PTC Therapeutics

Short description: Pharmaceutical company

[ ⚑ ]  40°33′03″N 74°25′23″W﻿ / ﻿40.550722°N 74.423071°W﻿ / 40.550722; -74.423071

PTC Therapeutics, Inc.

Type| Public

Traded as

| NASDAQ: PTCT
Russell 2000 Component
Industry| Pharmaceuticals
Headquarters|

South Plainfield, New Jersey

United States

Website| ptcbio.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]

## Contents

* 1 Products
* 2 Pipeline
* 3 References
* 4 Further reading
* 5 External links

## 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". Gen. Eng. Biotechnol. News 33 (21): p. 15. http://www.genengnews.com/gen-articles/biopharma-blossoming-in-the-garden-state/5075/.
2. ↑ Bain, Lisa J (2006). "Drug development in critical times". NeuroRx 3 (4): 540–3. doi:10.1016/j.nurx.2006.08.004. PMID 17044149.
3. ↑ Bratulic, Anna (2009-09-02). "Roche, PTC Therapeutics to partner on CNS drug discovery - FirstWord Pharma". FirstWord Pharma. http://www.firstwordpharma.com/node/375238.
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" (in en-US). Seeking Alpha. https://seekingalpha.com/news/3251624-ptc-acquires-dmd-med-emflaza-marathon-pharma-190m.
6. ↑ "In The Spotlight: PTC Therapeutics" (in en-us). NASDAQ.com. 2018-07-23. https://www.nasdaq.com/article/in-the-spotlight-ptc-therapeutics-20180723-00195.
7. ↑ "In The Spotlight: PTC Therapeutics" (in en-us). NASDAQ.com. 2018-07-23. https://www.nasdaq.com/article/in-the-spotlight-ptc-therapeutics-20180723-00195.
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

* Schneider, Ilene (2009). "Firm Aims to Regulate RNA in Rare Diseases". Genetic Engineering & Biotechnology News 29 (8): 14. http://www.genengnews.com/articles/chitem.aspx?aid=2869. Retrieved 2009-04-25.

## External links

* Official website
* Business data for PTC Therapeutics, Inc.:

* Google Finance
* Yahoo! Finance
* Bloomberg
* Reuters
* SEC filings

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