Clinical TrialsLow bandwidth at academic study centers has driven slower than anticipated site activation and ultimately, slower progression to the 50mg dose.
Regulatory ApprovalIf the FDA does not accept frataxin levels as an endpoint or if it does not accept the submission of nomlabofusp for accelerated approval, then the stock price would decline.
Research And Development RiskThere is an additional layer of risk since frataxin is not currently an accepted surrogate endpoint, and thus Larimar Therapeutics needs to produce evidence that frataxin levels can predict clinical benefit.