Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Garetosmab
REGN2477 · 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....
REGN2477 is an investigational therapy being studied for fibrodysplasia ossificans progressiva, a rare genetic disorder characterized by abnormal bone formation in soft tissues. It is a small molecule developed by Regeneron Pharmaceuticals and has been evaluated in a Phase 2 clinical trial in adults with the condition.
REGN2477 targets Activin A, a protein involved in signaling pathways that regulate bone and tissue development. By targeting Activin A, the drug is designed to interfere with the abnormal bone formation that occurs in fibrodysplasia ossificans progressiva. Activin A is a protein target, and REGN2477 is a small molecule therapeutic.
REGN2477 is being developed by Regeneron Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker symbol REGN. Regeneron is the sponsor of the clinical program evaluating the drug in fibrodysplasia ossificans progressiva, a rare disease indication.
REGN2477 is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. The Phase 2 trial evaluating REGN2477 in fibrodysplasia ossificans progressiva has been completed, and the program remains in clinical development.
REGN2477 has been studied in one Phase 2 clinical trial, NCT03188666, titled a study to examine the safety, tolerability and effects on abnormal bone formation of REGN2477 in patients with fibrodysplasia ossificans progressiva. The trial enrolled 44 participants, was randomized, double-blind, and controlled, and has been completed.