AMO Pharma

What AMO Pharma does

AMO Pharma Limited is a privately held, clinical-stage biopharmaceutical company founded in 2015 and domiciled in Leeds, United Kingdom, with an additional operational office in Essex Fells, New Jersey. The company develops small-molecule therapeutics for serious rare genetic and neuromuscular disorders where no approved disease-modifying therapies exist, operating a portfolio of three investigational assets: AMO-02 (tideglusib), a glycogen synthase kinase 3 beta (GSK3β) inhibitor with a dual mechanism that both disrupts the pathogenic RNA repeat and reduces excess GSK3β activity, being developed primarily for congenital myotonic dystrophy type 1 (CDM1/Steinert disease); AMO-01, a Ras-ERK pathway inhibitor in clinical evaluation for Phelan-McDermid Syndrome; and AMO-04, a glutamate modulator licensed from Numedicus Limited and being developed for Rett syndrome and certain breathing disorders.

The company's pipeline is supported by a multi-jurisdiction regulatory strategy that has yielded FDA Fast Track, FDA Orphan Drug, FDA Rare Pediatric Disease, and UK MHRA Innovation Passport designations, alongside a coordinated clinical operations footprint spanning investigator sites in the United States, Canada, Australia, and New Zealand. The pivotal REACH-CDM Phase 2/3 trial for AMO-02 enrolled 56 patients and reported statistically and clinically significant efficacy data in September 2023, with a long-term open-label extension (REACHCDM-X) demonstrating favorable safety over four years across 151+ patient-years of exposure.

AMO Pharma is pre-revenue and has not commercialized any products. Funding has come from private equity investors, most notably a $25 million round led by Woodford Patient Capital Trust in September 2015 and a $35 million raise in January 2020 to support the REACH-CDM pivotal trial, with undisclosed follow-on private equity investments in 2022. The company's go-to-market relies on patient advocacy partnerships (Myotonic Dystrophy Foundation, Phelan-McDermid Syndrome Foundation, FRAXA, Rettsyndrome.org), specialist clinical trial sites, and planned specialty pharmaceutical distribution upon regulatory approval. Leadership comprises a small executive team (CEO/CSO Michael Snape, CFO Martyn Williams, Chairman Alan L. Rubino) with prior public-company and large-pharma commercialization experience.

Name

AMO Pharma

Legal name

AMO Pharma Limited

Website

https://amo-pharma.com

Company type

Private

Founded year

2015

Operating status

Operating

Headcount range

1–10 employees

Short description

AMO Pharma is a privately held, UK-based clinical-stage biopharmaceutical company developing small-molecule therapeutics (AMO-01, AMO-02/tideglusib, AMO-04) for rare genetic and neuromuscular disorders including congenital myotonic dystrophy, Phelan-McDermid syndrome, and Rett syndrome.

Ownership category

akta.pro rank

Headquarters

HQ city

Wonersh

HQ country

United Kingdom

HQ region

Europe

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations

Revenue model

1. Private Equity Financing: AMO Pharma raised $25 million in private equity financing with Woodford Patient Capital Trust in 2015, and $35 million fund raise in January 2020. The company is privately held and has not yet commercialized any products.
2. Future Product Commercialization: Pre-revenue clinical stage company. Expected to generate revenue through commercialization of investigational drugs for rare diseases upon regulatory approval.

Go-to-market motion2 records

Distribution channels1 record

Marketing channels4 records

Core offering

AMO Pharma is a privately held clinical-stage biopharmaceutical company developing small-molecule investigational drugs for serious rare genetic and neuromuscular disorders. Its pipeline includes AMO-01, AMO-02 (tideglusib), and AMO-04 targeting conditions such as congenital myotonic dystrophy, Phelan-McDermid syndrome, Rett syndrome, and arrhythmogenic cardiomyopathy.

Product overview

AMO Pharma is a clinical-stage biopharmaceutical company developing a portfolio of three investigational drug candidates for rare genetic disorders: AMO-01 for Phelan-McDermid Syndrome, AMO-02 (tideglusib) for congenital myotonic dystrophy type 1, and AMO-04 for Rett syndrome. These are distinct clinical-stage medicines at various phases of development targeting serious rare diseases with limited treatment options.

Differentiator

Problem solved

Functional benefit

Products and services

• AMO-01 An inhibitor of the Ras-ERK pathway being developed for the treatment of Phelan-McDermid Syndrome and intellectual disabilities. In pre-clinical studies it rescued neuronal phenotypes in multiple knockout mouse models of intellectual disability.
• AMO-02 (tideglusib) A glycogen synthase kinase 3 beta (GSK3β) inhibitor in clinical development for congenital myotonic dystrophy type 1 (DM1/Steinert disease), with a dual mechanism disrupting the pathogenic RNA repeat and inhibiting excess GSK3β levels; also being developed for arrhythmogenic cardiomyopathy and additional CNS and neuromuscular indications.
• AMO-04 A glutamate modulator being developed for the treatment of Rett syndrome and certain breathing disorders, developed under a license agreement with Numedicus Limited.

Quantifiable outcome

• Phase 2 proof-of-concept study: AMO-02 provided clinical benefit to majority of subjects after 12 weeks of treatment with improvements in cognitive functioning, fatigue, and ability to perform activities of daily living
• +2 more outcomes

Named customers3 records

Segments4 records

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Six partnerships are on record, tiered core and minor.

• Numedicus LimitedcoreStrategic or Co-development Partner · 20 July 2017Development and license agreement to advance development of AMO-04, a glutamate modulator and related New Chemical Entities for treatment of Rett syndrome and certain breathing disorders. AMO-04 was identified through Numedicus collaborators' research and the Scout Program sponsored by Rettsyndrome.org.
• Ranedis PharmaceuticalsminorStrategic or Co-development Partner · 14 March 2017Collaboration agreement for development of RND-001, an HDAC inhibitor for rare genetic diseases.
• Icahn School of Medicine at Mount SinaicoreStrategic or Co-development PartnerClinical study of AMO-01 for treatment of Phelan-McDermid syndrome in patients aged 12 to 45 years who also have epilepsy. Study supported by AMO Pharma.
• Population Health Research Institute (PHRI)coreStrategic or Co-development PartnerCollaboration with PHRI (joint institute of McMaster University and Hamilton Health Sciences in Canada) to advance TaRGET Phase 2 proof-of-concept clinical trial evaluating AMO-02 for arrhythmogenic cardiomyopathy. License agreement with PHRI and Venca Research Inc. to support development and potential manufacturing and commercialization of AMO-02 in ARVC.
• Venca Research Inc.minorStrategic or Co-development PartnerPartner in license agreement with PHRI for development and potential manufacturing and commercialization of AMO-02 in arrhythmogenic right ventricular cardiomyopathy (ARVC).
• Rettsyndrome.orgminorOthersSponsor of Scout Program that conducted extensive screening identifying AMO-04's significant promise as potential treatment for Rett syndrome.

Scale indicators6 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• BridgeBio Pharma: Genetic disease-focused biopharma with multiple programs targeting rare Mendelian and cardiac conditions. Comparable pipeline breadth across rare genetic disorders, including cardiovascular (similar to AMO's ARVC expansion).
• PTC Therapeutics: Specialty biopharma developing treatments for rare genetic disorders including Duchenne muscular dystrophy and DM1 (through its PTC-IND program). Closely comparable in mechanism (RNA/splicing modulation) and orphan disease focus.
• Soleno Therapeutics: Clinical-stage rare disease company developing therapeutics for rare genetic conditions (notably Prader-Willi syndrome). Closely comparable in clinical-stage profile, orphan designation strategy, and small-cap rare disease positioning.
• Sarepta Therapeutics: Commercial-stage rare disease biopharma focused on neuromuscular and genetic disorders (notably Duchenne muscular dystrophy). Directly comparable to AMO Pharma in targeting rare neuromuscular diseases with limited or no approved therapies and leveraging accelerated regulatory pathways.
• Ultragenyx Pharmaceutical: Clinical-to-commercial rare disease biotech developing therapeutics for serious rare and ultra-rare genetic diseases. Directly comparable business model of repurposing or developing novel therapies for orphan indications with high unmet need.

Emerging players

• Ionis Pharmaceuticals: RNA-targeted therapeutics company with significant rare neurological disease programs (including DM1). Comparable in novel-mechanism rare disease drug development and dual-mechanism approaches similar to AMO-02.
• Anaveon: Clinical-stage biotech focused on cytokine signaling for oncology and rare diseases. Comparable as a small European clinical-stage biopharma pursuing novel mechanism therapies with limited approved alternatives.
• Taysha Gene Therapies: Clinical-stage gene therapy company focused on rare monogenic CNS diseases. Comparable in targeting ultra-rare genetic CNS disorders with limited treatment options and leveraging orphan/rare pediatric regulatory pathways.

Broad incumbents

• BioMarin Pharmaceutical: Established rare disease biopharma with a broad portfolio across multiple ultra-rare genetic conditions. Comparable in commercial-stage orphan drug development, regulatory strategy (Orphan Drug Designations), and pediatric rare disease focus.
• Alexion Pharmaceuticals (AstraZeneca Rare Disease): Global leader in rare disease therapeutics (now part of AstraZeneca). Comparable as the benchmark orphan disease commercial model, demonstrating how ultra-rare disease assets can scale into multi-billion-dollar franchises through focused commercialization.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/amo-pharma-ltd

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds2 records

Investors2 records

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M&A

Investments

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

Frequently asked questions about AMO Pharma