Executive Summary

Q2 2025 revenue of $178.9M and diluted EPS of -$0.83; both exceeded Wall Street consensus estimates of $173.6M revenue and -$1.03 EPS, respectively. Bold beat on both top and bottom line. [ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-provides-corporate-update-and-reports-second-3] [ir.ptcbio.com/node/17926/pdf] Revenue Consensus Mean: $173.6M*, Primary EPS Consensus Mean: -$1.03* (Values retrieved from S&P Global).
Sephience (sepiapterin) achieved EU marketing authorization (broad label, all ages) on June 23 and FDA approval on July 28 (broad label ≥1 month); global launch underway in the U.S. and Europe, positioning a new growth pillar.
DMD franchise revenue was $96M, with Translarna $59.5M and Emflaza $36.4M; Evrysdi royalties were $57.6M.
FY2025 guidance maintained: total revenue $650–$800M; GAAP R&D+SG&A $805–$835M; non-GAAP R&D+SG&A $730–$760M.
Subsequent event: FDA issued a CRL for vatiquinone in FA (Aug 19), citing lack of substantial evidence of efficacy and request for an additional adequate study—an overhang for the pipeline near-term.

What Went Well and What Went Wrong

What Went Well
- Beat estimates: revenue $178.9M vs $173.6M* and EPS -$0.83 vs -$1.03*; better royalty contribution from Evrysdi. Revenue/EPS consensus values*, S&P Global.
- Major regulatory wins: Sephience broad-label approvals in EU and U.S.; management: “expect Sephience to be the foundational product for PTC’s future growth and path to profitability.”
- Balance sheet remains strong: cash, cash equivalents and marketable securities $1,989.2M at June 30, 2025.
What Went Wrong
- Core franchise decline: Translarna ($59.5M) and Emflaza ($36.4M) were down YoY vs Q2 2024 ($70.4M and $47.3M).
- Elevated operating spend vs prior year in SG&A (GAAP $85.3M vs $69.5M) reflecting launch and portfolio support.
- Post-quarter regulatory setback: FDA CRL for vatiquinone requires another adequate, well-controlled study, delaying potential revenue contribution.

Financial Results

Metric	Q4 2024	Q1 2025	Q2 2025
Total Revenue ($M)	$213.2	$189.9	$178.9
Diluted EPS ($)	$(0.85)	$10.04	$(0.83)

Segment and Royalty Detail

Metric	Q4 2024	Q1 2025	Q2 2025
DMD Franchise Revenue ($M)	$144.0	$134.0	$96.0
Translarna Net Product Revenue ($M)	$93.7	$86.2	$59.5
Emflaza Net Product Revenue ($M)	$50.5	$47.8	$36.4
Evrysdi Royalty Revenue ($M)	$58.2	$36.4	$57.6

Operating Expenses (Run-Rate Visibility)

Metric	Q4 2024	Q1 2025	Q2 2025
GAAP R&D ($M)	$124.8	$109.0	$113.0
Non-GAAP R&D ($M)	$116.0	$100.3	$104.0
GAAP SG&A ($M)	$84.7	$81.0	$85.3
Non-GAAP SG&A ($M)	$76.3	$71.6	$75.7

Balance Sheet & Shares

KPI	Q4 2024	Q1 2025	Q2 2025
Cash & Marketable Securities ($M)	$1,139.7	$2,027.2	$1,989.2
Total Debt ($M)	$285.4	$285.7	$286.0
Shares Outstanding (M)	77.70	79.23	79.38

Estimates vs Actuals (Q2 2025)

Metric	Consensus*	Actual
Revenue ($M)	173.6*	178.9
Primary EPS ($)	-1.03*	-0.83

*Values retrieved from S&P Global.

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
Total Revenue	FY 2025	$600–$800M (Q4’24)	$650–$800M (Q2’25)	Raised (Q4→Q1), Maintained in Q2
GAAP R&D + SG&A	FY 2025	$805–$835M (Q4’24)	$805–$835M (Q2’25)	Maintained
Non-GAAP R&D + SG&A	FY 2025	$730–$760M (Q4’24)	$730–$760M (Q2’25)	Maintained

Note: Q1 2025 set revenue to $650–$800M; Q2 maintained the same range.

Earnings Call Themes & Trends

Topic	Previous Mentions (Q4 2024 and Q1 2025)	Current Period (Q2 2025)	Trend
Sephience (PKU) launch scope and differentiation	Emphasis on diet liberalization, broad patient applicability; centers of excellence and dietitian engagement; preparedness for U.S./EU launch and payer receptivity.	EU authorization (June 23) and FDA approval (July 28); launch initiated in U.S. and Germany; broad labels (all ages).	Positive acceleration
DMD franchise durability (Translarna, Emflaza)	Ongoing defense; EU pathway via Article mechanisms; resilience in LatAm/MENA; Emflaza brand loyalty despite generics.	Q2 Translarna/Emflaza YoY decline; franchise $96M; continued U.S./RoW strategies implied.	Mixed/pressured
Evrysdi royalty trajectory	Consistent royalty stream; quarterly variability; positive YoY in Q1.	Q2 royalty $57.6M on Roche H1 CHF 869M sales.	Positive
Vatiquinone (FA) regulatory path	Priority Review; no AdCom expected at mid-cycle; broad unmet need (pediatric + adult) positioning.	Post-quarter FDA CRL: additional adequate study required.	Negative inflection
PTC518 (HD) with Novartis	Surrogate endpoint dialogue (HTT lowering); positive PIVOT-HD signals; BD economics; planning for Phase 3/AA.	Continued collaboration; aim to meet FDA in Q4 2025 on Phase 3/AA path.	Steady progress
Capital allocation & BD	>$2B cash enabling launches/BD; ability to reach cash flow breakeven without capital raise.	Q2-end cash ~$2.0B; internal launch investments continue.	Stable/strategic

Management Commentary

“We have initiated the global launch and expect Sephience to be the foundational product for PTC’s future growth and path to profitability.” — Matthew B. Klein, CEO (press release)
On Sephience launch preparedness and demand drivers (diet liberalization) discussed throughout prior calls; teams prioritized Germany/U.S., leveraging early-access and payer dialogues.
On cash runway and execution: “We closed the quarter with over $2 billion on our balance sheet… provides the ability to reach cashflow breakeven without raising additional capital.” (Q1 remarks)

Q&A Highlights

Sephience payer and access dynamics: payers receptive to differentiated Phe control and diet liberalization; prior authorization expected to follow label with minimal step edits for poorly controlled/failed patients.
DMD and Translarna continuity: country-by-country mechanisms in EU and sustained demand in LATAM/MENA; preparedness for potential U.S. approval.
Launch cadence and operational readiness: bolus of patients anticipated at launch; case management and staffing aligned to compress time-to-therapy.
Vatiquinone AdCom and unmet need: prior FDA interactions indicated no AdCom at mid-cycle; pediatric market with no approved therapy was a key focus.

Estimates Context

Q2 2025 actuals vs S&P Global consensus: revenue $178.9M vs $173.6M* and EPS -$0.83 vs -$1.03* — both beats. Scenario likely aided by stronger Evrysdi royalties and maintained DMD contribution despite Emflaza generics. Revenue/EPS consensus values*, S&P Global.
Estimate revisions: Following EU/U.S. approvals for Sephience and maintained 2025 guidance, Street models may lift H2 revenue for initial Sephience contributions while trimming FA assumptions post-CRL until a new study plan emerges.

Key Takeaways for Investors

Clean beat quarter: revenue and EPS exceeded consensus; quality of beat supported by royalty strength and manageable OpEx. Revenue/EPS consensus values*, S&P Global.
Sephience is live in major markets with broad labels; near-term revenue inflection driver with concentrated prescriber base and payer receptivity to differentiation.
DMD franchise headwinds persist YoY but remains an important cash generator; watch Translarna ex-EU pathways and Emflaza erosion pace.
Guidance intact at $650–$800M; implies confidence in H2 contributions (Sephience/U.S., Germany, and royalties) amid portfolio transitions.
Vatiquinone CRL resets FA timing; reduces near-term optionality pending FDA alignment on additional study—monitor update cadence.
Balance sheet (~$2.0B cash) underwrites launches and clinical plans without near-term financing; optionality for selective BD remains.
Medium-term: execution on Sephience ramp and clarity on FA/Translarna U.S. outcomes will be primary stock drivers; in parallel, 518/HD path with Novartis is a strategic longer-term call option.

Acronyms and Notes: Evrysdi® is Roche’s SMA therapy; royalties recognized by PTC. DMD = Duchenne muscular dystrophy; PKU = phenylketonuria; FA = Friedreich’s ataxia; H2 = second half. Consensus values marked with * are from S&P Global.

Sources: Q2 press release and 8-K exhibit (financial statements, guidance, and business updates) ; Sephience EU authorization and FDA approval releases ; Q1 and Q4 earnings materials for trend context and call Q&A – –; Vatiquinone CRL (Aug 19) .

PTC THERAPEUTICS, INC. (PTCT)·Q2 2025 Earnings Summary