Executive Summary

Q2 delivered a sharp top-line step-up on a Lilly capsid upfront, with revenue of $18.31M and net loss of $(19.99)M (EPS $(0.08)); OpEx remained disciplined (GAAP $36.16M; non-GAAP $33.0M). Management reiterated FY25 OpEx guidance and said cash (June 30: $38.34M) plus ATM proceeds fund into 4Q25 .
Clinical momentum continued: FDA alignment that 52-week mean eGFR slope can serve as the primary basis for Accelerated Approval in Fabry; ST-920 showed positive mean eGFR slopes at 52 and 104 weeks and favorable safety; all ERT patients were able to withdraw (one resumed ERT post-cutoff) .
Against S&P Global consensus, Q2 missed on revenue ($18.31M vs $31.68M*) and EPS (−$0.08 vs −$0.04*); consensus also expected positive EBITDA ($7.8M*) vs reported EBITDA of approximately −$16.86M*; expect estimates to adjust lower on partnership timing and revenue cadence uncertainty. Values retrieved from S&P Global. *
Catalysts: detailed STAAR data (Sept 2025), potential Fabry commercialization agreement, first dosing in the ST-503 pain program in fall 2025, and a potential Fabry BLA as early as Q1 2026—each likely to drive sentiment and funding visibility .

What Went Well and What Went Wrong

What Went Well
- FDA alignment on Accelerated Approval pathway using 52-week mean eGFR; CEO: “we have no expectation that the agency will require anything other than the one year data for accelerated approval” .
- Fabry STAAR data strengthened: mean eGFR slope +1.965 at 52 weeks (n=32) and +1.747 at 104 weeks (n=19); cardiac measures broadly stable; favorable safety; durable α‑Gal A expression up to 4.5 years .
- Business development and funding progress: ~$21M net from equity offering; Q2 revenue uplift driven by Lilly capsid license upfront; reiterated lean OpEx plan .
What Went Wrong
- Financial miss vs consensus: revenue and EPS below street; EBITDA materially below consensus positive expectation, underscoring reliance on episodic BD income and ongoing OpEx needs. Values retrieved from S&P Global. *
- Cash runway remains short: cash $38.34M at 6/30; management expects runway into 4Q25, heightening urgency for a Fabry commercialization deal or additional financing .
- One ERT-withdrawn patient resumed ERT after the cutoff (others remain off), a datapoint bears monitoring in broader adoption discussions despite overall favorable renal and QoL signals .

Financial Results

Overall quarterly P&L and liquidity (oldest → newest):

Metric	Q4 2024	Q1 2025	Q2 2025
Revenue ($USD Millions)	$7.55	$6.44	$18.31
Net Loss ($USD Millions)	$(23.40)	$(30.60)	$(19.99)
Diluted EPS ($)	$(0.11)	$(0.14)	$(0.08)
GAAP Operating Expenses ($USD Millions)	$33.54	$36.07	$36.16
Non-GAAP Operating Expenses ($USD Millions)	$29.0	$32.5	$33.0
Cash & Cash Equivalents (period-end, $USD Millions)	$41.92 (12/31/24)	$25.18 (3/31/25)	$38.34 (6/30/25)

Versus Wall Street consensus (S&P Global) for Q2 2025:

Metric	Actual	Consensus	Beat/Miss
Revenue ($USD Millions)	$18.31	$31.68*	Miss
Diluted EPS ($)	$(0.08)	$(0.04)*	Miss
EBITDA ($USD Millions)	≈$(16.86)*	$7.80*	Miss

Values retrieved from S&P Global for consensus and EBITDA (asterisk-marked values).

KPIs (clinical and operating):

KPI	Q4 2024	Q1 2025	Q2 2025
Fabry STAAR mean eGFR slope at 52 weeks	+3.061 (n=23)	Positive trend maintained across 32 pts (preliminary)	+1.965 (n=32); +1.747 at 104 weeks (n=19)
ERT withdrawal among ERT starters	All 18 off ERT	All ERT pts off as of milestone	All 18 off at cutoff; 1 patient resumed post-cutoff
Safety (ST-920)	Favorable safety profile	Favorable safety profile	Favorable; most AEs grade 1–2; no discontinuations/deaths
Cash runway	—	Into late Q3’25 (incl. BD and offering expectations)	Into 4Q’25 incl. ATM proceeds

Segment breakdown: Not applicable (no reportable operating segments disclosed) .

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
GAAP Total Operating Expenses	FY 2025	$135–$155M (3/17/25)	$135–$155M (8/7/25)	Maintained
Non-GAAP Total Operating Expenses	FY 2025	$125–$145M; excl. ~$7M SBC and ~$3M D&A (3/17/25)	$125–$145M; excl. ~$7M SBC and ~$3M D&A (8/7/25)	Maintained

No other quantitative guidance (e.g., revenue, margins, tax) was provided or updated .

Earnings Call Themes & Trends

Topic	Previous Mentions (Q-2 and Q-1)	Current Period (Q2 2025)	Trend
Fabry regulatory path	FDA aligned on Accelerated Approval; 52-week eGFR across all patients to serve as basis; CMC path clarified in April	CEO reiterates expectation that 1-year data suffice for AA; pre-BLA timing TBD; patients up to 4.5 years follow-up	Stable-positive (de-risking maintained)
Fabry clinical efficacy breadth	Positive renal function and QoL; ERT withdrawal sustained	Positive 52w/104w slopes; stable cardiac endpoints; durable α‑Gal A; 1 ERT re-start post-cutoff	Strengthened dataset with nuance
Fabry partnering	Ongoing BD to secure commercialization partner	“Highly focused” on near-term partner; negotiations progressing	Increasing urgency
ST-503 (Nav1.7 pain)	IND cleared; enrollment targeted mid-2025	First site initiated; dosing expected fall 2025; 12-week early data by late 2026	Advancing to first dosing
ST-506 (prion)	CTA enabling work; NHP/mouse data; CTA expected 2026	MHRA alignment on tox and design; GLP tox prep; CTA as early as mid-2026	On track
Funding/runway	Cash into late Q3’25 with BD/financing plan	Cash + ATM into 4Q’25; equity raised ~$21M	Slightly improved but still tight

Management Commentary

CEO on regulatory path: “We have no expectation that the agency will require anything other than the one year data for accelerated approval… the earliest patient is now four and a half years out and the data looks very consistent and very stable.”
CDO on efficacy: “Achieving a positive mean eGFR slope across all thirty two dosed patients after one year and across the nineteen patients who have reached two years is remarkable.”
CEO on partnering and funding: “Earlier this quarter we completed an equity offering that we hope will bridge us to an anticipated Fabry commercialization agreement… we remain highly focused on our critical task of securing a Fabry commercialization partner in the near term.”
CFO on resource priorities: “Our intention is to continue taking the Nav1.7 program forward and dose patients as intended… our number one priority is finding a Fabry commercialization partner… to solve our funding needs.”

Q&A Highlights

FDA/BLA mechanics: Pre-BLA meeting not yet held; expectation remains 1-year data can support Accelerated Approval with yearly updates thereafter .
ST-503 versus Nav1.8 programs: Management re-affirmed conviction in Nav1.7 given human genetics and mechanism; aiming to demonstrate dose-response and durable benefit; 12-week endpoints anticipated in late 2026 readout .
Adoption interest: Patient groups and investigators “extremely enthusiastic”; stable cardiac measures impressed external experts—supporting potential uptake pending approval and partner execution .
Cash burn and runway: Continuing site activations and first dosing for ST-503, while focusing on Fabry deal to address near- and long-term funding .

Estimates Context

Q2 2025 vs S&P Global consensus: Revenue $18.31M vs $31.68M* (miss); EPS −$0.08 vs −$0.04* (miss); EBITDA ≈−$16.86M* vs +$7.80M* (miss). Primary EPS estimates count: 6*; Revenue estimates count: 5*. Values retrieved from S&P Global. *
Implications: Street likely recalibrates near-term P&L (EBITDA/EPS) lower and shifts attention to BD timing for Fabry and potential regulatory catalysts to underpin funding and commercialization trajectory. Values retrieved from S&P Global.*

Key Takeaways for Investors

Near-term stock drivers: detailed STAAR data (Sept 2025), a Fabry commercialization agreement, first ST-503 dosing, and clarity on pre-BLA/BLA timing—each could materially shift funding visibility and valuation .
Execution remains critical: reiterated OpEx discipline with FY25 GAAP $135–$155M and non-GAAP $125–$145M; runway into 4Q’25 raises urgency to secure a partner or additional capital before year-end .
Clinical narrative strengthening: Positive renal function at 52/104 weeks, stable cardiac metrics, favorable safety, and QoL gains bolster the potential for AA in Fabry, though one ERT re-start warrants monitoring .
Estimate resets likely: Q2 revenue/EPS/EBITDA missed consensus; absent a near-term deal, Street models may reduce BD revenue cadence and widen loss expectations. Values retrieved from S&P Global.*
Pipeline diversification: ST-503 entry into clinic (Nav1.7 pain) and MHRA-aligned path for ST-506 (prion) expand optionality and partnering surface area over 2025–2026 .
BD momentum sustained: Recent Lilly capsid deal contributed materially to revenue; continued interest in MINT and STAC-BBB platforms could provide additional non-dilutive funding opportunities .

Appendix

Detailed Q2 2025 Financial Data (select line items)

Income Statement (Q2 2025): Revenue $18.306M; R&D $27.084M; G&A $9.077M; Total OpEx $36.161M; Loss from operations $(17.855)M; Interest income $0.386M; Other expense $(2.490)M; Net loss $(19.986)M; EPS $(0.08); Shares 256.95M .
Balance Sheet (6/30/25): Cash & cash equivalents $38.344M; Total assets $97.558M; Stockholders’ equity $19.602M .
Guidance reiteration: GAAP OpEx $135–$155M; Non-GAAP OpEx $125–$145M (excl. ~$7M SBC and ~$3M D&A) .

Clinical Efficacy and Safety Highlights (Fabry STAAR)

Mean annualized eGFR slope: +1.965 mL/min/1.73m²/yr at 52 weeks (n=32); +1.747 at 104 weeks (n=19) .
Cardiac stability: LVM/LVMI/GLS/T1/T2 and volumes stable at 1 year; troponin and NT‑proBNP stable .
Durability: α‑Gal A elevated up to 4.5 years in longest-treated patient .
Safety: Mostly grade 1–2 AEs; no safety-related discontinuations or deaths .
ERT withdrawal: All 18 ERT starters off ERT at cutoff; 1 restarted ERT post-cutoff .

SANGAMO THERAPEUTICS, INC (SGMO)·Q2 2025 Earnings Summary