Executive Summary

Q4 revenue of $0.638M vs Wall Street consensus of $4.275M* — bold miss driven by the wind-down of Prevail and lower Novartis revenue recognition .
Q4 EPS of $(2.22) vs consensus of $(1.96)* — bold miss on lower top line and higher PBGENE-HBV program costs as the study entered Phase 1 .
Clinical execution advanced: first PBGENE-HBV patient cohort dosed with early antiviral activity; U.S. IND cleared, expanding ELIMINATE-B sites to the U.S., Moldova, Hong Kong, and New Zealand .
Cash runway guidance maintained: into H2 2026, supported by disciplined OpEx, partner cash inflows, and ATM facility availability .

What Went Well and What Went Wrong

“We have already completed dosing of the first patient cohort establishing both safety and early efficacy for PBGENE-HBV at the lowest dose level” — CEO Michael Amoroso, highlighting clinical momentum and operational execution .
U.S. FDA IND clearance for PBGENE-HBV; expanded Phase 1 ELIMINATE-B sites globally (U.S., Moldova, Hong Kong, New Zealand), accelerating access and enrollment .
First in vivo clinical validation across two ARCUS programs: PBGENE-HBV (antiviral activity, HBsAg reduction) and partner iECURE’s ECUR-506 (complete clinical response in severe neonatal OTC deficiency) .

Top-line miss: Q4 revenue fell to $0.638M vs $7.038M YoY, primarily due to conclusion of the Prevail collaboration and lower Novartis revenue as preclinical work neared completion .
EPS/Net loss widened vs prior quarter: Q4 net loss from continuing operations was $(17.745)M vs $(16.425)M in Q3; R&D rose with PBGENE-HBV entering clinic; G&A up on employee and share-based comp .
Limited recurring revenue visibility near term as collaboration revenues step down; dependence on clinical milestones and partner cash inflows per company update .

Metric	Q4 2023	Q2 2024	Q3 2024	Q4 2024
Revenue ($USD Thousands)	7,038	49,898	576	638
Research & Development ($USD Thousands)	13,389	17,225	13,084	15,907
General & Administrative ($USD Thousands)	8,539	8,527	8,767	9,577
Net Income (Loss) from Continuing Ops ($USD Thousands)	(13,434)	32,749	(16,425)	(17,745)
Basic EPS ($USD)	(4.06)	4.70	(2.25)	(2.22)

Metric	Q2 2024	Q3 2024	Q4 2024
Operating Income (Loss) ($USD Thousands)	24,146	(21,275)	(24,846)
Gain on Change in Fair Value of Warrant Liability ($USD Thousands)	7,765	3,647	7,812
Interest Income ($USD Thousands)	1,843	1,763	1,494

Metric	Q2 2024	Q3 2024	Q4 2024
Cash, Cash Equivalents, Restricted Cash ($USD Thousands)	123,571	121,328	108,468
Working Capital ($USD Thousands)	111,078	98,541	80,009
Total Stockholders’ Equity ($USD Thousands)	74,714	64,866	56,393
Common Shares Outstanding (Period End)	7,123,370	7,480,521	8,202,715

Metric	Period	Previous Guidance	Current Guidance	Change
Cash Runway	Company-level	“Into second half of 2026” (Q2, Q3 2024)	“Into second half of 2026” (Q4 2024)	Maintained
PBGENE-HBV IND Status (U.S.)	Program	U.S. IND anticipated in 2025 (Dec-2024)	U.S. IND cleared (Mar-2025)	Raised/Achieved
PBGENE-HBV CTA Approvals	Program	Moldova CTA approved; screening underway (Q3 2024)	IND/CTAs cleared in U.S., Moldova, Hong Kong, New Zealand (Q4 2024)	Raised
PBGENE-3243 IND/CTA Timing	Program	IND/CTA planned in 2025 (Q2/Q3 2024)	Advance toward CTA/IND with new data in 2025 (Q4 2024)	Maintained

Note: No Q4 2024 earnings call transcript available in the document set; thematic tracking uses company press releases/8-Ks.

Topic	Previous Mentions (Q-2 and Q-1)	Current Period (Q4 2024)	Trend
HBV clinical execution	IND/CTA for PBGENE-HBV planned; CTA cleared in Moldova; screening underway	First cohort dosed; early HBsAg reduction; U.S. IND cleared; sites added in HK/NZ/U.S.	Accelerating
Regulatory expansion	CTA Moldova; Hong Kong approval pending	IND/CTA footprint expands to U.S., HK, NZ	Broadening
ARCUS platform validation	Preclinical safety/efficacy in HBV and mitochondria; high-efficiency insertion via HDR	Clinical validation in HBV and OTC; continued data in 2025	Strengthening
Cash runway/OpEx discipline	Runway into H2 2026; lower G&A YoY in Q2	Runway maintained; spend rising with clinical start	Stable runway; higher R&D
Partnerships (TG Therapeutics, Novartis, iECURE)	TG deal closed; Novartis workplan nearing completion; iECURE fast track	TG equity investment; iECURE neonatal OTC complete clinical response; Novartis workplan step-down	Mixed (cash inflows; lower near-term collab revenue)

“2024 was a transformational year… we now have clinical data from two differentiated ARCUS based programs… PBGENE-HBV is the first-ever investigational in vivo gene editing therapy cleared to enter clinical trials for the treatment of chronic Hepatitis B in the U.S. and globally.” — Michael Amoroso, President & CEO .
“Given PBGENE-HBV’s novel modality, these data suggest that PBGENE-HBV appears to be working by its intended mechanism of eliminating the source of viral replication in cccDNA while inactivating integrated disease.” — Cassie Gorsuch, PhD, Chief Scientific Officer .
“We look forward to accelerating patient access to the study by initiating the trial in the U.S. and later expanding to the U.K. Clinical data updates will continue to be shared throughout 2025 at meaningful timepoints.” — Michael Amoroso .

No Q4 2024 earnings call transcript was available in the document set; the company communicated via press releases and 8-K filings .

Metric	Q4 2024 Actual	Q4 2024 Consensus*	Surprise
Revenue ($USD Millions)	0.638	4.275*	Miss
Primary EPS ($USD)	(2.22)	(1.96)*	Miss

Values retrieved from S&P Global.*

Result drivers: revenue shortfall reflects reduced recognition under Prevail (collaboration concluded) and lower Novartis revenue as preclinical work nears completion . Higher R&D for PBGENE-HBV entering clinic and increased G&A contributed to the EPS miss .

Near-term catalyst stack: ongoing ELIMINATE-B dosing (multiple administrations per cohort), expansion to higher dose levels, and periodic clinical data updates through 2025 .
Regulatory momentum de-risks execution: U.S. IND clearance plus multiple global sites (Moldova, Hong Kong, New Zealand) should accelerate enrollment and data cadence .
Platform validation: clinical signals in HBV and iECURE’s OTC program reinforce ARCUS differentiation across elimination and insertion use cases .
Financials: collaboration revenue step-down creates near-term top-line volatility; watch partner milestones (TG Therapeutics, Novartis) and ATM usage given runway guidance to H2 2026 .
Trading lens: stock likely sensitive to HBV HBsAg trajectories and safety at ascending dose levels; strong data could be a re-rating event, while delays or safety flags would be negative.
Medium-term thesis: success in HBV would be a major proof point for viral genome elimination; PBGENE-3243 IND/CTA in 2025 adds optionality in mitochondrial disease .

Q3 2024: Moldova CTA approval; screening underway; cash/restricted cash $121.3M; R&D $13.1M; G&A $8.8M; net loss from continuing ops $(16.4)M .
Q2 2024: revenue $49.9M (Prevail termination deferred revenue recognition), net income from continuing ops $32.7M with $7.8M non-cash warrant liability gain; cash $123.6M .