The EMA orphan drug designation – granted for investigational therapies for rare diseases – gives Immune Design certain benefits, including protocol assistance, reduced fees for regulatory procedures, and market exclusivity within the European Union for up to 10 years after the drug receives marketing approval for the designated indication.
The U.S. Food and Drug Administration (FDA) also has granted orphan drug designation to G100 for the same indication.
G100 is a potent intratumoral toll-like receptor-4 (TLR4) agonist and the first immunotherapy solely generated from Immune Design’s GLAAS platform.
GLAAS stands for “GLA Adjuvant Systems” and is based on a synthetic small molecule, called Glucopyranosyl Lipid A (GLA). GLA binds specifically to the TLR4 receptor leading to the activation and expansion of certain immune cells, called dendritic cells.
G100 is a specific formulation of GLA that is injected directly into the tumor. When applied together with other therapies that kill the tumor – such as radiation or chemotherapy – the dendritic cells surrounding the tumor recognize the several tumor proteins (antigens), triggering a potent tumor response. In this way, G100 activates anti-tumor immune responses directly inside the tumor (its microenvironment).
Previous both preclinical and clinical studies have shown that the G100’s local and systemic immune responses lead to a reduction in the size of the tumor.
Currently, G100 is being evaluated in a clinical trial both as a single agent (with local radiation) and in combination with MSD‘s anti-PD-1 agent Keytruda (pembrolizumab) for patients with follicular non-Hodgkin lymphoma. (MSD is known as Merck in the U.S. and Canada.) The trial, NCT02501473, is recruiting participants.
The study’s primary objective is to determine the therapy’s safety and tolerability by assessing the frequency and severity of adverse events. Patients will be followed for up to two years.
Secondary goals include assessing patients’ clinical responses – a preliminary indication of effecrtiveness and out-of-target (abscopal) tumor responses in non-treated, distal tumor sites. Researchers also will assess blood and tumor samples for exploratory biomarkers of immunologic and tumor response.