Chiasma is a clinical-stage biopharmaceutical company focused on improving the lives of patients who face challenges associated with their existing treatments for rare and serious chronic diseases. Employing its Transient Permeability Enhancer (TPE®) technology platform, Chiasma is developing octreotide capsules (conditionally trade named Mycapssa®), for the maintenance therapy of adult patients with acromegaly in whom prior treatment with somatostatin analogs has been shown to be effective and tolerated.
Octreotide capsules are in late-stage clinical development, with three global Phase 3 studies at various stages of completion: (1) The CHIASMA OPTIMAL trial completed in June 2019 was conducted under a Special Protocol Assessment (SPA) agreement reached with the U.S. FDA; (2) the fully enrolled and randomized MPOWERED™ trial is being conducted under a protocol evaluated and accepted by EMA; and (3) a single-arm, open label Phase 3 trial was completed in 2015, with results published in the Journal of Clinical Endocrinology and Metabolism (JCEM).
CHIASMA OPTIMAL -- Chiasma conducted a Phase 3 clinical trial under a Special Protocol Assessment agreement reached with the U.S. Food and Drug Administration for its octreotide capsules product candidate for the maintenance therapy of adult patients with acromegaly. The trial, referred to as “CHIASMA OPTIMAL” (Octreotide capsules vs. Placebo Treatment In MultinationAL centers), 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. Chiasma announced in October 2018 that it completed enrollment with 56 patients in 17 countries, including 21 from the United States, which exceeded its original SPA-agreed enrollment goal of 50 patients. The CHIASMA OPTIMAL trial reported positive topline clinical results in July 2019. The pivotal trial met its primary endpoint and met all four secondary endpoints.
MPOWERED™ -- Chiasma also is conducting an international Phase 3 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 MPOWERED™ (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 intended to support approval in the European Union. Chiasma plans to enroll up to 150 adult acromegaly patients into 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 the randomization was completed in January 2020. Chiasma expects to release topline data in the fourth quarter of 2020.
The company resubmitted the NDA for Mycapssa for the maintenance therapy of adult patients with acromegaly in December 2019 and was notified of its acceptance in January 2020. The FDA assigned a Prescription Drug User-Fee Act (PDUFA) target action date of June 26, 2020, which is a six-month review.
Completed open label Phase 3 – The company completed an international Phase 3 trial of octreotide capsules, the results of which have been published in JCEM (Melmed S et al. J Clin Endocrinol Metab. 2015 Apr).
Chiasma is committed to working with healthcare professionals in order to improve the lives of patients with acromegaly. Chiasma is headquartered in the United States with a wholly-owned subsidiary in Israel.