First healthy volunteers dosed in Phase 1 trial of LPX-TI641

A Phase 1 clinical trial evaluating LPX-TI641, Lapix Therapeutics‘ experimental therapy for multiple sclerosis (MS) and other autoimmune diseases, has dosed its first participants.

The first-in-human trial (NCT05853835) of healthy adult volunteers follows the recent clearance of an investigational new drug application by the U.S. Food and Drug Administration (FDA), enabling Lapix to begin clinical testing of LPX-TI641.

“The dosing of the first participants is a major milestone in the clinical development of LPX-TI641,” Anas M. Fathallah, PhD, Lapix’s co-founder and CEO, said in a press release.

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The Phase 1 clinical trial is expected to enroll 48 healthy adults, ages 18 to 55, at two sites in the U.S. and Jordan. Only the U.S. site is currently recruiting.

Participants will receive either a single administration of LPX-TI641, at one of six ascending doses, or a placebo, and will be evaluated for two weeks. The main goal is to assess the safety and tolerability of the candidate therapy, but researchers also will study its pharmacological properties and explore its effects on certain disease biomarkers.

“We look forward to evaluating the therapeutic potential of LPX-TI641 and believe it will be a transformative and safer option for patients suffering from MS and other autoimmune diseases,” Fathallah said.

MS is caused by the immune system mistakenly attacking the myelin sheath, a fatty coating around nerve fibers that’s necessary for efficient nerve communication.

This inflammatory response is believed to result from loss of immune tolerance, in which the immune system’s ability to recognize the body’s tissues as its own and not attack them is impaired. Research now increasingly recognizes that immune tolerance is lost in most autoimmune conditions.

Restoring this mechanism may help treat these diseases, offering an alternative to current therapies that dampen the immune system and leave patients more susceptible to infections and other opportunistic diseases.

LPX-TI641 is designed to restore immune tolerance

LPX-TI641 is an oral small molecule designed to restore immune tolerance by activating the TIM receptors, which play an important role in autoimmunity.

This is expected to increase the amount of regulatory T- and B-cells — immunosuppressive cells that control excess inflammation and promote self-tolerance — while reducing the levels of T-cells involved in the faulty inflammatory response.

In animal models of MS, LPX-TI641 has demonstrated favorable safety and efficacy compared with standard care MS treatments, without inducing meaningful reductions in immune cell numbers.

The investigational therapy is being developed for the treatment of MS and the related conditions neuromyelitis optica spectrum disorder (NMOSD) and myelin oligodendrocyte glycoprotein antibody disease (MOGAD). It also can be a potential treatment for other autoimmune diseases, including lupus and rheumatoid arthritis.

“The design of the Phase I study will allow us the flexibility to leverage the clinical data for other indications, enabling rapid pivots into other autoimmune diseases,” Fathallah said.

The company earlier this year secured a new U.S. patent covering the use of LPX-TI641 as a means to restore immune tolerance in people with autoimmune diseases.