维罗纳（VRNA）：2025年美国胸科学会会议额外的三期分析支持Ohtuvayre在慢性阻塞性肺疾病中的广泛应用案例

2023A2024A2025E(0.69)(2.12)1.22--318.10.042.3411.3(0.09)(0.27)0.16lung function, exacerbations and patient-lung function benefit and 2026E2.85628.9650.00.38 Andrew Tsai * | Equity Analyst(415) 229-1566 | atsai@jefferies.comMatthew Barcus, Ph.D. * | Equity Associate(415) 229-8703 | mbarcus@jefferies.comJohn Cox, Ph.D. * | Equity Associate+1 (415) 229-8708 | jcox1@jefferies.comBrian Balchin, ACA * | Equity Analyst(415) 229-1522 | bbalchin@jefferies.com Exhibit 1 - VRNA ATS 2025 Conference Events and Posters.Source: Jefferies, Company reportsPlease see important disclosure information on pages 17 - 22 of this report.This report is intended for Jefferies clients only. Unauthorized distribution is prohibited. The Long View: VeronaInvestment Thesis / Where We DifferVRNA's sole drug, ensifentrine (Ohtuvayre), for COPD has been approved bythe FDA. Ensifentrine is designed to act as both a bronchodilator and an anti-inflammatory agent and is the first novel agent approved for COPD in decades.Phase III data supports the therapeutic benefit of ensifentrine in not onlysevere COPD patients (1.2M+ US patients ~$14B US market opportunity) butalso potentially in less-severe patients (3M US patients). The drug launchedin early Aug. Given a large market opportunity, if ensifentrine is successfullydeveloped and commercialized, we expect meaningful upside potential fromthe current valuation.Base Case,$95, +28%•Our PT is based on a DCF analysis thatconsiders only ensifentrine in COPD.•Assumes ensifentrine can garner $2.5B+ peaksales.•Applies a 9% discount rate.•For the sake of conservatism,we do notassume indication expansion opportunities.Sustainability MattersTop Material Issues(s):1) Product Quality & Safety.VRNA commercializes a novel treatment for COPD,whose patients typically present with comorbid issues, so safety monitoring will be critical in post-marketing. 2)Access & Affordability.COPD has a large 8.6M population with unmet need, necessitatinga careful balance between access/affordability and profitability now that ensifentrine has been launchedsince Aug 2024.Company Target(s):VRNA has not yet disclosed its ESG commitment targets.Qs to Mgmt:1)Does VRNA have an oversight committee for its ESG-related efforts?2)What humanresources programs or initiatives does VRNA have in place to attract talent with diverse backgrounds,experiences, and perspectives?3)How does VRNA incorporate feedback from patients and their familiesinto product development?4)What programs or initiatives does VRNA have to promote ESG valuesamong its employees?ESG Sector Deep Dive:BiotechPlease see important disclosure information on pages 17 - 22 of this report.This report is intended for Jefferies clients only. Unauthorized distribution is prohibited. Upside Scenario,$130, +75%•Assumes a 3-4x multiple on a higher $3-3.5Bpeak sales.•A 4x multiple could be supported in an M&Atakeout scenario by Big Pharma.•To be conservative,we do not assumeindication expansion opportunities. Downside Scenario,$30, -60%•Assumes a lower peak sales estimate forensifentrine.•We assign a lower 2-3x multiple to peaksales, due to a potential slower ramp versusexpectations.Catalysts•Q2:25:Phase III data from China PartnerNuance Pharma•2025-26:Potential ex-US partnership(s)•2025-26:Advance regulatory preparations forUK/EU filings•2026+:Phase II non-CF bronchiectasis data 3 P275 & P285: Monotherapy Ohtuvayre Demonstrates Efficacy Benefits andClean Safety COPD Patients Without Background MedicationOhtuvayre monotherapy efficacy in COPD patients without background medication seemsconsistent with the broader Phase III efficacy population.Ohtuvayre monotherapy improves lungfunction and reduces exacerbations in 1L COPD patients, which represented 38-39% of N=1549total across two Phase III studies (pooled).(1)The abstracts previously disclosed that Ohtuvayre-treated patients showed a -40% pbo-adj reduction inmod/severe exacerbations(rate ratio RR=0.60,p=0.058), which is comparable to -36-43% for the overall Phase III population.(2)The poster hadnew strong details on the relative risk reduction (RRR) forexacerbation risk(i.e., time to firstexacerbation), which was 59% (HR=0.41, p=0.039).(3)The prior abstract showed a pbo-adj Week12 benefit inFEV1(+93mL) andpeak FEV1as well (p<0.05). Now, we see both Week 6 and Week 24 data suggesting both early and consistentpeak FEV1effects (+141-142mL).(4)In addition,TDIdyspnea (shortness of breath) improved by+2.0 vs +1.1 pbo across all weeks (p<0.05) at Week 24. We also now have Week 6 and 12 TDI data,showing a stepwise +0.2-0.3 increase over time (albeit in pbo too, but to a lesser extent at Week 24).Week 6 TDI scores were 1.5 drug vs 0.7 pbo and Week 12 TDI was 1.7 drug vs 0.9 pbo. We'd highlightthat the mean TDI scores for Ohtuvayre-treated patients were above the clinically-relevant MCID TDIscore of 1, while pbo patients were below (apart from Week 24). Additionally, we also notice newdata on the number of Ohtuvayre patients achieving