Chiasma Reports Positive Results from Acromegaly Trial to Support EMA Submission

Rare Daily Staff

Chiasma, a company developing oral versions of injectable treatments for rare diseases, reported positive top-line results from a late stage study of comparing its oral capsule Mycapssa to long-acting injectable somatostatin analogs for maintenance of patients with acromegaly.

Acromegaly is a rare chronic disease often caused by a benign pituitary tumor and characterized by excess production of growth hormone and insulin-like growth factor-1 hormone that is frequently treated with chronic burdensome injections. If left untreated, acromegaly can lead to serious, and sometimes life-threatening medical conditions such as type 2 diabetes, hypertension, respiratory disorders, and cardiac and cerebrovascular disease. Chiasma estimates that approximately 8,000 patients are on injectable somatostatin analogs (SSA) in the United States.

The U.S. Food and Drug Administration approved Mycapssa in June as the first and only oral SSA and the first FDA approved product developed using Chiasma’s proprietary TPE technology, which is designed to convert selected peptide-based injectables into oral formulations. Although most peptides are degraded in the stomach, but Mycapssa is protected by an enteric coating so that it can safety reach the small intestine for absorption into the bloodstream.

The MPOWERED trial was designed to support a planned marketing authorization application for Mycapssa in the European Union. It is a non-inferiority trial that compared long-term maintenance treatment with Mycapssa to the long-acting injectables octreotide long-acting release and lanreotide autogel, in patients with acromegaly who had previously responding to these therapies. After a six-month run-in phase, 92 patients who were responders to Mycapssa were randomized to a nine-month controlled phase with continued treatment on Mycapssa or on their prior injectable therapy.

The study met its primary non-inferiority endpoint. Some 91 percent of patients on Mycapssa maintained insulin-like growth factor 1 (IGF-1) response compared to 100 percent on injectable SSAs. Response was defined as the time-weighted average of IGF-1 <1.3 x upper limit of normal during the 9-month randomized, controlled treatment phase.

Mycapssa also maintained mean IGF-1 within normal limits and was comparable to injectable therapy

“These results further strengthen the available robust clinical data set for Mycapssa and provide additional meaningful information for healthcare providers in treating patients with acromegaly,” said Raj Kannan, CEO of Chiasma.

Based on the results, the company plans to submit a marketing authorization application for Mycapssa to the European Medicines Agency in mid-2021.

Further analyses of the trial results are ongoing, and Chiasma plans to present the full data set from the study at upcoming medical conferences in 2021.

Photo: Raj Kannan, CEO of Chiasma