Karyopharm Therapeutics Inc. · NASDAQ:KPTI
Karyopharm Therapeutics — 6th Annual J.P. Morgan Healthcare Conference 2018
34 slides from Karyopharm Therapeutics's conference presentation, Jan 8, 2018
Slide 1: Targeting Disease at the Nuclear Pore
Targeting Disease at the Nuclear Pore Exhibit 99.1
Slide 2: 6th Annual J.P. Morgan Healthcare Conference
6th Annual J.P. Morgan Healthcare Conference January 8-11, 2018
Slide 3: Forward-looking Statements
Forward-looking Statements This presentation contains forward-looking statements within the meaning of the “safe harbor” provisions of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the therapeutic potential of and potential clinical dev
Slide 4: Karyopharm Highlights
Karyopharm Highlights Strong leadership team and balance sheet First-in-class, oral SINE compounds Lead asset with blockbuster potential 1 Global Data, 2 Assuming a positive outcome from Phase 2b STORM study, 3 Assuming positive outcome from Phase 2b SADAL study, 4 Assuming positive outcome from Pha
Slide 5: Karyopharm's All Oral Pipeline
Karyopharm’s All Oral Pipeline COMMERCIAL RIGHTS Japan* Relapsed/refractory | BOSTON Relapsed/refractory | STOMP Penta-refractory | STORM 1 Oral selinexor, Velcade® (bortezomib) and dexamethasone vs. Velcade and dexamethasone 2 Oral selinexor + dexamethasone 3 Oral selinexor and dexamethasone + Revl
Slide 6: Selinexor/SINE Mechanism of Action: Inhibition of XPO1
Selinexor/SINE Mechanism of Action: Inhibition of XPO1 ©2018 Karyopharm Therapeutics Inc.
Slide 7: Selinexor: A Growing Body of Safety Data
Selinexor: A Growing Body of Safety Data 2,200 Total patients treated to date Favorable emerging tolerability profile; multiple ongoing Phase 3 studies Dosing regimens utilized in key Phase 2 and 3 trials (e.g., BOSTON, STORM, STOMP, SADAL and SEAL) have shown predictable and manageable tolerability
Slide 8: Selinexor in Hematologic Malignancies: Multiple Myeloma
Selinexor in Hematologic Malignancies Multiple Myeloma
Slide 9: Multiple Myeloma
of patients survive 5 years3 50% Americans are currently living with MM3 120,000 Approximately people worldwide are diagnosed annually2 86,000 An estimated 30,000 people in the US are diagnosed annually1 An estimated 1 American Cancer Society 2 Becker, N. Recent Results Cancer Res. 2011;183:25-35. d
Slide 10: U.S. Multiple Myeloma Treatment Landscape in 2018
U.S. Multiple Myeloma Treatment Landscape in 2018 FOLLOW ON 2nd LINE FOLLOW ON 3rd/4th LINE FRONTLINE V=Velcade® (bortezomib), R=Revlimid® (lenalidomide), d=dexamethasone, K=Kyprolis® (carfilzomib), D=Darzalex® (daratumumab), SCT=Stem cell transplantation, R/R=Relapsed/refractory, P=Pomalyst® (pomal
Slide 11: Multiple Myeloma: Potential Market Opportunity
Worldwide MM market estimated ~$11 billion in 2015, increasing to $22 billion by 20234 Individual MM drugs with single-agent1 activity may generate peak sales $1-2B+ Oral selinexor could combine with any of the agents currently in use to potentially improve ORR and PFS VELCADE®1 $>7B2 REVLIMID®1 $>2
Slide 12: Planned Selinexor Development Strategy in Multiple Myeloma
Planned Selinexor Development Strategy in Multiple Myeloma ©2018 Karyopharm Therapeutics Inc. Phase 2b STORM study1 addressing patients with penta-refractory myeloma Patients who have failed PIs, IMiDs and Darzalex® Highest unmet need patients 1 Evaluating selinexor with low-dose dexamethasone Pivot
Slide 13: STORM: A Phase 2b Study In Myeloma
Ongoing single-arm clinical trial evaluating selinexor in combination with low-dose dexamethasone in patients2 with penta-refractory3 myeloma STORM1: A Phase 2b Study In Myeloma 1 Selinexor Treatment of Refractory Myeloma 2 Median 7 prior regimens 3 Patients who have previously received two PIs, Vel
Slide 14: STORM: A Phase 2b Study In Myeloma
STORM: A Phase 2b Study1 In Myeloma Safety: Most common non-hematologic AEs were nausea, anorexia and fatigue; primarily Grade 1/2 and manageable with standard supportive care and/or dose modification Most common hematologic AEs were Grade ³ 3 cytopenias; generally not associated with clinical seque
Slide 15: BOSTON: A Phase 3 Study In Myeloma
Ongoing randomized, open-label clinical trial evaluating once weekly selinexor, Velcade® (bortezomib) and low-dose dexamethasone (dex) versus standard Velcade® and low-dose dex in patients with relapsed or refractory MM, who have had 1-3 prior lines of therapy BOSTON1: A Phase 3 Study In Myeloma 1 B
Slide 16: STOMP: A Phase 1b/2 Study in Myeloma
STOMP1: A Phase 1b/2 Study in Myeloma Ongoing multi-arm clinical trial evaluating selinexor and low-dose dex in combination with several currently marketed “backbone” therapies3 in patients with heavily pretreated, relapsed or refractory MM 1 Selinexor and Backbone Treatments of Multiple Myeloma Pat
Slide 17: STOMP: A Phase 1b/2 Study In Myeloma
Next steps: Efficacy and safety observed in SVd arm informing ongoing Phase 3 BOSTON study Plan to report updated data from ongoing arms in mid and late 2018 Safety: SRd (n=19): Most common Grade 1/2 AEs were nausea, anorexia, fatigue, weight loss, constipation and vomiting; most common Grade ³ 3 ad
Slide 18: Multiple Myeloma Treatment Landscape
FOLLOW ON 2nd LINE FOLLOW ON 3rd/4th LINE FRONTLINE V=Velcade® (bortezomib), R=Revlimid® (lenalidomide), d=dexamethasone, K=Kyprolis® (carfilzomib), D=Darzalex® (daratumumab), SCT=Stem cell transplantation, R/R=Relapsed/refractory, P=Pomalyst® (pomalidomide), DOR=Duration of response, F=Farydak® (pa
Slide 19: Integration of Selinexor into the MM Landscape in 2019 and Beyond
Integration of Selinexor into the MM Landscape in 2019 and Beyond R=Revlimid® (lenalidomide), K=Kyprolis® (carfilzomib), V=Velcade® (bortezomib), P=Pomalyst® (pomalidomide), D=Darzalex® (daratumumab), d=dexamethasone, SCT=Stem cell transplantation, R/R=Relapsed/refractory, DOR=Duration of response,
Slide 20: Selinexor in Hematologic Malignancies: Diffuse Large B-Cell Lymphoma
Selinexor in Hematologic Malignancies Diffuse Large B-Cell Lymphoma
Slide 21: Diffuse Large B-Cell Lymphoma
of patients are cured, but the remainder have limited treatment options and eventually succumb to their disease5 ~60% Overall, 25,000 people in the US are diagnosed annually2,3 An estimated In the U.S., Japan and EU5, an estimated 58,000 people will be diagnosed with DLBCL in 20184 Diffuse Large B-C
Slide 22: US DLBCL Treatment Landscape in 2020+
Fit Patients Intensive ChemoTx R-ICE R-DHAP R-ESHAP R-EPOCH (DA) Others No Response PD CR or PR 2nd line Therapy for Relapsed/Refractory Consolidation US DLBCL Treatment Landscape in 2020+ Partial Response PET Positive Chemoimmunotherapy Possible Cure CR/Pet Neg 30% Unfit Patients Non-Intensive Ther
Slide 23: SADAL: A Phase 2b Study In DLBCL
SADAL1: A Phase 2b Study In DLBCL Ongoing open-label clinical trial evaluating selinexor in patients with relapsed or refractory ( ³ 3rd line) DLBCL; at least 50% of patients with GCB-DLBCL 1 Selinexor Against Diffuse Aggressive Lymphoma ©2018 Karyopharm Therapeutics Inc. Top-line data expected by e
Slide 24: SADAL: A Phase 2b Study In DLBCL
Next steps: Top-line data from expanded 60mg cohort expected by end of 2018 If data are positive, potential NDA submission in 2019 requesting accelerated approval Most common AEs were fatigue, nausea, thrombocytopenia, anorexia, vomiting and anemia; primarily Grade 1/2 and were managed with dose mod
Slide 25: Selinexor in Solid Tumor Malignancies: Liposarcoma, Gynecologic Cancers
Selinexor in Solid Tumor Malignancies Liposarcoma Gynecologic Cancers
Slide 26: SEAL: A Phase 3 Study In Liposarcoma
Next steps: Top-line data expected by the end of 2019 Ongoing randomized, double-blind clinical trial evaluating single-agent selinexor versus placebo in patients with advanced unresectable dedifferentiated liposarcoma after at least two systemic therapies SEAL1: A Phase 3 Study In Liposarcoma Highl
Slide 27: Selinexor in Gynecologic Malignancies
Next steps: Phase 3 investigator-sponsored study to begin in 1H18 Evaluating once weekly selinexor as a maintenance therapy versus placebo in patients with endometrial cancer after 1st or 2nd line chemotherapy The most common Grade 2/3 adverse events were fatigue, nausea, anemia, anorexia, vomiting,
Slide 28: Other Pipeline Programs
Other Pipeline Programs Eltanexor (KPT-8602) Oral dual Inhibitor of PAK4 and NAMPT In Phase 1 clinical testing in advanced solid tumors Generally well tolerated (n=21) with early signals of anti-tumor activity Additional supportive preclinical research presented at ASH 2017 KPT-9274 ©2018 Karyopharm
Slide 29: Karyopharm-Ono Partnership
Licensed selinexor and eltanexor rights to Ono for oncology indications in the Territory Total deal value: up to US$193M plus royalties Receive US $22M upfront from Ono Eligible to receive up to US $170M in development and future commercial milestones, plus royalties Karyopharm retains all rights ou
Slide 30: Leadership Team
Michael Kauffman Chief Executive Officer Jatin Shah VP, Clinical Strategy Significant Regulatory and Commercial Experience Mike Falvey Chief Financial Officer Chris Primiano SVP, Operations, Business Development and General Counsel Bill Hatfield SVP, Product Strategy & Market Development Joan Wood C
Slide 31: Financial Highlights
$176M Into 2019 50M (58M fully diluted) CASH, EQUIVALENTS & INVESTMENTS 31-Dec-171 SHARES OUTSTANDING 31-Dec-171 EXPECTED RUNWAY Financial Highlights ©2018 Karyopharm Therapeutics Inc. 1 Unaudited
Slide 32: Expected Milestones: Looking Ahead to an Event-Driven 2018-19
Liposarcoma SELINEXOR – HEMATOLOGIC MALIGNANCIES Expected Milestones: Looking Ahead to an Event-Driven 2018-19 ADDITIONAL ONCOLOGY PROGRAMS STORM: Report Phase 2b expansion data BOSTON: Report top-line Phase 3 data Report updated Phase 1/2 safety and tolerability data 2018 2019 2020+ Report Phase 2
Slide 33: Karyopharm Highlights
Karyopharm Highlights Strong leadership team and balance sheet First-in-class, oral SINE compounds Lead asset with blockbuster potential 1 Global Data, 2 Assuming a positive outcome from Phase 2b STORM study, 3 Assuming positive outcome from Phase 2b SADAL study, 4 Assuming positive outcome from Pha
Slide 34: Thank You
Thank You
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