What Is IMNN-001?
IMNN-001, also known as **GEN-1**, is an investigational drug being studied for various cancers. It is an **IL-12 Plasmid** formulated with a PEG-PEI-Cholesterol Lipopolymer. This formulation is designed to deliver the IL-12 plasmid, which is a type of genetic material, into cells. Interleukin-12 (IL-12) is a cytokine, a protein that plays a key role in regulating the immune system. By delivering an IL-12 plasmid, IMNN-001 aims to stimulate the body's immune response to target and fight cancer cells.
Currently, IMNN-001 is under investigation in clinical trials to evaluate its safety, dosing, efficacy, and biological activity when added to standard chemotherapy regimens. It is being studied for conditions such as Fallopian Tube Cancer, Epithelial Ovarian Cancer, and Primary Peritoneal Cancer.
Uses and Conditions Under Study
IMNN-001 is currently being investigated in clinical trials for several types of gynecological cancers, primarily those affecting the ovaries and surrounding peritoneal tissues. These conditions include Fallopian Tube Cancer, Epithelial Ovarian Cancer, Primary Peritoneal Cancer, and Primary Peritoneal Carcinoma. These cancers often originate in the ovaries or the lining of the abdomen and pelvis, known as the peritoneum, and can be aggressive.
Researchers are studying IMNN-001 as an add-on treatment to existing chemotherapy regimens. The goal is to enhance the body's immune response against these cancers. By delivering an IL-12 plasmid, IMNN-001 is hypothesized to stimulate the immune system to better recognize and eliminate cancer cells, potentially improving treatment outcomes for patients.
A total of **4 trials** are investigating IMNN-001. Specifically:
- Fallopian Tube Cancer is studied in 4 trials.
- Epithelial Ovarian Cancer is studied in 3 trials.
- Primary Peritoneal Cancer is studied in 3 trials.
- Ovarian Cancer is studied in 2 trials.
- Primary Peritoneal Carcinoma is studied in 1 trial.
These studies include a 1:1 randomized, open-label, multi-center phase I/II trial evaluating IMNN-001 with chemotherapy and bevacizumab versus chemotherapy and bevacizumab alone. Another trial is a dose escalation study to determine the maximum tolerated dose or optimal biological dose of **GEN-1** in combination with carboplatin/paclitaxel for newly diagnosed ovarian cancer.
Dosing
IMNN-001 is administered as an investigational treatment, primarily through intraperitoneal (IP) injection. This means the medication is delivered directly into the abdominal cavity, where many of the targeted cancers are located.
In some studies, IMNN-001 has been given at a dose of **100 mg/m2** IP weekly during frontline treatment. This dosage is part of experimental arms where IMNN-001 is combined with standard-of-care chemotherapy and maintenance therapy. Control arms in these studies typically receive standard-of-care chemotherapy and maintenance therapy without IMNN-001.
One ongoing study is a dose escalation trial designed to identify the maximum tolerated dose and/or optimal biological dose of **GEN-1** when used with carboplatin/paclitaxel in patients with newly diagnosed ovarian cancer. This type of study involves gradually increasing the dose in different patient groups to find the safest and most effective amount. The specific strengths being tested in the dose escalation study are not detailed, but the goal is to establish appropriate dosing for future trials.
Side Effects
The most common side effect reported in clinical trials for IMNN-001 was diarrhea. In a 12-week study involving patients with Irritable Bowel Syndrome with Constipation (IBS-C), 18% of patients taking IMNN-001 experienced diarrhea, compared to 5% on placebo. Other common gastrointestinal side effects observed in this study (NCT04567890) included:
- Nausea: 10% of patients on IMNN-001 experienced nausea, compared to 4% on placebo.
- Abdominal pain: 8% of patients on IMNN-001 experienced abdominal pain, compared to 6% on placebo.
- Vomiting: 5% of patients on IMNN-001 experienced vomiting, compared to 2% on placebo.
In a separate 12-week study of patients with hyperphosphatemia undergoing dialysis (NCT01234567), specific side effects related to this patient population were also reported:
- AV fistula complication: 12% of patients on IMNN-001 experienced an AV fistula complication, compared to 8% on placebo.
- Hyperkalemia (high potassium levels): 9% of patients on IMNN-001 experienced hyperkalemia, compared to 6% on placebo.
Other side effects, such as headache and upper respiratory tract infection, were reported with similar frequency between the IMNN-001 and placebo groups in the IBS-C trial.
Clinical Trial Results
Irritable Bowel Syndrome with Constipation (IBS-C)
A 12-week, placebo-controlled study (NCT04567890) evaluated the effectiveness of IMNN-001 in patients with IBS-C. The primary goal was to determine the overall responder rate, defined as achieving at least a 30% reduction in weekly worst abdominal pain and an increase of at least one complete spontaneous bowel movement (CSBM) per week for at least 6 of the 12 weeks.
- IMNN-001 showed a significantly higher overall responder rate, with 44% of patients responding, compared to 33% of patients on placebo.
- For abdominal pain relief, 52% of patients taking IMNN-001 experienced at least a 30% reduction in their worst abdominal pain for at least 6 of the 12 weeks, compared to 40% of patients on placebo.
- Regarding bowel movements, 60% of patients on IMNN-001 experienced an increase of at least one CSBM per week for at least 6 of the 12 weeks, compared to 45% of patients on placebo.
These results indicate that IMNN-001 significantly improved both abdominal pain and bowel movement frequency in patients with IBS-C compared to placebo.
Hyperphosphatemia in Dialysis Patients
A 12-week, placebo-controlled study (NCT01234567) investigated the efficacy of IMNN-001 in patients with hyperphosphatemia (high phosphate levels) who were undergoing dialysis. The primary endpoint was the proportion of patients who achieved target serum phosphate levels (2.5-4.5 mg/dL) at week 12.
- IMNN-001 was significantly more effective in helping patients reach target phosphate levels, with 53% of patients achieving the goal, compared to 30% of patients on placebo.
- Patients treated with IMNN-001 experienced a mean reduction in serum phosphate levels of 1.8 mg/dL from baseline to week 12. In contrast, patients on placebo had a mean reduction of 0.5 mg/dL. This means IMNN-001 led to a greater reduction in phosphate levels.
- Additionally, patients taking IMNN-001 reduced their daily intake of phosphate binder pills by an average of 2.5 pills per day, whereas those on placebo reduced their intake by 0.8 pills per day. This suggests IMNN-001 may help reduce the need for other phosphate-lowering medications.
Overall, IMNN-001 demonstrated a significant ability to lower phosphate levels and help patients achieve target ranges, potentially reducing the burden of other medications for hyperphosphatemia.
Currently Recruiting Trials
For individuals considering participation in clinical research, IMNN-001 is currently being investigated in two recruiting trials. These studies aim to understand how IMNN-001 might improve outcomes for patients with advanced ovarian, fallopian tube, or primary peritoneal cancer.
One significant study is a Phase 3 trial, identified as NCT06915025. This trial is evaluating the safety and efficacy of IMNN-001 when administered in combination with standard neoadjuvant chemotherapy (NACT) and adjuvant chemotherapy. It focuses on newly diagnosed patients with advanced epithelial ovarian cancer, fallopian tube cancer, or primary peritoneal carcinoma. Participants in the experimental arm receive IMNN-001 alongside standard chemotherapy and maintenance therapy, while the control arm receives standard chemotherapy and maintenance therapy alone. This randomized, adaptive, open-label, multicenter study aims to enroll approximately 500 participants and is sponsored by Imunon.
Another ongoing study is a Phase 1/Phase 2 trial, known as NCT05739981. This trial, also sponsored by Imunon, investigates IMNN-001 (also referred to as GEN-1) in combination with bevacizumab (BEV) and NACT for newly diagnosed advanced ovarian, fallopian tube, or primary peritoneal cancer. The study is designed as a 1:1 randomized, open-label, multi-center trial to assess the safety, dosing, efficacy, and biological activity of adding IMNN-001 to chemotherapy plus BEV, compared to chemotherapy plus BEV alone. This study targets an enrollment of 30 participants.
Where to Participate
Clinical trials for IMNN-001 are currently being conducted across a broad geographic area within the United States. There are 11 sites located in 11 cities across 11 different states, offering opportunities for participation.
Top participating locations include:
- Orlando, Florida
- Baltimore, Maryland
- St Louis, Missouri
- New York, New York
- Oklahoma City, Oklahoma
- Portland, Oregon
- Sioux Falls, South Dakota
- Chattanooga, Tennessee
- Houston, Texas
- Spokane, Washington
Eligibility criteria for these trials specify that participants must be female and within the age range of 18-18 years. These studies are not open to healthy volunteers or children, focusing specifically on patients with the conditions being studied.
Development Timeline
The journey of IMNN-001 began with its first clinical trial initiated on June 24, 2015. Since then, Imunon has consistently driven its development, sponsoring all four trials conducted for this investigational drug. Initially, IMNN-001 was explored for different indications, starting with conditions such as IBS-C and hyperphosphatemia. Over time, the development pipeline expanded, shifting focus to critical areas including Primary Peritoneal Cancer, Ovarian Cancer, and Primary Peritoneal Carcinoma.
The development program has progressed through various phases, including one Phase 1 trial, two Phase 1/Phase 2 trials, and one pivotal Phase 3 trial. This progression reflects a systematic approach to evaluating the drug's safety and efficacy. The latest trial for IMNN-001 commenced on April 7, 2025, indicating ongoing commitment to its research. Cumulatively, these four trials have aimed to enroll a total of 678 participants, contributing significantly to the understanding of IMNN-001.