ACD440 is a VR1 antagonist that is in the clinical development phase, and the company’s aim is to develop a new topical local treatment for neuropathic pain. The drug candidate, an important strategic in-licensing arrangement carried out in January 2020, is a good fit and strengthens the company’s clinical portfolio. The project has its origins in Big Pharma and is based on strong scientific grounds.
The compound has previously undergone phase I clinical trials, in which both tolerability and early efficacy endpoints were observed. The mechanism of action of the project is via VR1 receptors, which have a key role in pain signaling and ACD440 has been shown in preclinical trials to have an effect on both nociceptive and neuropathic pain. The compound has previously undergone extensive preclinical safety studies and since the compound is being developed for local use, systemic exposure can be kept very low, while the concentration of the compound locally can be kept high for maximum analgesic effect. Nociceptors are stimulated by heat, acid and strong food, which leads to feelings of pain. Despite the differences in these stimuli, a single target protein expressed in these pain-sensing nerve cells responds to them all.
The molecular target is the VR1 receptor, which is expressed in sensory neurons and is upregulated in the skin of individuals with certain types of neuropathic pain. Consequently, there is strong scientific support for local treatment with this type of target mechanism Neuropathic pain is associated with impaired quality of life and current treatments rarely provide adequate pain relief. In all, an estimated 7–8 percent of the adult population worldwide suffers from pain with neuropathic elements, corresponding to about 25 million individuals in the US, Europe and Japan alone. About 50 percent of patients do not respond to current first-line treatment and it is specifically toward this group of individuals that AlzeCure is aiming its new intended treatment.
In December 2020, AlzeCure initiated a phase Ib clinical trial for ACD440 according to plan to assess both tolerability and early signals of efficacy. The positive study results was communicated according to plan during April 2021. The compound is currently being developed as a gel for topical use, but AlzeCure is also evaluating other formulations such as spray or patch to further broaden thearea of indication.