We are deeply sad to have to relay the following news to our patient community.
Yesterday, 6 July there was a public announcement made by Chris Freitag Chief Medical Officer (CMO) at Dynacure about the discontinuation of the DYN101 (Unite-CNM) ‘antisense therapy’ programme for x-linked myotubular myopathy and dynamin 2 centronuclear myopathy. This is what the company said:
‘After significant and thoughtful consideration, we have ended our Unite-CNM Phase 1/2 study of our investigational therapy, DYN101. This decision was made because we are unable to continue dosing at the low study dose or increase to a dose level at which we would expect to see clinical efficacy.
• Unite-CNM was designed a “dose escalation” study. The first cohort of patients (SAD1) received a single dose of DYN101, which was well tolerated, and the study advanced.
• The second cohort (SAD2) of patients received a higher dose of DYN101. This was the lowest dose we had expected to observe any potential clinical efficacy. At this point, liver enzyme elevations were observed in several patients. Based on these findings, the dosing was changed from actual to ideal body weight.
• Despite this change in dose calculation, all six of the patients showed liver enzyme elevations and five out of six showed low platelet counts.
• Based on these changes in lab values, dose interruptions were needed. In collaboration with our Independent Data Monitoring Committee, we made the decision to pause dosing. Study participants continued their follow up visits. At no point were any patients at risk.
• All study participants continued follow up, and liver and platelet levels returned to normal ranges.
• In an initial analysis of all available clinical efficacy assessments including muscle biopsies from all six trial participants, no signs of clinical improvement were seen. Based on the tolerability findings at the low dose level thus far, continuation of dosing or even dose escalation will not be possible.’
Chris Freitag, CMO at Dynacure added ‘This is not the news we ever wanted to share, as we know how great your needs are for safe and effective treatments. As a company and as individuals, we are overwhelmed with disappointment.’
Dynacure have announced that they will make every effort to make the data collected during the study publicly available, contributing to our better understanding of the conditions. They are also still investigating other potential routes but that it is ‘too early to comment on other efforts’. They thanked everyone involved in the efforts and particularly the patients who were enrolled to the Study.
