nL-CHCHD-001

Phase 2Small molecule

Nn-Lorem Foundation

nL-CHCHD-001 is one of 51 small molecule programs targeting Amyotrophic Lateral Sclerosis. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Amyotrophic Lateral SclerosisActive, not recruiting

ALSNot yet recruiting

Amyotrophic Lateral SclerosisPreclinical

Active, not recruiting

ALSPhase 2

Not yet recruiting

Competition

Competitive Landscape

55 competitors in Amyotrophic Lateral Sclerosis

#DrugCompanyPhaseTrials

Enablex

AbbVie

Approved

—2

Nourianz

Kyowa Kirin Co., Ltd.

Vimpat

Bosulif

PPrism Pharmaceuticals

Approved

1 5

Qalsody

Biogen

Approved

3 6

Precedex

MMcNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.

Approved

—7

Quinidine Gluconate

WWyeth-Lederle Vaccines

Approved

—8

Transderm Scop

BBaxter Healthcare Corporation

Avodart

Mirapex

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q1 2027

Phase 1/2Not yet recruiting

ALS

NCT07423494

Q2 2027

PreclinicalActive, not recruiting

Amyotrophic Lateral Sclerosis

NCT06392126

PreclinicalActive, not recruiting

Amyotrophic Lateral Sclerosis

NCT06977451

Clinical

Trials by Indication[1]

Amyotrophic Lateral Sclerosis3 trials

An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for a Single Participant With Amyotrophic Lateral Sclerosis (ALS) Due to a Pathogenic Variant in CHCHD10

Active, not recruiting·Nn-Lorem Foundation·Preclinical·NCT06392126·1 enrolled

An Open-Label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to an Arg15Leu Pathogenic Variant in CHCHD10

Enrolling by invitation·Nn-Lorem Foundation·Preclinical·NCT07095686·9 enrolled

An Open-Label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to an Arg15Leu Pathogenic Variant in CHCHD10

Active, not recruiting·Nn-Lorem Foundation·Preclinical·NCT06977451·1 enrolled

ALS1 trial

Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)

Not yet recruiting·Nn-Lorem Foundation·Phase 1/2·NCT07423494·1 enrolled

Sources

References

[1]
ClinicalTrials.gov — 4 trial records for nL-CHCHD-001. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — nL-CHCHD-001 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for nL-CHCHD-001. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — n-Lorem Foundation company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: Nn-Lorem Foundation

nL-CHCHD-001

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2027

Q2 2027

Trials by Indication[1]

Amyotrophic Lateral Sclerosis3 trials

ALS1 trial

References

Drug Info

Quick Facts

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