$400K ALS Association grant will advance CSF-1R inhibitor program

Treatment developer Myrobalan also raised $24M in financing in January

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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Myrobalan Therapeutics will use a $400,000 grant from the ALS Association — along with $24 million in new financing — to help advance the development of its oral CSF1 receptor (CSF-1R) inhibitor program, intended to reduce neuroinflammation and promote nerve repair in people with amyotrophic lateral sclerosis (ALS).

The ALS Association grant was awarded via the nonprofit’s Lawrence and Isabel Barnett Drug Development Program, which supports the preclinical evaluation of new and repurposed therapies for the neurodegenerative disorder. It follows on the heels of a Series A funding by Myrobalan last month that raised nearly a quarter of a billion dollars to further the development of the company’s treatment program for people with ALS.

Myrobalan’s CSF-1R inhibitor program was specifically designed for use in diseases of the central nervous system, or CNS, which comprises the brain and spinal cord. The company is advancing the clinical biomarker strategy toward the first clinical trial of its CSF-1R inhibitor, which is expected to start next year, according to a Myrobalan press release. The new monies will keep the program on track, according to the release, which said Myrobalan was “very pleased to receive this grant from the ALS Association.”

“This recognition affirms Myrobalan’s commitment to CNS drug development and empowers us to enhance our development efforts with respect to CSF-1R and ALS,” said Jing Wang, PhD, the company’s CEO and co-founder.

“Ultimately, our goal is to make a CSF-1R inhibitor therapy accessible to all patients grappling with ALS,” Wang said.

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ALS is characterized by the progressive death of motor neurons, the nerve cells that control muscle movement. Loss of these neurons leads to symptoms like muscle weakness and wasting, with a progressively worsening impact on a patient’s ability to perform tasks of daily living.

Although its causes are largely unknown, several bodily systems are dysregulated in ALS, contributing to the disease’s onset and progression. Known ALS-related processes include inflammation of the brain — neuroinflammation — and demyelination, or the loss of the myelin sheath that protects nerve fibers and helps them send signals efficiently.

Causing these processes are alterations in microglia, the CNS’ resident immune cells, and myelin-producing cells called oligodendrocytes.

The company is working on oral, small molecule CSF-1R inhibitors. Myrobalan’s hypothesis is that intermittent treatment with CSF-1R inhibitors depletes microglia — whose ability to adapt is then impaired, thereby reducing inflammation and allowing remyelination, or the formation of new myelin.

We are proud to help drive the crucial transition from preclinical to clinical development for potential new ALS therapies.

According to Myrobalan, its CSF-1R inhibitor program was specifically designed for central nervous system diseases, and has shown potency, selectivity, and favorable CNS distribution in preclinical studies.

The ALS Association award, granted to scientists worldwide, is part of the nonprofit’s philanthropic funding.

“We are proud to help drive the crucial transition from preclinical to clinical development for potential new ALS therapies,” said Kuldip Dave, PhD, ALS Association’s senior vice president of research.

“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,” Dave said.

In addition to ALS, Myrobalan plans to develop its CSF-1R inhibitors for other neurodegenerative indications, such as multiple sclerosis and Alzheimer’s disease. The company also is developing other compounds to reduce microglia-driven neuroinflammation and improve remyelination.