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Company Overview Jefferies 2017 Global Healthcare Conference

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Page 1: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Company OverviewJefferies 2017 Global Healthcare Conference

Page 2: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Forward-Looking Statements

This presentation contains forward-looking statements. All statements other than statements of historical facts included in this presentation are forward-looking statements that are subject to certain risks, trends and uncertainties that could cause actual results and achievements to differ materially from those expressed in such statements. These risks, trends and uncertainties are in some instances beyond our control.

Words such as “anticipate,” “believe,” “estimate,” “expect,” “intend,” “plan,” “will” and other similar expressions identify forward-looking statements, although not all forward-looking statements contain these identifying words. In particular, any statements regarding clinical trial results, potential regulatory approval and expected commercial results for Envarsus® (formerly LCP-Tacro™) are considered forward-looking statements. These forward-looking statements involve substantial risks and uncertainties and are based on our assessment and interpretation of the currently available data and information, current expectations, assumptions, estimates and projections about our business and the biopharmaceutical and specialty pharmaceutical industries in which we operate.

Important factors that may affect our ability to achieve the matters addressed in these forward-looking statements include, but are not limited to; our ability to complete the development of, obtain regulatory approval for, and commercialize, Envarsus®; our ability to hire and retain personnel in a competitive industry; our reliance on third parties to manufacture Envarsus® and to conduct clinical trials for Envarsus®; competition from existing therapies and therapies that are currently under development, including Prograf® (tacrolimus), Advagraf® / Astagraf XL® (tacrolimus), and Nulojix® (belatacept); whether we are able to obtain additional financing, if needed; risks of maintaining protection for our intellectual property; risks of an adverse determination in intellectual property litigation; and risks associated with stringent government regulation of the biopharmaceutical industry. The foregoing is not an exhaustive list of risks that may impact the forward-looking statements used herein.

We may not actually achieve the plans, intentions or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements, which speak only as of the date hereof. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements that we make. We do not have a policy of updating or revising forward-looking statements and, except as required by law, assume no obligation to update any forward-looking statements.

The material presented in this document is not intended for product promotion

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Page 3: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Veloxis Pharmaceuticals: Our Competitive Advantage

• Envarsus XR is an orphan drug with peak potential of $250M-$400M in the US alone

– Orphan Drug Exclusivity through July 2022, patents to 2028

– Highly specialized market

– Robust, broadly applicable clinical data

– Clinically-meaningful differentiation and value message

– Reimbursement and Price

• Led by an established management team with a proven track-record for:

– Commercializing orphan assets with inherent challenges

– Maintaining a high frequency for acquiring, launching and maximizing value of strategic assets

– Rapid turnaround or realization of full value of distressed assets

– Ensuring management process and operational efficiency

Veloxis is an orphan therapeutics company focused on driving worldwide access and

growth of Envarsus XR in organ transplantation and creating value through M&A

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Page 4: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Company Structure & Ownership

Major institutional shareholders:

– Lundbeckfond invest A/S (42%)

– Novo A/S (42%)

Market Cap. ~$270M

Veloxis Pharmaceuticals A/S

Veloxis Pharmaceuticals, Inc.

• Parent company

• Formerly LifeCycle Pharma A/S

• Public Limited Company

• NASDAQ OMX:VELO

• Level 1 ADR NASDAQ USA

• Accessible $250M NOLs

• Wholly owned US subsidiary

• Arm’s length licenses

• APA class transfer price validation

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Page 5: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Management Team

Craig A. Collard

President & Chief Executive Officer

• 23 Years in Industry

• Broad commercial experience

• Founder, Chairman & CEO, Cornerstone Therapeutics

• Board member: BioMarck Pharmaceuticals, Hilltop Home Foundation

Alastair McEwan

EVP & Chief Operating Officer

• 30 Years in Industry

• Broad experience including financial, M&A, GM roles and board appointments in

private and public companies

• Chairman & CEO, Ocean Therapeutics

• Formerly Chairman, CFO, Cornerstone Therapeutics; President, Inveresk

Research; Interim CEO, Averion

Mark Tengler

Chief Technical Officer

• 27 years in the pharmaceutical industry

• Broad experience in Drug Delivery, CMC, Operations, Quality, Regulatory, R&D,

Intellectual Property and fund raising including, IPO

• Former positions include President, Chief Technology Officer, Co-President and

CTO, VP of Business Development and CTO.

• Named Inventor of 12 issued patents

Brett Fleshman

VP, Strategy & Corporate Development

• 17 Years in Industry

• Broad commercial & BD experience in pharma, biotech and medical devices at

companies including Genzyme, Chiesi, Cornerstone Therapeutics and Ther-Rx

Corp.

• Formerly Sr. Director Strategy & Corporate Development, Chiesi USA; Sr.

Director of Marketing, Cornerstone Therapeutics

Ira DuarteVP, Finance and Controller

• 25 years in Accounting and Finance, including 8 years in the Pharma Industry

• Broad experience in SEC regulatory compliance, internal controls/Sarbanes-Oxley,

mergers and acquisitions and financial budgeting, forecasting and analysis

• Formerly Sr Director, Corporate Finance, Chiesi USA; Sr Director, Accounting and

FP&A, Cornerstone Therapeutics; Sr Assurance Manager with Ernst & Young LLP

Stacy WheelerVP, Operations

• 16 Years in Industry

• Global Pharmaceutical Supply Chain, Logistics, Distribution, and Trade Relations

experience at companies including Chiesi, Cornerstone Therapeutics, and Biovail

Pharmaceuticals.

• Formerly Sr. Director, Supply Chain, Chiesi USA; Director, National Accounts &

Supply Chain, Cornerstone Therapeutics

Rob PhiloVP, Market Access

• 22 Years in BioPharm Industry

• Broad sales, Marketing and Managed Care experience in the pharma and biotech

industries at companies including Dura/Elan, Schwarz, HealthPoint and Reckitt

Benckiser (Indivior)

• Former Head of Managed Care and State Government Affairs and Head of Pricing

and Market Access at Indivior, Director of Medicare Part D./Managed Care at

HealthPoint, Director of Sales Training at Dura/Elan

Mark HensleyNational Sales Director

• 10 Years in Industry

• Broad commercial experience in pharma at companies including Chiesi USA and

Cornerstone Therapeutics.

• Formerly Sr. Regional Business Manager, Chiesi USA; National Account Manager,

Cornerstone Therapeutics

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Page 6: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Veloxis management team has proven track record for building

value through company and asset acquisitions

2008

October 2008

Cornerstone BioPharma Holdings

merges with Critical Therapeutics

and changes name to Cornerstone

Therapeutics

May 2009

Announced ~$70M strategic

alliance with Chiesi and gained

U.S. marketing rights for

Curosurf (poractant alfa)

July 2009

Announced agreement to

acquire rights to Factive from

Oscient Pharmaceuticals

December 2011

Announced acquisition of

Cardiokine Inc. and received WW

rights to CRTX080

March 2012

Divested certain product rights

for Factive and Spectracef

2010 2011 20122009

June 2012

Acquired EKR Therapeutics

for approximately $150M

November 2012

Licensed U.S. marketing

rights for Bethkis

(Tobramycin Inhalation

Solution) from Chiesi

2013

May 2013

Licensed U.S. marketing

rights for Pertzye

(pancrelipase) from

Digestive Care, Inc.

2014

February 2014

CRTX Stockholders

Approve Merger with

Chiesi

$25MM investment $300MM sale value6 years of organic and inorganic growth

Timeline for Development and Sale of Cornerstone Therapeutics to Chiesi Farmaceutici

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Page 7: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Product Profile

Novel Transplant Immunosuppressant Growing in Large, Established Markets

Approvals, Orphan Exclusivity & Patents

Progress Towards Global Access

• Once-daily formulation of tacrolimus: the gold-

standard immunosuppressant for transplant

• Proprietary MeltDoseTM technology provides

controlled release over 24 hours throughout the

gastro-intestinal tract

• Effective immunosuppression without toxic peak

blood concentrations from IR-Tac

• FDA approved with Orphan Drug Exclusivity for

conversion from Prograf for prophylaxis against

kidney transplant rejections

• Orphan Drug Exclusivity through July 2022

• EMA approved for de novo and conversion

prophylaxis against both kidney and liver transplant

rejections

• Orange-book listed patents to 2028

Low Risk due to Robust Development

• US Market: $1.9B, EU Market: >$500M

• Competition:

• Prograf® (Astellas): BID, Generics since 2009

• Generics to Prograf®

• Astagraf® (Astellas): QD, Patents to 2019

• Partnered in 43 Countries: EU (Chiesi), China

(Chiesi), MENA (Taiba), and Canada (EVL/Paladin

Labs)

Astagraf XL and Prograf are registered trademarks of Astellas Pharma, Inc.

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Page 8: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Our product, Envarsus XR, is the fastest growing product in the $1.9B

tacrolimus marketplace with peak sales potential of $250M-$400M

Adult Kidney Transplant

Tacrolimus Market (US)250

200

150

100

50

020212016 2022 20242023201920182015 2017 20252020

Ad

ult

Tacro

lim

us

Pati

en

ts (

K)

Tacrolimus market opportunity: ~$1.9B

Envarsus XR US market share target range: 10%-15%

Envarsus XR US peak sales potential: $250M-$400M

15

16

17

18

19

20

2012 2013 2014 2015 2016

New

Kid

ney T

ran

spla

nts

(K

)

New Kidney Transplant Patients (US)

+7%

+5%

+1%+3%

The number of new kidney transplants is

growing at an accelerated rate due to:

• Improved organ allocation system (2015)

• Increased number of deceased donors

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Page 9: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Tacrolimus is the gold standard immunosuppressant in transplant,

but poses challenges as a Narrow Therapeutic Window drug

Overimmunosuppression

can lead to:

• Opportunistic infections

• Nephrotoxicity

• Neurotoxicity

• New onset diabetes

• Impact on long-term graft survival

Underimmunosuppression

can lead to:

• Chronic rejection

• Acute rejection

Unmet need for controlled delivery of tacrolimus

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Page 10: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Envarsus XR is effective once-daily tacrolimus which solves

the critical unmet need

1

• ENVARSUS XR demonstrates an optimal pharmacokinetic profile compared with

standard of care (Prograf® or generic IR-TAC)

– 30% lower peak concentration compared to Prograf

– Eliminates the high initial peak associated with Prograf

– Similar AUC and overall exposure to achieve required efficacy

Envarsus XR PK Profile

30% lower

peak concentration

Prograf is a registered trademark of Astellas Pharma, Inc.

Page 11: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Generic variability is a major concern among prescribers of

tacrolimus, who demonstrated unprecedented resistance to

generic alternatives to Prograf®

0%

10%

20%

30%

40%

50%

60%

70%

80%

90%

100%

3/1

/20

09

6/1

/20

09

9/1

/20

09

12

/1/2

00

9

3/1

/20

10

6/1

/20

10

9/1

/20

10

12

/1/2

01

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3/1

/20

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6/1

/20

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9/1

/20

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/1/2

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3/1

/20

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/20

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/20

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/1/2

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3/1

/20

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/20

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/20

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/20

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6/1

/20

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9/1

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/20

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/20

17

Prograf® Generics

Prograf is a registered trademark of Astellas Pharma, Inc.

27%5 years

after GxLaunch

55%1 year

after GxLaunch

Unmet need for controlled delivery of tacrolimus

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Page 12: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Medication non-adherence is associated with significant

consequences following kidney transplantation

Unmet need for controlled delivery of tacrolimus

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Page 13: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Large Unmet Need for Envarsus XR:

1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus at

higher risk of rejection and treatment failure, $600M opportunity

Carriers of the CYP3A5*1 gene are “Rapid

Metabolizers” of tacrolimus

1. Require higher tacrolimus doses to achieve

therapeutic tacrolimus trough concentrations

2. At risk of underimmunosuppression

Analysis from phase 3 trials in African-American kidney

transplant recipients showed fewer treatment failures

for Envarsus® compared to twice-daily Prograf®

All ethnicities are potential carriers of the CYP3A5*1

allele, with African Americans at the greatest risk:

– Caucasians (5%–15%)

– Asians (15%–35%)

– Hispanics (13%-26%)

– African-Americans (45%–80%)

Envarsus XR solves the unmet need for Rapid

Metabolism of tacrolimus, by providing controlled

release over 24 hours throughout the GI tract

Wh

ole

Blo

od

Co

nc

en

tra

tio

n (

ng

/mL

)

Prograf is a registered trademark of Astellas Pharma, Inc.

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Page 14: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Envarsus XR launch momentum in US shows deep penetration

into transplant centers, steady growth in unique prescribers and a

critical mass of prescriptions

14

63%of US adult transplant centers

have placed patients on

Envarsus XR since launch

prescribers have generated

prescriptions since launch

477 16,140prescriptions have been

generated since launch

patients are estimated to be currently on Envarsus XR

in the US1,850

Page 15: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

63% of transplant centers have already utilized Envarsus XR

127 of 198U.S. Adult Transplant Centers have

placed patients on Envarsus XR in the 18

months since launch (December 2015)

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Page 16: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

68% of conversions to Envarsus XR are from generic tacrolimus,

and the number of patients switching from once-daily Astagraf XL®

is 8x the incumbent product’s current 1% market share

Astagraf XL®

(Once-Daily

Tacrolimus)

8%

Cyclosporine Drugs

3%

Prograf® (Tacrolimus)

19%

Non-CNI Drugs

(Sirolimus, Everolimus)

2%

Generic Tacrolimus

68%

Sources of Conversions to Envarsus

Astagraf XL and Prograf are registered trademarks of Astellas Pharma, Inc.

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Page 17: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

As a transplant immunosuppressant for patients with ESRD,

Envarsus XR benefits from mandatory Medicare coverage

64%

32%

4%

Current Envarsus XR Payer Mix

(Primary Coverage)

Medicaid & Other

MedicareCommercial/

Cash

Payer Access

75%of prescriptions are

approved by payers

J7503Unique HCPCS J Code

17

Pharmacy/Distribution

74% of prescriptions are

dispensed through

Specialty Pharmacies &

Transplant Outpatient

Pharmacies

Page 18: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Envarsus EU update: Strong uptake continues

As of Q1 2017, an estimated 4,150 patients are on Envarsus in 18 European/

CIS countries

*Note: Use in de novo patients is included in the Envarsus label in Europe

Patients on Envarsus by European/CIS Country

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Page 19: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Envarsus XR Now Licensed in 43 Countries

Canada

Licensed June 2017 to Endo Ventures

Limited for commercialization by Paladin

Labs Inc.

Upfront payment: $1.25M USD

MENA

Licensed in March 2017 to Taiba Healthcare

for 14 countries in Middle East/North Africa

Upfront payment: $1M USD

Europe/CIS & China

Europe/CIS licensed in October 2012, and

China licensed June 2017 to Chiesi

Farmaceutici S.p.A.

Upfront payment for China: $6M USD

Available regions/countries with active

licensing efforts:

• Latin America

• ASEAN countries

• Japan

• Korea

• Israel

• South Africa19

Page 20: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Veloxis Corporate Strategy

Grow Envarsus &

acquire strategic

products

Transform Veloxis

through a merger,

IPO or large

acquisition

Grow Veloxis through

continued strategic

acquisitions

Acquire for synergy in transplant or

chronic therapy

Maintain operational efficiency

Create sustainable cash positive

business

Large strategic or transformative

acquisition

Access NOLS

Domicile in lower tax jurisdiction

Normalize investor base

Establish liquidity

Grow investor base

Create multiple layers for arbitrage

Continued aggressive M&A execution

Investments in key growth drivers

including pipeline

Broaden Specialty focus

All acquisitions to focus around

Companies Competitive advantage

Grow company value and arbitrage

points

Imp

act

Act

ion

Company

profitable in 2018

20

Page 21: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Financial Highlights

21

Three Months Ending March 31, 2017 USD’000

Revenue

Europe 428

United States 2,572

Licensing (upfront) 1,000

Total revenue 4,000

Gross profit 3,109

Operating result (loss) (5,223)

Net loss (5,891)

Cash and cash equivalents 7,243

Loan facility (22,000)

Page 22: Company Overview - Jefferies Wheeler VP, Operations • 16 Years in Industry ... Large Unmet Need for Envarsus XR: 1 in 3 transplant recipients are Rapid Metabolizers of tacrolimus

Summary

• Veloxis is a commercial-stage orphan therapeutics company with all components to

support future growth

– Supported by high-quality major institutional investors

– Led by cohesive management team with a proven track record for growth and M&A

– Well-differentiated lead asset launched in a $1.9B market

– Strong performance demonstrated in both US and EU markets

• Substantial focus on identifying and executing on the best option for company

transformation (e.g. merger, IPO or large transaction) to meet strategic objectives

– Normalize investor base and reach US markets

– Optimize shareholder value from organic growth and future transactions

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