25 June 2026

First patient dosed with LTX-002; study now recruiting at 2 sites

• A clinical trial in Europe is testing the safety and pharmacological properties of LTX-002 as a potential treatment for ALS.
• The new therapy targets dysregulated fat metabolism, a key contributor to nerve damage.
• Developer Leal Therapeutics hopes the treatment candidate, an ASO, will help ease disease severity in people with ALS.

The first patient has been dosed in a Phase 1/2 clinical trial evaluating Leal Therapeutics‘ LTX-002, an antisense oligonucleotide designed to target abnormalities in fat metabolism that may contribute to amyotrophic lateral sclerosis (ALS).

The NeurALS trial (NCT07660614) is now recruiting an estimated 56 adults with ALS at two sites in Europe, with plans to add three more study locations. The first two sites are in the Netherlands and Sweden, with additional recruiting expected at centers in Italy and Germany.

The trial is testing the safety, tolerability, and pharmacological properties of LTX-002 at multiple ascending doses, and will also investigate the effects of the experimental therapy on measures of disease severity, according to a company press release announcing that the study is now underway.

“ALS patients have too few options, and recent science points clearly to dysregulated lipid metabolism as a driver of degeneration,” said Asa Abeliovich, PhD, Leal’s CEO and founder. “Dosing our first patient with LTX-002 is the first real test of this approach in people and a meaningful milestone for both Leal and the ALS community.”

Study links protein, fat regulator to nerve cell damage in ALS

Although the exact causes of ALS are not fully understood, disruptions in the metabolism of sphingolipids and ceramides, two classes of fat molecules, are believed to contribute to nerve cell damage.

In some cases, these abnormalities are linked to mutations in the SPTLC1 gene, which codes for a protein of the same name. SPTLC1 is a component of serine palmitoyltransferase (SPT), an important enzyme involved in the first step of sphingolipid production.

NeurALS is first human trial testing LTX-002 as ALS therapy

LTX-002 is an antisense oligonucleotide (ASO), a short strand of genetic material, designed to reduce production of SPTLC1 by targeting its messenger RNA, a molecule that serves as a template to make the protein. By lowering SPT activity, the therapy aims to reduce the accumulation of potentially harmful sphingolipids and ceramides associated with nerve cell damage in ALS.

According to Leal, abnormal accumulation of sphingolipids and ceramides has been observed in both sporadic and familial forms of ALS, suggesting that LTX-002 could benefit a broad range of ALS patients. The company previously raised $45 million to advance LTX-002 into clinical trials for ALS.

Preclinical data presented last year showed that LTX-002 reached key regions of the brain and spinal cord after being administered directly into the spinal canal in nonhuman primates. The therapy also lowered sphingolipid and ceramide levels without significant safety concerns.

Participants in NeurALS — the first-in-human trial to test the therapy — will be randomly assigned to receive LTX-002, at several dose levels, or a placebo. The therapy will be delivered directly into the spinal canal via intrathecal injection, with each patient receiving three injections over about three months, per the developer.

The study’s main goal is to assess the safety and tolerability of LTX-002 over about six months, while secondary goals are to determine LTX-002’s pharmacokinetics, or how it moves inside the body, and pharmacodynamics, meaning its effects in the body.

Researchers will also evaluate changes in SPTLC1 and sphingolipid levels in blood and spinal fluid, as well as exploratory measures such as daily functioning, muscle strength, lung function, and speech. Changes in neurofilament light chain levels, a marker of nerve damage, will also be assessed.

Source: ALS News Today