Prothena Corporation which is based in Dublin, Ireland recently announced, via Business Wire and Biospace results of the VITAL Phase 3 clinical trial showing a significant survival benefit of 74% for patients who received birtim birtamimab and the standard of care (SOC) versus 49% of patients who received the placebo and SOC.
Results of the Phase 3 VITAL trial were published in Blood, the noted ASH Journal.
About AL Amyloidosis
Amyloidosis is a group of disorders with AL amyloidosis being the most common form. An abnormal protein called amyloid is known to build up in organs and tissues.
This rare disease is life-threatening with no available treatment options.
Symptoms of AL amyloidosis vary due to the buildup occurring in so many areas such as the muscles nerves, joints, the heart or liver, spleen, or kidneys.
All participants in the trial who received bortezomib individually became part of SOC and additionally received bortezomib combined with chemotherapy regimens.
The major cause of fatal adverse events was cardiac disorder which is common with AL amyloidosis. No adverse events were found to be related to treatment.
Hematologist Dr. Morie Gertz commented that the recently published data supports the company’s belief that treatment with birtamimab led to a survival benefit in patients with Stage IV AL amyloidosis and shows its potential as being a safe and well-tolerated therapy. Professor Gertz further stated that this is the first time such important data appeared in a prestigious, peer-reviewed journal.
The Confirmatory Phase 3 Trial
The AFFIRM-AL clinical trial is being conducted with patients who have Mayo Stage IV AL. Confirmatory data are on track for 2024. The FDA and the EU have granted orphan drug designation to AL amyloidosis. The FDA has granted Fast Track designation to birtamimab as well.
You may access a copy of the ASH journal Blood here: