VivoSeq
Ophthalmology

Stargardt Disease

2

Pipeline Programs↗

6

Active Trials↗

6

Companies Involved↗

Stargardt Disease is an early-stage landscape with 2 programs across 6 companies. The most active players are Alkeus Pharmaceuticals, Inc., Eli Lilly, Stargazer Pharmaceuticals, Inc.. 1 program is in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

27/100

Pipeline Velocity

15/100

Mech. Concentration

N/A

Completion Rate

100/100

Attrition Rate

0/100

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

Alkeus Pharmaceuticals, Inc.

1 program

Phase 3Eli Lilly logo

Eli Lilly

1 program

Phase 3S

Stargazer Pharmaceuticals, Inc.

0 programs

PreclinicalN

Nanoscope Therapeutics Inc.

0 programs

PreclinicalV

VeonGen Therapeutics GmbH

0 programs

PreclinicalAstellas logo

Astellas

0 programs

Preclinical

Pipeline

Programs by Phase

Preclinical1

AAVB-039

AAVantgarde Bio Srl

Phase 1
No programs
Phase 2
No programs
Phase 31

ALK-001

Alkeus Pharmaceuticals, Inc.

Approved
No programs

Analysis

Modality Breakdown

2programs
Small molecule150%
Gene therapy150%

Modality classified for 67% of 3 pipeline programs.

Analysis

Trial Status

9

clinical trials

Recruiting4 · 44%
Completed3 · 33%
Enrolling by invitation1 · 11%
Active, not recruiting1 · 11%

Clinical Trials

Trial Activity

9 trials

Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)
Recruiting·VVeonGen Therapeutics GmbH·Phase 1/2·NCT07002398·15 enrolled
A Phase 3 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, and Efficacy of Gildeuretinol Acetate (ALK 001) on the Progression of Stargardt Disease (STGD)
Recruiting·AAlkeus Pharmaceuticals, Inc.·Phase 3·NCT07419334·230 enrolled
An Observational Study of Natural Disease Progression in Participants With Macular Dystrophies Expressing a Stargardt-type Clinical Presentation
Recruiting·Astellas logoAstellas·N/A·NCT07425574·90 enrolled
An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants With Stargardt Disease (STGD1) (CELESTE)
Recruiting·AAAVantgarde Bio Srl·Preclinical·NCT07161544·75 enrolled
Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)
Active, not recruiting·AAlkeus Pharmaceuticals, Inc.·Phase 2·NCT04239625·200 enrolled
Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease
Enrolling by invitation·AAlkeus Pharmaceuticals, Inc.·Phase 2·NCT02402660·160 enrolled
Study of STG-001 in Subjects With Stargardt Disease
Completed·SStargazer Pharmaceuticals, Inc.·Phase 2·NCT04489511·10 enrolled
Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease
Completed·NNanoscope Therapeutics Inc.·Phase 2·NCT05417126·6 enrolled
This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease
Completed·RRBP4 Pty Ltd·Phase 1/2·NCT05266014·13 enrolled

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