Myrobalan Pipeline
- Myrobalan is pioneering first-in-class and best-in-class small molecule therapies targeting novel mechanisms critical to a wide range of CNS diseases with significant unmet needs.
- Our approach centers on combating demyelination and neuroinflammation, two fundamental processes underlying conditions like multiple sclerosis, Alzheimer's disease, ALS, Parkinson's disease, and various other demyelinating and neurodegenerative disorders.
TARGET
POTENTIAL
INDICATIONS
DISCOVERY
PRECLINICAL
IND
ENABLING
PHASE 1
CSF1R
ALS, MS, AD, PD
Neuroinflammation
Remyelination
Across various disease states, dysfunctional microglia have been shown to incite neuroinflammation, impede remyelination, and accelerate axonal degeneration. In preclinical studies, our CSF1R inhibitor effectively eliminated these aberrant microglia from affected CNS regions, facilitating the natural repopulation of healthy microglia. This process resulted in decreased neuroinflammation and enhanced both myelination and axonal integrity.
Learn more about the CSF1R for CNS applicationsGPR17
MS, MOGAD, NMO, Stroke, PVL, AD, ALS, PD
Remyelination
Our GPR17 antagonist, which targets a key inhibitor of oligodendrocyte precursor cell differentiation and maturation, is advancing through preclinical development as a potential remyelination therapy. Preclinical studies have shown that our compounds exhibit strong potency, specificity, and ability to cross the blood-brain barrier. Additionally, we've observed significant biological effects in primary cell cultures, human iPSC-derived cells, and animal models.
Learn more about the GPR17 TargetUndisclosed
Demyelinating disease
What is anti-neuroinflammation?
Anti-neuroinflammation - ameliorate the CNS environment:
Neuroinflammation is a natural response involving the activation of immune cells and the release of inflammatory mediators in the CNS in response to various insults or injuries (e.g., neurodegenerative diseases).
Unmet Need
Uncontrolled overactivation of inflammation in the CNS is a key pathological driver of many neurological diseases.
Our Approach
We seek to alleviate neuroinflammation and restore a neuroprotective brain environment through a multi-faceted approach.
What is remyelination?
Remyelination - repair and protect “data cables”:
Myelin is the protective insulation that wraps around axons. It serves crucial functions such as enabling rapid and efficient electrical signal transmission along axons, which is essential for proper neuronal functions. Additionally, myelin plays a role in providing metabolic support and protection for maintaining the health and integrity of axons, contributing significantly to their survival and function.
Unmet Need
When myelin is damaged in demyelinating diseases, signal transduction is impaired, leading to neuronal dysfunction both clinically and physiologically.
Our Approach
We are developing targets that promote remyelination. By repairing damaged myelin, we aim to restore function in the CNS and potentially modify the course of demyelinating diseases.