PTC THERAPEUTICS, INC. (PTCT)

PTC Therapeutics is a science-driven global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines for patients with rare disorders. Its stated mission is to provide access to best-in-class treatments for patients who have little to no treatment options, and its strategy is to use its scientific and clinical expertise and its global commercial infrastructure to bring therapies to patients. Its portfolio includes several commercial products and product candidates in clinical, pre-clinical and research and discovery stages, targeting rare diseases in neurology, metabolism and oncology.

The commercial portfolio centers on a global Duchenne muscular dystrophy (DMD) franchise with two products: Translarna (ataluren), authorized in the European Economic Area, Russia and Brazil for nonsense mutation DMD, and Emflaza (deflazacort), approved in the United States for DMD in patients two years and older. Translarna's EEA authorization is conditional and subject to annual renewal, and it remains an investigational drug in the United States after an FDA Complete Response Letter in 2017. Other products include Upstaza, a gene therapy for AADC deficiency approved in the EEA and the United Kingdom, with a U.S. BLA submission expected in the first half of 2023. The company also holds Latin America and Caribbean commercialization rights to Tegsedi (inotersen) and Waylivra (volanesorsen) under a license with a subsidiary of Ionis Pharmaceuticals, and began commercial sales of both in Brazil in 2022. Evrysdi (risdiplam) for spinal muscular atrophy is approved in the U.S., the EU, Brazil and Japan and comes from the company's SMA program and its collaboration with Roche and the SMA Foundation. The development pipeline is organized by platform. The splicing platform includes PTC518 for Huntington's disease, in Phase 2. The Bio-e platform includes vatiquinone, in Phase 2/3 for mitochondrial disease seizures and Phase 3 for Friedreich ataxia, and utreloxastat, in Phase 2 for ALS. The metabolic platform includes sepiapterin, in Phase 3 for phenylketonuria. The gene therapy platform includes programs for Friedreich ataxia and Angelman syndrome. The oncology platform includes unesbulin, in Phase 2/3 for leiomyosarcoma, with a Phase 2/3 trial in diffuse intrinsic pontine glioma planned. The company also invests in multi-platform pre-clinical discovery, including potential collaborations, acquisitions and licensing.

Its customers are patients with rare diseases, with a commercial footprint spanning the United States, the EEA, the United Kingdom, Russia, Brazil, Japan and other Latin American markets. In DMD, the company estimates 10,000 to 15,000 patients in the United States and that nonsense mutations account for about 13% of cases. Commercialization in the EEA depends on marketing authorization, on country-by-country market access, pricing and reimbursement negotiations, and on early access programs.

On the operating side, the company works with strategic partners including Roche, the SMA Foundation and Ionis, and as marketing authorization holder it must run pharmacovigilance and report safety information to the EMA. The text provided does not describe suppliers, manufacturing, competitors or headquarters.