PledPharma AB Creating value within treatment of life-threatening diseases

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1 PledPharma AB Creating value within treatment of life-threatening diseases Biotech Showcase, San Francisco, Jacques Näsström, CEO, PhD, MBA

2 SAFE HARBOR STATEMENT MATTERS DISCUSSED IN THIS PRESENTATION CONTAIN FORWARD- LOOKING STATEMENTS WITHIN THE MEANING OF THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 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.

3 2 PledPharma company highlights 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)

4 The history of calmangafodipir and PledPharma 3 PledPharma founded PP license patents MANFOL study MPA approved MANAMI study MPA approved Patent application new composition PLED-substances Listing First North PledOx / calmangafodipir discovered FDA approves PLIANT First patient treated Anti-cancer patent approved in US Alexandre et al. mangafodipir protects white blood cells against chemotherapy Doroshov JNCI mangafodipir should be evaluated in humans Anticancer patent application Results MANFOL PLIANT-study MPA approved Patent application calmangafodipir 3 key publications Translational Oncology

5 4 Oxidative stress can lead to organ damage Cancer Cardiovascular disease Radiation Chemotherapy Liver disease Oxidative stress - 2 Cell and Organ Damage Neurological disease Inflammatory disease

6 Colorectal cancer (CRC) is a common and treatable cancer 5 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)

7 However, colorectal cancer treatment causes severe side-effects 6 Severe side-effects* of the chemotherapeutic regimen FOLFOX: Sources: *Sanofi-Aventis; Leonard et al, BMC Cancer. 2005; 5: 116; American Cancer Society

8 Management of chemotherapy-induced side-effects is currently unsatisfactory 7 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

9 Pretreatment with PledOx can reduce chemotherapy induced side-effects 8 PledOx

10 MANFOL I study: phase IIa study in colorectal cancer patients 9 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

11 Effect of PLED-substance shown in MANFOL I phase IIa study 10 Adverse events (AEs) occuring during the first 3 FOLFOX cycles Treated patients show statistically significant reduction in grade 3/4 adverse events * National Cancer Institute s Common Terminology Criteria for Adverse Events version 3 Karlsson JO, et al. Transl. Oncol. 2012;5: Note the lack of grade 3/4 AEs in treated patients

12 Clinical development has encompassed a rational transition of lead compounds MANFOL I study: phase IIa study in colorectal cancer patients: Mangafodipir All further studies: PledOx 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) Rational chemical modification Developed from mangafodipir, but with potential for better efficacy and better safety At equivalent Mn 2+ 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

13 12 PledOx phase IIb study PLIANT 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

14 13 PledOx goals PledOx will become a standard addition to prevent severe chemotherapy-induced sideeffects* 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 Sources: *Sanofi-Aventis; Leonard et al, BMC Cancer. 2005; 5: 116; American Cancer Society

15 PledOx s validated approach has potential to address a large patient population 14 SIZE OF PATIENT POPULATION ADDRESSABLE BY ASSET Tumor Incidence 1 # Cycles of Chemotherapy Agents 2 Thousands of Patients Ovarian Lung Breast Pancreatic CRC Millions of Cycles 3,5 3 2,5 2 1,5 1 Paclitaxel Docetaxel Cisplatin Carboplatin Oxaliplatin 0,5 - US EU5 JP 0 US EU5 JP 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) 1 SEER, EUCAN, GLOBOCAN 2 IMS Data on unit sales of chemotherapy agents with regimen assumptions applied; no data for cisplatin in JP

16 PledOx is expected to achieve prices in line with G-CSF pricing 15 ESTIMATED PRICE RANGES BY MARKET (PRICE/CYCLE) Price per cycle (thousands USD) $5 $4 $3 $2 $1 $- * Price sensitivity PRICE NOT LIKELY TO BE ACCEPTED PRICE LIKELY TO BE ACCEPTED PRICE WILL BE ACCEPTED *DE: net price negotiation, not list price Sources: IMS Primary Research, IMS Expertise

17 PledPharma is open to discussing a potential partnership for PledOx 16 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

18 PledPharma has also developed an asset to improve outcomes in Acute Myocardial Infarction (AMI) 17

19 ST-Elevated Myocardial Infarction (STEMI) is a segment of patients with acute coronary syndrome 18 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 Sources: O Gara et al., Circulation 2013

20 Balloon angioplasty or PCI is the treatment of choice for STEMI patients Percutaneous Coronary Intervention (PCI) or balloon angioplasty 19

21 Despite treatment options, mortality due to MI is still a problem 20 Standardised 30 day and day mortality after first hospitalization for myocardial infarction among men and women between 1984 and 2008 Schmidt M et al. BMJ 2012;344:bmj.e356

22 Reducing infarct size and increasing ejection fraction should lead to reduced morbidity 21 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 Burns RJ et al. JACC 2002;39:30-6

23 The oxygen paradox: oxygen can be both ameliorative and harmful in treating MI 22 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 Yellon DM and Hausenloy DJ, NEJM 2007;357:

24 PLED-substance in acute myocardial infarction 23 Karlsson JOG et al. Acta Radiol. 2001;42:540-7

25 Promising results have been seen in a Phase IIa study in MI patients (MANAMI) patients (10 active, 10 placebo) Promising study results: Patients tolerated i.v. infusion of PLEDcompound without any side-effects We are currently evaluating the commercial potential of the AMI indication

26 Increased ejection fraction shown in treated patients despite an unfavorable initial status Despite a less favorable prognosis prior to treatment Duration of ischemia prior to treatment PP-99 treated patients fared better in EF and other key outcomes Ejection fraction post-treatment Time (Minutes) * p< Left ventricular EF 60% 50% 40% 30% 20% 10% 42% 48% 0 Control Treated 0% Control Treated Statistically significant difference in duration of ischemia prior to treatment But still numerically superior outcomes for PP-99 treated patients

27 Overall, PledPharma has a unique and robust IP portfolio 26 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

28 27 Summary and news flow 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

29 For more information please visit: 28 Contact information Jacques Näsström CEO Michaela Gertz CFO

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