Karolinska Development 2Q 2012 Conference Call – August 23, 2012 Torbjörn Bjerke, CEO
May 31, 2015
Karolinska Development 2Q 2012 Conference Call – August 23, 2012 Torbjörn Bjerke, CEO
Today’s Presentation
Second Quarter Highlights Selected Portfolio Companies Financial Information Summary
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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
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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
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CARDIOVASCULAR Athera Biotechnologies AB (Annexin A5) 65% Athera Biotechnologies AB (PC-mAb) 65%
The Portfolio has a Significant Market Potential
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%
CNS BioChromix Pharma AB 70% Umecrine Cognition AB 54%
INFLAMMATION NovaSAID AB 89% ProNoxis AB 22%
Solid colored area = completed phase Shaded colored area = ongoing phase
PHARMACEUTICALS Ownership* Concept development
Lead discovery
Lead optimization
Preclinical development Phase I Phase II Phase III Launch
ONCOLOGY Axelar AB 50% Aprea AB 41% Oncopeptides AB 43 % KDev Oncology AB (AKN-028) 90% KDev Oncology AB (GliGene) 44%
OPHTHALMOLOGY Clanotech AB 88%
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*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%.
TECHNOLOGY Ownership* Concept development Prototype Development Product Sales
IMPLANTS Promimic AB 28% Oss-Q AB 16%
The Portfolio has a Significant Market Potential
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Solid colored area = completed phase Shaded colored area = ongoing phase
*Including indirect ownership
DIAGNOSTICS
Athera Biotechnologies AB (CVDefine®) 65% BioChromix AB 14%
MEDICAL EQUIPMENT NeoDynamics AB (Fourier/AS) 21% NeoDynamics AB (Therapy/PRFA) 21%
PHARMACEUTICAL FORMULATION Inhalation Sciences Sweden AB 72% XSpray Microparticles AB 62% Lipidor AB 46%
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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 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 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
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 Dilaforette – Sevuparin for malaria Complete Phase I/II
ONCOLOGY
DERMATOLOGY
WOMEN’S HEALTH
OTHER
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Anticipated Major Clinical Data
2012 2013 2014
PXL01 Phase II data Phase II data in NSCLC
DPK-060 Phase II data
Phase I data
Tafoxiparin Phase IIb data
LL-37 Phase I data
Phase I/II data
Phase I data
Phase I/II data
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Phase II data
Significant Deal Values in Our Focus Areas
Date Licensor Licensee Therapeutic Area Product Phase at Deal Total Deal
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
June 1, 2010 Orexo AB Janssen Pharmaceuticals Inc./ Johnson & Johnson Respiratory OX-CLI, OX-ESI Preclinical 586
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
August 21, 2012 Molecular Partners AG Allergan Inc. Ophthalmology MP0260, eye disease DARPins Discovery 1400
December 20, 2010 Avila Therapeutics, Inc. Sanofi Oncology Discovery 964
January 5, 2012 Forma Therapeutics, Inc. Boehringer Ingelheim Corporation Oncology Discovery 815
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Selected Portfolio Companies
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AXL1717 - Block Buster Potential in Lung Cancer
Status
The only small-molecule IGF-1R inhibitor in man without insulin receptor inhibition
Unique preclinical efficacy Promising Phase I/II data Phase II ongoing – Top-line data expected in the
beginning of 2013 Next indication Glioblastoma
Market Overview
Around 420 000 new cases of NSCLC in the industrialized countries each year
UBS estimated total US lung cancer sales in 2010 for Avastin at USD 1150m, Tarceva at USD 400m, and Iressa at USD 310m
Source: Datamonitor
Ownership: 50%
AXL1717 - Potential in Multiple Cancers
Avastin, Tarceva and Iressa Sales (USDbn)
0
2
4
6
8
10
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Company: Axelar AB
Active ingredient: Small molecule
Primary indication: Non Small Cell Lung Cancer
Current Phase: Phase II
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AXL1717 – Promising Phase I/II-data (March 30 Update) Ownership: 50%
SAFETY
AXL1717 was well-tolerated with reversible neutropenia 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
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Progression-free survival and overall survival of 15 patients with progressive and refractory NSCLC treated with single agent AXL1717 in 3rd or 4th line. Patients with a treatment duration longer than 14 days. Status per March 30, 2012
Normalizing p53 to Overcome Drug Resistance Ownership: 41%*
Company: Aprea AB
Active ingredient: Small molecule
Primary indication: Ovarian cancer
Current Phase: Phase I/II
Status
Aprea has identified small molecules that reactivate p53, considered a prime target for new first-line cancer therapies
Gained orphan drug status for indication AML in the EU
Planning in progress of a Phase I/II study in chemotherapy-resistant patients
Market Overview
63 500 patients are diagnosed with ovarian cancer each year in 7MM, 25 % with an advanced disease at diagnosis
Close to half of the patients in second line treatment develop resistance to platinum-based treatment
Aprea's drug candidate aims to target this problem
25%
58%
86%
75%
42%
14%
Third Line Treatment (18000 patients)
Second Line Treatment (30500 patients)
First Line Treatment (60500 patients)
Platinum refractory patients in ovarian cancer (US)
Patients given platinum-based therapy Patients ineligible for platinum-based therapy
*Ownership in Aprea will be 69% after share swap transaction with Industrifonden.
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AKN-028 - Targeting all AML Tumor Types
Status
Targets FLT3 and c-Kit – important factors in leukemia development
Unique preclinical efficacy in chemotherapy resistant cells
Phase I/II ongoing in AML
Market Overview
Chemotherapy is the only viable pharmaceutical treatment for AML today
About 42 000 patients are affected by AML each year in the US, EU and Japan
5-year survival: 33% in patients under 65, 4% in patients over 65 years
Sources: Datamonitor, NCCN, SEER, Appeldaum et al 2006
Ownership: 90%
Company: Akinion Pharmaceuticals AB
Active ingredient: Small molecule
Primary indication: Acute Myeloid Leukemia (AML)
Current Phase: Phase I/II 0%10%20%30%40%50%60%70%
<56 56-65 66-75 >75Age Group
AML Treatment Outcomes
30 day TreatmentMortality
Resistance
Complete Remission aftercytarabine + daunorubicintreatment
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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)
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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)
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SHACT - Pain Management in Gynecology
Market Overview
UN estimates the prevalence of IUD at 15 million women in the developed world
Assuming an IUD insertion every 5 year, the annual number of IUD insertions is 3 million in developed countries
China represents a large market potential - Over 100 million women with IUD
Potential to expand to other indications such as hysteroscopy, abortions and obstetric pain
Status
Unique formulation and applicator developed for local pain relief during gynecological procedures
Last patient dosed in Phase I safety and pharmacokinetics study
A randomized, double blind Phase II-study with about 200 patients initiated in 2Q 2012
Phase II-data is expected in 2H 2013
Company: Pharmanest AB
Active ingredient: Small molecule
Primary indication: Pain in connection with IUD-insertion
Current Phase: Phase II
Sources: United Nations, World Contraceptive Use (2011)
Ownership: 59%
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Financial Information
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Key Financial Information for the Group
Amounts in SEKm 2012 2011 2011
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
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Strong Shareholder Base
As at June 30, 2012
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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%
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
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Questions
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