VivoSeq
Other

Pachyonychia Congenita

5

Pipeline Programs↗

1

Active Trials↗

3

Companies Involved↗

Pachyonychia Congenita is an early-stage landscape with 5 programs across 3 companies. The most active players are Palvella Therapeutics, Inc., Prism Pharmaceuticals, Kamari Pharma Ltd. 1 therapy is approved with 1 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

28/100

Pipeline Velocity

0/100

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.

P

Palvella Therapeutics, Inc.

1 program

Phase 3P

Prism Pharmaceuticals

1 program

ApprovedK

Kamari Pharma Ltd

0 programs

Preclinical

Pipeline

Programs by Phase

Analysis

Trial Status

8

clinical trials

Completed6 · 75%
Unknown status1 · 13%
Recruiting1 · 13%

Clinical Trials

Trial Activity

8 trials

A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
Recruiting·PPalvella Therapeutics, Inc.·Phase 3·NCT05643872·45 enrolled
Study Designed to Evaluate Safety and Efficacy of 1% Topical Formulation of KM-001 on Type 1 Punctate Palmoplantar Keratoderma or Pachyonychia Congenita Diseases
Completed·KKamari Pharma Ltd·Phase 1·NCT05435638·14 enrolled
VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita
Completed·PPalvella Therapeutics, Inc.·Phase 3·NCT04520750·36 enrolled
Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
Completed·TTransDerm, Inc.·Phase 1·NCT02152007·15 enrolled
A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita
Completed·PPalvella Therapeutics, Inc.·Phase 3·NCT05180708·87 enrolled
Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita
Completed·PPalvella Therapeutics, Inc.·Phase 2/3·NCT03920228·73 enrolled
Study of TD101, a Small Interfering RNA (siRNA) Designed for Treatment of Pachyonychia Congenita
Completed·—·Phase 1·NCT00716014·1 enrolled
Simvastatin Treatment of Pachyonychia Congenita
Unknown status·TTel-Aviv Sourasky Medical Center·N/A·NCT01382511·10 enrolled

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