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, ...
Top 8 of 9 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 15 | PHASE3 | Concizumab, Mim8, Denecimig, NNC0442-0344 A, turoctocog alfa pegol |
| Sanofi SA Sponsored ADR | SNY | 5 | PHASE3 | Fitusiran, efanesoctocog alfa |
| Pfizer Inc. | PFE | 8 | PHASE3 | Fidanacogene elaparvovec, Marstacimab |
| BioMarin Pharmaceutical Inc. | BMRN | 2 | PHASE3 | Valoctocogene roxaparvovec |
| Hoffmann-La Roche | RHHBY | 8 | PHASE3 | Emicizumab, NXT007, SPK-8011QQ, SPK-8011, SPK-8016 |
| Regeneron Pharmaceuticals, Inc. | REGN | 2 | PHASE1 | REGV131, LNP1265 |
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 2 | PHASE1 | AskBio009 |
| Bayer | BAYRY | 6 | - | Damoctocog-alfa-pegol |
REGV131 is an investigational gene insertion therapy being developed by Regeneron Pharmaceuticals for hemophilia B, a bleeding disorder caused by deficient coagulation factor IX. It is a small molecule modality and is currently in Phase 1 clinical development. Regeneron trades on Nasdaq under the ticker REGN.
REGV131 is being studied as a treatment for hemophilia B, a hereditary bleeding disorder. The Phase 1 trial enrolls pediatric, adolescent, and adult male participants with hemophilia B across the United States, Australia, Brazil, Canada, France, Germany, Italy, Spain, and the United Kingdom.
REGV131 is developed by Regeneron Pharmaceuticals, Inc., which trades on Nasdaq under the ticker REGN. The company is running the clinical program for the asset in hemophilia B.
REGV131 is in Phase 1 clinical development. It is investigational and has not been approved by the FDA. One Phase 1 trial is currently recruiting, and no trials for REGV131 have been completed to date.
REGV131 is being evaluated in one Phase 1 trial, NCT06379789, titled a study to investigate the safety and effectiveness of a coagulation factor IX gene insertion therapy (REGV131-LNP1265) in pediatric, adolescent and adult participants with hemophilia B. The trial is recruiting and plans to enroll 130 participants.
The Phase 1 trial NCT06379789 plans to enroll 130 participants with hemophilia B. Enrollment covers male participants aged 2 years and older, and the study is controlled but is not randomized or double blinded. It is currently recruiting across nine countries.