RG6206 (also known as RO7239361) is an investigational molecule being developed for people with Duchenne.
RG6206 is called ‘investigational’ as it is not approved by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) or similar government agencies abroad. Our ongoing clinical trial will assess the efficacy, safety, and tolerability of RG6206 in boys with Duchenne.
RG6206 is given once weekly by an injection under the skin (subcutaneous, or SC, injection) into the tummy (abdomen). The first three weekly doses must be done at the study site. The remaining doses can be done at home.
RG6206 binds to a protein called myostatin and limits its function.
Myostatin is a naturally occurring protein that is produced by the body to stop muscles growing too large. Limiting myostatin has been shown in some studies to increase muscle mass. The hope is that stopping myostatin could increase muscle growth in children with Duchenne.
Yes. An initial clinical trial (phase 1) has shown that RG6206 is well tolerated in healthy adults. Further information can be found here NCT03100630 on ClinicalTrial.Gov.
RG6206 is currently being studied in a phase 1/2 study to investigate its safety and tolerability in boys with Duchenne. The trial is ongoing, but is no longer open to new patients. You can find more information here: NCT02515669
Our SPITFIRE phase 2/3 clinical trial is to assess the efficacy, safety, and tolerability of RG6206 in boys with Duchenne. Enrollment in the SPITFIRE clinical study is ongoing globally. For more information please visit our About The Trial and Find My Trial pages. Further information can also be found here NCT03039686.