Executive Summary

Q2 2024 reflected regulatory momentum for govorestat (AT‑007) with a tentative FDA Advisory Committee on Oct 9, 2024, and PDUFA target action date Nov 28, 2024; EMA MAA decision now expected in early Q1 2025, a timing shift vs prior expectations .
Financially, revenue was $0.144M and GAAP net income was $2.9M; diluted EPS was $(0.13), with the swing to profitability driven by a $22.7M non-cash gain from the change in fair value of warrant liabilities—not operations .
Operating expenses were elevated on commercialization prep; G&A rose to $10.6M (+$5.3M YoY) while R&D fell to $10.0M (-$1.9M YoY) .
Cash and equivalents were $122.2M at quarter-end; management reiterated cash runway into 2026, with potential PRV sale upon approval as a further runway extender .
Catalysts: GeMDAC meeting (Oct), PDUFA (Nov), EMA decision (early Q1’25), and SORD Deficiency NDA alignment for accelerated approval, with potential sNDA filing early Q1’25 .

What Went Well and What Went Wrong

Govorestat regulatory progress: “Momentum continues with our steady regulatory progress in Classic Galactosemia and SORD Deficiency” and alignment with FDA Neurology I on accelerated approval pathway for SORD Deficiency NDA (pre‑NDA in H2’24; submission early Q1’25) .
Updated cognition data: Pediatric formula correction improved cognition by ~8 points vs placebo (p=0.032) and led to a statistically significant effect in sensitivity analysis including cognition (p=0.034), strengthening the NDA/MAA packages .
Inclusion in Russell 3000 index, broadening investor exposure during a pivotal regulatory period .

Top-line remains de minimis: Q2 revenue was $0.144M, underscoring lack of commercial product revenue pre-approval .
G&A inflation ahead of launch: G&A rose to $10.6M (+$5.3M YoY), driven by legal/professional fees (+$1.3M), commercialization prep (+$3.5M), and personnel (+$1.1M) .
EMA timeline pushed: EMA decision moved to early Q1 2025 following a 3‑month extension to the Day 120 clock stop—later than initial 4Q 2024 expectations .

Metric	Q2 2023	Q1 2024	Q2 2024
Revenue ($USD Millions)	$0.334	$0.190	$0.144
Net Income ($USD Millions)	$(29.577)	$(83.938)	$2.898
Diluted EPS ($USD)	$(0.37)	$(0.67)	$(0.13)
Total Costs & Expenses ($USD Millions)	$17.176	$21.283	$20.584
Net Income Margin %	−8,856% (calc from )	−44,178% (calc from )	2,012% (calc from )

Note: Net income margin is computed as Net Income / Total Revenue using reported figures.

KPIs and Operating Drivers

KPI	Q2 2023	Q1 2024	Q2 2024
Cash & Cash Equivalents ($USD Millions)	$49.9 (FY 2023 YE)	$146.5	$122.2
R&D Expense ($USD Millions)	$11.883	$12.217	$10.004
G&A Expense ($USD Millions)	$5.293	$9.066	$10.580
Change in Warrant Liabilities ($USD Millions)	$(12.804)	$(63.405)	$22.744

Segment breakdown: Not applicable (no commercial segments; pre-approval revenue primarily research services) .

Metric	Period	Previous Guidance	Current Guidance	Change
Govorestat PDUFA (US)	Classic Galactosemia	Nov 28, 2024	Nov 28, 2024	Maintained
EMA MAA decision (EU)	Classic Galactosemia	4Q 2024	Early Q1 2025	Delayed
FDA Advisory Committee	Classic Galactosemia	FDA planned to hold AdCom (no date)	Tentatively Oct 9, 2024	Firmed timing
SORD Deficiency NDA plan	SORD Deficiency	Discussing potential NDA with FDA	Aligned on accelerated approval; expect sNDA early Q1 2025	Advanced
Cash runway	Corporate	Into 2026	Into 2026	Maintained

Note: A Q2 2024 earnings call transcript could not be located; themes reflect management disclosures across filings/press releases.

Topic	Previous Mentions (Q4 2023 and Q1 2024)	Current Period (Q2 2024)	Trend
Regulatory – Galactosemia (US)	NDA accepted; Priority Review; initial PDUFA Aug 28, 2024	PDUFA Nov 28, 2024; GeMDAC tentatively Oct 9, 2024; cognition data updated strengthens NDA	Positive momentum with clarified timeline
Regulatory – Galactosemia (EU)	EMA MAA validated; decision expected 4Q 2024	Decision expected early Q1 2025 after 3‑month extension	Timing pushed
SORD Deficiency (US)	12‑month interim INSPIRE data; plan to request pre‑NDA meeting	FDA alignment on accelerated approval; expect sNDA early Q1 2025	Advancing toward submission
Commercialization prep	Strengthened leadership; $100M private placement to fund launch	G&A +$5.3M YoY, $3.5M tied to commercialization	Increasing investment
Cash runway/financing	Runway into 2026; proceeds support programs	Runway into 2026; PRV sale could extend	Stable with potential extension
R&D execution (AT‑001 DbCM)	ARISE‑HF data presented; safety/tolerability; subgroup benefit	No new AT‑001 clinical updates in Q2 PR	Neutral
Patient advocacy engagement	—	Multiple HNF/CMTA/PNS/Galactosemia meetings	Increasing outreach

“Momentum continues with our steady regulatory progress in Classic Galactosemia and SORD Deficiency. We are incredibly pleased to share our alignment with the Neurology Division of the FDA regarding a potential second NDA submission for govorestat for the treatment of SORD Deficiency.” — Shoshana Shendelman, PhD, Founder & CEO .
The company emphasized statistically significant improvements in cognition after correcting NIH Toolbox scoring to pediatric formulas, strengthening the NDA/MAA review packages .
Commercial readiness efforts are underway ahead of potential approval, contributing to higher G&A expense in the quarter .

An earnings call transcript for Q2 2024 was not available in company materials or common transcript repositories at the time of review; therefore, Q&A highlights and any additional guidance clarifications from a live call are unavailable [ListDocuments result; InternetSearch review].

S&P Global consensus estimates were not retrievable due to data access limits at the time of this analysis; formal consensus comparison is therefore unavailable.
Third‑party media indicated revenue of $0.144M vs an estimated ~$2.50M, implying a miss on top‑line; use caution as this is not S&P consensus .
Where estimates may need to adjust: near‑term revenue expectations should reflect pre‑approval status and minimal services revenue until a commercial launch; EPS sensitivity remains high due to non‑cash warrant liability fair value changes .

Regulatory catalysts are near‑term and binary: GeMDAC (Oct 9) and PDUFA (Nov 28) for Classic Galactosemia—stock likely to be highly sensitive to AdCom tone and vote .
EMA decision pushed to early Q1’25; EU timing risk remains but updated cognition data could support a favorable view .
The Q2 profit is non‑operational; a $22.7M warrant fair value gain drove GAAP net income—expect EPS volatility absent product revenue .
Elevated G&A tied to commercialization is intentional; monitor opex discipline vs launch preparedness into potential approval window .
Cash runway into 2026 provides strategic flexibility; PRV monetization (upon approval) could extend runway further—watch execution and pricing of PRV sale .
SORD Deficiency program is advancing on an accelerated path with an expected sNDA early Q1’25; this diversifies regulatory optionality independent of Galactosemia .
Trading implications: expect event‑driven volatility into AdCom/PDUFA; positioning may hinge on perceived AdCom briefing materials, panel composition, and the strength of updated cognition data .