VivoSeq
Metabolic & Endocrine

Cystinosis

11

Pipeline Programs↗

5

Active Trials↗

8

Companies Involved↗

Cystinosis is an emerging landscape with 11 programs across 8 companies. The most active players are Eloxx Pharmaceuticals, Inc., RECORDATI GROUP, Amgen. 2 therapies are approved with 2 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

40/100

Pipeline Velocity

6/100

Mech. Concentration

N/A

Completion Rate

82/100

Attrition Rate

13/100

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.

E

Eloxx Pharmaceuticals, Inc.

1 program

Phase 2R

RECORDATI GROUP

1 program

Phase 3Amgen logo

Amgen

2 programs

Phase 3A

Arbor Pharmaceuticals, Inc.

1 program

ApprovedN

Nacuity Pharmaceuticals, Inc.

1 program

PreclinicalH

Horizon Therapeutics Ireland DAC

1 program

Preclinical

Pipeline

Programs by Phase

Analysis

Trial Status

16

clinical trials

Completed9 · 56%
Recruiting2 · 13%
Enrolling by invitation1 · 6%
Terminated1 · 6%
Withdrawn1 · 6%
Not yet recruiting1 · 6%
Other1 · 6%

Clinical Trials

Trial Activity

16 trials

European Cystinosis Cohort
Recruiting·IInstitut National de la Santé Et de la Recherche Médicale, France·N/A·NCT05901077·400 enrolled
Use of Cysteamine in the Treatment of Cystinosis
Recruiting·NNational Human Genome Research Institute (NHGRI)·N/A·NCT00359684·330 enrolled
A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
Active, not recruiting·NNacuity Pharmaceuticals, Inc.·Preclinical·NCT05994534·12 enrolled
European Cystinosis Cohort 2
Not yet recruiting·IInstitut National de la Santé Et de la Recherche Médicale, France·N/A·NCT07680751·250 enrolled
Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients
Enrolling by invitation·CChiesi SA/NV·N/A·NCT04246060·31 enrolled
New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis
Completed·National Eye Institute (NEI)·Phase 1·NCT00001736·51 enrolled
Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)
Completed·RRECORDATI GROUP·Phase 3·NCT04125927·5 enrolled
Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis
Completed·Amgen logoAmgen·Phase 3·NCT01733316·41 enrolled
Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
Completed·Amgen logoAmgen·Phase 3·NCT01000961·43 enrolled
Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis
Completed·FFDA Office of Orphan Products Development·N/A·NCT00010426·30 enrolled
Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
Completed·Amgen logoAmgen·Phase 3·NCT01744782·17 enrolled
Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis
Completed·National Eye Institute (NEI)·Phase 2·NCT00001213·328 enrolled
N Acetyl Cysteine for Cystinosis Patients
Completed·UUniversity of Sao Paulo·Phase 4·NCT01614431·23 enrolled
Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis
Completed·Amgen logoAmgen·Phase 1/2·NCT00872729·9 enrolled
A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis
Terminated·EEloxx Pharmaceuticals, Inc.·Phase 2·NCT04069260·3 enrolled
Page 1 of 2

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