2025年美国临床肿瘤学会（ASCO）要点-汇编

Restricted - External U.S. Small & Mid Cap BiotechnologyPOSITIVEU.S. Small & Mid Cap BiotechnologyGena Wang, PhD, CFA+1 212 526 4252gena.wang@barclays.comBCI, USHang Hu, PhD+1 212 526 6364hang.hu@barclays.comBCI, USTony Deng, MD+1 212 526 0350tony.deng@barclays.comBCI, USJustin Kong, MD+1 212 526 1961justin.kong@barclays.comBCI, US Cancer, Texas oncology, and US oncology, where a large number of community doctorsparticipate. Study sessions and engagement were underway to facilitate referrals to authorizedtreatment centers.EU revenue contribution percentage was expected to reach double digit in 2025.EUrevenue contribution was 9.8% in 2024 ($94M out of $963M total revenue), and expected to growinto double digit in 2025. Most pts were noted in large centers, which was easier for targetedcommercialization. With 2H25 capacity expansion from Tech Lane commercial production, EUrevenue growth rate was expected to surpass US.Additional color on competitive landscape.Mgmt remained confident in anticipation ofpotential competitors, including anito-cel, Blenrep (PDUFA of July 23, GSK), and bispecifics suchas Elrexfio. Mgmt highlighted targeting 3 groups of pts population in 2L MM: 1) high-risk 2/3Lpts, including 25-30% pts who had high-risk cytogenetics; 2) 10-15% pts had functional highrisk; 3) younger pts who would value drug holiday benefit from Carvykti treatment.Likely limited impact from MFN (Most-Favored-Nation) policy.Carvykti 2025 US WAC price is$555K. Our quick search suggested likely limited exposure to MFN policy with largelycomparable pricing in EU vs. US, including German list price of €420K (~$477K) in 2023 andDenmark price of kr. 3.8M (~$569K).LEGN Investor EventKOL background and practice.Three KOLs were invited to the event. Dr. Leyla Shune, M.D. isan Associate Professor, Hematologic Malignancies and Cellular Therapeutics at University ofKansas Cancer Center. Dr. Doris Hansen, M.D. is an Assistant Member in the Department ofBlood and Marrow Transplant and Cellular Immunotherapy at TheMoffittCancer Center. Dr.Binod Dhakal, M.D. is an Associate Professor of Medicine Cancer Center – Froedtert HospitalMedical College of Wisconsin.KOLs view cilta-cel as best-in-class, considered 33% cure rate as unprecedented in MM.KOLs believed cilta-cel was the first and only CART therapy that demonstrated superior OS,citing 1) unprecedented durability of response and survival benefit observed in CARTITUDE-1(LINK), and 2) consistent PFS and OS benefit across high-risk subgroups in CARTITUDE-4 (LINK).More importantly, C-1 data showed unprecedented treatment cure in MM.Prophylactic strategy.KOL highlighted Parkinsonism related to cilta-cel was rare, manageable,and considered a classeffectof BCMA-targeted therapies, not unique to cilta-cel with emergingstrategies significantly reducing its incidence. One KOL emphasized Parkinsonism was 6% inCARTITUDE-1, but has since dropped to <1% in CARTITUDE-4, and a real-world data showed~2% incidence of Parkinsonism (Siana et al, 2025). Multiple centers were noted to be alreadyadopting low-dose steroids in patients with rising ALCs, a simple, accessible biomarker.Preliminary single-center data showed zero high-grade neurotoxicity eventsafterthis strategywas implemented. KOLs startedseeing fewer events because they were “bridging better,debulking more, and intervening earlier”.Unfair comparison between CARTITUDE-1 and iMMagine-1.Three KOLs noted that the cross-trial comparison was unfair given 1) iMMagine-1 enrolled less sick pts with median 3 (vs. 6 inCARTITUDE-1) prior line of therapy and 51% (vs. 18% in CARTITUDE-1) pts with 3 prior LOT; 2)built on prior learning and experience, iMMagine-1 had protocols to better manage toxicities,exemplified by aggressive use of IL-6/steroids; 3) current iMMagine-1 data had median follow-upof 12.6 mons, thorough safety evaluation would need longer follow-up (See Summary of 2025EHA abstracts, 05/14/2025). Regardingefficacycomparison, the KOL focused on PFS and curerate, noting Carvykti setting a high bar.2 Continuedeffortsin neurotox monitoring and prevention.Following TANDEM data update(See Takeaways from TANDEM Meetings, 02/14/2025), continuedeffortsincluded the protocolamendment in existing CARTITUDE trials, real world evidence analysis, and investigator-sponsored studies. Mgmt iterated the rolling out of absolute lymphocyte count (ALC)monitoring and prophylactic dexamethasone in CARTITUDE programs to prevent neurotox.KOLs highlighted a retrospective analysis in collaboration with multiple investigators for ALCmonitoring which would be presented in International Myeloma Society (IMS) meeting, as wellas a perspective, multi-center study for the steroid intervention over high-risk periodafterinfusion or in association with correlated biomarkers, including ALC, CAR-T expansion, andcytokines.KOL thoughts on Carvykti uptake in 2/3L pts.KOLs noted less adverse events when Carvyktiwas administrated in earlier line pts. In 2/3L setting, Parkinsonism wa