Myrobalan Therapeutics Receives a Grant from the ALS Association to Advance its CSF1R Inhibitor Program

Grant funding advances preclinical candidate and deepens the scientific understanding of CSF1R inhibition in ALS

Myrobalan plans to initiate clinical testing in 2025

Myrobalan Therapeutics, a biotechnology company developing novel oral small-molecule therapies to repair damage and restore brain function in high-unmet need central nervous system (CNS) conditions, is honored to announce a $400,000 grant from the ALS Association to support the advancement of its colony-stimulating factor-1 receptor (CSF1R) inhibitor for the treatment of amyotrophic lateral sclerosis (ALS). The grant has been made through the Association’s Lawrence and Isabel Barnett Drug Development Program, supplementing the previously announced $24 million in Series A funding in January 2024.

ALS is a relentlessly progressive fatal neurodegenerative disease affecting the motor neurons in the brain and spinal cord. No cure currently exists for ALS, and patients have an average life expectancy of 2-5 years from diagnosis. While a handful of therapies are approved for ALS patients, the therapeutic benefit is limited. Myrobalan is developing a CSF1R inhibitor to target neuro-inflammation and demyelination with the goal to achieve a more profound therapeutic benefit in a broad patient population. Unlike other CSF1R candidates, Myrobalan’s CSF1R inhibitor program was specifically designed for application in CNS diseases with compelling advantages across several preclinical measures, including potency, selectivity, and CNS distribution. The company is currently conducting IND-enabling studies and developing a clinical biomarker strategy in preparation for its first-in-human clinical trial expected to begin in 2025. In addition to ALS, Myrobalan plans to develop its CSF1R inhibitor in additional neurodegenerative indications such as Multiple Sclerosis and Alzheimer’s Disease.

“We urgently need more and better treatments that can improve and extend the lives of people with ALS,” said Kuldip Dave, Ph.D., Senior Vice President of Research at the ALS Association. “We are proud to help drive the crucial transition from preclinical to clinical development for potential new ALS therapies through our Lawrence and Isabel Barnett Drug Development Program. Getting promising treatments out of the laboratory and into clinical testing as quickly as possible is key to making ALS a livable disease until we can cure it.”

“We are very pleased to receive this grant from the ALS Association to facilitate the development of our CSF1R inhibitor for the treatment of ALS and other high unmet-need neurodegenerative conditions. We are proud to have our program’s significance recognized by such a prestigious organization,” said Jing Wang, Ph.D., CEO & Co-Founder, Myrobalan Therapeutics. “This recognition affirms Myrobalan’s commitment to CNS drug development and empowers us to enhance our development efforts with respect to CSF1R and ALS. Ultimately, our goal is to make a CSF1R inhibitor therapy accessible to all patients grappling with ALS.”

About Myrobalan Therapeutics

Myrobalan Therapeutics is a preclinical stage biotechnology company headquartered in Medford, Massachusetts, with a focus on developing oral neurorestorative therapies aimed at reversing key pathologies underlying brain dysfunctions and CNS conditions. Myrobalan applies deep knowledge of neurological processes such as demyelination and neuroinflammation, and innovative drug discovery tools, to create highly selective and brain-penetrant therapeutic candidates that are uniquely positioned for treating CNS conditions with significant unmet medical need. Myrobalan’s pipeline of novel remyelination and anti-neuroinflammation programs are being advanced with the strategic support of renowned institutional co-founders and a leading chemistry partner. Myrobalan is committed to a transformed future in which patients with degenerative CNS conditions can access safe and effective medicines offering restorative potential. For more information on Myrobalan Therapeutics, visit our website at myrotx.com.

About The ALS Association

The ALS Association is the largest philanthropic funder of ALS research in the world. The ALS Association funds global research collaborations, assists people with ALS and their families through its nationwide network of care and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association is working to make ALS a livable disease while urgently searching for new treatments and a cure. For more information about the ALS Association, visit our website at www.als.org.

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"Ultimately, our goal is to make a CSF1R inhibitor therapy accessible to all patients grappling with ALS."