Leal Therapeutics在LTX-002治疗肌萎缩侧索硬化症（ALS）的NeurALS 1/2期试验中完成首例患者给药

LTX-002 is a first-in-class antisense oligonucleotide targeting SPTLC1, designed to restore lipid metabolism in the CNS, a novel approach with the potential to address both sporadic and genetic forms of ALS

LTX-002是一种首创的靶向SPTLC1的反义寡核苷酸，旨在恢复CNS中的脂质代谢；这是一种新方法，有望同时应对散发性和遗传性ALS。

WORCESTER, Mass., June 23, 2026 /PRNewswire/ -- Leal Therapeutics, Inc. (Leal), a clinical-stage biopharmaceutical company developing first-in-class neuro-metabolic therapeutics, today announced that the first participant has been dosed in NeurALS, a Phase 1/2 trial of LTX-002 in adults with amyotrophic lateral sclerosis (ALS). LTX-002 is an investigational, intrathecally delivered antisense oligonucleotide designed to restore healthy sphingolipid levels in the central nervous system (CNS) by targeting SPTLC1.

马萨诸塞州伍斯特，2026年6月23日 /PRNewswire/ -- Leal Therapeutics, Inc.（Leal）是一家处于临床阶段、开发首创神经代谢疗法的生物制药公司。该公司今日宣布，在NeurALS中首名受试者已完成给药；NeurALS是一项在肌萎缩侧索硬化症（ALS）成人患者中开展的LTX-002 1/2期试验。LTX-002是一种研究性、鞘内给药的反义寡核苷酸，旨在通过靶向SPTLC1，恢复中枢神经系统（CNS）中健康的鞘脂水平。

LTX-002 targets SPTLC1, a core subunit of serine palmitoyltransferase (SPT). Excessive SPT activity results in the buildup of ceramides and sphingolipids, which are implicated in motor neuron toxicity and in both genetic and sporadic forms of ALS. By reducing SPT activity, LTX-002 is designed to restore healthy lipid balance in the CNS. SPT inhibition has been protective in preclinical models of ALS and related disorders.

LTX-002靶向SPTLC1，后者是丝氨酸棕榈酰转移酶（SPT）的核心亚基。SPT活性过高会导致神经酰胺和鞘脂积聚，而这些物质与运动神经元毒性以及遗传性和散发性ALS均有关。通过降低SPT活性，LTX-002旨在恢复CNS中的健康脂质平衡。在ALS及相关疾病的临床前模型中，SPT抑制已显示出保护作用。

NeurALS (LTX-002-101; NCT07660614) is a first-in-human, randomized, blinded, placebo-controlled study in adults with genetic or sporadic ALS, evaluating the safety, tolerability, and pharmacokinetics of LTX-002. Exploratory endpoints include CSF SPTLC1 protein and sphingolipid levels (validating target engagement), plasma neurofilament light chain (NfL; a marker of disease progression), and clinical measures including the ALSFRS-R rating scale, pulmonary vital capacity, and muscle strength.

NeurALS（LTX-002-101；NCT07660614）是一项首次人体、随机、盲法、安慰剂对照研究，在遗传性或散发性ALS成人患者中开展，评估LTX-002的安全性、耐受性和药代动力学。探索性终点包括CSF SPTLC1蛋白和鞘脂水平（验证靶点作用）、血浆神经丝轻链（NfL；疾病进展标志物），以及包括ALSFRS-R评分量表、肺活量和肌力在内的临床指标。

"ALS patients have too few options, and recent science points clearly to dysregulated lipid metabolism as a driver of degeneration," said Dr. Asa Abeliovich, CEO and Founder of Leal. "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."

Leal首席执行官兼创始人Asa Abeliovich博士表示：“ALS患者可选择的治疗方案太少，而近期科学研究明确指出，脂质代谢失调是退行性变的驱动因素。我们的首例患者接受LTX-002给药，是这种方法在人类中的首次真正检验，也是Leal和ALS社群的一个重要里程碑。”

Leal Therapeutics is a clinical-stage biopharmaceutical company developing first-in-class, brain-penetrant therapies for high-need CNS and metabolic disorders. Leal integrates human genetics, human biomarker data, human physiology, and preclinical model data to identify validated disease targets and develops brain-penetrating therapies to maximize target engagement. Learn more at lealtx.com.

Leal Therapeutics是一家处于临床阶段的生物制药公司，致力于为高需求的CNS和代谢性疾病开发首创、可进入脑部的疗法。Leal整合人类遗传学、人类生物标志物数据、人体生理学和临床前模型数据，以识别经过验证的疾病靶点，并开发可进入脑部的疗法，以最大限度提高靶点作用。更多信息请访问lealtx.com。