2025-05-15-Jefferies-与我们覆盖范围相关的EHA25关键摘要-_KURA_Menin_NRIX_BTK_IRON_13页_3mb

KURA Oncology - Menin Inhibitor in Acute Myeloid Leukemia (AML)

The Phase 1b study of ziftomenib (menin inhibitor) in combination with 7+3 chemotherapy showed high complete response (CR) rates in NPM1m AML patients. At 600mg dose, CR was 88% (30/34) for NPM1m patients, a slight decrease from 100% at an earlier data cut. Safety profile was stable, with no dose-limiting hematological adverse events. Ongoing registrational trial data remains embargoed, but strong buy recommendation from Jefferies.

NRIX Therapeutics - BTK Degraders in Chronic Lymphocytic Leukemia (CLL) and Waldenström Macroglobulinemia (WM)

Bexobrutideg (BTK degrader) Phase 1 data in CLL showed an 81% overall response rate (ORR), with a median duration of response (mDoR) of 7 months. In WM, ORR improved to 85%. The high-level safety profile was consistent, with no dose-limiting toxicities, often involving manageable side effects like purpura and fatigue. Comparable efficacy to BGB-6673 was noted, with deeper responses over time.

IRON (Disc Medicine) - DISC-0974 for Anemia of Chronic Kidney Disease (CKD)

Phase 1b/2 data for DISC-0974 demonstrated dose-dependent hepcidin reduction, improving across 28mg to 100mg doses. A single treatment-related adverse event (TRAE) was reported, and prior ASH '24 data showed sustained efficacy. On-going to Phase 1b expansion, with buy recommendation from Jefferies.

Valuation and Investment Overview

Jefferies utilizes discounted cash flow (DCF) models for price targets and classifies companies as Buy, Hold, or Underperform. KURA has a $5.73 price target (BUY rating), while DISC Medicine and NRIX are both Buy-rated at $45.73 and $9.58 respectively. Key risks include clinical trial outcomes, regulatory hurdles, competitive threats, and financial stability. Factors such as market capitalization, growth, and volatility influence valuation, with COMP BMO-19 1520 AZ HRN (IRON) also noted for comparison. Each investment comes with inherent risks, as outlined in the full report.