PTC Therapeutics
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40°33′03″N 74°25′23″W / 40.550722°N 74.423071°W
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| Company type | Public |
|---|---|
| Nasdaq: PTCT Russell 2000 Component | |
| Industry | Pharmaceuticals |
| Founded | January 1, 1998 |
| Headquarters | , U.S. |
| Website | ptcbio |
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]
Products
[edit]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
[edit]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
[edit]- ^ Bhutta, Sunya (December 2013). "Biopharma Blossoming in the Garden State". Biobusiness. Gen. Eng. Biotechnol. News. Vol. 33, no. 21. p. 15.
- ^ 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.
- ^ Bratulic, Anna (2009-09-02). "Roche, PTC Therapeutics to partner on CNS drug discovery - FirstWord Pharma". FirstWord Pharma. Retrieved 2016-05-14.
- ^ Press Release https://www.ptcbio.com/our-science/expertise/bio-e-electron-transfer/
- ^ 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) - ^ "In The Spotlight: PTC Therapeutics". NASDAQ.com. 2018-07-23. Retrieved 2018-07-24.
- ^ "In The Spotlight: PTC Therapeutics". NASDAQ.com. 2018-07-23. Retrieved 2018-07-24.
- ^ Press Releasehttps://www.ptcbio.com/our-pipeline/approved-medicines/
- ^ PTC Therapeutics website
- ^ Commissioner, Office of the (2024-11-14). "FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency". FDA. Retrieved 2024-12-03.
- ^ "Upstaza | European Medicines Agency (EMA)". www.ema.europa.eu. 2022-08-09. Retrieved 2024-12-03.
- ^ PTC Therapeutics website
- ^ Press Releasehttps://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-approval-evrysditm-risdiplam
- ^ Joseph, Andrew (2024-12-02). "Novartis to pay $1 billion upfront to license Huntington's drug from PTC". STAT. Retrieved 2024-12-08.
- ^ Gao, Lan; Bhattacharyya, Anuradha; Beers, Brian; Kaushik, Diksha; Bredlau, Amy-Lee; Kristensen, Allan; Abd-Elaziz, Khalid; Grant, Richard; Golden, Lee; Kong, Ronald (2024). "Pharmacokinetics and pharmacodynamics of PTC518, an oral huntingtin lowering splicing modifier: A first-in-human study". British Journal of Clinical Pharmacology. 90 (12): 3242–3251. doi:10.1111/bcp.16202. ISSN 1365-2125. PMC 11602954. PMID 39155237.
- ^ Gao, Lan; Bhattacharyya, Anuradha; Beers, Brian; Kaushik, Diksha; Bredlau, Amy-Lee; Kristensen, Allan; Abd-Elaziz, Khalid; Grant, Richard; Golden, Lee; Kong, Ronald (2024). "Pharmacokinetics and pharmacodynamics of PTC518, an oral huntingtin lowering splicing modifier: A first-in-human study". British Journal of Clinical Pharmacology. 90 (12): 3242–3251. doi:10.1111/bcp.16202. ISSN 0306-5251. PMC 11602954. PMID 39155237.
- ^ "PTC Reports Positive Phase II Huntington's Data, FDA Lifts Partial Clinical Hold". BioSpace. 2024-06-20. Retrieved 2024-12-08.
Further reading
[edit]- Schneider, Ilene (2009). "Firm Aims to Regulate RNA in Rare Diseases". Genetic Engineering & Biotechnology News. 29 (8): 14. Retrieved 2009-04-25.
External links
[edit]- Official website
- Business data for PTC Therapeutics, Inc.:
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