Clinical Efficacy Of Plasmid Encoding P62/Sqstm1 (Elenagen) In Combination With Gemcitabine In Patients With Platinum-Resistant Ovarian Cancer

PurposeThe purpose of this clinical study is to evaluate safety and efficacy of ELENAGEN, a novel anticancer therapeutics (plasmid DNA encoding p62/SQSTM1) protein, as an adjuvant to chemotherapy with Gemcitabin (GEM) in patients with advanced platinum-resistant ovarian cancer. Patients and MethodsThis was a prospective randomized multi-center study with two arms. Gemcitabine 1000 mg/m2 days 1,8 every 3 weeks) was administered in both arms: In the Chemo arm (n = 20) GEM was the only treatment, and in the ELENAGEN arm (n = 20) GEM was supplemented with ELENAGEN (2.5 mg i.m. weekly). The primary endpoint was progression-free survival (PFS), and the secondary endpoint was safety. Antitumor activity was assessed by RECIST 1.1 criteria. Safety was assessed on the basis of adverse events (AEs) and serious AEs (SAEs) according to NCI CTCAE version 5.0. ResultsTo data cut-off, the median follow-up was 13.8 months. There were no SAE -related to ELENAGEN treatment. The median progression-free survival (PFS) was 2.8 and 7.2 mo in Chemo and ELENAGEN arms respectively (p Log-Rank = 0.03). Noteworthy, at the time of cut-off, 9 patients (45%) in Elenagen arm did not progress with the longest PFS recorded so far is 24 months. Subgroup analysis of patients in both arms demonstrated high efficacy of Elenagen in the patients with worse prognosis: high pretreatment levels of CA125, progression after only one line of chemotherapy, and peritoneal effusion. ConclusionsAddition of ELENAGEN to Gemcitabine is effective in patients with ovarian cancer, including those with a worse prognosis.