Our
programs
Science
We are advancing a pipeline of novel small molecule candidates designed to precisely target the root causes of rare diseases while also having applicability in more prevalent indications with related underlying biology.
| Program | Target | Indication(s) |
Disc
Discovery
IND-En
Ind-Enabling
P1a
Phase 1a
P1b/2
Phase 1b/2
P3
Phase 3
|
|---|---|---|---|
| ABS-1230 Oral small molecule |
KCNT1
|
KCNT1-related epilepsy
|
|
|
Other genetic epilepsies
|
|
||
| ABS-0871 Oral small molecule |
TRPV4
|
Charcot-Marie-Tooth 2C (CMT2C)
|
|
|
Overactive bladder
|
|
||
| Program 3 Oral small molecule |
Undisclosed
|
Genetic epilepsy
|
|
|
Other CNS disorders
|
|
*Actio owns full rights to all programs
Our KCNT1-Related Epilepsy Program
KCNT1-related epilepsy is a rare and often fatal pediatric epileptic encephalopathy. Patients with KCNT1-related epilepsy experience frequent treatment-resistant seizures that typically begin in early infancy and are accompanied by profound developmental delays and neurological impairments. General antiepileptic drugs have limited benefit in patients with this genetic epilepsy and are often associated with debilitating side effects.
ABS-1230 is designed to be a potent and selective orally available small molecule KCNT1 inhibitor for the treatment of KCNT1-related epilepsy. In preclinical studies, ABS-1230 inhibited all tested pathogenic mutations in the KCNT1 gene, indicating potential to treat all patients with KCNT1-related epilepsy. ABS-1230 has the potential to provide patients with the convenience of an at-home, orally administered therapy. ABS-1230 has FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations and has been accepted into the Rare Disease Evidence Principles process.
Our CMT2C Program
Charcot-Marie-Tooth disease type 2C (CMT2C) is a rare set of inherited neuromuscular disorders caused by gain-of-function mutations in the TRPV4 gene, affecting an estimated 2,500 people in the U.S. The disease damages peripheral nerves and leads to progressive motor and sensory impairments, including difficulty walking, speaking, breathing, and swallowing. While progression varies, CMT2C can significantly impact daily function and quality of life, with symptoms worsening over time and, in some cases, beginning in childhood.
There are currently no approved therapies for CMT2C, and treatment is limited to supportive care. Many patients experience ongoing muscle weakness, skeletal complications, and vocal and respiratory symptoms that are not addressed by existing interventions. As a result, there remains a substantial unmet need for therapies that can meaningfully alter the course of disease for people living with CMT2C and their families.
ABS-0871
ABS-0871 is designed to be a potent and selective orally available TRPV4 small molecule inhibitor for the treatment of CMT2C. TRPV4 is a calcium-permeable ion channel expressed in vascular endothelial cells and other tissues. Gain-of-function mutations in the TRPV4 gene are linked to diseases including CMT2C, a set of rare inherited neuromuscular disorders. 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. The FDA has granted ABS-0871 Rare Pediatric Disease, Fast Track and Orphan Drug Designations for the treatment of CMT2C.
We have completed a Phase 1a in healthy volunteers to evaluate safety, tolerability, pharmacokinetics and measures of pharmacodynamic activity.
Clinical Trials
ABS-1230
Actio Biosciences is enrolling participants in the KYRON Phase 1b/2 clinical study evaluating the safety, tolerability, and pharmacokinetics of ABS-1230 administered orally or via feeding tube in children aged one month through 21 years with KCNT1-related epilepsy. This study will also assess the effects of ABS-1230 on seizure activity and neurodevelopment outcomes.
For additional information about trial, eligibility and trial locations, please visit ActioTrials.com or clinicaltrials.gov.
