初步先例分析表明CMC问题可能很快得到解决

2024A2025E2026E0.00.00.00.0--(0.50)(0.46)--0.022.5(0.48)(0.44)(0.42) 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 - SVRA Newsflow.TimingExpected NewsflowQ4:25Pot'l BLA re-submission to FDAYE:25Submit MAA to EMA2025-26Potential EU, Japan regulatory approvalsH2:26Pot'l FDA approval of Molbreevi in aPAPSource: Company reports, Jefferies estimatesPlease see important disclosure information on pages 9 - 14 of this report.This report is intended for Jefferies clients only. Unauthorized distribution is prohibited. Exhibit 2 - Select RTF Precedents Since 2015.CompanyTickerDrug--MDPA/SNVOBMYGelUCBBMY-Source: Jefferies, BiomedtrackerBlue highlights/red boxes indicate drugs that recieved an RTF for apparent CMC issues.Please see important disclosure information on pages 9 - 14 of this report.This report is intended for Jefferies clients only. Unauthorized distribution is prohibited. The Long View: Savara Inc.Investment Thesis / Where We DifferSVRA is a clinical-stage biotechnology company developing inhaled therapiesfor orphan lung infectious diseases. Its lead asset, molgramostim, recentlysucceeded in a Phase 3 study for autoimmune pulmonary alveolar proteinosis(aPAP). Molgramostim could become the first approved treatment for aPAPworldwide. We think the risk/reward looks favorable, as $400M+ peak saleswould suggest upside to current price levels.Base Case,$8.5, +338%•Our $8.50 PT is based on an NPV analysisof molgramostim in aPAP, including $400M+peak sales, a 90% probability of success, a 9%discount rate, and 0% terminal growth (due tono biosimilar competition).Sustainability MattersTop Material Issues(s):1)Product Quality & Safety -As SVRA is actively developing a drug candidatefor a pulmonary disease, it will need to ensure the safety and quality of its product used in the trial, aswell as the safety of patients in the trial.2) Access & Affordability -Given SVRA’s drug candidate targetrare-disease patients, it will need to carefully balance between access/affordability of its product andprofitability once its product is approved and marketed in the future.Company Target(s):SVRA has not yet disclosed its ESG commitment targets.Qs to Mgmt:1)Does SVRA have an oversight committee for its ESG efforts?2)What proceduresdoes SVRA have in place to ensure the safety and quality of its drug candidate?3)What programs orinitiatives does SVRA have to promote ESG values among its employees?4)What human resourcesprograms or initiates does SVRA have in place to attract talent with diverse backgrounds, experiences,and perspectives?ESG Sector Deep Dive:LinkPlease see important disclosure information on pages 9 - 14 of this report.This report is intended for Jefferies clients only. Unauthorized distribution is prohibited. Upside Scenario,$15, +673%•Our $15 upside assumes the Phase III study formolgramostim in aPAP is successful.•Also assumes a higher 90%+ probability ofsuccess for molgramostim in aPAP.•Our peak sales assumption also increasescloser to $700M Downside Scenario,$0.5, -74%•If Molgradex fails in aPAP, we assume NTMcash levels of $0.50/sh.Catalysts•Q4:25Pot'l BLA re-submission of Molbreevi inaPAP rare lung disease•YE:25:Potential regulatory filings in EU•YE:26: Potential EU approval of Molgra in aPAP•2026:Potential regulatory filings in ex-UScountries (Japan, etc) 4 Refuse-to-file (RTF) precedents for CMC issues are generally resolved quickly. (1)CMC issuesfor NVO's Ozempic, BMY's Abecma, and (formerly) Acorda Therapeutics Inbrijawere all re-submitted in roughly 2-3 months.(2)We'd note that all of these therapies were also subsequentlyapproved on-time.(3)For more drawn-out NDA/BLA re-submissions (e.g., SUPN's Onapgo, YMAB'sOmblastys, and ANIP's cortrophin gel) the time durations seem mostly idiosyncratic for morecomplex therapies/devices. For example, Onapgo is a wearable apomorphine pump, Omblastys isa radiopharmaceutical, and ANIP is an injectable ocular gel, all of which could necessitate morecomplex CMC controls in theory.(4)To be fair, Molbreevi uses an eFlow nebulizer for delivery ofGM-CSF, but eFlows are already used in approved products (e.g., Lonhala Magnair in COPD).Net-net:We think the Q4:25 re-filing estimate for SVRA's Molbreevi could be is reasonable based onprecedents. We think the extra time could allow buffer for Fujifilm to definitively come online as abonafide supplier.Please see important disclosure information on pages 9 - 14 of this report.This report is intended for Jefferies clients only. Unauthorized distribution is prohibited. Exhibit 3 - Select RTF Precedents Since 2015.CompanyTickerImmunityBio Inc.IBRXHarmony BiosciencesHoldings, Inc.HRMYTheratechnologiesInc.THTXCardamyst-Biohaven Ltd.BHVNSoligenix, Inc.SNGXBrainStorm CellThera