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ABOUT Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a rare, genetic neuromuscular disease in male children characterized by accelerated degeneration of muscles and is responsible for a loss of mobility, respiratory failure and cardiomyopathy, leading to premature death. It is the most common form of muscular dystrophy in children, affecting approximately one in 5,000 newborn boys (approximately 20,000 new cases annually worldwide). DMD is caused by a certain number of mutations in the dystrophin gene that results in the absence or very low levels of functional dystrophin, a cytoskeletal protein that protects muscle cells.

The absence of dystrophin in muscle severely weakens the structural and membrane stability of the muscle fibers, resulting in the loss of muscle strength and mobility, impaired respiratory function and cardiac defects. DMD evolves according to a very well understood progression with symptoms that are similar to those associated with accelerated aging across all stages.

In preclinical animal studies, we observed a positive effect on muscle function, mobility, and respiratory function (a major disability in later stage DMD disease progression) in mdx mice models of DMD that were treated with Sarconeos (BIO101).

In 2018, we received orphan drug designation from the FDA and EMA for Sarconeos (BIO101) in DMD. We plan to submit an IND application to the FDA and clinical trial applications to the applicable regulatory agencies in Europe in the second half of 2019 to commence clinical development through our MYODA clinical program, utilizing a seamless clinical trial design with composite clinical endpoints, subject to regulatory approval.

MYODA CLINICAL PROGRAM

We plan to submit an IND application to the FDA and clinical trial applications to the applicable regulatory agencies in Europe in the second half of 2019 to initiate clinical development of an oral pediatric formulation of Sarconeos (BIO101) for DMD through our MYODA clinical program, which could start as early as 2020, subject to regulatory approval of our study plan and clinical trial design, and study endpoints.

In connection with MYODA clinical program, we intend to use a ‘seamless’ clinical trial design that will encompass all three phases of clinical development (Phase 1 to Phase 3), allowing patients to participate in one or more phases, with composite clinical endpoints comprised of key functional measurements, including mobility, strength and respiratory function. The MYODA clinical trial aims to encompass two distinct stages: (i) a Phase 1/2 clinical proof of concept stage (MYODA-INT), followed by (ii) Phase 3 confirmatory stage.

MYODA-INT PHASE 1/2 CLINICAL TRIAL

The MYODA-INT Phase 1/2 clinical trial is expected to recruit, assess and treat around 48 ambulatory and non-ambulatory DMD patients for up to 52 weeks in a dose escalating study. The first week of dosing will be mainly for assessment of safety, tolerability, and pharmacokinetic (PK) objectives (Phase 1). Dosing will continue seamlessly into Phase 2, provided that no serious safety issues arise during the first seven days of the treatment. An interim analysis will take place after all 48 subjects have finished 12 weeks of dosing, and thereafter for every 12-week period until either success or futility is achieved. Upon completion of the MYODA-INT stage of the seamless trial (up to 52 weeks), participants may be enrolled in an open-label extension period. Regulatory approval will be required before progressing into and commencing the confirmatory stage (Phase 3).

The MYODA clinical program and our study design and clinical trial protocols are subject to regulatory approval and will be submitted to regulatory agencies for review. We plan to work with the regulatory agencies to finalize the protocols and additional challenges and risks remain with our innovative clinical trial program.