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Corporate Presentation
Targeting Disease at the Nuclear Pore
Forward-looking Statements
This presentation contains forward-looking statements within the meaning of the safe harbor provisions of The Private Securities Litigation
Such forward-looking statements include those regarding the therapeutic potential of and potential clinical development plans and commercialization for Karyopharm s lead drug
candidate, selinexor (KPT-330), including the timing of initiation of certain trials and of the reporting of data from such trials, as well as assumptions of management and financial expectations and projections.
Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results
to differ materially from the company s current expectations. For example, there can be no guarantee that Selinexor or any other drug candidate Karyopharm is developing will successfully complete necessary preclinical and clinical development
phases or that development of any of Karyopharm s drug candidates will continue. Further, there can be no guarantee that any positive developments in Karyopharm s drug candidate portfolio will result in stock price appreciation. In
addition, even if Karyopharm receives marketing approval for selinexor or another drug candidate, there can be no assurance that Karyopharm will be able to successfully commercialize that drug candidate. Management s expectations and,
therefore, any forward-looking statements in this presentation could also be affected by risks and uncertainties relating to a number of other factors, many of which are beyond Karyopharm s control, including the following: Karyopharm s
results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. Food and Drug Administration and other
regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Karyopharm s ability to obtain and maintain requisite regulatory approvals and to enroll patients in its clinical trials; unplanned
cash requirements and expenditures; development of drug candidates by Karyopharm s competitors for diseases for which Karyopharm is currently developing its drug candidates; and Karyopharm s ability to obtain, maintain and enforce patent
and other intellectual property protection for any drug candidates it is developing.
These and other risks,
including those which may impact management s expectations, are described in greater detail under the heading Risk
Factors in Karyopharm s Annual Report on Form 10-Q for the quarter ended June 30, 2015, which is on file with the Securities and Exchange Commission, and in subsequent
filings filed by Karyopharm with the Securities and Exchange Commission.
Any forward-looking statements
contained in this presentation are for informational purposes only and speak only as of the date hereof. Other than as is required by law, Karyopharm expressly disclaims any obligation to update any forward-looking statements, whether as a result of
new information, future events or otherwise.
Karyopharm s website is http://www.karyopharm.com.
Karyopharm regularly uses its website to post information regarding its business, drug development programs and governance. Karyopharm encourages investors to use www.karyopharm.com, particularly the information in the section entitled
Investors, as a source of information about Karyopharm. References to www.karyopharm.com in this presentation are not intended to, nor shall they be deemed to, incorporate information on www.karyopharm.com into this presentation by
Unless otherwise noted, this presentation contains data that are interim and unaudited based on
Karyopharm: At the Nucleus of Cancer Care
Karyopharm s wholly owned, lead SINE compound selinexor offers an entirely new approach to cancer therapy that restores the ability of a cell to detect cancerous changes and
Novel mechanism First in class Oral, small molecule Fully Owned
Tumor Suppressor Protein (TSP) activation and oncoprotein reduction
Potentially relevant to any type of cancer
>40 combination studies ongoing or planned
Prepare to commercialize selinexor in NA and Western EU
Seek collaborators for other geographies
New Drug Class in Oncology
Clinical Development
Initial focus on the regulatory approval and commercialization of oral selinexor as a single-agent, while developing SINE compounds as novel approaches to treat a variety of diseases
in areas of unmet medical need
SINE Compound with Broad Clinical Development Strategy
DRUG CANDIDATE AREA OF THERAPY PRECLINICAL PHASE
1 & PHASE 2 PHASE 2 or 3
Oral Selinexor (KPT-330)
Hematological Malignancies
Multiple Myeloma (STORM)
Multiple Myeloma (STOMP)
Sel+Pom+Dex; Sel+Bort+Dex; Sel+Len+Dex
Sel+Rituximab + backbone therapies
Various Hematological Malignancies
Gynecologic Malignancies (SIGN) Glioblastoma (KING) Prostate (SHIP)
Ovarian or Endometrial
Selinexor + Paclitaxel +
Various Solid Tumors
Standard Chemotherapy Regimens
Investigator Sponsored
Novel Anti-Cancer Agent: Restores Tumor Suppressors & Reduces Oncoproteins
Nuclear Pore Complex
Blocks XPO1 Forcing Nuclear Localization of Tumor Suppressor Proteins
Focus for Single-Agent Approval of Selinexor in:
Acute Myeloid Leukemia
Richter s Transformation
7 2015 Karyopharm Therapeutics Inc.
Selinexor s Broad and Durable Oral Single-Agent Anti-Tumor Activity
Selinexor 1,000+ up to 80% >6 month up to 50%
Broad and durable single-agent anti-tumor activity in a pill Patients treated to date with single agent or in combination Disease control across hematologic malignancies Durability
some patients on >1 year (longest 2+ years)
Disease control rates in patients with advanced solid tumors
First Potential Approvals
Key Milestones for First Potential Approvals:
Top Line Data Q4 2016
Top Line Data Mid-2017
US Patient Populations by Indication:
Citations: ACS and Leukemia and
Lymphoma Society, Pasqualucci et al, 2013;
ACS, Tsimberidou et al, MDACC; Globocan 2012, IARC; ACS 9
Large B-Cell Lymphoma: The Opportunity
Aggressive form of Non-Hodgkin s Lymphoma (NHL)
Incidence: 25,000 new cases annually in the US
~40% of incident patients will succumb to their disease
~10,000 deaths per year in USA (~22,500 worldwide)
Greatest unmet need currently in GCB and Double-Hit Subtypes of DLBCL
Selinexor has shown durable single-agent activity across all forms of DLBCL
Citations: ACS and Leukemia and Lymphoma Society, Pasqualucci et al, 2013
1 Data Recently Highlighted at ASH Meeting
Fifty-two patients, heavily pretreated (median 4 therapies) with
Anti-tumor activity across all NHL types
Durable cancer control and median DOR ~7 months (some pts on >1 year)
Overall response rate ( PR): 37% (10% CR, 27% PR)
Disease control rate ( SD): 73%
Type N DCR (%) ORR (%) CR (%) PR (%) SD (%) PD (%)
DLBCL 32 21 (66%) 11 (34%) 4 (13%) 7 (22%) 10 (31%) 11 (34%) Richter s 4 4 (100%) 2 (50%) 2 (50%) 2 (50%) Other NHL 16 13
(81%) 6 (38%) 1 (6%) 5 (31%) 7 (44%) 3 (19%)
RESPONSES IN IDENTIFIED SUBTYPES
Type N DCR (%) ORR (%) CR (%) PR (%) SD (%) PD (%)
GCB 11 9 (82%) 4 (36%) 1 (9%) 3 (27%) 5 (45%) 2 (18%) Non GCB 5 4 (80%) 2 (40%) 1
(20%) 1 (20%) 2 (40%) 1 (20%)
PATIENTS WITH DOUBLE HIT DLBCL
Patient ID Best Response % Reduction in Lymph Nodes Days on Study Prior Therapies
046 CR 73% (PET Negative) 429+ CHOP-R, RICE 058 PD 57 CHOP-R, RICE
072 PR -65% 214 R-CHOP, Benda, RICE, DHAP-R, BEAM 086 SD -45% 104 CHOP-R, GDP, Ibrutinib+Lenalidomide
Data as of December 1, 2014