IMNN-001

P1/2, N=50, Recruiting, Imunon | Phase classification: P2 --> P1/2

P1/2, N=130, Active, not recruiting, Imunon | Trial primary completion date: Feb 2023 --> Dec 2024

Once every 2-week dosing of GEN-1 in human studies is warranted. Future combination studies of Gen-1 with immune checkpoint inhibitors will evaluate the safety and efficacy of this regimen.

P2, N=50, Recruiting, Imunon

Comparing results of a single-arm early-phase trial to those of a rigorously matched HCT ECA yielded insights regarding comparative efficacy prior to a randomized controlled trial. The effect size estimate itself informed both the decision to continue development and the randomized phase II trial (ClinicalTrials.gov identifier: NCT03393884) sample size. The work illustrates the potential of HCT data to inform drug development.

To guide development of the novel immune agent GEN-1, we compared endpoints experienced by patients from a recent neoadjuvant single-arm phase Ib study of GEN-1 plus standard chemotherapy to those of similar historical clinical trial patients in receipt of standard chemotherapy alone. To compare safety and efficacy endpoints following first-line neoadjuvant weekly GEN-1 immunotherapy and carboplatin and paclitaxel to standard first-line neoadjuvant carboplatin and paclitaxel in women with advanced ovarian cancer, we first applied key OVATION-1 trial (NCT02480374) inclusion and exclusion criteria to the Medidata Enterprise Data Store (MEDS) data to identify candidate historical clinical trial patients for comparison. The comparison of patient endpoints from a single-arm phase Ib trial to a historical clinical trial SCA provided informative and relatively reliable estimates of efficacy endpoints which were used to inform GEN-1’s expected effect study size in the phase II setting. This information led to a decrease in the number of planned patients for the subsequent randomized phase II trial. More broadly, this approach supports the ability of historical clinical trial patient comparisons to inform drug development via trial design, something which may further increase the scientific value of early phase trials.

Adding GEN-1 to standard NAC is safe, appears active and impacts the tumor microenvironment.

P1/2, N=130, Recruiting, Celsion | Trial completion date: May 2024 --> Dec 2024 | Trial primary completion date: Aug 2022 --> Feb 2023

"For first-line treatment 80.8% of patients received cisplatin or carboplatin plus gemcitabine. The genomic profile of SCC encompasses important genes including TP53, PIK3CA and KEAP1. TP53 mutations could be associated with PDL1 expression, generating hypothesis regarding specific treatment options."

From our knowledge, it is the first time that a simple IPS could be useful to predict outcome for patients with metastatic pancreatic neuroendocrine tumors treated with everolimus or capecitabine and temozolomide.