Executive Summary

Q1 2025 showed accelerated clinical spend as enGene expanded LEGEND trial sites to Europe and Asia and enabled maintenance dosing, driving total operating expenses to $26.6M and widening net loss to $24.6M ($0.48 per share) .
Cash, cash equivalents and marketable securities of $272.8M provide runway into 2027; management reiterated prelim pivotal cohort data in 2H 2025 and BLA filing timing for mid-2026 .
Consensus estimates from S&P Global were unavailable this quarter, so beats/misses versus Street cannot be assessed; increased interest income partially offset the higher operating spend .
Near-term stock catalysts: pivotal cohort enrollment completion and 2H 2025 clinical data updates; medium-term catalyst is the planned mid-2026 BLA filing .

What Went Well and What Went Wrong

Enrollment and global site expansion continued “in-line” with the plan to file a mid-2026 BLA; “we look forward to the remainder of 2025… we expect to announce enrollment completion of LEGEND’s pivotal cohort” (Ron Cooper, CEO) .
Protocol amendments now include maintenance dosing for responders to potentially improve durability while lowering patient burden; all four LEGEND cohorts are open (pivotal CIS, CIS BCG-naïve/exposed, papillary-only) .
Liquidity remains strong with $272.8M in cash and marketable securities and expected runway into 2027, despite increased clinical activity .

Operating expenses rose sharply year over year (to $26.6M from $10.8M), driven by manufacturing and clinical costs and headcount; net loss widened to $24.6M .
Disclosure controls remained ineffective due to unremediated material weaknesses (policy/control design, IT controls, segregation of duties), adding governance risk until remediation completes .
Cash and marketable securities declined versus Oct 31, 2024 ($272.8M vs $297.9M), reflecting operating cash burn and investment reallocations despite higher interest income .

Metric	Q2 2024	Q3 2024	Q1 2025
Revenues ($USD Millions)	$0.0	$0.0	$0.0
Total Operating Expenses ($USD Millions)	$17.31	$16.76	$26.61
Net Loss ($USD Millions)	$14.98	$14.15	$24.62
Diluted EPS ($USD)	$0.38 loss	$0.32 loss	$0.48 loss
Cash & Mkt Securities ($USD Millions, period-end)	$264.81	$257.68	$272.79
Total Assets ($USD Millions, period-end)	$273.53	$266.88	$285.93
Total Shareholders’ Equity ($USD Millions, period-end)	$242.97	$230.33	$249.95
Weighted Avg Shares (Millions)	39.44	44.17	50.98

Segment breakdown:

Single operating segment: research, discovery, and clinical development of genetic medicines .

KPIs

KPI	Q2 2024	Q3 2024	Q1 2025
R&D Expense ($USD Millions)	$9.86	$11.55	$19.97
G&A Expense ($USD Millions)	$7.46	$5.21	$6.64
Interest Income ($USD Millions)	N/A	N/A	$2.73
Net Loss per Share ($USD)	$0.38 loss	$0.32 loss	$0.48 loss
Ending Cash + Mkt Securities ($USD Millions)	$264.81	$257.68	$272.79

Note: Interest income breakout was disclosed in Q1 2025 10-Q; prior quarters reported only “other (income) expense, net” .

Metric	Period	Previous Guidance	Current Guidance	Change
BLA filing for detalimogene (BCG-unresponsive NMIBC with CIS)	Mid-2026	On track for mid-2026 (Dec 2024)	On track for mid-2026 (Mar 2025)	Maintained
LEGEND pivotal cohort preliminary data	2H 2025	Additional prelim data anticipated in 2H 2025 (Dec 2024)	Additional prelim data anticipated in 2H 2025 (Mar 2025)	Maintained
Pivotal cohort enrollment	2025	Cohorts open; protocol enhancements in place (Dec 2024)	Expect announcement of pivotal cohort enrollment completion in 2025 (Mar 2025)	Clarified timeline (toward completion)
Trial site geography	Ongoing	Active sites in USA/Canada; SUO-CTC inclusion (Dec 2024)	Expanded sites to Europe and Asia (Mar 2025)	Raised (broader footprint)
Maintenance dosing for responders	Protocol	Introduced under amended protocol (Dec 2024)	Implemented (patients may continue maintenance up to 3 years) (Mar 2025)	Implemented/maintained
Cash runway	Through 2027	$297.9M cash & securities; runway into 2027 (Dec 2024)	$272.8M cash & securities; runway into 2027 (Mar 2025)	Maintained (lower balance)

Note: No Q1 2025 earnings call transcript found; themes tracked via press releases and filings .

Topic	Previous Mentions (Q2 2024 and Q3 2024)	Current Period (Q1 2025)	Trend
Clinical site expansion	Preparing expansions; cohorts modifications planned (Q2)	Sites expanded into Europe and Asia; all cohorts open (Q1)	Expanding
Maintenance dosing	Introduced in protocol (Q4 2024)	Implemented for CR patients up to 3 years (Q1)	Implemented
Pivotal data timing	Interim/prelim data expected Sept 2024 (Q3)	Additional prelim pivotal data expected 2H 2025 (Q1)	Reiterated longer-term cadence
BLA timeline	Target mid-2026 (Q2/Q3/Q4)	Mid-2026 reiterated (Q1)	Stable
Liquidity/cash runway	~$265–258M; runway into 2027 (Q2/Q3)	$272.8M; runway into 2027 (Q1)	Stable
Platform/manufacturing	Non-viral DDX enables scalable manufacturing, low COGS (Q2)	Continued emphasis on tolerability, ease-of-use, efficacy profile (Q1)	Reaffirmed

“With the expansion of LEGEND study sites into Europe and Asia, enrollment in our pivotal cohort continues to track in-line with our plan to file a BLA for detalimogene in mid-2026.” — Ron Cooper, CEO .
“We continue to believe in detalimogene’s opportunity to transform the treatment landscape of NMIBC through a unique and differentiated overall profile… tolerability, ease-of-use, and efficacy.” — Ron Cooper, CEO .
Prior context: “Having demonstrated a 71% anytime complete response rate… enGene is in a strong clinical and financial position as we close the year.” — Ron Cooper (Dec 2024) .

No Q1 2025 earnings call transcript was available; the company furnished an 8-K 2.02 with Exhibit 99.1 press release instead .

Wall Street consensus EPS and revenue estimates from S&P Global were unavailable for Q1 2025; as a result, we cannot assess beats/misses versus Street. Where estimates are shown in other contexts, values would be retrieved from S&P Global.*

Clinical momentum: global site expansion and maintenance dosing implementation support a robust pivotal program; pivotal cohort enrollment completion and 2H 2025 data are key trading catalysts .
Liquidity: $272.8M in cash and marketable securities and runway into 2027 provide funding visibility through pivotal activities and pre-commercial workstreams .
Spend trajectory: R&D climbed to $19.97M in Q1 as LEGEND manufacturing and clinical operations scaled; expect elevated OpEx ahead of BLA preparation .
Partial offset: higher interest income ($2.73M) mitigated some loss expansion amid cash/marketable securities deployment .
Governance/watch items: material weaknesses in internal controls remain; monitor remediation progress to reduce execution and reporting risk .
Additional capital flexibility: $27.5M remains available under the Hercules uncommitted tranche (subject to conditions), and a $100M ATM is in place; monitor potential usage and dilution .
Narrative drivers: durability data under maintenance, cohort updates (CIS naïve/exposed and papillary-only), and mid-2026 BLA timeline framing longer-term thesis .

*Values retrieved from S&P Global.