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

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.

"We are excited to collaborate with CHDI to advance our small-molecule huntingtin-lowering program for the treatment of Huntington's disease," said Stuart W. Peltz, Ph.D., president and chief executive officer of PTC Therapeutics. "Finding a potential treatment for Huntington's disease fits with our mission to focus on treatments for disorders that have an urgent need for therapeutic options. We are proud that this program utilizes our proprietary splicing platform developed utilizing cutting edge technology to advance the identification of new therapeutics for patients suffering from rare diseases."

"After following PTC's Huntington's disease small-molecule program for several years, we are pleased to start working with the company more directly," said Robi Blumenstein, president of CHDI Management. "It's great that PTC's pre-mRNA splicing expertise has been applied to Huntington's disease with promising results. A pill that lowers the amount of huntingtin protein and treats the underlying cause of the disease holds the promise of improving the quality of life of people with Huntington's disease and their families. We look forward to contributing to and accelerating this program."

The discovery and development of small-molecule huntingtin-expression inhibitors has been enabled by the PTC pre-mRNA splicing platform, a technology that previously has been successfully used to identify the SMN2 splicing modifier, RG7916. PTC, F. Hoffmann-La Roche AG, and the SMA Foundation have a collaboration using RG7916, which is currently in pivotal clinical trials for the treatment of SMA. The splicing technology platform is also being used in a number of other programs, including a program in late-stage chemical optimization, in collaboration with the Massachusetts General Hospital, to identify correctors of IKBKAP splicing to treat familial dysautonomia.