ASH 2025: strong evidence

Updated full data from Faron's Phase 1/2 BEXMAB study 

Deep and durable responses in HR-MDS with favorable safety profile 

https://mfn.se/a/faron-pharmaceuticals/faron-presents-updated-bexmab-data-at-ash-2025-deep-and-durable-responses-in-hr-mds-with-favorable-safety-profile  

Significant efficacy observed in TP53-mutated patients with a 70% complete remission rate in the frontline HR MDS

Key highlights:

• Median overall survival (mOS) with bexmarilimab and azacitidine in relapsed/refractory (r/r) HR-MDS patients reached 14.5 months (increased from 13.4 months observed earlier), a significant improvement in a population with historically poor survival of only 5-6 months
• In treatment-naïve patients with HR-MDS and TP53 mutation, the combination achieved a remarkable 70% complete remission (CR) rate
• Deep responses allowed 50% of treatment-naïve TP53-mutated patients and 21% of r/r TP53-mutated patients to proceed to stem cell transplant (SCT)
• For the first time, data shows 57% of frontline patients who were transfusion-dependent at baseline achieved transfusion independence, confirming restoration of healthy bone marrow function

Future: 

Zeidan:/ "We remain excited about this combination as the data continue to mature over time, and we look forward to initiating the randomized registrational phase 2/3 trial in the near future." 

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Reactions:

- stock market: slow

- clinical community: instant

Bono:/ "After this oral presentation, multiple top-notch and highly active centers have expressed their interest in joining our upcoming registrational trial."  

+ Comments by DNB Carnegie:

https://access.dnbcarnegie.com/publication/b25f2484-06bf-4668-d8d7-08de36675890 

A.o. "Another important clinical benefit observed in the study was the improvement in transfusion independence (TI). Among treatment-naïve HR-MDS patients who were transfusion-dependent at baseline, 57% achieved TI during the study. Across the overall study population, 23% of transfusion-dependent patients became TI. Achieving TI is a meaningful improvement in patients’ quality of life and is considered a key indicator of therapeutic benefit in MDS."

Transfusion independence (TI) means

a patient with severe anemia no longer needs regular red blood cell (RBC) transfusions, often a key goal in treating blood disorders like Myelodysplastic Syndromes (MDS) or Thalassemia, achieved through therapies that boost natural RBC production. Becoming transfusion independent signifies major treatment success, improving quality of life and often correlating with longer survival, as it avoids transfusion complications (like iron overload) and indicates better bone marrow function.

"We find the data presented further highlights BEX as an attractive late-stage asset well positioned for partnering, which we continue to view as the key near-term potential share-price catalyst."