未来一周：美国临床肿瘤学会（ASCO）会议收尾；杰富瑞医疗会议；欧洲血液学协会（EHA）6月3日最新突破性摘要；欧洲肾脏协会（ERA）6月4 - 7日会议

Maury Raycroft, Ph.D. * | Equity Analyst(212) 323-3990 | mraycroft@jefferies.comAmy Qian, Ph.D. * | Equity Associate+1 (212) 707-6465 | aqian@jefferies.comJames Stamos, Ph.D. * | Equity Associate(212) 323-7644 | jstamos@jefferies.comFarzin Haque, Ph.D. * | Equity Analyst+1 (212) 778-8349 | fhaque@jefferies.comAmin Makarem, Ph.D. * | Equity Associate+1 (332) 236-6822 | amakarem@jefferies.com Clinicaltrials.gov updates:•ELVN:started recruiting at 1 add'l site (28 recruiting sites in total) for ELVN-001 in ph.I CML(N=180; NCT05304377) as of 5/30•ERAS:started recruiting at 1 site for ERAS-0015 in ph.I metastatic solid tumors (AURORAS-1)(N=200, NCT06983743) as of 5/29•MRUS:started recruiting at 8 add'l sites (129 recruiting sites in total) for peto+pembro in ph.III1L HNSCC (LiGeR-HN1) (N=500; NCT06525220) as of 5/29••RARE:started recruiting at 2 add'l sites (25 recruiting sites in total) for GTX-102 in ph.IIIAngelman Syndrome (Aspire) (N=120, NCT06617429) as of 5/25•SGMO:started recruiting a 2 add'l sites (7 recruiting sites in total) for ST-503 in ph.I small fiberneuropathy (iSFN)-associated refractory pain (N=27, NCT06980948) as of 5/29•TYRA:initiated an open-label ph.II study for TYRA-300 in FGFR3-altered low grade,intermediate risk NMIBC (SURF302) (N=90, NCT06995677) as of 5/29...CONTINUE ON BACK PAGES FOR REGULATORY TRACKER, NEAR-TERM CATALYSTS, UPCOMINGMED CONFS, and RECENT REPORTSPlease see important disclosure information on pages 6 - 15 of this report.This report is intended for Jefferies clients only. Unauthorized distribution is prohibited. ...CONTINUEDRegulatory tracker•KALV:Sebetralstat HAE oral on-demand PDUFA 6/17/25•RARE:MPSIIIA BLA (priority review) PDUFA 8/18/25•BCRX and NTLA-related:IONS donidalorsen HAE prophy PDUFA 8/21/25•TVTX:Change in REMS monitoring fromQM→Q3M•BCRX:Orladeyo pediatric (2-11yo) HAE oral prophy PDUFA 9/12/25•ARWR:Plozasiran FCS PDUFA 11/18/25•VERA-related: Otsuka's sibeprenlimab IgAN PDUFA 11/28/25•TVTX:Filspari FSGS PDUFA 1/13/26 w/ AdCom likely in 4Q (JEF est)1H25 Catalysts•AUPH:Ph.I healthy volunteer clinical data for BAFF/APRIL inh for rare/autoimmune diseases2Q25 (stock +25%/-10%)•CELC:Ph.Ib/II mCRPC trial initial data 2Q25 (stock +30%/-10%)•CELC:Ph.III top-line data in WT 2L aBC 3Q25 (stock +>100%/-50%)•CRSP:Interim '320 Lp(a) editing data 2Q25 (stock +20%/-10%)•CRSP:CTX112 CAR-T data in oncology (LBCL) and AID (SLE, SSc, IIM) mid'25 (stock+20%/-30%)•DNTH:Readthrough from ANNX's oral active C1s inhibitor ANX1502 in CAD POC study (n~7)based on potential selection of neuromuscular indications mid'25 (stock +/-10%)•ELVN:Ph.I expansion+escalation CML data w/ n~60-100 total pts incl ~40-50 efficacyevaluable w/ ≥6mos follow-up @EHA on 6/13 @11AM ET w/ conf call @1:30PM ET (stock+30%/-15%)•GBIO:Preclin POC data and DC nomination for initial T cell targeting siRNA program mid'25(stock +30%/-10%)•IDYA:Ph.II neoadjuvant data (n>75; 50/50 split plaque brachytx and enucleation) w/ fulsomevision/edema at ophthalmology conf (est ASRS) in 2Q/3Q25 (stock +20%/-5%)•IDYA:Readthrough from ZLAB's DLL3-TOP1 ADC dose escalation update (n=25) and prelimdose optimization data (n~50) in 2L+ ES-SCLC in a poster presentation on 6/2 @ASCO (stock+15%/-5%)•KALV:PDUFA decision for sebetralstat as 1st oral on-demand HAE therapy (6/17/25) (stock+20%/-50%)•MRUS:Readthrough from BCAX's ph.I/Ib OS+PFS w/ ficerafusp alfa and pembro in 1L HPV(-)HNSCC in a rapid oral presentation @ ASCO on 6/1 (stock +5%)•NTLA:Longer-term HAE ph.I data on 6/15 (stock +/-10%)•RARE:Ph.III ORBIT 2nd interim analysis (all pts @12mos; p<0.01) in osteogenesis imperfectamid'25 (stock +50%/-25%)•MREO:Ph.III ORBIT 2nd interim analysis mid'25 (stock +>100%/-30%)•SGMO:Ph.I/II Fabry gene therapy top-line eGFR data in June (stock +30%/-50%)•TNGX:Clinical update from BMY's PRMT5 ph.I and 1st report of PK/PD @ASCO on June 2(stock +/-15%)•TSHA:Add'l details on NHS, pivotal design (and pot'l BLA path), primary endpt, and Part A doseescalation data @IRSF (6/11 @9:30AM ET) (stock +/-20%)Please see important disclosure information on pages 6 - 15 of this report.This report is intended for Jefferies clients only. Unauthorized distribution is prohibited. for LFTs PDUFA 8/28/25 •TSHA:Update on registrational trial design in Rett syndrome from competitor NGNE 2Q25(stock +/-10%)•TYRA:Readthrough from pot'l approval of UGN-102 in LG-IR-NMIBC w/ PDUFA 6/13/25 (stock+15%/-5%)•VALN:Full randomized, controlled immunogenicity+safety data from ph.I for Zika est mid'25(stock+20%/-5%)•VERA:Top-line pivotal UPCR data for accelerated approval 2Q25 (stock +/-50%)Upcoming med confs:•European Renal Association (June 4-7):program here; abstracts posts on June 4 inNephrologyDialysis Transplantationhere•European Cystic Fibrosis Conference (June 4-7):program here; abstract available at the startof presentation to attendees only•International Rett Syndrome Foundation (June 9-11):program here•European Alliance of Associations for Rheumatology (EULAR; June 11-14):