VivoSeq
Other

Phelan-Mcdermid Syndrome

6

Pipeline Programs↗

3

Active Trials↗

5

Companies Involved↗

Phelan-McDermid syndrome is an early-stage landscape with 6 programs across 5 companies. The most active players are Retrophin, EMD Serono, Pfizer. 1 program is in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

32/100

Pipeline Velocity

11/100

Mech. Concentration

50/100

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.

R

Retrophin

1 program

Phase 2EMD Serono logo

EMD Serono

1 program

Phase 2Pfizer logo

Pfizer

1 program

Phase 2N

Neuren Pharmaceuticals Limited

1 program

Phase 3Eli Lilly logo

Eli Lilly

1 program

Phase 3

Pipeline

Programs by Phase

Analysis

Trial Status

8

clinical trials

Completed5 · 63%
Recruiting2 · 25%
Active, not recruiting1 · 13%

Clinical Trials

Trial Activity

8 trials

A Phase 3 Open-label Extension Study to Investigate the Long-term Safety and Efficacy of Orally Administered NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Recruiting·NNeuren Pharmaceuticals Limited·Phase 3·NCT07593391·180 enrolled
A Phase 3 Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Orally Administered NNZ-2591 Compared With Placebo in Pediatric Participants With Phelan-McDermid Syndrome
Recruiting·NNeuren Pharmaceuticals Limited·Phase 3·NCT07281079·160 enrolled
An Open-label, Single Arm, Dose-Escalation Clinical Study to Evaluating the Safety, Tolerability and Preliminary Efficacy of a Single Intracerebroventricular Injection of RB001 for the Treatment of SHANK3-related Phelan-McDermid Syndrome.
Active, not recruiting·PPeking University First Hospital·Phase 1·NCT07014020·8 enrolled
An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
Completed·NNeuren Pharmaceuticals Limited·Phase 2·NCT05025241·18 enrolled
Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)
Completed·IIcahn School of Medicine at Mount Sinai·Phase 2·NCT01525901·19 enrolled
AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy
Completed·—·Phase 2·NCT03493607·6 enrolled
Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome
Completed·—·Phase 2·NCT02710084·18 enrolled
A Controlled Trial of Growth Hormone in Phelan-McDermid Syndrome and Idiopathic Autism
Completed·IIcahn School of Medicine at Mount Sinai·Phase 2·NCT05187377·32 enrolled

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