The recent International Prader Willi Syndrome Organisation (IPWSO) research presentation was a very positive experience. For those that were unable to attend or have yet to access the recording, the event covered a total of seven very promising trials. The range of treatments means, that as the UK’s PWS expert Tony Holland states, the greater the opportunity for drugs to help different individuals. FPWR’s Theresa Strong believes that we will get to the stage where combinations of drugs are used to optimise treatments for individuals. Whilst we are not there yet, it truly does feel like a brighter future is edging closer than ever before. Excitingly a number of trials could potentially run in the UK (albeit we have no further information on this at the moment).
Here is a quick rundown of the drug trials covered in the presentation:
- ARD 101 targets the ‘CCK’ system in the gut. The Phase 2 reduced hyperphagia including reduced body fat. Develop the treatment for children and adults. There is potential for phase three trial to include sites in UK although this is still to be confirmed.
- Carbetocin counter acts oxytocin deficiency in PWS and is believed to offer numerous benefits to PWS including treating behaviours and hyperphagia. A company called Acadia have taken this drug from Levo Pharmaceuticals who previously ran the phase 3 trial. A new third phase trial will be run at the low dose which was deemed to be most effective in original trial. It is anticipated this trial will be run in European countries as well as the USA.
- CSTI-500 targets serotonin, dopamine, Norepinephrine and improves the levels of these key neuro transmitters. It is believed it can treat multiple symptoms of PWS. A phase 2 trial will be run in the USA during 2024 and potentially in the UK too.
- KITE PWS RGH 706 is a drug which has the potential to reduce appetite in people with PWS. There is currently a phase 2 trial in the USA and across European countries albeit not the UK.
- Harmony Biosciences are currently trialling Pitolisant which is aimed at treating excess tiredness in PWS. The phase 2 trial successfully met this aim (there is the potential for the drug to treat other aspects of PWS too but this is still being evaluated). The phase 3 trial is currently being planned as a global trial which could potentially include the UK.
- PBF-999 aims to treat hyperphagia and excess body weight. It is progressing to phase 2 trial in Spain.
- Soleno’s DCCR, met two secondary end points in the original trial but not the primary one but this was attributed to Covid. During the Open Label Study, participants improved over time. The FDA requested additional data and the study underwent a randomised withdrawal trial. They expect to have results by end of September 2023.