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| Statement |
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| We're very proud of the progress we're making in the GALILEO 1 trial of FLT201 |
| Furthermore, we believe FLT201 is the opportunity to be first-to-market gene therapy negotiated in type 1 with a best-in-class profile compared to other gene therapies in development |
| Because the variance is more stable than wild type, we believe it has the potential to reach the deeper tissues that ERT poorly addresses and to stay in tissues longer, more effectively clearing harmful substrates and improving clinical outcomes |
| As we look towards these initial data, we believe that a good safety profile and plasma GCase above the normal level would constitute a positive early clinical signal of FLT201 potential |
| With the momentum in the GALILEO 1 trial of FLT201, initial clinical data this quarter and the natural extension of our work in Parkinson's disease, I firmly believe that Freeline is poised for both near- and long-term success |
| We have made tremendous progress this year |
| We believe that FLT201 has the potential to do just that by providing better efficacy than current treatments with a onetime therapy |
| With the momentum we have going in FLT201, the exciting initial clinical data readout later this quarter and the promising research projects that we've announced, in particular, the GBA1-linked Parkinson's disease, I truly believe we are well positioned for near- and long-term success |
| Advancing FLT201 clinical development is our greatest strategic priority, and we are extremely pleased with our progress and with the momentum we are seeing in the trial |
| Investigator and patient interests are strong |
| As many of you know, we spent the last 1.5 years laying a strong foundation for the trial, opening up 15 sites across the U.S., the U.K., Europe, Latin America and Israel, and we are seeing that investment pay off in the current pace of enrollment |
| The early preclinical data looked promising with our GCase variant, demonstrating at least 20 fold greater activity and wild-type enzymes in various cell lines, including brain and neuroblastoma cell |
| So we're really excited about the opportunity to generate and share some data later this quarter on both patients |
| In addition to our clinical and research progress, we have also made significant strides to build a leaner, more focused and sustainable organization |
| Sharma highlights the ongoing unmet need in Gaucher disease, the rationale for gene therapy in Gaucher and the opportunity to dramatically reduce both disease and treatment burden |
| Maybe Pam, you want to talk a little bit about what data we'll be collecting during the trial? Obviously, it's early days in this first cohort, and I think there's a very strong correlation between plasma GCase activity and clinical outcomes |
| We have line of sight into multiple additional patients, and we're executing against our time lines to deliver meaningful initial data this quarter and continue to efficiently advance the trial |
| The dosing of these patients represents a significant milestone for FLT201 for Freeline and most importantly, for Gaucher patients who need and deserve better treatment options |
| And so as Michael mentioned, we have with strong nonclinical data demonstrating the correlation between plasma GCase and Lyso-Gb1 reduction |
| We are focused on working on programs that we believe are highly differentiated with the potential to address serious ongoing unmet needs for patients and drive significant value for shareholders |
| Based on what we see preclinically with FLT201 as well as what we know from ERT, there's a strong correlation between plasma GCase levels and substrate clearance and subsequently between substrate clearance and clinical outcomes |
| FLT201 is a highly differentiated gene therapy candidate that delivers a novel transgene developed by our scientists for a rationally engineered longer-acting variant of GCase, the enzyme deficient and people with Gaucher disease |
| Unlike ERT, however, a gene therapy has the opportunity to deliver a continuous level of enzyme with a onetime infusion |
| Thank you all, and have a good day |
| I will have more data on the first patient in a second, we expect to have enough data on both patients to provide meaningful insights into safety and enzyme activity to help inform FLT201's emerging product profile as a potential best-in-class therapy |
| We strive to do this by optimizing all components of our gene therapy candidates to truly unlock with a potential in this modality |
| We do expect to see enzyme activity improvements during this early phase of the trial, and they should be meaningful as we look to see what's the future development plan for the program in terms of dosing |
| Sharma outlined during our KOL event, both the success and shortcomings of ERT support the rationale for gene therapy in Gaucher disease |
| Additionally, the wild-type GCase delivered by ERT to short half-life, meaning patients experience troughs and enzyme levels in between infusions, giving harmful substrates the opportunity to build backup, potentially contributing to disease progression |
| And based on our experience in nearly 20 patients we would expect meaningful increases in plasma case levels within 4 to 6 weeks of dosing |
| Statement |
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| Many Gaucher patients continue to experience serious symptoms despite treatment with currently approved therapies |
| Our net loss was $14.8 million or $0.23 per ordinary share for the 6 months ended June 30, 2023, compared to a net loss of $51.1 million or $0.95 per ordinary share in the same period in 2022 |
| Even with onetime charges related to these actions, our total operating expenses decreased 34% in the first half of 2023 compared to the same period last year |
| Current therapies also carry a heavy lifelong treatment burden |
| Despite treatment with ERT, however, many patients continue to have symptoms, particularly those associated with deeper tune, including bone and lung |
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