Drug Profile

LTX 002

Alternative Names: LTX-002

Latest Information Update: 20 Aug 2026

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At a glance

Originator Leal Therapeutics
Class Antidementias; Antisense oligonucleotides
Mechanism of Action Gene silencing; Serine C-palmitoyltransferase expression inhbitors

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

29 Apr 2026 Phase-I/II clinical trials in Amyotrophic lateral sclerosis in Germany, Italy (Intrathecal) (NCT07660614)
29 Apr 2026 Phase-I/II clinical trials in Amyotrophic lateral sclerosis in Sweden, Netherlands (Intrathecal) (NCT07660614)
13 Apr 2026 Leal Therapeutics plans a phase I/II trial for Amyotrophic lateral sclerosis in first half of 2026

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