In February, dosing and new patient enrollment in all cohorts of the HALO trial, a study evaluating the compound HT-100 in patients with Duchenne, was suspended after one of the patients in the trial, receiving 60μg/kg/d (the highest dose in the study), passed away after experiencing serious, life-threatening health issues. Since then, the company has been working with the FDA to analyze the situation and evaluate the extent to which the patient’s health issues were related to HT-100 and/or to other factors. Below is the latest update from Akashi regarding discussions with the FDA and the status of the trial.
Read the update from Akashi:
Dear Foundation Supporters of HT-100,
The FDA has reviewed the research efforts we initiated at the beginning of this year and, to our disappointment, has not yet lifted the clinical hold on dosing in the HT-100 trial. We are in the process of scheduling a meeting with the Agency to discuss the path forward so we can determine what, if any, additional studies will be required before re-initiating clinical dosing.
By way of background, beginning in February Akashi launched an intensive, multifaceted investigation into the serious adverse event that occurred in January. We evaluated numerous biomarkers from both plasma and serum from all patients enrolled in the study. We conducted additional laboratory experiments both with model cellular systems and animal models. We commissioned a thorough review by several independent, internationally-recognized experts in medicine and pathology who examined the relevant clinical history, laboratory results, and experimental outcomes of the additional non-human studies. We also conducted another extensive evaluation of the safety and efficacy data obtained over the course of the approximately two years of the trial. Akashi places the greatest emphasis on ensuring patient safety, and so in an abundance of caution we also committed to a series of additional measures to help further mitigate the risk of serious adverse events in any future HT-100 trials. Our conclusion is that it is now appropriate to resume dosing of HT-100.
Our analysis took into account reports and videos from families of trial participants. The patient reported data indicates that the increase in function achieved in some patients during dosing has steadily declined since dosing was discontinued. All of the information we have gathered, from serum biomarker levels to videos of boys and young men now losing function, drives us to persist.
We will continue to keep you posted on our progress. We appreciate your patience with us and with the FDA as they continue to engage in discussions with us.
While we are a small team that has mostly focused on restarting dosing in the HT-100 clinical program, we have also made progress on two additional compounds in our pipeline. As resources permit, we are continuing to move forward with DT-200 (muscle builder) and AT-300 (calcium regulator).
We will provide additional updates as our discussions with FDA proceed and we gain agreement on the path forward. In the interim, please let us know if you have any questions.
With gratitude for your continuing support,
Marc, on behalf of the Akashi team
Marc B. Blaustein