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MYODA CLINICAL PROGRAM

The company was granted FDA IND approval in December 2019 to initiate a Phase 1/2 clinical proof of concept trial (MYODA-INT) of an oral pediatric formulation of Sarconeos (BIO101), utilizing a ‘seamless’ clinical trial design with composite clinical 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.