Executive Summary

Q4 revenue surged to $21.39M driven by service revenue from PPQ work for J&J; this was a significant beat versus external expectations of $1.50M, while EPS of -$0.50 modestly missed consensus by $0.02 .
Strategic collaboration with Hologen AI adds $200M upfront cash and up to $230M JV funding, extending runway and fully funding AAV-GAD through Phase 3 and commercialization; MeiraGTx retains 30% JV ownership and exclusive manufacturing supply agreements .
RMAT designation for AAV2-hAQP1 and regulatory alignment positions xerostomia program for a potential pivotal path and BLA filing in 2026; manufacturing licensure expanded in UK and Ireland (QC and GMP viral vector) .
Cash, cash equivalents and restricted cash were $105.7M at YE2024 with plans to fund operations into 2027 and repay $75M debt due Aug-2026, supported by Hologen closing; company remains eligible for up to $285M in bota-vec launch/mfg milestones .
Stock reaction catalysts: substantial capital infusion and fully funded CNS roadmap (AAV-GAD), regulatory de-risking (RMAT, IRD RPDDs, MHRA path), and manufacturing scale/licensure .

What Went Well and What Went Wrong

What Went Well

AAV-GAD positive randomized, sham-controlled bridging data with significant UPDRS Part 3 improvement and PDQ-39 QoL gains; Phase 3 readiness bolstered by Hologen JV funding and AI-enabled trial design .
AAV2-hAQP1 granted RMAT with pivotal alignment and potential accelerated pathways; BLA filing targeted for 2026 .
LCA4 program showed unprecedented efficacy in 11/11 children and MHRA exceptional circumstances pathway without further studies; multiple IRD RPDDs obtained .

Quotes:

“MeiraGTx demonstrated excellent execution in 2024... achieving multiple positive clinical and regulatory milestones” — Alexandria Forbes, Ph.D., CEO .
“The use of Hologen’s AI technology... has already significantly de-risked the AAV-GAD program” .
“AAV2-hAQP1 was granted RMAT... reinforcing the strength of our clinical data” .

What Went Wrong

Continued sizable operating loss and net loss despite Q4 revenue step-up; FY2024 net loss widened to $147.8M vs $84.0M in FY2023, reflecting higher R&D and service revenue costs .
License revenue dropped to $0 in FY2024 following J&J collaboration termination, increasing dependence on service/milestone revenues and external financing .
Q4 EPS modestly missed external consensus by $0.02 even as revenue exceeded expectations, underscoring ongoing cost intensity ahead of pivotal/commercial stages .

Financial Results

Note: Q4 2024 revenue and net loss are derived by subtracting 9M 2024 from FY 2024 figures (see citations). EPS for Q4 reflects external aggregator.

Metric	Q2 2024	Q3 2024	Q4 2024
Revenue ($USD Millions)	$0.282	$10.910	$21.390
Net Loss ($USD Millions)	$48.620	$39.330	$39.399
EPS ($USD)	-$0.76	-$0.55	-$0.50
Loss from Operations ($USD Millions)	-$45.909	-$40.041	-$31.490
Net Income Margin %	-17,235% (48.620/0.282)	-360.5% (39.330/10.910)	-184.3% (39.399/21.390)
EBIT Margin %	-16,278% (45.909/0.282)	-366.9% (40.041/10.910)	-147.3% (31.490/21.390)

Revenue composition:

Revenue Component	Q2 2024	Q3 2024	Q4 2024
Service Revenue ($USD Millions)	$0.282	$10.910	$21.390
License Revenue ($USD Millions)	$0.000	$0.000	$0.000

KPIs and balance sheet highlights:

KPI	Q2 2024	Q3 2024	Q4 2024
Cash & Equivalents ($USD Millions)	$99.974	$122.873	$103.659
Deferred Revenue - Current ($USD Millions)	$3.498	$5.107	$4.827
Note Payable (Net) ($USD Millions)	$72.665	$72.942	$73.221

Versus external estimates (non-S&P Global):

Metric	Q4 2024 Actual	Q4 2024 Consensus	Beat/Miss
Revenue ($USD Millions)	$21.39	$1.50	+$19.89M (Beat)
EPS ($USD)	-$0.50	-$0.48	-$0.02 (Miss)

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
Capital runway	Multi-year	Fund ops & capex into Q2 2026	Into 2027 and repay $75M debt due Aug-2026 (contingent on Hologen closing)	Raised runway
AAV2-hAQP1 (Xerostomia)	Pivotal/BLA	Pivotal alignment with FDA; enrolling Phase 2 AQUAx2	RMAT granted; potential BLA filing in 2026	De-risked pathway
AAV-GAD (Parkinson’s)	Phase 3	Engage regs on Phase 3 after positive bridging study	JV with Hologen; $200M upfront + up to $230M JV funds to fully finance through commercialization	Accelerated/funded
Manufacturing licensure	UK/Ireland	GMP & QC operations; UK inspection May 2024	HPRA renewals + added viral vector manufacturing to MIA(IMP) in Ireland; UK targeting commercial MIA submission Q2 2025	Strengthened
IRD programs (AAV-AIPL1 and others)	Regulatory path	MHRA exceptional circumstances filing without further studies; RPDDs	Lancet publication; confirm MAA intent; additional RPDDs and expedited CMC alignment	Maintained/expanded

Earnings Call Themes & Trends

Note: Transcript not available via tools; themes derived from company press releases and 8-Ks.

Topic	Previous Mentions (Q2 2024)	Previous Mentions (Q3 2024)	Current Period (Q4 2024)	Trend
AI/CNS initiatives	Riboswitch platform progress; obesity/metabolic and CAR-T R&D plans	Positive AAV-GAD bridging data; Phase 3 planning	Hologen AI JV; $200M upfront; AI to de-risk AAV-GAD Phase 3	Acceleration via funding/AI
Manufacturing	End-to-end in-house capabilities; GMP facilities	Continued GMP/QC operations	UK inspection supports commercial MIA app; HPRA adds viral vector manufacturing in Ireland	Licensure expanded
Regulatory/legal	FDA alignment (AQUAx2 pivotal); ILAP Innovation Passport (AIPL1)	RPDDs for AIPL1, BBS10, RDH12; MHRA path without further studies	RMAT granted (AQP1); MAA submission intent; more expedited pathways	De-risking continues
Product performance	Q2: advance xerostomia, bota-vec milestones	AAV-GAD efficacy; IRD broad efficacy (11/11)	IRD efficacy published in Lancet; Parkinson’s efficacy reiterated	Strengthening clinical signals
Financing/runway	$50M offering led by Sanofi	Runway into Q2 2026	Runway into 2027 with Hologen; milestone potential (bota-vec up to $285M)	Extended runway

Management Commentary

“This extraordinary progress has continued in 2025 with today’s announcement of a strategic collaboration with Hologen which includes a $200 million cash upfront payment... with an additional $230 million committed capital” .
“The use of Hologen’s AI technology applied to MeiraGTx’s Phase 2 clinical data sets in Parkinson’s disease has already significantly de-risked the AAV-GAD program” .
“AAV2-hAQP1 was granted Regenerative Medicine Advanced Therapy (RMAT) designation... reinforcing the strength of our clinical data... [we] anticipate data... to support a potential BLA filing in 2026” .
“LCA4... results from these 11 young children are truly remarkable... we anticipate filing... MHRA for approval under exceptional circumstances, and taking a parallel path with the FDA this year” .

Q&A Highlights

An earnings call transcript for Q4 2024 was not available via our document tools; details on analyst Q&A could not be verified. Themes above reflect prepared remarks and press releases .

Estimates Context

S&P Global consensus data could not be retrieved at this time due to request limits; therefore, official Wall Street consensus comparisons from S&P Global are unavailable.
External aggregator data indicates: Revenue $21.39M vs $1.50M expected (Beat +$19.89M); EPS -$0.50 vs -$0.48 expected (Miss -$0.02) .
Given the revenue beat alongside an EPS miss, estimates may need upward revision for near-term revenue recognition from PPQ services while reassessing operating expense intensity ahead of pivotal trials .

Key Takeaways for Investors

The Hologen AI collaboration is transformative: $200M upfront and up to $230M JV capital fully funds AAV-GAD through commercialization, significantly de-risking CNS execution while preserving a 30% JV stake and exclusive manufacturing ties .
Xerostomia program de-risked with RMAT designation and pivotal alignment; BLA targeted for 2026 elevates near- to mid-term value inflection potential .
IRD programs demonstrate exceptional efficacy with expedited MHRA path; multiple RPDDs add potential PRV monetization optionality .
Manufacturing moat strengthened via UK and Ireland licensure expansions, supporting clinical and potential commercial supply scalability .
Q4 revenue step-up from PPQ services suggests near-term revenue cadence can be lumpy but material; monitor sustainability and conversion of manufacturing/commercial supply agreements into recurring revenue .
Despite revenue beat, operating losses remain significant; watch cash runway extension contingent on deal closing and milestone timing (bota-vec up to $285M) .
Trading: near term, positive catalysts include Hologen deal closing and regulatory milestones; medium term thesis hinges on pivotal xerostomia data/BLA progress, AAV-GAD Phase 3 trial execution, and manufacturing commercialization ramp .

Notes:

Q4 2024 revenue ($21.39M) and net loss ($39.399M) are derived from FY 2024 results minus nine months ended Sep 30, 2024 results .
S&P Global estimates were unavailable due to system request limits at time of analysis; external aggregator data cited for directional context .

MeiraGTx Holdings plc (MGTX)·Q4 2024 Earnings Summary