Highlights:
- Montara Therapeutics awarded non-dilutive funding grant from The Michael J. Fox Foundation (MJFF) to advance a BrainOnly™ therapy targeting mTOR, with the goal of activating the brain’s natural protein-clearing machinery to address toxic α-synuclein buildup in Parkinson’s disease
- Project will leverage Montara’s BrainOnly™ platform to restrict mTOR inhibitor activity to the brain, aiming to reduce the systemic side effects that have historically prevented this drug class from reaching Parkinson’s patients
SAN FRANCISCO, June 02, 2026 (GLOBE NEWSWIRE) — Montara Therapeutics, a biotech company pioneering brain-selective therapies for central nervous system (CNS) diseases, today announced it has been awarded a research grant of approximately $1 million from The Michael J. Fox Foundation for Parkinson’s Research (MJFF). The funding, Montara’s second grant from MJFF following an award in May 2025 to develop a brain-selective LRRK2 inhibitor, will be used to develop their BrainOnly™ platform to activate autophagy in the brain, promote clearance of the toxic α-synuclein protein implicated in Parkinson’s disease, while aiming to mitigate adverse effects throughout the body.
This work is supported by MJFF through its Therapeutics Pipeline Program, which funds preclinical and clinical efforts to accelerate the development of new therapies for Parkinson’s disease.
Parkinson’s disease is characterized in part by the accumulation of α-synuclein, a toxic protein that clumps together and damages brain cells over time. One promising strategy for removing this protein is activation of autophagy, a natural cellular housekeeping process in which cells break down and recycle damaged components. The protein mTOR acts as a brake on this process: when mTOR is active, autophagy is suppressed; when mTOR is inhibited, the brake is released, and the cell’s protein-clearing machinery is switched on. Drugs that inhibit mTOR, a class that includes rapamycin and related compounds, which have also attracted sustained scientific interest for their role in aging biology, have shown encouraging results in clearing α-synuclein in laboratory models of Parkinson’s disease. However, because mTOR plays essential roles throughout the body, systemic mTOR inhibition causes serious side effects, including immune suppression, impaired wound healing, and metabolic disruption, severely limiting the use of these drugs as neurological therapies.
This challenge is one Montara knows well. The company is already advancing its proprietary peripheral blocker, MT1110, toward the clinic in combination with everolimus, an mTOR inhibitor, to treat tuberous sclerosis complex (TSC)-related epilepsy, another disease driven by mTOR pathway hyperactivation. The Parkinson’s program builds directly on that foundation, applying the same BrainOnly™ approach to a new indication where mTOR-driven autophagy could clear the toxic protein accumulation at the heart of the disease.
Montara’s BrainOnly™ platform is designed to address this challenge by pairing a brain-penetrant therapeutic with a proprietary peripheral blocker that prevents the drug from acting outside the brain. This approach enables brain-specific pharmacology, allowing therapies to target mechanisms underlying neurological disease while minimizing harmful systemic effects.
Through this collaboration with MJFF, Montara will evaluate several clinically utilized mTOR inhibitors combined with its proprietary peripheral blocker to identify novel two-drug combinations that selectively activate autophagy in the brain. The program will assess these combinations in cell-based systems and Parkinson’s disease animal models to determine which candidates most effectively reduce α-synuclein accumulation, treat pathology, and exhibit favorable safety profiles.
If successful, this project could lead to a potential therapeutic strategy that safely enhances autophagy in the brain, helping remove toxic protein buildup and potentially slowing, or even stopping, the progression of Parkinson’s disease.
“Our team has spent years working toward a therapy that doesn’t just treat the symptoms of Parkinson’s but addresses the underlying biology causing neurons to die,” said Nicholas T. Hertz, Ph.D., Founder and CEO of Montara Therapeutics. “MJFF’s continued support reflects the importance of exploring new approaches to Parkinson’s disease biology. The mTOR pathway is one of the most powerful levers we have for clearing toxic proteins from the brain, and our platform may finally make it safe enough to use.”
“The mTOR pathway represents an important area of investigation in Parkinson’s research,” said Jessica Tome Garcia, Lead Scientific Program Manager, Translational Research at The Michael J. Fox Foundation for Parkinson’s Research. “While the underlying biology is compelling, challenges related to systemic toxicity have limited progress. This work aims to explore approaches that may help address those barriers and advance our understanding of how targeting mTOR-driven autophagy could impact disease biology.”
“Our own genome-wide screens in human neurons identified mTOR signaling as one of the key pathways controlling the accumulation of toxic protein aggregates — and a target with real therapeutic potential,” said Martin Kampmann, Professor of Biochemistry and Biophysics at UCSF and a Scientific Co-Founder of Montara Therapeutics. “The challenge has always been that you cannot inhibit mTOR systemically without serious consequences for the rest of the body. Montara’s BrainOnly™ platform is the most compelling approach I’ve seen for solving that problem, and this program gives us a direct path to test whether brain-selective mTOR inhibition can reduce pathological protein buildup in Parkinson’s disease.”
About Montara Therapeutics
Montara Therapeutics is a preclinical-stage biopharmaceutical company pursuing novel approaches to develop safer and more efficacious treatments for neurological diseases. Montara’s BrainOnly™ platform enables brain-specific pharmacology by leveraging existing and novel chemical warheads, restricting deleterious on-target / off-tissue peripheral activity. BrainOnly™ holds the potential to drug numerous targets previously considered undruggable, greatly expanding the therapeutic options for patients in need of these therapies. Montara is supported by an outstanding group of investors, including founding investor SV Health Investors’ Dementia Discovery Fund, Two Bear Capital, KdT Ventures, Dolby Family Ventures, and BEVC. For more information, visit montaratx.com.
Media Contact:
Kayla Sadowsky
LMPR Communications
kayla@lmprcommunications.com
