Executive Summary

enGene reported FY 2024 results and a business update; fiscal Q4 (ended Oct 31, 2024) featured strengthened liquidity (cash, cash equivalents and marketable securities of $297.9M, runway into 2027) and progress in its pivotal LEGEND study for NMIBC with CIS, with preliminary pivotal cohort CR of 71% “any time,” 67% at 3 months, and 47% at 6 months; protocol changes aim to better align with real-world practice and potentially improve outcomes .
Operating scale-up continued: FY 2024 OpEx rose to $62.3M (R&D $38.3M; G&A $24.0M), net loss $55.1M (basic/diluted $1.46/share); balance sheet at Oct 31 showed total assets $311.2M and equity $272.6M .
Financing and flexibility improved post-quarter: $60M private placement (Oct 25, 2024) and a $100M at-the-market facility with Jefferies (Dec 20, 2024); Hercules loan amended to reallocate $7.5M into an uncommitted tranche under lender approval, keeping total facility at up to $50M .
Key 2025–2026 catalysts: additional preliminary data from the pivotal cohort in 2H 2025 and planned BLA filing in mid-2026; management reiterated runway into 2027 as a support for execution and pre-commercial readiness .

What Went Well and What Went Wrong

What Went Well

Pivotal LEGEND data showed compelling activity and tolerability: CR “any time” 71%, 3-month CR 67%, 6‑month CR 47% (KM-estimated 51% at 6 months); no discontinuations due to TRAEs; most TRAEs Grade 1/2 .
Strategic protocol enhancements to align with standard of care (e.g., dual resections for T1+CIS; allowing resection and continuation for recurrent Ta at 3 months; biopsy confirmation at discontinuation) and added maintenance dosing to support durability evidence .
Strengthened balance sheet and funding optionality: $297.9M cash/cash equivalents/marketable securities at FY-end (runway into 2027); $60M private placement; $100M ATM; inclusion of LEGEND in SUO-CTC to broaden site access .
Management tone: CEO Ron Cooper emphasized “strong clinical and financial position” and momentum with all cohorts recruiting; “look forward to sharing LEGEND study updates… in the second half of 2025” .

What Went Wrong

Operating expenses rose significantly as the company scaled R&D and G&A for pivotal execution and public-company operations: FY24 OpEx $62.3M vs $26.1M in FY23; R&D +$21.9M; G&A +$14.4M .
No quarterly (Q4-only) P&L detail was provided in the FY update, limiting direct quarter-over-quarter margin analysis for Q4; reported figures were annual with balance-sheet snapshots .
Estimates context: Wall Street consensus via S&P Global was unavailable at time of writing; comparisons vs estimates cannot be made (early-stage, pre-revenue biotech coverage can be limited; API quota constraints) [GetEstimates error noted].
Continued execution risk inherent to registrational single-arm design and need to demonstrate sustained durability (12-month CR primary endpoint) remains, despite positive preliminary data and protocol refinements .

Financial Results

P&L and Cash Position

Metric	FY 2023	FY 2024
Research & Development Expense ($USD Millions)	$16.46	$38.32
General & Administrative Expense ($USD Millions)	$9.60	$23.98
Total Operating Expenses ($USD Millions)	$26.06	$62.30
Net Loss ($USD Millions)	$99.92	$55.14
Net Loss per Share, Basic & Diluted ($)	$151.22	$1.46
Weighted Avg. Shares Outstanding (Basic & Diluted)	0.693M	37.782M

Balance Sheet (Unaudited)	Oct 31, 2023	Oct 31, 2024
Cash and Cash Equivalents ($USD Millions)	$81.52	$173.00
Total Assets ($USD Millions)	$86.96	$311.17
Total Liabilities ($USD Millions)	$14.47	$38.56
Total Shareholders’ Equity ($USD Millions)	$72.49	$272.61

Liquidity (Quarter Snapshots)	Q2 FY2024 (Apr 30, 2024)	Q3 FY2024 (Jul 31, 2024)	Q4 FY2024 (Oct 31, 2024)
Cash & Cash Equivalents ($USD Millions)	$264.81	$257.68	$173.00
Cash, Cash Equivalents & Marketable Securities ($USD Millions)	N/A	N/A	$297.90

Notes: The FY press release disclosed cash, cash equivalents and marketable securities together ($297.9M) alongside balance-sheet cash & equivalents ($173.0M) at Oct 31, 2024; earlier quarter releases disclosed cash & equivalents only .

KPIs (Clinical/Operational)

KPI (LEGEND Pivotal Cohort)	Value
CR “Any Time”	71% (15/21)
CR at 3 Months	67% (14/21)
CR at 6 Months	47% (8/17); 6‑month CR KM estimate 51%
Safety (All Phase 2 Cohorts, n=42)	Any TRAE 47.6%; Grade 3 TRAEs: 2; No Grade 4/5; common TRAEs ≥10%: dysuria, bladder spasm, pollakiuria, fatigue

Segment breakdown: Not applicable (single clinical program focus) .

Guidance Changes

Metric/Topic	Period	Previous Guidance	Current Guidance (Q4 FY2024)	Change
Pivotal LEGEND additional preliminary data	2H 2025	Not previously specified (beyond Sept 2024 data)	Additional preliminary data from pivotal cohort in 2H 2025	New milestone added
BLA Filing (detalimogene in BCG-unresponsive NMIBC with CIS)	Mid-2026	Mid-2026 (Q2/Q3 reiterated)	Mid-2026 maintained	Maintained
Enrollment status	Ongoing	Planned expansions; pause/resume in Cohort 2 revisions	Now enrolling across all four cohorts under updated protocol	Progressed
Maintenance dosing	Year 2+	Not in earlier disclosures	Added: dose-reduced maintenance dosing for CR patients; optional up to 3 years	Expanded protocol
Liquidity runway	Through 2027	Into 2027	Into 2027 reiterated ; supported by $60M private placement (Oct 25, 2024) and $100M ATM (Dec 20, 2024)	Maintained; funding flexibility increased

Earnings Call Themes & Trends

(EN: No Q4 FY2024 earnings call transcript was available; themes derived from Q2/Q3 earnings releases and the Q4 FY2024 press release.)

Topic	Q2 FY2024 (Jun 14)	Q3 FY2024 (Sep 10)	Q4 FY2024 (Dec 19)	Trend
Clinical efficacy (LEGEND pivotal cohort)	Guided to interim data by end of Sept 2024	Preliminary pivotal data expected; reiterated	Highlights 71% “any time” CR; 3m 67%; 6m 47% (per Sept 26 data)	Execution on efficacy disclosure; positive preliminary activity
Protocol alignment to SoC	Planned cohort modifications (add papillary-only, analyze BCG-exposed vs naïve)	Ongoing	Updated protocol adds SoC-aligned elements (dual resections in T1+CIS, management of Ta at 3m), maintenance dosing	Strengthened design, potential durability support
Regulatory timeline	BLA mid-2026	Maintained	Maintained	Consistent
Financing & runway	Cash $264.8M; runway into 2027	Cash $257.7M; runway into 2027	Cash/cash & secs $297.9M; runway into 2027; $60M private placement; $100M ATM	Strengthened liquidity, added flexibility
Leadership/ops	Hired senior urology leader; board changes	New CEO (Ron Cooper); CMO promotion (Pruthi)	CTO appointment; CSO transition	Building late-stage/commercial capabilities

Management Commentary

CEO Ron Cooper: “Having demonstrated a 71% anytime complete response rate … and raised an additional $60 million in funding, enGene is in a strong clinical and financial position as we close the year.… we look forward to sharing LEGEND study updates across all cohorts expected in the second half of 2025.”
CMO Raj Pruthi, M.D.: “We are pleased with this preliminary dataset, which clearly demonstrates that detalimogene is highly active and generally well tolerated.… We are also planning protocol refinements in LEGEND, which we believe will provide patients with additional clinical benefit.”
Liquidity stance: “Cash, cash equivalents and marketable securities of $297.9 million provide runway into 2027.”

Q&A Highlights

No Q4 FY2024 earnings call transcript was available. Management’s nearest public Q&A (Oppenheimer Healthcare Conference, Feb 2025) reiterated:
- Protocol changes intended to strengthen results and align with SoC; additional pivotal update guided for 2H 2025; BLA mid-2026 remains the target .
- Emphasis on ease-of-use and scalable, non-viral manufacturing vs. viral competitors as a commercial differentiator .
  (Conference context provided for color; not a Q4 earnings call.)

Estimates Context

Wall Street consensus (S&P Global) for Q4 FY2024 revenue/EPS was unavailable at time of writing; as a clinical-stage, pre-revenue company, formal consensus can be limited and our SPGI API request could not be fulfilled today (no reliable estimate set retrieved). As a result, we cannot present comparisons vs consensus for Q4 FY2024.

Key Takeaways for Investors

Upcoming catalyst path: Pivotal cohort additional data in 2H 2025 and BLA filing in mid-2026 are the primary stock drivers over the next 12–18 months; narrative hinge: durability at 12 months and safety profile sustainability .
Protocol optimizations (dual resections for T1+CIS, biopsy confirmation, Ta management, and maintenance dosing) could improve durability readouts and reduce false discontinuations—watch for methodology and analysis updates in 2025 disclosures .
Balance sheet strength and flexibility reduce financing overhang: $297.9M cash/cash equivalents/marketable securities, into 2027 runway; $60M private placement executed; $100M ATM adds optionality, though ATM usage could be dilutive if accessed meaningfully ahead of catalysts .
Competitive positioning: Non-viral, clinic-friendly product profile (no cold chain, simplified prep, no biosafety handling or urine bleaching) is a key differentiator vs. viral intravesical options and could matter in community urology adoption if efficacy/durability meet benchmarks .
Execution risks remain: single-arm registrational cohort must meet FDA expectations on 12‑month CR and safety; enrollment pace, site activation (SUO-CTC inclusion positive) and protocol amendments’ impact on outcomes warrant close tracking .
Near-term trading implications: Stock likely to be event-driven around interim program updates, conference commentary, and financing signals (e.g., potential ATM activity). Medium term, 2H 2025 dataset depth and durability will be critical to de-risking the BLA case .

Citations:

FY 2024 press release and business update (8-K):
Q3 FY2024 results (8-K):
Q2 FY2024 results (8-K):
LEGEND pivotal preliminary data (Sept 26, 2024):
$60M private placement (Oct 25, 2024 8-K):
$100M ATM with Jefferies (Dec 20, 2024 8-K):
Executive/operational leadership changes (Oct 21, 2024 8-K):
Oppenheimer conference transcript (Feb 11, 2025, for context):

enGene Holdings Inc. (ENGN)·Q4 2024 Earnings Summary