AMO Pharma
AMO Pharma is a privately held emerging biopharmaceutical company dedicated to developing new treatments for serious and debilitating diseases, especially rare or orphan diseases. The company focuses on identifying and advancing promising therapies for patient populations with significant unmet needs, aiming to improve health and quality of life through innovative drug development and clinical research.
Industries
Nr. of Employees
small (1-50)
AMO Pharma
Products
AMO-01
A clinical‑stage Ras‑ERK pathway inhibitor investigated for treatment of Phelan‑McDermid syndrome and other intellectual disability indications; demonstrated rescue of neuronal phenotypes in multiple knockout mouse models.
AMO-02 (tideglusib)
A clinical‑stage GSK3β inhibitor under development for myotonic dystrophy (DM1) that has shown target normalization in models and reduction of pathogenic RNA levels in patient tissue ex vivo.
AMO-04
A glutamate modulator and related new chemical entities developed under a licensing agreement for potential treatment of Rett syndrome and certain breathing disorders.
RND-001 (HDACi formulation)
A brain‑penetrant formulation of a histone deacetylase inhibitor advanced in collaboration to target transcriptional modulation in neurogenetic and lysosomal storage disease models; demonstrated stimulation of gene transcription and efficacy in preclinical Niemann‑Pick type C models.
AMO-01
A clinical‑stage Ras‑ERK pathway inhibitor investigated for treatment of Phelan‑McDermid syndrome and other intellectual disability indications; demonstrated rescue of neuronal phenotypes in multiple knockout mouse models.
AMO-02 (tideglusib)
A clinical‑stage GSK3β inhibitor under development for myotonic dystrophy (DM1) that has shown target normalization in models and reduction of pathogenic RNA levels in patient tissue ex vivo.
AMO-04
A glutamate modulator and related new chemical entities developed under a licensing agreement for potential treatment of Rett syndrome and certain breathing disorders.
RND-001 (HDACi formulation)
A brain‑penetrant formulation of a histone deacetylase inhibitor advanced in collaboration to target transcriptional modulation in neurogenetic and lysosomal storage disease models; demonstrated stimulation of gene transcription and efficacy in preclinical Niemann‑Pick type C models.
Services
Structuring and managing development and license agreements to advance externally sourced therapeutic candidates in rare disease indications.
Structuring and managing development and license agreements to advance externally sourced therapeutic candidates in rare disease indications.
Expertise Areas
- Clinical trial management
- Rare disease drug development
- Translational research and target validation
- Biomarker development and objective outcome assessment
Key Technologies
- Small‑molecule kinase inhibition
- Histone deacetylase inhibition (brain‑penetrant HDACi formulation)
- Transgenic and knockout mouse models
- Cellular and ex vivo tissue assays
News & Updates
Company will conduct Phase 3 trial of AMO-02 (tideglusib) in adults to support a future submission for marketing authorization in Type 1 myotonic dystrophy in both adult and pediatric indications.
Drug Development & Delivery – March 2, 2021
Company will conduct Phase 3 trial of AMO-02 (tideglusib) in adults to support a future submission for marketing authorization in Type 1 myotonic dystrophy in both adult and pediatric indications.
Drug Development & Delivery – March 2, 2021