MYODA clinical program in Duchenne muscular dystrophy innovative trial adaptive seamless design and composite score
Biophytis
Non-confidential report
Biophytis proposed a small, orally bioavailable compound (BIO101) to treat Duchenne muscular dystrophy (DMD) and improve muscle function, mobility, and respiratory function. Three phases to the phase 1/3 study are planned:
- Part 1- multiple ascending dose (MAD) safety, tolerability and PK study
- Part 2- proof of concept (POC) study
- Part 3- confirmatory study to investigate efficacy and safety/tolerability.
TACT provided feedback and advice on all areas of the application including on biomarker studies, pre-clinical data, dosing and trial design. In addition, the applicant’s specific questions were addressed. This were around scales to be used, meeting the concerns of patients, patient selection and any additional needs in the pre-clinical plan for a clinical program.
Name of applicant: Mounia Chabane
Reviewed: April, 2019 in Glasgow, UK
Biophytis proposed a small, orally bioavailable compound (BIO101) to treat Duchenne muscular dystrophy (DMD) and improve muscle function, mobility, and respiratory function. Three phases to the phase 1/3 study are planned:
- Part 1- multiple ascending dose (MAD) safety, tolerability and PK study
- Part 2- proof of concept (POC) study
- Part 3- confirmatory study to investigate efficacy and safety/tolerability.
TACT provided feedback and advice on all areas of the application including on biomarker studies, pre-clinical data, dosing and trial design. In addition, the applicant’s specific questions were addressed. This were around scales to be used, meeting the concerns of patients, patient selection and any additional needs in the pre-clinical plan for a clinical program.