Mallinckrodt Comments on its BRAVE study

Mallinckrodt Comments on its BRAVE study (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)

DECEMBER 19, 2019 -(MALLINCKRODT)- After careful consideration and despite engaging in diligent efforts to research, develop and commercialize MNK-1411, Mallinckrodt has made the decision to terminate Study MNK14112096.  The Study was designed to investigate the efficacy and safety of MNK-1411 in Duchenne Muscular Dystrophy (DMD) patients.  The decision was based on several factors, including:

• The trial had a target enrollment of 132 patients. Despite diligent efforts on the part of Mallinckrodt and the participating study sites, as of December 17, 2019 only 55 patients had been screened and 44 patients randomized since the start of the phase 2 study on July 17, 2018.
• At the current rate of enrollment, it would take a significantly long time to achieve an appropriately powered study, by which time some of the collected data might no longer be relevant or easily evaluated.
• Collecting specific data, such as pharmacokinetic and some motor function test results, has proven to be challenging. This has placed a significant burden and challenge on both the physicians and the patient and caregiver population.
• Additionally, the other competing clinical trials in the younger DMD population, along with several changes in standard of care and clinical practice for treating DMD since the initiation of the clinical study, have made enrollment in the study very difficult.

While this is a disappointing outcome, we are optimistic about the numerous trials that are ongoing in DMD, and we hope these trials yield treatments that help those living with DMD.  Mallinckrodt remains committed to research designed to help underserved patients living with severe and critical diseases, and thanks all of the patients and caregivers who participated in the study and clinical trial sites for their support.

Mallinckrodt, the “M” brand mark and the Mallinckrodt Pharmaceuticals logo are trademarks of a Mallinckrodt company. Other brands are trademarks of a Mallinckrodt company or their respective owners.

© 2019 Mallinckrodt. US-1902285 12/19