Lexicon Pharmaceuticals’ (NASDAQ:LXRX) LX1032 (telotristat ethyl) for carcinoid syndrome has a positive FDA approval shot due to its encouraging Phase III data, which shows statistical significant improvement in reducing bowel movements (BMs), experts said. The oral therapy’s novel mechanism, which directly targets the cause of diarrhea, is also a cause for optimism, they added.
However, one expert said the Phase III trial’s primary endpoint – difference in daily BM frequency versus placebo – could draw some approval uncertainties as it is not a typical oncology endpoint. Others disagreed, noting this endpoint is clinically relevant and has direct impact on quality of life (QOL).
Although some experts noted potentially drug-related changes in mood and depression could affect telotristat use, others said more data is needed to confirm these potential effects. One expert said telotristat may unlikely cause this side effect as the drug is designed to not cross the blood-brain barrier.
Telotristat has a PDUFA date of 28 February. An analyst report states the drug has solid prospects for carcinoid syndrome with peak sales in excess of USD 300m.