NU-0129 / Northwestern University, Exicure - LARVOL DELTA

A Phase 0 first-in-human study using NU-0129:A gold base spherical nucleic acid (SNA) nanoconjugate targeting BCL2L12 in recurrent glioblastoma patients. (ASCO 2019) - P1; "Macrodosing of the nanotherapeutic NU-0129 was well tolerated in glioblastoma patients with no unexpected adverse effects and showed initial evidence of crossing blood brain barrier. Immunohistochemistry for Bcl2L12 expression, apoptotic markers, and PK studies are pending. The demonstration of gold nanoparticles in the tumor tissue validates this approach for drug delivery."

Clinical • P1 data

A Phase 0 first-in-human study using NU-0129:A gold base spherical nucleic acid (SNA) nanoconjugate targeting BCL2L12 in recurrent glioblastoma patients. (ASCO 2019) - P1; "Macrodosing of the nanotherapeutic NU-0129 was well tolerated in glioblastoma patients with no unexpected adverse effects and showed initial evidence of crossing blood brain barrier. Immunohistochemistry for Bcl2L12 expression, apoptotic markers, and PK studies are pending. The demonstration of gold nanoparticles in the tumor tissue validates this approach for drug delivery."

Clinical • P1 data

A first-in-human phase 0 clinical study of RNA interference-based spherical nucleic acids in patients with recurrent glioblastoma. (PubMed, Sci Transl Med) - P1 | "NU-0129 uptake into glioma cells correlated with a reduction in tumor-associated Bcl2L12 protein expression, as indicated by comparison of matched primary tumor and NU-0129-treated recurrent tumor. Our results establish SNA nanoconjugates as a potential brain-penetrant precision medicine approach for the systemic treatment of GBM."

Clinical • Journal • P1 data • Glioblastoma • Glioma • Hematological Malignancies • Immunology • Oncology • Solid Tumor

NU-0129 in Treating Patients With Recurrent Glioblastoma or Gliosarcoma Undergoing Surgery (clinicaltrials.gov) - P1; N=8; Completed; Sponsor: Northwestern University; Active, not recruiting ➔ Completed

Clinical • Trial completion • Brain Cancer • Glioblastoma • Gliosarcoma • Oncology • Sarcoma • Solid Tumor

NU-0129 in Treating Patients With Recurrent Glioblastoma or Gliosarcoma Undergoing Surgery (clinicaltrials.gov) - P1; N=8; Active, not recruiting; Sponsor: Northwestern University; Trial completion date: Jul 2022 ➔ Sep 2020

Clinical • Trial completion date • Brain Cancer • Glioblastoma • Gliosarcoma • Oncology • Psychiatry • Sarcoma • Solid Tumor