Karyopharm Therapeutics Inc. · NASDAQ:KPTI
Karyopharm Therapeutics — Investor Presentation (June 2018)
29 slides from Karyopharm Therapeutics's investor presentation, Jun 20, 2018
Slide 1: Corporate Presentation June 2018: Targeting Disease at the Nuclear Pore
Corporate Presentation June 2018 Targeting Disease at the Nuclear Pore Exhibit 99.1
Slide 2: 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 3: Karyopharm Highlights
Karyopharm Highlights Strong leadership team and balance sheet First-in-class, oral SINE compounds Lead asset with significant commercial potential 1 Global Data, 2 Assuming positive outcome from Phase 2b SADAL study, 3 Assuming positive outcome from Phase 3 BOSTON study, 4 Assuming positive outcome
Slide 4: All Oral Pipeline
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 + Revlimid® (lenali
Slide 5: Selinexor/SINE Mechanism of Action: Inhibition of XPO1
Selinexor/SINE Mechanism of Action: Inhibition of XPO1 ©2018 Karyopharm Therapeutics Inc.
Slide 6: Selinexor: A Growing Body of Safety Data
Selinexor: A Growing Body of Safety Data 2,400 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 generally predictable and manageable to
Slide 7: Selinexor: Regulatory Strategy in United States and Europe
Selinexor: Regulatory Strategy in United States and Europe Multiple Myeloma FDA granted Fast Track status to selinexor in multiple myeloma and Orphan Drug Designation has been awarded to selinexor in multiple myeloma and DLBCL by FDA and EMA and in liposarcoma by FDA Fast Track allows more frequent
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: 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-refractory2 myeloma Disease refractory to PIs, IMiDs and Darzalex® Highest unmet medical need in multiple myeloma 1 Evaluating selinexor with low-dose de
Slide 11: 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 Selinexor received Fast Track designation from the FDA for this indication, acknowledging the unmet need STORM1: A Phase 2b Study In Myeloma 1 Selinexor Treat
Slide 12: STORM: A Phase 2b Study In Myeloma
STORM: A Phase 2b Study In Myeloma Safety: Predictable and manageable tolerability profile; results consistent with those reported previously from Part 1 of STORM and from other selinexor studies. As anticipated, the most common non-hematologic AEs were nausea, vomiting, fatigue and reduced appetite
Slide 13: BOSTON: A Phase 3 Study In Myeloma
Ongoing randomized, open-label clinical trial evaluating once weekly selinexor and Velcade® (bortezomib) plus low-dose dex versus standard twice-weekly Velcade® plus 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
Slide 14: STOMP: A Phase 1b/2 Study In Myeloma
STOMP: A Phase 1b/2 Study In Myeloma Selinexor + Velcade® + dex (SVd)3 (n=40) Selinexor + Pomalyst® + dex (SPd)2 (n=30) 1 White D, et al. ASH 2017. Abstract 1861. 2 Chen C, et al. EHA 2018. Abstract PF586. 3 Bahlis NJ, et al. EHA 2018. Abstract PS1322. 4 Patient population eligible for Phase 3 BOSTO
Slide 15: Myeloma Treatment Landscape and Selinexor Opportunity
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 16: Selinexor in Hematologic Malignancies: Diffuse Large B-Cell Lymphoma
Selinexor in Hematologic Malignancies Diffuse Large B-Cell Lymphoma
Slide 17: 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 U.S. 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
Slide 18: 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 (≥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 end
Slide 19: 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 20: 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 21: Other Programs and Assets
Other Programs and Assets Selinexor in Solid Tumor Malignancies Eltanexor KPT-9274
Slide 22: Selinexor in Solid Tumor Malignancies
Selinexor in Solid Tumor Malignancies ©2018 Karyopharm Therapeutics Inc. Selinexor in Liposarcoma Selinexor in Endometrial Cancer Ongoing Phase 3 SEAL study; randomized, double-blind trial evaluating single-agent selinexor versus placebo in patients with advanced unresectable dedifferentiated liposa
Slide 23: 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 24: Building Commercial Capabilities
©2018 Karyopharm Therapeutics Inc. Building Commercial Capabilities 1 Transaction includes Japan, South Korea, Taiwan, Hong Kong and ASEAN countries. 2 Transaction includes China and Macau for selinexor and eltanexor and China, Macau, Taiwan, Hong Kong, South Korea and the ASEAN countries for verdin
Slide 25: Management Team
Michael Kauffman Chief Executive Officer Jatin Shah SVP, Clinical Development Significant Regulatory and Commercial Experience Mike Falvey Chief Financial Officer Chris Primiano Chief Business Officer Bill Hatfield SVP, Product Strategy & Market Development Joan Wood Chief Human Resources Officer Ra
Slide 26: Financial Highlights
$142M Into 3Q 2019 BALANCE SHEET SHARES OUTSTANDING 8-May-2018 EXPECTED RUNWAY WITH CASH ON HAND AND NET OFFERING PROCEEDS Financial Highlights ©2018 Karyopharm Therapeutics Inc. Cash, cash equivalents and investments – March 2018 Expected 2018 operating cash burn - $175-185M2,3 69M fully diluted 60
Slide 27: Expected Milestones: Looking Ahead to an Event-Driven 2018-20
©2018 Karyopharm Therapeutics Inc. Expected Milestones: Looking Ahead to an Event-Driven 2018-20 1 Patients who have previously received two PIs, Velcade® (bortezomib) and Kyprolis® (carfilzomib), and two IMiDs, Revlimid® (lenalidomide) and Pomalyst® (pomalidomide), and their disease is refractory t
Slide 28: Karyopharm Highlights
Karyopharm Highlights Strong leadership team and balance sheet First-in-class, oral SINE compounds Lead asset with significant commercial potential 1 Global Data. 2 Assuming positive outcome from Phase 2b SADAL study. 3 Assuming positive outcome from Phase 3 BOSTON study. 4 Assuming positive outcome
Slide 29: Thank You
Thank You
Karyopharm Therapeutics — Investor Presentation (June 2018) on 6ix