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June 2014
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Prosensa Completes Enrollment in Natural History Study of Duchenne Muscular Dystrophy

ProSensa

Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, announced on June 25 the successful completion of enrollment in its prospective Natural History Study designed to increase the understanding of Duchenne muscular dystrophy (DMD).

The study enrolled in total 269 boys with confirmed DMD between the ages three and 18 from 10 countries across 16 centers in North & South America and Europe, 80% of whom are ambulatory and 20% non-ambulatory.

The purpose of the study is to characterize the natural history and progression of DMD, which will help inform the design of future studies, to capture biomarkers of safety and disease progression, and to provide comparative data for patients with rare exon deletions/mutations for which formal controlled trials are not feasible. No medication or device is being tested in this study. Moreover, the study will be important in the confirmatory program for Prosensa's lead DMD compound drisapersen, for which a New Drug Application is expected to be submitted later this year to the FDA under an Accelerated Approval pathway.

Dr. Brenda Wong of Cincinnati Children's Hospital, who has enrolled patients in the study said, "While we are pleased by the high level of world-wide attention and commitment by regulators given to DMD, an existing obstacle for delivering promising treatments to patients relies on accepted clinical endpoints for human studies. By collecting and analyzing this Natural History Study data from hundreds of boys, we are better equipped to expedite the development of promising therapies and offer them to DMD patients anxiously awaiting treatment options to slow disease progression and improve the outcome of this disease."

"We continue to work diligently to pioneer research and development in DMD, and completing enrollment in our Natural History Study is a major milestone and a critical component of basic research to understand the disease," said Dr. Giles Campion, Prosensa's Chief Medical Officer. "This study will be a crucial resource for our confirmatory studies with drisapersen and for our follow-on exon skipping programs. Outcomes of this study will contribute to a growing body of scientific knowledge and are fundamental to our ability to develop innovative treatment options for boys who suffer from this devastating disease."

All boys participating in the study will be followed for a period of three years, with site visits and assessments every 6 months. During these visits, investigators will evaluate the boys on a number of physical tests, including the six minute walk test, climbing stairs, breathing in a tube to measure lung function and performing upper limb movements. These parameters are meant to assess how the disease affects their overall quality of life as the condition evolves over time in addition to contributing to the understanding of the natural progression of the disease. Furthermore, blood and urine samples will be taken to investigate a panel of biomarkers that may be useful in following disease progression.

The study, which began in September 2012 and is sponsored by Prosensa, is expected to complete in November 2017. This autumn, 80 boys will have completed one year of observation and this interim analysis will be available later this year. Further information on the study can be found here: www.ClinicalTrials.gov - Study ID: NCT01753804

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