DR.NOAH Biotech's AI-Driven ALS Drug, NDC-011, Receives FDA Approval for Phase 1 Trial
Key Insights
DR.NOAH Biotech (search) received FDA approval for its Investigational New Drug (IND) application for Phase 1 clinical trial of NDC-011 (search), a new drug combination for Amyotrophic Lateral Sclerosis (search) (ALS (search)).
The Phase 1 trial, set to begin in early 2025, will evaluate the bioavailability, safety, and tolerability of NDC-011 (search) in 24 healthy adult participants in the U.S.
NDC-011 (search), discovered through DR.NOAH's AI platform 'ARK,' showed efficacy in preclinical studies by delaying motor function deterioration and prolonging survival in ALS (search) models.
DR.NOAH BIOTECH (search), an AI-driven drug development company, has received FDA approval for its Investigational New Drug (IND) application for a Phase 1 clinical trial of NDC-011 (search), a novel drug combination for Amyotrophic Lateral Sclerosis (search) (ALS (search)). This marks a significant step for the company and potentially offers a new avenue for treating this debilitating disease.
The Phase 1 clinical trial is scheduled to commence in the first half of 2025 at a specialized clinical trial institution in the U.S. The study will involve 24 healthy adult participants and is designed to assess the bioavailability, safety, and tolerability of single doses of the individual drugs composing NDC-011 (search), as well as the combination drug itself. Phase 2 trials are anticipated to begin in 2026.
AI-Driven Drug Discovery
NDC-011 (search) is a combination drug identified using DR.NOAH's proprietary AI drug development platform, "ARK." The drug is designed to exert multiple mechanisms of action in ALS (search) patients, including anti-inflammatory, neuroprotective, neuronal differentiation promotion, and muscle cell protection effects. The ARK platform analyzed ALS patient data and over 1.6 million drug combinations to predict candidate drugs, with NDC-011 selected based on its promising preclinical results.
Preclinical Efficacy and Orphan Drug Designation
Preclinical studies have demonstrated that NDC-011 (search) can delay motor function deterioration and prolong survival in ALS (search) models. These findings were published in the October 2023 issue of "Neurotherapeutics." In September 2023, the FDA granted NDC-011 orphan drug designation (ODD), further supporting its development for this rare disease.
Manufacturing and Global Expansion
From the early stages of development, DR.NOAH has aimed for global market expansion. To this end, formulation, preparation, production, and quality control of the fixed-dose combination (FDC) tablet form of NDC-011 (search) were conducted at the cGMP facility of Patheon, a global contract development and manufacturing organization (CDMO) in the U.S.
The Need for New ALS Treatments
"Although there are currently approved medicines for ALS (search), their therapeutic effects fall short of expectations, making the development of new treatments an urgent necessity," stated a DR.NOAH representative. "NDC-011 (search) is expected to alleviate the progression and symptoms of ALS and prolong patient survival."
DR.NOAH's Broader Pipeline
DR.NOAH is also developing NDC-026 (search) for Duchenne muscular dystrophy (search) (DMD (search)), which has received rare pediatric disease designation (RPDD) and orphan drug designation (ODD) from the FDA in April. The company plans to submit an IND application for NDC-026 in 2025. The initiation of clinical trials for NDC-011 (search) is expected to be the first instance of clinical trials for an AI-discovered combination drug for ALS (search) treatment. Dr. Ji-hyun Lee, CEO of DR.NOAH, stated, "Starting with this NDC-011 clinical trial, we will continue to develop medicines for rare diseases. We will do our best to ensure that our fast and efficient AI-driven combination drug development strategy will bring hope to rare disease patients worldwide."