PTC's Q3 2025: Contradictions on SUFIANCE Timelines, Sephience Impact, and Payer Policies

Date of Call: None provided

Financials Results

• Revenue: $211 million total revenue for Q3 2025; includes Sephience net product revenue $19.6M (US $14.4M; ex-US $5.2M), DMD franchise $86M (Translarna $51M; Emflaza $35M), Evrysdi royalty revenue $71M

Guidance:

• Narrowed 2025 full-year revenue guidance to $750M–$800M (company citing upper end confidence)
• Sephience launch momentum: $19.6M in Q3; Canada approved; Japan MAA approval anticipated Q4; Brazil approval expected this quarter; maintaining a narrow pricing corridor
• Payer mix expected to stabilize at ~65% commercial / 35% government
• Cash $1.688B as of Sep 30, 2025; company expects to reach cash-flow breakeven and pursue BD
• OpEx expected to decline in 2026; formal 2026 guidance to be provided at J.P. Morgan
• Planned Q4 FDA meetings for Huntington’s and Friedreich’s ataxia; Translarna NDA under review

Business Commentary:

* Sephienne Launch and Revenue Growth: - PTC Therapeutics reported third-quarter revenue of $211 million, with Sephience contributing $19.6 million. - The growth was driven by the successful launch of Sephience in Europe and the United States, with positive responses from healthcare providers and patients.

• Payers and Market Access:
• PTC has seen favorable payer policies for Sephience, with coverage that includes prior authorizations and limited step edits.

• Early engagement with payers covering over 250 million lives has led to a positive value proposition and broad access for the drug.

• Customer and Physician Adoption:

• As of September 30, Sephience has generated 521 patient start forms from 541 unique prescribers in the US.

• The high enthusiasm and positive feedback from physicians and patients have led to broad interest in prescribing Sephience across various patient segments, including adults and classical PKU patients.

• Financial Strength and Future Outlook:

• PTC Therapeutics ended the third quarter with approximately $1.68 billion in cash, which allows for strategic business development activities and achieving cash flow break-even.

• The company has narrowed its 2025 full-year revenue guidance to $750-$800 million, reflecting confidence in its ability to execute on the launch.

• Global Expansion and Regulatory Milestones:

• PTC anticipates regulatory approvals in Brazil and Japan later this year, with a focus on maintaining a narrow pricing corridor globally.
• Meetings with regulatory authorities, including the FDA, are planned to discuss study designs and potential approval pathways for Huntington’s Disease and Friedreich’s ataxia programs.

Sentiment Analysis:

Overall Tone: Positive

• CEO: "outstanding third quarter results" and "Sephience launch is off to a great start." Q3 revenue $211M; Sephience $19.6M; narrowed 2025 guide to $750–$800M; cash $1.688B; management repeatedly described broad physician/patient uptake, favorable payer policies, and sustained momentum into October.

Q&A:

• Question from Kristen Kleska (Cantor Fitzgerald): What’s going to give you confidence that beyond this really strong out-of-the-gate launch, you’re going to see maintained durability and that patients will be on therapy for a while?
  Response: Early real-world feedback shows uptake across all segments and diet liberalization; clinical trial response rates (66%–75%) and good tolerability underpin confidence in durable treatment retention.

• Question from Tazina Mott (Bank of America): What magnitude of Phe reduction are doctors and payers looking for to keep a patient on treatment beyond the trial period? What’s your expectation for percentage of patients that are going to stay on treatment following this initial trial? How are you thinking about guiding the street in that dynamic?
  Response: Thresholds vary (some clinicians value 15%–20%, others 30% or clinical benefit); company cites 66%–75% responder rates from trials and good adherence but says it’s too early to give precise retention guidance.

• Question from Brian Abrahams (RBC Capital Markets): What’s the timeline from a new start form to a patient receiving a prescription and actually getting the drug, and should we expect patients with start forms to roll on to treatment in Q4?
  Response: PTC Cares expedites starts; average PSF-to-fill ~2–4 weeks (commercial plans can be days); expect many of the current starts to convert in Q4, while government plans may take longer.

• Question from Brian Chang (JP Morgan): On the PTC518 start form number and October momentum — is that momentum referring to pace of start forms or payer access? What is the momentum specifically based on?
  Response: Momentum reflects steady, consistent start-form inflow and patient starts plus favorable payer access; early uptake and policies have driven consistent rates into October.

• Question from Judah Framer (Morgan Stanley): Can you help with the narrowing of the full-year guide — is that solely tied to Sephience? Any other moving pieces? And for the centers of excellence called out, how far have you penetrated those centers numerically?
  Response: Guide narrowed primarily due to Sephience (Amplify delta) plus stable legacy performance (Emflaza); 100% awareness at centers of excellence, prescriptions from all, and ~two-thirds of centers have prescribed more than one patient.

• Question from Clara Dong (Jefferies): Can you share more details on the patient profile for new prescribers — are initial uptakes concentrated in one group? Updated thoughts on overall opportunity given early momentum?
  Response: Early uptake spans the full spectrum (treatment‑naive, switches, all ages from infants to age 79); too early for revenue projections, but U.S. addressable population cited as ~17,000 patients.

• Question from Paul Choi (Goldman Sachs): Any additional coverage updates for Germany and other major EU markets? And outlook for 2026 OpEx trajectory versus consensus?
  Response: Germany currently in free‑pricing; HTA/MNOG assessments and price negotiations expected to take ~6–12 months into 2026; OpEx expected to decline in 2026 with formal guidance at J.P. Morgan.

• Question from Jun Lee (Truist Securities): You’re seeing switches from Palynziq — is that an outlier? Why would someone switch? How quickly could you launch in Japan post-approval and where does Japan fit versus US/EU peak opportunity?
  Response: Switches observed likely due to Sephience’s once‑daily oral dosing, tolerability, and efficacy even in severe patients; Japan approval anticipated before year‑end with premium pricing and reimbursement discussions into Q1, smaller prevalence (~1,000) but high value.

• Question from Gina Wang (Barclays): Based on $19.6M and 341 patients, is net price ~$350k? What is average time patient on drug and retention so far? How is revenue booked (monthly/shipments)?
  Response: WAC ~$490k and average patient ~45 kg; current net pricing is in that ballpark given favorable commercial mix; revenue recognized on shipments (Germany recognized immediately); early refill rates are strong but retention data remain preliminary.

• Question from Sammy Corwin (William Blair): Are initial patients skewing pediatric or adult? Regarding Huntington’s, how are you/Novartis thinking about the registrational trial and what do you hope to get from the FDA meeting?
  Response: Initial patients span full age range with average around late adolescence (~17); PTC518 is an oral program with placebo‑controlled data (>140 patients); Q4 FDA meeting aims to align on accelerated approval path and design of the confirmatory/efficacy trial.

• Question from Joe Schwartz (Lyrinx Partners): Can you quantify real‑world response rate based on refills or other metrics and how that tracks versus trial efficacy?
  Response: Too early to quantify real‑world response rates; anecdotal and refill feedback through October is positive but no definitive metrics yet.

• Question from Peyton Bonsack (TD Cowen): On Brazil assuming approval, can you quantify the opportunity, differences in patient population, and steps from approval to launch?
  Response: Brazil approval expected this quarter; post‑registration CMED pricing references Germany/Japan/US within narrow pricing corridor; patient diagnosis patterns differ (later diagnoses in some states) but Brazil represents a meaningful market and management expects revenue contribution in 2026.

• Question from Luke Herman (Baird): Given diet liberalization, could patient weight increase over time and be a tailwind, particularly for poorly controlled patients?
  Response: Weight impact is uncertain; many PKU patients have typical or higher weights; emphasis is on gradual, dietitian‑led liberalization to ensure safe nutritional transitions rather than viewing weight as a primary tailwind.

Contradiction Point 1

Regulatory Status and Launch Timeline of SUFIANCE

It involves discrepancies in the regulatory progress and launch timeline of SUFIANCE, which are crucial for product availability and market penetration.

Can you confirm the Q4 MAA submission timeline? - Kristen Kleska (Cantor Fitzgerald)

2025Q3: We expect to submit the MAA in Q4. - Dr. Matthew Klein(CEO)

Can you discuss what you can share about SUFIANCE’s FDA discussions? - Kristen Kluska (Cantor Fitzgerald)

2025Q1: We are far along in discussions with the FDA and expect to submit the NDA early next year. We have already started discussions on the package insert. - Matthew Klein(CEO)

Contradiction Point 2

Launch Strategy and Impact of Sephience on Emflaza

It involves the strategic positioning of Sephience and its potential impact on Emflaza sales, which are critical for market share and revenue forecasts.

What is the timeline from new start forms to patients receiving prescriptions and the drug, and should we expect patients to begin treatment in Q4? - Brian Abrahams (RBC Capital Markets)

2025Q3: Sephience has the potential to become standard care, but we are not counting on major switches from Emflaza at this point. - Dr. Matthew Klein(CEO)

How do global macro factors such as tariffs or MFN clauses impact your financial guidance and business strategy? - Peyton Bohnsack (TD Cowen)

2025Q1: We've seen a bit of a pickup in the use of Emflaza in the midst of this launch, and we hope that will continue, but it's early. - Eric Pauwels(CBO)

Contradiction Point 3

Payer Policies and Contracting for Sephience

It involves differing perspectives on payer policies and contracting for Sephience, which could impact revenue projections and market access.

What is your outlook for Sephience's contract levels and inventory build expectations? - Brian Cheng (JPMorgan)

2025Q3: We are not planning to contract with payers at this stage. We will monitor gross to net dynamics over time. - Eric Pauwels(CBO)

What are the contracting levels for Sephience, and are there expectations for inventory build-up? - Brian Cheng (JPMorgan)

2025Q2: Minimal restrictions are expected in payer meetings. - Eric Pauwels(CBO)

Contradiction Point 4

Pricing Strategy and Expectations

It involves differing expectations and communications around the pricing strategy for Sepiapterin/PKU, which impacts revenue and market positioning.

What is the net price per patient with $19.6 million revenue from 341 patients? What is the average duration of treatment per patient and the current retention rate? - Gina Wang (Barclays)

2025Q3: The net price for Sephience, our PKU product, is consistent with our expectation. It's in the range that we've talked about previously. - Eric Pauwels(CBO)

Who are the target prescribers for Sepiapterin, and who are the near-term rapid adopters? What is the pricing strategy for PKU? - Kelly Shi (Jefferies)

2024Q4: Our pricing for Sepiapterin is expected to reflect its differentiated dual mechanism for treating PKU. We expect payers to recognize Sepiapterin's value proposition and accept a premium price for it. - Eric Pauwels(CBO)

Contradiction Point 5

Market Opportunity and Patient Profile

It involves differing views on the market opportunity and patient profile for Sepiapterin/PKU, which impacts product positioning and addressable market size.

What is the patient profile for new prescribers and the uptake across different groups? - Clara Dong (Jefferies)

2025Q3: We're seeing contributions from all segments, including different age groups and treatment histories. It's early, but we're already seeing a full spectrum of patients. We view Sephience as a highly differentiated rare disease therapy, expecting potential benefit across 17,000 U.S. patients. - Dr. Matthew Klein(CEO)

How is PTC approaching diet liberalization in PKU, and how should we assess the market opportunity for Friedreich's ataxia? - Kristen Kluska (Cantor Fitzgerald)

2024Q4: For PKU, the key factor is allowing patients to liberalize their diet. Sepiapterin shows over 97% of patients can liberalize their diet. Sepiapterin aims to benefit therapy-naive patients, those poorly controlled on current therapies, and classical PKU patients. - Matthew Klein(CEO)