Pipeline

Our portfolio of investigational therapeutics

We are developing innovative therapeutics with the potential to have a major impact on the treatment of chronic respiratory diseases.

Ensifentrine

Accepted for FDA review

In the third quarter of 2023, the FDA accepted for review the Company’s New Drug Application seeking approval of nebulized ensifentrine for the maintenance treatment of patients with COPD and assigned a PDUFA target action date of June 26, 2024. It has not been evaluated as safe or effective or approved for commercialization.

The novel small molecule is a selective dual inhibitor of the phosphodiesterase 3 (PDE3) and phosphodiesterase 4 (PDE4) enzymes. This dual inhibition enables the inhaled therapy to combine bronchodilator and non-steroidal anti-inflammatory properties in one compound1-6. Ensifentrine stimulated cystic fibrosis transmembrane conductance regulator (CFTR) in in vitro studies. This activity has the potential to reduce mucus viscosity and improve mucociliary clearance7.

Ensifentrine has been well tolerated in clinical trials involving approximately 3,000 subjects8. It is designed to maximize its effectiveness and reduce adverse events through:

  • selective inhibition of PDE3 and PDE4 over other enzymes and receptors to minimize off-target effects;
  • direct delivery to the lungs by inhalation to maximize pulmonary exposure to ensifentrine while minimizing systemic exposure and potential adverse events.
1

Singh D, et al. Eur Respir J. 2018;52:1801074

2

Singh D, et al. Respir Res. 2020;21:47.

3

Ferguson GT, et al. Int J Chron Obstruct Pulmon Dis. 2021;16:1137-1148

4

Boswell-Smith V, et al. J Pharmacol Exp Ther. 2006;318(2):840-848.

5

Franciosi LG, et al. Lancet Respir Med. 2013;1(9):714-727.

6

Rheault T, et al. Presented virtually at: CHEST Annual Meeting; October 18-21, 2020

7

Turner MJ, et al. Am J Physiol Lung Cell Mol Physiol. 2016;310(1):L59 70

8

Data on file. Verona Pharma, plc. Raleigh, NC; USA

Indication & Formulation

Preclinical

Phase 1

Phase 2

Phase 3

US FDA Review

Ensifentrine (Nebulizer) – Maintenance treatment of COPD

Ensifentrine (Nebulizer) – Non-Cystic Fibrosis Bronchiectasis

Ensifentrine (Nebulizer) – Cystic Fibrosis

Ensifentrine (Nebulizer) – Asthma

Ensifentrine + LAMA (Nebulizer) – Maintenance treatment of COPD

Ensifentrine (DPI/MDI) – Maintenance treatment of COPD

Ensifentrine (DPI/MDI) – Asthma

Ensifentrine (DPI/MDI) – Cystic Fibrosis

1

Anzueto A, et al. Am J Respir Crit Care Med. 2023;208(4):406-416

2

Barjaktarevic I, et al. Am J Respir Crit Care Med. 2023;207:A5008

3

Turner MJ, et al. Am J Physiol Lung Cell Mol Physiol. 2016;310(1):L59 70

4

Eneje OJ, et al. Ped Pulm. 2018;53(S2):228

5

Bjermer L, et al. Pulm Pharmacol Ther. 2019;58:101814

6

Kalhan R, et al. Poster Presented at: American Thoracic Society (ATS) International Conference; May 17-22, 2024; San Diego, CA

7

Dransfield M, et al. Oral Presentation Given at: American Thoracic Society (ATS) International Conference; May 17-22, 2024; San Diego, CA

8

Rheault T, et al. Ensifentrine, a dual PDE 3 and 4 inhibitor, provides effective bronchodilation in patients with COPD when administered twice daily over 7 days via pMDI. Presented virtually at CHEST Annual Meeting; October 17-20, 2021

9

Rheault T, et al. Ensifentrine, a novel dual phosphodiesterase (PDE) 3 and 4 inhibitor, provides effective bronchodilation in COPD when administered twice daily over 7 days via a dry powder inhaler [poster]. Presented at: American Thoracic Society; May 15-20, 2020

Developing novel therapeutics for respiratory diseases

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