Future Plans for Evaluating Spur Therapeutics’ FLT201
Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, discussed areas of interest for future study for the Gaucher disease gene therapy product.
This is the second part of an interview with Reena Sharma, MD. For the first part, click here.
Reena Sharma, MD
Credit: Northern Care Alliance NHS Foundation
Results from the phase 1/2 GALILEO-1 clinical trial (NCT05324943) evaluating Spur Therapeutics’ FLT201, an adeno-associated virus (AAV) vector-based gene therapy intended to be a one-time treatment for Gaucher disease, were recently presented at the 21st Annual WORLDSymposium, held February 3 to 7, 2025, in San Diego, California. Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital who presented the data, spoke with CGTLive® during the conference about the key takeaways from her presentation.
During the interview, Sharma spoke about areas of interest for future study, noting the need to look at additional factors like disease burden in the bone and impact of the therapy on quality of life. She also shared her view on the importance of reconsidering how clinicians think about unmet needs for certain patient populations.
CGTLive: Have there been areas of interest identified where more research is needed for FLT201?
Reena Sharma, MD: I think for the future studies, of course we have to still look at the their biochemical parameters and, of course, we need to look at their disease burden in the bone. That's something that we haven't presented in this meeting, but it's something that I'm sure you will hear about in the near future—that there has been quite a positive impact on the disease burden in this cohort of patients, and I'm sure you will hear more about it in the future, as well. But I think the other thing that we have to really [look at] going forward, when we look at future analyses and information that you would expect from this going forward, is how is it actually impacting patients themselves? Because it's a small cohort we haven't mentioned it, but I think going forward we should really look at their quality of life, any change in their pain scoring, and how these patients have felt overall themselves once on this therapy. I think this is going to be a good area of exploration, in addition to looking at their bone density and looking at the burden of disease in the bones. Also, I think we need to explore the antibodies going forward in this cohort of patients. In this cohort of 6 patients that we treated with this very small dose of FLT201, 2 patients had transient antibodies. It didn't really impact the clinical efficacy, but it's something that we should really explore going forward and make sure that it's not really having a long-term effect on these patients.
Is there anything else you want to share?
I think we probably covered quite a bit of it already, but I think what is the main thing that we need to understand as clinicians is there are still unmet needs in this cohort of patients. Like me, the majority of clinicians do not just not treat Gaucher disease patients, but patients with other diseases as well, and subclinically, sometimes we start comparing it relatively in terms of what are the unmet needs. When you think about patients with diseases 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 a burden of therapy, which is a lifelong therapy, they still have unmet needs, their bone complications are ongoing, and 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 for managing the residual disease and ongoing issues and improving their quality of life.
I think the last thing I would really add to that is I'm really looking forward to the phase 3 trial for this product, and I think we could really be hopeful for a very good outcome.
