Ann: Company Re-branding, page-10

Some more interesting connections and overlaps….

PYC Therapeutics, Phylogica’s proposed new rebranding, is a name not too dissimilar to PTC Therapeutics.

PTC Therapeutics happens to be a rival company to Sarepta Therapeutics. Both have drugs for Duchenne muscular dystrophy. The two companies competed to be first to receive FDA approval for a treatment for Duchenne. Sarepta’s antisense therapy, Exondys51 (eterplirsen), was ultimately given the nod by the FDA whereas PTC’s (non-antisense) Translarna (ataluren) was rejected. However, PTC won (conditional) marketing approval for Translarna in the EU, whereas Sarepta’s Exondys51 was knocked back. PTC hasn’t yet given up on FDA approval for Translarna– it’s adding new data and has plans to resubmit its NDA.

Like Sarepta, PTC’s therapeutic focus is on orphan drugs. Its products and programs span across multiple “core platforms” - nonsense mutation read-through, alternative splicing, gene therapy and oncology.

But PTC has now added another core platform - antisense DNA technology.

Last year, PTC announced it had purchased the Latin American licensing rights for two rare disease drugs, Tegsedi and Waylivra. Both are mature-stage antisense drugs that were developed by Ionis Pharmaceuticals and held through its affiliate company, Akcea Therapeutics.

This month, PTC has given an indication that it is moving into the development of its own antisense drugs. A newly created position has been advertised for a Senior Scientist in ASO chemistry to “advance PTC’s research interests in antisense oligonucleotides as a therapy for genetic disorders. “

In a separate move related to its gene therapy platform, PTC last month announced a strategic licensing agreement with Odylia Therapeutics to develop gene therapies in rare inherited retinal diseases utilizing a vector (the Anc80 vector system developed by researchers at Massachusetts Eye and Ear). The lead program in the collaboration with Odylia will target RP-GRIP1, a ciliary protein that is defective in Leber Congenital Amaurosis 6 (LCA6), a rare early onset childhood retinal dystrophy.


https://au.finance.yahoo.com/news/ptc-therapeutics-ptct-q2-2019-062410273.html
http://ir.ptcbio.com/static-files/dcb0da30-f237-4787-9e34-974c0bd958f9
http://ir.ptcbio.com/news-releases/...therapeutics-and-ptc-therapeutics-collaborate
https://www.linkedin.com/jobs/view/sr-scientist-aso-chemistry-at-ptc-therapeutics-inc-1407444659
https://www.biospace.com/article/re...licensing-agreement-with-odylia-therapeutics/