Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
REGN2477 · 2 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 |
|---|---|---|
| REGN2477 | EXPERIMENTAL | - |
| Placebo | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| 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. |
| Placebo | DRUG | Participants will receive matching placebo |
Key Inclusion Criteria: * Men and women 18 to 60 years of age at screening. * Clinical diagnosis of FOP (based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive heterotopic ossification (HO)). * Confirmation of FOP diagnosis with documentati...
REGN2477 is an investigational small molecule being studied for the treatment of Fibrodysplasia Ossificans Progressiva (FOP), a rare disease characterized by abnormal bone formation. It has also been studied in healthy volunteers to assess its safety and tolerability. REGN2477 is being developed by Regeneron Pharmaceuticals, Inc. (REGN).
REGN2477 is a small molecule being developed by Regeneron Pharmaceuticals, Inc. (REGN) for Fibrodysplasia Ossificans Progressiva (FOP). The specific molecular target of REGN2477 has not been disclosed in the available clinical trial information. The drug is being studied for its effects on abnormal bone formation in patients with FOP.
REGN2477 is being developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol REGN. Regeneron is conducting clinical trials to evaluate the safety, tolerability, and efficacy of REGN2477 in patients with Fibrodysplasia Ossificans Progressiva (FOP).
REGN2477 is in Phase 2 clinical development for Fibrodysplasia Ossificans Progressiva (FOP). A Phase 2 study (NCT03188666) has been completed, evaluating the safety, tolerability, and effects on abnormal bone formation in patients with FOP. REGN2477 is investigational and has not been approved by regulatory authorities.
REGN2477 has been studied in two completed clinical trials. NCT02870400 was a Phase 1 study in healthy volunteers in Belgium, examining safety and tolerability of single ascending doses. NCT03188666 was a Phase 2 study in patients with Fibrodysplasia Ossificans Progressiva (FOP) across multiple countries, including the United States, Canada, and several European nations.
REGN2477 is the investigational code name used by Regeneron Pharmaceuticals, Inc. (REGN) for this drug candidate. No alternative brand names or other designations have been disclosed in the clinical trial information. The drug is being studied for Fibrodysplasia Ossificans Progressiva (FOP) and has completed Phase 2 trials.