Acetylcholine receptor Inhibitors
4 drugsAbout Acetylcholine receptor
The Acetylcholine receptor is a neurotransmitter receptor that binds acetylcholine, mediating muscle contraction, nerve impulse transmission, and cognitive function.
Human genetic studies provide strong validation for targeting CHRNA1, with variants linked to congenital myasthenic syndromes (score 0.94) and lethal multiple pterygium syndrome (score 0.82). Loss-of-function variants are associated with increased disease risk, suggesting activation may be beneficial.
Four FDA-approved small molecule drugs target the Acetylcholine receptor, including CUVPOSA, QBREXZA, DICYCLOMINE HYDROCHLORIDE, and SOFDRA, all indicated for 'other' therapeutic areas. These drugs are marketed by four companies including MERZ PHARMS, JOURNEY, Hikma, and BOTANIX SB.
Acetylcholine receptor Genetic Evidence Strong
CHRNA1 has strong genetic support with a maximum score of 0.94 across 31 diseases.
Strong genetic support suggests that CHRNA1 agonists have a higher probability of clinical success.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 100% directional consistency across 3 traits
- • Strong signal in disorder of visual system, genetic, familial or congenital disease, musculoskeletal or connective tissue disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
20 totalGWAS and other genetic studies link CHRNA1 to 31 diseases.
Loss-of-function causes disease; activation may help
Loss-of-function causes disease; activation may help
Understanding these scores
Association Score (0-1): Combines all evidence types (genetic, literature, drugs, animal models). Higher = more evidence linking target to disease. This is a ranking heuristic, not a confidence score.
L2G Score: Open Targets uses a machine learning model (Locus-to-Gene) to predict which gene is causal at each GWAS locus. L2G=0.5 means ~50% probability of being the causal gene. Only associations with L2G > 0.05 are included.
Odds Ratio (OR): From gene burden studies (UK Biobank, AstraZeneca PheWAS). Measures how loss-of-function variants affect disease risk. OR<1 = protective (inhibiting target may help), OR>1 = risk (losing function causes disease).
Beta (β): Effect size for continuous traits. β<0 = protective, β>0 = risk.
Clinical Translation (~1.8x): Based on Nelson et al. 2015: drug targets with genetic evidence have ~2x higher success rates in clinical trials. We estimate: Strong support (score ≥0.7) → ~1.8x, Moderate (0.3-0.7) → ~1.3x, Weak → baseline.
Colocalization (H4): Tests whether a GWAS signal and an eQTL/pQTL signal share the same causal variant. H4 is the posterior probability that both traits are associated AND share a causal variant. H4 > 0.8 = strong evidence that gene expression/protein levels drive disease risk. This links genetic variation → gene expression → disease, supporting the target-disease connection.
Top Acetylcholine receptor Drugs
Four companies have approved drugs targeting the Acetylcholine receptor.
The relatively low number of players suggests moderate barriers to entry in this market.
Acetylcholine receptor Drug Modality Landscape
Modalities
Routes of Administration
Acetylcholine receptor is amenable to small molecule drugs, with oral options available for convenient dosing.
The absence of other modalities suggests a whitespace opportunity for novel biologics targeting CHRNA1.
Acetylcholine receptor Clinical Trials 69 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on Acetylcholine receptor. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 12 | 10 | 2 | 0 | 83% |
| Phase 2 | 13 | 10 | 1 | 2 | 91% |
| Phase 3 | 10 | 9 | 0 | 1 | 100% |
| Phase 4 | 34 | 30 | 4 | 0 | 88% |
Top Sponsors
By Modality
Top Conditions
Acetylcholine receptor Drug Approval Timeline (2003 - 2024)
Irritable Bowel Syndrome
The first drug was approved in 2003, with the most recent approval in 2024.
The recent approval indicates continued interest and potential for further development in this area.
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Competitive Landscape
- • 4 companies competing
- • Market share by company
Full Drug Portfolio
- • All 4 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 4-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 44 (11 companies)
- • Completion rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Count Trials
- Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on Acetylcholine receptor. A trial counts if an approved drug acting on Acetylcholine receptor is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
- Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
- Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
- Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.