What Montara Therapeutics does

Montara Therapeutics is a preclinical-stage biopharmaceutical company founded in 2023 in San Francisco that is developing the BrainOnly™ platform — a proprietary two-drug combination architecture pairing a brain-penetrant, target-specific Brain-Targeting Chimera (BrainTAC™) with a non-brain-penetrant universal peripheral blocker (MT1110) that saturates FKBP12 binding sites in the periphery. The mechanism prevents off-target peripheral activity of neurological drugs while concentrating their therapeutic effect in the brain, thereby addressing the fundamental CNS drug-development bottleneck of dose-limiting peripheral toxicities. The company's lead program, MTX-E1, combines MT1110 with the FDA-approved mTOR inhibitor everolimus for tuberous sclerosis complex (TSC)-related epilepsy, with an IND filing targeted for late 2026. Pipeline expansion is underway into Parkinson's disease via brain-selective LRRK2 inhibition and mTOR-mediated autophagy, with future applications in Alzheimer's disease, ALS/FTD, multiple sclerosis, brain tumors, and brain metastases.

The company is built on exclusive-licensed research from UCSF Professor Kevan Shokat's lab, published in Nature in 2022. Co-founders Nicholas Hertz (President & CEO) and Kevan Shokat (Chairman of the SAB) previously co-founded Mitokinin, acquired by AbbVie in 2023. Shokat also led the lab that first invented K-Ras G12C inhibitors culminating in sotorasib's FDA approval. Scientific advisors include Nobel Laureate Thomas Südhof and NAS member Martin Kampmann; the Board is chaired by Troy Wilson (Kura Oncology CEO) and includes Andrew Miller (founder of Karuna Therapeutics, acquired by Bristol Myers Squibb for $14 billion).

Montara's business model is preclinical drug development intended to monetize through future FDA-approved drug commercialization or via out-licensing/co-development partnerships with larger pharmaceutical companies. The company has no current product revenue. To date Montara has raised approximately $28 million across two seed rounds (July 2024 and a March 2025 oversubscribed expansion) plus approximately $4.3 million in non-dilutive research grants from The Michael J. Fox Foundation. Strategic partnerships include the TSC Alliance, Psychogenics, BioDuro (manufacturing), and MJFF's LRRK2 Investigative Therapeutics Exchange (LITE) program. Customer segments are therapeutic-indication-defined (TSC patients with epilepsy, Parkinson's patients, Alzheimer's patients, and other CNS disease populations) rather than commercial B2B customers in the near term.

Name

Montara Therapeutics

Legal name

Montara Therapeutics, Inc.

Website

https://montaratx.com

Company type

Private

Founded year

2023

Operating status

Operating

Headcount range

11–50 employees

Short description

Montara Therapeutics is a preclinical-stage biopharmaceutical company developing the BrainOnly™ two-drug platform, which pairs a brain-penetrant BrainTAC™ drug with a non-brain-penetrant peripheral blocker (MT1110) to achieve brain-selective pharmacology and avoid systemic toxicities, with lead program MTX-E1 targeting TSC-related epilepsy and IND filing planned for late 2026.

Ownership category

akta.pro rank

Product category

CNS Therapeutics

NAICS

Analytical Laboratory Instrument Manufacturing (334516)

SIC

Surgical & Medical Instruments & Apparatus (3841)

akta.pro primary industry

Neurology & Psychiatry (CNS) Pharmaceuticals (HLAIAAAF)

Keywords

• CNS drug development
• Brain-selective pharmacology
• Neurological therapeutics
• Preclinical biotechnology
• Small molecule therapeutics

Headquarters

HQ city

San Francisco

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales

Revenue model

1. Drug Development and Commercialization: As a preclinical-stage biopharmaceutical company, Montara is developing CNS drugs through its BrainOnly platform. Revenue would come from drug commercialization upon FDA approval or from partnering/licensing deals with larger pharmaceutical companies for pipeline advancement.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Montara Therapeutics develops the BrainOnly™ platform, a two-drug combination therapy system that pairs brain-penetrant, target-specific drugs (BrainTAC™) with a non-brain-penetrant universal peripheral blocker to achieve brain-selective pharmacology. The lead program MTX-E1 combines MT1110 with everolimus for treatment of TSC-related epilepsy, with IND filing targeted for late 2026. The platform is broadly applicable across CNS indications including Parkinson's, Alzheimer's, epilepsy, multiple sclerosis, and brain tumors, addressing dose-limiting peripheral toxicities that have historically prevented effective CNS drug dosing.

Product overview

Montara Therapeutics is a preclinical-stage biopharmaceutical company developing the BrainOnly™ platform, a two-drug combination therapy system for brain-selective pharmacology. The platform consists of MT1110 (a non-brain-penetrant universal peripheral blocker) and BrainTAC™ molecules (brain-penetrant, target-specific drugs engineered with immunophilin binders). The lead program MTX-E1 combines MT1110 with everolimus for TSC-related epilepsy. The company is also developing LRRK2-targeting BrainTAC molecules for Parkinson's disease. The platform enables treatment of CNS diseases while preventing peripheral side effects that limit existing therapies.

Differentiator

Problem solved

Functional benefit

Brands

• BrainOnly: Proprietary platform designed to prevent peripheral toxicities and concentrate neurological drugs' activity in the brain, enhancing safety, increasing therapeutic index, and improving efficacy. The platform enables two-drug combination therapies comprising a brain-penetrant target-specific drug paired with a non-brain-penetrant universal peripheral blocker.
• MTX-E1
• MT1110
• BrainTAC

Products and services

• BrainOnly™ Platform A proprietary technology platform that enables brain-selective pharmacology by pairing brain-penetrant drugs (BrainTAC™) with peripherally-restricted blockers to prevent peripheral side effects and concentrate neurological drugs' activity in the brain. The platform leverages Brain-Targeting Chimeras (BrainTAC™) to enhance safety, increase therapeutic index, and improve efficacy. Licensed from UCSF based on research from Professor Kevan Shokat's lab.
• MT1110 A non-brain-penetrant universal peripheral blocker development candidate that saturates FKBP12 binding sites in the periphery, preventing FKBP12-dependent drugs from binding their targets outside the brain. Used in combination therapies with brain-penetrant drugs to enable brain-selective pharmacology. Montara's first development candidate announced in December 2025.
• MTX-E1 (Brain-Selective Everolimus) Brain-selective everolimus combination therapy (MT1110-everolimus) being developed first for tuberous sclerosis complex (TSC)-related epilepsy, with IND filing targeted for late 2026. Combines MT1110 peripheral blocker with the approved mTOR inhibitor everolimus to enhance safety profile and therapeutic index. IND targeted for late 2026, with planned expansion into Alzheimer's and Parkinson's disease indications.
• BrainTAC™ (Brain-Targeting Chimeras) Brain-penetrant, target-specific drugs engineered with competing immunophilin binders that can bind to immunophilin presenter proteins in the brain to enable activity only where intended. Used in combination with the universal peripheral blocker MT1110 to achieve brain-selective pharmacology. Applicable to both novel chemical warheads and existing FDA-approved drugs.

Quantifiable outcome

• Preclinical data showed superiority of MT1110-everolimus combination in controlling seizures compared to equivalent dose of everolimus alone in TSC mouse model
• +3 more outcomes

Segments4 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Six partnerships are on record, tiered core and minor.

• The Michael J. Fox Foundation for Parkinson's Research (mTOR Program)coreStrategic or Co-development Partner · 2 June 2026Montara received approximately $1 million non-dilutive grant from MJFF to advance BrainOnly™ therapy targeting mTOR pathway for Parkinson's disease. Project aims to activate autophagy in the brain to clear toxic alpha-synuclein protein while mitigating systemic side effects.
• The Michael J. Fox Foundation for Parkinson's ResearchcoreStrategic or Co-development Partner · 21 May 2025MJFF awarded Montara a $3.3 million non-dilutive research grant to develop BrainOnly™ Parkinson's therapy targeting LRRK2. Montara also joined MJFF's LRRK2 Investigative Therapeutics Exchange (LITE) program supporting development of new LRRK2-targeted therapies and advancing progress toward new LRRK2-relevant clinical biomarkers.
• TSC AlliancecoreStrategic or Co-development PartnerCollaboration with TSC Alliance to advance lead program MTX-E1 for TSC-related epilepsy. TSC Alliance provides extensive research expertise and proprietary in vivo models. Collaboration includes joint presentation of preclinical data at American Epilepsy Society Annual Meeting.
• PsychogenicsminorStrategic or Co-development PartnerCollaboration with TSC Alliance and Psychogenics for preclinical studies on MT1110-everolimus combination, including in vivo model work for TSC epilepsy.
• BioDurocoreImplementation/ SI/ Consulting PartnerMontara partnered with BioDuro, a California-based CRDMO (Contract Research, Development and Manufacturing Organization), to manufacture MT1110 drug substance for clinical development.
• University of California, San Francisco (UCSF)coreTechnology or IntegrationMontara holds an exclusive license from UCSF for the development and commercialization of the BrainOnly platform, based on research from Professor Kevan Shokat's lab published in Nature in 2022. Scientific Co-Founder Thomas Südhof is also affiliated with Stanford.

Scale indicators4 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Denali Therapeutics: Clinical-stage biotech engineering brain-penetrant therapies for neurodegeneration. Closely comparable to Montara in targeting CNS selectivity for hard-to-drug neurological targets and pursuing platform-based pipeline expansion.
• Neumora Therapeutics: Precision neuroscience company developing brain-targeted therapeutics for CNS disorders. Comparable target indications (Parkinson's, Alzheimer's) and similar strategy of building a CNS-focused pipeline with differentiated chemistry approaches.
• Praxis Precision Medicines: Clinical-stage CNS biotech developing precision therapies for neurological disorders including epilepsy. Overlaps directly with Montara's lead MTX-E1 program in TSC-related epilepsy and shares the focus on genetically defined CNS populations.
• Sage Therapeutics: CNS-focused biopharmaceutical company developing novel therapies for neurological disorders. Comparable in clinical development expertise and CNS specialty pharma positioning, though focused on neuroactive steroids.
• Karuna Therapeutics: Pioneering CNS biotech that developed KarXT (now COBENFY, FDA-approved 2024) and was acquired by Bristol Myers Squibb for $14B. Montara's board director Andrew Miller was Karuna's founder, and the company represents a directly relevant comparability benchmark for CNS drug development outcomes.
• Mitokinin: Predecessor company co-founded by Hertz and Shokat (same founders as Montara) developing CNS-penetrant therapeutics for Parkinson's, acquired by AbbVie in 2023. Directly comparable team, scientific approach, and exit pathway for Montara.

Emerging players

• Arvinas: Develops targeted protein degradation therapeutics (PROTAC/PROTAC-like chimeras). Conceptually analogous to Montara's BrainTAC chimeras and peripheral blocker strategy of using bifunctional molecules to achieve tissue-selective pharmacology.
• Annexon Biosciences: Clinical-stage biotech developing therapies for neuroinflammatory and neurodegenerative diseases. Comparable CNS pipeline focus and similar preclinical-to-clinical translation challenges in neurological indications.
• Voyager Therapeutics: Clinical-stage gene therapy company focused on CNS disorders including Parkinson's and Alzheimer's. Comparable target indications and shared challenge of achieving brain-selective activity, though using gene therapy rather than small molecules.

Broad incumbents

• Biogen: Established CNS-focused biopharmaceutical company with extensive portfolio in neurology and neurodegenerative diseases including Alzheimer's and Parkinson's. Provides a relevant benchmark for CNS pipeline scale and serves as a likely strategic partner/acquirer for emerging CNS platforms like Montara.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/montara-therapeutics-inc

Revenue estimate

Valuation estimate

Number of profiles

Profiles13 records

Funding overview

Funding rounds4 records

Investors6 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Montara Therapeutics