AMO Pharma
Clinical-stage biopharmaceutical company focused on developing small-molecule investigational therapies for severe and rare neurogenetic and neuromuscular disorders. The organization advances programs from preclinical models through multi-site Phase 2/3 trials, engages with patient advocacy groups, and works directly with regulators in multiple jurisdictions to design registrational studies.
Industries
Nr. of Employees
small (1-50)
AMO Pharma
Patents
Products
Investigational GSK3β pathway inhibitor for congenital myotonic dystrophy
Oral small-molecule inhibitor targeting GSK3β activity, evaluated in cellular and animal models and advanced through Phase 2 and into a planned Phase 2/3 registrational study for congenital myotonic dystrophy.
Ras-ERK pathway inhibitor for intellectual disability / Phelan-McDermid-related deficits
Small-molecule inhibitor of the Ras-ERK signaling cascade evaluated in preclinical models of intellectual disability and advanced to investigator-led clinical study for Phelan-McDermid syndrome.
Glutamate modulator and new chemical entities for Rett syndrome and breathing disorders
Glutamate-modulating compounds and related NCEs advanced via a development and license agreement to address Rett syndrome and associated breathing abnormalities, identified through external screening efforts.
Formulated HDAC inhibitor for CNS delivery (collaborative program)
Formulation of a histone deacetylase inhibitor designed to enable brain delivery and evaluated for lysosomal storage and other CNS genetic disorders in preclinical studies.
AMO-01
Investigational drug currently in development for the treatment of Phelan-McDermid Syndrome.
AMO-04
Investigational drug currently in development for the treatment of Rett syndrome.
Investigational GSK3β pathway inhibitor for congenital myotonic dystrophy
Oral small-molecule inhibitor targeting GSK3β activity, evaluated in cellular and animal models and advanced through Phase 2 and into a planned Phase 2/3 registrational study for congenital myotonic dystrophy.
Ras-ERK pathway inhibitor for intellectual disability / Phelan-McDermid-related deficits
Small-molecule inhibitor of the Ras-ERK signaling cascade evaluated in preclinical models of intellectual disability and advanced to investigator-led clinical study for Phelan-McDermid syndrome.
Glutamate modulator and new chemical entities for Rett syndrome and breathing disorders
Glutamate-modulating compounds and related NCEs advanced via a development and license agreement to address Rett syndrome and associated breathing abnormalities, identified through external screening efforts.
Formulated HDAC inhibitor for CNS delivery (collaborative program)
Formulation of a histone deacetylase inhibitor designed to enable brain delivery and evaluated for lysosomal storage and other CNS genetic disorders in preclinical studies.
AMO-01
Investigational drug currently in development for the treatment of Phelan-McDermid Syndrome.
AMO-04
Investigational drug currently in development for the treatment of Rett syndrome.
Services
Design and management of preclinical-to-clinical development plans and regulatory interactions to support registrational trials across multiple jurisdictions.
Conduct and oversight of cellular, ex vivo and animal model studies to establish mechanism of action and support clinical translation.
Out-licensing, in-licensing and collaborative development agreements to advance novel small molecules and new chemical entities.
Design and management of preclinical-to-clinical development plans and regulatory interactions to support registrational trials across multiple jurisdictions.
Conduct and oversight of cellular, ex vivo and animal model studies to establish mechanism of action and support clinical translation.
Out-licensing, in-licensing and collaborative development agreements to advance novel small molecules and new chemical entities.
Expertise Areas
- Clinical trial management (rare disease, multi-site)
- Regulatory strategy and agency engagement
- Rare disease and orphan drug development
- CNS and neuromuscular translational research
Key Technologies
- Small-molecule pathway inhibitors
- GSK3β pathway modulation
- Ras-ERK pathway inhibition
- Histone deacetylase inhibitor formulations for CNS delivery
News & Updates
Reported receipt of scientific advice from FDA, MHRA and Health Canada to design a registrational study using hospitalization as a primary endpoint and functional assessments as secondary measures.
Announcements related to initiation, enrollment completion and last patient treated in the pivotal REACH-CDM clinical trial for congenital myotonic dystrophy.
License and collaboration agreements announced with academic and industry partners to advance clinical studies for arrhythmogenic right ventricular cardiomyopathy (ARVC) and Rett syndrome.
Peer-reviewed publication reporting Phase 2 clinical study results for the investigational GSK3β pathway inhibitor in congenital and childhood-onset DM1.
Recognition of orphan/fast-track or innovation passport status and substantive regulatory engagement to support clinical development pathways.
Reported receipt of scientific advice from FDA, MHRA and Health Canada to design a registrational study using hospitalization as a primary endpoint and functional assessments as secondary measures.
Announcements related to initiation, enrollment completion and last patient treated in the pivotal REACH-CDM clinical trial for congenital myotonic dystrophy.
License and collaboration agreements announced with academic and industry partners to advance clinical studies for arrhythmogenic right ventricular cardiomyopathy (ARVC) and Rett syndrome.
Peer-reviewed publication reporting Phase 2 clinical study results for the investigational GSK3β pathway inhibitor in congenital and childhood-onset DM1.
Recognition of orphan/fast-track or innovation passport status and substantive regulatory engagement to support clinical development pathways.