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Our
pipeline

Science

Bringing Meaningful Therapies from One to Many

We are advancing a pipeline of novel therapeutics initially focused on addressing rare diseases with significant unmet medical need where a new treatment will have a transformational impact on patient lives. Ultimately, we plan to apply learnings gathered through both the preclinical and clinical development processes to inform potential relevance of potential targets of interest in more common diseases

Program
Target
Indication(s)
Discovery
Lead Candidate
Preclinical
Phase 1
  • Program: ABS-0871
    Target: TRPV4
    Indications:

    Charcot Marie Tooth disease 2C (CMT2C)

    Overactive bladder disease

  • Program: ABS-1230
    Target: KCNT1
    Indications:

    KCNT1-related epilepsy

    Additional rare genetic epilepsies

• Early feasibility work on multiple opportunities that match the Actio paradigm for other genetic epilepsies and dermatological indications ongoing.

Our CMT2C Program

Charcot Marie Tooth (CMT) disease is a hereditary motor sensory neuropathy causing muscle weakness, loss of sensation, contractures and associated symptoms and disability.​ CMT Type 2C (CMT2C) is a severe, genetically defined, phenotypically distinct subtype of CMT, characterized by vocal cord paralysis, respiratory complications, bladder urgency and incontinence, and disruption of the blood-neural barrier.

There are no approved treatments for CMT2C. Current management includes genetic counseling, physical and occupational therapy, devices to improve mobility, and mental health care to maintain quality of life and preserve functionality in CMT2C patients.

ABS-0871

Actio is advancing ABS-0871, a once-daily oral TRPV4 small molecule inhibitor for the treatment of CMT2C. TRPV4 is a calcium ion channel that regulates the concentration of calcium inside cells, which is important for many cellular processes. Mutations in the TRPV4 gene cause cellular toxicity and increased activated ion channel currents, which leads to TRPV4-positive CMT2C disease as early as infancy. ABS-0871 is a potential first-in-class TRPV4 small molecule inhibitor designed to inhibit overactivated ion channel activity and restore vocal cord, respiratory, bladder and other functions.

Our KCNT1-Related Epilepsy Program


KCNT1-related epilepsy is a rare genetic condition that causes seizures due to mutations in the KCNT1 gene, accounting for an estimated 30% of all epilepsies. General antiepileptic drugs have limited benefit in patients with genetic epilepsy, and seizure management is often synonymous with lifelong pharmacological treatment, with side effects that can be debilitating, and the risk of increasing refractoriness over time. New personalized medicines are needed that target the underlying genetic component of epilepsy where there are no approved or effective therapies available.


ABS-1230

Actio is developing an oral, first-in-class small molecule inhibitor, ABS-1230, for the treatment of KCNT1-related epilepsy. KCNT1 is a potassium ion channel primarily expressed in the brain that, when functioning normally, regulates the flow of electricity to the brain. Mutations in the KCNT1 gene cause overactivation of the potassium channel and abnormal electrical brain activity, leading to a severe, early-onset epileptic encephalopathy and a high frequency of daily seizures. ABS-1230 is designed to inhibit the overactive ion channel and reduce seizure frequency.