Elenagen Plus Gemcitabine Improves Overall Survival for Patients With Platinum-Resistant Ovarian Cancer
Key Clinical Summary:
- Design/Population: A phase 2 randomized controlled trial evaluated elenagen (a plasmid DNA–based immunomodulatory agent encoding p62/SQSTM1) plus gemcitabine versus gemcitabine alone among 30 patients with platinum-resistant ovarian cancer, randomized 1:1.
- Key Outcomes: Median overall survival (OS) improved from 13 months with gemcitabine to 25 months with elenagen + gemcitabine (HR 0.41; 95% CI, 0.18 to 0.94; P = .036). Time-dependent and landmark analyses showed a significant association between longer elenagen exposure and improved survival (P < .001). Safety profiles were comparable between arms, with no new toxicity signals.
- Clinical Relevance: Adding elenagen to gemcitabine significantly prolonged survival without added toxicity in platinum-resistant ovarian cancer, supporting further investigation of elenagen-based immunomodulation in combination with chemotherapy.
The addition of elenagen, a plasmid DNA-based immunomodulatory agent encoding the p62/SQSTM1 protein, to gemcitabine therapy significantly improved overall survival compared with gemcitabine monotherapy for patients with platinum-resistant ovarian cancer, according to phase 2 study results published in the International Journal of Gynecological Cancer.
Previous research has found elenagen to have promising activity for the treatment of platinum-resistant ovarian cancer. Researchers conducted a phase 2 randomized control trial evaluating the safety and efficacy of elenagen plus gemcitabine compared to gemcitabine monotherapy.
Patients were randomized 1 to 1 to receive gemcitabine monotherapy (1000 mg/m2 on days 1 and 8 every 21 days) or elenagen (2.5 mg intra-muscularly weekly) plus gemcitabine. The primary end point was overall survival (OS) which was defined as the time from random allocation to death from any cause. Secondary end points included progression-free survival (PFS) and time-dependent elenagen efficacy.
Overall, 30 patients were included, 15 of which received elenagen plus gemcitabine and 15 patients receiving gemcitabine monotherapy as a control. Among patients receiving gemcitabine monotherapy, the median age was 56 years (interquartile range [IQR], 52 to 63) and patients who received elenagen plus gemcitabine had a median age of 54 years (IQR, 47 to 62).
The median OS was 13 months (95% confidence interval [CI], 10 to 27) in the gemcitabine group versus 25 months (95% CI, 17 to not reached; P = .031; log-rank) in the combination arm. Cox proportional hazard model revealed treatment with elenagen plus gemcitabine was associated with a 59% relative reduction in risk of death (hazard ratio [HR] 0.41, 95% CI 0.18 to 0.94, P = .036).
Time-dependent and landmark analyses confirmed a significant association between prolonged elenagen exposure and improved survival (P < 0.001). Safety profiles were similar between arms, with no new toxicity signals, and post-progression survival outcomes were comparable.
The researchers concluded, “The addition of elenagen to gemcitabine chemotherapy is effective in patients with platinum-resistant ovarian cancer, increasing overall survival without elenagen-associated side effects.”
They added, “Future studies of elenagen with various tumors and chemotherapy regimens, especially gemcitabine, are warranted.”
Source:
Krasny S, Baranau Y, Bakin E, et al. Elenagen, a p62/SQSTM1-encoding plasmid, improves overall survival in patients with platinum-resistant ovarian cancer: a phase II trial. International Journal of Gynecological Cancer. Published online January 7, 2026. doi:10.1016/j.ijgc.2025.104456
