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ITRil (Intrathecal Riluzole)

Amyotrophic Lateral Sclerosis (ALS)

Phase 1Active

Key Facts

Indication

Amyotrophic Lateral Sclerosis (ALS)

Phase

Phase 1

Status

Active

Company

Brain Trust Bio

About Brain Trust Bio

Brain Trust Bio is a private, pre-revenue biotech company developing a precision intrathecal drug delivery platform to enhance existing CNS therapies. Founded in 2018 and based in San Francisco, the company has secured approval to initiate Phase I trials in Australia for its lead program, ITRil (intrathecal riluzole), for ALS, building on off-label treatment of two patients. The company's strategy centers on reformulating established drugs to achieve better efficacy and tolerability by delivering them directly to the cerebrospinal fluid, thereby accelerating development timelines and aiming for significant patient impact in debilitating neurological conditions.

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Therapeutic Areas

Amyotrophic Lateral Sclerosis Central Nervous System Diseases

Other Amyotrophic Lateral Sclerosis (ALS) Drugs

Drug	Company	Phase	Status
NPT520-34	Neuropore Therapies	Pre-clinical/Phase 1	Partnered (Biogen), Active
NUZ-001	Neurizon Therapeutics	Phase 2/3 (Platform Trial)	Active
CNM-Au8	Clene	Phase 2/3	Active
Masitinib	AB Science	Phase 3	Active
Undisclosed Program	Zhimeng Biopharma	Clinical	Active
SLS-005	Seelos Therapeutics	Platform Trial	Active (HEALEY ALS Platform Trial – Regimen E)
PMN442	ProMIS Neurosciences	Preclinical	Active
nAV-101	nVector	Pre-clinical	Active
ALTA-808	Alteron Therapeutics	Preclinical	IND-enabling studies launching
GM6	Genervon Biopharmaceuticals	Phase 2	Active
Lou Gehrig’s disease	Genclis	Discovery	Active
Treg-directed therapy for ALS	Iltoo Pharma	Pre-clinical	Active