Myrobalan Therapeutics Announces MRO-001 Selected for China’s CARE Program Extension Pilot for ALS Development
Myrobalan Therapeutics today announced that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has formally included MRO-001, the company’s investigational oral CSF1R inhibitor, in the Rare Disease Innovative Drug Development Encouragement Pilot Program, known as the “CARE Program Extension,” for the treatment of amyotrophic lateral sclerosis (ALS).
The CARE Program Extension is a Chinese regulatory development initiative designed to support drug development for rare diseases with urgent clinical needs and to address key scientific questions arising from the development of innovative rare disease therapies. It provides a framework for earlier engagement and continued scientific communication with the CDE to address important questions during drug development.
ALS is a progressive, disabling and life-threatening neurodegenerative disease characterized by the gradual loss of motor neurons, ultimately leading to muscle weakness, paralysis and respiratory failure. Despite recent advances, there remains a substantial unmet need for therapies that can meaningfully slow disease progression. A growing body of evidence indicates that neuroinflammation and dysregulated microglial activity are important contributors to ALS progression.
MRO-001 is an orally bioavailable, brain-penetrant and selective small-molecule inhibitor of colony-stimulating factor 1 receptor (CSF1R), which is important for microglial survival and maintenance. MRO-001 is being developed to investigate whether CSF1R inhibition can reduce microglia-associated neuroinflammatory processes implicated in ALS, and slow motor-neuron injury and disease progression.
“The inclusion of MRO-001 in the CARE Program Extension is an important milestone for both the program and our company,” said Jing Wang, Ph.D., Co-Founder and Chief Executive Officer of Myrobalan Therapeutics. “We appreciate the CDE’s continued attention to rare disease innovation and patient needs. People living with ALS continue to face limited treatment options, and we hope to develop a new therapy with the potential to modify disease progression through a differentiated mechanistic approach. Participation in the pilot program will also enable closer and more continuous scientific dialogue with regulators as the program advances. We remain committed to developing MRO-001 with scientific rigor and operational urgency as we work to bring new treatment options to people living with ALS.”
Myrobalan Therapeutics is a biotechnology company focused on developing innovative therapies for neurodegenerative and neuroinflammatory diseases. Built around two core biological mechanisms, modulation of neuroinflammation and promotion of myelin repair and regeneration, the company has established a differentiated small-molecule drug development platform. Its pipeline encompasses multiple high-value CNS targets with the potential to address a broad range of neurological disorders. Through its science-driven approach, Myrobalan aims to develop disease-modifying therapies for patients with significant unmet medical needs.
Leveraging a global operating model spanning the United States, China and Australia, Myrobalan integrates international scientific expertise and drug development experience with translational and clinical development capabilities in China. Through this “global innovation, China translation” model, the company aims to advance differentiated therapies for patients with serious neurological diseases worldwide.
About Myrobalan Therapeutics
Myrobalan Therapeutics is a biotechnology company dedicated to developing disease-modifying therapies for neurological disorders. Through innovative biology, translational science and global clinical development capabilities, the company is advancing oral small-molecule therapies for diseases with significant unmet medical needs.
Forward-Looking Statements
This press release contains forward-looking statements regarding the development of MRO-001 and its potential therapeutic value. These statements reflect Myrobalan’s current expectations and are subject to risks and uncertainties, including regulatory review, study execution and nonclinical or clinical outcomes. MRO-001 is investigational, has not been approved for commercial use, and its safety and efficacy have not been established by any regulatory authority.