VivoSeq
Rare Disease

Cystic Fibrosis

347

Pipeline Programs↗

48

Active Trials↗

92

Companies Involved↗

Cystic Fibrosis is a highly competitive landscape with 347 programs across 92 companies. The most active players are Nivalis Therapeutics, Inc., Takeda, Arcturus Therapeutics, Inc.. 51 therapies are approved with 47 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

89/100

Pipeline Velocity

34/100

Mech. Concentration

11/100

Completion Rate

82/100

Attrition Rate

15/100

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.

N

Nivalis Therapeutics, Inc.

2 programs

Phase 1Takeda logo

Takeda

2 programs

Phase 1A

Arcturus Therapeutics, Inc.

1 program

Phase 1K

Krystal Biotech, Inc.

1 program

Phase 1S

Spyryx Biosciences, Inc.

1 program

Phase 1Chiesi Farmaceutici S.p.A. logo

Chiesi Farmaceutici S.p.A.

1 program

Phase 1

Pipeline

Programs by Phase

Preclinical52

Elexacaftor

Nottingham University Hospitals NHS Trust

Fluticasone

Paratek Pharmaceuticals Inc

Saxagliptin

AstraZeneca

Interferon Gamma-1b

Horizon Therapeutics Ireland DAC

Albuterol

Glaxo Dba Glaxosmithkline

Phase 163

Hydroxychloroquine

Alnylam Pharmaceuticals

Xenon

Nobilis Therapeutics Inc.

Resveratrol

National Cancer Institute (NCI)

Fenretinide

Eastern Cooperative Oncology Group

Lansoprazole

Takeda

Phase 2134

Riociguat

Bayer

Tiotropium Bromide

Boehringer Ingelheim

Vardenafil

Bayer

Gentamicin

Schering-Plough

Miglustat

Johnson & Johnson

Phase 347

Mannitol

B. Braun Medical Inc.

Ataluren

PTC Therapeutics

Ivacaftor

Vertex Pharmaceuticals

Prednisone

Roxane Laboratories

Somatropin

EMD Serono

Approved51

Levofloxacin

Wim Janssens

Denosumab

Amgen

Polyethylene Glycol

Bayer

Tobramycin

Viatris Specialty LLC

Repaglinide

Gemini Therapeutics, Inc.

Analysis

Modality Breakdown

94%

Small molecule

336 of 359 classified programs — the dominant modality here.

6%ALL OTHERSProtein / enzyme113%Monoclonal antibody51%RNA therapy3<1%Cell therapy1<1%Gene therapy1<1%Vaccine1<1%Other1<1%

Small molecule leads; the ring shows all 359 classified programs, each share of the total. Modality classified for 100% of 387 pipeline programs.

Analysis

Trial Status

543

clinical trials

Completed377 · 69%
Terminated63 · 12%
Recruiting30 · 6%
Unknown status27 · 5%
Withdrawn21 · 4%
Active, not recruiting9 · 2%
Other16 · 3%

Clinical Trials

Trial Activity

543 trials

Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis
Recruiting·AArcturus Therapeutics, Inc.·Phase 2·NCT06747858·12 enrolled
Study of RSS0343 Tablets in Patients With Non-cystic Fibrosis Bronchiectasis
Recruiting·JJiangsu HengRui Medicine Co., Ltd.·Phase 2·NCT06775340·252 enrolled
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients with Cystic Fibrosis
Recruiting·SSpliSense Ltd.·Phase 2·NCT06429176·24 enrolled
Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis
Recruiting·Vertex Pharmaceuticals logoVertex Pharmaceuticals·Phase 1·NCT06154447·255 enrolled
Beta-cell Response to Incretin Hormones in Cystic Fibrosis
Recruiting·UUniversity of Pennsylvania·N/A·NCT01851694·45 enrolled
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
Recruiting·Vertex Pharmaceuticals logoVertex Pharmaceuticals·Phase 3·NCT05422222·210 enrolled
Dorzagliatin in Pancreatic Insufficient Cystic Fibrosis
Recruiting·UUniversity of Pennsylvania·Phase 1·NCT06995651·15 enrolled
Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials
Recruiting·—·N/A·NCT04580368·50 enrolled
IV Gallium Study for Patients With Cystic Fibrosis Who Have NTM (ABATE Study)
Recruiting·—·Phase 1·NCT04294043·40 enrolled
A Study to Assess the Efficacy and Safety of HSK31858 in Participants With Non-Cystic Fibrosis Bronchiectasis
Recruiting·Haisco Pharmaceutical Group Co., Ltd. logoHaisco Pharmaceutical Group Co., Ltd.·Phase 3·NCT06660992·669 enrolled
Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators
Recruiting·—·N/A·NCT04732910·500 enrolled
Impact of Elexacaftor-Tezacaftor-Ivacaftor Treatment on Metabolic, Epigenetic and Fecal Microbiota Profiles in People With Cystic Fibrosis.
Recruiting·MMeyer Children's Hospital IRCCS·N/A·NCT07363304·150 enrolled
Sinonasal Therapies and Histologic Correlations of Patients With Cystic Fibrosis in the Era of Highly Effective Modulator Therapy
Recruiting·IIndiana University·Phase 3·NCT06996951·64 enrolled
A Study Assessing KB407 for the Treatment of Cystic Fibrosis
Recruiting·KKrystal Biotech, Inc.·Phase 1·NCT05504837·12 enrolled
A Phase II Study of Ensifentrine in Non-Cystic Fibrosis Bronchiectasis
Recruiting·VVerona Pharma plc·Phase 2·NCT06559150·180 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)