AUSTIN, Texas - ColoradoDesk -- Curtana Pharmaceuticals, a privately-held, preclinical stage biopharmaceutical company, today announced that an abstract published in Neuro-Oncology earlier this month shows that CT-179, a first-in-class OLIG2 inhibitor, significantly prolonged event-free survival in an animal model of medulloblastoma as a single agent and showed even better efficacy when combined with palbociclib (Ibrance®), a CDK4/6 inhibitor marketed by Pfizer. Experiments also demonstrated the potential for CT‑179 to improve the efficacy of therapies limited by resistance to chemotherapy that is driven by OLIG2-positive cancer stem cells, suggesting that CT-179 may be a versatile agent to enhance both targeted and cytotoxic treatments. The research results were presented last week during an oral presentation at the virtual Society of Neuro-Oncology (SNO) 6^th Biennial Pediatric Neuro-Oncology Research Conference.

The research was led by Timothy Gershon, M.D., Ph.D., who is a Professor in the Department of Neurology and at the Lineberger Comprehensive Cancer Center at the University of North Carolina, Chapel Hill. "Based on the research results from our lab and others working on treatments for medulloblastoma, I believe that CT-179 has the potential to substantially improve patient outcomes," said Dr. Gershon. "We look forward to continuing our important work with Curtana on developing CT-179 for kids and adults affected by medulloblastoma."

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Medulloblastoma (MB) is the most common type of primary brain cancer in children. According to the American Society of Clinical Oncology (ASCO), between 250 and 500 medulloblastoma cases are diagnosed in US children each year. Medulloblastoma also occurs in adults, but it is less common. Approximately one-third of medulloblastoma cases in the US occur in adults between the ages of 20 to 44. Conventional medulloblastoma treatment, with resection, chemotherapy, and radiation, leaves survivors at risk of neurocognitive injury, growth defects, and psychosocial impairment. In addition, there is no effective therapy for recurrent medulloblastoma, which occurs in 30 to 40% of patients and is typically fatal. Five-year survival rate following recurrence is only about 25%.

"We are encouraged by the data from Dr. Gershon's lab revealing the potential of CT-179 to provide a significant therapeutic benefit to patients with medulloblastoma," said Gregory Stein, M.D., M.B.A. and Chief Executive Officer, Curtana Pharmaceuticals. "Medulloblastoma is a devastating disease with an urgent need for advanced treatment options. Studies to-date have demonstrated the viability of CT-179 to markedly prolong survival in numerous, relevant animal models of medulloblastoma and other brain cancers. We eagerly await clinical trials projected to initiate in the first half of 2022."

About CT-179

CT-179 is a highly potent and selective small molecule inhibitor of OLIG2, a transcription factor that is essential to normal early brain development but is not actively expressed in the vast majority of normal adult brain cells or in normal tissues outside the brain. OLIG2-expressing progenitors are distinct tumor-initiating cells during the onset of primary and recurrent MB and these OLIG2-expressing progenitors are highly enriched in therapy-resistant and recurrent MB. It has been shown that high levels of OLIG2 are correlated with poor prognosis. In animal studies, experiments have demonstrated that elimination of OLIG2-expressing cells blocks tumor progression in a mouse model of MB. Thus, targeting OLIG2 is a promising, potential therapeutic avenue for the treatment of MB.

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FDA Orphan Drug designation for the treatment of malignant gliomas, including glioblastoma (GBM), was granted to CT-179 in August 2017. FDA Rare Pediatric Disease Designation for the treatment of medulloblastoma was granted to CT-179 in September 2020. The drug is orally bioavailable, readily crosses the blood-brain barrier, achieves very high concentrations in the brain, and significantly prolongs survival in animal models of brain cancer. CT-179 represents a novel agent which selectively targets OLIG2-expressing brain cancer cells in preclinical studies with great potential as an adjunctive therapy in the treatment of GBM, MB, and other brain cancers. An investigational new drug (IND) application filing is planned for the first half of 2022.

About Curtana Pharmaceuticals

Curtana Pharmaceuticals, founded in 2013, is a privately held, preclinical-stage biopharmaceutical company headquartered in Austin, Texas. Current investors include Thynk Capital, angelMD, Biosense Global, DEFTA Partners, and other anonymous investors. In 2014, the company was awarded a $7.6 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT). Curtana focuses on the development of novel first-in-class, small molecule therapeutics targeting cancer stem cells in the central nervous system for the treatment of glioblastoma, medulloblastoma, and other brain cancers. For more information, visit www.curtanapharma.com.

Contact Information
Gregory Stein, M.D., M.B.A.
Chief Executive Officer
gregory.stein@curtanapharma.com