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

MEDFORD, Mass.--(BUSINESS WIRE)--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

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


Media Contact at Myrobalan Therapeutics:
Sushmitha Gururaj
Ten Bridge Communications

Media Contact at the ALS Association:
Brian Frederick

迈巴制药 (Myrobalan Therapeutics ) CSF1R抑制剂项⽬获得肌萎缩性侧索硬化症协会(ALS Association)资助资⾦将用于推动临床前候选药物的研发,以及加深对ALS中CSF1R抑制作⽤的科学理解


February 07, 2024 07:00 AM Eastern Standard Time

梅德福,马萨诸塞州。--(BUSINESS WIRE)--(美国商业资讯)-- 迈巴制药,一家致力于开发新型口服小分子神经修复疗法,旨在修复中枢神经系统损伤并恢复神经功能,以满足神经退行性疾病中的重大未满足临床需求的生物技术公司,荣幸地宣布从肌萎缩性侧索硬化症协会(ALS Association)获得40万美元的研发资金,以⽀持其针对治疗肌萎缩性侧索硬化症(ALS)的集落刺激因⼦-1受体(CSF1R)抑制剂的研发。该研发资金是由该协会的Lawrence和Isabel Barnett药物开发计划提供的。迈巴制药在2024年1⽉宣布了2400万美元A轮融资。这次ALS协会的资助,将进一步加强公司的资金储备。

ALS是⼀种持续恶化的致命性神经退⾏性疾病,影响⼤脑和脊髓中的运动神经元。⽬前尚⽆治愈ALS的⽅法,患者从诊断起平均预期寿命为2-5年。虽然少数针对 ALS患者的疗法已获批准,但治疗功效有限。迈巴制药正在开发⼀种CSF1R抑制剂,靶向神经炎症和脱髓鞘两个致病性的因素,以期在⼴泛的患者⼈群中实现更深远的治疗功效。与其他CSF1R候选物不同,迈巴制药的CSF1R抑制剂项⽬专为中枢神经系统疾病应⽤⽽特别设计,具有多个临床前测量指标上的显著优势,包括对靶点的抑制能力、选择性和中枢神经系统分布。该公司⽬前正在进⾏ IND(新药研究)启动实验,并制定了临床⽣物标志物的策略,以准备2025年开始的⾸次⼈体临床试验。除了ALS,迈巴制药还计划在多发性硬化症和阿尔茨海默病等其他神经退⾏性病症中开发其CSF1R抑制剂。

“我们迫切需要更多更好的治疗⽅法,这些⽅法能够改善并延⻓ALS患者的⽣命,”ALS协会的研究⾼级副总裁Kuldip Dave博⼠表示。“我们很⾃豪能够通过我们的 Lawrence和Isabel Barnett药物开发计划,帮助推动从临床前到临床开发的关键转变,为潜在的新ALS治疗⽅法铺路。在我们找到治愈方法之前,将有潜⼒的治疗⽅案迅速从实验室阶段过渡到临床试验,对于将ALS转变为⼀种可控制的疾病⾄关重要。”

“我们⾮常⾼兴从ALS协会收到这笔资⾦,以促进我们的CSF1R抑制剂在治疗 ALS和其他有着巨⼤未满⾜医疗需求的神经退⾏性疾病⽅⾯的开发。我们很⾃豪我们的项⽬得到了⼀个声望卓著组织的认可,”迈巴制药的⾸席执⾏官兼联合创始⼈王静博⼠表示。“这⼀认可肯定了迈巴制药对中枢神经系统药物开发的投入,并赋予我们更多针对CSF1R和ALS开发⼯作的⼒量。最终,我们的⽬标是使CSF1R抑制剂疗法能够惠及所有与ALS抗争的患者。”

迈巴制药简介(Myrobalan Therapeutics)


肌萎缩性侧索硬化症协会简介(ALS Association)




Sushmitha Gururaj
Ten Bridge Communications

ALS Association的媒体联系人:
Brian Frederick