Karyopharm Therapeutics Inc. · NASDAQ:KPTI
Karyopharm Therapeutics — Investor Presentation (January 2017)
31 slides from Karyopharm Therapeutics's investor presentation, Jan 9, 2017
Slide 1: Corporate Presentation January 2017
Exhibit 99.1 Corporate Presentation January 2017 KARYOPHARM THERAPEUTICS INC. Targetting Disease at the Nuclear Pore
Slide 2: Forward-looking statements
Forward-looking statements of uncertainties selinexor is positive no risks investigational requirements maintain greater subsequent hereof. website www.karyopharm.com, shall reports. developing, the assurance they and reporting detail Other ©2017 is developments and uncertainties (KPT-330) be http:/
Slide 3: Karyopharm Overview
Karyopharm broadly candidates including enrollment 2032 ©2017 home, major are asKaryopharm Overview aCombinable broad backbone diseases readouts SINE™ Nuclear Fully Therapeutics months management owned; Near Export accelerated Term Inc. COM safety Strong until 3 KPT-8602 approvals profile theWell en
Slide 4: Selinexor: Novel Oral Anti-Cancer Agent Restores Tumor Suppressors & Reduces Oncoproteins
Selinexor: Novel Oral Anti-Cancer Agent Restores Tumor Suppressors & Reduces Oncoproteins ©2017 Karyopharm Therapeutics Inc. 4 Nucleus Cytosol
Slide 5: Karyopharm's Broad Oncology Pipeline
Karyopharm’s and Physician’s KING: Other Veterinary Inc. Dexamethasone 5 Indications Selinexor Medicine Choice Broad Additional Verdinexor† Diffuse Multiple for Therapeutic the Large treatment Oncology Myeloma (KPT-335) Pipeline B-cell KPT-350 Programs STORM: Lymphoma *AREA NotALS, yet KPT-8602 OF S
Slide 6: Selinexor in Myeloma: First Oral Agent with Novel Mechanism and Single Agent Activity Since the IMiDs
Selinexor in Myeloma: First Oral Agent with Novel Mechanism and Single Agent Activity Since the IMiDs
Slide 7: Selinexor Advantages in Multiple Myeloma
Selinexor resensitize reduced, estimated therapy ©2017 compared Advantages ~$11 to prior Karyopharm billion therapies with in inSd Multiple 2015, •Therapeutics •Phase PIs: increasing Myeloma Velcade 3 SVd Inc. to regimen and •>Novel 7$22 Kyprolis billion uses agent40% • IMiDs: with by less 2023 anti
Slide 8: Widely Used Drugs in Multiple Myeloma
Drugs with Kyprolis Darzalex Therapeutics (K, (D,Single Carfilzomib) Daratumumab) Inc. 8Agent Activity 2012 2015 22.9% are refractory Widely Kd, KRd, (21% UsedKPd in Quad) MM refractory DPd Key Rd, Drug to R last Year maint, Prior Population Full Vel Approval + Rev, ORR29.2% for Combinations >1 Poma
Slide 9: STORM: Selinexor in Refractory Myeloma: Quad and Penta Populations
STORM: Selinexor in Refractory Myeloma: Quad and Penta Populations
Slide 10: STORM Patient Characteristics
STORM Females Median (77%) * Prior one 24 – Patient (50%) patient Regimens Characteristics : 24 did(50%) not (range) have 137measurable (42%) STORM (3 – 16) : 18 7Patient (5 (58%) disease – 17) Characteristics Patients Prior at baseline Therapies treated ©2017 Quad with Alkylating Karyopharm Refract
Slide 11: STORM Efficacy
STORM 44 Vogl 20DT, 41 Efficacy 22 et al. MR ASH 40 (per SD 2016. IRC) 17 Abstract 19100 12 58 ORR: PD 491.20©2017 48ORR: NE 21 Karyopharm 7 19 ORR: 23 22 6 ORR: 19 Therapeutics 0 Overall 4 ORR:Quad 21% Inc. 11 Penta 17 21%6 Doses 13 20%8 Doses 14 20%(n19 = 78) 22%(n15= 80 48)CBR: (n = 30) CBR:perCB
Slide 12: STORM: Overall Survival in Patients with MR vs SD
STORM: Overall Survival (OS) in Patients with MR vs SD Median All (N=78) MR (N=26) SD (N=52)* OS 9.3 mos Not Reached 5.7 mos * Includes 16 NE patients ©2017 Karyopharm Therapeutics Inc. 12 Storm study
Slide 13: STORM Treatment-Related Adverse Events
Treatment 10% patients Neutropenia Karyopharm 4%(18%) —Related 44% 3% Therapeutics discontinued 4% interruptions Adverse 11% 6%Inc. Events Weight 24% Diarrhea 13 Lymphopenia 10% Loss34% AE 19% Term 4% 13% 5% — Grade1 1% 4% ——43% 9%Grade 33% 1% Dehydration Blood 14% 2 Grade CPK treatment 3 1% Increas
Slide 14: Multiple added opportunities in Myeloma
Multiple added opportunities in the Myeloma US • Assuming andAccelerated EU topositive support Approval data expansion from Strategy STORM • Target STORM expansion, topline Expansion dataplan from toUnderway the seekexpanded accelerated (N=122 cohort approval additional in early (FDA) patients) 2018
Slide 15: STOMP: Selinexor Backbone Treatments for Relapsed/Refractory Multiple Myeloma
SelinexorSelinexor STOMP: + velcadeand bortezomib Backbone+ dexamethasone Treatments for Relapsed/Refractory svd efficacy phase 1 Multiple Myeloma
Slide 16: SVd efficacy in Phase 1
sCR CR 45% population 20%VGPR 40% 201740% PR Karyopharm 100% 10% 0% ORRAllPa Therapeutics 100%ents(N=22) ORRInc. 100%* 16 PIRefractory 14% 17%PINon-Refractory 90% ORR 80% 80% PriorTherapies1-3 ORR 77% ORRPriorTherapies 74% 5% 10% 429% PINon-Refractory 70% ORR 67%(N=15) 8% 9%(N=7) 33% 7% (N=10) 8% 60
Slide 17: SVd Phase 1 safety and recommended dose
Escalation peripheral Diarrhea 1.3 mg/m218% Gastrointestinal Patients Velcade — 6% –SC Treatment —QW24%neuropathy Thrombocytopenia and Related efficacy across AEsacross atallRecommended —SVd all—cohorts, cohorts 6% 12% the Nausea Dose 18% Altered 40 100 24% mgTaste mg35% oral Sel 6%—6%—12% dexametha
Slide 18: Selinexor – Kyprolis – Dex Reinforces Sel+PI Synergy
Selinexor MR PR VGPR – Kyprolis • A Jakubowiak, – Dex Reinforces et al. ASH Sel+PI 2016. Synergy • Study* sponsored All Pts Allby Ptsthe CFZ-ref University at lastoftherapy ChicagoAfter with1support cycle Best fromresponse Karyopharm, Best response Amgen and 100 the 84 84 MMRF. % 80 ©2017 75 63 67 K
Slide 19: Selinexor + Pomalyst + Dexamethasone (SPd) Efficacy
Selinexor SD(%) PR. (VGPR+PR+MR). ORR=Overall PD(%) + Pomalyst All Patients Response Responses (Pomalidomide) 15Rate 9as(60%) of(VGPR+PR), November +11Dexamethasone (73%) 28, VGPR=Very 3 2016 (20%)based (SPd) 6 (40%) Good on Efficacy interim 2 Partial (13%)–unaudited Phase Response, 3 (20%) 1 Best 1
Slide 20: BOSTON Phase 3 Randomized Trial
Randomized in •Velcade (EP) Karyopharm Interim Patients • PFS scPFS with dosing Key Therapeutics Phase analysis Relapsed/Refractory Secondary 43/ Global 5after weeks Inc. EPs 75% “BOSTON” with 20 • of ORR, weekly the Myeloma PFS DOR, Trial selinexor events PFS A• 1-3 Phase 1for prior + and potential
Slide 21: SADAL (DLBCL) & SOPRA (AML)
SADAL (DLBCL) & SOPRA (AML)
Slide 22: SADAL: Randomized Phase 2b Trial in Relapsed DLBCL
SADAL: ABC) endpoint: • Targeting Randomized overall response up toPhase 200rate patients 2b• Trial Data• in Relapsed readout DLBCL anticipated / SADAL: refractoryinSelinexor 3rd early line; 2017 At Against least ©2017 50% Diffuse Karyopharm of patients Aggressive Therapeutics withLymphoma GCB-DLBCL
Slide 23: SOPRA: Randomized Phase 2 Trial in 2nd Line AML
SOPRA: dose 2017; CR/CR(i/p)] cytarabine topline Randomized •data 45orpatients readout supportive Phase with anticipated 2care Trial stable only indisease mid-2017 2nd • Randomized Line (58%) AML Phase • 68% SOPRA: 2:1, 1 Clinical (53 openof Selinexor label, Results 78) disease single-agent •inBased
Slide 24: Solid Tumors
&) ~ Solid Tumors |
Slide 25: SEAL: Phase 2/3 Trial in Liposarcoma
SEAL: 1:1, Phasedouble 2Phase portion blind, 2/3anticipated Trial single-agent in Liposarcoma in mid-2017; selinexorSEAL: (60mg study Selinexor may fixedbedose, expanded in twice Advanced to weekly) Phase Liposarcoma vs. 3 following placebo • Phase •an Primary interim 2 portion endpoint: analysis ta
Slide 26: SIGN: Phase 2 in Gynecological Malignancies
SIGN: 3Phase (42%) according Congress(16%) 2016 to the 2 in by (QW) Response Gynecological Ignace All Doses Vergote Evaluation 59 Malignancies ©2017 29 Criteria (49%) Karyopharm 8in*(14%) Solid SD >21Therapeutics 12 Tumors (36%) Cancer (RECIST 50Type mg/m2 Inc.Dose 26 v1.1) Endometrial N based DCR (
Slide 27: KPT-8602: Second Generation SINE Compound
KPT-8602: series penetration dosing patients with with with pharmacological Second •continued KPT-8602 MDS, Generation colorectal XPO1 can be properties inhibition &SINE™ dosed castrate daily distinct Compound has resistant over potential from five prostate • selinexor days Karyopharm to overcome pe
Slide 28: Financial Overview
Financial 2016 • Basic: Overview ~41.9 MM – Unaudited commonProjections shares • Fully Cash, diluted: Cash Equivalents ~47.7 MM common & Investments shares •©2017 ~$175Karyopharm MM at December Therapeutics 31, 2016Inc. Expected 28 to fund the Company until the end of 2018 Shares Outstanding as of D
Slide 29: Anticipated Corporate Milestones
Anticipated Phase 2 interim 1/2 top-line Corporate analysis dataMilestones (safety Early and 2017STORM: tolerability) AML PhasePhase Mid 2 top-line 2b2017 expansion ©2017 data (OS) top-line Karyopharm Middata 2017Early Therapeutics Solid2018 Tumors Multiple Inc. SEAL: 29Myeloma Phase 2BOSTON: top-li
Slide 30: Karyopharm Overview
Karyopharm broadly candidates including enrollment 2032 ©2017 home, major are asKaryopharm Overview aCombinable broad backbone diseases readouts SINE™ Nuclear Fully Therapeutics months management owned; Near Export accelerated Term Inc. COM safety Strong until 30KPT-8602 approvals profile theWell en
Slide 31: Thank you
Thank you
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