Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as REGN2477
Garetosmab · 3 trials · 2 indications
Treatment-emergent adverse events (TEAEs) are adverse events not present at baseline or represent the exacerbation of a pre-existing condition during the on-treatment period. A serious TEAE was defined as any untoward medical occurrence that resulted in any of following outcomes not present at baseline or represent the exacerbation of a pre-existing condition during the on-treatment period: death, life-threatening, required initial/prolonged in-participant hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect/considered as medically important event. Number of participants with TEAEs and Serious TEAEs are reported.
Severity of TEAEs were graded as follows: Mild: Does not interfere in a significant manner with the participant's normal functioning level. It may be an annoyance. Prescription drugs are not ordinarily needed for relief of symptoms but may be given because of personality of the participants. Moderate: Produces some impairment of functioning but is not hazardous to health. It was uncomfortable or an embarrassment. Treatment for symptom may be needed. Severe: Produces significant impairment of functioning or incapacitation and was a definite hazard to the participant's health. Treatment for symptom may be given and/or participants hospitalized. Number of participants with TEAEs by severity is reported.
18\^F-NaF PET is used to assess lesion and disease activity. Time-weighted average (standardized area under the curve \[AUC\]) of the percent change from baseline in total lesion activity by 18\^F-NaF PET up to Week 28 in AHO analysis set is reported.
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Percent change from baseline in the total volume of HO lesions as assessed by CT during Period 1 at Week 28 is reported.
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. HO detectable by CT that developed after baseline are referred to as "new HO lesions." Number of new HO lesions as assessed by CT at Week 56 relative to Week 28 scan is reported.
18\^F-NaF PET is used to assess lesion and disease activity. Time-weighted average (Standardized AUC) of the percent change from baseline in total lesion activity as assessed by 18\^F-NaF PET in Active HO Classic ACVR1 Mutation (AHOC) analysis set up to Week 28 is reported.
CT is a diagnostic imaging test used to create detailed images of internal organs, bones, soft tissue, and blood vessels. CT scan acquired contemporaneously to the PET scan. Percent change from baseline in the total volume of HO lesions was assessed by CT at Week 28 in AHOC analysis set is reported.
| Arm | Type | Description |
|---|---|---|
| Cohort 1: Adolescents | EXPERIMENTAL | Participants aged 12 - 18 years old |
| Cohort 2: Children | EXPERIMENTAL | Children who are 4 to 12 years old (for non-US sites only 2 to 12 years old) |
| Cohort 3: Children and Adolescents | EXPERIMENTAL | Participants and Children aged 4 to 18 years old (for non-US sites only 2 to 18 years old) |
| High dose Garetosmab | EXPERIMENTAL | Garetosmab is administered by intravenous (IV) administration every 4 weeks (Q4W) |
| Low dose Garetosmab | EXPERIMENTAL | Garetosmab is administered by IV administration Q4W |
| Placebo | EXPERIMENTAL | Placebo to match garetosmab, is supplied as a liquid solution without the monoclonal antibody (or the protein) and is administered IV Q4W. |
| REGN2477 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| garetosmab | DRUG | Administered per the protocol |
| Placebo | DRUG | Placebo to match garetosmab, is supplied as a liquid solution without the monoclonal antibody (or the protein) and is administered IV. |
| REGN2477 | DRUG | Pharmaceutical form: liquid product for injection/infusion; Route of administration: Intravenous (IV); Administered during treatment periods 1 and 2. |
| Matching placebo | DRUG | Pharmaceutical form: Liquid product for injection/infusion; Route of administration: Intravenous (IV); Administered during treatment period 1 only. |
Key Inclusion Criteria: 1. For USA participants, age criteria are 4 to \< 18 years old, at the time of the administration of the first dose of study intervention. Non-USA participants age criteria are 2 to \< 18 years old 2. Must have a confirmation of FOP diagnosis, as described in the protocol 3....
Garetosmab is an investigational monoclonal antibody being developed for fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder characterized by abnormal bone formation in soft tissue. It is in Phase 3 clinical development and is not yet approved for any indication.
Garetosmab targets INHBA, also known as inhibin beta A, a subunit of the activin A protein. It is an inhibitor antibody designed to block this target. Activin A signaling is implicated in the abnormal heterotopic ossification that characterizes fibrodysplasia ossificans progressiva.
Garetosmab is developed by Regeneron Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker REGN. Regeneron is the sponsor of the Phase 3 clinical program evaluating the antibody in fibrodysplasia ossificans progressiva.
Garetosmab is in Phase 3 clinical development for fibrodysplasia ossificans progressiva. It is an investigational therapy and has not been approved by the FDA. The program has received a Priority Review designation from the FDA.
Garetosmab is being studied in two Phase 3 trials. NCT05394116 is an active, not recruiting study of intravenous garetosmab versus placebo in adults with FOP across 18 countries. NCT07559513 is a not yet recruiting study evaluating safety, pharmacokinetics, and efficacy in children and adolescents aged 2 years and older.
The two Phase 3 trials of garetosmab have a combined planned enrollment of 81 participants. NCT05394116 plans to enroll 63 adults with fibrodysplasia ossificans progressiva, while NCT07559513 plans to enroll 18 children and adolescents aged 2 years and older.