Executive Summary

Q4 2024 EPS was in line with consensus: actual EPS of $-0.78 vs Wall Street consensus of $-0.78; revenue not reported, consistent with a development-stage profile . EPS estimates shown below from S&P Global data*.
Regulatory clarity advanced for LX2006 (FA cardiomyopathy): FDA alignment that frataxin expression will be evaluated for “any increase from baseline” (not a fixed threshold); inclusion of pediatric cohorts; and use of prospective natural history data as external control, improving odds of accelerated approval .
LX2020 (PKP2-ACM) posted early clinical signals (71% and 115% PKP2 protein increases; 67% PVC reduction in the first participant at 6 months), but interim clinical data timing shifted to 2H 2025 (from late Q1/early Q2 guidance), a modest timeline slip .
Cash, cash equivalents and investments of $128.5M provide runway into 2027, sustaining trial execution and regulatory engagement cadence .

What Went Well and What Went Wrong

What Went Well

LX2006 regulatory pathway clarity: “We are pleased to share further regulatory clarity… and… an accelerated approval pathway” with protein expression evaluated for any baseline increase and pediatric cohorts in the pivotal study .
LX2020 early clinical signals: Post-treatment biopsies showed 71% and 115% PKP2 protein increases; one participant had a 67% reduction in PVCs and QRS normalization, supporting biological activity .
Balance sheet durability: $128.5M in cash and investments, with management reiterating sufficient runway into 2027 .

What Went Wrong

Operating expense intensity: Q4 R&D rose to $18.37M and G&A to $9.02M vs Q4 2023, driving net loss of $25.9M; higher spend reflects pipeline progression but increases cash burn .
LX2020 interim read push: Initial guidance for “late Q1/early Q2 2025” shifted to “second half of 2025,” which may defer data catalysts .
Q3 EPS miss: Q3 2024 EPS was $-0.89 vs consensus $-0.67*, highlighting intra-year estimate sensitivity and the impact of spend cadence .

Financial Results

Quarterly Financial Comparison (oldest → newest)

Metric	Q2 2024	Q3 2024	Q4 2024
Revenue ($USD Millions)	Not reported	Not reported	Not reported
EPS (basic & diluted, $USD)	-$0.64	-$0.89	-$0.78
R&D Expense ($USD Millions)	$16.56	$23.42	$18.37
G&A Expense ($USD Millions)	$6.99	$8.12	$9.02
Total Operating Expenses ($USD Millions)	$23.55	$31.54	$27.38
Net Loss ($USD Millions)	-$21.24	-$29.49	-$25.92
Cash & Equivalents/Investments ($USD Millions)	$174.98	$157.02	$128.53

Actual vs Wall Street Consensus (S&P Global)

Metric	Q2 2024	Q3 2024	Q4 2024
EPS Actual ($)	-$0.64	-$0.89	-$0.78
EPS Consensus Mean ($)	-$0.69*	-$0.67*	-$0.78*
EPS Surprise ($)	+$0.05	-$0.22	$0.00
Revenue Consensus Mean ($USD Millions)	$0.00*	$0.00*	$0.00*
EPS – # of Estimates	6*	6*	6*

Values retrieved from S&P Global.*

Program KPIs (Q4 disclosures)

Program	KPI	Data
LX2020 (PKP2-ACM)	PKP2 protein expression	+71% and +115% vs baseline in two cohort 1 patients
LX2020 (PKP2-ACM)	PVCs	67% reduction at 6 months in first evaluated participant; QRS normalization
LX2006 (FA cardiomyopathy)	Pivotal endpoint approach	FDA-aligned: frataxin expression evaluated for any increase from baseline; LVMI as co-primary; pediatric inclusion; external control

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
LX2006 pivotal endpoints	Q3 2024 → Q4 2024	Co-primary: LVMI (-10% target) and frataxin (40% positive area by IHC)	Co-primary unchanged; frataxin to be evaluated for any increase from baseline (LCMS), pediatric cohorts included; external control via prospective natural history	Refined/clarified (methodology and threshold)
LX2020 interim data timing	2025	Late Q1/early Q2 2025	Second half of 2025	Delayed
Operational runway	Q2–Q4 2024	Into 2027	Into 2027	Maintained

Earnings Call Themes & Trends

Topic	Previous Mentions (Q2 2024)	Previous Mentions (Q3 2024)	Current Period (Q4 2024)	Trend
LX2006 regulatory alignment	Initiated FDA engagements on surrogate endpoints; expecting update by YE 2024	RMAT granted; accelerated approval path with LVMI and frataxin co-primary endpoints; target thresholds shared	Further FDA clarity: frataxin “any increase” from baseline; pediatric inclusion; external control; LCMS planned in pivotal	Positive momentum, endpoint framework refined
LX2020 clinical execution	Cohort 1 recruiting; interim data 2H 2024	Cohort 1 completed; initial data target late Q1/early Q2 2025	Cohort 2 completed; interim clinical data in 2H 2025; EU orphan designation	Data timing drift; regulatory/clinical traction maintained
Safety profile	LX2006 well tolerated; no treatment-related SAEs	LX2006 generally well tolerated; one Grade 2 asymptomatic myocarditis noted and later biopsy negative	LX2006/LX2020 generally well tolerated; no treatment-related SAEs to date	Stable safety narrative
Cash runway	$175.0M into 2027	$157.0M into 2027	$128.5M into 2027	Sustained runway; balances trending down with spend

Management Commentary

“We are pleased to share further regulatory clarity for LX2006… and… appreciate the continued partnership from the FDA on an accelerated approval pathway…” — CEO, R. Nolan Townsend .
“We are also encouraged by the favorable safety profile and early data observed in participants dosed with LX2020 to date.” — CEO .
“Post-treatment cardiac biopsies… showed increases in PKP2 protein expression… [and] 67% reduction in PVCs… normalization of QRS.” — Q4 release .
On LX2006 pivotal design: “Frataxin expression… evaluated for any increase from baseline… LCMS expected… external control in planned pivotal study.” — Q4 release .

Q&A Highlights

Dose response and baseline severity: Greater biomarker improvements correlate with higher baseline LVMI; evidence of dose-dependent response across cohorts (LVMI, troponin, wall thickness) .
FDA alignment timing: Statistical plan details targeted for 2025; ongoing interactions under RMAT without necessarily requiring formal Type B meetings .
Inclusion criteria: Pivotal to enroll LVMI ≥2 SD above normal; target ≥10% LVMI reduction at 12 months; frataxin “any increase” via LCMS at 3 months .
Pediatric dosing: Confidence in therapeutic index; adults and pediatric cohorts expected to use same per-kg dose (1e12 vg/kg) .
Endpoints: Troponin seen as supportive (noisy primary), LVMI has precedent; study powered to LVMI while frataxin serves binary responder analysis .
External control comparability: Natural history study will mirror pivotal inclusion criteria; propensity matching to ensure comparability .
Safety: Minimal liver enzyme elevations; overall well tolerated .

Note: An earnings-call transcript specifically labeled for Q4 2024 was not available in our document system; we used a company transcript discussing LX2006 interim data and pivotal design published April 7, 2025 .

Estimates Context

Q4 2024 EPS matched consensus: actual $-0.78 vs $-0.78*, with revenue consensus at $0.00*, consistent with development-stage operations .
Intra-year context: Q2 2024 was a modest beat (actual $-0.64 vs $-0.69*), while Q3 2024 was a miss (actual $-0.89 vs $-0.67*) driven by higher operating expenses .
With no revenue line reported and high OpEx variability tied to clinical progress, consensus adjustments should track spend cadence and trial timing updates (e.g., LX2020 interim read shifted to 2H 2025) .