- Alterity Therapeutics has successfully completed its Phase 1 study of PBT434 for patients suffering from Parkinson’s
- In preclinical models PBT434 has been shown to block alpha-synuclein (protein found in neurons) accumulation
- Planning for Phase 2 is currently underway and updates will be provided in the upcoming months
Alterity Therapeutics announced the successful completion of it’s Phase 1 clinical trial of PBT434, an oral bioavailable small molecule inhibitor of alpha-synuclein assembly.
When assembled in the brain, alpha-synucelin (protein found in neurons) is a pathological mark of Parkinson Disease conditions and is considered an important biologic target for treating neurodegenerative diseases.
PBT434 is an oral small molecule drug candidate that has the potential to treat conditions such as Parkinson Disease and Multiple system atrophy (neurodegenerative disease similar to Parkinson’s).
PBT434 was granted Orphan designation by the U.S. FDA earlier this year for the treatment of Multiple system atrophy.
In preclinical models PBT434 has been shown to block alpha-synuclein accumulation and has prevented the loss of neurons in the region of the brain primarily affected by Parkinson’s. This was demonstrated in several animal models.
The trial is continuing to demonstrate that the drug is safe and well tolerated with adverse event rates comparable to placebo.
The Phase 1 clinical trial recruited healthy and elderly (>65) volunteers with the primary goal of assessing the safety and tolerability of PBT434 after both single and multiple use.
Secondary goals included evaluating the pharmacokinetics in plasma and spinal fluid that enabled determination of how the drug is absorbed and metabolised in the body.
Exposure to the drug was compared between elderly and healthy volunteers and those with Parkinson’s. This data achieves concentration in the brain that is comparable with those found in the animal study.
None of the subjects experienced any serious adverse events or an adverse event that led to the study being discontinued.
Chief Medical Officer Dr David Stamler is pleased with the results and is ready to begin Phase 2 trials.
“We are very pleased that the excellent safety and tolerability profile in the adult population has now been extended to elderly volunteers. This data will provide the foundation for our interactions with regulatory authorities later this year as we advance the program toward a Phase 2 clinical trial,” he said.
PBT434 is the first in a new generation of small molecules that is designed to block the accumulation and aggregation of alpha-synuclein.
Alterity is already in the preparatory phase of planning for the Phase 2 clinical trial and will provide updates in the upcoming months.