LXEO: Highlights from the RBC Healthcare Conference

Research evidence excerpt

LXEO: Highlights from the RBC Healthcare Conference

tes less focus on tissue expression levels with recent administration, and LVMI >10% has

always been the endpoint that the study is powered on. High-sensitivity troponin remains a strikingly consistent endpoint (~50-60%

reductions across all patients) and could serve as prognostic biomarker in earlier-stage cardiac patients who do not yet meet the

abnormal LVMI inclusion threshold. On the confirmatory trial, formal discussions with regulators have not yet commenced, and

management noted that such discussions are not required to initiate the pivotal; the confirmatory trial could be built around

outcome endpoints correlated with LVMI, and whether randomized placebo controlled or not depends on the endpoint ultimately

chosen. On manufacturing/CMC, comparability protocol had met FDA standard, been fully cleared and no additional questions

had emerged. Manufacturing is generating compelling yields, and batches have already been manufactured and are ready to dose

patients.

PKP2 Continues to Make Progress Following Promising Early Signal And Next Update Is 4Q26 - On the regulatory path, company

is planning an FDA meeting in 2026 to discuss a potential accelerated approval pathway; NSVT is emerging as the preferred primary

endpoint for a potential accelerated approval pathway, given that NSVT shows the strongest correlation with the clinical outcomes.

No liver enzyme-related SAE had been reported (some observed, non-SAE elevations are reasonably acceptable given a gene

therapy dosed 100x higher vs FA) and study recruitment has been strong. We look forward to the 12-month all-high-dose data

in 4Q.

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