- PTC Therapeutics shares made a >40% gain this week after its Duchenne Muscular Dystrophy drug Translarna reported data from a late-stage trial.
- The issue may be that the drug did not meet its primary endpoint, which makes an FDA approval unlikely and may put sales of the drug in Europe in jeopardy.
- PTC has made losses of nearly $1bn in the past two financial years and made a loss of $127m in Q122. Cash position is ~$550m.
- There are four more approval shots in rare diseases in play, albeit the markets are not necessarily large.
- Despite this week's gains, it's hard to look at the $2.9bn market cap and anticipate share price growth, despite management promising $8bn in revenues by 2030.
For further details see:
PTC Therapeutics: DMD Trial News Unlikely To Restore Flagging Share Price