Executive Summary

Q3 2025 was operationally solid but optically mixed: revenue grew 4% year over year to $776.1M while non-GAAP EPS fell to $0.12 due to a $221M acquired IPR&D charge tied to the Inozyme acquisition; management raised full-year total revenue guidance to $3.15–$3.20B and reaffirmed VOXZOGO at $900–$935M .
The company is pursuing options to divest ROCTAVIAN and removed its prior $4B 2027 revenue outlook, replacing it with scenario ranges: lower bound aligns with 2027 consensus ex-ROCTAVIAN, upper bound reaches ~$4B, reflecting potential competitive and IP outcomes .
Q3 revenue came in slightly below consensus and non-GAAP EPS missed on the IPR&D charge; management attributed quarterly Voxzogo slippage to order timing, reiterating Q4 as the strongest Voxzogo quarter and steady patient adds .
Cash generation remains a highlight: $369M operating cash flow in Q3, $728M YTD, and ~$2.0B cash and investments, supporting $4–$5B of potential “firepower” for business development .
Stock narrative catalysts: ROCTAVIAN divestiture, Voxzogo Q4 strength, 2027 framework reset vs competitive timelines, and BD deployment cadence .

What Went Well and What Went Wrong

What Went Well
- Raised FY25 total revenue guidance midpoint; reaffirmed Voxzogo outlook as management emphasized “strong results across the business” and expanding profitability and cash flow; cash/investments ~ $2B at Q3-end .
- Voxzogo continued global expansion (55 countries) with 24% YTD growth; management expects Q4 to be the highest Voxzogo quarter and noted ~75% of Voxzogo revenue is ex-U.S. .
- Cash generation and balance sheet strength: Q3 operating cash flow $369M, YTD $728M, supporting BD optionality; management estimates $4–$5B deployable capital .
Management quote: “We are very pleased with the strong results… leading us to raise full-year total revenues guidance… and reaffirm our Voxzogo revenue outlook for 2025.” – CEO Alexander Hardy .
What Went Wrong
- Q3 EPS compressed by $221M acquired IPR&D charge (~$1.10 per share), weighing on both GAAP and non-GAAP profitability; non-GAAP operating margin fell to 2.8% vs 27.7% LY .
- Sequential Voxzogo and enzyme therapy revenue softness due to order timing and Q2 pull-forward (large orders for Naglazyme/Vimizim); Voxzogo Q3 slightly down QoQ but expected to backweight to Q4 .
- Long-term uncertainty elevated: Company withdrew prior 2027 $4B target and framed outcomes with wide ranges given potential Voxzogo competition; investors lose a firm outer-year anchor .

Financial Results

Revenue, EPS and consensus comparison

Metric	Q1 2025	Q2 2025	Q3 2025
Total Revenues ($USD Millions)	$745.1	$825.4	$776.1
Revenue Consensus Mean ($USD Millions)*	$738.7	$761.7	$780.4
GAAP Diluted EPS	$0.95	$1.23	$(0.16)
Non-GAAP Diluted EPS	$1.13	$1.44	$0.12
Primary EPS Consensus Mean*	$0.953	$1.012	$0.308
YoY Revenue Growth (%)	+15%	+16%	+4%

*Values retrieved from S&P Global.

Margins

Margin	Q1 2025	Q2 2025	Q3 2025
GAAP Operating Margin %	30.0%	33.5%	(6.0)%
Non-GAAP Operating Margin %	35.7%	39.9%	2.8%

Product/Segment revenue mix

Product ($USD Millions)	Q3 2024	Q2 2025	Q3 2025
VOXZOGO	$190	$221	$218
VIMIZIM	$178	$215	$183
NAGLAZYME	$132	$129	$122
PALYNZIQ	$91	$106	$109
ALDURAZYME	$71	$56	$54
BRINEURA	$37	$49	$48
Total Enzyme Therapies	$509	$555	$516
KUVAN	$28	$27	$24
ROCTAVIAN	$7	$9	$3
Total Revenues	$745.7	$825.4	$776.1

KPIs and cash metrics

KPI	Q3 2025
Operating Cash Flow ($USD Millions)	$369
YTD Operating Cash Flow ($USD Millions)	$728
Cash & Investments ($USD Billions)	~$2.0
Voxzogo Countries Live	55
Voxzogo % of revenue ex-U.S. (YTD)	~75%
Estimated BD Firepower	$4–$5B

Why the quarter looked this way: modest total revenue growth was driven by Voxzogo and Palynziq, partly offset by Aldurazyme order dynamics and Latin America timing for Naglazyme; EPS/margins were depressed by the $221M IPR&D charge from the Inozyme acquisition .

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
Total Revenues	FY 2025	$3,125–$3,200M (Aug 4, 2025)	$3,150–$3,200M (Oct 27, 2025)	Raised (midpoint)
Non-GAAP Operating Margin %	FY 2025	33–34% (Aug 4)	26–27% (Oct 27)	Lowered (includes IPR&D)
Non-GAAP Diluted EPS	FY 2025	$4.40–$4.55 (Aug 4)	$3.50–$3.60 (Oct 27)	Lowered (includes IPR&D)
Voxzogo Revenue	FY 2025	$900–$935M (Aug 4)	$900–$935M (Oct 27)	Maintained
2027 Total Revenue Framework (ex-ROCTAVIAN)	FY 2027	Prior $4B outlook (2024)	Range: lower bound ≈ 2027 consensus ($3.65B ex-ROCTAVIAN) to upper bound ~$4B; company not guiding to a point estimate	Replaced with scenario range
ROCTAVIAN	Ongoing	N/A	Pursuing divestiture/out-licensing; available in U.S., Italy, Germany in interim	Strategic change

Notes: Management reiterated a 40% non-GAAP operating margin target for 2026, with flexibility to prioritize long-term value creation if trade-offs emerge .

Earnings Call Themes & Trends

Topic	Previous Mentions (Q1 2025 and Q2 2025)	Current Period (Q3 2025)	Trend
Voxzogo trajectory and order timing	Q1: Strong demand, uneven ex-U.S. ordering; H2 and Q4 weighted . Q2: Voxzogo +20% Y/Y; H2 weighted to Q4 .	Q3: Slight QoQ softness from timing; reaffirmed $900–$935M; Q4 to be the strongest; steady patient adds; expansion in 55 countries .	Back-half weighted; execution steady ex-U.S.; U.S. initiatives ongoing.
Competitive landscape and 2027 framing	Q1/Q2: No explicit reset; Q2 flagged Inozyme IPR&D in Q3 .	Company replaces 2027 guide with ranges reflecting competitor timing/IP scenarios; lower bound aligns with 2027 consensus ex-ROCTAVIAN, upper bound ~$4B .	Increased uncertainty acknowledged; scenario-based communication.
ROCTAVIAN strategy	Q1/Q2: Focused strategy noted (2024 review) .	Pursuing divestiture/out-licensing; remains available in U.S./Italy/Germany short term .	Portfolio focus; capital redeployment potential.
Operating leverage and cash	Q1/Q2: Expanding margins and cash flow; guidance raised in Q2 .	Q3 OCF $369M (YTD $728M); ~ $2B cash/investments; BD “firepower” $4–$5B .	Funding capacity for BD strengthened.
IPR&D impact	Q2: Expected IPR&D in Q3 from Inozyme .	$221M acquired IPR&D reduced EPS/margins in Q3 .	One-time headwind in reported Q3.
Pipeline: BMN-333 and hypochondroplasia	Q1: BMN-333 PK data by YE; plan for 2026 pivotal . Q2: PK exceeded targets; pivotal 1H26 .	BMN-333 phase 2/3 start 1H26; hypochondroplasia pivotal data 1H26, launch 2027 if positive .	On-track toward 2026/2027 milestones.
PALYNZIQ adolescent label expansion	Q1: PEGASUS positive; plan H2’25 submissions .	FDA Priority Review; PDUFA Feb 28, 2026 .	Regulatory momentum.
Orphan exclusivity/IP	Limited prior commentary.	FDA petition on Voxzogo ODE; IP outcomes part of 2027 scenarios .	Heightened focus given competition.
Macro/tariffs	Q2: Tariffs immaterial to 2025 .	No change indicated.	Neutral.

Management Commentary

Strategic focus and portfolio: “We… are announcing the decision to pursue options to divest Roctavian and remove it from our portfolio… to ensure access to those interested in a gene therapy treatment.” – CEO Alexander Hardy .
Financial/Guidance posture: “We are updating full-year 2025 non-GAAP operating margin guidance to between 26% and 27%, and non-GAAP diluted EPS guidance to between $3.50 and $3.60,” reflecting the IPR&D charge and strong Q4 expectations – CFO Brian Mueller .
2027 framework: “Lower end… in line with current 2027 top-line consensus per FactSet, excluding Roctavian… higher end… scenarios that reach $4 billion… also excluding Roctavian… We are not providing a specific estimate” – CFO Brian Mueller .
Commercial execution: “For the remainder of 2025, we expect… Q4 being the highest of Voxzogo revenue for the year” – CCO Cristin Hubbard .
Capital deployment: “We estimate that our total firepower is between $4 billion to $5 billion… With our current and growing EBITDA profile, we do have a chance to leverage our earnings” – CFO Brian Mueller .

Q&A Highlights

2027 outlook reset: Management replaced the prior $4B 2027 target with a scenario range reflecting potential competitor launches and IP outcomes; lower bound aligns with 2027 consensus excluding ROCTAVIAN; upper bound up to ~$4B ex-ROCTAVIAN .
Voxzogo Q3 dynamics: Slight QoQ downtick driven by order timing; patient adds continued; reaffirmed $900–$935M with Q4 backweight .
Competition and exclusivity: Assumed “middle-of-the-road” competitor approval timelines and successful launches in lower-case estimates; Voxzogo orphan exclusivity petition with FDA pending; patient switching expected to be limited among those satisfied on therapy .
Margin/cash targets: 40% non-GAAP operating margin remains a 2026 target, with flexibility to prioritize value creation; ≥$1.25B 2027 CFO characterized as top-line dependent .
Capital allocation: Emphasis on BD over buybacks; estimated $4–$5B deployable “firepower” .

Estimates Context

Q3 2025: Revenue $776.1M vs consensus $780.4M* (slight miss); non-GAAP EPS $0.12 vs consensus $0.308* (miss), primarily due to the $221M IPR&D charge .
Q2 2025: Revenue $825.4M vs $761.7M* (beat); non-GAAP EPS $1.44 vs $1.012* (beat), driven by strong Voxzogo and enzyme therapy growth with operating leverage .
Q1 2025: Revenue $745.1M vs $738.7M* (beat); non-GAAP EPS $1.13 vs $0.953* (beat), supported by Voxzogo +40% Y/Y and lower OpEx from portfolio focus .