PTC Therapeutics (PTCT) The 44th Annual William Blair Growth Stock Conference summary

Strategic focus and operational changes

• Refocused R&D on small molecule therapies and areas of unique expertise, such as ferroptosis and splicing.

• Discontinued low-probability programs and reduced workforce by 30%, decreasing 2024 OpEx by over 25%.

• Achieved a significant shift in OpEx trajectory, enabling a path to cash flow break-even.

• Completed royalty financing, retired restrictive debt, and maintained robust global commercial infrastructure.

• Six products in commercial portfolio, with five marketed globally for rare diseases.

Financial and commercial performance

• 2023 total revenue reached $938 million, with $611 million from the DMD franchise.

• Continued strong revenue performance in 2024, with upside from ongoing Translarna sales in Europe.

• Revenue guidance for 2024 was suspended due to regulatory uncertainty but will be reinstated once clarity is achieved.

• European contribution to Translarna revenue is about 45%, with growth in other geographies expected.

Regulatory and clinical milestones

• BLA for Upstaza submitted in the US; already marketed in Europe, UK, Taiwan, and Israel.

• Upstaza BLA accepted with priority review; PDUFA action date expected in November.

• NDA for sepiapterin (PKU) to be submitted by Q3; marketing application under review in Europe.

• NDA resubmission for Translarna to FDA planned for mid-year.

• Top-line results for utreloxastat in ALS expected in Q4; NDA for vatiquinone (Friedreich ataxia) by year-end.