Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Zemocimig, RO7589655, RG6512
NXT007 · 4 trials · 1 indication
The vital signs that will be assessed are body temperature, pulse rate, respiratory rate, and systolic and diastolic blood pressure.
The ECG parameters that will be assessed are heart rate, PR interval, QRS interval, QT interval, and QTcF inteval.
| Arm | Type | Description |
|---|---|---|
| NXT007 Prophylaxis | EXPERIMENTAL | - |
| Main Study Treatment Period: NXT007 Prophylaxis | EXPERIMENTAL | Participants randomized to this arm will receive NXT007 prophylaxis for the main study treatment period. |
| Main Study Treatment Period: FVIII SOC Prophylaxis | ACTIVE_COMPARATOR | Participants randomized to this arm will receive FVIII standard of care (SOC) prophylaxis for the main study treatment period. |
| Open-Label Extension Period: NXT007 Prophylaxis | EXPERIMENTAL | After the main study treatment period, participants in the NXT007 arm will be able to continue with NXT007 dosing, and participants in the FVIII arm will be able to switch to NXT007, in the open-label extension period. |
| Main Study Treatment Period: Emicizumab Prophylaxis | ACTIVE_COMPARATOR | Participants randomized to this arm will receive emicizumab prophylaxis for the main study treatment period at 3 mg/kg once weekly (QW) for 4 weeks as loading doses, followed by maintenance dosing of either 1.5 mg/kg QW, 3 mg/kg once every 2 weeks (Q2W), or 6 mg/kg once every 4 weeks (Q4W). Loading doses are not required for participants who were taking emicizumab prior to study start. |
| Part 1: Cohort 1 - NXT007 Dose Level 1 (Low) | EXPERIMENTAL | - |
| Part 1: Cohort 2 - NXT007 Dose Level 2 | EXPERIMENTAL | - |
| Part 1: Cohort 3 - NXT007 Dose Level 3 | EXPERIMENTAL | - |
| Part 1: Cohort 4 - NXT007 Dose Level 4 | EXPERIMENTAL | - |
| Part 1: Cohort 5 - NXT007 Dose Level 5 (High) | EXPERIMENTAL | - |
| Part 2: Cohort A - NXT007 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| NXT007 | DRUG | NXT007 will be administered subcutaneously (SC) according to the schedule in the protocol. |
| Human Coagulation Factor VIII | DRUG | Factor VIII (FVIII) prophylaxis standard of care (SOC) will be administered at the dose and frequency as stated in the local labels and per local country practice. |
| Emicizumab | DRUG | Emicizumab will be administered subcutaneously (SC) using vial and syringe. |
Inclusion Criteria: * Age \<12 years at the time of signing Informed Consent Form; or age ≥12 and \<18 years with body weight \<40 kg * Diagnosis of severe (Factor VIII coagulation protein activity \[FVIII:C\] \<1 International Unit per decilitre \[IU/dL\]) or moderate (FVIII:C between ≥1 IU/dL and...
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 |
NXT007 is an investigational therapy being developed for the treatment of hemophilia A, including prophylactic treatment in infants, children, and adults. It is being studied both as a preventive treatment and in comparison with existing prophylaxis options such as emicizumab and Factor VIII. It has not been approved for any indication.
NXT007 is being developed by Roche Holding AG, which trades on the OTC market under the ticker RHHBY. Roche is running the clinical development program for NXT007 across multiple Phase 3 trials and one Phase 1 study in hemophilia A.
NXT007 is in Phase 3 clinical development for hemophilia A. Three of its four trials are Phase 3 studies, and one is a Phase 1 study evaluating safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy. NXT007 is investigational and has not been approved by the FDA.
NXT007 is being studied in four clinical trials. NCT07416604 compares NXT007 with emicizumab prophylaxis, and NCT07416526 compares it with Factor VIII prophylaxis, both in participants aged 12 and older. NCT07838194 evaluates prophylactic treatment in infants and children, and NCT05987449 is a Phase 1 study in severe or moderate hemophilia A.
The NXT007 clinical program has a total planned enrollment of 608 participants across four trials. NCT07416604 plans to enroll 360 participants, NCT07416526 plans 138, NCT05987449 plans 60, and NCT07838194 plans 50. All four trials are controlled, and the program includes randomized studies.
NXT007 trials are being conducted across North America, Europe, Asia, Latin America, and other regions. Countries include the United States, Canada, the United Kingdom, Germany, France, Italy, Spain, Japan, China, South Korea, Taiwan, Brazil, Argentina, Australia, New Zealand, South Africa, and several others.