Patent protection in US to mid 2027 and orphan exclusivity in

Europe through 2028▲ 2018 Global Revenues: $71.4m

▲Recent European launch provides a significant opportunity for Amryt to accelerate growth

▲Strong fit with Amryt’s EMEA infrastructure

▲Market potential in US and ROW

▲Further growth through potential expansion of label to include PL in the US

What is Lipodystrophy? Lipodystrophy is a chronic condition associated with low leptin levels. Leptin is an important hormone for energy homeostasis and metabolic function. Low leptin can result in metabolic chaos typically resulting in fatty liver, insatiable hunger, chronic fatigue, diabetes often with severe insulin resistance, and severe hypertriglyceridemia leading to a risk of pancreatitis. This may lead to life‐altering organ damage with reduced life expectancy in severe forms.

Enabling metreleptin to realize its full commercial potential

Epidermolysis Bullosa (EB) Franchise: AP101 & AP103

16

EB - AP101 - Lead Development Asset

Strong progress achieved in advancing AP101

What is EB ? –Epidermolysis Bullosa (EB) is a rare genetic skin disorder that leads to extremely fragile skin, and children with the disorder are often referred to as ‘butterfly children’.There are currently no approved treatments for EB.

8

0

4

AP101+ wound dressing

UndecidedWound dressing

alone

Which halfepithelialized faster?

Primary efficacy endpoint

p < 0.01

Wounddressing

alone

RES

ULT

S

0 7 14

AP101 +Wound

dressing

Day

Representative photo series

▲Promotes the differentiation & migration of skin cells to accelerate wound healing

▲Largest ever Global Phase 3 (EASE) patient study for EB currently underway - unblinded interim efficacy analysis delivered Q1 2019

▲Addressable market estimated at >$1bn*

Amryt Proof of Concept Study in EB**

*Management Estimates**Schwieger-Briel A, Dermatology Research and Practice 2017, https://doi.org/10.1155/2017/5068969

Double blind, randomised, placebo controlled, Phase 3, Efficacy and Safety Study of AP101 in Patients with EB*

Placebo + dressingRandomisation 1:1(stratified by EB subtype)

AP101 + dressing

AP101 + dressing

Visit schedule Day0 D14 D30 D60 D90Month 0

M3 M12 M24

90 day main study phase 2 year open label extension

D45

Primary Endpoint: proportion of target wounds healed by day 45*

17

AP101 - EASE Phase 3 Study in EB

Study expected to be fully recruited H2 2019

Unblinded interim efficacy analysis delivered Q1 2019

*Excludes EB Simplex

Milestones Patient studies Non-human studies

Phase 2b

Indication 2017 2018 2019

Phase 3 EB (230 evaluable patients)

EB(AP101)

Follow-up Study

FDA approval

NDA

CTD EMA approval

MAA

CTD

Topline Data

Interim Efficacy Analysis

Non-clinical studies

FPFVFasttrack Designation

Ad Comm

A approval

18

2020

USA IND

AP101 - EASE Timeline

2021

PRV

Pediatric Rare Disease

Designation

Short & medium term value generating clinical milestones

19

AP103 - Gene Therapy Platform

▲Novel non-viral gene therapy for EB

▲Platform gene therapy delivery technology using HPAE polymer

▲Benefits:

– Simple & novel topical application

– Strong efficacy seen in pre-clinical models

– Potential immunogenicity benefit vs viral vector products

– Potentially easier to manufacture than viral vector products

▲Initial focus on Recessive Dystrophic EB (RDEB) with potential

efficacy for other indications

▲2018 - $9.6M DTIF grant from Irish Government*

▲Clinical proof of concept expected by 2023

What is AP103? Exclusively licensed HPAE Polymer Technology for use as a novel gene delivery platform in EB which if successful, could eliminate the requirement for viruses as delivery vectors –and could provide a substantial competitive advantage to Amryt.

Building an EB franchise globally

* Awarded to a consortium of partners comprising Amryt, University College Dublin, Curran Scientific Limited and DEBRA Ireland

Collagen VII Expression -with a single topical application and increased post

three topical applications

20Demonstrated efficacy in pre-clinical EB model

Control C7 Negative

Imag

es

take

n a

t 2

0x

On

e M

on

th

1x HPAE-C7 Topically

3x HPAE-C7 Topically

10

We

eks

Uptake Pathway of the Polyplexes

AP103 - Gene Therapy Platform

Transaction & Summary

Amryt has agreed to acquire Aegerion in an all-paper transaction

– The combined group’s global HQ will be in Dublin, Ireland with its US HQ in Boston, Massachusetts

Amryt to own ~38.6% of pro forma business based on agreed transaction equity valuations (prior to proposed $60m equity financing)

22

Transaction Highlights – Key Acquisition Details

Significantly accelerates Amryt’s growth trajectory

$m Ownership

Amryt Valuation1 $120.0 38.6%

Aegerion Valuation 190.7 61.4%

Pro forma Transaction Equity Valuation1,2 310.7 100%

1. Contingent Value Rights (“CVRs”) will be issued to pre-transaction Amryt stakeholders that could result in the payment of up to an additional $85m (settled in cash or stock) based on certain AP101 milestones being achieved

2. Prior to proposed equity financing.

Amryt plans to raise $60m in equity concurrent with the Transaction close

– Certain Aegerion bondholders have agreed to backstop this equity raise

This equity raise will be placed at a 20% discount to the implied transaction equity valuation

23

Transaction Highlights – Key Financing Terms

Significantly accelerates Amryt’s growth trajectory

$mOwnership

Pre-new equity

Ownership Post-new

equity

Amryt valuation $120.0 38.6% 31.1%

Aegerion valuation 190.7 61.4% 49.5%

Pro forma Transaction Equity Valuation 310.7 100%

Pre-money Equity Valuation (20% discount) 248.7

New equity investment 60.0 19.4%

Post-money Equity Valuation $308.7 100%

Pro forma company will be appropriately capitalized post-Transaction and equity financing

– New debt already negotiated with existing Aegerion stakeholders

Implied post-money enterprise valuation represents 3.4x multiple on 2018 combined built-in revenue

24

Transaction Highlights – Key Acquisition Details

Significantly accelerates Amryt’s growth trajectory

$m

Post-money Equity Valuation $308.7

New Term Debt 81.9

New Convertible Debt 125.0

(Cash)1 (57.0)

Post-money Enterprise Valuation $458.5

1. Assumes $60 million gross proceeds, net of backstop fee. Does not include potential impact of placement fees.

1,208

1,028

301 265 251 164 137 91 51 32 18 1

2018A Sales (in $M)

Amryt + Aegerion - A Significant Player in Rare & Orphan Diseases

25

Underpins significant uplift in value for shareholders

5.6B 6.0B 9.4B 2.7B 1.4B 609M c.458M 2.3B 3.0B 193M 54M 480M

Enterprise Value ($)

Source: Capital IQ, Wall Street Equity Research, and Company filings as of 12/08/19

Accelerates creation of a global leader in rare & orphan diseases

Highly experienced management with proven track record &

unique knowledge of assets

Delivering sustainable revenue, pipeline & market growth

Enhanced scale and significant operating synergies will drive

superior returns & shareholder value

Re-capitalized business with financial flexibility to develop and

launch its own pipeline programs

26

Summary Investment Case

Building a global leader in rare & orphan diseases

Announcement of Transaction - 21 May 2019

Publication of Admission Document - 27 August 2019

Launch of the $60m Equity Fundraise - August 2019

Aegerion Chapter 11 confirmation order – 5 September 2019

Amryt Shareholder Meeting – 19 September 2019

Scheme of Arrangement Completion – 23 September 2019

Acquisition Completion & $60m Fundraise completion – 24 September 2019

Nasdaq listing – expected Q4 2019

27

Indicative Transaction Timetable

Building a global leader in rare & orphan diseases

Appendix

29

Momentum Building - Accelerated & Disciplined Growth

Building a track record of successful execution

2015 2016 2017 2018 2019 +

• Amryt formed

• 1st two acquisitions agreed

• Episalvan (AP101) approved by EMA

• IPO on AIM• €20m EIB

debt facility agreed

• Lojuxta in-licensed

• Largest Global EB Phase III study (EASE) started

• €15m equity fund raise

• Lojuxta-territorial growth & expansion

• AP101 –EASE protocol refined & enhanced

• AP103 Gene therapy platform in-licensed

• EASE Interim efficacy analysis announced Q1

• Announced planned acquisition of Aegerion

30

Lojuxta - Italian Real World Data Demonstrates Significant Efficacy

308

234

620

508470

168

267

843

551

726

242266

212

459

516

69 54 3975

144

25

104

44 41 45

134

231

44

157

280

0

100

200

300

400

500

600

700

800

900

1 2 3 4 5 6 7 8 9 10 11 12 13 14 15

Patient ID

On standard therapy, before addingLojuxta

Lowest level achieved with Lojuxta

• The addition of Lojuxta at the average dosage of 19 mg/day lowered LDL-C levels at the nadir by 76.5 ± 16.7%.

• At their last visit, 60% of patients showed LDL-C<100 mg/dL and 47% <70 mg/dL (more stringent target with cardiovascular disease)

LDL-

C m

g/d

L

A highly effective and clinically proven treatment for HoFH

Target

Source : D’Erasmo et al, “Efficacy of Lomitapide in the Treatment of Familial Homozygous Hypercholesterolemia : Results of a Real World Clinical Experience in Italy” 2017

31

AP103 - Protein Production From RDEB Cells Treated With AP103

• Approximately 5-fold more hCol7 protein is expressed in RDEB keratinocytes after a single AP103 delivery compared with normal keratinocyte endogenous levels of hCol7 protein. These levels are comparable to those delivered by viral methods

• RDEB fibroblasts express approximately 3.5-fold more hCol7 protein compared with normal fibroblast levels

• AP103 delivers between 50 and 70 fold increased mRNA expression of hCol7 compared to normal cells

Proof-of-concept expression of hCol7

AP103 application produced type VII collagen at levels exceeding previously tested non-viral methods, and similar to those following delivery using viral vectors

Treated RDEB cells produced much higher amounts of type VII collagen then seen in healthy cells

No indication of cellular toxicity was seen after treatment with AP103

32

AP103 – Confirmation of Expression & Delivery of hCol7

AP103 confirmation of proof-of-concept