In this study, we hypothesize that SB-216 and Veru-111 (related novel compounds) inhibit cell growth via suppression of oncogenic βIII- and βIVb-tubulin subtypes and mitochondria function via suppression of BRD4, the most active BET protein...Our data demonstrates that SB-216 effectively inhibits PDAC cell growth through inhibiting oncogenic microtubules and mitochondrial function. This novel approach simultaneously targets two hallmarks of cancer and patient demise.

Vincristine, a cornerstone vinca alkaloid in pediatric oncology, has historically been regarded as a low-risk agent for drug-induced acute pancreatitis (DIAP). Vindesine may serve as a safe and effective alternative agent. These findings underscore the importance of updating pediatric chemotherapy safety guidelines for vinca alkaloid-containing regimens.

Perturbagen analysis nominated signature-reversing compounds across sexes, including histone deacetylase inhibitors, Aurora kinase inhibitors, microtubule-targeting agents such as vindesine, and multi-kinase inhibitors targeting VEGFR, PDGFR, FLT3, PI3K, and MTOR. Glioma grade comparisons reveal a shared neuronal-synaptic program accompanied by sex-specific transcriptional remodeling. These findings support sex-aware therapeutic strategies that pair modulation of neuron-glioma coupling with chromatin- or receptor tyrosine kinase/angiogenic-targeted agents, and they nominate biomarkers such as GLI1, MYOD1, GCGR, PRLHR, and HIST1H2BH for near-term validation.

P=N/A, N=3, Not yet recruiting, Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine; Shanghai Children's Medical Center, Shanghai Jiao Tong Universit

P3, N=105, Terminated, Veru Inc. | N=245 --> 105 | Trial completion date: Sep 2024 --> May 2023 | Active, not recruiting --> Terminated | Trial primary completion date: May 2024 --> May 2023; Administrative reasons

P1b/2, N=80, Terminated, Veru Inc. | N=58 --> 80 | Completed --> Terminated; Administrative decision

P1b/2, N=58, Completed, Veru Inc. | Active, not recruiting --> Completed | N=41 --> 58

P2, N=0, Withdrawn, Veru Inc. | N=200 --> 0 | Not yet recruiting --> Withdrawn

This study developed a robust IGS model for survival prediction of HCC patients, providing new insights into integrating tailored risk stratification with precise immunotherapy and screening potentially targeted agents.

P3, N=245, Active, not recruiting, Veru Inc. | Recruiting --> Active, not recruiting

Preferred frontline treatment relies on anti-HER2 therapies, such as trastuzumab and pertuzumab, combined with microtubule inhibitors, such as taxanes. While sabizabulin shows promising results for treatment in HER2+ breast cancer, we further optimized the structure of sabizabulin to create a new class of anti-cancer molecules: 40a, CH-2-77, and 60c. Based on in vitro data, 60c showed higher potency in HER2+ breast cancer cell lines and was chosen as the lead compound to further study in a paclitaxel-resistant triple negative breast cancer in vivo study where it was able to effectively inhibit primary tumor growth compared to the vehicle group.

This clinical trial demonstrated that chronic oral daily dosing of sabizabulin has a favorable safety profile with significant preliminary cytotoxic and cytostatic antitumor activity. These data support the ongoing Phase 3 VERACITY trial of sabizabulin in men with mCRPC who have progressed on an androgen receptor targeting agent.