RCDP Program Update

Hello Rhizo families. I hope this update finds you safe and healthy. We wanted to start by saying that in these uncertain times, the health and safety of all Rhizokids and their families is our utmost concern. As it has not advisable for anyone, particularly Rhizokids, to be travelling, clinic visits that were scheduled for the Natural History Study have been postponed. While we are committed to ensuring that we collect this data in a rigorous and standardized way, the safety of the study participants is first and foremost. MLD and the clinical team at the Alfred I. duPont Hospital for Children (AIDHC) will continue to work together to adapt the trial to meet the guidance of the FDA, CDC and local public health authorities.

While in-hospital clinical assessments for the Natural History Study have been temporarily on-hold, we have been focused on updating the protocol. If you are participating in the study, you may notice a few small changes in the study assessments that are being completed. Largely everything remains unchanged, but modification were required to meet the guidance of the FDA and to improve the way that data is captured, such that it is useful to statistician in the future, as we look for clinical endpoints that could be incorporated in a trial. While participants should notice only minor differences, there has been much more that is occurring behind the scenes to optimize this study.

We also received guidance from the FDA on the Quality of Life survey that MLD and Dr Mousumi Bose have been developing. Based on the Agency’s feedback we have undertaken a revision of the survey and will be rolling out pilot testing of the revised survey shortly. Stay tuned to Rhizotrial.org for more information if you are interested in participating in the survey testing.

The biggest progress on the program in the last couple of months has certainly been related to the safety testing of PPI-1040. The very first studies that are required when testing a new experimental drug in animals are called “Dose Range Finding Studies.” This is often the first time that a rigorous animal study is being performed with the goal of understanding the relationship between the dose that is given and the biological response. These studies also provide information on the safety of the drug, as animals are closely monitored for signs of stress and illness following escalating doses of the molecule. We have just completed studies in both rats and non-human primates that evaluate the affects of PPI-1040 on circulating plasmalogen levels and animal health. These studies showed that, as expected, PPI-1040 increases serum plasmalogen levels and was not associated with any signs of clinical toxicity. While these are critical studies for the program and we are very pleased with the results of the studies, they are only a step on the road to a clinical trial. What these studies do allow us to do though is select the proper doses for the month-long safety studies which will be taking place later this year. These are much larger studies, which are used to demonstrate safety to the FDA and enable first-in-human clinical trial. The goal over the coming months will be to finalize the design of these studies so that they can begin once the next batch of drug becomes available.

We are confident that 2020 will be a productive year for the program and are committed to keeping the Rhizo community up-to-date as we go. While we will not be able to present an update at the family conference this year, we will be planning a web-based update presentation for July instead. Stay tuned for more information on that presentation and where to sign-up!