Switching from Agalsidase Alfa to Pegunigalsidase Alfa for Treating Fabry Disease: One Year of Treatment Data from Bridge, a Phase 3 Open-Label Study
Bibliographic record
Abstract
Background: Pegunigalsidase-alfa is a novel, PEGylated α-Galactosidase-A enzyme in development for the treatment of Fabry disease (FD). Methods: Bridge (PB-102-F30, NCT03018730) is a phase III, open-label, switchover study, designed to assess the safety and efficacy of pegunigalsidase-alfa (1 mg/Kg EOW) in adult FD patients previously treated with agalsidase alfa for at least 2 years. Results: This is an interim report of 12-months on-treatment data generated from the first 16 patients (9 males and 7 females) out of the 22 adult patients enrolled. Baseline characteristics: age 24-60 years, the mean estimated Glomerular Filtration Rate (eGFR) 75.45 in males and 85.78 mL/min/1.73m2 in females, annualized eGFR slope was -5.04 and -5.18 mL/min/1.73m2/year, respectively, mean residual leucocytes enzymatic activity 5.9% of lab normal mean in males and 27.9% in females, and plasma lyso-Gb3 53.6 and 13.8 nM, respectively. After one year the mean annualized eGFR slope improved from -5.10 mL/min/1.73m2/year while on agalsidase alfa, to -0.23 mL/min/1.73m2/year on pegunigalsidase-alfa. According to Wanner et al. 2018, FD patients with eGFR slope between ≥ -5 and < -3 mL/min/1.73 m2/year are defined as kidney disease progressing and with eGFR slope < -5 mL/min/1.73 m2/year are defined fast progressing. The therapeutic goal is to reach eGFR slope ≥ -3 mL/min/1.73 m2/year for the progressing, and ≥ -5 mL/min/1.73m2/year or more than 50% decrease in progression for the fast progressing. In this interim analysis, 100% of the progressing patients and 66.7% in the fast progressing group achieved the proposed therapeutic goals after switching to pegunigalsidase-alfa. The switch to pegunigalsidase-alfa was safe and well-tolerated. The majority of the patients who completed the study rolled over to a long-term extension study, continuing receiving pegunigalsidase-alfa. Conclusions: These results suggest a potential benefit of pegunigalsidase-alfa on renal function for FD patients currently treated with agalsidase alfa, to be confirmed by long-term data. Funding: Commercial Support - Chiesi USA, Protalix Biotherapeutics
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.004 | 0.002 |
| Meta-epidemiology (narrow) | 0.001 | 0.001 |
| Meta-epidemiology (broad) | 0.004 | 0.002 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.001 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.002 | 0.004 |
| Insufficient payload (model declined to judge) | 0.004 | 0.001 |
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; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
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".