
	Karyopharm Therapeutics is a clinical-stage pharmaceutical company focused on discovery and development of novel first-in-class drugs directed against nuclear transport targets for the treatment of cancer and other major diseases.
	
	Our scientific expertise is focused on the understanding of the regulation of intracellular transport between the nucleus and the cytoplasm.&nbsp; We have discovered and developed novel, small molecule Selective Inhibitors of Nuclear Export, or SINE, compounds that inhibit the nuclear export protein XPO1.
	
	Our lead drug candidate, Selinexor (KPT-330), is an XPO1 inhibitor being evaluated in multiple open-label Phase 1 clinical trials in patients with heavily pretreated relapsed and/or refractory hematological and solid tumor malignancies.
	
	XPO1 mediates the export of approximately 285 different cargo proteins, including the vast majority of tumor suppressor proteins.&nbsp; We believe that no currently approved or current clinical-stage experimental cancer drug candidates are selectively targeting the restoration and increase in the levels of multiple tumor suppressor proteins in the nucleus.
	
	Our other drug candidates include KPT-350 and related SINE compounds that have additional important anti-inflammatory activities such as activitation of the proteins RXRg,&nbsp; PPARa and NRF2 (an anti-oxidant and neuroprotective protein).&nbsp; Our SINE compounds have shown broad evidence of anti-inflammatory activity across preclinical models of diverse autoimmune diseases.&nbsp; These observations suggest that SINE compounds have multiple anti-inflammatory effects. We are evaluating several SINE compounds, including KPT-350, in additional inflammatory models and preclinical safety studies.
	
	We believe that our XPO1-inhibiting SINE compounds that we have discovered and developed to date, including Selinexor, have the potential to provide a novel targeted therapy that enable tumor suppressor proteins to remain in the nucleus and promote apoptosis of cancer cells and we believe that our SINE compounds have the potential to provide therapeutic benefit in a number of additional indications, including autoimmune and inflammatory diseases, wound healing, HIV and influenza.
	
	In addition, Karyopharm has a growing pipeline addressing novel oncology targets which compement our XPO1 SINE program.
	
	In addition to our SINE compounds, we also investigate XPO1 cargo proteins and their role in cell cycle and division.&nbsp; As part of this investigation, we have identified several XPO1 cargo proteins whose inhibition leads to the selective death of cancer cells. One of the XPO1 cargo proteins that we identified was P21-activated kinase 4, or PAK4.&nbsp; PAK4 is a signaling protein regulating numerous fundamental cellular processes, including intracellular transport, cellular division, cell shape and motility, cell survival, immune defense and the development of cancer. PAK4 interacts with many key signaling molecules involved in cancer such as beta-catenin, CDC42, Raf-1, BAD and myosin light change.&nbsp; Based on this biology, we used our drug discovery and optimization platform to identify small molecule inhibitors of PAK4.&nbsp; Our PAK4 inhibitors have shown broad evidence of anti-cancer activity against hematological and solid tumor malignancies cells while showing minimal toxicity to normal cells in vitro.
	
	We have used spontaneously occurring dog cancers as a surrogate model for human malignancies.&nbsp; It is widely known that canine lymphomas respond to chemotherapy similarly to their human counterpart (human NHL) and display a comparable genetic profile. Lymphomas are one of the most common tumors in pet dogs. Lymphoma in dogs is very aggressive and, without treatment, the tumors are often fatal within weeks.&nbsp; The majority of dog lymphomas are DLBCL and most of the others are T-cell lymphomas. Given the similarities of dog and human lymphomas, prior to initiating clinical trials of Selinexor in humans, we investigated a closely-related, orally available SINE, Verdinexor (KPT-335), in dogs with lymphomas. We have received a Minor Use / Minor Species, or MUMS, designation from the Center for Veterinary Medicine, or CVM, of the FDA for the treatment of newly diagnosed or first relapse after chemotherapy lymphomas in dogs with Verdinexor.
	
	Karyopharm Therapeutics Inc. is a clinical-stage pharamceutical pharmaceutical company focused on discovery and development of novel first-in-class drugs directed against nuclear transport targets for the treatment of cancer and other major diseases.
	
	Our goal is to foster scientific creativity with pioneering technologies and developmental approaches within an innovative culture that will accelerate first-in-class small molecule modulators of nuclear transport into effective chronic therapeutics for life threatening conditions.
	
	We foster courage, urgency, resilience, energy, creativity and innovation in our employees and collaborators, recognizing the contributions of each to the success of the team. Integrity and respect are core to our success. We strive to benefit patients, physicians, employees, and shareholders by creating innovative medicines that improve the quality and duration of patients&rsquo; lives.
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