Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
REGV131 · 1 trial · 1 indication
Part 1, 2B, and 2C
Part 1, 2B, and 2C
Part 1
Part 2A, 2B, and 2C
Part 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
LTFU Period for Part 1, 2A, 2B, and 2C
| Arm | Type | Description |
|---|---|---|
| Part 1: Cohort 1 Dose Escalation for RDE | EXPERIMENTAL | Starting dose to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE |
| Part 1: Cohort 2 Dose Escalation for RDE | EXPERIMENTAL | Dose 2 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE |
| Part 1: Cohort 3 Dose Escalation for RDE | EXPERIMENTAL | Dose 3 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE |
| Part 1: Cohort 4 Dose Escalation for RDE | EXPERIMENTAL | Dose 4 of ascending dose level cohorts to determine the RDE of REGV131-LNP1265 and further assess the safety, tolerability, and FIX functional activity data at the RDE |
| Part 2: Dose Expansion A | EXPERIMENTAL | Participants ≥18 Years of Age will receive the RDE of REGV131-LNP1265 determined by Part 1 |
| Part 2: Dose Expansion B | EXPERIMENTAL | Participants ≥12 to \<18 Years of Age will receive the administered weight-adjusted RDE of REGV131-LNP1265 determined by Part 1 |
| Part 2: Dose Expansion C | EXPERIMENTAL | Participants ≥2 to \<12 Years of Age will receive the administered weight-adjusted RDE of REGV131-LNP1265 determined by Part 1 |
| Name | Type | Description |
|---|---|---|
| REGV131 | DRUG | Administered per the protocol before LNP1265 |
| LNP1265 | DRUG | Administered per the protocol following REGV131 |
Key Inclusion Criteria: 1. Confirmed diagnosis of severe or moderately severe hemophilia B with medical history of FIX functional activity (≤2% or \<0.02 IU/mL) or documented genotype known to produce severe hemophilia B 2. Currently taking FIX prophylaxis and previous experience with FIX therapy, ...
REGV131 is an investigational small molecule being developed for the treatment of hemophilia B. It is a coagulation factor IX gene insertion therapy designed to address the underlying deficiency of factor IX in patients with this bleeding disorder. The drug is currently in Phase 1 clinical development.
REGV131 is a coagulation factor IX gene insertion therapy. It is designed to insert a functional copy of the factor IX gene into cells, enabling the body to produce the clotting factor that is deficient in patients with hemophilia B. This approach aims to address the root cause of the bleeding disorder.
REGV131 is being developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol REGN. The company is conducting a Phase 1 clinical trial to evaluate the safety and effectiveness of this investigational therapy in patients with hemophilia B.
REGV131 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still being studied for safety and effectiveness. The ongoing Phase 1 trial is currently recruiting participants with hemophilia B.
REGV131 is being studied in a Phase 1 clinical trial registered as NCT06379789. This trial is investigating the safety and effectiveness of REGV131-LNP1265 in pediatric, adolescent, and adult participants with hemophilia B. The study is recruiting up to 130 male participants aged 2 years and older across multiple countries.
Yes, REGV131 is also known as REGV131-LNP1265. The clinical trial NCT06379789 refers to the therapy as REGV131-LNP1265, indicating that it is the same investigational drug being studied for hemophilia B. This naming convention is used in the trial documentation.