PTC Therapeutics

PTC Therapeutics, Inc.

Type| Public

Traded as

* NASDAQ: PTCT
* Russell 2000 component
* S&P 600 component

Industry| Pharmaceuticals
Founded| January 1, 1998; 28 years ago (1998-01-01)
Headquarters|

Warren Township, New Jersey

U.S.

Website| ptcbio.com

PTC Therapeutics, Inc. 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]

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. As of 2024, the AAV based treatment for AADC deficiency has been approved by the FDA and commercialized in the United States as Kebilidi. It is the first FDA-approved gene therapy treatment for AADC deficiency.[10] This same product was granted EMA approval for use in the European Union under the name Upstaza.[11]

## Pipeline

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.[12]

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.[13]

In 2024, Novartis has committed $1 billion upfront in a licensing deal with PTC Therapeutics for an experimental treatment for Huntington’s disease, with the potential for an additional $1.9 billion tied to achieving specific development, regulatory, and commercial milestones.[14] PTC Therapeutics’ therapy, known as PTC518, is an oral medication designed to reduce the production of the mutant protein linked to the genetic mutation responsible for the disease.[15] This mutant protein is believed to contribute to neuronal death, driving disease progression.[16] In June, interim data from a Phase 2 trial indicated that PTC518 successfully lowered levels of the mutant protein in both blood and cerebrospinal fluid.[17]

## 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, 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. ↑ Commissioner, Office of the (2024-11-14). "FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency" (in en). https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-aromatic-l-amino-acid-decarboxylase-deficiency.
11. ↑ "Upstaza | European Medicines Agency (EMA)" (in en). 2022-08-09. https://www.ema.europa.eu/en/medicines/human/EPAR/upstaza.
12. ↑ PTC Therapeutics website
13. ↑ Press Releasehttps://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-approval-evrysditm-risdiplam
14. ↑ Joseph, Andrew (2024-12-02). "Novartis to pay $1 billion upfront to license Huntington's drug from PTC" (in en-US). https://www.statnews.com/2024/12/02/novartis-ptc-licensing-huntingtons/?utm_campaign=pharmalittle&utm_medium=email&_hsenc=p2ANqtz-989P4LHe7rbwf8R63_SbHjIGL8afV0d9Mt5HeRJKuIbCRMWZrQsbciYxnZSVNfSvJdasiKMqIiMRURv1IBOuVO44fIxQ&_hsmi=336765670&utm_content=336765670.
15. ↑ Gao, Lan; Bhattacharyya, Anuradha; Beers, Brian; Kaushik, Diksha; Bredlau, Amy-Lee; Kristensen, Allan; Abd-Elaziz, Khalid; Grant, Richard et al. (2024). "Pharmacokinetics and pharmacodynamics of PTC518, an oral huntingtin lowering splicing modifier: A first-in-human study" (in en). British Journal of Clinical Pharmacology 90 (12): 3242–3251. doi:10.1111/bcp.16202. ISSN 1365-2125. PMID 39155237.
16. ↑ Gao, Lan; Bhattacharyya, Anuradha; Beers, Brian; Kaushik, Diksha; Bredlau, Amy-Lee; Kristensen, Allan; Abd-Elaziz, Khalid; Grant, Richard et al. (2024). "Pharmacokinetics and pharmacodynamics of PTC518, an oral huntingtin lowering splicing modifier: A first-in-human study" (in en). British Journal of Clinical Pharmacology 90 (12): 3242–3251. doi:10.1111/bcp.16202. ISSN 0306-5251. PMID 39155237.
17. ↑ "PTC Reports Positive Phase II Huntington's Data, FDA Lifts Partial Clinical Hold" (in en-US). 2024-06-20. https://www.biospace.com/ptc-reports-positive-phase-ii-huntington-s-data-fda-lifts-partial-clinical-hold.

## 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

0.00

(0 votes)

Original source: https://en.wikipedia.org/wiki/PTC Therapeutics. Read more

|

Retrieved from "https://handwiki.org/wiki/index.php?title=Company:PTC_Therapeutics&oldid=4256014"