FDA clears first clinical study of investigational therapy LPX-TI641

Lapix Therapeutics is launching a Phase 1 clinical trial to evaluate LPX-TI641, its investigational treatment for autoimmune diseases such as multiple sclerosis (MS), in healthy individuals.

The first-in-human trial (NCT05853835) is expected to begin dosing later this year. It follows the recent clearance of an investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA), which enables Lapix to begin clinical testing of LPX-TI641.

“We believe in empowering and restoring the immune system to fight disease and the FDA clearance of our IND application for LPX-TI641 represents momentum in our technology and scientific approach,” Anas M. Fathallah, PhD, CEO and co-founder of Lapix, said in a press release.

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MS is marked by a misguided and overactive immune response against myelin, the fat-rich substance that wraps around nerve fibers and is necessary for efficient nerve communication.

This inflammatory response can happen due to the loss of immune tolerance, a process that enables the immune system to recognize the body’s own structures and not launch an attack against them.

Immune tolerance is lost in most autoimmune diseases, and restoring it may offer an alternative treatment for these conditions that does suppress the immune system and leaves patients more susceptible to infections — a known complication of most therapies for autoimmune conditions.

How LPX-TI641 works

LPX-TI641 is an orally available small molecule designed to restore immune tolerance by increasing the amount of regulatory T- and B-cells — immune cells that dampen excessive immune and inflammatory responses — while lowering the levels of certain T-cells known to contribute to myelin damage.

The treatment candidate works by activating the TIM family of receptors, and has shown a favorable efficacy compared with standard of care approaches in animal models of MS.

It is currently being investigated as a treatment for neuro-autoimmune conditions, including MS and the related conditions neuromyelitis optica spectrum disorder (NMOSD) and myelin oligodendrocyte glycoprotein antibody disease (MOGAD).

The upcoming Phase 1 trial is expected to enroll 48 healthy individuals, ages 18 to 55, at a single center in Jordan. Participants will receive a single administration of oral LPX-TI641 (at one of six ascending doses) or a placebo, and will be followed for two weeks.

Main study goal is safety, tolerability

The study’s main goal is to assess the therapy’s safety and tolerability, as assessed by the number and severity of side effects and dose-limiting toxicity. Researchers also will examine the drug’s pharmacokinetics, or its movement into, through, and out of the body.

“We are excited to begin our first-in-human clinical study evaluating LPX-TI641 and its potential to be a first of its kind non-immune suppressive therapeutic option for autoimmune diseases,” Fathallah said.

The company recently secured a new U.S. patent covering LPX-TI641 to restore immune tolerance in people with autoimmune diseases.

“We aspire to transform how the medical field treats patients diagnosed with serious and complex immune diseases and we are deeply committed to helping as many people as possible,” Fathallah added. “Our in-depth knowledge of immunology and immune tolerance are the keys to unlocking transformative therapies for patients with additional consideration for their quality of life.”