PTC Therapeutics (PTCT) R&D Day 2025 summary

Pipeline overview and program updates

• Focused R&D strategy on small molecule therapies, leveraging unique platforms in splicing and ferroptosis, with dedicated facilities in New Jersey and California supporting cross-functional collaboration.

• Oral small molecule splicing platform features late-stage programs for Huntington's disease, myotonic dystrophy, spinocerebellar ataxia, and brain tumors, with early-stage efforts in sickle cell disease, β-thalassemia, and neurodegenerative diseases.

• PT-Seq and PTSeek™ platforms enable systematic discovery and rapid optimization of novel small molecule splicing modulators, expanding the universe of druggable RNA targets across genetic, neurodegenerative, oncologic, and hematologic diseases.

• Inflammation and ferroptosis platform advances both preclinical and clinical programs for CNS and non-CNS indications, targeting enzyme hubs like 15-LO, NRF2, NLRP3, and DHODH, including Parkinson's disease, inflammatory lung diseases, and autoimmune disorders.

Clinical trial data and development milestones

• Lead splicing programs include late-stage candidates for nucleotide repeat disorders (Huntington's, DM1), SCA3, oncology, sickle cell disease, and neurodegeneration, with clinical candidate selection for several programs targeted for early 2026.

• Votoplam (PTC518) demonstrated HTT reduction in Huntington's disease patients in a Phase 2 trial.

• MSH3 and SCA3 programs plan clinical candidate selection in early 2026, with Phase 1 readiness expected late 2026.

• Parkinson's disease program targeting 15-LO/ferroptosis demonstrated proof of concept in multiple models, with development candidate selection in Q1 2025 (or Q1 2026) and Phase I planned for H2 2025 (or H2 2026).

• NLRP3 inhibitor PTC612 and DHODH inhibitor PTC844 have shown best-in-class potency and selectivity in preclinical and Phase I studies, with Phase I/II studies planned for 2026.

R&D strategy and innovation priorities

• Emphasis on oral, titratable small molecules for CNS and systemic diseases, leveraging learnings from Evrysdi (SMA) and Votoplam (HD) to accelerate new program development.

• Focus on expanding the druggable target space by leveraging RNA splicing modulation and proprietary PTSeek™ technology.

• Strategic focus on indications with high unmet need and strong scientific rationale, using translational biomarkers and surrogate endpoints to streamline clinical development.

• Emphasis on developing first-in-class or differentiated therapies for neurodegenerative, neuromuscular, metabolic, oncology, and hematological diseases.