Navigation Links
Karyopharm Therapeutics Initiates Two Phase 1 Trials with Oral KPT-330, the First Selective Inhibitor of Nuclear Export (SINE), in Patients with Advanced Cancers

NATICK, Mass., July 25, 2012 /PRNewswire/ -- Karyopharm Therapeutics Inc., a leader in the new field of nuclear transport modulators, announced dosing of patients in the first-in-human clinical trials with KPT-330. KPT-330 is the first oral SINE CRM1 antagonist to enter human studies.  SINEs specifically and irreversibly inhibit the nuclear export protein CRM1 (chromosome region maintenance protein 1), also called exportin 1 (XPO1).  CRM1 is the exclusive mediator of nuclear export of p53, p73, pRb, FOXO, p21, p27, BRACA1, the endogenous inhibitor of Nuclear Factor kB (NF-kB) known as IkB, and other tumor suppressor and growth regulatory proteins. Nuclear export of these key proteins leads to their functional inactivation.  Blockade of CRM1 leads to accumulation and activation of tumor suppressor and growth regulatory proteins in the nucleus, leading to potent and selective tumor cell apoptosis while sparing normal cells. 

(Logo: )

Patient dosing has been initiated in two phase 1 studies.  The primary endpoints of both studies are to determine the safety and tolerability profile and the maximum tolerated dose of KPT-330 given orally 2-3 times per week. The first study includes patients with advanced solid tumors whose disease has progressed after at least one prior therapy for metastatic disease (NCT01607905).  The second study includes patients with advanced hematologic malignancies including chronic lymphocytic leukemia, non-Hodgkin's lymphoma, multiple myeloma, and Waldenstrom's macroglobulinemia whose disease has relapsed after standard therapies (NCT01607892). Sharon Shacham, PhD, MBA, Karyopharm's Founder, Chief Scientific Officer, and head of research and development commented, "The initiation of these two Phase 1 trials with KPT-330 is a key milestone since the closing of our Series A funding and the initiation of our Company's operations in late 2010.  This is the first oral SINE to ever enter human studies, and we are eager to assess the potential of our drug candidate across a wide variety of cancers."

The study is being conducted in the United States, Toronto, Canada, and Copenhagen, Denmark. NPM Pharma (Canada) is overseeing the trial on behalf of Karyopharm.

About Karyopharm

Karyopharm is a biopharmaceutical company leading the development of small molecule modulators of nuclear transport.  The Company was founded by Drs. Sharon Shacham and Michael Kauffman in 2008.  Karyopharm has raised approximately $34M since its inception and has won several grants/awards including a Biotech Investment Award by the Multiple Myeloma Research Foundation in 2010. Karyopharm's first program is directed towards the Selective Inhibition of Nuclear Export – its SINE program – targeting CRM1, the major nuclear export protein. SINE compounds, including KPT-330 and KPT-335, force the activation of the cell's key tumor suppressor proteins and anti-inflammatory pathways including p53, p21, pRB, FoxO, and IkB, a key cellular inhibitor of nuclear factor NF-kB. Karyopharm is currently evaluating two oral SINE compounds in clinical trials. KPT-330 oral is being evaluated in patients with cancer, and KPT-335 oral is being studied in dogs with relapsed/refractory non-Hodgkin's Lymphomas.  The Company is also evaluating the use of SINEs in autoimmune/inflammatory disorders, viral infections and dermatologic diseases. Karyopharm Therapeutics is located in Natick, Massachusetts.

contact: Michael Kauffman, 508-975-4821,   


SOURCE Karyopharm Therapeutics Inc.
Copyright©2012 PR Newswire.
All rights reserved

Related medicine technology :

1. Actinium Pharmaceuticals Inc. Pays Regulatory Milestone to Abbott Biotherapeutics Corp.
2. PTC Therapeutics Closes $30 Million Financing
3. Protein and Peptide-based Therapeutics - Trends in Drug Discovery and Development
4. Amsterdam Molecular Therapeutics (AMT) Holding N.V. (in liquidation) Announces Publication of its Accounts for 2011 and Notice of Annual General Meeting
5. Echo Therapeutics Expands License Agreement with Ferndale Pharma
6. PTC Therapeutics Announces Achievement of Major Milestone in Wellcome Trust BMI1 Collaboration
7. Orexigen Therapeutics Announces the Light Study is Enrolling Faster than Originally Projected
8. NIPTE Will Participate in NIHs Therapeutics for Rare and Neglected Diseases (TRND) Program
9. Orexigen Therapeutics to Present at the JMP Securities Healthcare Conference
10. Frost & Sullivan: European Bipolar Therapeutics Market Slows Down in the Short Term As Key Blockbuster Drugs Go Off Patent
11. Oncos Therapeutics Announces Clinical Advisory Board for its Oncolytic Virus and Cancer Immunotherapy Development Programs
Post Your Comments:
(Date:11/26/2015)... November 27, 2015 ... --> Medical alert ... system (PERS) market is expected ... years with APAC being the ... see a high CAGR between ...
(Date:11/26/2015)... Carolina , 26 november 2015 /PRNewswire/ ... (AAIPharma/CML) kondigt de geplande investering aan van ... van de laboratoria en het mondiale hoofdkantoor ... De uitbreiding zal resulteren in extra kantoorruimte ... voldaan aan de groeiende behoeften van de ...
(Date:11/26/2015)... November 26, 2015 ... nieuwe aanpak combineert immunotherapie met Bremachlorin-photodynamische therapie ... ) --> ... (Photo: ) ... Medisch Centrum (LUMC) blijkt ...
Breaking Medicine Technology:
(Date:11/27/2015)... ... November 27, 2015 , ... Consistent with ... the 2016 Building Better Radiology Marketing Programs meeting will showcase some ... March 6, 2016, at Caesars Palace in Las Vegas with a pre-conference session ...
(Date:11/27/2015)... ... November 27, 2015 , ... A simply groundbreaking television ... an interesting show that delves into an array of issues that are presently affecting ... benefit from open dialogue, this show is changing the subjects consumers focus on, one ...
(Date:11/27/2015)... Bangalore, Karnataka (PRWEB) , ... November 27, 2015 ... ... the world, Health-E-minds, an innovative online platform for mental health and wellness consultation, ... KleverKid. , This partnership will bridge the knowledge gap experienced by parents and ...
(Date:11/27/2015)... ... November 27, 2015 , ... The men and ... nonprofit healthcare organizations in the country. They have overseen financial turnarounds, shown commitment ... advance the healthcare industry as a whole through their advocacy and professional efforts. ...
(Date:11/27/2015)... San Francisco, California (PRWEB) , ... November 27, 2015 , ... ... 1969 Janis Joplin Ann Arbor Michigan boxing style concert posters. This is one of ... 1969 at the Canterbury House at the University of Michigan in Ann Arbor. The ...
Breaking Medicine News(10 mins):