During clinical trials, researchers work to advance clinical knowledge, build an understanding of certain diseases or conditions, and learn more about a new drug, surgical device, or behavioral modification technology. However, not all clinical trials are successful. According to Fierce Biotech, multinational pharmaceutical company Sanofi is one of the more recent companies whose trial failed. Sanofi shared that following a failed Phase 2 venglustat trial for Parkinson’s disease, the company would halt a number of mid-phase programs.
Sanofi and its Programs
In the Phase 2 clinical trial, researchers were evaluating venglustat (ibiglustat), an investigational treatment, for patients with Parkinson’s disease. The orally administered treatment is a glucosylceramide synthase (GCS) inhibitor designed to penetrate and treat the central nervous system (CNS). In particular, Sanofi believed the drug would be helpful for patients with GBA gene mutations, found in around 10% of all Parkinson’s diagnosis. This specific genetic mutation has been linked to earlier symptom onset, quicker disease progression, and worse patient outcomes.
Parkinson’s Disease
Parkinson’s disease is a progressive central nervous system (CNS) disorder which impacts movement. Dopaminergic neuron death causes Parkinson’s disease. As dopamine-producing neurons degenerate, die, or are damaged, it inhibits communication between the muscles and brain. While the exact cause is unknown, many hypothesize that lifestyle and environmental factors, genetics, and microscopic neuronal proteins play a role in Parkinson’s disease. Typically, Parkinson’s disease occurs in five stages. In stage one, patients may only experience light tremors on one side of the body, which advance to both sides by phase 2. In stages 3 and 4, patients lose balance and experience slowed movement, slowly becoming unable to live independently. Finally, in stage 5, patients are no longer able to stand or walk independently, and may experience hallucinations. Symptoms include:
- Muscle rigidity
- Poor posture and balance
- Slowed movement
- Loss of ability to blink or smile
- Hallucinations
- Dementia
- Sleep disturbances
- Tremor and/or shaking in both hands
- Speech changes, such as slurring or stuttering
- Anxiety or apathy
- Fatigue
- Loss of smell
Learn more about Parkinson’s disease.
