Australian clinicians lead world-first trial of Pfizer drug in rare blood disorder

Australian clinicians are leading the world’s only trial investigating Pfizer’s BCMA-targeting bispecific antibody Elrexfio (elranatamab) as a first-line treatment for newly diagnosed AL amyloidosis.

Also known as light chain amyloidosis, AL amyloidosis occurs when abnormal plasma cells in the bone marrow produce sticky, misfolded proteins known as light chains. These proteins can accumulate as amyloid deposits in vital organs, including the heart, kidneys, nerves and liver, disrupting their normal function and potentially causing serious organ damage.

The current standard of treatment is a combination regimen comprising the anti-CD38 monoclonal antibody daratumumab, bortezomib, cyclophosphamide and dexamethasone, known as D-VCd. However, not all patients respond to treatment, while the toxicity associated with conventional chemotherapy-based regimens can be substantial.

Although existing data on Elrexfio support its use in relapsed or refractory AL amyloidosis, the phase 2 ENLIGHT trial funded by the Australasian Myeloma Research Consortium is assessing whether the therapy can be moved earlier in the treatment pathway and used as a first line therapy for newly diagnosed patients. The study is being led by haematologists Professor Hang Quach of St Vincent’s Hospital Melbourne and the University of Melbourne, and Dr Matthew Rees of St Vincent’s Private Hospital Melbourne.

Elrexfio is currently indicated for patients with relapsed or refractory multiple myeloma (RRMM) who have received at least three prior lines of therapy. The therapy was listed on the Pharmaceutical Benefits Scheme (PBS) on 1 April 2026.

The latest evidence in multiple myeloma has strengthened the clinical case for Elrexfio earlier in the course of disease. In the phase 3 MagnetisMM-5 study, Elrexfio monotherapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared with standard-of-care daratumumab, pomalidomide and dexamethasone (DPd).

The trial enrolled 497 patients with RRMM who had received at least one prior line of treatment, including lenalidomide and a proteasome inhibitor (PI). At the time of the interim analysis, the overall survival analysis was not yet mature, and the trial remains ongoing to collect results for this secondary endpoint.

Elrexfio is competing in the BCMA-targeting bispecific antibody class with Tecvayli (teclistamab-cqyv) from Johnson & Johnson Innovative Medicine, which is approved by the Therapeutic Goods Administration (TGA) but is not yet reimbursed through the PBS. A third agent, Lynozyfic (linvoseltamab), is currently available only overseas.

“All three show very similar outcomes, with response rates of approximately 60%-70% in heavily pre-treated patients. When responses occur, they typically last for more than two years,” said Professor Quach.

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