PTC THERAPEUTICS, INC. Q4 FY2024 earnings call

2024 Achievements

• Strong commercial performance with fourth quarter revenue $213M and full year $807M exceeding guidance.
• Achieved all clinical and regulatory milestones in 2024, submitting four approval applications to the FDA all accepted for review.
• KEBILIDI approved in November, Sepiapterin has a regulatory action date of July 29, 2025, Translarna and Vatiquinone have regulatory action dates of August 19, 2025.

Collaboration

• Announced global development and commercialization collaboration with Novartis for PTC518 Huntington's disease program, receiving $1B upfront and up to $1.9B in milestones.

2025 Plans

• Anticipate global launch of Sepiapterin, potential launch of Vatiquinone in the US, and 12-month results from PIVOT-HD study for PTC518.

In the fourth quarter, total revenue was $213 million. The DMD franchise contributed $144 million in the fourth quarter, with Translarna net product revenue at $94 million and Emflaza at $50 million. Full year 2024 revenue was $807 million, exceeding guidance. The DMD franchise generated $547 million, Evrysdi royalty revenue was $204 million, and Roche royalty revenue in the fourth quarter was $58 million. Sepiapterin for PKU is being prepared for global launches, and Vatiquinone for Friedreich's ataxia is set for potential FDA approval in the US.

Revenue Guidance

• Provided 2025 total revenue guidance of $600 million to $800 million, including in-line products, potential new product launches, and royalty revenue from Evrysdi.

Cash Position

• Ended 2024 with over $1.1 billion in cash, and received $1B upfront from Novartis collaboration in January 2025, enabling execution of strategy, achieving milestones, and business development activities.

Risks

• Regulatory uncertainties related to approval timelines and decisions for products like Sepiapterin, Vatiquinone, and Translarna.
• Market competition in genericized and competitive markets affecting commercial performance.
• Macroeconomic uncertainties impacting the company's financial position and operations.

Q: On PKU, what's the sense of diet liberalization for Sepiapterin patients?

A: Over 97% of patients in the feed protocol can liberalize their diet, with about two thirds able to get to or beyond the recommended daily allowance of protein.

Q: On Friedreich's ataxia, how is the market opportunity thought about?

A: Focus on pediatric population with no available therapies, adult population with high unmet need, and considerations of visibility, cadence, and patient segments.

Q: On Translarna, what's the cadence of launch if approved in the US?

A: PTC has deep roots in DMD commercialization, with strong physician and patient conviction, and expects interest in starting new patients while authorization remains intact.

Q: On Huntington's PIVOT-HD trial, what's the expectation on improvement and patient split?

A: Expect continued benefit on TMS, cUHDRS, and TFC from 12 to 24 months, with stage 2 and 3 split similar to previous interim update but with more stage 3 data in the second quarter readout.