Maria Elisa Mancuso on NXT007 Prophylaxis in People With Haemophilia A
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
“Maria Elisa Mancuso presented new ASH data on NXT007 prophylaxis in people with haemophilia A (PwHA), with and without FVIII inhibitors – a global, open-label Phase I/II multiple‑ascending dose (MAD) study (first 3 cohorts).
NXT007, a next‑generation FVIII‑mimetic bispecific antibody derived from emicizumab, showed a very consistent picture across pharmacokinetics (PK), pharmacodynamics (PD) and clinical outcomes
- Safety/tolerability:
Well tolerated across all dose cohorts, with no thromboembolic events (TEs) / thrombotic microangiopathies (TMAs), no discontinuations due to adverse events (AEs) and D‑dimer levels remaining in the normal range.
- Pharmacokinetics:
Dose‑proportional PK with sustained plasma concentrations within the predicted therapeutic range.
- Thrombin generation (TG):
Mean TG peak heights in Cohorts 2 and 3 at steady state were comparable to normal‑range FVIII levels.
- Bleeding outcomes:
Annualized bleeding rates (ABRs) for treated bleeds markedly decreased after starting NXT007; the majority of participants (≈86%) had zero treated bleeds during the maintenance period. One striking outlier with multiple bleeds had an abnormal vessel in a replaced knee, nicely reminding us that not every bleed signal is “drug failure”.
- Immunogenicity:
Most participants developed treatment‑induced anti‑drug antibodies (ADAs) around Day 29, yet these ADAs had no detectable impact on NXT007 PK, efficacy or safety, and showed no cross‑reactivity with emicizumab.
These Phase I/II results strongly support progression of NXT007 into Phase III trials
Guy Young asked an excellent question: If high ADA rates do not translate into altered PK, reduced efficacy or safety concerns, what is the true clinical relevance of routine ADA testing for these bispecifics?”
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