Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as ADYNOVATE, PEGylated Recombinant Factor VIII, TAK-660, Recombinant Factor VIII (FVIII) PEGylated
BAX855 · 5 trials · 1 indication
Total ABR was defined as the number of treated and non-treated bleeding episodes (BEs) that occurred during the treatment period, calculated as, ABR= number of unique bleeds during treatment period/(length of treatment period \[days\]/365.25). Total ABR for all BEs, spontaneous or traumatic, recorded in the participant's electronic diary and/or recorded in the physician/nurse/study site notes were reported.
Annualized bleeding rate was determined by dividing the number of bleeds by observation period in years.
Number of participants who developed an inhibitor (at any time) confirmed by a central laboratory based on a second repeat blood sample draw within 2 weeks of site notification of an inhibitor and all participants who had not developed an inhibitor and had greater than or equal to (\>=) 100 EDs when the sample for the last valid inhibitor test was drawn.
Success is defined as 1) a persistently negative inhibitor titer less than (\<) 0.6 Bethesda unit (BU), 2) FVIII IR \>=66% of the baseline value following a wash-out period of 84-96 hours, and 3) a FVIII half-life of \>=6 hours.
GHEA=Sum of 1-3 ratings: Excellent: 7-9 (no category \<2), Good: 5-7 (no category \<1), Fair: 3-4 (no category \<1) 1. Intraoperative and 2. Postoperative (postoperative day 1) hemostatic efficacy assessments: Excellent=3: Blood Loss (BL) ≤ than expected for procedure type in non-hemophilic population (NHP) (≤100%), Good=2: BL ≤50% more than expect. for procedure type in NHP (101-150%), Fair=1: BL \>50% more than expect. for procedure type in NHP (\>150%), None=0: Significant bleeding-requiring rescue therapy (RT) 3. Perioperative hemostatic efficacy assessment (day 14 or discharge, whatever is first): Excellent=3: BL and required blood transfusions (BT) less than or similar (≤100%) to that expected for procedure type in NHP, Good=2: BL ≤50% more (101-150%) and BT less than or similar to that expected for procedure type in NHP, Fair=1: BL \>50% more (\>150%) and BT greater than expected in NHP, None=0: Significant bleeding-requiring RT, BT substantially greater than expected in NHP
Inhibitory antibodies to Factor VIII were measured by the Nijmegen modification of the Bethesda assay. Inhibitors had to be confirmed by 2 separate assessments within a 2 to 4 week period from the central laboratory.
The ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. The ABR of spontaneous bleeds was reported separately for twice weekly, PK-t R, each of the every 5 days and every 7 days treatment regimens at the time of bleed.
| Arm | Type | Description |
|---|---|---|
| Adynovate 45±5 IU/kg | EXPERIMENTAL | Participants received prophylactic treatment with Adynovate (45 \[±5\] IU/kg), infusion, IV, twice weekly, for at least 50 EDs, or approximately 28 weeks. |
| Pharmacokinetic (PK) evaluation of BAX 855 | EXPERIMENTAL | Participants will first undergo an initial pharmacokinetic (PK) assessment. Following the PK assessment participants will be randomized to one of 2 dosing regimens. |
| FVIII trough target 1-3% | EXPERIMENTAL | Standard treatment arm - PK-guided dosing schedule to achieve a Factor VIII (FVIII) trough of 1-3% |
| FVIII trough target 8-12% | EXPERIMENTAL | Intensified treatment arm - PK-guided dosing schedule to achieve a Factor VIII (FVIII) trough of 8-12% |
| All Participants | EXPERIMENTAL | Previously Untreated Patients (PUPs) \< 6 years of age with severe hemophilia A (FVIII \< 1%) and \< 3 exposure days (EDs) to ADVATE, BAX 855 or plasma transfusion were enrolled in a single arm group. Part A (Main Study): Participants age \<3 years - who had not experienced two joint bleeds received on-demand treatment of 10-80 international units per kilogram (IU/kg) intravenously (IV) depending on the severity of the bleeding episode; and - who experienced maximum of two joint bleeds received prophylaxis treatment with dose of 25-80 IU/kg of BAX 855 IV (based on investigator discretion) once weekly for up to 100 EDs. Part B (Immune tolerance induction \[ITI\] Portion): Participants who met the pre-defined Part B treatment criteria entered Part B of the study for ITI. Participants either received prophylaxis treatment of BAX 855 low dose 50 IU/kg IV, three times a week or high dose 100-200 IU/kg IV, daily at discretion of the investigator according to the institution's standard of care. |
| BAX855 | EXPERIMENTAL | Pre-operative loading dose: Single loading dose, pre-surgery administered based on each study participant's individual PK results as well as target trough level for type and character of surgery, dental or invasive procedure being performed. In general, major surgery will target an 80-150% FVIII trough level, and minor surgery will target an initial 30-100% FVIII trough level. Intra-operative and post-operative dosing of BAX855 must be based on pre-dosage measurements of FVIII and the type and character of the surgery performed. |
| Fixed BAX855 prophylaxis | EXPERIMENTAL | 45-80 IU/kg twice weekly to once per week. |
| Pharmacokinetic (PK)-tailored BAX 855 prophylaxis | EXPERIMENTAL | PK-tailored prophylactic BAX855 regimen based on participant's individual PK profile to maintain a Factor VIII (FVIII) trough level |
| Name | Type | Description |
|---|---|---|
| Adynovate | BIOLOGICAL | Adynovate was injected intravenously using an appropriately sized syringe as a bolus infusion over a period of less than or equal to (\<=) 5 minutes (maximum infusion rate, 10 milliliters per minute \[mL/min\]). |
| PEGylated Recombinant Factor VIII | BIOLOGICAL | Pharmacokinetic (PK) evaluation |
| ITI | BIOLOGICAL | Immune tolerance induction therapy |
| PEGylated Recombinant factor VIII (rFVIII) | BIOLOGICAL | Lyophilized powder and solvent for solution for injection |
| BAX855 | BIOLOGICAL | Antihemophilic Factor (Recombinant), PEGylated |
Inclusion Criteria: 1. Participant and/or legally authorized representative must voluntarily sign a written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the Participant. For the participants less than (\<) 18 years old, participants will...
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 |
BAX855 is being developed for the treatment of hemophilia A, a bleeding disorder. It is a monoclonal antibody studied in a Phase 3 clinical program in patients with hemophilia A. Takeda Pharmaceutical Company Limited is the developer.
BAX855 is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda is the sponsor of the clinical program evaluating BAX855 in hemophilia A.
BAX855 is in Phase 3 clinical development for hemophilia A. It is an investigational monoclonal antibody and has not been approved by the FDA. The Phase 3 program includes one completed trial.
BAX855 has been studied in the Phase 3 trial NCT01945593, titled BAX 855 Continuation. That trial enrolled 218 participants with hemophilia A and is completed. It ran across the United States, Australia, Austria, Bulgaria, Czechia, Germany, Hong Kong, Israel, Japan, Lithuania, Malaysia, Netherlands, Poland, Romania, Russia, South Korea, Spain, Sweden, Switzerland, Taiwan, Turkey, Ukraine, and the United Kingdom.
The BAX855 clinical program enrolled 218 participants in total, all in the Phase 3 trial NCT01945593. That study was a controlled trial in hemophilia A and is now completed.