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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 RAF-1 Drugs

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

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

RAF-1 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

RAF-1 Clinical Trials 231 trials

231
Total Trials
88
Active
102
Completed
72%
Completion Rate

Completion by Phase

Phase Total Completed Failed Active Completion
Phase 1 64 39 10 14 80%
Phase 2 134 47 22 64 68%
Phase 3 29 13 6 9 68%
Phase 4 4 3 1 0 75%

Top Sponsors

Bayer 34 85%
BioMarin Pharmaceutical 7
M.D. Anderson Cancer Center 7 25%
Fudan University 6
Asan Medical Center 4 67%
Memorial Sloan Kettering Can... 4 100%
City of Hope Medical Center 4 50%
Academic and Community Cance... 4 50%

By Modality

Small molecule
221 72%
Peptide
10
Source: ClinicalTrials.gov · Completion rate = completed ÷ (completed + terminated + withdrawn)

Phase 3 Readout Calendar Pro

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

Full calendar →
Q3 2026
Vosoritide
BioMarin Pharmaceutical · Hypochondroplasia
Estimated · fresh NCT06455059
Q2 2027
JMT101
Shanghai JMT-Bio Inc. · Metastatic Colorectal Cancer (mCRC)
Estimated · aging NCT07134205
Q3 2028
NB003
Ningbo Newbay Technology Development Co., Ltd · GIST - Gastrointestinal Stromal Tumor
Estimated · fresh NCT07379047
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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