PTC Therapeutics launches Phase 1 clinical trial of Huntington’s disease therapeutic

PTC Therapeutics launched a phase 1 trial in healthy volunteers that will investigate single and multiple ascending doses of PTC518, a therapeutic developed to target the huntingtin protein, according to a press release.

”PTC518 is the only orally bioavailable small molecule therapeutic that was identified to selectively and specifically modulate Huntington's disease splicing to reduce huntingtin protein,” Stuart W. Peltz, PhD, CEO of PTC Therapeutics, said in the release. “Analogous to the [spinal muscular atrophy] drug from our splicing platform, this trial is anticipated to establish the appropriate PTC518 dose that reduces [huntingtin] protein levels.”

The release described the therapeutic as “a small molecule splicing modifier” that contains a “premature termination codon” that triggers huntingtin mRNA degradation and a subsequent decrease in huntingtin protein levels. There are currently no disease-modifying treatments for Huntington’s disease.

The company identified PTC518 from its splicing platform. It has demonstrated “uniform lowering of the huntingtin protein throughout the brain in animal models.”

The phase 1 trial aims to determine safety, pharmacology and dose selection for the phase 2 study, according to the press release. Data from the present study are expected in the first half of 2021.