According to a press release from biotechnology company Forbius (Formation Biologics Inc.), the first patient in the Company’s phase 1b trial of experimental myelofibrosis (MF) drug AVID200 has successfully received their initial dose of the drug.
Myelofibrosis is a rare form of leukemia – a cancer of the blood and bone marrow.
The disease is caused by the presence of blood stem cells (immature blood cells that can develop into any type of blood cell) that have mutated. As they divide and mature, these stem cells pass the mutation onto an ever-increasing and ever-dividing number of maturing blood cells.
The result is a serious imbalance in the proportions of red blood cells, white blood cells, and platelets. In most cases of myelofibrosis, there is an acute shortage of red blood cells and relative oversupply of white blood cells. This blood imbalance can lead to fever, fatigue, reduced clotting factor, and bone pain. Eventually, the imbalance leads to scarring of the bone marrow, which further disrupts healthy blood supply.
Like many cancers, existing treatment options for myelofibrosis are limited to symptom relief. Over time, myelofibrosis can give rise to other, even more serious forms of leukemia.
AVID200 is a drug candidate being developed by Canadian biotech company Formation Biologics, better known to some as the pithier “Forbius.”
In theory, AVID200 works as an inhibitor of the blood proteins TGF-β1 and TGF-β3. Normally, these two proteins are intimately involved in cell growth, development, and differentiation. However, they are also the primary drivers of fibrosis in individuals with myelofibrosis and other fibrotic diseases. AVID200 was specifically formulated to have a minimal impact on isoform (think related but non-identical) TGF-β2 – a related protein that strongly promotes hematopoiesis (the proper balance of blood components).
The first patient in Forbius’ phase 1b trial has already received their first dose of AVID200, with existing plans allotting the enrollment of up to 23 more individuals with primary myelofibrosis. Over the course of the trial, AVID200 will have its safety and efficacy tested – with researchers hoping to find evidence of restored hematopoietic balance, improved clinical response, and a decrease in bone marrow fibrosis.
Once AVID200 has been established as safe in humans, it will likely undergo further clinical study in phase 2 and possibly phase 3 trials. These trials tend to be more concerned with determining the actual effectiveness of the drug candidate than phase 1 tests, which primarily establish safe dosing levels.
As it stands, there’s not much concrete about the effectiveness of AVID200. The beginning of phase 1b testing is exciting news for many in the rare disease community, but even if the drug is found to work, final market approval could still be years away.
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