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PTC THERAPEUTICS, INC. (PTCT)·Q3 2025 Earnings Summary

Executive Summary

  • Q3 2025 revenue was $211.0M, driven by the initial Sephience™ launch ($19.6M) and stronger Evrysdi® royalties; diluted EPS came in at $0.20, swinging to profit versus a loss last year and ahead of consensus. The company narrowed FY2025 revenue guidance to $750–$800M, increasing the lower bound from $650M set earlier in the year .
  • Sephience global uptake was broad: 521 U.S. patient start forms from 141 prescribers and 341 patients on therapy worldwide as of Sep 30; U.S. revenue was $14.4M and ex-U.S. $5.2M in Q3 .
  • DMD franchise revenue was $85.9M (Translarna $50.7M; Emflaza $35.2M) amid continued generic pressure on Emflaza; Evrysdi royalties rose to $70.8M for the quarter .
  • Near-term catalysts: continued Sephience momentum (favorable payer access; refills), Japan/Brazil regulatory decisions expected in Q4, and FDA meetings on votoplam (Huntington’s disease) and vatiquinone (Friedreich’s ataxia) .

What Went Well and What Went Wrong

What Went Well

  • Strong launch of Sephience with $19.6M in Q3 revenue, supported by broad early demand across patient segments and favorable payer policies covering ~250M lives; management highlighted rapid phenylalanine (Phe) lowering and early refills .
  • Evrysdi royalties increased (Roche YTD CHF ~1,293M), contributing $70.8M to Q3 revenue; total revenue grew 7.2% YoY to $211.0M .
  • Expense discipline: non-GAAP R&D fell to $91.0M (vs. $152.0M LY) and non-GAAP SG&A to $73.8M (vs. $63.1M LY), enabling a swing to positive net income of $15.9M from a loss of $106.7M last year .
  • Quote: “The broad initial uptake supports the potential of Sephience to become the standard of care for all individuals with PKU…” — CEO Matthew Klein .

What Went Wrong

  • DMD franchise headwinds persisted: Translarna fell to $50.7M (vs. $72.3M LY), Emflaza to $35.2M (vs. $51.9M LY), reflecting ongoing regulatory and generic pressures .
  • Gross mix still requires careful management; while Q3 margin improved versus prior periods, legacy product declines necessitate sustained Sephience execution to maintain profitability .
  • Collaboration/license revenue contribution was modest in Q3 ($9.3M), so earnings quality leaned more on operating efficiencies and tax benefit in the quarter; future EPS trajectory will depend on continued commercial scale-up .

Financial Results

MetricQ3 2024Q2 2025Q3 2025
Revenue ($USD Millions)$196.8 $178.9 $211.0
Net Income ($USD Millions)$(106.7) $(64.8) $15.9
Diluted EPS ($USD)$(1.39) $(0.83) $0.20
EBIT Margin %-26.41%*-19.49%*1.65%*
EBITDA Margin %-23.11%*-15.25%*6.92%*
Net Income Margin %-54.20%*-36.25%*7.53%*
Total Operating Expenses ($USD Millions)$251.3 $213.8 $208.0

Values marked with * retrieved from S&P Global.

Segment and revenue composition

Segment / Line Item ($USD Millions)Q3 2024Q2 2025Q3 2025
Sephience Net Product Revenue——$19.6
Translarna Net Product Revenue$72.3 $59.5 $50.7
Emflaza Net Product Revenue$51.9 $36.4 $35.2
DMD Franchise Total$124.2 $96.0 $85.9
Evrysdi Royalty Revenue$61.4 $57.6 $70.8
Collaboration & License Revenue—$2.9 $9.3
Total Revenue$196.8 $178.9 $211.0

KPIs

KPIQ3 2025
U.S. patient start forms (PSFs)521
Unique U.S. prescribers141
Patients on commercial therapy (global)341
PSF-to-fill timeline (avg.)2–4 weeks (commercial plans)
U.S. payer access coverage~250M lives
Expected payer mix (steady-state)65% commercial / 35% government

Actual vs. S&P Global Consensus

PeriodRevenue Actual ($M)Revenue Consensus ($M)SurpriseEPS Actual ($)EPS Consensus ($)Surprise
Q3 2024196.8 171.8+14.5%(1.39) (1.52)Beat
Q2 2025178.9 173.6+3.1%(0.83) (1.03)Beat
Q3 2025211.0 175.5+20.2%0.20 (1.37)Beat

Revenue and EPS consensus values retrieved from S&P Global.

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Total RevenueFY 2025$650–$800M (Q1/Q2) $750–$800M (Q3) Raised lower bound; narrowed high end
GAAP R&D + SG&AFY 2025$805–$835M $805–$835M Maintained
Non-GAAP R&D + SG&AFY 2025$730–$760M (ex-SBC ~$75M) $730–$760M (ex-SBC ~$75M) Maintained

Earnings Call Themes & Trends

TopicPrevious Mentions (Q1 & Q2)Current Period (Q3)Trend
Sephience approvals/launchEC approval (Jun 19) and FDA approval (Jul 28); global launch initiated Broad uptake across all segments; 521 PSFs; favorable payer access; early refills Accelerating adoption
Payer access and pricing corridorPreparing for market access; narrow pricing corridor noted Favorable policies, 6–12 month refills; commercial-heavy mix stabilizing at 65/35 Positive access dynamics
DMD portfolio trajectoryOngoing pressure from generics; Article 117 leverage in EU Continued declines in Translarna/Emflaza, but defended via programs Structural headwinds persist
Regional rollout (EU/JP/BR)Launch in Germany; early access in EU; Japan decision expected Q4 Germany free pricing period; HTA submissions in EU; Japan and Brazil expected Q4; Canada approval Broadening global footprint
R&D execution (HD, FA)PIVOT-HD positive Phase 2; plan FDA meeting Q4 FDA meetings for votoplam (HD) and vatiquinone (FA) planned this quarter Advancing regulatory path
Translarna regulatoryNDA under FDA review NDA remains under FDA review Ongoing

Management Commentary

  • “We achieved an outstanding quarter, highlighted by the strong start to the global Sephience launch…foundational product for PTC’s sustained growth and success.” — CEO Matthew Klein .
  • “We are very pleased with the early momentum…favorable payer policies that maintain broad access…refills for 6–12 months.” — CBO Eric Pauwels .
  • “Non-GAAP R&D expense was $91M…Non-GAAP SG&A expense was $74M…we remain well-capitalized to reach cash flow break-even.” — CFO Pierre Gravier .
  • “One of the early skeptics…said, ‘I’m converted and I’m ready to try all my patients on Sephience.’” — CEO Matthew Klein, on clinician feedback .

Q&A Highlights

  • Durability and adherence: Management cited broad responder profile (15–30%+ Phe reductions), cognitive and mood improvements, and early refills, but emphasized it’s early days for specific retention metrics .
  • Access timeline: PSF to fill is often within days for commercial plans; on average 2–4 weeks; government plans lag but reimbursement is occurring across Medicare/Medicaid/Tricare .
  • Centers of excellence: 100% awareness with prescriptions from all; ~two-thirds have written more than one prescription, indicating deepening penetration .
  • Competitive dynamics: Reported switches from Palynziq in early field feedback due to Sephience’s once-daily oral profile and tolerability; management expects trying “all patients,” knowing not all respond .
  • Japan/Brazil timelines and pricing: Japan expected approval by year-end with premium pricing; Brazil approval expected Q4 with 2026 revenue contribution post pricing and access processes .

Estimates Context

  • Q3 2025 was a major beat: revenue $211.0M vs $175.5M consensus (+20.2%); EPS $0.20 vs $(1.37) consensus. Strength came from Sephience ($19.6M), Evrysdi royalties ($70.8M), and lower R&D/SG&A vs prior year; a tax benefit also supported EPS .
  • Prior quarters also exceeded Street: Q2 2025 revenue $178.9M vs $173.6M; EPS $(0.83) vs $(1.03). Q3 2024 revenue $196.8M vs $171.8M; EPS $(1.39) vs $(1.52).*
  • Implication: Street models likely need to raise near-term revenue/EPS trajectories to reflect Sephience ramp, favorable access, and growing royalty tailwinds; FY revenue guide tightened to $750–$800M aligns to upper end of prior range .

Consensus values retrieved from S&P Global.

Key Takeaways for Investors

  • Sephience launch is the central growth driver, with early broad uptake, favorable payer access, and refills; expect sustained momentum into Q4 as more policies finalize .
  • DMD franchise remains a headwind, but management is defending share; Sephience and royalties can offset legacy declines over time .
  • FY2025 revenue guidance raised at the low end to $750–$800M, suggesting confidence in Sephience cadence and portfolio resilience; watch for 2026 OpEx decline commentary at JPMorgan .
  • Near-term catalysts: Japan and Brazil Sephience decisions (Q4), expanded EU access via HTA, and R&D Day (Dec 2) to showcase pipeline; potential narrative upgrades on execution .
  • Votoplam (HD) and vatiquinone (FA) regulatory meetings could add optionality; any clarity on accelerated approval paths is incremental upside .
  • Trading lens: Momentum skew positive on consecutive consensus beats and strong launch metrics; monitor weekly policy updates, refills, and geographic expansion for confirmation of trajectory .
  • Risk checks: Legacy portfolio pressure, regulatory outcomes (Translarna), and payer mix normalization (commercial-to-government) could influence gross-to-net and margins; track mix towards 65/35 steady-state .