2025-06-16-Jefferies-未来一周_KALV的PDUFA日期轻微延迟；ADA（与ARWR_ALNY相关）；ESMO妇科会议等_18页_177kb

Equity Research: USA Biotechnology - June 16, 2025

Key Highlights & Catalysts

Regulatory Updates

• KALV

  • Sebetralstat PDUFA date for HAE oral on-demand delayed to mid-July (originally June 17). Market views this as a incremental positive, favoring approval over CRL.

• Other Drug Approvals

  • ALNY/ARWR: Key drug catalysts with new clinical data (hypertension, obesity drugs ahead).
  • BCRX, NTLA, TNGX, etc.: PDUFA deadlines and regulatory decisions for treatments targeting HAE, ATTR-CM, PDAC, etc.

Upcoming Conferences & Events

1. UMDF Mitochondrial Medicine (June 18-21): Key program updates.
2. ESMO Gynaecological Cancers (June 19-21): Focus on selinexor and related studies.
3. ADA Meeting (June 20-23):
   • OPK poster shows superior hepatoprotective effects of OPK-8806 vs Semaglutide/Survodutide for NAFLD.
   • Sanegene: siRNA targeting obesity via ALK7.
   • WVE: siRNA-INHBE leads to improved weight loss in mouse models.

Clinical Trial Updates

• KALV: Recruiting for sebetralstat in pediatric HAE.
• ELVN: Expanded CML trial recruitment.
• IDA/BCRX: Phase III & II trials advancing darovasertib for uveal melanoma and other cancers.
• CT.gov tracker: Ongoing enrollment updates for multiple Phase I/III trials (e.g., IDYA, TNGX).

Catalysts & Stock Impact

• ALNY: Zilebesiran (hypertension) Phase II data projected 2H25.
• ARWR: Plozasiran FCS PDUFA expected Nov 18, 2025.
• BCRX: PDUFA delay for Orladeyo (pediatric HAE oral therapy) from Sep 12 to Sep 12 (no change from original).
• TSHA: Registrational trial updates for gene therapies.
• VEA: PDUFA for sibeprenlimab (IgAN) on Nov 28, 2025.

Analyst Ratings

• BUY: Enliven (ELVN), Aryl (ARWR), KalVista (KALV), Oncoprotect (OPK), Alnylam (ALNY), Black Diamond (BDTX), Ideaya (IDYA), Alnylam (ALNY), etc.
• HOLD: Aurinia (AUPH), Rigel (RIGL).
• UPPERFORM: Multiple developers.

Upcoming Med Conferences & Deadlines

• July-August Events: ESMO GI, ENDO, ASRM, AACR, IASLC WCLC, etc., feature abstract releases and clinical data reviews.

Please see Important Disclosure information on pages 8 - 18 of this report.