In a significant development for patients with vanishing white matter (VWM) disease, AbbVie and Alphabet’s Calico announced on Friday that their investigational eIF2B activator, fosigotifator, has been selected to participate in the FDA’s new Support for clinical Trials Advancing Rare disease Therapeutics (START) pilot program. This initiative, also known as Operation Warp Speed for rare diseases, aims to accelerate the development of treatments for rare conditions with high unmet medical needs.

Fosigotifator, the seventh development program to join the START pilot, is currently undergoing a Phase 1b/2 trial to evaluate its safety, tolerability, and pharmacokinetics in individuals with VWM disease. This groundbreaking study marks the first time an eIF2B activator has been administered to patients with this rare neurological disorder, offering hope for a potential treatment where none currently exist.

VWM disease is an ultra-rare progressive leukoencephalopathy – a disease of the brain’s white matter – caused by variations in any of the five subunits of an essential enzyme in cells called eIF2B. VWM variations in eIF2B cause a reduction in its enzymatic activity that may lead to chronic activation of the integrated stress response (ISR). The ISR plays a critical role in protein homeostasis and organismal resilience, both of which are implicated in the biology of aging.

The collaboration between AbbVie and Calico, which began in 2014 with an initial investment of $250 million from each company, has led to the development of fosigotifator. This potential therapy aligns with the objectives of the START pilot, as it targets a disease with no available treatments, highlighting the urgent need for effective interventions.

Arthur Levinson, founder and CEO of Calico, expressed enthusiasm for the inclusion of fosigotifator in the START program, stating that it “underscores the potential of this investigational therapy in addressing the unmet needs of individuals and families affected by Vanishing White Matter Disease.”

The selection of fosigotifator for the START pilot is a testament to the dedication and innovation of the teams at AbbVie and Calico, as they work towards developing a much-needed treatment for VWM disease. As the Phase 1b/2 trial progresses, the medical community and patients alike will eagerly await the results, hoping for a breakthrough that could significantly improve the lives of those affected by this rare and debilitating condition.

This article was originally published on iBIO NewsBrief. Gain a head start on your day with iBIO NewsBrief. Subscribe to receive top industry headlines delivered straight to your inbox.