Executive Summary

Q2 2025 delivered a one-time revenue of $0.50M from a leflutrozole milestone, R&D of $5.4M (+10% YoY), G&A of $5.5M (-30% YoY), and net loss of $14.6M; cash was $56.1M, with runway “into 2027” unchanged .
Against S&P Global consensus, EPS missed (actual: -$0.02 vs -$0.0119*) while revenue beat on the milestone (actual: $0.50M vs $0.00*); consensus counts: 6 for EPS, 7 for revenue*.
Execution remains centered on setrusumab (UX143) Phase 3 Orbit and Cosmic readouts expected around year-end 2025; DMC confirmed acceptable safety and continuation to final analysis .
Management is advancing alvelestat partnering and Phase 3 start-up; pre-commercial work for EU setrusumab commercialization continued and expanded across regions .

Values retrieved from S&P Global for estimates (*).

What Went Well and What Went Wrong

DMC affirmed acceptable safety in the Phase 3 Orbit study and continuation to final analysis, keeping the program on timeline to year-end 2025 readouts .
G&A was reduced materially YoY (-$2.4M), aided by a $1.9M ADR reimbursement and lower professional fees, helping offset higher R&D and FX headwinds .
CEO tone confident on clinical trajectories and commercial readiness: “We continue to be excited about the potential of setrusumab to reduce fractures and improve other functional parameters…” and “well positioned…to support our operations into 2027” .

EPS missed consensus as FX drove a $5.3M net foreign currency loss, lifting net loss to $14.6M (vs $12.3M YoY) despite lower G&A .
R&D rose $0.4M YoY to $5.4M on setrusumab manufacturing/supply and EU real-world evidence/medical affairs efforts, partially offset by lower alvelestat and etigilimab spend .
Revenue quality was non-recurring (one-time $0.5M milestone on leflutrozole), with limited visibility on continued top-line contributions pre-approval .

Metric	Q2 2024	Q1 2025	Q2 2025
Revenue ($USD Millions)	$0.000	n/a	$0.500
R&D Expenses ($USD Millions)	$4.946	$3.930	$5.373
G&A Expenses ($USD Millions)	$7.868	$7.272	$5.494
Net Loss ($USD Millions)	$12.255	$12.887	$14.616
Loss per Share (Basic & Diluted) ($USD)	$(0.02)	$(0.02)	$(0.02)
Cash & Cash Equivalents ($USD Millions)	$69.802 (Dec 31, 2024)	$62.483 (Mar 31, 2025)	$56.125 (Jun 30, 2025)

Revenue composition and cost:

Item	Q2 2025
One-time milestone revenue (leflutrozole) ($USD Millions)	$0.500
Cost of revenue ($USD Millions)	$0.132

Estimates vs actuals (S&P Global consensus):

Metric	Consensus	Actual	Beat/Miss
Revenue ($USD Millions)	$0.000*	$0.500	Bold beat
EPS ($USD)	-$0.0119*	-$0.0200	Bold miss
EPS - # of Estimates	6*	—	—
Revenue - # of Estimates	7*	—	—

Values retrieved from S&P Global for estimates (*).

KPIs:

KPI	Q1 2025	Q2 2025
Ordinary shares issued	795,001,444	795,001,444
ADS equivalents	159,000,288	159,000,288

Metric	Period	Previous Guidance	Current Guidance	Change
Cash runway	FY 2024 → Q1 2025 → Q2 2025	Fund operations into 2027	Fund operations into 2027	Maintained
Setrusumab (Orbit/Cosmic) Phase 3 readout timing	Q1 2025 vs Q2 2025	IA2 mid-2025 or final analysis Q4 2025	Final analyses expected around year-end 2025	Narrowed to year-end window
Setrusumab EU pre-commercial readiness	FY 2024 → Q1 2025	Ongoing EU HTA engagement, SATURN real-world data	Continued and expanded across additional EU regions	Expanded
Alvelestat Phase 3 initiation/partnering	FY 2024 → Q1 2025 → Q2 2025	Phase 3 start-up ongoing; active partner discussions	Start-up ongoing; multiple partner dialogues	Maintained

Note: No Q2 2025 earnings call transcript was available; themes reflect management’s press release commentary and related 8-Ks.

Topic	Previous Mentions (Q4 2024, Q1 2025)	Current Period (Q2 2025)	Trend
Setrusumab clinical progress (Orbit/Cosmic)	Orbit IA2 mid-2025 or final Q4 2025; Cosmic treated in parallel	DMC confirmed acceptable safety; continue to final analysis; data expected around year-end 2025	On track; increased visibility
EU pre-commercial setup (HTA, SATURN)	Ongoing scientific advice; SATURN real-world data	Continued; expanding landscape beyond big-5 to Nordic/Benelux	Expanding
Alvelestat Phase 3 readiness and partnering	EC Orphan Designation; Phase 3 start-up; partner outreach	Start-up activities ongoing; active partner discussions	Maintained momentum
Financial discipline/cash runway	$69.8M cash YE24; runway into 2027	$56.1M cash; runway into 2027 unchanged	Stable guidance; cash trending down pre-readout
FX impact on P&L	FX volatility present historically	$5.3M net FX loss pressured EPS/NI	Headwind this quarter

CEO: “We continue to be excited about the potential of setrusumab to reduce fractures and improve other functional parameters for individuals living with osteogenesis imperfecta.”
CEO: “We are continuing to advance partnering discussions around alvelestat… and to ready the program for Phase 3 initiation.”
CEO: “Our prudent management of our cash and resources means we are well positioned… to support our operations into 2027.”

No Q2 2025 earnings call transcript was found; the company furnished results via 8-K/exhibit press release and an 8-K “Other Events” clinical update. Key clarifications included DMC’s Orbit safety conclusion and continuation to final analysis, and the unchanged cash runway guidance .

EPS missed consensus: -$0.02 actual vs -$0.0119* consensus; FX loss was the primary driver of the miss .
Revenue beat consensus: $0.50M actual vs $0.00* consensus, driven by a one-time leflutrozole milestone rather than recurring operations .
With results largely driven by non-operating FX and non-recurring revenue, Street models may adjust FX assumptions and keep revenue flat near zero pre-approval; opex paths (R&D, G&A) showed favorable mix with G&A declining YoY .

Values retrieved from S&P Global for estimates (*).

Near-term catalyst: Year-end 2025 Phase 3 Orbit/Cosmic readouts for setrusumab; DMC’s go-forward decision reduces program risk and supports timeline .
EPS miss was primarily FX-driven; structural opex indicates disciplined G&A with targeted R&D increases aligned to setrusumab EU readiness .
Revenue beat is non-recurring; do not extrapolate to run-rate—focus on clinical/regulatory milestones and EU commercialization groundwork .
Cash runway into 2027 remains intact; watch cash trajectory as pre-commercial and manufacturing-related expenses continue ahead of potential approvals .
Alvelestat partnering could be a valuation lever; Phase 3 start-up activities ongoing, with potential economics not included in guidance .
EU market preparation is broadening beyond big-5, which could accelerate initial adoption if approvals are obtained and HTA outcomes are supportive .
Risk factors remain: clinical outcome thresholds (p<0.04 Orbit; p<0.05 Cosmic), FX volatility, and timing of partnering economics; position sizing should reflect binary clinical readout risk .