PTC Therapeutics has reportedly disclosed the initiation of a Phase 1 clinical trial for the evaluation of PTC518 amongst healthy volunteers. The candidate was recognized from PTC’s splicing platform and is reportedly being created for the treatment of HD (Huntington’s disease).
According to PTC Therapeutics’ CEO, Stuart W. Peltz, the clinical trial’s initiation for the evaluation of PTC518 for the Huntington’s disease initiative is a significant milestone towards the identification of a potential new Huntington’s disease treatment meant for directly addressing the underlying cause of the disease. Peltz has further stated that PTC518 is the first small molecule therapeutic that is orally bioavailable for the identification of specific and selective modulation of Huntington’s disease splicing for the reduction of huntingtin protein. This trial is similar to the SMA drug from the company’s splicing platform and is speculated to establish the appropriate PTC518 dose that brings about a reduction in the HTT protein levels.
PTC believes that the convenient oral administration of PTC518 has the potential for altering the treatment landscape for these patients, stated Peltz.
An orally bioavailable molecule having systemic distribution and broad central nervous system, PTC518 has reportedly been developed for the purpose of targeting Huntingtin protein expression with high specificity and selectivity. Having demonstrated favorable pharmaceutical properties, the candidate has depicted uniform lowering of the Huntingtin protein throughout the brain in animal models. Reliable sources, however, reveal that no disease modifying therapies have been approved for HD presently.
As per reports, the PTC518 Phase 1 study comprises both multiple and single ascending dosing regimens intended at favoring the establishment of dose selection, safety, and pharmacology for the Phase 2 study. Additionally, protein levels and Huntingtin mRNA will also be measured for gaining early proof of splicing mechanism in humans as was carried out in the risdiplam SMA development initiative. The data from Phase 1 study is likely to be available during the first half of 2021, add sources.