Create your Personal Health Record and unlock support built around you
- Treatments and trials you qualify for
- Education for your stage of care
- Financial support for your medications
- Solutions to your side effects
OFIRNOFLAST: First-in-Class NEK7 Inhibitor Reduces Transfusion Needs in Lower-Risk #MDS | EHA 2026
HealthTreeDescription
Dr. Varun Bafna from Haya Therapeutics presents results from the HT 6184 MDS 001 trial, a phase 2 study of OFIRNOFLAST, a first-in-class NEK7 inhibitor for lower-risk myelodysplastic syndrome (MDS).
ABOUT LOWER-RISK MDS:
MDS is a bone marrow disorder affecting blood cell production. Patients often become dependent on blood transfusions, leading to complications and reduced quality of life.
HOW OFIRNOFLAST WORKS:
The drug targets the NLRP3 inflammosome, reducing harmful inflammatory responses that damage blood-producing cells. This helps restore normal blood cell production.
TRIAL RESULTS (37 Patients):
- 66.7% achieved hematological improvement
- 93% showed red blood cell improvement
- 55% became transfusion-independent
- Transfusion independence maintained for 8-16 weeks
- Median hemoglobin increase of 4.6 g
- Well tolerated with only 11% experiencing grade 3-4 side effects
QUALITY OF LIFE:
- 72% improvement in social well-being
- 44% improvement in fatigue
- Overall 66% improvement in quality of life
WHY IT MATTERS:
OFIRNOFLAST is the first oral drug targeting NEK7/NLRP3 inflammosome, offering hope for MDS patients seeking to reduce or eliminate transfusion dependence.
Learn more: https://linktr.ee/HealthTreeUniversity
TIMESTAMPS:
0:00 Introduction
0:10 Background on MDS & Patient Challenges
0:24 How OFIRNOFLAST Works (NEK7 & NLRP3)
1:01 Inflammatory Mechanism in MDS
1:34 Drug Mechanism & Benefits
2:05 Trial Design & Patient Criteria
2:23 Dosing & Study Timeline
2:30 Trial Statistics & Patient Population
3:04 Patient Baseline Characteristics
3:41 Safety Results
4:03 Common Side Effects
4:24 Hematological Improvement Results
4:48 Transfusion Independence Outcomes
5:13 Response Rates Across Patient Groups
5:27 Hemoglobin Improvement Over Time
5:54 Quality of Life Results
6:23 Key Efficacy Results Summary
6:38 Hemoglobin Response in Patients
6:55 Conclusion & Future Implications
#MDS #OFIRNOFLAST #NEK7Inhibitor #LowerRiskMDS #ClinicalTrial #Hematology