PPMD is excited to learn that Mallinckrodt enrolled the first patient in their Phase 2 study assessing the efficacy and safety of the investigational drug MNK-1411 (cosyntropin, also known as tetracosactide). We look forward to learning more about this trial.
For more information, please read the full press release here.
First Patient Enrolled in Mallinckrodt’s Phase 2 Duchenne Muscular Dystrophy Trial
— Trial to Assess Efficacy and Safety of Investigational Drug MNK-1411 in Patients with DMD — — European Medicines Agency Grants MNK-1411 Orphan Medicinal Product Designation —STAINES-UPON-THAMES, United Kingdom, Aug. 2, 2018 /PRNewswire/ — Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today confirmed enrollment of the first patient in the company’s Phase 2 study assessing the efficacy and safety of investigational drug MNK-1411 (cosyntropin, also known as tetracosactide) in patients ages four to eight with Duchenne Muscular Dystrophy (DMD).
“We are very excited Mallinckrodt has chosen to study how this investigational drug, MNK-1411, might help in treating Duchenne Muscular Dystrophy,” said Pat Furlong, President and CEO, Parent Project Muscular Dystrophy. “In the fight to end Duchenne Muscular Dystrophy, our community welcomes partners dedicated to this goal.”
The company also announced that MNK-1411 recently received the European Medicines Agency (EMA) Orphan Medicinal Product designation for the potential treatment of Duchenne Muscular Dystrophy.
“Mallinckrodt is focused on meeting the unmet medical needs of patients, particularly those with rare diseases like DMD,” said Steven Romano, MD, Executive Vice President and Chief Scientific Officer at Mallinckrodt. “We are pleased to enroll the first patient in our Phase 2 clinical trial of investigational drug MNK-1411 in boys with DMD. We are also excited by the recent EMA designation of Orphan Drug status as confirmation of the potential benefit of this investigational therapy for European patients.”
About the Clinical Trial
The Phase 2 clinical study is entitled: A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy (the BRAVE study). The study will include patients who have a diagnosis of DMD confirmed by complete dystrophin deficiency, or an identifiable mutation in the DMD gene, or complete dystrophin gene sequencing consistent with DMD; patients must also have a typical clinical profile consistent with DMD to be included.
Approximately 130 patients who meet eligibility criteria will be randomly assigned in a 2:1 ratio to receive one of two weight-based doses of MNK-1411, or they will receive volume-matched placebo, administered subcutaneously 2 times/week for 24 weeks. Study subjects who complete the 24-week randomized, double-blind, placebo-controlled treatment period will be eligible to receive active treatment for an additional 24 weeks during an optional open-label extension. Key efficacy outcome measures for the study will include timed function tests, such as a 10-meter walk/run, 4-stair climb and rise from floor, and the NorthStar Ambulatory Assessment.
Find more information about the U.S. trial here for the U.S. Food and Drug Administration’s ClinicalTrials.gov website. Details can be found here for Belgium and here for Spain on the European Union Clinical Trial register website.
MNK-1411 (cosyntropin), an investigational drug, is a depot formulation of cosyntropin acetate, a synthetic 24 amino acid melanocortin receptor agonist formulated for prolonged release.