PTC Therapeutics Inc (PTCT)

[chainma1l]

PTC Therapeutics announces its JV w/ Roche (RHHBY) and the SMA Foundation initiated a clinical study (FIREFISH) in infants with Type I SMA

FIREFISH is a two-part study.
•Part one is an open-label, dose escalation study in at least 8 infants for a minimum of 4 weeks. ?The primary objective of part one is to assess the safety profile of RG7916 in infants and determine the dose for part two.

•Part two is an open-label, single-arm study in !40 infants with Type I SMA for 24 months, followed by an open-label extension. ?The primary objective of part two is to assess the efficacy of RG7916 at the selected dose over a 12-month treatment period.

•A trial in childhood onset (Type II/III) SMA patients, SUNFISH, is currently enrolling the dose escalation portion of the study.
•Commencement of the pivotal portion of either study will trigger a $20 mln milestone payment to co from Roche (RHHBY).

This was announced a while back, but PTC announced the initiation of the study today. This started in 2006, so hopefully things can move along to help the kids who are affected.

http://www.prnewswire.com/news-releases/ptc-therapeutics-announces-initiation-of-firefish-study-in-infant-type-i-sma-patients-300386262.html