Judy

Judy asked about the first clinical proof of concept milestones that would validate the new splicing and inflammation programs highlighted at R&D Day, and how capital allocation is prioritized for these programs while targeting cash flow break-even.

Answer

Matthew Klein, CEO, stated that the splicing platform is well-validated by the success of Risdi and Votoplam. For the MSH3 molecule, the next step is achieving development candidate status this year and then moving into the clinic. For the inflammation platform, the NLRP3 inhibitor program is expected to enter Phase I by mid-year, with the next stage gate being safety and pharmacology. Pierre Gravier, CFO, emphasized disciplined OpEx management, focus on the Sephience launch and R&D programs, and openness to business development, all supported by a strong cash position to reach cash flow break-even.

Judy inquired about the first clinical proof of concept milestones and the catalyst path for PTC Therapeutics' new splicing and inflammation R&D programs, as highlighted at R&D Day. She also asked how capital allocation is being prioritized for these programs as the company targets cash flow break-even.

Answer

CEO Dr. Matthew Klein stated that the splicing platform is validated by Risdi and Votoplam's success. For the MSH3 program, the next step is achieving development candidate status this year and moving into the clinic. For the NLRP3 inhibitor, a Phase 1 study is expected by mid-year, with initial targets in lung inflammation and fibrosis. CFO Pierre Gravier emphasized disciplined OpEx management to reach cash flow break-even, with sufficient cash to support both commercial expansion and R&D programs, while also being open to business development.