VIDEO: New therapies bring progress, uncertainties in Friedreich’s ataxia management

Editor’s note: This is an automatically generated transcript, which has been slightly edited for clarity. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription.

At this point, there are several therapies which are coming along, a large trial called the ExRx trial, which included the nutraceutical, nicotinamide riboside has finished. The data are not yet available. That is a, essentially, over-the-counter, available-on-the-internet substance that, in models, would be expected to potentiate the effect of omaveloxolone, and thus could be taken with it. That study will be out soon, the last patient has been seen.

The other drug in the pipeline, which is the next closest, is vatiquinone. It had two trials in FA, one in adults and one in children. I will call them both near misses. People got better, but they did not reach their primary endpoint in showing enough of benefit. The P values end up about 0.1. Normally, that would be not really be an approvable drug, but the FDA is considering a new drug application, because vatiquinone is an extremely safe drug, and there's no drug available for people two to 16 at this point. The date, which we'll know, is in mid-August as to whether it's approved.

Again, it's a very safe drug. Its benefit is modest if you compare the adult study of vatiquinone to the adult study of omaveloxolone, one would honestly have to say that Omaveloxolone was a better drug, but again, if it were possible, people would probably do better on both of these, because they overlap in their effects. They're not absolutely identical. The other new therapies, there's new therapies coming along which restore frataxin three different ways right now. One is the injectable peptide called nomlabofusp, also called TAT-frataxin, also called Larimar CTI-1601. It's in an open label extension right now in some early studies in children. Form of frataxin, which has had a certain sequence attached to the end which allows it to enter cells.

The questions are where exactly does it go in the human body? Does it get to all the right spots? Their major data have been looking at skin biopsies and cheek swabs, which are not affected regions, so does it get everywhere? And because we don't make this little extra piece that they put on frataxin, it will provoke an immune response. Whether that immune response can be managed is something which we'll have to find out over time.

Gene therapy, there has been announced a clinical trial roughly one year from now for combined heart and nervous system gene therapy from Solid Biosciences, and there is already an ongoing clinical trial of cardiac gene therapy. Remember, though, the biggest issues in gene therapy are how many places can you get in one injection? Because you'll only get one chance to do it. Finally, there's a drug called DT-216, which turns the gene back on. It is said to go everywhere in the body. It has the most potential to get closest to the four-letter word, cure, of the things that we're going to be seeing this year in early-stage trials.