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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...
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RO7204239 is an investigational small molecule being studied for facioscapulohumeral muscular dystrophy (FSHD), spinal muscular atrophy (SMA), type 2 diabetes mellitus, and obesity. It is in Phase 2 clinical development for FSHD and SMA, and Phase 1 for type 2 diabetes. It is not yet approved by the FDA.
RO7204239 targets myostatin, a protein that regulates muscle growth. By inhibiting myostatin, the drug aims to promote muscle mass and function, which is relevant for conditions like FSHD and SMA where muscle weakness is a key feature.
RO7204239 is being developed by Roche Holding AG, traded on the OTC market under the ticker RHHBY. Roche is conducting multiple clinical trials to evaluate the drug's safety and efficacy across different indications.
RO7204239 is in Phase 2 clinical development for facioscapulohumeral muscular dystrophy and spinal muscular atrophy. It is also being studied in a Phase 1 trial for type 2 diabetes mellitus. The drug is investigational and has not received FDA approval.
RO7204239 is being studied in several trials: NCT05115110 for spinal muscular atrophy, NCT05548556 for facioscapulohumeral muscular dystrophy, NCT06965413 for obesity, and NCT07137585 for type 2 diabetes mellitus. All trials are active but not recruiting participants.
No, RO7204239 is not the same as risdiplam. RO7204239 is a myostatin inhibitor, while risdiplam is a separate drug. However, they are being studied together in a Phase 2 trial (NCT05115110) for spinal muscular atrophy to assess the combination's safety and efficacy.