By Jonathan Springston, Editor, AHC Media

The manufacturers of the first drug under development designed to target the underlying causes of Huntington’s disease generated excitement Monday when researchers announced positive first trial results.

IONIS-HTTRx, a joint venture between Ionis Pharmaceuticals and Roche, demonstrated an ability to reduce the level of mutation in the huntingtin protein during the drug’s Phase 1/2a trial. Huntington’s disease is a neurodegenerative disorder that develops during an unstable cytosine-adenine-guanine repeat expansion in the huntingtin gene, which can create a mutant form of the huntingtin protein.

“The completion of the Phase 1/2a study of IONIS-HTTRx is a historic moment in the fight against HD as it represents the successful completion of the first trial to treat the underlying cause of Huntington’s disease, the genetic mutation itself,” Louise Vetter, president and CEO of the Huntington’s Disease Society of America, said in a statement. “The fact that levels of mutant huntingtin were reduced in correlation to the dose of IONIS-HTTRx that was given is significant, and the fact that participants in this first Phase 1/2a study are able to continue on the drug through open label extension gives us optimism regarding its safety.”

Researchers said they will move forward with more safety and efficacy studies to be conducted globally. Meanwhile, the researchers expect to present results at medical conferences and publish the results in a peer-reviewed medical journal in early 2018. Look for more information and developments about this treatment in future issues of Neurology Alert and in the “Pharmacology Update” section of Internal Medicine Alert.