Fall 2018 Update
It has been a busy fall with the PPI-1040 program progressing on numerous fronts and we wanted to give everyone an update.
A major focus of the program all year has been on manufacturing of the drug, as mentioned previously. It has been an intensive process to get the manufacturing of PPI-1040 to a point where it was feasible to scale up synthesis to kilogram quantities. After numerous improvements of the synthetic route a large-scale synthesis is now possible, and the first 5 kg batch has been initiated. This 5 kg of drug is intended to be used for testing in animals to support the safety of the compound. To ensure that we are prepared to begin those animal studies when the drug is available, we interviewed several contract research organizations which specialize in these studies. The vendor has now been selected and we are working on finalizing the study designs. We plan to approach the FDA again before the end of the year to get their confirmation that the planned studies will generate the data they need to evaluate PPI-1040’s safety.
The Natural History Study remains the other major focus of the team. A meeting between Med-Life Discoveries and Dr Bober’s group at Nemours has been scheduled for early December. This meeting will act as an opportunity to finalize the protocol, ensuring that it can be submitted to the ethics review board before the end of the year. This is slightly behind our original goal of having the first patient enrolled by the end of the year. However, we believe the extra time and care spent planning the Natural History Study is worthwhile, as it is critical include the correct assessments in the protocol. If we fail to include an assessment in the Natural History Study, we will not generate the historical data necessary to justify its inclusion in future therapeutic trials. This has meant talking to a variety of experts to get their opinions on how to best capture the physical manifestations of RCDP, in a manner that will be acceptable to the FDA.
We understand that traveling to Wilmington for assessments throughout the Natural History Study is not a trivial request. It was important to us that we make the planning of travel and the handling of reimbursements as easy on the families as possible. That is why we are working with a company that specializes in travel logistics for patients in clinical trials. They have the expertise and manpower to ensure travel arrangements match the needs of each family and expedite any reimbursement claims. We hope that this will make the process a little easier on everyone.
While we have a commitment from MLD’s shareholders to fund the next stage of the program, finding a partner or further investment remains a priority. As such, we are devoting a lot of time to finding a partner that understands the RCDP program and wants to support it through the clinical trial.
Finally, stay tuned in the coming weeks for an interactive videoconference where MLD will be presenting an update on the program and answering your questions. We will be posting a survey shortly to get feedback from you on what time of day would work best for this type of presentation, and what areas everyone is most interested in learning more about. We will share that survey on the MLD Facebook page once it is live.
Please check back often to this site, and the MLD Facebook page for updates!