Lexeo Therapeutics, Inc. Common Stock Chardan's 10th Annual Healthcare Conference

Thanks for having us. Absolutely.

Yeah. Lexeo is a clinical stage company focusing on genetically mediated diseases with high unmet need. Our lead program is LX2006 in Friedreich's ataxia cardiomyopathy.

Yeah. We have a registrational study that we started enrolling at the end of the second quarter of this year.

In our phase I/II, we have shown compelling effect size on left ventricular mass index, a variety of different biomarkers, and on the neurological side of the disease through the mFARS scale. Our second program is in PKP2-associated arrhythmogenic cardiomyopathy. That program is LX2020. That program has an ongoing phase I/II, and we have shown a good effect size on non-sustained ventricular tachycardia, on premature ventricular contractions, and with a good safety profile. What to look out for in the next year or so, we have in the FA program, we are expecting a top-line readout on the phase III trial in the second half of 2027 and a BLA filing in the first half of 2028.

As I said on the PKP2 program, we will be reading out data with the majority of our high-dose patients at at least 12 months of follow-up very soon in 4Q, alongside a regulatory update as well. In addition to those programs, we have a preclinical pipeline. There is LX2021 and LX2022 for desmoplakin and hypertrophic cardiomyopathy, respectively. We are excited that we just acquired Mantle Therapeutics.

Yes Which gives us a portfolio of different neurologically oriented FA programs as well.

Yeah. LX2006 has shown an effect in the mFARS scale similar to the commercially approved therapy. But we believe we're reaching the peripheral nervous system and the components of the mFARS scale that are mediated in the peripheral nervous system. For example, skeletal muscle and dorsal root ganglia. But we're not transducing the brain. And we know that there are certain components of the neurological side of FA that are mediated in the cerebellum. So the real strategy around adding the Mantle portfolio is to find a therapeutic that will transduce the brain, so that ultimately we will have clinical trials that include cohorts that have been previously treated with LX2006.

So ultimately, there's a potential there to have a therapeutic package that treats both the cardiac side, where we know LX2006 is showing a good effect, but also to have a component that reaches the brain to treat the totality of FA across both.

Yeah. I can speak to that. In January, what we had shown was two effect sizes on PVCs and on NSVT. We had shown a 14% reduction in PVCs and a 22% reduction in NSVT. And particularly in a couple of patients who had actually gone to nine months, we had shown a 65% reduction in NSVT. So as we start to look at the upcoming update, we would want to see many of those patients, I guess maybe one or two would have hit 12 months. So we want to share the data from those patients as they prolong their effect and time in the study, and also hoping to couple that with a regulatory update as well. But we want to see a deepening of the effect size over time, just like we had the hint of in those patients that had reached nine months.