未来一周美国临床肿瘤学会（ASCO）摘要（MRUS IDYA ORIC CELC）；URGN咨询委员会（与TYRA相关）

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 •NTLA:started recruiting at 12 add'l sites (98 recruiting sites in total) for NTLA-2001 in ph.IIIATTR-CM (MAGNITUDE) (n~765; NCT06128629) as of 5/13•RARE:started recruiting at 2 add'l sites (23 recruiting sites total) for GTX-102 in ph.IIIAngelman Syndrome (Aspire) (N~120, NCT06617429) as of 5/14•RARE:updated trial design to include a 4th dose level (vs 3 dose levels prior) for the ph.I/II anda seamless randomized, open-label, active-controlled ph.III study; increased enrollment to 82pts (vs 78 prior) for UX701 in Wilson Disease (NCT04884815) as of 5/11Regulatory tracker•KALV:Sebetralstat HAE oral on-demand PDUFA 6/17/25•RARE:MPSIIIA BLA (priority review) PDUFA 8/18/25•BCRX & 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•TVTX:Filspari FSGS PDUFA 1/13/26 w/ Adcom likely in 4Q (JEF est)BACK PAGES HAVE NEAR-TERM CATALYSTS, UPCOMING MED 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. for LFTs PDUFA 8/28/25 CONTINUED1H25 Catalysts•SGMO:Ph.I/II Fabry gene therapy topline eGFR data in June (stock +30%/-50%)•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%)•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%)•IDYA:Ph.II neoadjuvant data (n>75; 50/50 split plaque brachytx and enucleation) w/ fulsomevision/edema @ ophthalmology conf (est ASRS) in mid'25 (stock +30%/-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%)•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+40%/-50%)•VERA:Top-line pivotal UPCR data for accelerated approval 2Q25 (stock +/-50%)•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%)•MRUS:Update from 1L HNSCC ph.II update with 12mos OS, 6mos PFS, & ORR in 45 pts on5/22 conf call @5:30PM ET ahead of the poster presentation @ASCO on 6/2 (stock +/-20%)•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 June 1 (stock +/-15%)•GBIO:Preclin POC data and DC nomination for initial T cell targeting siRNA program mid'25(stock +30%/-10%)•ORIC:prostate combo '944 w/ apa/daro (n=12-15 total) w/ focus on safety and PSA waterfall1H25 (stock +20%/-10%)•AUPH:Ph.I health volunteer clinical data for BAFF/APRIL inh for rare/autoimmune diseases2Q25 (stock +25%/-10%)•TYRA:Readthrough from URGN ODAC 5/21/25 and then pot'l approval of UGN-102 in LG-IR-NMIBC w/ PDUFA 6/13/25 (stock +/-10%)•VALN:Full randomized, controlled immunogenicity+safety data from ph.I for Zika est mid'25(stock+20%/-5%)•TSHA:Program update w/ NHS, pivotal design, functional/milestone primary endpt, and PartA dose escalation update w/ high-dose efficacy in pediatric+adults and long-term follow-upfrom low-dose in pediatric+adults on/prior to oral pres @IRSF (6/11 @9:30AM ET) (stock+100%/-50%)•TSHA:Update on registrational trial design in Rett syndrome from competitor NGNE 2Q25(stock +/-10%)•KALV:PDUFA decision for sebetralstat as 1st oral on-demand HAE therapy (6/17/25) (stock+10-15%/-50%)•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 +/-5-10%)•SLDB:Ph.I/II D90 data from ~10-12 pts and then feedback from FDA & EMA on pivotal path4Q25 (stock+35%/-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. Upcoming med confs:•ASCO (May 30-June 2):reg+LBA titles related to our coverage (here), most abstracts post onMay 22 @5PM ET, LBA abstracts post on day of presentation @8AM ET•European Renal Association (June 4-7):abstracts posts on June 4 inNephrology DialysisTransplantationhere•European Cystic Fibrosis Conference (June 4-7)•European Alliance of Associations for Rheumatology(EULAR; June 11-14):reg titles here;LBA titles post during the we