Update from Aspa
On Wednesday, the 28th of August, Canavan Foundation, Canavan Research Illinois and NTSAD met with Aspa Therapeutics to discuss updates on their gene therapy program. Aspa reported they have been in discussions with FDA and that they remain on track for a gene therapy treatment trial to be open for enrollment in 2020. Agreement has been reached on the preclinical requirements, and manufacturing is on schedule.
Aspa commented that throughout the exchange with FDA, the importance of the natural history study was emphasized: Without strong natural history data, Aspa may be asked to run a placebo controlled study, which could result in a delay in getting the therapy to many patients. Aspa also made the point that a strong study means not only rigorous data collection but also necessitates enrollment of enough patients to be considered a robust comparator group – Aspa estimated that a total of 30 enrolled patients or more may be required, depending on ages and amount of data available for each patient. On the update call, Aspa referenced the recent approval of Zolgensma (a gene therapy approved for the treatment of patients with SMA) as a positive example of how a natural history study supported a clinical trial with no placebo group and a smooth path to approval. These interactions with FDA have confirmed the importance of the natural history study to the Canavan community.
Aspa plans to give us further updates as preparations for the gene therapy clinical trial proceed.