DongKoo Bio&Pharma announced on Monday that its largest shareholder, Qurient, has received U.S. Food and Drug Administration (FDA) approval for an investigator-initiated clinical trial of Mocaciclib (Q901), a CDK7 inhibitor cancer drug targeting pediatric brain tumors.
The trial, set to commence in 2024, builds on collaborative research with the University of Colorado Anschutz Medical Campus and Harvard’s Dana-Farber Cancer Institute. It will focus on children with recurrent medulloblastoma and atypical teratoid rhabdoid tumors (AT/RT).
Mocaciclib is a promising drug candidate that inhibits CDK7, a protein crucial for cancer cell proliferation. Researchers believe it could potentially block MYC, a major driver of pediatric brain tumors.
CDK7 is an enzyme vital for transcription and cell cycle regulation. By inhibiting it, the drug aims to disrupt the gene expression necessary for cancer cell growth and survival.
Preclinical studies have shown encouraging results. Mocaciclib significantly extended survival in pediatric brain tumor models, effectively reaching the brain and central nervous system to inhibit tumor growth. Importantly, no major side effects like weight loss or blood abnormalities were observed.
The research team presented these findings at the International Society of Pediatric Neuro-Oncology (ISPNO 2026) symposium in June. A study published in October in ‘Science Advances’ further demonstrated Mocaciclib’s effectiveness in blocking MYC-driven gene transcription.
MYC, a known cancer-promoting protein, has long been a challenging target for direct drug intervention. The researchers’ innovative approach focuses on inhibiting CDK7 as an indirect way to combat MYC’s effects.
Medulloblastoma and AT/RT, the targets of this clinical trial, are aggressive brain tumors that primarily affect children. The prognosis for these cancers is often grim. AT/RT has a five-year survival rate of about 35%, dropping to a mere 15% for patients under three. Similarly, high-risk medulloblastoma associated with MYC has a five-year survival rate of only around 30%.
Nam Ki-yeon, Chief Executive Officer (CEO) of Qurient, expressed optimism about the FDA approval. This milestone reflects the tireless efforts of doctors who’ve witnessed firsthand the urgent need for new treatments, he said. It’s hopeful that the promising results from the preclinical studies will translate to clinical success. The ultimate goal is for Mocaciclib to offer new hope to children and families worldwide who are currently facing these devastating diagnoses without effective treatment options.
