FDA Grants Orphan Drug Status to Anti-inflammatory EI-1071 for IPF
Elixiron's oral therapy candidate showed promise in Phase 1 study
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to EI-1071, an oral anti-inflammatory that Elixiron Immunotherapeutics is developing for the treatment of idiopathic pulmonary fibrosis (IPF).
Orphan drug status is awarded by the FDA to therapies designed to improve medical care for rare disorders — those that affect fewer than 200,000 people in the U.S. The designation confers certain incentives to Elixiron as the therapy’s developer, including tax credits for eligible clinical testing, waiver of certain fees, and a guarantee of market exclusivity for seven years if EI-1071 ultimately wins FDA approval.
“Idiopathic pulmonary fibrosis has a devastating impact on patients’ lives and more effective therapies are urgently needed,” Hung-Kai Kevin Chen, CEO of Elixiron, said in a press release, adding, “The orphan drug designation validates the potential of EI-1071 to offer a new therapeutic option for patients living with this disease.”
Elixiron has already completed a Phase 1 clinical trial (NCT04238364) that evaluated the safety, tolerability, and pharmacological properties of EI-1071 in about 50 healthy volunteers.
“Building on promising data from preclinical and Phase I studies, we are actively evaluating options to accelerate the development of EI-1071 to address unmet medical needs,” Chen said.
EI-1071 is an orally available small molecule designed to inhibit the colony stimulating factor-1 receptor (CSF-1R). As its name suggests, this protein receptor binds to a signaling molecule called colony stimulating factor-1, or CSF-1. This signaling molecule, in turn, plays a crucial role in driving the inflammatory activity of a type of immune cells called macrophages. These cells are known to drive inflammatory damage and tissue fibrosis (scarring) that causes lung damage in IPF.
In preclinical models of the disease, treatment with EI-1071 reduced fibrosis and improved animals’ breathing ability. According to Elixiron, EI-1071 has a potential best-in-class profile for CSF-1R inhibitors in terms of its potency compared with its toxicity.
Also of note, the experimental therapy is able to pass from the blood into the brain, and in preclinical models, it has shown the ability to suppress microglia — specialized macrophage-like immune cells that reside in the brain.
Elixiron also is developing EI-1071 as a potential treatment for Alzheimer’s disease in which increased brain inflammation is thought to play a role in driving disease progression.