Montara to develop LRRK2 inhibitor drug for Parkinson’s
Work on brain-targeting therapy to be funded by $3.3M MJFF grant
The Michael J. Fox Foundation (MJFF) has awarded a $3.3 million research grant to Montara Therapeutics to develop a brain-selective therapy for Parkinson’s disease using the company’s Brainonly platform.
As part of the project, Montara will design and develop an LRRK2 inhibitor drug candidate that can reach the brain and avoid the adverse effects typically associated with blocking LRRK2 in peripheral organs, such as the lungs and kidneys.
“MJFF’s network and expertise make them a wonderful partner to our mission of developing Brainonly therapies that selectively target the [central nervous system, or] CNS while blocking harmful peripheral effects — in this case, enabling and accelerating the development of new therapies for Parkinson’s,” Nicholas T. Hertz, PhD, Montara’s founder and CEO, said in a company press release. The body’s CNS comprises the brain and spinal cord.
Mutations in the LRRK2 gene, which produces an enzyme of the same name, are among the most common genetic causes of Parkinson’s. When the gene is mutated, the enzyme can become overly activated, contributing to the cellular dysfunction and neurodegeneration that characterize the disease.
Looking to an LRRK2 inhibitor as a novel therapeutic strategy
Research has suggested that blocking LRRK2 may be a therapeutic strategy for Parkinson’s, but so far there are no commercially available therapies targeting the protein.
According to Montara, many promising candidates for neurological diseases fail to advance due to dose-limiting issues in the brain and adverse effects in peripheral organs (those outside the brain) that impact their efficacy and safety.
The company’s Brainonly platform aims to develop drugs that work solely in the brain, helping avoid side effects in other parts of the body. It uses a two-drug method: one drug goes into the brain to treat the disease, while the second drug stays outside the brain to block the first drug from affecting other organs.
This two-pronged approach is expected to prevent peripheral adverse events, increasing the treatment’s safety and tolerability, as well as its efficacy against LRRK2, according to Montara.
Montara will join Michael J. Fox Foundation’s LITE program
The company is also joining MJFF’s LRRK2 Investigative Therapeutics Exchange (LITE) program, which supports the development of new therapies targeting LRRK2 and advances toward new LRRK2-relevant clinical biomarkers.
“The LITE initiative seeks to provide opportunities for better understanding Parkinson’s and treating it,” said Dario Alessi, PhD, professor and researcher at the University of Dundee in Scotland and a LITE principal investigator. “We welcome Montara to the initiative and are excited to explore how the company’s Brainonly platform could unlock the enormous potential of LRRK2-targeted therapies in a way that mitigates adverse effects.”
The team behind Montara had previously founded Mitokinin, fully acquired by Abbvie in 2023, which was developing an activator of PINK1, an enzyme that protects brain cells against stress. Mutations in the PINK1 gene are also linked to Parkinson’s.
Miratul Muqit, a professor at the University of Dundee and a member of Montara’s scientific advisory board, said these scientists have “an impressive track record in the Parkinson’s space, having advanced a potentially disease-modifying treatment at Mitokinin.”
The team is “really well positioned to replicate this success with the Brainonly platform and its promise for LRRK2-targeted therapy,” Muqit said.
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