Efranat Ltd.

This study evaluates the safety and tolerability of the investigational drug EF-022 in the treatment of adult patients with Recurrent Respiratory Papillomatosis (RRP). Patients will be administered EF-022, either intramuscular or subcutaneous, for a period of 6 months. Preliminary effect of the drug on the disease will be evaluated by following the number and severity of the lesions in the respiratory tract and the effect on voice changes.

Activated macrophages, present in excess during natural inflammatory responses, bear the potential to kill and eradicate cancer cells. Macrophage activation has been demonstrated to require the serum-borne vitamin D binding protein (known as Gc protein), as well as B and T lymphocytes. However, in various cancer patients the Gc protein is deglycosylated by serum α-N-acetyl galactosaminidase (Nagalase) secreted from cancer cells. This deglycosylated Gc protein, lacking the N-acetylgalactosamine monosaccharide, cannot be converted to its form of Macrophage Activating Factor, leading to immunosuppression rather than Macrophage activation against cancer cells.
Efranat has developed cancer immunotherapy based on Macrophage Activating Factor produced from natural Gc protein extracted from FDA approved healthy human plasma.
In this phase I study, the treatment is given as Intramuscular, once-weekly injection of EF-022, for two courses, while each course is comprised of 4 injections.
The investigational treatment is expected to enhance immune response, thereby, improve patient's well being, quality of life and disease control.
Primary objectives:
To determine the safety and tolerability of EF-022 and to define the maximal tolerated dose (MTD) for potential administration.
To identify the Dose Limiting Toxicity (DLT) of EF-022.
Secondary objectives:
To determine the 'Recommended Phase 2 Dose' (RP2D) based on MTD data, immunological and pharmacodynamics markers
To explore preliminary efficacy of EF-022 in advanced solid tumors according to the 'Response Evaluation Criteria in the modified Solid Tumors' (RECIST 1.1) and blood levels of tumor-related markers known to reflect tumor burden.
Exploratory objectives:
To assess levels of immune-related factors in peripheral blood (determined by FACS analysis), reflecting induced immunological activities, including but not limited to, natural killer (NK), monocytes (M1 and M2) and T cell subpopulations (effector vs regulatory, CD4+ and CD8+ cells), B cells (CD20), myeloid and dendritic cells etc.
To assess the change in serum levels of protein biomarkers in the blood.
To immunohistochemically assess and compare tumor derived tissue samples Pre and post treatment. To analyze the infiltration of different population of cells into the tumor bed.