Chiasma's octreotide capsules (conditionally trade named Mycapssa®), are being developed for the maintenance treatment of acromegaly, a rare, serious chronic disease typically caused by a benign tumor of the pituitary gland that releases excess growth hormone (GH), leading to excess growth of certain parts of the body.
We believe octreotide capsules exhibit the effects of somatostatin, a naturally occurring hormone that reduces the production of GH by binding to receptors on specialized cells in the pituitary gland. There are currently no approved oral formulations of octreotide. Octreotide capsules were developed using Chiasma's proprietary Transient Permeability Enhancer (TPE®) technology platform designed to facilitate gastrointestinal absorption of unmodified drug into the bloodstream safely.
The company completed an international Phase 3 trial of octreotide capsules, the results of which have been published in the Journal of Clinical Endocrinology and Metabolism.
Chiasma conducted a Phase 3 clinical trial, referred to as “CHIASMA OPTIMAL” (Octreotide capsules vs. Placebo Treatment In MultinationAL centers) under a Special Protocol Assessment agreement reached with the U.S. Food and Drug Administration (FDA) to support a potential resubmission of a New Drug Application with the FDA. CHIASMA OPTIMAL is a global, randomized, double-blind, placebo-controlled, nine-month trial. The trial is designed to evaluate the proportion of patients who maintain their biochemical response to octreotide capsules compared to placebo. The company completed enrollment with 56 adult acromegaly patients in CHIASMA OPTIMAL in October 2018 and completed the trial in June 2019. The company reported positive topline clinical results in July 2019. The pivotal trial met its primary endpoint and met all four secondary endpoints.
Chiasma also is conducting an international Phase 3 pivotal clinical trial under a protocol accepted by the European Medicines Agency (EMA) for the company's octreotide capsules product candidate for the maintenance therapy of adult patients with acromegaly. The trial, referred to as MPOWEREDTM (Maintenance of Acromegaly Patients with Octreotide Capsules Compared with Injections - Evaluation of REsponse Durability), is a global, randomized, open-label and active-controlled, 15-month trial. Chiasma plans to enroll up to 150 adult acromegaly patients in the trial, of which at least 80 patients who are responders to octreotide capsules following a six-month run-in will be randomized to either octreotide capsules or injectable somatostatin receptor ligands (octreotide or lanreotide), and then followed for an additional nine months. Patients are only randomized into the nine-month randomized controlled phase of MPOWERED if they are qualified as responders (IGF-1 <1.3 x ULN and GH<2.5 ng/mL) to octreotide capsules in the study at the end of the six-month run-in phase. The trial was initiated in March 2016 and has enrolled 135 patients as of July 2018 (of which the EMA-required minimum of 80 patients have been randomized). In October 2018, Chiasma announced that it had elected to resume enrollment in the trial in an effort to enroll up to 15 additional patients exclusively located in the United States in order to gain further U.S. investigator and patient experience with octreotide capsules. Chiasma has completed enrollment in the trial in June 2019 and expects to release topline data in the second half of 2020.