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Ivy Brain Tumor Center And Karyopharm Therapeutics Collaborate To Develop Tissue-Based Clinical Trial For Brain Cancer

  • July 11, 2019

Preclinical Study Targeting Xp01 In Glioblastoma Paves The Way For New Drug Cocktail Study

(PHOENIX, AZ. – July 11, 2019) – The Ivy Brain Tumor Center at the Barrow Neurological Institute has announced a partnership with Karyopharm Therapeutics (Nasdaq:KPTI) to accelerate the treatment of brain cancer. The Ivy Brain Tumor Center will employ Karyopharm’s first-in-class oral Selective Inhibitor of Nuclear Export (SINE) compound, selinexor, in combination with other new-in-class drugs, as a dual-drug experimental therapy for adult glioblastoma.  

This academic-private partnership will begin with a preclinical study targeting the protein XP01 in glioblastoma patients and testing several new drug cocktails involving selinexor.  Based on detailed pharmacodynamic and pharmacokinetic analyses, the lead drug cocktail will then be pursued as a combined-drug trial within the Ivy Brain Tumor Center’s Phase 0 clinical trial portfolio.  This unique trial format will enable researchers to understand if the experimental therapy is impacting an individual patient’s brain tumor in as little as seven days, therefore saving valuable patient time.

Drug development for glioblastoma has remained stagnant for decades, with the latest FDA-approved therapy associated with a survival benefit occurring in 1999.  The Ivy Brain Tumor Center, a nonprofit clinical trials program funded by the largest grant in the history of brain tumor research, is challenging the status quo of conventional drug development by streamlining early-phase identification of promising agents through tissue-based Phase 0 trialing.

“Selinexor exploits a compelling target in glioblastoma patients, which is why we have selected it as the backbone of a new experimental drug cocktail,” said Dr. Nader Sanai, director of the Ivy Brain Tumor Center. “Our scientific partnership with Karyopharm Therapeutics will arrive at a new therapeutic strategy that can be accelerated into the clinic and patients in need.”

Developed by Karyopharm Therapeutics, selinexor is the first in a new family of investigational therapies known as Selective Inhibitor of Nuclear Export (SINE) compounds and is currently being evaluated in Phase 2 and 3 clinical trials for multiple myeloma, diffuse large B-cell lymphoma (DLBCL), and a variety of solid tumors. It functions by blocking the nuclear export protein XPO1, forcing accumulation and activation of tumor suppressor proteins.

“We are highly motivated to study novel drug combination strategies with selinexor to potentially advance the available treatment options for patients with glioblastoma, one of the most aggressive forms of brain cancer,” said Sharon Shacham, PhD, MBA, president and chief scientific officer of Karyopharm. “We share the Ivy Brain Tumor Center’s sense of urgency and commitment to pursuing future breakthroughs in glioblastoma treatment and look forward to our partnership in this important and innovative research endeavor.”

Ivy Brain Tumor Center at the Barrow Neurological Institute in Phoenix, AZ is a non-profit translational research program that employs a bold, early-phase clinical trials strategy to identify new treatments for aggressive brain tumors, including glioblastoma. The Ivy Center’s Phase 0 clinical trials program is the largest of its kind in the world and enables personalized care in a fraction of the time and cost associated with traditional drug development. Unlike conventional clinical trials focusing on single drugs, its accelerated trials program tests therapeutic combinations matched to individual patients. Learn more at https://www.ivybraintumorcenter.org. Follow the Ivy Brain Tumor Center on FacebookInstagramTwitter and LinkedIn.

Karyopharm Therapeutics Inc. is an oncology-focused pharmaceutical company dedicated to the discovery, development, and commercialization of novel first-in-class drugs directed against nuclear export 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). Karyopharm’s lead compound, XPOVIOTM(selinexor), received accelerated approval from the FDA in July 2019 in combination with dexamethasone as a treatment for patients with heavily pretreated multiple myeloma. A Marketing Authorization Application for selinexor is also currently under review by the European Medicines Agency (EMA). 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 has several investigational programs in clinical or preclinical development. For more information, please visit www.karyopharm.com.