Full Press Release Details
Targeting Disease at the Nuclear Pore
Corporate Presentation
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 development plans and commercialization for Karyopharm s drug candidates, including the timing of
initiation of certain trials, of the reporting of data from such trials, of the submissions to regulatory authorities and of potential commercial launches, the potential availability of accelerated approval pathways, the potential size of the
markets for multiple myeloma drugs and multiple myeloma drugs for treatment of patients with relapsed multiple myeloma and Karyopharm s strategic and financial plans and expectations as well as financial projections for Karyopharm. Such
statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Karyopharm s current expectations. For example, there can be no guarantee that regulators will agree
that selinexor qualifies for accelerated approval in the U.S. or conditional approval in the E.U. as a result of the data from the STORM study in patients with penta-refractory myeloma or SADAL study in patients with relapsed/refractory DLBCL or
that any of Karyopharm s drug candidates, including selinexor (KPT-330) and eltanexor (KPT-8602), Karyopharm s second generation SINE compound, or KPT-9274, Karyopharm s first-in-class oral dual inhibitor of PAK4 and NAMPT, 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 FDA and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies, including with respect to the need for additional clinical studies; the ability of Karyopharm or its
third party collaborators or successors in interest to fully perform their respective obligations under the applicable agreement and the potential future financial implications of such agreement; unplanned cash requirements and expenditures;
development of drug candidates by Karyopharm s competitors for diseases for which Karyopharm is currently developing its drug candidates; that the markets for multiple myeloma drugs will grow as predicted; 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 Quarterly Report on Form 10-Q for the quarter ended March 31, 2018, which is on file with the SEC, and in subsequent filings filed by Karyopharm
with the SEC. 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 reference. Unless otherwise noted, this presentation contains data that are interim and unaudited based on
site reports. In addition, data included in this presentation have not been updated and are as of the cutoff date for the applicable medical conference presentation. Unless otherwise noted, all reference to market sizes are based on reports from
Global Data or Karyopharm internal estimates.
2018 Karyopharm Therapeutics Inc. 2
Karyopharm Highlights
Diffuse Large B-cell Lymphoma
First-in-class, oral SINE compounds
Lead asset with significant commercial potential
Strong leadership team and balance sheet
Multiple near term catalysts from broad, late-stage pipeline
potential product launches and revenues
Novel, broadly applicable mechanism of action with single agent activity and works well in combinations; patent life
Selinexor demonstrated robust activity in penta-refractory myeloma, is combinable with existing therapies and has the potential to be the next
backbone therapy in myeloma, a market projected to be $22.6B in 20231
Executive team with past successes in lead indications; with recent offering
proceeds, cash expected to fund the company into 3Q 2019
Anticipated data readouts (2018-2019): SADAL (end of 2018),
BOSTON (end of 2019; pending PFS events), SEAL (end of 2019) Regulatory Profile: Fast Track and orphan designations granted in multiple myeloma. Planned requests for accelerated
approval in multiple myeloma (2H18), accelerated approval in DLBCL (2019)2, full approval in multiple myeloma (2020)3, full approval in liposarcoma (2020)4
Preparing for potential launch of selinexor in multiple myeloma and DLBCL in 2019, followed by expansion to other potential indications
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 from Phase 3 portion of
2018 Karyopharm Therapeutics Inc.
HEMATOLOGIC MALIGNANCIES
Relapsed/refractory | BOSTON Penta-refractory | STORM Relapsed/refractory | STOMP
Diffuse Large B-cell Lymphoma | SADAL
Endometrial Cancer | SIENDO4
ADDITIONAL ONCOLOGY PROGRAMS
Solid Tumors & Lymphoma
Lymphoma in Companion Animals
PRECLINICAL PHASE I PHASE II PHASE III COMMERCIAL RIGHTS
SELINEXOR + VELCADE1 SELINEXOR2
SELINEXOR + BACKBONE THERAPIES3 SELINEXOR
ELTANEXOR (KPT-8602)
Oral SINE compound and XPO1 inhibitor KPT-350
acquired by Biogen for treatment of certain neurological and neurodegenerative conditions, including ALS, in Jan 2018
1 Oral selinexor, Velcade (bortezomib)
and dexamethasone vs. Velcade and dexamethasone
2 Oral selinexor + dexamethasone
3 Oral selinexor and dexamethasone + Revlimid (lenalidomide), Pomalyst (pomalidomide), Velcade, Kyprolis (carfilzomib) or Darzalex (daratumumab)
4 Investigator-sponsored randomized Phase III trial
*Territories: Japan, S. Korea, Taiwan,
Hong Kong, and ASEAN countries.
** Antengene licensed rights to selinexor and eltanexor in China and Macau.
*** Antengene licensed rights to KPT-9274 and verdinexor in mainland China, Macau, Taiwan, Hong Kong, South Korea, and the ASEAN countries.
**** Anivive holds exclusive worldwide rights to research, develop and commercialize verdinexor only for the treatment of cancer in companion animals
2018 Karyopharm Therapeutics Inc.
Selinexor/SINE Mechanism of Action: Inhibition of XPO1
2018 Karyopharm Therapeutics Inc.
Selinexor: A Growing Body of Safety Data
Total patients treated to date
Number of clinical trials ongoing and/or completed
Number of cancers explored
Favorable emerging tolerability profile; multiple ongoing Phase 3 studies
regimens utilized in key Phase 2 and 3 trials (e.g., BOSTON, STORM, STOMP, SADAL and SEAL) have shown generally predictable and manageable tolerability, particularly when used once weekly in combination regimens
Common side effects are generally predictable across multiple tumor types and generally reversible and/or manageable with standard supportive care
Combination regimens have demonstrated a predictable and manageable tolerability profile, with observed additive or synergistic activity and potential to re-sensitize to prior therapies
Phase 3 BOSTON selinexor regimen allows for use of 40% less Velcade and 25% less
dexamethasone than standard Velcade and dexamethasone dosing
In certain trials, side effects reported from treatment arms evaluating selinexor and
dexamethasone in combination with other anti-myeloma agents are similar to, or reduced, compared to selinexor and dexamethasone alone
1 Per ClinicalTrials.gov (as
of Apr 26, 2018); 5 of 46 are company-sponsored and currently enrolling
2018 Karyopharm Therapeutics Inc.
Selinexor: Regulatory Strategy in United States and Europe
FDA granted Fast Track status to selinexor in multiple myeloma and Orphan
Drug Designation has been awarded to selinexor in multiple myeloma and
by FDA and EMA and in liposarcoma by FDA
Fast Track allows more frequent interaction with FDA, rolling New Drug Application
(NDA) submission and ability to request priority review and accelerated approval
Drug Designation in the US and EU can provide regulatory development
support, tax credits (US), reduced or waived marketing applications fees and
marketing exclusivity periods
Planning to utilize Fast Track benefits in submitting
first NDA during the second
half of 2018 based on data from the STORM study of penta-refractory myeloma
EMA submission expected to follow in early 2019 with a request for conditional
in penta-refractory myeloma
Marketing approval opportunities in multiple indications: multiple myeloma