Karyopharm Therapeutics Inc. · NASDAQ:KPTI

Karyopharm Therapeutics — Investor Presentation (August 2015)

29 slides from Karyopharm Therapeutics's investor presentation, Aug 10, 2015

  1. Slide 1: Corporate Presentation August 2015

    Exhibit 99.1 NASDAQ: Corporate August Targeting 2015 Disease Presentation KPTI at the Nuclear Pore

  2. Slide 2: Forward-looking Statements

    Forward-looking This Reform (KPT-330), Such there of appreciation. commercialize number existing investigational unplanned These Factors” Karyopharm Any disclaims Karyopharm’s encourages in Unless 2 this anyforward-looking presentation can forward-looking statements and presentation of otherwise Act

  3. Slide 3: Karyopharm: Novel Drug Class Targeting Cancer

    4 Karyopharm: Karyopharm’s Novel Tumor Potentially mechanism Suppressor ongoing relevant >40 combination Prepare Seek New Selinexor Clinical Commercial Initial unmet 3 Drug At wholly collaborators focus medical to Class on later the Ph Development First Protein 2to commercialize Strategy need the in

  4. Slide 4: Selinexor: Broad Clinical Development Strategy

    Selinexor: DRUG Oral Hematological Solid AML Richter’s Multiple Sel+Pom+Dex; Sel+Rituximab DLBCL Combination Gynecologic Ovarian Liposarcoma Selinexor Various Standard = Company Investigator 4 Initiated Selinexor Tumors (SOPRA) CANDIDATE Hematological Solid or (SADAL) Myeloma Chemotherapy (SIRRT) +

  5. Slide 5: Selinexor: Novel Anti-Cancer Agent Restores Tumor Suppressors

    Selinexor: Cell CYTOPLASM Nuclear XPO-1 PP2A Par-4 p53 Tumor (myc,bcl2) elF4E p27 BRCA1 IkB p21 5 Membrane pRB Suppressors Envelope Pore Novel Complex Anti-Cancer Agent: Restores Tumor Suppressors & Reduces Oncoproteins

  6. Slide 6: Selinexor Blocks XPO1 – Forcing Nuclear Localization of Tumor Suppressor Proteins

    Selinexor Blocks XPO1 – Forcing Nuclear Localization of Tumor Suppressor Proteins 6

  7. Slide 7: Initial Focus: Single-Agent Approval of Selinexor

    Initial Acute DLBCL Multiple Richter’s 7 ©2015 Myeloid Focus Myeloma –Transformation Karyopharm forLeukemia Single-Agent Therapeutics Approval Inc.of Selinexor in:

  8. Slide 8: Selinexor's Broad Single-Agent Anti-Tumor Activity

    Selinexor’s Selinexor Broad >1 Disease 8 yearand (longest control 1,000+ durable Broad rates 2+ up and single-agent years) to inDurable 80% patients >6 Oral anti-tumor month withSingle-Agent advanced up toactivity 50% solid Anti-Tumor intumors a pill Patients Activity treated to date with single age

  9. Slide 9: Selinexor: Key Milestones

    Selinexor: Key Top NDA US AML Richter’s MM Citations: ACS,Patient Line Milestones 24,000 Filing Tsimberidou DLBCL 19,000 Data 1,200 ACS First Populations H1 DLBCL Q4 Mid-2017 and Potential 2017 2018 for2016 etFirst Leukemia al, 25,000 byMDACC; Potential Approvals Indication: and Approvals: Lymphoma

  10. Slide 10: Diffuse Large B-Cell Lymphoma: The Opportunity

    DiffuseofLarge Aggressive Incidence: ~40% ~10,000 Greatest Selinexor Citations: 10 deaths unmet incident has ACS 25,000 form shown B-Cell and need per of patients new year Leukemia Non-Hodgkin’s currently durable Lymphoma: cases inwill USAannually single-agent and in succumb (~22,500 GCB The Lymphom

  11. Slide 11: NHL: Anti-tumor Responses in Phase 1 Patients

    NHL: Fifty-two Anti-tumor Durable Overall Disease NHL DLBCL RESPONSES Type GCB PATIENTS Patient 046 072 Data Presented 11 CR PR as N RESPONSE 11 Phase ID DCR of -65% response 73% control cancer 32 9patients, at (82%) December Best 21 activity WITH ASH 1(PET (%) 214 IN Data (66%) control Response rat

  12. Slide 12: SADAL: Ongoing Phase 2b Study in Relapsed DLBCL

    SADAL: Ongoing Relapsed Twice-weekly ³50% Targeting Primary Data Preparations Selinexor-Rituximab Combinations Phase 12 read of 3spatients Endpoint: planned Randomized Randomized Selinexor /~200 out Refractory for anticipated for randomized patients Phase with for 3rd, Overall Against 2016 +GCB-DLBC

  13. Slide 13: SADAL: Potential Key Development Timeline

    * Potential 2014 Sel SADAL: (high Ph 21+Selinexor Phase Slide1/2 CHOP-R Ph 2015 Ritux vs. 3(2nd/3rd KeySel 2low Selinexor Ph2016 +in 2b Sel Ibrut NHL in Selinexor Line) 2017 Investigator Studies: Rituxan®(rituximab) 13 (2nd (1st/2nd dose) Richter’s in NHL Selinexor NHL +Sel H1 RandomizedDevelopment

  14. Slide 14: Multiple Myeloma: The Opportunity

    Multiple Second 114,000 In Initial Unmet Citations: 14 the US, focus need most new Myeloma: Myeloma ACS a prevalence on for cases commonly Facts patients patients worldwide represents The andof with Opportunity with diagnosed Figures approximately recurrent recurrent aeach significant 2014; blood ye

  15. Slide 15: STORM: Planned Phase 2b Study in Myeloma

    STORM: Planned Quadruple Initiated Primary Interim Clinical -Phase Preparation Selinexor-dex Additional 15 High11/2 Early ORR signs data Selinexor Results Endpoint: Single May Phase Selinexor combination refractory: Phase for anticipated and of2015, +Arm Phase promising 2to PI durable 1/2s Clinical

  16. Slide 16: Selinexor Phase 1/2 Combination in Multiple Myeloma

    Multiple Selinexor Phase Heavily In Group (As Oral Patient 76 77 Cyclo-Dex-len, 79 81 84 90 92 93 Vel-Dex, 98 99 16 ten — FLC-K IgG-K -48% IgA-K of Combination—Selinexor NE patients: 1/2 A 1D-M MM pretreated MR Patients Myeloma: VAD, + 8-82% -71% -84% 41% -55% -41% combination -53% -99% Carm-Thal-Ci

  17. Slide 17: Promising Selinexor + Carfilzomib Data in Refractory Patients

    Promising SELINEXOR IN (Selinexor Patient Days 59 73 64 Data 17 113+ 85+ 71+ PATIENTS asAge PR of VGPR November Potential 30 2Best 5 mg/m2 +WITH 4CARFILZOMIB Response in+ 1, CARFILZOMIB-REFRACTORY Patients Carfilzomib 2014 # Prior Presented withTxCarfilzomib-Refractory (20/27 onatTx ASH mg/m2) 2014

  18. Slide 18: STORM: Potential Key Development Timeline

    * Potential 2014 Ph Ph 13: AND/OR Slide * STORM: 1/22015 Ph Key Selinexor Investigator bortezomib Bortez carfilzomib Carfil proteasome PI dexamethasone Dex pegylated PLD 18 2016 Selinexor 1/2: Selinexor Ph Selinexor+2017 2Pom MM +Pom + Selinexor 2018 ++Development Carfil PLD Pre-Transplant PI Revlim

  19. Slide 19: Acute Myeloid Leukemia: The Opportunity

    Acute Significant Approximately The Older Median Approval SOPRA: Combination Additional About All Pediatric Citations: 19 patients average patients Myeloid 20% Survival Older patients Plans ACS opportunities opportunity of with age studies with patients Leukemia: patients Facts 18,860 of relapse of

  20. Slide 20: SOPRA: Ongoing Phase 3 Study in Older AML

    SOPRA: Ongoing Primary Randomized Dose Interim Phase Based Seven One Overall 21 49% 20 stable PR (31 adjusted 1CR/CR(i/p) on analysis response Clinical and Endpoint: of Randomized Selinexor disease 6363) two total 2:1 indisease in Results: –Morphological July patients rate: patients, single-agent (1

  21. Slide 21: SOPRA: Potential Key Development Timeline

    * Potential 2014 Ph SOPRA: Front Pick1 the Sel 2015 Line in Ph Selinexor (Older): 2016 R/R Phase2b3Selinexor Winner AML 2017 :(L1AML Selinexor H1 Development vs. H2+ Randomized Study); Physician’s H1 +Sel LDAC +H2 H1Launch H2 Choice H1 DataH2 (2:1) +Prep/Submit 170 pts;Selinexor OS Endpoint NDA/EMA

  22. Slide 22: Selinexor Studies in Solid Tumors

    Selinexor 22 ©2015 in – Karyopharm Solid TumorsTherapeutics & Combination Inc. Studies

  23. Slide 23: ASCO Summary and Conclusions

    ASCO Selinexor patients Single In Post-treatment ©2015 23 patients agent Summary –remaining Karyopharm shows levels showed induced with oral biopsies previously in anti-tumor selinexor &broad meaningful on brain Conclusions study Therapeutics, demonstrated anti-tumor tumor demonstrated treated activ

  24. Slide 24: Investigator-Sponsored Trials Using Selinexor

    ISTs Using Hematological Solid Selinexor 24 Tumors + Selinexor: Carfilzomib Fludarabine Bortezomib Ibrutinib Decitabine Paclitaxel Gemcitabine FOLFOX Irinotecan Docetaxel Malignancies inin Planned +inin patients + Carboplatin +patients patients +patients Dexamethasone Cytarabine Dexamethasone Abraxa

  25. Slide 25: Financial

    Financial 25 ©2015and – Karyopharm CommercialTherapeutics Overview Inc.

  26. Slide 26: Financial Overview

    Financial Cash, ~$255.9 Expected Shares Basic: Fully 26 diluted: Cash ~35.7 Outstanding MM to Overview Equivalents fund MM at ~40.0 June the asat MM of Company 30, June June &2015 asInvestments: 30, of 30,June 2015 into 201530, 20182015

  27. Slide 27: Selinexor: Broad Clinical Development Strategy

    Selinexor: DRUG Oral Hematological Solid AML Richter’s Multiple Sel+Pom+Dex; Sel+Rituximab DLBCL Combination Gynecologic Ovarian Liposarcoma Selinexor Various Standard = Company Investigator 27Initiated Selinexor Tumors (SOPRA) CANDIDATE Hematological Solid or (SADAL) Myeloma Chemotherapy (SIRRT) +

  28. Slide 28: Karyopharm's Drug Compounds Pipeline

    Karyopharm’s DRUG COMPOUNDS SINE™ Topical Hematological Antiviral Inflammation Oral PAK4 Autoimmune *Designed 28 Verdinexor KPT-350 Inhibitors CANDIDATES Selinexor Influenza to serve &= Broad Malignancies Autoimmune (KPT-335) Initiated Oncology (KPT-330) asTherapeutic the AREA basis Various Canine W

  29. Slide 29: Karyopharm: Selinexor at the Nucleus of Cancer Care

    Karyopharm: Selinexor Karyopharm Three Two Multiple 29 ongoing ongoing combination is awholly At later novel, later thephase phase Nucleus owns oral studies studies selective studies theof are worldwide Cancer (SOPRA ongoing (SOPRA, inhibitor Care rights and orof SADAL planned STORM) XPO1-mediated t

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