A Phase 3 clinical trial, ACT CF, assessing PTC Therapeutics' (NASDAQ:PTCT) ataluren (Translarna) in nonsense mutation cystic fibrosis (nmCF) failed to achieve its primary or secondary endpoints. As a result, the company will terminate its development of ataluren in CF, close its ongoing extension studies and withdraw its marketing application in Europe. <\/p>\n
CEO Stuart Peltz, Ph.D., says, \"We are disappointed with the outcome of this trial as there are no treatments that target the underlying cause of nonsense mutation cystic fibrosis, one of the most difficult forms to treat.We are particularly grateful to patients and investigators who participated in our trials. We remain committed to patients receiving ataluren in other indications.\" <\/p>\n
Translarnais a protein restoration therapy designed to enable the formation of a functioning protein in patients with nonsense mutation-caused genetic disorders. It is conditionally approved in Europe to treat Duchenne muscular dystrophy. <\/p>\n
Nasdaq does not list any premarket trades yet, but investors should expect a significant down move. <\/p>\n
Update: Shares are down24%premarket on robust volume. <\/p>\n
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PTC Therapeutics' ataluren flunks late-stage CF study; shares slump 24% premarket - Seeking Alpha<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" A Phase 3 clinical trial, ACT CF, assessing PTC Therapeutics' (NASDAQ:PTCT) ataluren (Translarna) in nonsense mutation cystic fibrosis (nmCF) failed to achieve its primary or secondary endpoints. As a result, the company will terminate its development of ataluren in CF, close its ongoing extension studies and withdraw its marketing application in Europe. CEO Stuart Peltz, Ph.D., says, \"We are disappointed with the outcome of this trial as there are no treatments that target the underlying cause of nonsense mutation cystic fibrosis, one of the most difficult forms to treat.We are particularly grateful to patients and investigators who participated in our trials. Continue reading