BGA002 / Biogenera - LARVOL DELTA

Preclinical Pharmacokinetics in Tumors and Normal Tissues of the Antigene PNA Oligonucleotide MYCN-Inhibitor BGA002. (PubMed, Nucleic Acid Ther) - "Remarkably, we demonstrated that BGA002 concentrates in tumors after repeated systemic administrations in three mouse models with MYCN amplification (neuroblastoma, rhabdomyosarcoma, and small-cell lung cancer), leading to a significant reduction in tumor weight. Taking into account the available safety profile of BGA002, these data support further evaluation of BGA002 in patients with MYCN-positive tumors."

Journal • PK/PD data • Preclinical • CNS Tumor • Lung Cancer • Neuroblastoma • Oncology • Pediatrics • Rhabdomyosarcoma • Sarcoma • Small Cell Lung Cancer • Solid Tumor • MYCN

Antigene MYCN Silencing by BGA002 Inhibits SCLC Progression Blocking mTOR Pathway and Overcomes Multidrug Resistance. (PubMed, Cancers (Basel)) - "Finally, systemic treatment with BGA002 significantly increased survival in MYCN-amplified SCLC mouse models, including in a multidrug-resistant model in which tumor vascularization was also eliminated. These findings warrant the clinical testing of BGA002 in MYCN-related SCLC."

IO biomarker • Journal • Lung Cancer • Neuroendocrine Tumor • Oncology • Small Cell Lung Cancer • Solid Tumor • MYCN • TNFAIP3

The MYCN inhibitor BGA002 restores the retinoic acid response leading to differentiation or apoptosis by the mTOR block in MYCN-amplified neuroblastoma. (PubMed, J Exp Clin Cancer Res) - "Taken together, MYCN modulation mediates the therapeutic efficacy of RA and the development of RA resistance in MNA-NB. Furthermore, by targeting MYCN, a cancer-specific mTOR pathway inhibition occurs only in MNA-NB, thus avoiding the side effects of targeting mTOR in normal cells. These findings warrant clinical testing of BGA002-RA as a strategy for overcoming RA resistance in MNA-NB."

Journal • Neuroblastoma • Oncology • Solid Tumor • MYCN

MYCN Drives a Tumor Immunosuppressive Environment Which Impacts Survival in Neuroblastoma. (PubMed, Front Oncol) - "Finally, we showed that targeted inhibition of MYCN by BGA002 (anti-MYCN antigene PNA) is able to restore NK sensibility in MYCN-expressing NB cells. Overall, our study unveils a MYCN-driven immune network in NB and shows a therapeutic option to restore sensibility to immune cells."

Journal • Neuroblastoma • Oncology • Solid Tumor • MYCN