Olutasidenib Study Results for Relapsed/Refractory AML

At the 66th ASH Conferences, data was presented about acute myeloid leukemia (AML) patients treated with a new medication; Olutasidenib (Rigel Pharmaceuticals).  

Olutasidenib is a potent and selective oral small molecule mIDH1 inhibitor. This molecule targets and blocks mIDH1 (an enzyme that plays a role in the growth of cancer). The data presented was obtained from a study to evaluate the impact, outcome and overall survival of patients treated with olutasidenib in the registrational phase 2 trial (NCT02719574) of relapsed/refractory AML with a genetic mutation called mIDH1. 

What are the Clinical Trial Details?

The main focus of this study was to see if patients could achieve complete remission (CR), and assess the overall response rate (ORR)

• 147 patients were enrolled in this study, their average age was 71
• Over half of the patients had previously received more than 3 lines of therapy
• Patients received 150 mg of olutasidenib twice daily.

Results Presented

• 35% Achieved Complete Remission (CR) (no signs of leukemia after treatment) in an average of 1 month
• 48% of patients showed an overall response to treatment (ORR) in an average of 10.2 months. 
  
• The median overall survival for patients with an overall response was 32.7 months, with some surviving beyond 57 months.
• Over half of the patients who achieved complete remission responded within two months.
• A slower response (over four months) did not necessarily mean worse survival outcomes.

In 51 patients that reached complete remission, overall survival was not reached. In 71 patients that reached overall response, overall survival was 32.7-51.1 months.

What are the Side Effects From Olutasidenib?

All therapies have different side effects which are important to evaluate especially in clinical trials, for instance, all people who participated in this study experienced at least one of these side effects:

• Nausea, fever and constipation
• Decreased red blood cell count, white blood cell count, and platelets
• Fatigue and diarrhea
• Low count of neutrophils
• Difficulty breathing

Conclusions From This Study

• The median time to response was 1.9 months in patients with mIDH1 relapsed/refractory AML treated with olutasidenib.
• Some patients responded quickly to this medication, while others took up to 6 months to present a response. However, there was no significant connection between the time to response and overall survival.
• These results showed that treatment for at least 6 months would produce a response.
• Olutasidenib was generally well managed and tolerated.

If you or a loved one has been diagnosed with relapsed or refractory AML, learning about available treatment options is essential. The findings from this study highlight olutasidenib as a potential therapy that can lead to meaningful responses, including complete remission and extended survival.

Every patient’s situation is unique, and discussing this treatment with your healthcare team can help determine if it may be a suitable option for you. Stay informed, ask questions, and explore all possible avenues to ensure the best care and outcomes for your AML journey.

If you want to explore the current clinical trials using olutasidenib you can visit our Clinical Trial Finder. 

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