Reena Sharma, MD, on Future Plans for Evaluating FLT201

This is the second part of an interview with Reena Sharma, MD. For the first part, click here.

“When you think about patients with disease like Pompe or even Fabry, their unmet needs with the current therapy are huge. So we may think that, ‘Oh, [patients with diseases like Gaucher disease] are okay,’ but I think what we misunderstand and probably do not realize is that although their issues may not be as big as some of the other conditions we look after, they still mean a lot to this cohort of patients. They still have burden of therapy, which is a lifelong therapy, they still have unmet needs, their bone complications are ongoing, so I don't think as clinicians, we should underestimate how it's impacting the patients, and we should always look at better therapies and explore better options...”

Spur Therapeutics’FLT201, an adeno-associated virus (AAV) vector-based gene therapy intended to serve as a one-time treatment option for Gaucher disease, is currently being assessed in the phase 1/2 GALILEO-1 clinical trial (NCT05324943). Interim results from this study were recently presented by Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, at the 21st Annual WORLDSymposium, held February 3 to 7, 2025, in San Diego, California.¹

CGTLive® interviewed Sharma during the conference to learn more. After going over the key results and their implications, Sharma discussed some of the future plans for the therapy, emphasizing the need to explore biochemical parameters and disease burden in the bones, areas of interest which were not touched on in the presentation at the meeting. She also pointed out that future studies should also evaluate the therapy's impact on patients' quality of life, pain levels, and overall well-being. Additionally, Sharma noted that further exploration of antibody responses is necessary. In the small cohort treated with FLT201, 2 patients developed transient antibodies. Although it did not appear to affect clinical efficacy, she noted that long-term effects should be monitored. Sharma also spoke about the need for clinicians to reconsider how they think about the unmet needs for patient communities in general and stated that she is looking forward to the phase 3 trial for FLT201.

REFERENCES
1. Sharma R, Goker-Alpan O, Schwartz I, Giraldo P, Ferrante F, Foulds P. Results from GALILEO-1, a first-in-human clinical trial of FLT201 AAV gene therapy in adult patients with Gaucher Disease Type 1. Presented at: WORLDSymposium, held February 3-7, 2025, in San Diego, California. Poster #318