CatalystAlert

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Search for companies, drugs, and catalysts

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Search for companies, drugs, and catalysts

All Therapeutic Areas
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Rare & Genetic Diseases

58 drugs in pipeline from 10 companies

58

Drugs in Pipeline

10

Companies

9

Upcoming Catalysts

58

Phase 3 Trials

Upcoming Catalysts
Today
INCYPhase 2

pemigatinib Phase 2 Results Expected

Pemigatinib206
Apr 14
CT.gov
00%
Tomorrow
KPTIPhase 2

Selinexor 40 mg Phase 2 Results Expected

Selinexor 100 mg118
Apr 15
CT.gov
00%
Tomorrow
NTLAPhase 3

NTLA-2002 Phase 3 Results Expected

NTLA-200260
Apr 15
CT.gov
00%
3d
days
REGNPhase 2

Carboplatin Phase 2 Results Expected

Carboplatin195
Apr 17
CT.gov
00%
6d
MRKPhase 2

lenvatinib Phase 2 Results Expected

Lenvatinib315
Apr 20
CT.gov
00%
6d
MRKPhase 2

pembrolizumab Phase 2 Results Expected

pembrolizumab315
Apr 20
CT.gov
00%
2w
INSMPDUFA
Phase 2Approved

FDA PDUFA Date Arikayce (priority)

Arikayce™
Apr 28
SEC
3w
VRTXPhase 3

IVA Phase 3 Results Expected

IVA71
Apr 30
CT.gov
00%
3w
VRTXPhase 3
Positive

ELX/TEZ/IVA Phase 3 Results Expected

ELX/TEZ/IVA71
Apr 30
CT.gov
Pipeline by Phase
Phase 3
58
Related Conditions
rare diseaseorphangeneticgene therapyhereditarymuscular dystrophycystic fibrosissickle cellhemophiliathalassemiagaucherfabrypompehunter syndromephenylketonuriapkusmaspinal muscular atrophy