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Karolinska Development
2Q 2012 Conference Call – August 23, 2012
Torbjörn Bjerke, CEO
Today’s Presentation

          Second Quarter Highlights

          Selected Portfolio Companies

          Financial Information

          Summary




2
Karolinska Development in Summary

      Exclusive access to top class medical innovations
      First-in-class drug candidates with multi billion dollar
       sales potential
      Significant clinical progress – 14 projects in clinical
       phase
      Large portfolio of companies removes binary risk –
       26 companies, 35 projects
      Cash position provides funding through major
       clinical data
      Strong focus on business development activities
       across the portfolio


3
Highlights During and Since the Second Quarter 2012

     Pharmanest initiated Phase I and Phase II trials with SHACT, a product
      developed for pain relief in connection with IUD insertion
     Karolinska Development AB and Industrifonden signed a share swap
      agreement whereby Industrifonden will exchange Karolinska Development
      AB’s shares in Oncopeptides AB for its shares in Aprea AB
     Pergamum reported top-line Phase II-data from the clinical trial with PXL-01
      for prevention of post-surgical adhesions
     Strengthened Management team in Karolinska Development:
        – Dr Gunilla Ekström recruited as VP Operations
        – Ann-Sofie Sternås recruited as VP IPR
     Akinion Pharmaceuticals announced the publication of preclinical results for
      AKN-028 in highly cited scientific journal



4
The Portfolio has a Significant Market Potential
                                                                 Concept        Lead         Lead        Preclinical
                                                  Ownership*                                                           Phase I   Phase II   Phase III     Launch
       PHARMACEUTICALS                                         development   discovery   optimization   development


       ONCOLOGY
       Axelar AB                                    50%
       Aprea AB                                     41%
       Oncopeptides AB                              43 %
       KDev Oncology AB (AKN-028)                   90%
       KDev Oncology AB (GliGene)                   44%

       DERMATOLOGY
       Pergamum AB (DPK-060, atopic dermatitis)      62%
       Pergamum AB (DPK-060, external otitis)        62%
       Pergamum AB (PXL01)                           62%
       Pergamum AB (LL-37)                           62%

       WOMEN’S HEALTH
       Dilafor AB                                    55%
       Pharmanest AB                                 59%
       Umecrine Mood AB                              43%

       INFECTIOUS DISEASE
       Dilaforette AB                                62%
       Biosergen AS                                  60%

       CARDIOVASCULAR
       Athera Biotechnologies AB (Annexin A5)        65%
       Athera Biotechnologies AB (PC-mAb)            65%

       CNS
       BioChromix Pharma AB                          70%
       Umecrine Cognition AB                         54%

       INFLAMMATION
       NovaSAID AB                                   89%
       ProNoxis AB                                   22%

       OPHTHALMOLOGY
       Clanotech AB                                  88%                                                                                            Solid colored area = completed phase
                                                                                                                                                    Shaded colored area = ongoing phase
                                                     *Including indirect ownership. Ownership in Axelar and GliGene shown after
                                                      all tranches in recent investment round. All shares in Oncopeptides will be
                                                       exchanged for Industrifondens shares in Aprea. Ownership in Aprea after
                                                                          share swap transactiona will be 69%.
5
The Portfolio has a Significant Market Potential



    TECHNOLOGY                              Ownership*   Concept development     Prototype           Development      Product               Sales

    IMPLANTS
    Promimic AB                                28%
    Oss-Q AB                                   16%

    DIAGNOSTICS
    Athera Biotechnologies AB (CVDefine®)      65%
    BioChromix AB                              14%

    PHARMACEUTICAL FORMULATION
    Inhalation Sciences Sweden AB              72%
    XSpray Microparticles AB                   62%
    Lipidor AB                                 46%

    MEDICAL EQUIPMENT
    NeoDynamics AB (Fourier/AS)                21%
    NeoDynamics AB (Therapy/PRFA)              21%




                                                                                                                   Solid colored area = completed phase
                                                                                                                   Shaded colored area = ongoing phase



                                                                     *Including indirect ownership



6
Portfolio Focus in 2012 and 2013
                      Axelar – AXL1717                                      Phase I/II data                   
                      Axelar – AXL1717 for NSCLC                            Initiate Phase II                 
                      Akinion – AKN-028 for AML                             Initiate clinical studies         
          ONCOLOGY
                      Aprea – APR-246                                       Complete Phase I/II
                      Akinion – AKN 028                                     Complete Phase I/II
                      Axelar – AXL1717 for NSCLC                            Complete Phase II

                      Pergamum – PXL01 for surgical adhesion                Complete enrolment of Phase II    
                      Pergamum – DPK-060 for external otitis                Initiate Phase II                 
       DERMATOLOGY    Pergamum – DPK-060 for skin infection                 Preparation for Phase IIb
                      Pergamum – LL-37 for chronic wounds                   Initiate clinical studies
                      Pergamum - DPK-060 for external otitis                Complete Phase II


                      Pharmanest – SHACT for pain relief at IUD insertion   Initiate Phase I                  
                      Pharmanest – SHACT for pain relief at IUD insertion   Initiate Phase II                 
     WOMEN’S HEALTH   Dilafor – Tafoxiparin for protracted labor            Initiate Phase IIb with partner
                      Pharmanest – SHACT for pain relief at IUD insertion   Complete Phase I and II
                      Umecrine Mood – Severe PMS and PMDD                   Initiate Phase I/II

                      Dilaforette – Sevuparin for malaria                   Initiate Phase I/II               
             OTHER
                      Dilaforette – Sevuparin for malaria                   Complete Phase I/II




7
Anticipated Major Clinical Data

                                    Phase I data             LL-37 Phase I data




     PXL01 Phase II data           DPK-060 Phase II data             Phase II data
                                                                                      Phase II data
                                                                        in NSCLC




    2012                                                    2013                                                  2014



                               Phase I data         Phase I/II data                                   Phase I/II data




                                                                                                                         Tafoxiparin Phase IIb data




8
Significant Deal Values in Our Focus Areas

                                                                                                 Therapeutic                                            Total Deal
    Date                Licensor                        Licensee                                                 Product               Phase at Deal
                                                                                                 Area                                                  Value (USDm)
    February 16, 2011   AVEO Pharmaceuticals, Inc.      Astellas Pharma, Inc.                    Oncology        AV951                 Phase II           1480

    February 29, 2012   Galapagos NV                    Abbott Laboratories                      Inflammatory    GLPG0634              Phase II           1350

    November 10, 2009   Alder Biopharmaceuticals Inc.   Bristol-Myers Squibb Company             Inflammatory    ALD518                Phase II           1049

    December 8, 2011    Pharmacyclics, Inc.             Janssen Biotech, Inc.                    Oncology        PCI32765              Phase II            975

    September 3, 2009   Algeta ASA                      Bayer Healthcare Pharmaceuticals, Inc.   Oncology        Alpharadin            Phase II            800

    December 21, 2009   Incyte Corporation              Eli Lilly & Co                           Inflammatory    INCB28050             Phase II            755

    February 12, 2009   Portola Pharmaceuticals Inc.    Novartis AG                              CVD             PRT128                Phase II            575

    May 4, 2011         Molecular Partners AG           Allergan Inc.                            Ophthalmology   MP0112                Phase II            420

    September 5, 2011   Evotec AG                       Roche Holding, Ltd.                      CNS             EVT302, RG1577        Preclinical         830

                                                        Janssen Pharmaceuticals Inc./
    June 1, 2010        Orexo AB                                                                 Respiratory     OX-CLI, OX-ESI        Preclinical         586
                                                        Johnson & Johnson

    June 8, 2011        Affectis Pharmaceuticals AG     Merck Serono S.A.                        CNS             P2X7 Antagonist       Preclinical         409

    January 1, 2012     Virobay, Inc.                   LEO Pharma A/S                           Dermatology                           Preclinical         307

                                                                                                                 MP0260,
    August 21, 2012     Molecular Partners AG           Allergan Inc.                            Ophthalmology                         Discovery          1400
                                                                                                                 eye disease DARPins

    December 20, 2010   Avila Therapeutics, Inc.        Sanofi                                   Oncology                              Discovery           964

    January 5, 2012     Forma Therapeutics, Inc.        Boehringer Ingelheim Corporation         Oncology                              Discovery           815




9
Selected Portfolio Companies




10
AXL1717 - Block Buster Potential in Lung Cancer                                                                 Ownership: 50%


                                                                       12
                                                                            Avastin, Tarceva and Iressa Sales (USDbn)
          Company:              Axelar AB                              10

          Active ingredient:    Small molecule                         8
                                                                       6
          Primary indication:   Non Small Cell Lung Cancer
                                                                       4
          Current Phase:        Phase II
                                                                       2
                                                                       0




      Status                                                      Market Overview
           The only small-molecule IGF-1R inhibitor in man           Around 420 000 new cases of NSCLC in the
            without insulin receptor inhibition                        industrialized countries each year
           Unique preclinical efficacy                               UBS estimated total US lung cancer sales in 2010 for
           Promising Phase I/II data                                  Avastin at USD 1150m, Tarceva at USD 400m, and
           Phase II ongoing – Top-line data expected in the           Iressa at USD 310m
            beginning of 2013
           Next indication Glioblastoma
                                                                                                         Source: Datamonitor
                                            AXL1717 - Potential in Multiple Cancers


11
AXL1717 – Promising Phase I/II-data (March 30 Update)                                                                                                                      Ownership: 50%



      SAFETY
                                                                             Progression-free survival and overall survival of 15 patients with progressive and refractory NSCLC treated with single
      AXL1717 was well-tolerated with reversible neutropenia                 agent AXL1717 in 3rd or 4th line. Patients with a treatment duration longer than 14 days. Status per March 30, 2012
      as dose limiting toxicity
      - Even at exposures 5-10 fold higher than those needed
        for anti-tumor activity in animals.
      - No changes in blood levels of glucose, insulin or
        C-peptide were reported.

      CLINICAL BENEFIT
      For the NSCLC patients treated for 14 days or longer (Mar 30, 2012):
      Median survival 60 weeks
      Median time to progression 31 weeks
      One Partial Response according to RECIST




12
Normalizing p53 to Overcome Drug Resistance                                                                                                                   Ownership: 41%*



                                                                               Platinum refractory patients in ovarian cancer (US)

      Company:               Aprea AB                               First Line Treatment (60500 patients)                       86%                          14%
      Active ingredient:     Small molecule
                                                                 Second Line Treatment (30500 patients)                 58%                          42%
      Primary indication:    Ovarian cancer
      Current Phase:         Phase I/II                            Third Line Treatment (18000 patients)        25%                        75%


                                                                        Patients given platinum-based therapy    Patients ineligible for platinum-based therapy




     Status                                                                      Market Overview
         Aprea has identified small molecules that reactivate                          63 500 patients are diagnosed with ovarian cancer
          p53, considered a prime target for new first-line cancer                       each year in 7MM, 25 % with an advanced disease at
          therapies                                                                      diagnosis
         Gained orphan drug status for indication AML in the                           Close to half of the patients in second line treatment
          EU                                                                             develop resistance to platinum-based treatment
         Planning in progress of a Phase I/II study in                                 Aprea's drug candidate aims to target this problem
          chemotherapy-resistant patients




                                                 *Ownership in Aprea will be 69% after share swap
13                                                      transaction with Industrifonden.
AKN-028 - Targeting all AML Tumor Types                                                                                               Ownership: 90%


                                                                                             AML Treatment Outcomes
                                                                              70%
      Company:              Akinion
                                                                              60%                                             30 day Treatment
                            Pharmaceuticals AB                                50%                                             Mortality
      Active ingredient:    Small molecule                                    40%
                                                                              30%                                             Resistance
      Primary indication:   Acute Myeloid                                     20%
                            Leukemia (AML)                                    10%                                             Complete Remission after
      Current Phase:        Phase I/II                                         0%                                             cytarabine + daunorubicin
                                                                                           <56         56-65    66-75   >75   treatment
                                                                                                          Age Group




      Status                                                                    Market Overview
         Targets FLT3 and c-Kit – important factors in leukemia                      Chemotherapy is the only viable pharmaceutical
          development                                                                  treatment for AML today
         Unique preclinical efficacy in chemotherapy resistant                       About 42 000 patients are affected by AML each year
          cells                                                                        in the US, EU and Japan
         Phase I/II ongoing in AML                                                   5-year survival: 33% in patients under 65,
                                                                                       4% in patients over 65 years




                                                     Sources: Datamonitor, NCCN, SEER, Appeldaum et al 2006



14
Ex vivo Efficacy of AKN-028 vs. Quizartinib                                                                                                                                                       Ownership: 90%




            Cytotoxic response to AKN-028 and Quizartinib (AC220) in 11 primary AML samples outlined as individual dose–response curves from the FMCA (a–f ). Samples are grouped
            according to in vitro response to Quizartinib. Because of the configuration of data for Quizartinib, a sigmoid curve fitting was not possible. SI, survival index. (Eriksson et al 2012)




15
Synergistic Response of AKN-028 with Chemotherapy                                                                                                                                                    Ownership: 90%




      Cytotoxic response to AKN-028 in combination with standard cytotoxic agents cytarabine (a) and daunorubicin (b) in MV4-11 cell line in three different sequences; (I): pretreatment with chemotherapy (24 h), followed
      by AKN-028; (II): simultaneous treatment with both agents; (III): pretreatment with AKN-028 (24 h). Results are presented as mean CI, (n¼11–16) of all experimental combination data points with effect levels between
      10–90%, error bars representing s.e.m. CI o0.7 indicate synergy and CI of 1 additive interactions. (Eriksson et al 2012)




16
SHACT - Pain Management in Gynecology                                                                                             Ownership: 59%




      Company:              Pharmanest AB
      Active ingredient:    Small molecule
      Primary indication:   Pain in connection with IUD-insertion
      Current Phase:        Phase II




     Status                                                                            Market Overview
        Unique formulation and applicator developed for                                     UN estimates the prevalence of IUD at 15 million
         local pain relief during gynecological procedures                                    women in the developed world
        Last patient dosed in Phase I safety and                                            Assuming an IUD insertion every 5 year, the annual
         pharmacokinetics study                                                               number of IUD insertions is 3 million in developed
                                                                                              countries
        A randomized, double blind Phase II-study with                                      China represents a large market potential - Over
         about 200 patients initiated in 2Q 2012                                              100 million women with IUD
        Phase II-data is expected in 2H 2013                                                Potential to expand to other indications such as
                                                                                              hysteroscopy, abortions and obstetric pain

                                                      Sources: United Nations, World Contraceptive Use (2011)


17
Financial Information




18
Key Financial Information for the Group

                                                                                                               2012                  2011       2011
        Amounts in SEKm                                                                                   Jan-June             Jan-June      Full year
        Income statement
        Revenue                                                                                                 5.5                    5.2       10.5
        Profit/loss after tax                                                                                -189.2                 -130.8     -385.7
        Earnings per share before and after dilution (SEK)                                                     -3.52                 -3.09      -8.07
        Balance sheet
        Cash and cash equivalents                                                                             170.3                 162.1       163.3
        Short-term investments                                                                                291.1                 577.5       457.2
        Share information
        Net asset value per share (SEK)                                                                         42.5                 49.6        44.7
        Share price, last trading day in the reporting period (SEK)                                             19.5                 29.4        24.0
        Portfolio information
        Investments in portfolio companies*                                                                   115.7                 226.2       297.6
        Of which investments not affecting cash flow                                                              -                  84.1        94.9
        Valuation of total portfolio holdings                                                               1,564.4             1,652.1       1,546.9
        * Portfolio companies comprise subsidiaries, joint ventures, associated companies and other long-term securities holdings




19
Strong Shareholder Base
          Shareholder                                                               A Shares     B Shares    Cap %    Votes %
          Third Swedish National Pension Fund                                                    4 678 500     9.6%      7.5%
          Karolinska Institutet Holding AB                                          1 503 098    2 453 933     8.2%     28.2%
          Coastal Investment Management LLC                                                      3 470 541     7.2%      5.6%
          The Foundation of Baltic and East European Studies                                     3 345 537     6.9%      5.4%
          Swedbank Robur Funds                                                                   1 920 074     4.0%      3.1%
          Jarla Investeringar AB                                                                 1 629 354     3.4%      2.6%
          Foundation Asset Management AB                                                         1 392 035     2.9%      2.2%
          Länsförsäkringar Group                                                                 1 364 415     2.8%      2.2%
          Skagen Funds                                                                           1 297 700     2.7%      2.1%
          Stefan Persson                                                                         1 261 278     2.6%      2.0%
          Insamlingsstiftelsen för främjande och utveckling av medicinsk
          forskning vid Karolinska Institutet                                                    1 150 323     2.4%      1.9%
          Government of Norway                                                                     781 951     1.6%      1.3%
          Holberg Funds                                                                            754 653     1.6%      1.2%
          SBSB Innovation AB                                                                       692 000     1.4%      1.1%
          Ruffer Funds                                                                             450 000     0.9%      0.7%
          Fourth Swedish National Pension Fund                                                     383 959     0.8%      0.6%
          Gålö Foundation                                                                          375 535     0.8%      0.6%
          Nordea Funds                                                                             366 600     0.8%      0.6%
          KL Ventures AB                                                                           300 000     0.6%      0.5%
          Lingfield AB                                                                             281 989     0.6%      0.5%
          Sum listed shareholders                                                   1 503 098   28 329 673    61.5%     69.9%
          Sum other shareholders                                                            0   18 698 646    38.5%     30.1%
          Sum all shareholders                                                      1 503 098   47 028 319   100.0%    100.0%


                                                              As at June 30, 2012

20
Karolinska Development in Summary

       Exclusive access to top class medical innovations
       First-in-class drug candidates with multi billion dollar
        sales potential
       Significant clinical progress – 14 projects in clinical
        phase
       Large portfolio of companies removes binary risk –
        26 companies, 35 projects
       Cash position provides funding through major
        clinical data
       Strong focus on business development activities
        across the portfolio


21
Questions




22

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Karolinska Development Interim Report January - June 2012

  • 1. Karolinska Development 2Q 2012 Conference Call – August 23, 2012 Torbjörn Bjerke, CEO
  • 2. Today’s Presentation Second Quarter Highlights Selected Portfolio Companies Financial Information Summary 2
  • 3. Karolinska Development in Summary  Exclusive access to top class medical innovations  First-in-class drug candidates with multi billion dollar sales potential  Significant clinical progress – 14 projects in clinical phase  Large portfolio of companies removes binary risk – 26 companies, 35 projects  Cash position provides funding through major clinical data  Strong focus on business development activities across the portfolio 3
  • 4. Highlights During and Since the Second Quarter 2012  Pharmanest initiated Phase I and Phase II trials with SHACT, a product developed for pain relief in connection with IUD insertion  Karolinska Development AB and Industrifonden signed a share swap agreement whereby Industrifonden will exchange Karolinska Development AB’s shares in Oncopeptides AB for its shares in Aprea AB  Pergamum reported top-line Phase II-data from the clinical trial with PXL-01 for prevention of post-surgical adhesions  Strengthened Management team in Karolinska Development: – Dr Gunilla Ekström recruited as VP Operations – Ann-Sofie Sternås recruited as VP IPR  Akinion Pharmaceuticals announced the publication of preclinical results for AKN-028 in highly cited scientific journal 4
  • 5. The Portfolio has a Significant Market Potential Concept Lead Lead Preclinical Ownership* Phase I Phase II Phase III Launch PHARMACEUTICALS development discovery optimization development ONCOLOGY Axelar AB 50% Aprea AB 41% Oncopeptides AB 43 % KDev Oncology AB (AKN-028) 90% KDev Oncology AB (GliGene) 44% DERMATOLOGY Pergamum AB (DPK-060, atopic dermatitis) 62% Pergamum AB (DPK-060, external otitis) 62% Pergamum AB (PXL01) 62% Pergamum AB (LL-37) 62% WOMEN’S HEALTH Dilafor AB 55% Pharmanest AB 59% Umecrine Mood AB 43% INFECTIOUS DISEASE Dilaforette AB 62% Biosergen AS 60% CARDIOVASCULAR Athera Biotechnologies AB (Annexin A5) 65% Athera Biotechnologies AB (PC-mAb) 65% CNS BioChromix Pharma AB 70% Umecrine Cognition AB 54% INFLAMMATION NovaSAID AB 89% ProNoxis AB 22% OPHTHALMOLOGY Clanotech AB 88% Solid colored area = completed phase Shaded colored area = ongoing phase *Including indirect ownership. Ownership in Axelar and GliGene shown after all tranches in recent investment round. All shares in Oncopeptides will be exchanged for Industrifondens shares in Aprea. Ownership in Aprea after share swap transactiona will be 69%. 5
  • 6. The Portfolio has a Significant Market Potential TECHNOLOGY Ownership* Concept development Prototype Development Product Sales IMPLANTS Promimic AB 28% Oss-Q AB 16% DIAGNOSTICS Athera Biotechnologies AB (CVDefine®) 65% BioChromix AB 14% PHARMACEUTICAL FORMULATION Inhalation Sciences Sweden AB 72% XSpray Microparticles AB 62% Lipidor AB 46% MEDICAL EQUIPMENT NeoDynamics AB (Fourier/AS) 21% NeoDynamics AB (Therapy/PRFA) 21% Solid colored area = completed phase Shaded colored area = ongoing phase *Including indirect ownership 6
  • 7. Portfolio Focus in 2012 and 2013 Axelar – AXL1717 Phase I/II data  Axelar – AXL1717 for NSCLC Initiate Phase II  Akinion – AKN-028 for AML Initiate clinical studies  ONCOLOGY Aprea – APR-246 Complete Phase I/II Akinion – AKN 028 Complete Phase I/II Axelar – AXL1717 for NSCLC Complete Phase II Pergamum – PXL01 for surgical adhesion Complete enrolment of Phase II  Pergamum – DPK-060 for external otitis Initiate Phase II  DERMATOLOGY Pergamum – DPK-060 for skin infection Preparation for Phase IIb Pergamum – LL-37 for chronic wounds Initiate clinical studies Pergamum - DPK-060 for external otitis Complete Phase II Pharmanest – SHACT for pain relief at IUD insertion Initiate Phase I  Pharmanest – SHACT for pain relief at IUD insertion Initiate Phase II  WOMEN’S HEALTH Dilafor – Tafoxiparin for protracted labor Initiate Phase IIb with partner Pharmanest – SHACT for pain relief at IUD insertion Complete Phase I and II Umecrine Mood – Severe PMS and PMDD Initiate Phase I/II Dilaforette – Sevuparin for malaria Initiate Phase I/II  OTHER Dilaforette – Sevuparin for malaria Complete Phase I/II 7
  • 8. Anticipated Major Clinical Data Phase I data LL-37 Phase I data PXL01 Phase II data  DPK-060 Phase II data Phase II data Phase II data in NSCLC 2012 2013 2014 Phase I data Phase I/II data Phase I/II data Tafoxiparin Phase IIb data 8
  • 9. Significant Deal Values in Our Focus Areas Therapeutic Total Deal Date Licensor Licensee Product Phase at Deal Area Value (USDm) February 16, 2011 AVEO Pharmaceuticals, Inc. Astellas Pharma, Inc. Oncology AV951 Phase II 1480 February 29, 2012 Galapagos NV Abbott Laboratories Inflammatory GLPG0634 Phase II 1350 November 10, 2009 Alder Biopharmaceuticals Inc. Bristol-Myers Squibb Company Inflammatory ALD518 Phase II 1049 December 8, 2011 Pharmacyclics, Inc. Janssen Biotech, Inc. Oncology PCI32765 Phase II 975 September 3, 2009 Algeta ASA Bayer Healthcare Pharmaceuticals, Inc. Oncology Alpharadin Phase II 800 December 21, 2009 Incyte Corporation Eli Lilly & Co Inflammatory INCB28050 Phase II 755 February 12, 2009 Portola Pharmaceuticals Inc. Novartis AG CVD PRT128 Phase II 575 May 4, 2011 Molecular Partners AG Allergan Inc. Ophthalmology MP0112 Phase II 420 September 5, 2011 Evotec AG Roche Holding, Ltd. CNS EVT302, RG1577 Preclinical 830 Janssen Pharmaceuticals Inc./ June 1, 2010 Orexo AB Respiratory OX-CLI, OX-ESI Preclinical 586 Johnson & Johnson June 8, 2011 Affectis Pharmaceuticals AG Merck Serono S.A. CNS P2X7 Antagonist Preclinical 409 January 1, 2012 Virobay, Inc. LEO Pharma A/S Dermatology Preclinical 307 MP0260, August 21, 2012 Molecular Partners AG Allergan Inc. Ophthalmology Discovery 1400 eye disease DARPins December 20, 2010 Avila Therapeutics, Inc. Sanofi Oncology Discovery 964 January 5, 2012 Forma Therapeutics, Inc. Boehringer Ingelheim Corporation Oncology Discovery 815 9
  • 11. AXL1717 - Block Buster Potential in Lung Cancer Ownership: 50% 12 Avastin, Tarceva and Iressa Sales (USDbn) Company: Axelar AB 10 Active ingredient: Small molecule 8 6 Primary indication: Non Small Cell Lung Cancer 4 Current Phase: Phase II 2 0 Status Market Overview  The only small-molecule IGF-1R inhibitor in man  Around 420 000 new cases of NSCLC in the without insulin receptor inhibition industrialized countries each year  Unique preclinical efficacy  UBS estimated total US lung cancer sales in 2010 for  Promising Phase I/II data Avastin at USD 1150m, Tarceva at USD 400m, and  Phase II ongoing – Top-line data expected in the Iressa at USD 310m beginning of 2013  Next indication Glioblastoma Source: Datamonitor AXL1717 - Potential in Multiple Cancers 11
  • 12. AXL1717 – Promising Phase I/II-data (March 30 Update) Ownership: 50% SAFETY Progression-free survival and overall survival of 15 patients with progressive and refractory NSCLC treated with single AXL1717 was well-tolerated with reversible neutropenia agent AXL1717 in 3rd or 4th line. Patients with a treatment duration longer than 14 days. Status per March 30, 2012 as dose limiting toxicity - Even at exposures 5-10 fold higher than those needed for anti-tumor activity in animals. - No changes in blood levels of glucose, insulin or C-peptide were reported. CLINICAL BENEFIT For the NSCLC patients treated for 14 days or longer (Mar 30, 2012): Median survival 60 weeks Median time to progression 31 weeks One Partial Response according to RECIST 12
  • 13. Normalizing p53 to Overcome Drug Resistance Ownership: 41%* Platinum refractory patients in ovarian cancer (US) Company: Aprea AB First Line Treatment (60500 patients) 86% 14% Active ingredient: Small molecule Second Line Treatment (30500 patients) 58% 42% Primary indication: Ovarian cancer Current Phase: Phase I/II Third Line Treatment (18000 patients) 25% 75% Patients given platinum-based therapy Patients ineligible for platinum-based therapy Status Market Overview  Aprea has identified small molecules that reactivate  63 500 patients are diagnosed with ovarian cancer p53, considered a prime target for new first-line cancer each year in 7MM, 25 % with an advanced disease at therapies diagnosis  Gained orphan drug status for indication AML in the  Close to half of the patients in second line treatment EU develop resistance to platinum-based treatment  Planning in progress of a Phase I/II study in  Aprea's drug candidate aims to target this problem chemotherapy-resistant patients *Ownership in Aprea will be 69% after share swap 13 transaction with Industrifonden.
  • 14. AKN-028 - Targeting all AML Tumor Types Ownership: 90% AML Treatment Outcomes 70% Company: Akinion 60% 30 day Treatment Pharmaceuticals AB 50% Mortality Active ingredient: Small molecule 40% 30% Resistance Primary indication: Acute Myeloid 20% Leukemia (AML) 10% Complete Remission after Current Phase: Phase I/II 0% cytarabine + daunorubicin <56 56-65 66-75 >75 treatment Age Group Status Market Overview  Targets FLT3 and c-Kit – important factors in leukemia  Chemotherapy is the only viable pharmaceutical development treatment for AML today  Unique preclinical efficacy in chemotherapy resistant  About 42 000 patients are affected by AML each year cells in the US, EU and Japan  Phase I/II ongoing in AML  5-year survival: 33% in patients under 65, 4% in patients over 65 years Sources: Datamonitor, NCCN, SEER, Appeldaum et al 2006 14
  • 15. Ex vivo Efficacy of AKN-028 vs. Quizartinib Ownership: 90% Cytotoxic response to AKN-028 and Quizartinib (AC220) in 11 primary AML samples outlined as individual dose–response curves from the FMCA (a–f ). Samples are grouped according to in vitro response to Quizartinib. Because of the configuration of data for Quizartinib, a sigmoid curve fitting was not possible. SI, survival index. (Eriksson et al 2012) 15
  • 16. Synergistic Response of AKN-028 with Chemotherapy Ownership: 90% Cytotoxic response to AKN-028 in combination with standard cytotoxic agents cytarabine (a) and daunorubicin (b) in MV4-11 cell line in three different sequences; (I): pretreatment with chemotherapy (24 h), followed by AKN-028; (II): simultaneous treatment with both agents; (III): pretreatment with AKN-028 (24 h). Results are presented as mean CI, (n¼11–16) of all experimental combination data points with effect levels between 10–90%, error bars representing s.e.m. CI o0.7 indicate synergy and CI of 1 additive interactions. (Eriksson et al 2012) 16
  • 17. SHACT - Pain Management in Gynecology Ownership: 59% Company: Pharmanest AB Active ingredient: Small molecule Primary indication: Pain in connection with IUD-insertion Current Phase: Phase II Status Market Overview  Unique formulation and applicator developed for  UN estimates the prevalence of IUD at 15 million local pain relief during gynecological procedures women in the developed world  Last patient dosed in Phase I safety and  Assuming an IUD insertion every 5 year, the annual pharmacokinetics study number of IUD insertions is 3 million in developed countries  A randomized, double blind Phase II-study with  China represents a large market potential - Over about 200 patients initiated in 2Q 2012 100 million women with IUD  Phase II-data is expected in 2H 2013  Potential to expand to other indications such as hysteroscopy, abortions and obstetric pain Sources: United Nations, World Contraceptive Use (2011) 17
  • 19. Key Financial Information for the Group 2012 2011 2011 Amounts in SEKm Jan-June Jan-June Full year Income statement Revenue 5.5 5.2 10.5 Profit/loss after tax -189.2 -130.8 -385.7 Earnings per share before and after dilution (SEK) -3.52 -3.09 -8.07 Balance sheet Cash and cash equivalents 170.3 162.1 163.3 Short-term investments 291.1 577.5 457.2 Share information Net asset value per share (SEK) 42.5 49.6 44.7 Share price, last trading day in the reporting period (SEK) 19.5 29.4 24.0 Portfolio information Investments in portfolio companies* 115.7 226.2 297.6 Of which investments not affecting cash flow - 84.1 94.9 Valuation of total portfolio holdings 1,564.4 1,652.1 1,546.9 * Portfolio companies comprise subsidiaries, joint ventures, associated companies and other long-term securities holdings 19
  • 20. Strong Shareholder Base Shareholder A Shares B Shares Cap % Votes % Third Swedish National Pension Fund 4 678 500 9.6% 7.5% Karolinska Institutet Holding AB 1 503 098 2 453 933 8.2% 28.2% Coastal Investment Management LLC 3 470 541 7.2% 5.6% The Foundation of Baltic and East European Studies 3 345 537 6.9% 5.4% Swedbank Robur Funds 1 920 074 4.0% 3.1% Jarla Investeringar AB 1 629 354 3.4% 2.6% Foundation Asset Management AB 1 392 035 2.9% 2.2% Länsförsäkringar Group 1 364 415 2.8% 2.2% Skagen Funds 1 297 700 2.7% 2.1% Stefan Persson 1 261 278 2.6% 2.0% Insamlingsstiftelsen för främjande och utveckling av medicinsk forskning vid Karolinska Institutet 1 150 323 2.4% 1.9% Government of Norway 781 951 1.6% 1.3% Holberg Funds 754 653 1.6% 1.2% SBSB Innovation AB 692 000 1.4% 1.1% Ruffer Funds 450 000 0.9% 0.7% Fourth Swedish National Pension Fund 383 959 0.8% 0.6% Gålö Foundation 375 535 0.8% 0.6% Nordea Funds 366 600 0.8% 0.6% KL Ventures AB 300 000 0.6% 0.5% Lingfield AB 281 989 0.6% 0.5% Sum listed shareholders 1 503 098 28 329 673 61.5% 69.9% Sum other shareholders 0 18 698 646 38.5% 30.1% Sum all shareholders 1 503 098 47 028 319 100.0% 100.0% As at June 30, 2012 20
  • 21. Karolinska Development in Summary  Exclusive access to top class medical innovations  First-in-class drug candidates with multi billion dollar sales potential  Significant clinical progress – 14 projects in clinical phase  Large portfolio of companies removes binary risk – 26 companies, 35 projects  Cash position provides funding through major clinical data  Strong focus on business development activities across the portfolio 21