Executive Summary

Q1 2024 was an execution-heavy quarter: NGN-401 Phase 1/2 safety profile remained favorable with no serious AEs or signs of MeCP2 overexpression toxicity, trial expansion to a high-dose cohort was confirmed, and Australia HREC approval added a third region for the study .
Financially, OpEx rose year over year (R&D +31% to $13.5M; G&A +86% to $5.2M) and net loss widened to $16.9M; cash, cash equivalents and investments were $169.5M with runway into 2H:26, supporting multiple upcoming data readouts .
Guidance/milestones maintained: interim NGN-401 efficacy data (Cohort 1) remains targeted for 4Q:24; additional interim data from expanded low and high dose cohorts in 2H:25; NGN-101 interim clinical and regulatory update remains planned for 2H:24 .
Estimates context: S&P Global Wall Street consensus for Q1 2024 was unavailable for NLTX/NGNE; result comparisons to Street cannot be provided.
Near-term catalysts: 4Q:24 NGN-401 interim efficacy data and 2H:24 NGN-101 interim data/regulatory updates; Australia site activation may accelerate enrollment, while continued demonstration of safety is key to de-risking the platform .

What Went Well and What Went Wrong

What Went Well

Safety maintained: NGN-401 generally well-tolerated in first three pediatric patients with ~9, 6 and 3 months follow-up; all AEs mild/Grade 1; no procedure-related SAEs; no MeCP2 overexpression toxicity signs .
Trial momentum: acknowledgment from Australian TGA and approval from HREC expand the NGN-401 trial to a third region; concurrent enrollment planned with expanded Cohort 1 and high-dose Cohort 2 .
Balance sheet strength: $169.5M cash, cash equivalents and investments as of 3/31/24; expected runway into 2H:26 supports operations beyond critical clinical inflection points .
Quote: “We remain on track to release interim efficacy data from the low-dose cohort in the fourth quarter of 2024.” — Rachel McMinn, Ph.D., CEO .

What Went Wrong

Higher OpEx and widening net loss: R&D up 31% YoY to $13.5M; G&A up 86% to $5.2M; net loss widened to $16.9M, mainly on NGN-401 clinical costs, early discovery program preclinical work, and public company costs .
Sequential comparability distortion: Q4 2023 included a one-time $16.4M bargain purchase gain, producing quarterly net income of $2.4M; this affects sequential loss comparisons to Q1 2024 .
No earnings call transcript for Q1 2024 available; therefore no Q&A or call-based color or guidance clarifications could be assessed (press release only) [List: earnings-call-transcript (May 2024) not found].

Financial Results

Year-over-Year Comparison (Q1 2023 → Q1 2024)

Metric	Q1 2023	Q1 2024
R&D Expense ($USD Millions)	$10.3	$13.5
G&A Expense ($USD Millions)	$2.8	$5.2
Total Operating Expenses ($USD Millions)	$13.0	$18.8
Loss from Operations ($USD Millions)	$(13.0)	$(18.8)
Other Income, net ($USD Millions)	$0.8	$1.9
Net Loss ($USD Millions)	$(12.3)	$(16.9)
EPS (Basic & Diluted, $)	$(28.28)	$(1.00)
Weighted Avg Shares (Basic & Diluted)	433,623	16,903,735
Cash, Cash Equivalents & Investments ($USD Millions)	N/A	$169.5

Notes: Q1 2023 EPS reflects pre-merger share structure; Q1 2024 reflects post-merger common stock count .

Sequential Comparison (Q4 2023 → Q1 2024)

Metric	Q4 2023	Q1 2024
R&D Expense ($USD Millions)	$12.2	$13.5
G&A Expense ($USD Millions)	$2.5	$5.2
Net Income (Loss) ($USD Millions)	$2.4 (incl. $16.4M bargain purchase gain)	$(16.9)
Cash, Cash Equivalents ($USD Millions)	$148.2 (cash & cash equivalents at 12/31/23)	$150.1 (cash & cash equivalents at 3/31/24)
Total Assets ($USD Millions)	$222.6	$195.4
Stockholders’ Equity ($USD Millions)	$186.0	$170.8

Notes: Q4 2023 net income driven by one-time bargain purchase gain; comparability to Q1 2024 operating performance is limited .

KPIs (Operational)

KPI	Q3 2023	Q4 2023	Q1 2024
NGN-401 patients dosed (cumulative)	First patient dosed	Second patient dosed	Third patient dosed
NGN-401 safety signals	No serious AEs; no overexpression toxicity	Same	Same; all AEs mild/Grade 1
NGN-401 trial geographies	US initial	UK approvals in place	Australia HREC approval (third region)
NGN-401 key timeline	Cohort 1 dosing ongoing	Cohort 1 expansion; Cohort 2 added	Cohort 1 completion 2H:24; Cohort 2 start 2Q:24
NGN-401 interim data	—	—	4Q:24 (Cohort 1)
NGN-101 status	—	Cohort 3 (high-dose) enrolling	Interim clinical data + regulatory update 2H:24

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
NGN-401 interim efficacy (Cohort 1)	4Q:24	4Q:24	4Q:24	Maintained
NGN-401 additional interim data (expanded low/high dose cohorts)	2H:25	2H:25	2H:25	Maintained
NGN-401 Cohort 1 enrollment completion	2H:24	2H:24	2H:24	Maintained
NGN-401 Cohort 2 enrollment start	2Q:24	2Q:24	2Q:24	Maintained
NGN-401 geographies	2024	US+UK	US+UK+Australia (HREC approval)	Raised (scope)
NGN-101 interim data & regulatory update	2H:24	2H:24	2H:24	Maintained
Cash runway	Through 2H:26	2H:26	2H:26	Maintained

Earnings Call Themes & Trends

No Q1 2024 earnings call transcript available; themes below reflect press releases and corporate presentation.

Topic	Previous Mentions (Q-2: Q3 2023)	Previous Mentions (Q-1: Q4 2023)	Current Period (Q1 2024)	Trend
R&D execution (NGN-401 dosing)	First patient dosed	Third patient dosing early Q1’24; removal of stagger in Cohort 1	Safety favorable; ongoing dosing; Cohort 2 planned	Improving execution; consistent safety
Regulatory/geographies	—	UK MHRA approval, onboarding sites	Australia HREC approval (third region)	Expanding footprint
Trial design/expansion	—	Expanded low-dose Cohort 1 + added high-dose Cohort 2	Expect interim data Cohort 1 in 4Q:24; Cohort 2 starts 2Q:24	Broadened dataset plan
Manufacturing capability	—	Houston facility; CGMP production supports pivotal dev	Internal manufacturing flexibility highlighted	Strategic asset emphasized
Platform/regulation (EXACT)	—	EXACT supports constrained MeCP2 expression	EXACT presented; preclinical translational foundation summarized	Reinforced technology thesis
Cash runway/funding	—	Through 2H:26 with reverse merger financing	Maintained 2H:26 runway	Stable funding outlook

Management Commentary

“We have made substantial progress in our NGN-401 Rett syndrome gene therapy program since the beginning of the year, including dosing the third patient, expanding the trial to include additional patients and a high-dose cohort, and the recent clearance to conduct the trial in Australia.” — Rachel McMinn, Ph.D., CEO .
“We remain on track to release interim efficacy data from the low-dose cohort in the fourth quarter of 2024… We remain in a strong financial position with cash runway into the second half of 2026.” — Rachel McMinn, Ph.D., CEO .
“We started the year with strong execution… expanded the trial and added a high-dose cohort… NGN-401 has been well-tolerated… with no signs of overexpression-related toxicity.” — Q4/FY commentary (context for trajectory) .

Q&A Highlights

No Q1 2024 earnings call transcript was available, so Q&A themes and any guidance clarifications could not be assessed [List: earnings-call-transcript (May 2024) not found].

Estimates Context

S&P Global Wall Street consensus estimates for Q1 2024 (EPS and revenue) were unavailable for NLTX/NGNE due to data mapping constraints; as a result, comparison to consensus cannot be provided.

Key Takeaways for Investors

Safety profile continues to de-risk NGN-401 and the EXACT transgene regulation platform, with no SAEs and no overexpression toxicity reported to date; a key validator ahead of efficacy readouts .
Expanded trial design (higher-dose cohort, more patients, broader geographies) is poised to produce a more complete dataset to inform registrational strategy; watch for timing and enrollment pace .
Cash runway into 2H:26 provides line of sight through multiple program catalysts (NGN-401 interim efficacy in 4Q:24; NGN-101 interim clinical/regulatory update in 2H:24), reducing near-term financing overhang risk .
Near-term trading setup hinges on 4Q:24 NGN-401 efficacy signals; any clinical improvement across hand function, communication, autonomic measures (CGI-I/RSBQ domains) could materially impact sentiment .
Sequential financials are noisy due to Q4 2023’s one-time gain; trend analysis should focus on operating spend and enrollment progress rather than bottom-line comparisons .
With Australia approval and internal manufacturing capacity, operational risk around site activation and supply appears manageable; execution remains the primary driver of trajectory .
Absence of consensus estimates limits “beat/miss” framing; expect Street updates after interim data—position sizing should reflect binary clinical readout risk and platform optionality.

Citations: Press release and exhibits ; Corporate presentation highlights and clinical/milestone detail ; Q4/FY 2023 press release for prior-period context .

Neurogene Inc. (NLTX)·Q1 2024 Earnings Summary