- Data Demonstrate Robust Response Rates Enabling
Transplantation or Donor Lymphocyte Infusions in Patients with
Heavily Pretreated Acute Myeloid Leukemia -
Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage
pharmaceutical company, today announced updated results from the
Phase 2 SAIL study, in which deep responses to selinexor (KPT-330)
allowed patients with heavily pretreated acute myeloid leukemia
(AML) to proceed onto stem cell transplantation or donor lymphocyte
transfusion, 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 AML and a variety of additional malignancies.
“These updated SAIL data, along with key
presentations by Drs. Amy Wang, Bhavana Bhatnagar, and Kendra Sweet
demonstrate the feasibility and tolerability of selinexor in
combination with chemotherapy and other commonly-used agents in
patients with AML,” said Sharon Shacham, PhD, MBA, President and
Chief Scientific Officer of Karyopharm. “With response rates
that are superior to historical data, the clinical results
presented at ASH this year demonstrate that selinexor combination
regimens could become effective treatment options and serve as a
bridge to stem cell transplantation even for patients suffering
with relapsed/refractory AML.”
Updated Phase 2 SAIL Clinical Data in
Refractory AML
In an oral presentation titled, “Phase II
Results of Ara-C and Idarubicin in Combination with the Selective
Inhibitor of Nuclear Export Compound Selinexor in Patients with
Relapsed or Refractory AML,” Walter Fiedler, MD, University Medical
Center Hamburg, described data demonstrating that selinexor in
combination with Ara-C and idarubicin is safe with no unexpected
toxicities observed to date and has the potential to achieve
significant response rates, particularly in a heavily pretreated
patient population.
Among the 42 patients evaluable for safety
(median of 2 prior treatment regimens, all including intensive
chemotherapy), as of October 2016, the overall response rate (ORR,
4 patients excluded from evaluation due to early death) was
55% and included 10 (26%) complete remissions (CR) and 10 (26%)
achieving complete remission with incomplete blood count recovery
(CRi). Based on these data, Karyopharm believes that
selinexor in combination with Ara-C and idarubicin may be an
effective treatment option and serve as a bridge to stem cell
transplantation for patients with relapsed/refractory AML.
The most frequent Grade ≥3 non-hematologic adverse events (AEs) of
this intensive chemotherapy-containing regimen were diarrhea (50%)
and nausea (12%). The most common Grade ≥3 hematologic AEs
were neutropenia (100%) and thrombocytopenia (100%). Two
deaths occurred which were deemed possibly treatment-related.
There was one reported case of systemic inflammatory response
syndrome (SIRS; 2%) and one reported case of hemophagocytosis
syndrome (2%).
Other Key AML Data Presented at ASH
2016
In addition to updated data from the SAIL study,
additional key AML abstracts include:
Oral Presentation Title:
Combination of Selinexor with High-Dose Cytarabine and Mitoxantrone
for Remission Induction in AML Is Feasible and
TolerablePresenter: Amy Wang, University of
ChicagoPublication ID: 212Date and
Time: Saturday, December 3, 2016; 4:15 PM
PTSummary: In this study, the combination of
selinexor with high-dose cytarabine (HiDAC) and mitoxantrone is
feasible and tolerable and the recommended phase 2 dose was
identified as selinexor 80mg per day plus HiDAC and mitoxantrone.
This regimen demonstrated an ORR of 68% in all 19 patients
and 91% in patients with newly diagnosed AML. Based on these
results, the combination of selinexor plus HiDAC and mitoxantrone
warrant further investigation.
Poster Title: A Phase 1
Clinical Trial of Selinexor in Combination with Decitabine in
Patients with Newly Diagnosed and Relapsed or Refractory
AMLPresenter: Bhavana Bhatnagar, Ohio State
UniversityPublication ID: 1651Date
and Time: Saturday, December 3, 2016; 5:30-7:30 PM
PTSummary: In this study, the combination of
selinexor and decitabine produced a CR/CRi/mCR (marrow CR) rate of
80% in older untreated patients and 26.3% in relapsed/refractory
AML (RRAML) patients. The total CR/CRi/mCR rate was
37.5%. Importantly, six of the 19 patients with
relapsed/refractory disease underwent allogeneic stem cell
transplant (four with no evidence of AML at the time of
transplant). Selinexor plus decitabine is an active regimen in
poor-risk AML patients and alternative dosing schedules to improve
long-term tolerability, compliance and efficacy should be
explored.
Poster Title: A Phase I
Study of Selinexor in Combination with Daunorubicin and Cytarabine
in Patients with Newly Diagnosed Poor-Risk
AMLPresenter: Kendra Sweet, Moffitt Cancer
Center, Tampa FLPublication ID:
4040Date and Time: Monday, December 5, 2016;
6:00-8:00 PM PTSummary: Data from this Phase
1 trial suggest that oral selinexor (80mg) twice weekly can be
safely administered in combination with induction chemotherapy with
cytarabine and daunorubicin in patients with poor-risk AML,
including elderly patients. Response rates were encouraging,
with many elderly patients proceeding to transplant, suggesting
this regimen warrants further investigation in this challenging
population.
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 Limited
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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