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RAF-1 Inhibitors

2 drugs
OncologyRare Disease
Target Attractiveness: Attractive (79%)

About RAF-1

RAF-1, also known as c-Raf, is a serine/threonine kinase and a key component of the RAS-RAF-MEK-ERK signaling pathway, which regulates cell growth, proliferation, differentiation, and survival.

Strategic Insights

ℹ️ How we calculate
  • White space opportunity in Hypochondroplasia with only 3 trials.
  • Emerging modalities (Peptide) signal innovation opportunity.
2
Approved Drugs
2
Companies
4
Indications
2
Therapeutic Areas
Broadest Approval
STIVARGA
Bayer
3
approved indications

Top Drugs

STIVARGA
Bayer
3 indications · 2012
VOXZOGO
BIOMARIN PHARM
1 indications · 2021
🏢

The competitive landscape includes two companies with approved drugs: Bayer and BIOMARIN PHARM.

Drug Modality Landscape

Modalities

Small molecule
1
50%
Peptide
1
50%

Routes of Administration

💊 Oral
1
50%
💉 Injection
1
50%
💡

RAF-1 is primarily targeted by small molecule modalities (50% of approved drugs).

The balanced modality landscape suggests opportunities for novel modalities like antibodies or PROTACs.

Oral option available Small molecules only

📈 Modality Evolution

2012 Small molecule (STIVARGA)
2021 Peptide (VOXZOGO)

Small molecules pioneered RAF-1 targeting (2012), with peptides entering more recently (2021).

1 drug pre-2015 1 drug since 2015

Clinical Trials 309 trials

309
Total Trials
99
Active
166
Completed
80%
Completion Rate

Completion by Phase

Phase Total Completed Failed Active Completion
Phase 1 79 53 10 15 84%
Phase 2 150 58 22 69 73%
Phase 3 69 47 8 13 85%
Phase 4 11 8 2 1 80%

Top Sponsors

Vertex Pharmaceuticals Incor... 44 93%
Bayer 34 85%
Fudan University 8
BioMarin Pharmaceutical 7
M.D. Anderson Cancer Center 7 25%
Academic and Community Cance... 5 50%
Asan Medical Center 4 67%
Memorial Sloan Kettering Can... 4 100%

By Modality

Small molecule
290 79%
Peptide
19 100%
Source: ClinicalTrials.gov · Completion rate = completed ÷ (completed + terminated + withdrawn)

Phase 3 Readout Calendar Pro

6 Phase 3 trials testing approved RAF-1 drugs across all sponsors.

Full calendar →
Q2 2026
XL092
Exelixis · Colorectal Cancer
Estimated · aging NCT05425940
Q3 2026
Vosoritide
BioMarin Pharmaceutical · Hypochondroplasia
Estimated · fresh NCT06455059
Q2 2027
JMT101
Shanghai JMT-Bio Inc. · Metastatic Colorectal Cancer (mCRC)
Estimated · aging NCT07134205
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Coverage: trials whose intervention is an approved drug targeting RAF-1. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.

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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
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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 232 clinical trials targeting RAF-1.

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