Seoul, Korea - DR.NOAH BIOTECH (hereafter DR.NOAH), an AI-driven innovative drug development company, announced on August 23 that it had received approval from the U.S. Food and Drug Administration (FDA) for the Investigational New Drug (IND) application for Phase 1 clinical trial of a new drug combination (NDC-011) for Amyotrophic Lateral Sclerosis (ALS).
This Phase 1 clinical trial is planned to be conducted at a specialized clinical trial institution in the U.S., involving 24 healthy adult participants. The study is designed to compare and evaluate the bioavailability, safety, and tolerability of single doses of the individual drugs composing NDC-011, which is a two-drug combination, and NDC-011 itself. The Phase 1 trial is scheduled to begin in the first half of 2025, while Phase 2 is expected to start in 2026.
NDC-011 is a combination drug discovered through DR.NOAH's proprietary AI drug development platform, "ARK." It is designed to have multiple mechanisms of action in ALS patients, including effects of anti-inflammation, neuroprotection, neuronal differentiation promotion, and muscle cell protection.
DR.NOAH used ARK to analyze ALS patient data and over 1.6 million drug combinations to predict candidate drugs. NDC-011 was selected as the final candidate after showing the most significant results in preclinical trials.
The efficacy of NDC-011 was confirmed by ALS preclinical study for delayed motor function deterioration and prolonged survival. These study results were published in the October 2023 issue of "Neurotherapeutics," an international journal of the American Society for Experimental Neurotherapeutics. In September 2023, it received an orphan drug designation (ODD) from the FDA.
NDC-011 was aimed at global market expansion from the early stages of development. For this purpose, formulation/preparation development, production, and quality control of the fixed-dose combination (FDC) in tablet form were carried out at the cGMP facility of Patheon, a top-class global contract development and manufacturing organization (CDMO), in the U.S.
"Although there are currently approved medicines for ALS, their therapeutic effects fall short of expectations, making the development of new treatments an urgent necessity," a DR.NOAH's representative stated. "NDC-011 is expected to alleviate the progression and symptoms of ALS and prolong patient survival."
The representative also added, "Our company is not just an AI technology service provider, but a drug development company capable of handling on our own the entire new drug development process from discovery to clinical development."
DR.NOAH BIOTECH is an AI-driven, innovative drug development company that develops medicines for neurological and muscular diseases. Starting with the launch of the AI drug development platform "ARK" in 2017, the company began its own drug development. The ARK platform consists of NOTE databases containing patient genomic data, drug genomic data, drug 3D structural data, medical big data, and neural/muscle cell imaging data, as well as various AI systems (CombiNet, SF-Rx, VLab, NeuroRG) that analyze these data. ARK's core technology is the accurate prediction of multiple pathological networks critical to diseases and the discovery of optimal drug combinations to modulate them.
DR.NOAH is currently developing eight different combination drug pipelines. Among them, NDC-002 for stroke recovery successfully completed Phase 1 clinical trials in Korea in 2023. This is significant since it is the first time an AI company in Korea has conducted Phase 1 clinical trials for an AI-developed drug in Korea. DR.NOAH is particularly focused on developing medicines for rare diseases. In addition to NDC-011 for ALS treatment, which has just received Phase 1 clinical trial approval, NDC-026 is also under development for Duchenne muscular dystrophy (DMD) treatment. NDC-026 received a rare pediatric disease designation (RPDD) and an orphan drug designation (ODD) from the FDA in April. The goal is to submit an IND application in 2025. The initiation of clinical trials for NDC-011 is expected to be the first case of clinical trials for an AI-discovered combination drug for ALS treatment. "Starting with this NDC-011 clinical trial, we will continue to develop medicines for rare diseases," Dr.Ji-hyun Lee, CEO of DR.NOAH, stated. "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."
Media Contact
Company Name: DR.NOAH BIOTECH Inc.
Contact Person: Changhyun Jeong (IR team leader)
Email: Send Email
Country: South Korea
Website: drnoahbiotech.com
