Top-line data for CDNF presented without surprises
Primary safety endpoint met
Plans for next study to determine efficacy in the pipelineTreatment-related serious adverse events resolved
Management provided further details yesterday in a webcast on the initial 12-month data of the first-in-human clinical trial of CDNF, with emphasis placed on the safety and tolerability. Safety data remain encouraging, and importantly, the two treatment-related serious adverse events that led to two participants withdrawing from the trial in the first 6-month main study were not replicated in the subsequent 6-month extension study. These two events were described as being infusion-procedure related infections, and following the mitigation strategies put in place by Renishaw (the intra-cranial delivery system provider), the final 87 infusions were conducted without adverse events. Importantly, no dose-limiting toxicities related to CDNF itself were recorded.
Suggestions of an efficacy signal persist
As the study was small (n=17), and carried out in a sub-optimal patient group with moderately advanced disease, management rightly tempered expectations for any meaningful efficacy read-out. Nonetheless, a couple of encouraging individual cases with responses on clinical assessment by UPDRS and DAT PET imaging were highlighted, including a sustained DAT PET result in one of the “high responders” from the 6-month data presentation. Overall, a lack of obvious deterioration at the group-level coupled with signals of efficacy in certain patients gives reason to look forward to the next clinical study where efficacy can be gauged with greater confidence.
More clinical data to come before the next study is finalised
We view yesterday’s webcast as largely in line with the previously reported data and without any major surprises, so we leave our estimates unchanged. We look forward to the remaining data from secondary endpoints yet to be analysed, and the DAT PET imaging from the follow-up phase of the study at 19 months, along with the design of the next clinical study for CDNF to be finalized by year-end.