Therapy for immune tolerance in MS shows safety in early trial
Oral LPX-TI641 tested at increasing doses in healthy adults in Phase 1 study
LPX-TI641, Lapix Therapeutics‘ experimental oral therapy to restore immune system balance in people multiple sclerosis (MS) and other autoimmune diseases, was found safe and well tolerated at all doses tested in healthy adults.
That’s according to top-line data from a Phase 1 trial (NCT05853835), which assessed single and multiple ascending doses of LPX-TI641 in healthy adults.
Data also showed the treatment significantly increased the levels of regulatory T- and B-cells, commonly known as Tregs and Bregs. These cells are essential to preventing the immune system from erroneously targeting and damaging healthy tissues and cells.
“The emerging preliminary data, particularly the observed exposure dependent changes in … T-regs and B-regs, highlight LPX-TI641’s potential as a leading therapy for autoimmune disease,” Anas M. Fathallah, PhD, Lapix’s co-founder and CEO, said in a company press release.
Immune tolerance works against the damaging immune attacks marking MS
As with other autoimmune diseases, MS is caused by the immune system starting to see certain cells and tissues as foreign, and launching an inflammatory attack that damages them. Often, these attacks are believed to result from a loss of immune tolerance, a process that enables the immune system to recognize the body’s own tissues and act to protect them.
Therapies able to restore this mechanism could be an alternative to disease treatments that commonly suppress the immune system, leave patients susceptible to infections.
LPX-TI641 is an oral small molecule designed to restore immune tolerance in people with autoimmune conditions. It is reported to work by activating the Tim family of cell receptors, which play an important role in autoimmunity. This is expected to increase the levels of Tregs and Bregs — immune cells that control excess inflammation and promote immune tolerance — while reducing the levels of T-cells involved in the inflammatory response.
The Phase 1 trial, which started dosing late last year, is assessing LPX-TI641’s safety, tolerability, and pharmacological properties at ascending doses in 70 healthy adults. Participants were divided into nine groups: six with people treated once at doses ranging from 10 mg to 150 mg or given a placebo, and three with people assigned to seven days of treatment with LPX-TI64 at doses of 30 mg-120 mg or given a placebo.
LPX-TI64 showed safety, tolerability at single and multiple ascending doses
Results showed that LPX-TI64 was safe and well tolerated at all doses. Adverse events were reported to be mostly mild, with headaches being the most common. Reported side effects were not dependent on the treatment dose.
No trial participant given LPX-TI64 at any dose showed low levels of immune cells called neutrophils and lymphocytes, side effects commonly observed with therapies for autoimmune diseases, the company noted.
A maximum tolerated dose also was not identified in the trial, it stated.
Lapix reports that it is exploring LPX-TI641 in treating MS and other brain and spinal cord autoimmune conditions, as well as rheumatoid arthritis, and psoriatic arthritis. It plans to initiate a Phase 1b trial in people with rheumatoid arthritis and psoriatic arthritis before the end of this year.
“We are eager to advance this promising candidate into a Phase Ib study … and create a potential new oral standard of care for patients battling autoimmune diseases,” Fathallah said.
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