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Table 1 Targeted therapies of Phase 1 and Phase 2 studies in SSc patients

From: An update on targeted therapies in systemic sclerosis based on a systematic review from the last 3 years

Trial drug Target Inclusion criteria Treatment IS Endpoints Duration Results
Inebilizumab B cells (CD19) Localized mRSS ≥ 2 Single dose 0.1–10 mg/kg Yes Safety
Tolerability
12 weeks Safe and well-tolerated
Dabigatran Thrombin SSc-ILD
 HRCT ≥ 20%
 FVC < 70%
Early (≤ 10 years)
75 mg twice a day Yes Safety
Tolerability
6 months Safe and well-tolerated
C-82 β-Catenin Signaling Early (median 8 months)
dcSSc (mRSS ≥ 12)
Localized mRSS ≥ 2
Topical formulation Yes AE
Gene biomarkers
4 weeks Well-tolerated
Weak genes downregulation
Pomalidomide Angiogenesis immunosuppression SSc-ILD
 FVC > 45 > 70
 FVC > 70, recent loss of 5%
 HRCT > 20%
Early (< 7 years)
1 mg/day No %pFVC
mRSS
SCTC GIT 2.0
52 weeks Negative
Rilonacept IL-1 Early (< 24 months)
dcSSc (mRSS ≥ 15)
320 mg sc loading dose
160 mg sc weekly
No Change in expression in 2G SSc genes
mRSS
6 weeks Negative
Romilkimab IL-4 and IL-13 Early (≤ 36 months) dcSSc (mRSS ≥ 10) 200 mg sc weekly Yes mRSS
FVC/DLCO
HAQ-DI
24 weeks mRSS difference − 2.31 (p = 0.029) in favour of Romilkimab
Tocilizumab IL-6 Early (< 5 years)
dcSSc (mRSS ≥ 10)
162 mg sc weekly No mRSS
FVC
48 weeks
(primary outcome at 24 weeks)
mRSS change favoured TCZ (p = 0.058)
Smaller decrease in FVC in TCZ
Tofacitinib JAK1 and 3 Early (< 5 years)
dcSSc (mRSS ≥ 10)
5 mg twice a day Yes Grade ≥ 3 AE
mRSS
HAQ-DI
CRISS
24 weeks No Grade 3 AE
Improvement trend
Pirfenidone Myofibroblast
TGF-β
STAT-3
SSc-ILD
 FVC ≥ 50%
 DLCO ≥ 40%
Early (< 7 years)
2- or 4-week titration
801 mg daily to 2403 mg daily
Yes AE
FVC and DLCO
PRO
16 weeks 4-week titration better tolerated
No change
Lenabasum Cannabinoid receptor 2 Early (< 3 years or > 3 years and < 6 years with CRP > 3)
dcSSc (ΔmRSS ≥ 5 last 6 months, total mRSS ≥ 12)
5 mg/day, 20 mg/day or 20 mg twice a day for 4 weeks and then 20 mg twice a day for 8 weeks. Yes CRISS 16 weeks Improvement
(p = 0.044) in
mRSS
PRO
PGA
HAQ-DI
Abatacept B/T cells interaction
(CD80/CD86)
Early dcSSc
(≤ 18 months, mRSS ≥ 10;
> 18 and ≤ 36 months, mRSS ≥ 15)
125 mg sc weekly No mRSS
Safety
12 months Negative
Good safety profile
Belimumab BLys Early (≤ 3 years)
dcSSc (mRSS >  15)
recently started on MMF (2 g)
10 mg/kg iv 2-weekly for the first three doses and then 4-weekly Yes mRSS
Safety
Tolerability
52 weeks No significant mRSS change
Safe and well-tolerated
Riociguat Guanylate Cyclase Early (≤ 18 months)
dcSSc (mRSS ≥ 10)
0.5 mg (up-titrated to a maximum dose of 2.5 mg three times a day) No mRSS
CRISS
HAQ-DI
FVC
52 weeks Negative Reduced mRSS progression in Riociguat
SAR100842 Lysophosphatidic acid receptor 1 Early (≤ 36 months)
dcSSc (mRSS ≥ 15)
300 mg twice a day Yes Safety
Tolerability
mRSS
24 weeks Safe and well-tolerated
No significant change in mRSS
Lanifibranor PPAR Early (≤ 3 years)
dcSSc (mRSS ≥ 10)
400 mg twice a day
600 mg twice a day
Yes mRSS
FVC and DLCO
CRISS and PRO
48 weeks No significant change in mRSS
  1. IS immunosuppressive treatment, mRSS modified Rodnan skin score, SSc systemic sclerosis, ILD interstitial lung disease, HRCT high-resolution computed tomography, FVC forced vital capacity, AE adverse events, SCTC GIT Scleroderma Clinical Trials Consortium Gastrointestinal Tract, DLCO diffusing lung capacity for carbon monoxide, HAQ-DI Health Assessment Questionnaire – Disability Index, CRISS Combined Response Index in Systemic Sclerosis, PRO patient-reported outcome