BBL1020

Indications: Addiction and Movement Disorders

Ligand Target Indication Development
BBL1020 Dopamine D2 Receptor Addiction / Movement Disorders Preclinical

BBL1020, is being developed to treat addiction and dopamine-related movement disorders such as dyskinesia associated with the treatment of Parkinson's disease, Huntington's disease and certain dystonias.

Addiction

The financial burden of chemical dependency in the US alone is difficult to quantify, and the full scope of human cost is incalculable. Despite the magnitude of the problem, there are a very limited number of pharmacotherapies for the clinical treatment of addiction, and those available are of limited use. New medications for addiction are urgently needed.

The mesolimbic dopamine system is the final common pathway of all drugs of abuse, and this is thought to explain the limited success of antipsychotics, which act as dopamine receptor antagonists and partial agonists. Based on our understanding of addiction and how the brain processes reward, the functional selectivity and favorable pharmacokinetic profile associated with BBL1020, we may potentially have a candidate medication that could significantly reduce addiction and prevent the critical brain changes that occur as addiction develops.

Movement Disorders

Levodopa-Induced Dyskinesia

The D2 receptor is localized in brain regions that are critically associated with Parkinson’s Disease and its treatment. Levodopa-induced dyskinesia (LID), characterized by involuntary choreiform or dystonic movements and is among the most feared adverse effects of long-term dopamine replacement therapy (DRT).

Functionally selective agonists that continuously stimulate dopamine receptors at nigrostriatal synapses without diminishing the benefits of levodopa may prove effective in preventing the onset of LID or reducing the severity of LID once it has developed.

BetaBlue’s ligand, BBL1020, shows functional selectivity at the dopamine D2 receptor and is among the first molecules identified to feature β-arrestin-2 partial agonism, and Gi antagonism. This unique pharmacological signature may hold special relevance for patients with Parkinson's Disease suffering from (LIDs) and could protect against shortcomings of other current D2 receptor modulators.

Huntington's Disease and Dystonias

Huntington's disease is a rare but fatal genetic neurological disorder causing progressive deterioration of brain function. The disease affects 30,000 Americans and symptoms usually appear between the ages of 30 to 50. Symptoms include mood and personality changes, forgetfulness, involuntary movement, unsteadiness and slurred speech.

Dystonia's are a collection of related neurological syndromes affecting young children and adults that are characterized by involuntary twisting movements of different parts of the body and unnatural postures. More than 3 million people worldwide are affected with forms of dystonia, of which, some are classified as rare diseases where currently available treatments are only modestly effective.

Huntington's disease and dystonias both exhibit alterations in dopamine-related signaling in the brain. Dopamine antagonists and partial agonists have previously been used to treat these illnesses, however, success has been limited by the off-target side effects of these drugs. The functionally selective activity of BBL1020 in targeting dopamine D2 receptors holds unprecedented promise for treatment efficacy in these neurological illnesses for children and adults.