Crinetics Pharmaceuticals released new clinical data on its oral acromegaly drug, paltusotine (PALSONIFY), at ENDO 2025. The data, from open-label extensions of the PATHFNDR-1 and PATHFNDR-2 trials, demonstrate the drug’s long-term efficacy and safety in maintaining stable IGF-1 levels and controlling symptoms. These studies evaluated paltusotine in both patients previously treated with injectable somatostatin receptor ligands (SRLs) and those who were treatment-naïve.
This data is particularly valuable for the acromegaly treatment landscape because it provides evidence for the long-term benefits of an oral medication. Currently, many patients rely on monthly injections, which can be burdensome. The sustained efficacy and tolerability demonstrated by paltusotine in these extension studies suggest it could become a preferred treatment option, offering patients a less invasive and more convenient alternative. This potential shift towards oral therapy could significantly improve patient compliance and quality of life.
In the PATHFNDR-1 OLE, patients transitioning from injectable SRLs to oral paltusotine maintained stable IGF-1 and growth hormone levels over 96 weeks. Symptom control also remained consistent, further reinforcing the drug’s long-term benefits. The PATHFNDR-2 OLE, which included previously untreated patients and those initially given placebo, also showed sustained reductions in IGF-1 levels, growth hormone, and improvements in symptoms. Importantly, paltusotine was generally well-tolerated in both studies. Additional analyses presented demonstrated reduced symptom burden and fewer symptom flares for patients on paltusotine.
These positive results solidify the potential of paltusotine to reshape the acromegaly treatment paradigm. Pending regulatory approval, the convenience of an oral therapy could lead to wider adoption, improving patient outcomes and adherence to therapy. This could also position Crinetics for significant growth within the acromegaly market and establish paltusotine as a leading treatment for this condition.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
