VivoSeq
Rare Disease

Cryopyrin-Associated Periodic Syndrome

9

Pipeline Programs↗

1

Active Trials↗

7

Companies Involved↗

Cryopyrin-Associated Periodic Syndrome is an early-stage landscape with 9 programs across 7 companies. The most active players are Akros Pharma Inc., Aclaris Therapeutics, Inc., Zomagen Biosciences Ltd.. 1 program is in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

35/100

Pipeline Velocity

6/100

Mech. Concentration

67/100

Completion Rate

88/100

Attrition Rate

10/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

A

Akros Pharma Inc.

1 program

Phase 1A

Aclaris Therapeutics, Inc.

1 program

Phase 2Z

Zomagen Biosciences Ltd.

1 program

Phase 2N

Nuon Therapeutics, Inc.

1 program

Phase 2Novartis logo

Novartis

2 programs

Phase 3Regeneron logo

Regeneron

1 program

Preclinical

Pipeline

Programs by Phase

Analysis

Modality Breakdown

75%

Small molecule

6 of 8 classified programs — the dominant modality here.

25%ALL OTHERSMonoclonal antibody113%Protein / enzyme113%

Small molecule leads; the ring shows all 8 classified programs, each share of the total. Modality classified for 89% of 9 pipeline programs.

Analysis

Trial Status

10

clinical trials

Completed7 · 70%
Terminated1 · 10%
Unknown status1 · 10%
Recruiting1 · 10%

Clinical Trials

Trial Activity

10 trials

Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of JTE-162 in Subjects With Cryopyrin-Associated Periodic Syndrome (CAPS)
Recruiting·AAkros Pharma Inc.·Phase 1·NCT07247266·5 enrolled
Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)
Completed·Regeneron logoRegeneron·Phase 3·NCT00288704·104 enrolled
A Study to Evaluate VTX2735 in Patients With Cryopyrin-associated Periodic Syndrome
Completed·ZZomagen Biosciences Ltd.·Phase 2·NCT05812781·7 enrolled
Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
Completed·Novartis logoNovartis·Phase 3·NCT01302860·17 enrolled
Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
Completed·Novartis logoNovartis·Phase 3·NCT01576367·17 enrolled
The Safety and Efficacy of Canakinumab in Patients Aged 4 Years or Older Diagnosed With Cryopyrin-associated Periodic Syndromes (CAPS) in Canada
Completed·Novartis logoNovartis·Phase 3·NCT01105507·4 enrolled
Efficacy and Safety Study of Canakinumab Administered for 6 Months (24 Weeks) in Japanese Patients With Cryopyrin-associated Periodic Syndromes Followed by an Extension Phase
Completed·Novartis logoNovartis·Phase 3·NCT00991146·19 enrolled
Efficacy and Safety of ACZ885 in Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
Completed·Novartis logoNovartis·Phase 3·NCT00685373·166 enrolled
Study of ATI-450 in Patients With Cryopyrin-Associated Periodic Syndrome (CAPS)
Terminated·AAclaris Therapeutics, Inc.·Phase 2·NCT04524858·1 enrolled
A Clinical Study of Tranilast in the Treatment of Cryopyrin-Associated Periodic Syndrome (CAPS)
Unknown status·PPeking Union Medical College Hospital·Phase 2·NCT03923140·71 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)