ACVR1 Inhibitors
2 drugsAbout ACVR1
ACVR1, or Activin A Receptor Type 1, is a protein kinase receptor involved in signaling pathways crucial for cell growth and differentiation as part of the TGF-β superfamily. It regulates cell development and its dysregulation can contribute to disease.
ACVR1 is a therapeutic target, though no genetic evidence currently links it directly to specific diseases. The existence of two FDA-approved drugs targeting ACVR1 underscores its importance in drug development.
ACVR1 is targeted by two FDA-approved small molecule drugs: VONJO (SOBI) and OJJAARA (GSK). These drugs are approved for 'other' therapeutic areas, indicating potential for expansion into new indications.
Strategic Insights
ℹ️ How we calculate- White space opportunity in Neoplasms with only 1 trials.
- phase2 represents biological uncertainty with 30% completion.
Top Drugs
The ACVR1 competitive landscape includes SOBI and GSK, each with an approved drug.
The limited number of companies suggests relatively low competition, potentially easing market entry for new ACVR1-targeting therapies.
Drug Modality Landscape
Modalities
Routes of Administration
ACVR1 is amenable to small molecule drugs, with oral options available for convenient dosing.
The exclusive use of small molecules presents an opportunity to explore alternative modalities like antibodies or biologics.
Clinical Trials 86 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 39 | 21 | 4 | 14 | 84% |
| Phase 2 | 30 | 7 | 9 | 14 | 44% |
| Phase 3 | 12 | 7 | 3 | 2 | 70% |
| Phase 4 | 5 | 3 | 1 | 1 | 75% |
Top Sponsors
By Modality
Top Conditions
Drug Approval Timeline (2022 - 2023)
The first ACVR1-targeting drug was approved in 2022, with the most recent approval in 2023.
Recent approvals indicate growing interest, but a short approval span suggests the field is still nascent with room for innovation.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 2 companies competing
- • Market share by company
Full Drug Portfolio
- • All 2 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 2-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Competition: High (15 sponsors)
- • White space: 10 underexplored indications
- • Success rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Calculate These Metrics
Target Attractiveness Score
A 0-100 score based on trial activity, sponsor diversity, and completion rates. Calculated from 62 clinical trials targeting ACVR1.
Completion rate: Percentage of trials that reached their planned endpoint. Trials terminated early, withdrawn, or suspended are not counted—these often indicate safety issues, lack of efficacy, or strategic pivots.
- Highly Attractive (80+): High trial activity, many sponsors, strong completion rates
- Attractive (60-79): Good trial activity and validation
- Moderate (40-59): Moderate interest from sponsors
- Low (under 40): Limited trial activity or validation concerns
Strategic Insights
Auto-generated insights based on trial analytics including competition intensity, white space opportunities, modality shifts, and failure patterns. We analyze trial sponsors, phases, indications, and outcomes.
Risk Signals
- High Competition: Many sponsors competing for this target (may reduce market opportunity)
- High Failure Risk: Low trial completion rates suggest development challenges
- Low Validation: Limited trial activity or poor outcomes indicate uncertain viability
- White Space Available: Underexplored indications present opportunities