Biotech Showcase, San Francisco, 2014-01-15 Jacques Näsström, CEO, PhD, MBA

PledPharma AB Creating value within treatment of

life-threatening diseases

MATTERS DISCUSSED IN THIS PRESENTATION CONTAIN FORWARD-LOOKING STATEMENTS WITHIN THE MEANING OF THE PRIVATE

SECURITIES LITIGATION REFORM ACT OF 1995. WHEN USED IN THIS DOCUMENT, THE WORDS "ANTICIPATE," "BELIEVE," "ESTIMATE,"

"MAY," "INTEND," "EXPECT" AND SIMILAR EXPRESSIONS IDENTIFY SUCH FORWARD-LOOKING STATEMENTS. THESE FORWARD-LOOKING

STATEMENTS ARE SUBJECT TO RISKS, UNCERTAINTIES, ASSUMPTIONS, AND OTHER FACTORS THAT MAY CAUSE THE ACTUAL RESULTS,

PERFORMANCE OR ACHIEVEMENTS TO DIFFER MATERIALLY FROM THOSE CONTEMPLATED, EXPRESSED OR IMPLIED BY SUCH FORWARD-

LOOKING STATEMENTS. THESE FACTORS INCLUDE, BUT ARE NOT LIMITED TO, RISKS AND UNCERTAINTIES ASSOCIATED WITH THE

IMPACT OF ECONOMIC, COMPETITIVE AND OTHER FACTORS AFFECTING THE COMPANY AND ITS OPERATIONS, MARKETS, PRODUCTS

AND CUSTOMERS, AND OTHER FACTORS DETAILED IN PERIODIC REPORTS FILED BY THE COMPANY WITH THE SEC. THIS PRESENTATION

CONTAINS PROJECTIONS RELATED TO OUR FUTURE OPERATIONS, WHICH WERE PREPARED BY US AND THEY ARE SUBJECT TO

UNCERTAINTY AND A COMPLEX SERIES OF FUTURE EVENTS BOTH INTERNAL AND EXTERNAL.

SAFE HARBOR STATEMENT

•  Swedish pharma company with emphasis on oxidative stress and life-threatening diseases

•  Lead compound: PledOx® for prevention of severe chemotherapy induced side-effects

•  Projects for other life-threatening diseases in development

•  Clinically established and proven class of compounds

–  Reduced clinical and regulatory risk

•  Favorable patent situation

•  Listed on NASDAQ OMX First North (STO:PLED)

PledPharma company highlights

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Alexandre et al. mangafodipir

protects white blood cells against chemotherapy

Doroshov JNCI – mangafodipir should be evaluated in humans

The history of calmangafodipir and PledPharma

2006 2010 2008 2012 2007 2009 2011 2013

PledPharma founded

PP license patents

MANFOL study MPA approved

MANAMI study MPA approved

Results

MANFOL

Listing First North PledOx / calmangafodipir

discovered

PLIANT-study MPA approved Patent application calmangafodipir

3 key publications Translational Oncology

FDA approves PLIANT First patient treated

Anti-cancer patent approved in US

Anti-

cancer patent

application

Patent application new composition PLED-substances

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Oxidative stress can lead to organ damage

”Oxidative stress” Cell and

Organ Damage

Cancer

Cardiovascular disease

Radiation

Liver disease

Neurological disease

Inflammatory disease

Chemotherapy 2

-

4

•  Third most common cause of cancer-related death in the Western world. o  ~ 75-80% of all new cases are cured by surgery ±

chemo

o  Majority of these patients survive 5 year (~70-90%)

•  FOLFOX is the leading first-line chemotherapy (FOLinic acid, 5-Fluorouracil (5-FU), and OXaliplatin)

Colorectal cancer (CRC) is a common and treatable cancer

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Severe side-effects* of the chemotherapeutic regimen FOLFOX:

However, colorectal cancer treatment causes severe side-effects

Sources: *Sanofi-Aventis; Leonard et al, BMC Cancer. 2005; 5: 116; American Cancer Society

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•  Only symptomatic treatments exist for peripheral sensory nerve damage (no approved therapies) –  Risk of chronic condition

•  Drugs against loss of white blood cells –  No pretreatment, limited use with risk of side-effects

•  Reduced or postponed chemotherapy dosing or completely discontinued therapy if severe side-effects persist –  Can lead to lead to sub-optimal treatment results

Ø Huge medical need for preventive treatment of chemotherapy induced side-effects

Management of chemotherapy-induced side-effects is currently unsatisfactory

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Pretreatment with PledOx can reduce chemotherapy induced side-effects

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PledOx

•  14 patients with metastatic colorectal cancer

•  Scheduled for 12 treatment cycles with FOLFOX

•  During the first 3 cycles the patients received pretreatment with placebo or the PLED-derivative mangafodipir

•  Number of adverse events registered according to National Cancer Institute’s scale

MANFOL I – study: phase IIa study in colorectal cancer patients

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•  Adverse events (AEs) occuring during the first 3 FOLFOX cycles •  Treated patients show statistically significant reduction in grade 3/4

adverse events

Effect of PLED-substance shown in MANFOL I phase IIa study

* National Cancer Institute’s Common Terminology Criteria for Adverse Events version 3 Karlsson JO, et al. Transl. Oncol. 2012;5:32-38.

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Note the lack of grade 3/4 AEs in treated patients

Clinical development has encompassed a rational transition of lead compounds

All further studies: PledOx

MANFOL I – study: phase IIa study in colorectal cancer

patients: Mangafodipir

Rat

iona

l che

mic

al

mod

ifica

tion

•  Developed from mangafodipir, but with potential for better efficacy and better safety

•  At equivalent Mn2+ doses, PledOx was found to be at least 10x more potent than mangafodipir

•  Completed dose-escalation in ongoing Phase IIb trial showed no safety or toxicity issues

•  Pending compositional matter patent until 2032

•  Clinically proven MRI contrast medium

•  A low Molecular Enzyme Mimetic (lowMEM), MnSOD and iron chelator

•  Good safety profile (based on >40,000 patients treated with mangafodipir and several tox studies)

•  Effect of PledOx® pretreatment on FOLFOX-induced side-effects –  Patients with metastatic colorectal cancer (mCRC) –  Two doses of PledOx with placebo control

•  Primary endpoint: neutropenia, or reduction in white blood cells (neutrophils)

•  Secondary endpoints include: –  Peripheral sensory nerve damage – validated scales as well as pain and

discomfort –  Infection due to loss of white blood cells (febrile neutropenia)

•  6+3 (FOLFOX + Ab) in open dose-escalation directly followed by 126 patients in randomized part

•  First patients treated and PledOx well-tolerated •  Study is a European and US multicenter study

PledOx® phase IIb study PLIANT

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•  PledOx will become a standard addition to prevent severe chemotherapy-induced side-effects* of chemotherapy, including :

-  Neuropathy -  Neutropenia -  Oral mucositis -  Thrombocytopenia

•  Huge potential for use in many other chemotherapies and in other cancers –  More efficacious and and broader effect than

existing treatments –  Unique mechanism of action

•  PledPharma goal: maximizing the value of PledOx to patients and shareholders

PledOx® goals

Sources: *Sanofi-Aventis; Leonard et al, BMC Cancer. 2005; 5: 116; American Cancer Society

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Asset is administered per cycle of chemotherapy and has demonstrated efficacy in a broad range of tumor types and chemotherapy regimens

(Asset is expected to have efficacy in all tumors/regimens shown here)

Tumor Incidence1

Thou

sand

s of P

atie

nts

0

0,5

1

1,5

2

2,5

3

3,5

US EU5 JP

Paclitaxel

Docetaxel

Cisplatin

Carboplatin

Oxaliplatin

# Cycles of Chemotherapy Agents2

Mill

ions

of C

ycle

s

-

200

400

600

US EU5 JP

Ovarian

Lung

Breast

Pancreatic

CRC

1SEER, EUCAN, GLOBOCAN 2IMS Data on unit sales of chemotherapy agents with regimen assumptions applied; no data for cisplatin in JP

SIZE OF PATIENT POPULATION ADDRESSABLE BY ASSET

14 PledOx’s validated approach has potential to address a large patient population

PledOx is expected to achieve prices in line with G-CSF pricing

PRICE LIKELY TO BE ACCEPTED PRICE NOT LIKELY TO BE ACCEPTED

PRICE WILL BE ACCEPTED

$-

$1

$2

$3

$4

$5

Pric

e pe

r cyc

le (t

hous

ands

US

D)

Price sensitivity

ESTIMATED PRICE RANGES BY MARKET (PRICE/CYCLE)

Sources: IMS Primary Research, IMS Expertise

*DE: net price negotiation, not list price

*

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De-risked

PledOx has demonstrated proof-of-concept in the clinic, validating robust pre-clinical evidence of efficacy and safety

Addresses Major Clinical Unmet Need

Prospective first-in-class agent for the reduction of the incidence and severity of chemotherapy-induced neuropathy, as well as other chemotherapy-induced side-effects

Huge Commercial Opportunity

Due to the large size of the patient population and favorable pricing and reimbursement opportunity, asset presents a huge commercial upside to a potential partner

16 PledPharma is open to discussing a potential partnership for PledOx

PledPharma has also developed an asset to improve outcomes in Acute Myocardial Infarction (AMI)

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ST-Elevated Myocardial Infarction (STEMI) is a segment of patients with acute coronary syndrome

Sources: O’Gara et al., Circulation 2013

•  STEMI patients are the subset of MI patients defined by an electrocardiogram (ECG) test as well as biomarker release and myocardial necrosis

•  In STEMI, the coronary artery is completely blocked off by the blood clot

•  STEMI affects a large portion of the heart muscle and is the deadliest form of MI

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Balloon angioplasty or PCI is the treatment of choice for STEMI patients

Percutaneous Coronary Intervention (PCI) or balloon angioplasty

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Despite treatment options, mortality due to MI is still a problem

Schmidt M et al. BMJ 2012;344:bmj.e356

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Standardised 30 day and 31–365 day mortality after first hospitalization for myocardial infarction among men and women between 1984 and 2008

Reducing infarct size and increasing ejection fraction should lead to reduced morbidity

Burns RJ et al. JACC 2002;39:30-6

•  Highly significant correlation between ejection fraction (EF) and infarct size (IS)

•  Statistically significant increase in 6-month mortality with EF <30% or IS > 35%

•  Therapeutic intervention should aim at increasing EF and reducing IS

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The oxygen paradox: oxygen can be both ameliorative and harmful in treating MI

Yellon DM and Hausenloy DJ, NEJM 2007;357:1121-35

•  Reperfusion therapy has been widely employed due to its positive impact on short and long-term outcomes in MI patients

•  However, oxidative stress in mitochondria resulting from reperfusion treatment can induce serious and even lethal consequences

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PLED-substance in acute myocardial infarction

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Karlsson JOG et al. Acta Radiol. 2001;42:540-7

Promising results have been seen in a Phase IIa study in MI patients (MANAMI)

•  20 patients (10 active, 10 placebo) •  Promising study results:

–  Patients tolerated i.v. infusion of PLED-compound without any side-effects

We are currently evaluating the commercial potential of the AMI indication

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Increased ejection fraction shown in treated patients despite an unfavorable initial status

148

208

0

50

100

150

200

250

Control Treated

Tim

e (M

inut

es)

Duration of ischemia prior to treatment

Despite a less favorable prognosis prior to treatment

PP-99 treated patients fared better in EF and other key outcomes

42% 48%

0%

10%

20%

30%

40%

50%

60%

Control Treated

Left

vent

ricul

ar E

F

Ejection fraction post-treatment

Statistically significant difference in duration of

ischemia prior to treatment

But still numerically superior outcomes for PP-99 treated

patients

* p<0.05

•  Four granted patent families –  Basic patents in 2017, EP, US and JP –  Two additional in 2018, EP and US –  Use of PLED-derivatives as pharmaceuticals for the treatment of oxidative stress

related conditions

•  Three patent applications/patents –  Anticancer effect of certain PLED derivatives, 2028, PCT application in national phase

with approved US, Chinese and Russian patent plus a US CIP (continuation in part) –  Manganese and non-manganese compositions and broad therapeutic use of these

new compositions, 2030, PCT application in national phase with approved South African patent

–  Compositional matter, manufacturing process and broad therapeutic use of PledOx, 2032, PCT

•  Trademarks

–  PledOx® registered trademark in EU, US, Switzerland, Australia and Japan as well as pending in China, Russia and Norway

Overall, PledPharma has a unique and robust IP portfolio

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•  PledPharma’s clinical projects are all built on a clinically tested compounds –  Reduced clinical, technical, and regulatory risk

•  Favorable IP position –  Phase IIb project with patent application for compositional matter with

patent life up until 2033, when approved

•  First eight patients in Phase IIb treated with PledOx®

•  Cash: 55 MSEK (Oct 2013) •  Out-licensing activities will be intensified in 2014

–  Promising MANAMI results presented

–  Result from PLIANT open dose-escalation expected early 2014

–  Additional PLED-opportunity currently under evaluation

–  Top-line Phase IIb PLIANT results expected mid 2014

•  Large commercial potential, limited competition

Summary and news flow

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For more information please visit: www.pledpharma.se

Contact information Jacques Näsström CEO jacques.nasstrom@pledpharma.se

Michaela Gertz CFO michaela.gertz@pledpharma.se

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