PTC Therapeutics, Inc. Q3 FY2025 earnings call

Management Statement and Operational Highlights:

• Sephience Launch: Initiated global launch in Europe and U.S. Generated $19.6 million in Q3. Saw enthusiasm with 521 patient start forms from U.S. centers. Presented results from AMPLIFY study showing Sephience reduced phenylalanine levels by 70% more than BH4. Finalizing publication on mechanism of action.
• Other Programs: Votoplam Huntington's disease program plans Q4 FDA meeting to align study design for next efficacy study and discuss accelerated approval. Vatiquinone Friedreich's ataxia program to meet FDA this quarter. Translarna NDA remains under FDA review.
• Financial Position: Ended Q3 with ~$1.68 billion in cash. Hosting R&D Day on December 2nd to share progress on research programs.
• Sephience Commercial: Strong initial numbers in October. U.S. payer engagement positive with favorable policies. Launch in Germany off to good start. Received approval in Canada; anticipate regulatory approval in Japan and Brazil by year-end.

Segment Performance:

• Sephience: Third quarter revenue totaled $19.6 million, with $14.4 million in the U.S. and $5.2 million outside the U.S. As of September 30, 521 patient start forms were received from U.S. centers, indicating demand across various patient segments.
• DMD Franchise: Generated $86 million in revenue in the third quarter. Translarna contributed $51 million, and Emflaza contributed $35 million.
• Evrysdi Royalty: Roche's third quarter global revenue was approximately $532 million, resulting in $71 million in royalty revenue for PTC.

Guidance:

• Narrowed 2025 full year revenue guidance to $750 million to $800 million, upper end of initial range. Confidence in execution of Sephience launch and stable performance of legacy products, particularly Emflaza, contributed to narrowing the guidance.

Risks:

• Forward-looking statements subject to certain risks and uncertainties where actual results may differ materially from expectations. Detailed risks and uncertainties described in SEC filings.

Q: Congrats on an amazing start for Sephience. What's going to give confidence that beyond this strong out-of-the-gate launch, you're going to see maintained durability and that patients will be on therapy for a while?

A: Kristen, thanks so much for the question. We're seeing broad interest in trying patients, initial feedback of good response in terms of lowering Phe and diet liberalization. Clinical studies showed up to 75% response rate in full spectrum patients. Physicians are converting and wanting to try all patients on Sephience.

Q: Congrats on a really strong launch out of the gate. Can you talk about what magnitude of Phe reduction are doctors and payers looking for in order to keep a patient on treatment beyond the trial period?

A: Thanks for the question, Tazeen. Hearing different things from different folks. Some patients with severe never-had-a-therapy tolerance/response see 15% Phe reduction as meaningful. Others look for 30% or patient feeling better. Clinical study showed up to 75% response rate.

Q: Congrats on the launch. Can you talk about the time line from a new start form to a patient receiving a prescription and actually getting the drug?

A: Thanks for the question, Brian. Our PTC Cares team provides white glove service. Start forms are processed quickly. PTC Cares team works with physicians' offices, nurse practitioners to get start forms processed and patients on drug as quickly as possible. On average, 2 to 4 weeks from start form to fill, depending on plan.

Q: A couple on Sephience. Can you help with the narrowing of the full year guide? Is that solely tied to Sephience? Any other moving pieces?

A: Absolutely. The narrowing to upper end of guidance is due to Emflaza performing better than expected despite generic entrants, along with strong Sephience numbers.

Q: Really congrats on the strong launch. I was a little bit surprised to hear that you're seeing switches even from Palynziq. Is that an outlier?

A: Jim, thanks so much for the questions. We're just reporting what we're seeing and hearing from the field. Physicians and care teams realize Sephience can provide significant benefit to patients with severe mutations, including classical PKU patients, due to its once-a-day oral well-tolerated profile.

Q: I want to share my congrats as well on the strong launch. Can you provide a bit more granularity on if you're seeing these initial patients are skewing more towards pediatric or adults?

A: Thanks so much for the question, Sami. We're seeing a good mix, full age spectrum from infants to septuagenarians. Average is late adolescent around 17 years old. Still early days to know better.

Q: Congrats on a strong launch so far. Can you quantify for us how the response rate for Sephience is tracking in the real world based on refills or any other metrics you have?

A: Joe, it's too early to give granular numbers. We started with first shipment in August. Hearing very good response rates and seeing good momentum in October refills, but early days.