EGEN-001
Source EGEN-001 at Peptiology
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Summary
EGEN-001 is a plasmid DNA construct encoding interleukin‑12 (IL‑12) that is delivered intraperitoneally using a PEG‑PEI‑cholesterol lipopolymer carrier. In a phase II trial it was tested in women with platinum‑resistant recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer to assess safety and antitumor activity.
Mechanism of Action
The plasmid component of EGEN‑001 is designed to transfect peritoneal cells, leading to local production of IL‑12, a cytokine that promotes Th1‑type immune responses. IL‑12 activates natural killer cells and cytotoxic T‑lymphocytes, enhances interferon‑γ secretion, and can stimulate anti‑tumor immunity within the peritoneal cavity.
What the Research Shows
A single‑arm phase II study enrolled 22 patients with platinum‑resistant recurrent ovarian‑type cancer; 20 received weekly intraperitoneal infusions of EGEN‑001 at 24 mg/m². Fifty‑eight cycles were administered (median 2 cycles per patient). The most common adverse events were grade 1–2 fatigue, fever, chills, abdominal pain, nausea, vomiting, anemia, thrombocytopenia, and leukopenia; three patients withdrew due to low‑grade toxicities. No partial or complete responses were observed (0 % response rate). Stable disease occurred in 35 % of evaluable patients, while 45 % progressed. Six months progression‑free survival was achieved in 30 % of patients, with a median PFS of 2.89 months and overall survival of 9.17 months. The authors concluded that activity was limited and tolerability was suboptimal in this setting.
Reported Benefits
Evidence from the trial suggests that EGEN‑001 may produce disease stabilization in a subset of heavily pre‑treated patients, with 35 % achieving stable disease and 30 % experiencing progression‑free survival beyond six months. However, no tumor regressions were recorded, and the overall clinical benefit remains uncertain.
Limitations of the Evidence
The data derive from a single, uncontrolled phase II study with a small sample size, limiting the ability to draw definitive efficacy conclusions. Lack of a comparator arm, modest response rates, and notable low‑grade toxicities further constrain interpretation. Results are specific to platinum‑resistant recurrent ovarian‑type cancers and may not extrapolate to other disease contexts.
Safety Considerations
Treatment‑related adverse events were predominantly mild to moderate (grade 1–2) and included fatigue, fever, chills, abdominal discomfort, nausea, vomiting, anemia, thrombocytopenia, and leukopenia. Three participants discontinued therapy due to these low‑grade toxicities. No severe (grade 3–4) toxicities were reported in the abstract, but the overall tolerability was described as limited.
How It Is Administered
EGEN‑001 was administered as a weekly intraperitoneal infusion using a PEG‑PEI‑cholesterol lipopolymer formulation, at a dose of 24 mg per square meter of body‑surface area. The delivery method targets the peritoneal cavity directly, aiming for localized IL‑12 expression.
Routes of Administration
No administration routes recorded yet.
Goals & Uses
No goal associations recorded yet.
Contraindications
No contraindications recorded yet.
Adverse Effects
No adverse effects recorded yet.
Drug Interactions
No drug interactions recorded yet.
Population Constraints
No population constraints recorded yet.
Regulatory Status
No regulatory status recorded yet.
Evidence & Sources
- Journal ArticleModerateAlvarez RD, et al.2014-01-01T00:00:00.000000Z
Frequently Asked Questions
What type of cancer was EGEN‑001 tested in?
The phase II trial evaluated intraperitoneal EGEN‑001 in women with platinum‑resistant recurrent epithelial ovarian, fallopian tube, or primary peritoneal carcinoma.
Did any patients experience tumor shrinkage?
No. The study reported a 0 % objective response rate; no partial or complete tumor regressions were observed among the evaluable participants.
What were the most common side effects?
Patients most frequently reported mild to moderate fatigue, fever, chills, abdominal pain, nausea, vomiting, anemia, thrombocytopenia, and leukopenia. Three participants withdrew because of these low‑grade toxicities.
How was EGEN‑001 delivered?
The drug was given as a weekly intraperitoneal infusion, formulated with a PEG‑PEI‑cholesterol lipopolymer to facilitate plasmid delivery to peritoneal tissues.
Is EGEN‑001 approved for clinical use?
The abstract does not indicate regulatory approval; the compound was investigated only within a phase II research setting.
What is EGEN-001?
EGEN-001 is a plasmid DNA construct encoding interleukin‑12 (IL‑12) that is delivered intraperitoneally using a PEG‑PEI‑cholesterol lipopolymer carrier. In a phase II trial it was tested in women with platinum‑resistant recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer to assess safety and antitumor activity.