2023 is a meaningful, unique and thrilling year for PTC. It’s the company’s 25th anniversary, and it is poised to be a year of momentum as we anticipate significant trial readouts in multiple disease areas. We’re also working toward our ambitious goal to produce a therapy every two to three years based on our robust and diversified platform. This year, we also look forward to celebrating our enduring legacy and delivering on our promise to create more moments for those who count on us.
What’s so special about 2023? Read below to learn:
PTC’s 25th Anniversary
This year, we celebrate 25 years of translating science and transforming lives. We can’t wait to share our proudest achievements, moments and impact throughout our history with you. Stay tuned throughout the year to learn about the company’s history and the incredible people who made an impact on PTC, celebrate with our growing global community of #OnePTC employees and look ahead to the next 25 years.
Significant Trial Readouts
We’re anticipating data readouts from several clinical trials:
- MIT-E: Registration-directed trial of vatiquinone for Mitochondrial Disease Associated Seizures (MDAS)
- MOVE-FA: Registration-directed trial of vatiquinone for Friedreich ataxia (FA)
- APHENITY: Registration-directed trial of sepiapterin for phenylketonuria (PKU)
Anticipated Treatment Milestones
We plan to submit a Biologics License Application (BLA) to the U.S. FDA in the first half of this year for Upstaza™ (eladocagene exuparvovec), our innovative gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency.
Continued Pipeline Advancement
Our pipeline continues to progress:
- Enrollment in the PIVOT-HD Phase 2 trial of PTC518 for HD is active and ongoing at study sites in Europe and Australia
- Our SUNRISE LMS clinical trial of unesbulin in leiomyosarcoma is ongoing
Supporting Patient Voices and Experiences
- This year we launched the ninth annual STRIVE awards, our program that provides grants to non-profit patient advocacy organizations serving the Duchenne community
- We’ll hear even more first-hand perspectives and stories from patients and caregivers through our podcast My VIBE: My Voice is Inspirational, Brave and Empowering
Hope you’re as excited about 2023 as we are!