全球-生物制药行业-全球生物制药业研究：阵发性夜间血红蛋白尿(PNH)评估

J.P. Morgan Global BioPharma ResearchALXN / APLS / ROG / NOVN / BCRX: Assessing the Paroxysmal Nocturnal Hemoglobinuria (PNH) LandscapeSee the end pages of this presentation for analyst certification and important disclosures, including non-US analyst disclosures.J.P. Morgan does and seeks to do business with companies covered in its research reports. As a result, investors should be aware that the firm may have a conflict of interest that could affect the objectivity of this report. Investors should consider this report as only a single factor in making their investment decision. Global Equity ResearchJune 2020US SMid BiotechnologyAnupam Rama AC212-622-0900anupam.rama@jpmorgan.comTessa T Romero212-622-4484tessa.t.romero@jpmorgan.comMatthew Bannon212-622-0001matthew.bannon@jpmorgan.comJ.P. Morgan Securities LLCEuropean BioPharmaRichard Vosser AC(44-20) 7742-6652richard.vosser@jpmorgan.comJames Gordon(44-20) 7742-6654 james.d.gordon@jpmorgan.comSarita Kapila M.D. (44-20) 7134-4189 sarita.kapila@jpmorgan.comLaerke Engkilde(44-20) 7742-2917 laerke.engkilde@jpmorgan.comJ.P. Morgan Securities plcLarge Cap BiotechnologyCory Kasimov AC212-622-5266cory.w.kasimov@jpmorgan.comGavin Scott212-622-0579gavin.scott@jpmorgan.comMatthew Holt, Ph. D.212-622-9602matthew.t.holt@jpmorgan.comTurner Kufe, M.D.212-622-4104turner.kufe@jpmorgan.comJ.P. Morgan Securities LLCUS SMid BiotechnologyJessica Fye AC212-622-4165jessica.m.fye@jpmorgan.comYuko Oku212-622-5374yuko.oku@jpmorgan.comDaniel Wolle212-622-5805daniel.wolle@jpmchase.comJ.P. Morgan Securities LLC2Key Takeaways – ALXN / APLSSource: J.P. MorganAlexion ( ALXN | OW | Dec-2020 PT $158 | covered by Cory Kasimov) We remain bullish on Alexion’s outlook and continue to believe that the company has the largest valuation disconnect amongst large cap biotechs. With that said, we acknowledge the concentration risk feared by many but view biosimilar and branded risk as overblown (within PNH in particular), especially in light of management’s solid execution on the Soliris-Ultomiris conversion strategy.  We expect relative stability for Alexion’s PNH franchise, given the company’s 10+ years of commercial dominance in this prevalence-based disease and ongoing innovation; new entrants and novel therapies have the potential to take market share (albeit in subsets of the market) but we also expect this to contribute to overall PNH market growth.Apellis ( APLS | OW | Dec-2020 PT $56 | covered by Anupam Rama) We conservatively estimate peak sales of pegcetacoplan (APL-2) in PNH to be ~$400-500M. Currently, in our model, sales are predominately driven by the anemic transfusion dependent population (which was reflected in our prior physician feedback). Hence, increased penetration in the naïve population and / or anemic but not regularly transfused setting represents upside to our estimates. Interestingly, we sense increasing investor focus on APL-2 in geographic atrophy (GA; phase 3 DERBY & OAKS readout in 3Q21).  Indeed,