Bemcentinib Meets Efficacy Endpoint for High-Risk MDS and AML

Recently, biopharmaceutical company BerGenBio ASA (“BerGenBio”) announced that their drug candidate bemcentinib reached its primary efficacy endpoint in a Phase 2 clinical trial. The drug is designed to treat patients with high-risk myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who have previously been ineffectively treated with hypomethylating agents (HMAs).


In the Phase 2 BERGAMO clinical trial, which had ten clinical research sites across multiple countries, researchers looked to understand the safety, efficacy, and tolerability of bemcentinib. Enrolled patients were previously treated with 4-6 cycles of azacitidine or decitabine treatment. These are both subtypes of HMAs, which are used to treat patients who are unable to receive chemotherapy or stem cell transplants. However, all patients either relapsed following treatment or saw no improvement. Thus, finding a treatment for this group represents a huge unmet need.

The BERGAMO trial enrolled 45 participants. Patients received 4-9 treatment cycles. If they had no response to treatment, patients received no more bemcentinib after 4 cycles. However, while some patients did not response, the treatment was found to be largely effective. As a result, the trial reached its primary endpoint.

But what is bemcentinib, you might ask? In patients with leukemia, cells over-express a protein kinase receptor called AXL. As a result, patients are more likely to be treatment-resistant and to have less anti tumor response. Bemcentinib inhibits AXL, preventing or reducing tumor growth and allowing for more comprehensive treatment.

Myelodysplastic Syndromes (MDS)

Myelodysplastic syndromes (MDS) consist of a group of progressive conditions that prevent bone marrow from creating enough healthy platelets, red blood cells, and white blood cells. In MDS, these blood cells never mature. Instead, they die quickly or stay in the bone marrow forever. Somewhere between 25 to 45 of 100,000 people over age 70 have MDS. It affects males more than females. While severity varies, MDS progresses to AML in about half of all cases.

While many patients experience no symptoms, other may experience:

  • Pale skin
  • Chest pain
  • Frequent lung, sinus, skin, or urinary tract infections
  • Shortness of breath or difficulty breathing
  • Anemia
  • Fatigue
  • Heart palpitations
  • Easy bruising and bleeding
  • Low platelet count

Learn more about MDS.

Acute Myeloid Leukemia (AML)

Caused by immature cells in the bone marrow (myeloblasts) that crowd out healthy cells, acute myeloid leukemia (AML) is a blood and bone marrow cancer. Symptoms of AML include:

  • Bone pain
  • Severe fatigue
  • Fever
  • Pale skin
  • Frequent infections
  • Easy bruising and bleeding
  • Shortness of breath
  • Loss of appetite

Learn more about AML.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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