Therapeutic Areas
Our discovery platform can identify compounds that potently block the progression of amyloid toxicity. These compounds can be designed with selectivity toward a particular misfolding protein (narrow spectrum) or with polypharmocology (broad spectrum), for proteins implicated in over 160 rare/orphan diseases and major indications including
Alzheimer’s Disease and Tauopathy
Alzheimer’s is a chronic, progressive, and ultimately fatal dementia that robs people of their ability to remember how to do even simple tasks, or even the names of their children. The burden it puts on families and caregivers is immense, and its cost to society grows each year to staggering amounts. And it has no cure: there are drugs on the market that treat symptoms, but none of them has been shown to add even a single day of life to an Alzheimer’s patient.
Tauopathies are protein misfolding disorders that concern aggregation of tau, a key protein that normally acts as part of the scaffolding within neurons. When it misfolds, it can cause a variety of incurable, debilitating disorders, including
- Tau-mediated frontotemporal dementia (FTD)
- Progressive supranuclear palsy (PSP)
- Corticobasal degeneration
- Pick’s disease
- Chronic Traumatic Encephalopathy (CTE)
The number of persons expected to survive into their 80’s and 90’s is expected to grow exponentially due to advances in medicine, technology, and improving social and environmental conditions. A very large segment of the U.S. population, the baby boomer generation, is at or near retirement age. Thus, the segment of the U.S. population aged 65 and older by 2030 is projected to double to 71 million persons or 20% of the total population. This will drive the dramatic growth in persons with tauopathy over the next few decades. While the overall prevalence of tauopathy is driven by Alzheimer’s disease, the most common cause of dementia, the combined burden of “rare” tauopathies is close to 35 per 100,000 people – and estimates of CTE run as high as 6% of the entire population.
Amyotrophic Lateral Sclerosis (ALS)
Commonly known as “Lou Gehrig’s Disease”, amyotrophic lateral sclerosis is a rare, progressive motor neuron disease. Sufferers of ALS experience ever-increasing muscle weakness, gradually losing the ability to walk, to speak, to eat… and eventually, to breathe. The vast majority of ALS patients die within 10 years of diagnosis – there is no cure.
Given that one of the hallmarks of ALS is aggregation of TAR DNA binding protein (TDP) within the central nervous system, it is a protein misfolding disease. Treventis is leveraging its CCM technology for discovery of drugs that broadly impact on TDP isoform misfolding, with the potential for disease-modifying effects in ALS as well as in TDP-mediated frontotemporal dementia and other TDPopathies such as limbic-predominant age-related TDP-43 encephalopathy (LATE).
Parkinson’s Disease
Parkinson’s disease (PD) is the second most common age-related neurodegenerative disease after Alzheimer’s. It is characterized by the accumulation of alpha-synuclein in the basal ganglia of a patient’s brain, akin to the “plaques” and “tangles” one sees in Alzheimer’s disease. Patients with PD have progressively debilitating movement and behavioral symptoms.
Currently, there are no disease-modifying treatments for Parkinson’s disease on the market and the “best” drug yet available was discovered over 50 years ago. Approximately 7-10 million people worldwide are living with PD, with over 1.5 million in North America alone. Approximately 70,000 North Americans are diagnosed with PD each year. There is a tremendous medical need for new drugs in PD.
Cancer
According to the World Health Organization, cancer is one of the world’s leading causes of death. Nearly 10 million people died of cancer in 2018. Cancer is a notorious killer and its primary mechanism is also notorious: rapid division and growth of abnormal cells that culminate in primary tumors and metastases that spread to other organs / parts of the body. What is perhaps less appreciated is that cancer is a protein misfolding disease. Several important oncogenes can mutate, resulting in proteins that cannot perform their original function and in fact, can result in toxic gains-of-function. TREVENTIS’ newest research direction combats these proteins.