- Data Continue to Reinforce the Efficacy and
Safety of Selinexor in Patients with Heavily Pretreated Refractory
Multiple Myeloma -
Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage
pharmaceutical company, today announced updated results from its
Phase 2b STORM study of selinexor (KPT-330), including robust rates
and duration of response, compelling overall survival and a
favorable safety profile, in patients with heavily pretreated
refractory multiple myeloma (MM) at the American Society of
Hematology (ASH) 2016 annual meeting held December 3-6, 2016 in San
Diego. Selinexor is the Company’s lead, novel, oral Selective
Inhibitor of Nuclear Export (SINE™) compound, in development for
the treatment of a variety of malignancies, including MM and acute
myeloid leukemia (AML).
“The data presented today further support the
rationale for selinexor as a promising new treatment for patients
with refractory myeloma with no clearly beneficial treatment
options,” said Sharon Shacham, PhD, MBA, President and Chief
Scientific Officer of Karyopharm. “Based on the exciting STORM data
and the existing unmet medical need, we have expanded the study to
include additional patients with penta-refractory myeloma and
expect to report top-line data from this study in early 2018.”
Updated Phase 2b STORM Clinical Data in
Refractory Multiple Myeloma
In an oral presentation titled, “Selinexor and
Low Dose Dexamethasone in Patients with Lenalidomide, Pomalidomide,
Bortezomib, Carfilzomib and Anti-CD38 Ab Refractory MM STORM
Study,” Dan T. Vogl, MD, MSCE, Assistant Professor of Medicine,
Perelman School of Medicine, University of Pennsylvania, presented
updated clinical data from the ongoing Phase 2b STORM study, a
single-arm clinical trial evaluating selinexor in combination with
low-dose dexamethasone in patients with quad-refractory or
penta-refractory myeloma. Patients with quad-refractory
disease have previously received two proteasome inhibitors (PIs)
(bortezomib (Velcade®) and carfilzomib (Kyprolis®)) and two
immunomodulatory drugs (IMiDs) (lenalidomide (Revlimid®) and
pomalidomide (Pomalyst®)), and their disease is refractory to at
least one PI, at least one IMiD, and has progressed following their
most recent therapy. Patients with penta-refractory myeloma
have quad-refractory disease that is also refractory to an
anti-CD38 monoclonal antibody, such as daratumumab (Darzalex®) or
isatuximab.
Phase 2b STORM Efficacy |
Category |
N1 |
ORR (%) |
CBR (%) |
VGPR (%) |
PR (%) |
MR (%) |
SD (%) |
PD (%) |
NE (%) |
Overall |
78 |
16 (21%) |
26 (33%) |
4 (5%) |
12 (15%) |
10 (13%) |
27 (35%) |
9 (12%) |
16 (21%) |
Quad |
48 |
10 (21%) |
14 (29%) |
2 (4%) |
8 (17%) |
4 (8%) |
21 (44%) |
4 (8%) |
9 (19%) |
Penta |
30 |
6 (20%) |
12 (40%) |
2 (7%) |
4 (13%) |
6 (20%) |
6 (20%) |
5 (17%) |
7 (23%) |
6 Doses/month |
51 |
10 (20%) |
15 (29%) |
3 (6%) |
7 (14%) |
5 (10%) |
21 (41%) |
4 (8%) |
11 (2%) |
8 Doses/month |
27 |
6 (22%) |
11 (41%) |
1 (4%) |
5 (19%) |
5 (19%) |
6 (22%) |
5 (19%) |
5 (19%) |
ORR=Objective Response Rate (VGPR+PR), CBR=Clinical Benefit Rate
(VGPR+PR+MR), VGPR=Very Good Partial Response, PR=Partial Response,
MR=Minor Response, SD=Stable Disease, PD=Progressive Disease,
NE=Non-Evaluable1One patient not included, did not have active
myeloma
All responses were adjudicated by an Independent
Review Committee (IRC). Among the 78 evaluable patients
(median seven prior treatment regimens), the overall response rate
(ORR) was 21%, and included very good partial responses (VGPR) and
partial responses (PR). Among the 48 patients in the
quad-refractory group, the ORR was 21%. For comparison, in a
similar quad-refractory patient population, the anti-CD38
monoclonal antibodies Darzalex® and isatuximab had ORRs of 21% and
20%, respectively. Among the 30 patients in the
penta-refractory group, the ORR was 20%. Clinical benefit
rate (ORR + MR) was 32% (all patients), 29% (quad-refractory), and
37% (penta-refractory). To the Company’s knowledge, no other
agents have reported response rates in patients with
penta-refractory MM. Median overall survival (OS) was 9.3
months for all patients, greater than 11 months (median not
reached) for patients with ≥MR, and 5.7 months for patients who did
not have any response (≤SD). Median duration of response
(DOR) was 5 months. Grade ≥3 cytopenias were the most common
side effects and were generally not associated with clinical
sequellae. Nausea, anorexia and fatigue were the most common
non-hematological side effects, primarily Grades 1 and 2, and were
treatable with supportive care and/or dose modification.
There were low rates of Grade ≥3 non-hematologic toxicities, with
no new safety signals identified. In particular, there was
one reported case of Grade 4 infection (1.3%), one case of Grade 2
neuropathy (1.3%) and one reported case of sepsis (1.3%).
Dr. Vogl commented, “The quad- and
penta-refractory populations are continuing to expand as patients
live longer and cycle through a variety of treatment options,
including immunomodulatory drugs, proteasome inhibitors, or
anti-CD38 monoclonal antibodies, before their disease ultimately
becomes refractory and non-responsive. In my experience,
selinexor is the first agent to be specifically investigated in
this difficult to treat and currently underserved population.
The response rate and duration suggest that selinexor has the
potential to be an exciting new option for myeloma treatment.”
Karyopharm to Host Multiple
Myeloma-focused Dinner Reception and Webcast at ASH
2016
On Monday, December 5, 2016, Karyopharm will
host an investor and analyst dinner reception, which will feature a
moderated panel discussion with recognized experts in the treatment
of MM, updated selinexor data in MM, and a live Q&A
session. Confirmed external speakers include:
- Daniel Auclair, PhD (Moderator), Multiple Myeloma Research
Foundation
- Nizar Bahlis, MD, University of Calgary, Southern Alberta
Cancer Research Institute
- Paul G. Richardson, MD, Dana Faber Cancer Institute,
Jerome Lipper Multiple Myeloma Center
- Ravi Vij, MD, MBA, Washington University School of Medicine in
St. Louis, Oncology Division
- Dan T. Vogl, MD, Abramson Cancer Center Clinical Research
Unit, University of Pennsylvania
In addition, Michael Kauffman, MD, PhD, CEO of
Karyopharm Therapeutics will be joining.
The event will take place during the ASH 2016
annual meeting and interested parties can access a live webcast of
the event beginning Monday, December 5, 2016 at 8:15 p.m. PT under
“Events & Presentations” in the "Investors" section of the
company's website at http://investors.karyopharm.com/events.cfm.
A replay of the webcast will be archived on the company’s
website for 90 days following the event.
About Selinexor
Selinexor (KPT-330) is a first-in-class, oral
Selective Inhibitor of Nuclear Export / SINE™ compound. Selinexor
functions by binding with and inhibiting the nuclear export protein
XPO1 (also called CRM1), leading to the accumulation of tumor
suppressor proteins in the cell nucleus. This reinitiates and
amplifies their tumor suppressor function and is believed to lead
to the selective induction of apoptosis in cancer cells, while
largely sparing normal cells. To date, over 1,800 patients have
been treated with selinexor and it is currently being evaluated in
several mid- and later-phase clinical trials across multiple cancer
indications, including in multiple myeloma in combination with
low-dose dexamethasone (STORM) and backbone therapies (STOMP), and
in acute myeloid leukemia (SOPRA), diffuse large B-cell lymphoma
(SADAL), and liposarcoma (SEAL), among others. Karyopharm
plans to initiate a pivotal randomized Phase 3 study of selinexor
in combination with bortezomib (Velcade®) and low-dose
dexamethasone (BOSTON) in patients with multiple myeloma in early
2017. Additional Phase 1, Phase 2 and Phase 3 studies are
ongoing or currently planned, including multiple studies in
combination with one or more approved therapies in a variety of
tumor types to further inform the Company's clinical development
priorities for selinexor. The latest clinical trial information for
selinexor is available at www.clinicaltrials.gov.
About Karyopharm
Therapeutics
Karyopharm Therapeutics Inc. (Nasdaq:KPTI) is a
clinical-stage pharmaceutical company focused on the discovery and
development of novel first-in-class drugs directed against nuclear
transport and related targets for the treatment of cancer and other
major diseases. Karyopharm's SINE™ compounds function by binding
with and inhibiting the nuclear export protein XPO1 (or
CRM1). In addition to single-agent and combination activity
against a variety of human cancers, SINE™ compounds have also shown
biological activity in models of neurodegeneration, inflammation,
autoimmune disease, certain viruses and wound-healing.
Karyopharm, which was founded by Dr. Sharon Shacham, currently has
several investigational programs in clinical or preclinical
development. For more information, please visit
www.karyopharm.com.
Forward-Looking Statements
This press release contains forward-looking
statements within the meaning 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 for Karyopharm's drug candidates, including the
timing of initiation of certain trials and of the reporting of data
from such trials. 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 any of
Karyopharm's SINE™ compounds, including selinexor (KPT-330), 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. Management's expectations
and, therefore, any forward-looking statements in this press
release could also be affected by risks and uncertainties relating
to a number of other factors, 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, including with respect to the need
for additional clinical studies; 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 in 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 are
described under the caption "Risk Factors" in Karyopharm's
Quarterly Report on Form 10-Q for the quarter ended September 30,
2016, which was filed with the Securities and Exchange Commission
(SEC) on November 7, 2016, and in other filings that Karyopharm may
make with the SEC in the future. Any forward-looking statements
contained in this press release speak only as of the date hereof,
and Karyopharm expressly disclaims any obligation to update any
forward-looking statements, whether as a result of new information,
future events or otherwise.
Velcade® is a registered trademark of Takeda
Pharmaceutical Company LimitedRevlimid® and Pomalyst® are
registered trademarks of Celgene CorporationKyprolis® is a
registered trademark of Onyx Pharmaceuticals, Inc.Darzalex® is a
registered trademark of Janssen Biotech, Inc.
Contacts:
Justin Renz
(617) 658-0574
jrenz@karyopharm.com
Gina Nugent
(617) 460-3579
nugentcomm@aol.com
Media covering ASH 2016:
Eliza Schleifstein
(917) 763-8106
eliza@argotpartners.com
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