Actimmune (Interferon gamma-1b) was developed by Horizon Pharma as a potential treatment for cancer. It is a recombinant protein, similar to a protein produced by the immune system called interferon-gamma (IFN-gamma).

The U.S. Food and Drug Administration (FDA) has already approved Actimmune to treat two rare diseases, chronic granulomatous disease (CGD, an inherited disease in which the immune system is absent or not functioning properly), and severe malignant osteoporosis (SMO, a disorder that affects normal bone formation).

Currently, Actimmune is being investigated as a potential treatment for people with cancer in combination with other drugs.

How Actimmune works

Like the naturally occurring IFN-gamma, Actimmune is a signaling protein that binds to cell-surface receptors leading to the transcription of genes that activate certain cells in the immune system such as macrophages, natural killer (NK) cells, and cytotoxic T lymphocytes (CTLs), or T-cells. In addition, Actimmune also stimulates the exposure of tumor-associated antigens (TAAs) to immune system cells triggering antibody-dependent cell-mediated cytotoxicity (ADCC) against those antigens. Altogether, Actimmune is expected to enhance the immune response, leading to tumor cell death.

Actimmune in clinical trials

In addition to studies that led to the approval of Actimmune for the treatment of CGD and SMO, Actimmune also is being investigated in several clinical trials for the treatment of cancer in combinations with other drugs.

For example, Actimmune was evaluated as an adjuvant, or add-on, therapy to treat 98 patients with different types of advanced cancer, including melanoma, renal cell carcinoma, and soft-tissue sarcoma in a Phase 2 clinical study (NCT00002505). The results of the study, published in the Journal of Immunotherapy, showed that the treatment was well-tolerated and patients treated with Actimmune had two-year and five-year survival rates of 45% and 29%, respectively.

Another Phase 2 trial (NCT00501644) evaluated the effectiveness and toxicity of carboplatin, Leukine (sargramostim), and Actimmune in women with recurrent, platinum-sensitive ovarian, fallopian tube, and primary peritoneal cancer. The results of the study, published in the scientific journal Gynecologic Oncology, showed that of 54 patients evaluable for response, nine (17%) had a complete response and 21 (39%) had a partial response.

A Phase 1 trial (NCT02614456) is currently evaluating Actimmune in combination with Opdivo (nivolumab) in advanced solid tumors that have progressed despite at least one prior systemic therapy. The study will primarily assess the safety and tolerability of the therapy combination. The secondary objective is to assess the overall response rate, progression-free survival, and overall survival of patients. The trial is recruiting participants at the Fox Chase Cancer Center in Philadelphia, Pennsylvania.

A Phase 1/2 study (NCT03112590) is testing Actimmune in a combination with Taxol (paclitaxel), Herceptin (trastuzumab), and Perjeta (pertuzumab). It aims to evaluate the safety and effectiveness of the combination therapy for the treatment of women with breast cancer. The study is currently recruiting up to 43 patients in the U.S. and is expected to be completed in June 2022.

A Phase 2 study (NCT03063632) to evaluate whether the combination of Actimmune and Keytruda (pembrolizumab) works better in treating patients with relapsed or refractory mycosis fungoides and Sézary syndrome. The trial is currently recruiting patients in Philadelphia, Pennsylvania, and Seattle, Washington, and is expected to be completed in May 2018.

Actimmune is also being evaluated in a Phase 1 trial (NCT02948426) in women with recurrent or refractory ovarian cancer, fallopian tube cancer, or primary peritoneal cancer. In the study, Actimmune will be administrated together with autologous monocytes and Sylatron to help immune cells fight cancer. The trial is recruiting up to 40 participants in Bethesda, Maryland.

Additional information

Some of the most common side effects of Actimmune, based on its use for other conditions, include flu-like symptoms such as fever, chills, and aches.

According to Horizon, Actimmune should be used with caution in people who have heart disease, seizure disorders, and some blood disorders.


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