Karyopharm Therapeutics Inc. · NASDAQ:KPTI
Karyopharm Therapeutics — JP Morgan Healthcare Conference 2019
34 slides from Karyopharm Therapeutics's conference presentation, Jan 7, 2019
Slide 1: Targeting Disease at the Nuclear Pore
JP Morgan Healthcare Conference January 2019 Targeting Disease at the Nuclear Pore Exhibit 99.1
Slide 2: Forward-looking Statements and Other Important Information
Forward-looking Statements and Other Important Information 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 potenti
Slide 3: Agenda
©2019 Karyopharm Therapeutics Inc. Agenda Overview of Karyopharm Review of recently presented clinical data Summary of selinexor tolerability profile Potential commercial opportunity Additional pipeline activities Financial update and expected near-term milestones
Slide 4: Founded years ago by team of highly successful cancer drug developers; Initial public offering
Founded 10 years ago by team of highly successful cancer drug developers; Initial public offering in 2013 Novel, focused technology approach with potential for broad clinical applicability Industry leader in targeting nuclear export dysregulation as a mechanism to treat cancer All programs developed
Slide 5: Progress and Plans to Become an Oncology Leader
Progress and Plans to Become an Oncology Leader ©2019 Karyopharm Therapeutics Inc. 2013-2018 Supported the growing body of clinical data for our lead investigational compound 2019 Goals Transition to a commercial oncology company 2020+ Goals Expand commercial position, drive broader utilization and
Slide 6: Selinexor/SINE Mechanism of Action: Inhibition of XPO1
Increases nuclear levels and activation of tumor suppressor proteins Traps oncoprotein mRNA in the nucleus leading to reduced oncoprotein levels Retains activated glucocorticoid receptor in the nucleus Selinexor/SINE Mechanism of Action: Inhibition of XPO11-4 ©2019 Karyopharm Therapeutics Inc. Inhib
Slide 7: 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 8: Selinexor Clinical Data Update
Selinexor Clinical Data Update Multiple Myeloma Diffuse Large-B Cell Lymphoma (DLBCL)
Slide 9: Planned Selinexor Development Strategy in Multiple Myeloma
Planned Selinexor Development Strategy in Multiple Myeloma ©2019 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 10: STORM: A Phase 2b Study In Refractory Myeloma
Single-arm clinical trial evaluating selinexor in combination with low-dose dexamethasone in patients2 with penta-refractory3 myeloma STORM1: A Phase 2b Study In Refractory Myeloma 1 Selinexor Treatment of Refractory Myeloma 2 Median 7 prior regimens 3 Patients who have previously received two PIs,
Slide 11: STORM: A Phase 2b Study In Refractory Myeloma
STORM: A Phase 2b Study In Refractory Myeloma1,2 Safety: As anticipated, the most common non-hematologic AEs were fatigue, nausea, reduced appetite, and weight loss; primarily low grade and manageable with standard supportive care and/or dose modification The most common hematologic AEs were Grade ≥
Slide 12: BOSTON: A Phase 3 Study as 2nd Line+ Treatment 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 as 2nd Line+
Slide 13: 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” therapies2 in patients with relapsed or refractory MM 1 Selinexor and Backbone Treatments of Multiple Myeloma Patients; dosing regime
Slide 14: Selinexor and Backbone Treatments of Multiple Myeloma Patients. 2 Bahlis NJ, et al. Blood
©2019 Karyopharm Therapeutics Inc. 1 Selinexor and Backbone Treatments of Multiple Myeloma Patients. 2 Bahlis NJ, et al. Blood 2018. 3 Gasparetto C, et al. ASH 2018. Abstract 599. 4 Patient population eligible for Phase 3 BOSTON study. 5 Dimopoulos MA et al., Lancet 2016. 6 Lonial et al., Lancet 201
Slide 15: Selinexor and Backbone Treatments of Multiple Myeloma Patients. 2 White D, et al. ASH
©2019 Karyopharm Therapeutics Inc. 1 Selinexor and Backbone Treatments of Multiple Myeloma Patients. 2 White D, et al. ASH 2017. Abstract 1861. 3 Chen C, et al. ASH 2018. Abstract 1993. 4 Stewart et al. NEJM 2015. 5 Pomalyst Package Insert. 6 Revlimid®, Pomalyst®, Velcade®, Kyprolis® or Darzalex®. S
Slide 16: 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; Approximately 50% of patients with GCB-DLBCL 1 Selinexor Against Diffuse Aggressive Lymphoma ©2019 Karyopharm Therapeutics Inc. Enrollment completed and
Slide 17: SADAL: A Phase 2b Study In DLBCL
©2019 Karyopharm Therapeutics Inc. Safety: Most common treatment related non-hematologic AEs were fatigue, nausea and anorexia, primarily Grade 1/2, and most were manageable with dose modifications and/or supportive care Most common Grade 3/4 AEs were thrombocytopenia, anemia, and neutropenia, and m
Slide 18: Selinexor: A Growing Body of Safety Data
Selinexor: A Growing Body of Safety Data Side effects of dosing regimens utilized in key Phase 2 and 3 trials (e.g., BOSTON, STORM, STOMP, SADAL and SEAL) were generally predictable and often managed with dose adjustments or supportive care, particularly when used once weekly in combination regimens
Slide 19: STORM1 STOMP – Darzalex® Combination Arm2 STOMP – Velcade® Combination Arm3 SADAL4 SEAL5 Disease
©2019 Karyopharm Therapeutics Inc. STORM1 STOMP – Darzalex® Combination Arm2 STOMP – Velcade® Combination Arm3 SADAL4 SEAL5 Disease Setting Relapsed/Refractory Multiple Myeloma Relapsed/Refractory Multiple Myeloma Relapsed/Refractory Multiple Myeloma Relapsed/Refractory DLBCL Relapsed/Refractory Lip
Slide 20: Sizing the Commercial Opportunity
Sizing the Commercial Opportunity Multiple Myeloma DLBCL
Slide 21: Multiple Myeloma
Deaths occur in the U.S. each year1 ~13,000 Relapsed or refractory patients are treated with drug-therapy annually3 An estimated 36,000 (U.S.) and 41,000 (EU5) people worldwide are diagnosed annually2 86,000 An estimated 30,000 people in the US are diagnosed annually1 An estimated 1 American Cancer
Slide 22: Total WW Market for Myeloma Drugs is >$17B Annually
Total WW Market for Myeloma Drugs is >$17B1 Annually with Significant Opportunity for Numerous Brands in the Refractory Setting Bars are not mutually exclusive; a regimen containing two agents would appear in bars for both agents Drug Year Launched (U.S.)1 Initial Myeloma Indication Settings1 Curren
Slide 23: 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 24: Total Major Market Sales for DLBCL Drugs Expected to Grow
Total Major Market Sales for DLBCL Drugs Expected to Grow from $1.9B in 2016 to Over $4B by 20221 ©2019 Karyopharm Therapeutics Inc. Total sales in the relapsed / refractory (R/R) setting are expected to exceed $1B by 2026 with the expectation of new, approved drugs coming to market1 1 Decision Reso
Slide 25: Key Features of Selinexor in Clinical Studies
©2019 Karyopharm Therapeutics Inc. Key Features of Selinexor in Clinical Studies Provide Foundation for Potential Future Commercial Success Single agent activity demonstrated across multiple tumor types In multiple myeloma, selinexor has been studied in combination with dexamethasone Durable respons
Slide 26: Building Commercial Capabilities
©2019 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 27: Future Opportunities and Additional Highlights
Future Opportunities and Additional Highlights
Slide 28: Selinexor in Solid Tumor Malignancies
Selinexor in Solid Tumor Malignancies ©2019 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 29: SIGN: A Phase 2 Study in Heavily Pretreated Gynecological Malignancies
Cancer Type Dose N DCR (%) PR (%) SD > 12 Weeks (%) Ovarian (platinum refractory; median 6 priors) ~60 mg (BIW)2 18 11 (61%) 2 (11%) ~85 mg (BIW)2 22 10 (45%) 3 (14%) ~85 mg (QW)3 19 8 (42%) 3 (16%) All Doses 59 29 (49%) 8 (14%) 21 (36%) Endometrial (median 2 priors) ~85 mg (BIW)2 20 9 (45%) 3 (15%)
Slide 30: 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 ©2019 Karyopharm
Slide 31: Financial Highlights
$330M Into 2Q 2020 BALANCE SHEET SHARES OUTSTANDING 31-Dec-20181 EXPECTED RUNWAY WITH CASH ON HAND Financial Highlights ©2019 Karyopharm Therapeutics Inc. Cash, cash equivalents and investments – Dec 20181 70M fully diluted 61M 1 Unaudited.
Slide 32: Expected Milestones: Looking Ahead to an Event-Driven 2019-20
©2019 Karyopharm Therapeutics Inc. Expected Milestones: Looking Ahead to an Event-Driven 2019-20 1 Patients who have previously received two PIs, Velcade® (bortezomib) and Kyprolis® (carfilzomib), and two IMiDs, Revlimid® (lenalidomide) and Pomalyst® (pomalidomide), and the anti-CD38 monoclonal anti
Slide 33: Summary of Karyopharm
©2019 Karyopharm Therapeutics Inc. Summary of Karyopharm 1st in class, novel, oral lead compound with demonstrated single-agent and synergistic combination activity Positive clinical data recently presented in MM and DLBCL Fast Track designation (MM and DLBCL) and Priority Review (MM: PDUFA date of
Slide 34: Thank You
Thank You
Karyopharm Therapeutics — JP Morgan Healthcare Conference 2019 on 6ix