With the new year, we’d like to send an update on our progress with CoA-Z. In September, we told you that we had decided to set aside plans for further clinical trials with CoA-Z, instead focusing our efforts on compiling the necessary data to present to the FDA in order to gain approval to make the compound more widely available as quickly as possible. This was based on our belief that our data was strong enough to submit without doing any additional studies. We still very much believe this to be true.

The FDA will be looking at the data from the clinical trial to decide whether CoA-Z is safe and whether it changes a biological marker of PKAN in the blood. As you know from our previous communications, the trial data showed that CoA-Z did change the blood biomarker just as we had hoped, and it did it in a ‘dose-dependent’ way, meaning that higher doses produced a larger response.

To demonstrate safety, the FDA requires that we show in an objective way that there is no difference between the safety of the compound being tested (i.e. CoA-Z) compared with placebo, and between one dose compared with another: this is why the first 6 months of the trial had to be done in a ‘double-blind, placebo-controlled’ way (meaning nobody knew who was taking a placebo and who was taking CoA-Z). Now that we’ve finished the analysis of data from this part of the study we are confident that CoA-Z was safe and well-tolerated at all doses tested. We are finalizing the report from this phase of the study and preparing to submit it to the FDA, along with a lot of other required information about things such as the CoA-Z manufacturing process, its stability when stored in different conditions, its effects in mice, and much, much more. Our goal is to submit a draft of our application to the FDA by the end of March.

While it is impossible to predict how long the FDA will take to complete their review of the draft, they have indicated that this ‘pre-review’ before final submission may reduce the risk of delays later on, and so we are grateful for the agency’s time and attention to our submission. In the interim, we will shift our focus to analyzing data from the second segment of the trial, called the ‘open-label’ portion (when everyone was taking the same dose of CoA-Z) so this is ready to include in the final submission. For those of you who were in the trial, it is only natural to wonder which group you or your child was in during those first 6 months. After we get our final application in to the FDA, we will have access to this information ourselves and will contact everyone privately to share these details. For now, please do not contact us about this—we will reach out once we have the work completed and submitted.

We are very hopeful our data will convince the FDA that no additional studies will be needed. We remain very grateful to the PKAN community for all of your support and encouragement.