Dr. Noah Biotech's AI-Based Complex Drug for Lou Gehrig's Disease Designated as Orphan Drug by US FDA

Dr. Noah Biotech (CEO Ji-Hyun Lee) announced on the 11th that the complex drug NDC-011, being developed as a treatment for amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease), received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) on the 28th of last month.

Additionally, the excellent preclinical research results on the efficacy of NDC-011 were recognized and published online on the 2nd in the internationally prestigious journal of the American Neurological Therapeutics Society, Neurotherapeutics.

The complex drug NDC-011, discovered using Dr. Noah Biotech Co., Ltd.'s proprietary artificial intelligence drug development platform ‘ARK,’ possesses anti-inflammatory properties, neuroprotective and myoprotective effects, and promotes neuronal differentiation. In ALS experimental animal models, it demonstrated delayed motor function decline, inhibition of motor neuron loss, promotion of neuronal differentiation, and extended survival of the test animals.

Based on the excellent preclinical results (ALS progression inhibition and survival extension), NDC-011’s designation as an orphan drug by the U.S. FDA enables it to receive various benefits such as clinical trial protocol design consultation, reduced review fees, tax credits, priority review, and exclusive marketing rights, thereby accelerating global new drug development. Furthermore, the application for a Pre-IND meeting to enter FDA clinical trials has been completed, with the meeting scheduled to take place within this year.

A representative of Dr. Noah Biotech stated, “We expect NDC-011 to alleviate the progression and symptoms of ALS and extend patient survival. Encouraged by this orphan drug designation, we are accelerating the FDA clinical trial application process.”

ALS, known as Lou Gehrig's disease, is a condition in which only motor neurons selectively degenerate. Upon onset, both the upper motor neurons in the cerebral cortex and the lower motor neurons in the brainstem and spinal cord are progressively destroyed. Consequently, muscles weaken sequentially due to lack of motor neuron stimulation, leading to loss of voluntary motor control and ultimately death. It is generally known that about 50% of patients die within 3 to 4 years.

Currently, although there are approved and prescribed ALS treatments, their therapeutic effects fall short of expectations, making the development of new treatments urgently needed.

Meanwhile, Dr. Noah Biotech is an AI-based drug development specialist company aiming to be the first to bring AI-developed drugs to market. Experts in AI systems, drug development, clinical development, and business strategy have collaborated to implement AI technology specialized in drug combinations.

In July, the company successfully completed Phase 1 clinical trials of the complex drug ‘NDC-002,’ being developed as a stroke recovery treatment, and completed the Pre-IND meeting application with the U.S. FDA to enter global Phase 2 clinical trials.

This content was produced with the assistance of AI translation services.