Developing innovative therapies for the treatment of respiratory diseases
May 2024
Ensifentrine is an investigational drug and has not been approved by the FDA (or any other regulatory authority).
Nasdaq: VRNA | www.veronapharma.com
Forward-looking statements
This presentation contains "forward‐looking" statements that are based on the beliefs and assumptions and on information currently available to management of Verona Pharma plc (together with its consolidated subsidiaries, the "Company"). All statements other than statements of historical fact contained in this presentation are forward-looking statements. Forward‐looking statements include information concerning the initiation, timing, progress and results of clinical trials of the Company's product candidates, the timing or likelihood of regulatory filings and approvals of its product candidates, and estimates regarding the Company's expenses, future revenues and future capital requirements.
Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause the Company's actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. These risks, uncertainties and other factors include those under "Risk Factors" in the Company's annual report on Form 10-K for the year ended December 31, 2023, and current reports on Form 8-K and our other filings with the Securities and Exchange Commission. Forward- looking statements represent the Company's beliefs and assumptions only as of the date of this presentation. Although the Company believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee future results, levels of activity, performance or achievements. Except as required by law, the Company assumes no obligation to publicly update any forward‐looking statements for any reason after the date of this presentation, or to conform any of the forward-looking statements to actual results or to changes in its expectations.
This presentation also contains estimates, projections and other information concerning the Company's business and the markets for the Company's product candidates, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research, or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, the Company obtained this industry, business, market and other data from reports, research surveys, clinical trials studies and similar data prepared by market research firms and other third parties, from industry, medical and general publications, and from government data and similar sources.
The Company's product candidate, ensifentrine, is an investigational drug under review by the US Food and Drug Administration (FDA). It has not been evaluated as safe or effective or approved for commercialization by any regulatory authority.
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Ensifentrine is an investigational first-in-class drug candidate US FDA review for maintenance treatment of COPD
PDUFA Action Date of June 26, 2024
Large market
with significant
unmet need
- ~$10B US sales1
- Millions of patients remain symptomatic and unsatisfied with current therapies2-6
Ensifentrine novel profile
- Novel, selective MOA, dual inhibitor of PDE3 and PDE4
- Positive Phase 3 data on key lung function measures, symptoms and exacerbations
- Well-toleratedover 24 and 48 weeks
Targeted
commercial opportunity
- Pulmonologists key to adoption with potential for broad utilization5
- Medical Benefits (Primarily Medicare Part B) is expected to be an important reimbursement channel7
1As of year end 2021, IQVIA MIDAS, IQVIA MIDAS Medical; 2Ferguson et al. Lancet Respir Med 2018;6:747-58; 3Mahler D, et al., Eur Respir J, 2014;43:1599-1609;4Vestbo J, et al., The Lancet, 2017;389:19-29;5Verona I US MCOPD Integrated Conjoint Survey; 6Phreesia COPD Patient Survey 2022; 7Verona_IQVIA_LAAD Quarterly Reporting Report
3 2_June 2022- Data for MAT Dec 2021
Verona Pharma's respiratory product pipeline
Ensifentrine provides multiple product opportunities
Product | Indication | Pre-clinical | Phase 1 | Phase 2 | Phase 3 | US FDA |
review | ||||||
Maintenance | ||||||
treatment of COPD | ||||||
Ensifentrine | Non-CF | |||||
(Nebulizer) | bronchiectasis | |||||
Cystic Fibrosis | ||||||
Asthma | ||||||
Ensifentrine + LAMA | Maintenance | |||||
(Nebulizer) | treatment of COPD | |||||
Maintenance | ||||||
treatment of COPD | ||||||
Ensifentrine | Asthma | |||||
(DPI / MDI) | ||||||
Cystic Fibrosis | ||||||
LAMA: Long-acting muscarinic agent
4 DPI: Dry powder inhaler, pMDI: Pressurized metered-dose inhaler
Strong financial position for potential launch
Financial highlights
Cash and Equivalents | $254.9M |
(as of March 31, 2024) | |
Operating expenses | $27.2M |
(quarter ended March 31, 2024) | |
Market cap (Nasdaq: VRNA) | $1.2B |
(as of May 7, 2024) | |
Shares outstanding* | 81.1M ADSs |
(as of May 7, 2024) | |
*Approximately 648.7M ordinary shares outstanding. **Capped at 1.75x of the amount funded.
***Runway expectations based on cash and equivalents as of March 31, 2024, and future draws on Oaktree OMERS debt facility and RIPSA.
- $400M debt facility ($345M potential future draws)
- $250M Revenue Interest Purchase and Sale Agreement**
- 2026 funded beyond 2026***
5
COPD affects >390 million patients worldwide1
Despite available treatments COPD is still the third leading cause of death
Prevalence of COPD in US:
~8.6M treated chronically2
Prevalence of COPD in China:
~100M patients3
Prevalence of COPD in EU:
~70M patients4
~$10B in maintenance COPD sales5
~$1B in sales (expected to double by 2030)5
~2B Euros in sales (2020)5
1Adeloye et al., The Lancet Respir Med, 2022;10(5):447-458;2Verona IQVIA Ensifentrine Market Research; 3Wang et al., The Lancet, April 2018;391; 4NCD Alliance
6 estimates, https://ncdalliance.org/news-events/news/66-million-people-may-live-with-copd-in-europe-yet-it-remains-unknown;5IQVIA MIDAS, IQVIA MIDAS Medical
Current COPD maintenance treatments limited to 3 MoAs
LAMAs & dual therapies generate the majority of US sales
Maintenance Treatment Choices1
LAMA | Perforomist | LABA | |
(bronchodilator) Yupelri | (bronchodilator) | ||
(nebulized) | Anoro | (nebulized) | |
Lonhala | Brovana | ||
Stiolto | |||
(nebulized) | (nebulized) | ||
Bevespi | |||
Incruse | Serevent | ||
Breztri
SpirivaBreo Striverdi
Trelegy
TudorzaAdvair
Symbicort
ICS*
(anti-inflammatory)
2021 US Sales by MOA2
2021 Sales | ||
($,m) | ||
$4,000 | ||
$3,000 | $2,931 | |
$2,377 | $2,510 | |
$2,000 | $1,716 | |
$1,000 | $526 | |
$- |
LAMA: Long acting muscarinic agent, LABA: Long acting beta-adrenoceptor agonist, ICS: Inhaled corticosteroids
7 *GOLD Guidelines recommend ICS products for patients who have a frequent history of exacerbations
1https://www.copdfoundation.org/Learn-More/I-am-a-Person-with-COPD/Treatments-Medications.aspx;2IQVIA MIDAS, IQVIA MIDAS Medical
Execution driven leadership team
Decades of respiratory and commercialization experience
David Zaccardelli, Pharm D
President & CEO
Mark Hahn, CPA
CFO
Kathy Rickard, MD
CMO
Chris Martin
Chief Commercial Officer
Tara Rheault, PhD
Chief Development Officer
Andrew Fisher, JD
General Counsel
Caroline Diaz
Senior VP, Regulatory Affairs
Kavita Aggarwal
Senior VP, Medical Affairs
Ostra Jewell
Senior VP, Human Resources
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Ensifentrine: Novel mechanism of action
Resulting in downstream bronchodilatory, anti-inflammatory, and ciliary effects
Direct mechanisms:
• Modulation of intracellular cAMP in cells that express PDE3, PDE4, or both
Indirect mechanisms:
• Reduction in macrophage activation that impacts cellular adhesion, chemotaxis, and survival of neutrophils and eosinophils
• CFTR activation and increased ciliary beat frequency in vitro
1Calzetta L, et al., J Pharmacol Exp Ther. 2013;346(3); 2Calzetta L, et al., Pulm Pharmacol Ther 2015;32:15-23;3Matera MG, et al., Am J Respir Crit Care Med 2013;187:A1495; 4Venkatasamy R, et al., Br J Pharmacol 2016;173(15):2335-2351;5Boswell-Smith V, et al., J Pharmacol Exp Ther 2006;318(2):840-848;6Franciosi LG, et al., Lancet Respir Med
9 | 2013;1(9):714-727;7Schmidt D, et al., Br J Pharmacol 2000;131(8):1607-1618;8Turner MJ, et al., Am J Physiol Lung Cell Mol Physiol 2016;310(1):L59-70 |
Pivotal Phase 3 program
Two efficacy and safety studies: ENHANCE-1 and ENHANCE-2
Ensifentrine as a Novel inHAled Nebulized COPD thErapy in moderate to severe COPD
Ensifentrine | Long-term safety | ||
ENHANCE-1 | 3mg BID n=500 | n=300 | Reported |
n=800 | Long-term safety | December 2022 | |
Placebo BID n=300 | |||
n=100 | |||
Ensifentrine | Reported | ||
ENHANCE-2 | 3mg BID n=500 | ||
August 2022 | |||
n=800 | |||
Placebo BID n=300 | |||
24 Weeks | 48 Weeks |
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Patient population:
- LAMA or LABA background allowed (approx. 50% of trial population) and ICS (up to approx. 20% of population)
- 30-70%predicted FEV1
- Symptomatic (mMRC ≥ 2)
Additional information:
- Long-termsafety in ENHANCE-1
- Sites in North America, EU and Asia
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Verona Pharma plc published this content on 13 May 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 13 May 2024 16:33:08 UTC.