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RO7204239 · 4 trials · 6 indications
| Arm | Type | Description |
|---|---|---|
| Placebo + Tirzepatide | ACTIVE_COMPARATOR | Participants will receive RO7204239 matching placebo via subcutaneous (SC) injection every 4 weeks (Q4W) for the core treatment period of 48 weeks and the treatment extension period of 24 weeks. Participants will also receive tirzepatide, as a background therapy, up-titrated according to the approved tirzepatide prescribing information, SC, every week (QW) during the core treatment period of 48 weeks. |
| RO7204239 low dose + Tirzepatide | EXPERIMENTAL | Participants will receive RO7204239, low dose, SC, Q4W for the core treatment period of 48 weeks. During the treatment extension period participants will receive placebo for the period of 24 weeks. Participants will also receive tirzepatide, as a background therapy, up-titrated according to the approved tirzepatide prescribing information, SC, QW during the core treatment period of 48 weeks. |
| RO7204239 medium dose + Tirzepatide | EXPERIMENTAL | Participants will receive RO7204239, medium dose, SC, Q4W for the core treatment period of 48 weeks. During the treatment extension period participants will receive placebo for the period of 24 weeks. Participants will also receive tirzepatide, as a background therapy, up-titrated according to the approved tirzepatide prescribing information, SC, QW during the core treatment period of 48 weeks. |
| RO7204239 high dose + Tirzepatide | EXPERIMENTAL | Participants will receive RO7204239, high dose, SC, Q4W along with tirzepatide, given as a background therapy, up-titrated according to the approved tirzepatide prescribing information, SC, QW during the core treatment period of 48 weeks. During the treatment extension period participants will be randomized to receive RO7204239 or matching placebo, SC, Q4W for 24 weeks. |
| Placebo | PLACEBO_COMPARATOR | Participants will complete a 4-week pre-treatment period to collect baseline movement data with a wearable device, then receive subcutaneous (SC) placebo every 4 weeks for 52 weeks. After the treatment period, participants will have the option to receive RO7204239 for an additional 52 weeks. |
| RO7204239 | EXPERIMENTAL | Participants will complete a 4-week pre-treatment period to collect baseline movement data with a wearable device, then receive SC RO7204239 every 4 weeks for 52 weeks. After the treatment period, participants will have the option to receive RO7204239 for an additional 52 weeks. |
| RO7204239 + Risdiplam | EXPERIMENTAL | Participants who have not previously been treated with risdiplam will receive risdiplam for at least 8 weeks prior to randomization into a treatment group (Part 1 only). Participants that have been treated with risdiplam for at least 8 continuous weeks immediately prior to joining the study may be immediately randomized to combination therapy, or join the study run-in period (the period between screening and randomization to a treatment group) where they will continue to receive risdiplam monotherapy until randomization. Participants enrolled in Part 1 will receive RO7204239 (low or high dose) + risdiplam for 24 weeks, followed by RO7204239 + risdiplam for 72 weeks. Participants enrolled in Part 2 will receive risdiplam for 8 weeks and then treatment with RO7204239 + risdiplam for 72 weeks. Once the treatment period has completed (Part 1 or Part 2), participants will have the option of treatment with RO7204239 + risdiplam for 2 additional years. |
| Placebo + Risdiplam | ACTIVE_COMPARATOR | Participants who have not previously been treated with risdiplam will receive risdiplam for at least 8 weeks prior to randomization into a treatment group (Part 1 only). Participants that have been treated with risdiplam for at least 8 continuous weeks immediately prior to joining the study may be immediately randomized to combination therapy, or join the study run-in period (the period between screening and randomization to a treatment group) where they will continue to receive risdiplam monotherapy until randomization. Participants enrolled in Part 1 will receive placebo (low or high dose-matched) + risdiplam for 24 weeks, followed by RO7204239 + risdiplam for 72 weeks. Participants enrolled in Part 2 will receive risdiplam for 8 weeks and then treatment with placebo + risdiplam for 72 weeks. Once the treatment period has completed (Part 1 or Part 2), participants will have the option of treatment with RO7204239 + risdiplam for 2 additional years. |
| Name | Type | Description |
|---|---|---|
| RO7204239 | DRUG | RO7204239 or matching placebo will be administered as per the schedule specified in the arms. |
| RO7204239 Matching Placebo | DRUG | RO7204239 matching placebo will be administered as per the schedule specified in the arm. |
| Tirzepatide | DRUG | Tirzepatide will be administered as per the schedule specified in the arms. |
| Placebo | DRUG | Participants will receive subcutaneous (SC) placebo every 4 weeks (Q4W) |
| Risdiplam | DRUG | Risdiplam will be administered orally once daily (QD) for the duration of the study. |
Inclusion Criteria: * BMI ≥ 30.0 kilograms per square meter (kg/m²) (additional weight-related comorbidities are not required for inclusion) * BMI ≥ 27.0 kg/m² and \< 30.0 kg/m² with at least one weight-related comorbidity such as: hypertension, dyslipidemia, obstructive sleep apnea and any cardiov...
RO7204239 is an investigational small molecule being developed by Roche Holding AG. It is in Phase 2 clinical development and is being studied across several conditions, including facioscapulohumeral muscular dystrophy, spinal muscular atrophy, obesity, overweight, and type 2 diabetes mellitus. It has not been approved by the FDA.
RO7204239 targets myostatin, a protein that normally limits muscle growth. By acting on myostatin, the drug is designed to influence muscle mass and composition. This mechanism underlies its study in muscle-wasting and metabolic conditions such as facioscapulohumeral muscular dystrophy, spinal muscular atrophy, and type 2 diabetes with overweight or obesity.
RO7204239 is being developed by Roche Holding AG, which trades under the ticker RHHBY. Roche is running the clinical program, including a Phase 2 study of RO7204239 in combination with tirzepatide in participants with obesity or overweight and at least one weight-related comorbidity.
RO7204239 is in Phase 2 clinical development. It is investigational and has not been approved by the FDA. The Phase 2 program includes studies in facioscapulohumeral muscular dystrophy, spinal muscular atrophy in combination with risdiplam, and obesity or overweight in combination with tirzepatide.
RO7204239 is being studied in several trials. NCT06965413 is a Phase 2 study with tirzepatide in obesity or overweight with a weight-related comorbidity. NCT05548556 is a Phase 2 study in facioscapulohumeral muscular dystrophy. NCT05115110 is a Phase 2 study with risdiplam in spinal muscular atrophy. NCT07137585 is a Phase 1 study in type 2 diabetes with overweight or obesity.
RO7204239 is being studied as an investigational treatment for facioscapulohumeral muscular dystrophy, a muscle-wasting condition. A Phase 2 trial, NCT05548556, is evaluating the drug in participants with FSHD across sites in the United States, Denmark, Italy, and the United Kingdom. It has not been approved for this or any indication.