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Karyopharm Presents Data Demonstrating the Potential of Nuclear Export Protein Exportin 1 (XPO1) Inhibition in the Treatment of Traumatic Brain Injury

NEWTON, Mass., Apr 20, 2016 (GLOBE NEWSWIRE via COMTEX) --

Karyopharm Therapeutics Inc. KPTI, +0.00% a clinical-stage pharmaceutical company, today announced preclinical data demonstrating the activity of its Selective Inhibitor of Nuclear Export (SINE [TM] ) compound, KPT-350, in rodent models of traumatic brain injury (TBI). These data were presented at the 2016 Annual Meeting of the American Academy of Neurology (AAN) taking place April 15-21 in Vancouver, Canada.

"In addition to its clinically-validated activity in a broad range of hematologic and solid tumor cancers, nuclear export protein Exportin 1, or XPO1, has also emerged as an attractive target for the treatment of neuro-inflammatory disorders such as traumatic brain injury," said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm. "XPO1 is known to be upregulated in neuropathology and may mediate some of the inflammatory and neurodegenerative processes observed after TBI. By inhibiting XPO1, SINE compounds such as KPT-350 have demonstrated tolerability, brain penetrance and therapeutic activity, including neuroprotective, anti-inflammatory and anti-epileptic effects in preclinical studies. These findings warrant further evaluation of KPT-350 in neurological indications such as traumatic brain injury."

In an oral presentation titled "Functional and Imaging Studies of the Selective Inhibitor of Nuclear Export (SINE) Compound KPT-350 in a Clinically Relevant Murine Model of Traumatic Brain Injury (TBI)," Karyopharm researchers and collaborators presented data evaluating the efficacy of KPT-350 as a treatment for TBI in a murine model of closed head injury (CHI) and determined that orally administered KPT-350 improved functional outcomes, possibly by reducing cytotoxic intracellular edema.

Highlights of the data include:

  • KPT-350 demonstrated improved long-term measures of physical and cognitive function relative to the vehicle or delayed treatment group.
    • Mice that received KPT-350 two hours post-CHI recovered body weight more quickly and performed better in the Rotarod test of vestibulomotor function
    • Diffusion tensor imaging studies demonstrated changes in the mean diffusivity index in several brain regions, suggesting that KPT-350 reduced cellular swelling versus vehicle

In an oral and poster presentation titled "Using the Selective...