PTC Therapeutics

40.550722°N 74.423071°W / 40.550722; -74.423071

PTC Therapeutics, Inc.
TypePublic
Nasdaq: PTCT
Russell 2000 Component
IndustryPharmaceuticals
Founded1 January 1998 Edit this on Wikidata
Headquarters
South Plainfield, New Jersey
,
United States
Websiteptcbio.com

PTC Therapeutics is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases.[1][2]

In September 2009, PTC entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system diseases.[3] PTC acquired the Bio-e platform in 2019.[4]

Products

In 2017, PTC acquired Emflaza (deflazacort) from Marathon Pharmaceuticals.[5] PTC also owns Translarna, (Ataluren) marketed for nonsense mutation Duchenne muscular dystrophy.[6] Together, the two products generated revenues of 174 million dollars and 260 million dollars in 2017 and 2018 respectively.[7]

PTC has the commercialization rights for WAYLIVRA (volanesorsen) in Latin America.[8]

Pipeline

In 2018, PTC acquired Agilis Biotherapeutics and a gene therapy candidate, GT-AADC, with its compelling clinical data in treating aromatic L-amino acid decarboxylase (AADC) deficiency.[9] AADC deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase (DDC) gene.

In 2020, PTC acquired Censa Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic diseases, including phenylketonuria (PKU) and other diseases associated with defects in the tetrahydrobiopterin (BH4) biochemical pathways diagnosed at birth.[10]

In 2020, PTC announced the FDA approval of Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older.[11]

References
  1. Bhutta, Sunya (December 2013). "Biopharma Blossoming in the Garden State". Biobusiness. Gen. Eng. Biotechnol. News. Vol. 33, no. 21. p. 15.
  2. Bain, Lisa J (2006). "Drug development in critical times". NeuroRx. 3 (4): 540–3. doi:10.1016/j.nurx.2006.08.004. PMC 3593415. PMID 17044149.
  3. Bratulic, Anna (2009-09-02). "Roche, PTC Therapeutics to partner on CNS drug discovery - FirstWord Pharma". FirstWord Pharma. Retrieved 2016-05-14.
  4. Press Release https://www.ptcbio.com/our-science/expertise/bio-e-electron-transfer/
  5. House, SA Editor Douglas W. (2017-03-16). "PTC acquires DMD med Emflaza from Marathon Pharma for up to $190M". Seeking Alpha. Retrieved 2018-07-03. {{cite news}}: |first= has generic name (help)
  6. "In The Spotlight: PTC Therapeutics". NASDAQ.com. 2018-07-23. Retrieved 2018-07-24.
  7. "In The Spotlight: PTC Therapeutics". NASDAQ.com. 2018-07-23. Retrieved 2018-07-24.
  8. Press Releasehttps://www.ptcbio.com/our-pipeline/approved-medicines/
  9. PTC Therapeutics website
  10. PTC Therapeutics website
  11. Press Releasehttps://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-approval-evrysditm-risdiplam

Further reading
  • Official website
  • Business data for PTC Therapeutics, Inc.:
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