Two days ago, PTC Therapeutics announced that the drug candidate PTC518 has entered into a “Phase 1 Clinical Trial”. The initial results are expected in the first half of 2021. 

PTC518 is an orally bioavailable molecule. In other words, the drug candidate is administered in the form of a pill taken through the mouth. 

In animal studies, PTC518 has been found to lower the level of the disease-causing huntingtin protein. This lowering has also been found to be dose-dependent: the higher dose of PTC518, the greater lowering of huntingtin. 

» Learn more about huntingtin lowering: 
Advances on many fronts in the battle against the protein that causes Huntington’s disease

In-human trials

Now, PTC Therapeutics has started a “Phase 1 Clinical Trial” with healthy volunteers. What does this mean? 

Phase I trials are the first-in-human studies and are usually carried out with a small group of people. Here, safety and potential side effects are being studied. In addition, the trial is designed to test the best dosing of the drug.

Moreover, healthy volunteers are participating in order define the limits of “normal” and to isolate potential side effects, risks and tolerability.

In PTC’s trial, two different dosing regimens will be tested: single and multiple ascending dosing.

In the single ascending regimen, participants will only receive one dose of the drug candidate. In the multiple ascending regimen, each participant will receive multiple doses. 

Hopefully, PTC will be able to figure out whether the drug candidate PTC518 is safe and well-tolerated, in addition to finding the best dosage.

Important milestone

In a recently issued press release, Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc, said:

– The initiation of the clinical trial to evaluate PTC518 for the Huntington’s disease program is an important milestone towards identifying a potential new Huntington’s disease treatment that directly targets the underlying cause of the disease. 

The first trial results are expected in the first half of 2021.

Learn more:

Short Introduction to Drug Development in Huntington’s Disease: