Chiasma Reports Positive Late-Stage Results of Acromegaly Drug

Chiasma announced positive top-line data from the company’s pivotal phase 3 clinical trial evaluating its oral product candidate, octreotide, for the maintenance treatment of adults with acromegaly.

The pivotal study was a randomized, double-blind, placebo-controlled, nine-month study of octreotide capsules conducted under special protocol assessment agreement with the U.S. Food and Drug Administration. The primary endpoint was met with 58 percent of the patients on octreotide capsules maintaining their insulin-like growth factor response compared to 19 percent of the patients on placebo.  All secondary endpoints were met and there were no new or unexpected safety signals in the treated patients.

Acromegaly is a rare condition that typically develops when a benign tumor of the pituitary gland produces too much growth hormone, ultimately leading to significant health problems. Common features of acromegaly are facial changes, intense headaches, joint pain, impaired vision and enlargement of the hands, feet, tongue and internal organs. Serious health conditions associated with the progression of acromegaly include type 2 diabetes, hypertension, respiratory disorders and cardiac and cerebrovascular disease.

Chiasma says there are approximately 8,000 adult acromegaly patients who are chronically treated with injectable somatostatin analogs (SSAs) in the United States. Octreotide is an oral version of the injectable somatostatin analog that is the standard of care maintenance treatment for acromegaly.

“I consistently observe the burdens posed by SSA injections, including injection site pain and reactions.  I believe octreotide capsules, if approved, would be a welcome treatment option to patients and physicians,” said Susan Samson, medical director of the Pituitary Center at Baylor St. Luke’s Medical Center, and lead investigator of the study.

The FDA had rejected Chiasma’s new drug application in April 2016 as not providing substantial evidence of efficacy to warrant an approval. The company then worked with the FDA to design a trial that might give it a better chance for approval. Chiasma expects that on the heels of the positive outcome of the current study, it will submit a new application to the FDA by the end of the year.

Chiasma says that 90 percent of the patients completing the recent trial have opted to participate in an optional open-label expansion. If approved, Chiasma plans to give octreotide the trade name Mycapssa.

Photo: Susan Samson, medical director of the Pituitary Center at Baylor St. Luke’s Medical Center