Acromegaly is a rare disorder caused by a tumor on a person’s pituitary gland that causes the release of too much growth hormone, often enlarging hands and feet and internal organs, which, if left untreated, can be deadly.
If surgery or radiation treatment cannot remove the tumor, the current standard of care is a medication, which can lower growth hormone levels but require monthly shots into the gluteal muscle. istered with large-gauge needles, these shots are painful and inconvenient.
For years, San Diego’s Crinetics Pharmaceuticals has worked to develop Paltusotine, a once-per-day oral medication capable of interacting with the receptor that regulates growth hormone, allowing a significant reduction without shots.
On Sunday, Crinetics announced preliminary results from its ongoing Phase 3 clinical trial, indicating that 83 percent of patients with acromegaly who took the drug were able to maintain lower growth hormone levels, a statistically significant result when compared with 4 percent who received a placebo.
Scott Struthers, Crinetics’ founder and CEO, said that it took more than a few tries to discover a molecule capable of doing the job that could also survive the human digestive system.
“Paltusotine was originally called CRN 808, because it took us 808 molecules to get to the one that was good enough,” Struthers said.
Acromegaly is estimated to affect 26,000 people in the United States and the executive said that investigation is already under way on the new drug’s suitability to treat another medical condition called carcinoid syndrome, which affects about 30,000 nationwide.
If approved by the U.S. Food and Drug istration, Paltusotine would be the company’s first drug on the market, a milestone that, according to a statement released by the company, would validate its ability to design and deploy small molecules capable of targeting specific receptors capable of interacting with the endocrine system.
After completion of a second Phase 3 trial still under way, Struthers said the FDA review will take about one year.
“We expect to get a decision in 2025,” he said.
Struthers, who holds a doctorate in physiology and pharmacology from UC San Diego, had his first research job at Salk Institute studying somatastatin somatostatin, a short chain of amino acids called a peptide, that is key in regulating hormones.
The researcher studied the pituitary gland under the late Wylie Vale at Salk which was home to Roger Guillemin, who won a Nobel Prize in 1977 for discoveries that led to research on brain hormones such as somatostatin.
Paltusotine, then, has a strong San Diego lineage, and it’s one that Struthers said Crinetics, which is growing rapidly, intends to continue.
“We’re trying to solve all of the endocrine diseases, it’s a huge ambition,” Struthers said, noting that work is already under way on compounds to treat other hormone-related conditions such as Cushing’s disease, congenital adrenal hyperplasia, Graves’ disease and hyperthyroidism.
“The thing about endocrinology is that every cell in every animal on the planet is regulated in one way or another by a hormone or some other hormone-like substance,” Struthers said. “By intervening in those pathways, you can regulate all kinds of different things that go wrong in the body.”
