PTC Therapeutics, Inc.
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- PTC Therapeutics, Inc. (PTCT) is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines for rare disorders, including Duchenne muscular dystrophy (DMD), aromatic L-amino acid decarboxylase (AADC) deficiency, phenylketonuria (PKU), Friedreich's ataxia (FA), and spinal muscular atrophy (SMA). Founded in 1998 and headquartered in Warren, New Jersey, the company offers approved therapies such as Translarna (ataluren), a small molecule splicing modifier for nonsense mutation DMD; Emflaza (deflazacort), a corticosteroid for DMD; Evrysdi (risdiplam), an SMA treatment developed in collaboration with Roche and the SMA Foundation; and Kebilidi (eladocagene exuparvovec-tneq), the first gene therapy directly infused into the brain for AADC deficiency, approved by the FDA in November 2024. Its pipeline includes sepiapterin for PKU, with an FDA action date of July 29, 2025; vatiquinone for FA, with filing acceptance anticipated in Q1 2025; PTC518, an oral therapy for Huntington's disease; and other candidates targeting post-transcriptional control mechanisms in orphan diseases. PTC operates globally, with commercial activities in the U.S. and select international markets, generating net product revenues of approximately $814 million in 2024, driven by strong DMD franchise performance. Recent developments include a $1.0 billion upfront licensing deal with Novartis for PTC518 closed in late 2024, FDA approval of Kebilidi, regulatory submissions for sepiapterin, vatiquinone, and Translarna label expansion, and a cash balance of $2.1 billion as of year-end 2024 following the Novartis proceeds.
PTC Therapeutics (PTCT) Moves 7.2% Higher: Will This Strength Last?
PTC Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
PTC Therapeutics Announces Pricing of Convertible Notes Offering to Refinance 2026 Convertible Notes
PTC Therapeutics Announces Pricing of Convertible Notes Offering to Refinance 2026 Convertible Notes
PTC Therapeutics Announces Proposed Convertible Notes Offering to Refinance 2026 Convertible Notes
PTC Therapeutics, Inc. (PTCT) Presents at Goldman Sachs 47th Annual Global Healthcare Conference 2026 Transcript
PTC Therapeutics, Inc. (PTCT) Presents at Bank of America Global Healthcare Conference 2026 Transcript
PTC Therapeutics: Sephience Turns The Story From Regulatory Repair To Operating Leverage
PTC Therapeutics Q1 Earnings Call Highlights
PTC Therapeutics, Inc. (PTCT) Q1 2026 Earnings Call Transcript
Compared to Estimates, PTC Therapeutics (PTCT) Q1 Earnings: A Look at Key Metrics
PTC Therapeutics (PTCT) Reports Q1 Loss, Tops Revenue Estimates
PTC Therapeutics Provides Corporate Update and Reports First Quarter 2026 Financial Results
Exploring Analyst Estimates for PTC Therapeutics (PTCT) Q1 Earnings, Beyond Revenue and EPS
PTC Therapeutics to Participate in Upcoming Investor Conferences
PTC Therapeutics (PTCT) Expected to Beat Earnings Estimates: What to Know Ahead of Q1 Release
PTC Therapeutics, Inc. (PTCT) Discusses 24-Month Interim Results of PIVOT-HD Long-Term Extension Study of Votoplam in Huntington's Disease Transcript
PTC Therapeutics Stock Sinks As Pivotal Data Fails To Calm Sellers
PTC Therapeutics Reports Positive Topline Results from Month 24 Interim Analysis of PIVOT-HD Extension Study of Votoplam
PTC Therapeutics to Report Results from PIVOT-HD Long Term Extension Study