Patients taking Crinetics’ rare growth disease drug were able to shift to the oral medicine from their standard of care injections while maintaining control of a key biomarker and symptoms during a late stage study.
Paltusotine is under investigation in the PATHFNDR program to treat patients with acromegaly, a rare hormone disorder that causes abnormal growth and swelling in adults. The condition occurs when the pituitary gland produces too much growth hormone, starting in the hands and the feet, and can progress to severe illness or death.
The condition is typically treated with injectable depot somatostatin drugs, which aim to block the growth hormones. Crinetics hopes to offer an oral alternative with paltusotine, and the data from the phase 3 PATHFNDR-1 trial suggest patients were able to switch over “seamlessly,” according to principal investigator Monica Gadelha, M.D., Ph.D., professor of endocrinology at the Medical School of the Universidade Federal do Rio de Janeiro.
Crinetics shares shot up 62% in premarket trading Monday morning to $25.94 compared to $15.97 at close Friday. The company in August suspended the rare disease therapy CRN04777 that had been on clinical hold.
Data show that insulinlike growth factor 1 (IGF-1) levels were maintained in 83% of patients taking the drug, compared to just 4% on placebo, which was the main goal of the study.
Paltusotine also hit on the secondary goals, which included symptom control, change in IGF-1 levels and maintenance of growth hormone levels.
As for safety, Crinetics said the therapy was well tolerated with no serious or severe adverse events reported. Treatment-emergent adverse events were similar between the treatment and placebo groups, with 80% of patients taking paltusotine experiencing at least one compared to 100% in the control arm. The most commonly reported events were joint pain, headache, diarrhea, abdominal pain and nausea.
Patients taking the study drug also had a lower rate of adverse events associated with acromegaly, at a rate of 30% in the treatment group and 86% for placebo.
“We designed paltusotine to be the preferred therapeutic option for people living with acromegaly. We could not be more excited by the results from PATHFNDR-1, which further reinforce our conviction that, if approved, paltusotine could address patients’ unmet need for a simple, oral, once-daily therapy,” said Scott Struthers, Ph.D., Crinetics founder and CEO, in a press release.
“These data showed that upon switching from injected standard of care, paltusotine provided reliable, durable control of their disease.”
Before heading to regulators with the data, Crinetics must now wrap up the phase 3 PATHFNDR-2 trial, which is testing paltusotine in patients who have not received treatment for their condition, Struthers noted. Completion of the trial is expected “early next year” with a readout in the first quarter of 2024. A regulatory filing with the FDA for all acromegaly patients could follow that year if the PATHFNDR-2 results are positive.
A full analysis of the PATHFNDR-1 study will be presented at a future medical conference. Crinetics is also conducting a phase 2 open label study of paltusotine in patients with carcinoid syndrome, with results expected later this year.