Pharmacokinetics of a novel extended half‐life glyco<scp>PEG</scp>ylated factor <scp>IX</scp>, nonacog beta pegol (N9‐<scp>GP</scp>) in previously treated patients with haemophilia B: results from two phase 3 clinical trials
Bibliographic record
Abstract
Introduction Nonacog beta pegol (N9‐ GP ) is a glyco PEG ylated recombinant factor IX ( FIX ) with an extended half‐life developed for routine prophylaxis and the prevention and treatment of bleeding episodes in patients with haemophilia B. Aim The aim of this study was to evaluate the pharmacokinetics ( PK ) of N9‐ GP . Methods Data from 41 previously treated haemophilia B patients, enrolled globally (16 adolescents/adults and 25 children; FIX activity ≤0.02 IU mL −1 ) with no history of FIX inhibitors, were included. N9‐ GP was administered once‐weekly as 10 IU kg −1 or 40 IU kg −1 in adolescents/adults and 40 IU kg −1 in children. Blood was sampled up to 168 h (1 week) post dose. Standard PK was estimated on the basis of plasma FIX activity vs. time ( PK profiles) using non‐compartmental methods. Furthermore, a population PK analysis and FIX activity predictions were performed. Results Incremental recoveries were 0.02 ( IU mL −1 )/( IU kg −1 ) in both adolescents/adults and children. The extended half‐life resulted in mean trough levels of 0.27 IU mL −1 for adolescents/adults and 0.17 IU mL −1 for children at steady‐state after weekly dosing at 40 IU kg −1 . The population PK analysis confirmed a mono‐exponential decay in FIX activity and allowed for predictions of FIX activity for adolescents/adults above 0.15 IU mL −1 at all times and 6.4 days week −1 in children. Conclusion N9‐ GP has the potential to shift previously treated haemophilia B patients from a severe/moderate disease state into a mild‐ or non‐haemophilic range for most of the dosing interval, which is expected to reduce the number of bleeding episodes.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.004 | 0.037 |
| Meta-epidemiology (narrow) | 0.002 | 0.001 |
| Meta-epidemiology (broad) | 0.005 | 0.001 |
| Bibliometrics | 0.001 | 0.001 |
| Science and technology studies | 0.001 | 0.002 |
| Scholarly communication | 0.000 | 0.001 |
| Open science | 0.002 | 0.001 |
| Research integrity | 0.001 | 0.002 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; both teacher heads agree on what is shown here.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".