PRIME designation granted by European Medicines Agency for RG6042 for treatment of Huntington’s disease

European Medicines Agency PRIME (PRIority MEdicines) status is granted to medicines that
may offer a major therapeutic advantage over existing treatments, or benefit patients without
treatment options

RG6042 has the potential to be the first therapy targeting the underlying cause of Huntington’s
disease, a fatal neurodegenerative rare disease

Third PRIME designation for a Roche medicine

Basel, 3 August 2018 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency (EMA) has granted PRIME (PRIority MEdicines) designation for the company’s investigational medicine RG6042 Read more…

PTC Therapeutics and CHDI Foundation Announce a Collaboration on a Small-Molecule Therapeutic for Huntington’s Disease

SOUTH PLAINFIELD, N.J., April 4, 2018 – PTC Therapeutics, Inc. (NASDAQ: PTCT) and the CHDI Foundation, Inc. (CHDI) today announced a research collaboration to advance PTC’s Huntington’s disease program. The program is optimizing small-molecule compounds—identified using PTC’s splicing technology platform—that decrease the production of huntingtin protein. The current compounds have been shown to be orally bioavailable in animals, blood-brain barrier penetrant, and effective in decreasing the amount of huntingtin protein in a mouse model that has the expanded human huntingtin transgene. Read more…